There are many new medicines in testing, development and clinical trials that are currently not available or recently approved to the marketplace and many are having encouraging and positive results. There are also many approved meds that are in tests involved for other ailments/conditions that might work, where these new trials will determine. BiotecHOPE™ will seek to highlight these trials/efforts many of which are from smaller/newer biotech companies and/or are being developed in partnership with Universities, Hospitals, Medical Centers, larger Bio-Pharma companies.
Welcome to BiotecHOPE™ and thank you for visiting www.biotechope.com.
Mar. 14, 2021 3:08 PM ET. AZN, BNTX, JNJ, LLY, MRK, MRNA, NVAX, PFE, VIR
•Premiered Jan 29, 2021 Danny Gokey
Wake up and breathe in deeper than yesterday Take on the morning like your soul's been remade
Roll down the windows, let your cares fly away Good things are yours to claim, you don't have to wait
All across the sky New mercies rise And the future's bright This is a new day Everything bursting with hope
Coming alive This moment, moment You've got a freedom No looking back anymore Open your eyes
It's coming, coming This is a new day The old has gone away The old has gone away This is a new day
Don't let it slip away Don't let it slip away Go on and reignite impossible dreams
Become the one you never thought you could be, woah
All across the sky New mercies rise And the future's bright Oh-oh-ooh-ohh
This is a new day (come on) Everything bursting with hope (woah)
Coming alive This moment, moment You've got a freedom (you've got a freedom)
No looking back anymore (no looking back anymore)
Open your eyes It's coming, coming This is a new day The old has gone away The old has gone away
This is a new day Don't let it slip away Don't let it slip away This is a new day The old has gone away
The old has gone away This is a new day Don't let it slip away Don't let it slip away
Love hasn't give up on you Love hasn't give up on you Love is making the old things new
Today and every single day Love hasn't give up on you Love hasn't give up on you Love... (1, 2, 3)
A new day, a new day This is a new day Everything bursting with hope
Coming alive This moment, moment You've got a freedom (you've got a freedom)
No looking back anymore (no looking back anymore)
Open your eyes It's coming, coming This is a new day The old has gone away The old has gone away
This is a new day Don't let it slip away Don't let it slip away This is a new day The old has gone away
The old has gone away This is a new day (a new day, a new day)
Don't let it slip away Don't let it slip away This is a new day It's a new day It's a new day It's a new day
Yeah, yeah It's a new day It's a new day It's a new day
Songwriters: Danny Gokey, Colby Wedgeworth, Ethan Hulse
•Jul 20, 2020 Elevation Worship
Written by Steven Furtick, Chris Brown
Aug. 26, 2020 5:37 PM ET|
"The country behaved like a horror-movie character who believes the danger is over, even though the monster is still at large."
there is encouraging news coming from biotech/pharma companies with regard to vaccines and therapies to fight this virus.
"I know breakthrough is coming, By faith I see a miracle
My God made me a promise, And it won't stop now""
BiotecHOPE™ Every Victory
Apr 19, 2021Download PDF
AUSTIN, Texas, April 19, 2021 (GLOBE NEWSWIRE) -- XBiotech (NASDAQ: XBIT) announced today that the FDA has granted permission to commence clinical trials with its novel drug candidate for treating patients with pancreatic cancer. From 1992 to 2018 the death rate from pancreatic cancer steadily increased in the USA. It is now predicted that pancreatic cancer will claim 48,220 lives and be the 3rd leading cause of cancer death in the USA in 2021 (National Cancer Institute’s Surveillance, Epidemiology, and End Results (SEER) program).
Pancreatic cancer is typically identified at an advanced stage and treatment often includes surgery and aggressive chemotherapy. A current approved treatment involves combination chemotherapy including ONIVYDE and 5-fluorouracil, drugs that have significant toxicities and provide only modest response rates. XBiotech’s new drug candidate (XB2001) specifically targets a process potentially involved in the growth and spread of malignant tumors; and the drug also blocks inflammation associated with tissue injury, which may reduce toxicity associated with the chemotherapy and allow these drugs to be better tolerated and more effective.
The Phase I/II clinical study will evaluate XBiotech’s new drug candidate when added to the ONIVYDE/5-FU combination therapy. The clinical study is chaired by Dr. Shubham Pant, a leading researcher and oncologist at MD Anderson Cancer Center; and will involve at least 15 other top cancer centers around the United States. The Phase 1 portion of the study will examine increasing doses of XBiotech’s new drug and assess tolerability of the combination at escalating doses. Once a safe dose has been determined, the phase 2 portion will begin, enrolling 60 patients, which will be randomized to receive treatment with ONIVYDE/5-FU or ONIVYDE/5-FU combined with XB2001. Clinical endpoints in the study are safety, overall survival, objective response rate, progression free survival, time to treatment failure, clinical benefit response, number of severe adverse advents, as well as biological measures of experimental drug activity.
About True Human™ Therapeutic Antibodies
XBiotech’s True Human™ antibodies are derived without modification from individuals who possess natural immunity to certain diseases. With discovery and clinical programs across multiple disease areas, XBiotech’s True Human antibodies have the potential to harness the body’s natural immunity to fight disease with increased safety, efficacy and tolerability.
For more information, visit www.xbiotech.com.
Apr. 19, 2021 4:27 AM ET XBiotech Inc. (XBIT)
Apr 19, 2021 DownloadPDF Format (opens in new window)
BURLINGAME, Calif.--(BUSINESS WIRE)-- Humanigen, Inc. (Nasdaq: HGEN) (“Humanigen”), a clinical-stage biopharmaceutical company focused on preventing and treating an immune hyper-response called ‘cytokine storm’ with its lead drug candidate, lenzilumab™, today announced positive data from the Phase 1b portion of ZUMA-19 evaluating the efficacy and safety of lenzilumab in patients treated with CAR-T in diffuse large B-cell lymphoma (DLBCL). At the recommended Phase 2 dose of lenzilumab, the ORR was 100% and no patient experienced severe cytokine release syndrome (CRS) or severe neurotoxicity (NT).
ZUMA-19 was a clinical study designed to evaluate the efficacy and safety of lenzilumab and CAR-T (axicabtagene ciloleucel, Axi-Cel) in patients with relapsed or refractory DLBCL.
This study was a standard 3+3 design with three patients administered 600 mg lenzilumab (cohort 1) and three patients administered 1,800 mg lenzilumab (cohort 2) just prior to CAR-T. The recommended Phase 2 dose was determined to be 1,800 mg.
In the six study patients, the ORR was 83% (n=5) which included four complete responses (CR). In cohort 1, there was no severe CRS (≥ grade 3). One patient experienced grade 3 NT with a two-day duration. At the recommended Phase 2 dose (cohort 2), ORR was 100% (n=3) and the toxicity-free CR (CRS and NT < grade 2) was 66% (n = 2). There was no severe CRS or severe NT at the recommended Phase 2 dose. There were no adverse events attributed to lenzilumab across the study.
For more information, visit www.humanigen.com
Apr. 19, 2021 9:37 AM ET
April 15, 2021 6:45 am ET
Phase 3 MOVe-OUT Study of Molnupiravir in Outpatients to Proceed, Phase 2/3 MOVe-IN Study in Hospitalized Patients Will Not Proceed
KENILWORTH, N.J., & MIAMI--(BUSINESS WIRE)-- Merck (NYSE: MRK), known as MSD outside the United States and Canada, and Ridgeback Biotherapeutics today provided an update on the clinical development program for molnupiravir (MK-4482/ EIDD-2801), an investigational orally available antiviral therapeutic. Based on a planned interim analysis of data from the Phase 2, dose-finding portion (Part 1) of two ongoing placebo-controlled Phase 2/3 trials evaluating molnupiravir administered twice a day for five days in outpatients (MOVe-OUT) and hospitalized patients (MOVe-IN) with COVID-19, and from a previously completed Phase 2a dose-ranging study in outpatients, the decision has been made to proceed with the Phase 3 portion (Part 2) of MOVe-OUT in outpatients with COVID-19, evaluating the 800 mg dose of molnupiravir twice daily. Data from MOVe-IN indicate that molnupiravir is unlikely to demonstrate a clinical benefit in hospitalized patients, who generally had a longer duration of symptoms prior to study entry; therefore, the decision has been made not to proceed to Phase 3.
This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20210415005258/en/
About Molnupiravir Protocol MK-4482-006 (also known as EIDD-2801-2003)
Protocol 6 (MK-4482-006) is a Phase 2a, double-blind, placebo-controlled, randomized trial designed to compare the safety, tolerability, and antiviral activity of molnupiravir versus placebo as measured by viral RNA detection in symptomatic, outpatient (at baseline) adults at least 18 years old with SARS-CoV-2 infection as confirmed by viral RNA detection within seven days of symptom onset. Of 202 treated participants, molnupiravir was considered generally well tolerated and of the 4 serious adverse events reported, none were considered study drug related. Preliminary data from this study was previously presented at CROI 2021.
About Molnupiravir Nonclinical studies
Merck has conducted a comprehensive nonclinical program to characterize the safety profile of molnupiravir. This program included assays such as Big Blue and PIG-a which are designed to provide a robust measure of a drug or chemical’s ability to induce mutations in vivo. Animals were administered molnupiravir for longer and at higher doses (mg/Kg) than those employed in human studies. The totality of the data from these studies indicates that molnupiravir is not mutagenic or genotoxic in in vivo mammalian systems.
Molnupiravir (EIDD-2801/MK-4482) is an investigational, orally administered form of a potent ribonucleoside analog that inhibits the replication of multiple RNA viruses including SARS-CoV-2, the causative agent of COVID-19. Molnupiravir has been shown to be active in several preclinical models of SARS-CoV-2, including for prophylaxis, treatment, and prevention of transmission, as well as SARS-CoV-1 and MERS. Molnupiravir was invented at Drug Innovations at Emory (DRIVE), LLC, a not-for-profit biotechnology company wholly owned by Emory University. For more information on molnupiravir clinical trials please visit https://merckcovidresearch.com/
For more information, visit www.merck.com
Apr. 15, 2021 7:38 AM ET
April 13, 2021 08:30 ET | Source: Longeveron
Study meets primary safety endpoint; positive secondary efficacy assessments support potential benefit from Lomecel-B
Decline in cognitive function slower in patients who received low-dose Lomecel-B as compared with placebo
Quality of life metrics improved with Lomecel-B compared to placebo
Complete results being prepared for publication.
On track to initiate Phase 2 study in the second half of 2021.
MIAMI, April 13, 2021 (GLOBE NEWSWIRE) -- Longeveron Inc. (NASDAQ: LGVN) announced today the final results of its Phase I clinical study evaluating the safety and efficacy of intravenous (i.v.) administration of Lomecel-B, an allogeneic bone marrow-derived medicinal signaling cell (MSC) product, in subjects with mild Alzheimer’s disease. Preliminary results were previously reported in the Company’s S-1/A Registration Statement as part of Longeveron’s successful Initial Public Offering in the first quarter of 2021. The study met its primary safety endpoint, which paves the way for future trials in subjects with Alzheimer’s disease. Importantly, several pre-specified secondary efficacy endpoints and biomarker results support potential benefit from Lomecel-B. The complete trial results are currently being prepared for publication in a peer-reviewed journal, and will be posted on the Company’s website in the future. Longeveron also indicated they are on track to commence a Phase 2 study of Lomecel-B in Alzheimer’s disease in the second half of 2021.
The phase 1 trial, funded in part by an Alzheimer’s Association Part the Cloud Challenge on Neuroinflammation grant, used a randomized, placebo-controlled double-blind design testing single i.v. infusion of Lomecel-B 20 million cells (“low-dose”; (n=15)), Lomecel-B 100 million cells (“high-dose”; n=10)), or placebo (n=8). Subjects were followed for 52 weeks post-infusion.
Key findings from new and previously disclosed data:
Potential of Lomecel-B for Treating Alzheimer’s Disease
Medicinal Signaling Cells (MSCs), as the main ingredient of Lomecel-B, have numerous mechanisms of action that may potentially treat the complex pathology associated with Alzheimer’s disease. Beyond the hallmarks of beta amyloid deposits (plaques) and neurofibrillary tangles, Alzheimer’s disease is also characterized by inflammation in the brain (referred to as “neuroinflammation”), poor functioning of the blood vessels of the brain (“neurovasculature dysfunction”), and degeneration of brain cells (“neurodegeneration”), among other features. The properties of MSCs can potentially treat all of these aspects of Alzheimer’s disease pathology, and preclinical studies support this conclusion. Using mouse models of Alzheimer’s disease, MSCs were shown to be able to decrease inflammation in the brain, promote break-down and clearance of Aβ (the protein component of beta amyloid), decrease the protein that causes neurofibrillary tangles, promote new nerve cell formation (“neurogenesis”), and improve cognitive/behavioral performance.
Additional information about the Company is available at www.longeveron.com.
REQORSA Enhances Efficacy of Chemo-Immune Combination Therapy in KRAS-LKB1 Mutant NSCLC in Humanized Mice
REQORSA Overcomes Resistance to Targeted Therapy Osimertinib
AUSTIN, Texas — (April 12, 2021) — Genprex, Inc. (“Genprex” or the “Company”) (NASDAQ: GNPX), a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes, today announced that its collaborators presented positive preclinical data for the combination of TUSC2 immunogene therapy (REQORSA™) in combination with chemotherapy and immunotherapies for the treatment of non-small cell lung cancer (NSCLC). Collaborators also presented positive preclinical data for the use of REQORSA in combination with targeted therapies for the treatment of NSCLC. These data were presented in two presentations at the 2021 American Association of Cancer Research (AACR) annual meeting. The TUSC2 gene is a tumor suppressor gene and is the active agent in REQORSA.
“We are pleased to have these positive data that provide further support for the therapeutic potential of REQORSA in combination with immunotherapies and targeted therapies in NSCLC presented before an audience of the world’s leading cancer researchers. These data are particularly encouraging as we look to initiate our upcoming combination Acclaim-1 and Acclaim-2 clinical trials of REQORSA in NSCLC,” said Rodney Varner, President and Chief Executive Officer of Genprex. “We know that many patients inevitably develop resistance to immune checkpoint blockade therapy or EGFR-TKI therapy. These data show that REQORSA in combination with immunotherapies and targeted therapies may provide enhanced efficacy in NSCLC that has become resistant to these regimens, offering hope to a large patient population who currently has limited treatment options.”
For more information, please visit the Company’s web site at www.genprex.com
FibroGen Receives Fast Track Designation from the U.S. FDA for Pamrevlumab for the Treatment of Duchenne Muscular Dystrophy
SAN FRANCISCO, April 12, 2021 (GLOBE NEWSWIRE) -- FibroGen, Inc. (NASDAQ: FGEN) announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for the company’s anti-CTGF antibody, pamrevlumab, for the treatment of patients with Duchenne muscular dystrophy (DMD). This designation follows review of the Phase 2 clinical data from a single-arm trial in non-ambulatory patients with DMD, and represents recognition by the FDA that pamrevlumab has the potential to address an unmet medical need for this disease. Pamrevlumab is currently being evaluated in two Phase 3 trials for the treatment of DMD.
“Fast Track designation by the FDA for pamrevlumab in DMD underscores the high unmet medical need for patients suffering from this debilitating disease and potential to advance a new treatment option,” said Mark Eisner, M.D, M.P.H, Chief Medical Officer, FibroGen. “We look forward to working closely with the FDA on the development of pamrevlumab as a potential therapy for DMD.”
Pamrevlumab is a first-in-class antibody developed by FibroGen that inhibits the activity of connective tissue growth factor (CTGF), an important biological mediator in fibrotic and proliferative disorders. Pamrevlumab is in Phase 3 clinical development for the treatment of locally advanced unresectable pancreatic cancer (LAPC), Duchenne muscular dystrophy (DMD), and idiopathic pulmonary fibrosis (IPF). For information about pamrevlumab studies currently recruiting patients, please visit www.clinicaltrials.gov.
For more information, please visit www.fibrogen.com.
Pamrevlumab is a first-in-class antibody that inhibits the activity of connective tissue growth factor (CTGF), a common factor in chronic fibrotic and proliferative disorders, characterized by persistent and excessive fibrous tissue which can lead to organ dysfunction and failure, and in cancer, characterized by promotion of tumor growth. Pamrevlumab is in Phase 3 clinical development for the treatment of idiopathic pulmonary fibrosis (IPF) and for the treatment of locally advanced unresectable pancreatic cancer (LAPC) and Duchenne muscular dystrophy (DMD), and in Phase 2 clinical development for the treatment of COVID-19. The U.S. Food and Drug Administration has granted Orphan Drug Designation to pamrevlumab for the treatment of patients with IPF, LAPC, and DMD. Pamrevlumab has also received Fast Track designation from the U.S. Food and Drug Administration for the treatment of patients with IPF and LAPC. Across all clinical studies to date, pamrevlumab has consistently demonstrated a good safety and tolerability profile. For information about pamrevlumab studies currently recruiting patients, please visit www.clinicaltrials.gov.
April 12, 2021
– Primary endpoint achieved: the proportion of patients alive and free of mechanical ventilation at Day 29 was 12.3 percentage points higher with mavrilimumab versus placebo (p=0.1224 met predefined statistical threshold of p<0.2) –
– 65% reduction in risk of mechanical ventilation/death with mavrilimumab versus placebo (p=0.0175) –
– 61% reduction in risk of death with mavrilimumab versus placebo (p=0.0726) –
– Clinical improvement was observed on top of steroids and/or antivirals –
– Enrollment in the Phase 3 portion of the trial ongoing –
HAMILTON, Bermuda, April 12, 2021 (GLOBE NEWSWIRE) -- Kiniksa Pharmaceuticals, Ltd. (Nasdaq: KNSA) (“Kiniksa”), a biopharmaceutical company with a portfolio of assets designed to modulate immunological pathways across a spectrum of diseases, today announced the Phase 2 portion of the Phase 2/3 trial of mavrilimumab in non-mechanically-ventilated patients (Cohort 1) with severe COVID-19 pneumonia and hyperinflammation achieved its primary efficacy endpoint of the proportion of patients alive and free of mechanical ventilation at Day 29. Mavrilimumab is an investigational fully-human monoclonal antibody that targets granulocyte macrophage colony stimulating factor receptor alpha (GM-CSFRα).
“These data suggest that mavrilimumab may be a transformational treatment option for patients with severe pneumonia due to hyper-inflammatory syndromes, including COVID-19,” said Sanj K. Patel, Chief Executive Officer and Chairman of the Board of Kiniksa. “Additionally, they reinforce our belief in the potential broad utility of mavrilimumab, which has demonstrated positive clinical data across three indications: giant cell arteritis, rheumatoid arthritis, and severe COVID-19. We are engaged with various government agencies to potentially secure resources to help bring mavrilimumab to patients as soon as possible.”
The Phase 2/3 trial is a global, randomized, double-blind, placebo-controlled study designed to evaluate the efficacy and safety of mavrilimumab treatment in adults hospitalized with severe COVID-19 pneumonia and hyperinflammation.
Non-mechanically ventilated patients (Cohort 1) treated with mavrilimumab demonstrated a reduction in mechanical ventilation and death at Day 29 pooled across dose levels.
Mavrilimumab is an investigational fully-human monoclonal antibody that blocks activity of GM-CSF by specifically binding to the alpha subunit of the GM-CSF receptor. Mavrilimumab was dosed in over 550 patients with rheumatoid arthritis through Phase 2b clinical studies in Europe and achieved prospectively-defined primary endpoints of efficacy and safety. Kiniksa’s lead indication for mavrilimumab is giant cell arteritis (GCA), a rare inflammatory disease of medium-to-large arteries. A Phase 2 trial in GCA achieved both the primary and secondary efficacy endpoints with statistical significance. Kiniksa is also evaluating mavrilimumab in severe COVID-19 pneumonia and hyperinflammation. The FDA granted Orphan Drug designation to mavrilimumab for the treatment of GCA in 2020.
April 10, 2021 Download this Press ReleasePDF Format (opens in new window)
BOULDER, Colo.--(BUSINESS WIRE)-- Clovis Oncology, Inc. (NASDAQ: CLVS) announced that Phase 1 clinical data from studies exploring Rubraca in combination with Xtandi for the treatment of advanced prostate cancer (RAMP) and Rubraca monotherapy in advanced solid tumors in Japanese patients (RUCA-J) will be presented during week one of the American Association for Cancer Research Virtual Annual Meeting (AACR), taking place April 10-15, 2021.
“We remain committed to understanding how Rubraca may benefit patients with cancer, and the data presented at AACR further enhance our understanding in different patient populations and solid tumor types,” said Patrick J. Mahaffy, President and CEO of Clovis Oncology. “The Phase 1b RAMP data for the combination of Rubraca and Xtandi in unselected mCRPC patients help inform the Alliance for Clinical Oncology-sponsored CASPAR Phase 3 trial which is expected to begin enrolling patients soon, and we look forward to learning more about the combination.”
The presentations can also be viewed at https://www.clovisoncology.com/pipeline/scientificpresentations/ .
About Rubraca (rucaparib)
Rucaparib is an oral, small molecule inhibitor of PARP1, PARP2 and PARP3 being developed in multiple tumor types, including ovarian and metastatic castration-resistant prostate cancers, as monotherapy, and in combination with other anti-cancer agents. Exploratory studies in other tumor types are also underway.
Please click here for full Prescribing Information for Rubraca.
Please visit www.clovisoncology.com for more information.
About Alliance for Clinical Trials in Oncology
The Alliance for Clinical Trials in Oncology develops and conducts clinical trials with promising new cancer therapies, and utilizes the best science to develop optimal treatment and prevention strategies for cancer, as well as research methods to alleviate side effects of cancer and cancer treatments. The Alliance is part of the National Clinical Trials Network (NCTN) sponsored by the National Cancer Institute (NCI) and serves as a research base for the NCI Community Research Oncology Program (NCORP). The Alliance comprises nearly 10,000 cancer specialists at hospitals, medical centers, and community clinics across the United States and Canada. Learn more about the Alliance, visit www.AllianceforClinicalTrialsinOncology.org.
View source version on businesswire.com: https://www.businesswire.com/news/home/20210410005008/en/
Clinical and pathological data presented from the clinical trials of Bria-IMT™ alone and in combination with immune checkpoint inhibitors in advanced breast cancer indicates high responding subset with protracted progression-free survival:
• Data suggests a link with cancer grade, disease control, and progression-free-survival in patients treated with Bria-IMT™.
• Highest rate of disease control and longest progression free survival were observed in patients with Grade I/II tumors.
• Median overall survival of 12.5 months in patients with Grade I/II tumors versus 7.2-9.8 months in a recent study of third line breast cancer.
BERKELEY, Calif. and VANCOUVER, British Columbia, April 12, 2021— BriaCell Therapeutics Corp. (Nasdaq: BCTX, BCTXW) (TSX-V:BCT) (“BriaCell” or the “Company”), a clinical-stage biotechnology company specializing in targeted immunotherapies for advanced breast cancer, is pleased to announce the presentation of results from clinical studies with its lead product candidate, Bria-IMT™, summarized in a poster session held at the American Association for Cancer Research (AACR) Annual Meeting 2021, a virtual meeting, held over two weeks (Week 1: April 10-15; Week 2: May 17-21).
The findings indicate disease control in advanced breast cancer patients, including stable disease (SD), partial responses (PR) or complete responses (CR). Disease control was especially noted in patients with Grade I/II (i.e. well or moderately differentiated) tumors or those that matched Bria-IMT™ at 2 or more HLA alleles. Patients with low or undetectable levels of circulating cancer cells were more likely to benefit from therapy.
Analysis and Discussion:
• The Bria-IMT™ regimen with or without checkpoint inhibitors is able to induce an effective immune response and disease control in heavily pre-treated advanced breast cancer patients. The patients were all heavily pretreated and failed multiple prior regimens.
• Delayed Type Hypersensitivity (DTH) to Bria-IMT™ analysis identified a group with significantly higher rates of disease control and progression-free survival (8 months) in both monotherapy and combination therapy studies suggesting a robust immune response is predictive of clinical benefit in these patients.
• Highest levels of disease control and PFS was observed in patients who matched Bria-IMT™ at 2 or more HLA alleles in the monotherapy study but not in the combination therapy study supporting our strategy to develop Bria-OTS™, an off-the-shelf personalized immunotherapy for advanced breast cancer.
• Patients with Grade I/II tumors (median of 8 prior therapy regimens) were more likely to respond with disease control (67%) and longer progression free survival. The response was more pronounced in the patients in the combination therapy study suggesting additive or synergistic effects of checkpoint inhibitors when combined with the Bria-IMT™ regimen. Bria-IMT™, with a molecular signature most closely related to Grade I/II tumors, may result in disease control and clinical benefit especially in this subset of patients.
A copy of the poster is posted at the following: https://briacell.com/novel-technology/scientific-publications/.
For additional information on BriaCell, please visit: https://briacell.com/.
BriaCell is currently conducting a Phase I/IIa clinical trial of Bria-IMT™, BriaCell’s lead candidate, in a Combination Study with immune checkpoint inhibitors such as the Incyte drugs INCMGA00012, an anti-PD-1 antibody similar to pembrolizumab (KEYTRUDA®; manufactured by Merck & Co., Inc., and epacadostat, an orally bioavailable small-molecule inhibitor of indoleamine 2,3-dioxygenase 1 (IDO1). The Combination Study is listed in ClinicalTrials.gov as NCT03328026.
March 30, 2021
HOUSTON, March 30, 2021 /PRNewswire/ -- Moleculin Biotech, Inc., (Nasdaq: MBRX) (Moleculin or the Company), a clinical stage pharmaceutical company with a broad portfolio of drug candidates targeting highly resistant tumors and viruses, today announced that the U.S. Food and Drug Administration ("FDA") has approved its request for Fast Track Designation for its drug, Annamycin, for the treatment of soft tissue sarcoma (STS) lung metastases.
"We are pleased to receive our second Fast Track Designation from the FDA for Annamycin. We now have potential pathways for accelerated approval in two indications, STS lung metastases, and the treatment of relapsed or refractory acute myeloid leukemia," commented Walter Klemp, Moleculin's Chairman and CEO. "Not only does this make us eligible for accelerated approval and priority review for our NDA submission, but it serves as an important reminder of the unmet need in STS lung metastases. We are now focused on initiating our internally funded clinical trial in the US, possibly prior to mid-year. In addition, we recently announced a $1.5 million grant awarded in Poland for an investigator initiated clinical trial there for this indication which should start later this year."
Annamycin is a "next generation" anthracycline that has recently been shown in animal models to accumulate in the lungs at up to 30-fold the level of doxorubicin. Importantly, Annamycin has also demonstrated a lack of cardiotoxicity in recently conducted human clinical trials for the treatment of acute myeloid leukemia, so we believe that the use of Annamycin may not face the same usage limitations imposed on doxorubicin.
For more information about the Company, please visit http://www.moleculin.com.
Mar. 30, 2021 8:04 AM ET
On 25 March 2021, the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion, recommending the granting of a marketing authorisation for the medicinal product Copiktra, intended for the treatment of adult patients with relapsed or refractory chronic lymphocytic leukaemia (CLL) and refractory follicular lymphoma (FL). The applicant for this medicinal product is Verastem Europe GmbH.
Copiktra will be available as 15-mg and 25-mg hard capsules. The active substance of Copiktra is duvelisib, an anti-neoplastic agent (ATC code: L01EM04) which acts by inhibiting phosphatidylinositol 3-kinase p110δ (PI3K-δ) and PI3K-γ. These enzymes are involved in the proliferation and survival of malignant B-cell lines and primary CLL tumour cells, and in immunological pathways in the tumour microenvironment of malignant B cells.
The benefits of Copiktra are that it prolongs the survival time without any progression of the disease as compared to ofatumumab in patients with CLL who have received 2 or more prior lines of treatment and induces tumour responses in patients with FL who have received 2 or more prior treatments. The most common side effects are respiratory tract infections, neutropenia, anaemia, thrombocytopenia, headache, dyspnoea, cough, decreased appetite diarrhoea/colitis, nausea, vomiting, abdominal pain, constipation, rash, musculoskeletal pain, arthralgia, pyrexia, fatigue and increased transaminases.
Copiktra should be prescribed by physicians experienced in the treatment of cancer.
19 Studies found for: CH5126766 OR defactinib | Recruiting, Not yet recruiting, Active, not recruiting, Completed, Enrolling by invitation Studies
Mar. 26, 2021 7:53 AM ET
Oncology / Immunology, Press Releases, RNS Announcements | 24 Mar 2021
Hong Kong, Shanghai & Florham Park, NJ — Wednesday, March 24, 2021: Hutchison China MediTech Limited (“HUTCHMED”) (Nasdaq/AIM: HCM) has initiated a Phase Ib/II study of surufatinib in combination with BeiGene’s tislelizumab in patients with advanced solid tumors in the U.S. and Europe. The first patient was dosed on March 23, 2021. This trial is to explore potential synergistic activity of the novel, oral angio-immuno kinase inhibitor surufatinib with the anti-PD-1 antibody tislelizumab in enhancing overall antitumor activity from inhibition of angiogenesis along with stimulation of an immune response.
This is an open-label study to evaluate the safety, tolerability, pharmacokinetics and efficacy of surufatinib in combination with tislelizumab in patients with advanced solid tumors. The study consists of two parts: dose finding (Part 1) and dose expansion (Part 2). Part 1 will be conducted to determine the recommended Phase II dose (“RP2D”) and/or the maximum tolerated dose (MTD) of surufatinib in combination with tislelizumab in patients with advanced or metastatic solid tumors who have progressed on, or are intolerant to, standard therapies. Part 2 will be an open-label, multi-cohort design to evaluate the anti-tumor activity of surufatinib in combination with tislelizumab in patients with specific types of advanced or metastatic solid tumors, including neuroendocrine tumors, colorectal cancer, small cell lung cancer, gastric cancer, and soft tissue sarcoma. Patients will receive the RP2D determined in Part 1 of this study. Additional details may be found at clinicaltrials.gov, using identifier NCT04579757.
Surufatinib is a novel, oral angio-immuno kinase inhibitor that selectively inhibits the tyrosine kinase activity associated with vascular endothelial growth factor receptor (VEGFR) and fibroblast growth factor receptor (FGFR), which both inhibit angiogenesis, and colony stimulating factor-1 receptor (CSF-1R), which regulates tumor-associated macrophages, promoting the body’s immune response against tumor cells. Its unique dual mechanism of action may be very suitable for possible combinations with other immunotherapies, where there may be synergistic anti-tumor effects.
HUTCHMED currently retains all rights to surufatinib worldwide.
NETs in the U.S. and Europe: In the U.S., surufatinib was granted Fast Track Designations for development in pNET and epNET in April 2020, and Orphan Drug Designation for pNET in November 2019. A U.S. FDA NDA rolling submission was initiated in December 2020, to be followed by a marketing authorization application (MAA) submission to the European Medicines Agency (EMA) in Europe. The basis to support these filings includes the completed SANET-ep and SANET-p studies, along with existing data from surufatinib in U.S. epNET and pNET patients (clinicaltrials.gov identifier: NCT02549937).
epNETs in China: On December 30, 2020, surufatinib was granted drug registration approval by the National Medical Products Administration of China (“NMPA”) for the treatment of epNET. Surufatinib is marketed in China under the brand name Sulanda®. The approval was based on results from the SANET-ep study, a Phase III trial (clinicaltrials.gov identifier: NCT02588170) in patients with advanced epNETs conducted in China. The study met the pre-defined primary endpoint of progression-free survival (“PFS”) at a preplanned interim analysis. The positive results of this trial were highlighted in an oral presentation at the 2019 ESMO Congress and published in The Lancet Oncology in September 2020. Median PFS was significantly longer for patients treated with surufatinib at 9.2 months, compared to 3.8 months for patients in the placebo group (HR 0.334; 95% CI: 0.223-0.499; p<0.0001). Surufatinib had an acceptable safety profile, with the most common treatment-related adverse events of grade 3 or worse being hypertension (36% of surufatinib patients vs. 13% of placebo patients), proteinuria (19% vs. 0%) and anemia (5% vs. 3%).
pNETs in China: In 2016, we initiated the SANET-p study, which is a pivotal Phase III study in patients with low- or intermediate-grade, advanced pNET in China. It was terminated early as the pre-defined primary endpoint of PFS was met (clinicaltrials.gov identifier: NCT02589821) at a preplanned interim analysis, leading to a second NDA accepted by the NMPA in September 2020. The positive results of this study were presented at the 2020 ESMO Virtual Congress and published simultaneously in The Lancet Oncology, demonstrating that surufatinib reduces the risk of disease progression or death by 51% in patients, with median PFS of 10.9 months compared to 3.7 months on placebo (HR 0.491; 95% CI: 0.391-0.755; p=0.0011). The safety profile of surufatinib was manageable and consistent with observations in prior studies.
Biliary tract cancer in China: In March 2019, we initiated a Phase IIb/III study comparing surufatinib with capecitabine in patients with advanced biliary tract cancer whose disease progressed on first-line chemotherapy. The primary endpoint is overall survival (OS) (clinicaltrials.gov identifier: NCT03873532).
Immunotherapy combinations: We have entered into collaboration agreements to evaluate the safety, tolerability and efficacy of surufatinib in combination with anti-PD-1 monoclonal antibodies, including with tislelizumab (BGB-A317), Tuoyi® (toripalimab) and Tyvyt® (sintilimab), which are approved as monotherapies in China.
Tislelizumab (BGB-A317) is a humanized IgG4 anti-PD-1 monoclonal antibody specifically designed to minimize binding to FcγR on macrophages. In pre-clinical studies, binding to FcγR on macrophages has been shown to compromise the anti-tumor activity of PD-1 antibodies through activation of antibody-dependent macrophage-mediated killing of T effector cells. Tislelizumab is the first drug from BeiGene’s immuno-oncology biologics program and is being developed internationally as a monotherapy and in combination with other therapies for the treatment of a broad array of both solid tumor and hematologic cancers.
The NMPA has granted tislelizumab full approval for first-line treatment of patients with advanced squamous non-small cell lung cancer (NSCLC) in combination with chemotherapy. Tislelizumab has also received conditional approval from the NMPA for the treatment of patients with classical Hodgkin’s lymphoma (cHL) who received at least two prior therapies, and for the treatment of patients with locally advanced or metastatic urothelial carcinoma (UC) with PD-L1 high expression whose disease progressed during or following platinum-containing chemotherapy or within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy. Full approval for these indications is contingent upon results from ongoing randomized, controlled confirmatory clinical trials.
In addition, three supplemental Biologics License Applications for tislelizumab have been accepted by the Center for Drug Evaluation (CDE) of the NMPA and are under review for first-line treatment of patients with advanced non-squamous NSCLC in combination with chemotherapy, for the second- or third-line treatment of patients with locally advanced or metastatic NSCLC who progressed on prior platinum-based chemotherapy, and for previously treated unresectable hepatocellular carcinoma.
Currently, 15 potentially registration-enabling clinical trials are being conducted in China and globally, including 12 Phase 3 trials and two pivotal Phase 2 trials.
In January 2021, BeiGene and Novartis entered into a collaboration and license agreement to develop, manufacture, and commercialize tislelizumab in North America, Europe, and Japan.
Tislelizumab is not approved for use outside of China.
Tislelizumab (BGB-A317) is a humanized IgG4 anti–PD-1 monoclonal antibody specifically designed to minimize binding to FcγR on macrophages. In pre-clinical studies, binding to FcγR on macrophages has been shown to compromise the anti-tumor activity of PD-1 antibodies through activation of antibody-dependent macrophage-mediated killing of T effector cells. Tislelizumab is the first drug candidate produced from BeiGene’s immuno-oncology biologic program, and we believe it could serve as a key element of our immuno-oncology combination platform. Tislelizumab is being developed as a monotherapy and in combination with other therapies for the treatment of a broad array of both solid tumor and hematologic cancers.
For more information, please visit: www.hutch-med.com.
Mar. 24, 2021 3:17 AM ET
By: Mamta Mayani, SA News Editor
Patients screened in ORA-D-013-2 study which is recruiting 450 patients in 53 sites in the U.S., Europe and Israel
NEW YORK, March 23, 2021 /PRNewswire/ — Oramed Pharmaceuticals Inc. (Nasdaq: ORMP) (TASE: ORMP) (www.oramed.com), a clinical-stage pharmaceutical company focused on the development of oral drug delivery systems, announced today it has screened the first patients in its ORA-D-013-2 study, the second of two concurrent Phase 3 studies of its oral insulin capsule, ORMD-0801, for the treatment of type 2 diabetes (T2D).
The studies are taking place under U.S. Food and Drug Administration (FDA) approved protocols to treat T2D patients who have inadequate glycemic control over a period of 6 to 12 months. [Enrollment for the other Phase 3 study, ORA-D-013-1, is ongoing and has surpassed 25%]. The double-blinded, placebo-controlled, multi-center randomized studies will recruit a total of 1,125 patients to evaluate the efficacy and safety of ORMD-0801. Efficacy data for the studies will become available after all patients have completed the first 6-month treatment period.
About the Study
The ORA-D-013-2 study is recruiting 450 T2D patients with inadequate glycemic control who are managing their condition with either diet alone or with diet and metformin monotherapy. Patients will be recruited through 28 sites in the U.S. and 25 sites in Western Europe and Israel. The double-blind study will randomize patients 1:1 into two cohorts dosed with 8 mg of ORMD-0801 at night and placebo at night. The primary endpoint of the study is to compare the efficacy of ORMD-0801 to placebo in improving glycemic control as assessed by A1c over a 26-week treatment period, with a secondary endpoint of comparing ORMD-0801 to placebo in maintaining glycemic control over a 52-week treatment period.
About Oramed Pharmaceuticals
Oramed Pharmaceuticals is a platform technology pioneer in the field of oral delivery solutions for drugs currently delivered via injection. Established in 2006, with offices in the United States and Israel, Oramed has developed a novel Protein Oral Delivery (POD™) technology. Oramed is seeking to transform the treatment of diabetes through its proprietary lead candidate, ORMD-0801, which has the potential to be the first commercial oral insulin capsule for the treatment of diabetes. The Company has completed multiple Phase 2 clinical trials under an Investigational New Drug application with the U.S. Food and Drug Administration. In addition, Oramed is developing an oral GLP-1 (Glucagon-like peptide-1) analog capsule, ORMD-0901.
For more information, please visit www.oramed.com.
SOURCE Oramed Pharmaceuticals Inc.
Mar 23, 2021
Basel, March, 23, 2021 — Novartis today reported the first interpretable results of the Phase III VISION study evaluating the efficacy and safety of 177Lu-PSMA-617, a targeted radioligand therapy in patients with progressive PSMA-positive metastatic castration-resistant prostate cancer (mCRPC) compared to best standard of care alone. The trial met both primary endpoints of overall survival and radiographic progression-free survival1, helping to move closer the ambition of becoming the targeted treatment for >80% of patients with advanced prostate cancer. The safety profile was consistent with data reported in previous clinical studies1. Results from the VISION trial will be presented at an upcoming medical meeting and included in US and EU regulatory submissions.
177Lu-PSMA-617, a targeted radioligand therapy
“Patients with metastatic castration-resistant prostate cancer have a less than 1 in 6 chance of surviving 5 years2 and need new treatment options. These groundbreaking data confirm our belief in the potential of 177Lu-PSMA-617 to reimagine outcomes for these patients through phenotypic precision medicine. We intend to submit these data to regulatory authorities as soon as possible,” said John Tsai, Head of Global Drug Development and Chief Medical Officer for Novartis. “We would like to thank the patients who volunteered to participate in this study as well as the clinical teams at each of the trial sites. We would not be able to realize our commitment to reimagining medicine without the partnership of patients and their families.”
Radioligand therapy combines a targeting compound that binds to markers expressed by tumors and a radioactive isotope, causing DNA damage that inhibits tumor growth and replication. This therapeutic approach enables targeted delivery of radiation to the tumor, while limiting damage to the surrounding normal tissue. Novartis has established global expertise and specialized supply chain and manufacturing capabilities across its network of four radioligand therapy production sites, and is further increasing capacity to ensure delivery of radioligand therapies like 177Lu-PSMA-617 to patients in need.
177Lu-PSMA-617 is an investigational PSMA-targeted radioligand therapy for metastatic castration-resistant prostate cancer. It is a type of precision cancer treatment combining a targeting compound (ligand) with a therapeutic radioisotope (a radioactive particle)10-12. After administration into the bloodstream, 177Lu-PSMA-617 binds to prostate cancer cells that express PSMA13, a transmembrane protein, with high tumor-to-normal tissue uptake10,14,15. Once bound, emissions from the radioisotope damage tumor cells, disrupting their ability to replicate and/or triggering cell death. The radiation from the radioisotope works over very short distances to limit damage to surrounding cells14,16.
VISION is an international, prospective, randomized, open-label, multicenter, phase III study to assess the efficacy and safety of 177Lu-PSMA-617 (7.4 GBq administered by i.v. infusion every 6 weeks for a maximum of 6 cycles) plus investigator-chosen best standard of care in the investigational arm, versus best standard of care in the control arm17. Patients with PSMA PET-scan positive mCRPC, and progression after prior taxane and androgen receptor-directed therapy (ARDT), were randomized in a 2:1 ratio in favor of the investigational arm. The alternate primary endpoints were rPFS and OS. The study enrolled 831 patients1.
Find out more at https://www.novartis.com.
Mar. 23, 2021 4:07 AM ET
By: Mamta Mayani, SA News Editor
March 16, 2021
Patients treated with mirikizumab met the primary endpoint of clinical remission and all key secondary endpoints compared to placebo
- LUCENT-1 is the first and only Phase 3 study of an anti-IL-23p19 monoclonal antibody to demonstrate reduced bowel urgency in moderate to severe ulcerative colitis
- Safety results in this study were consistent with that of the previous mirikizumab study in ulcerative colitis and studies with the anti-IL-23p19 antibody class
INDIANAPOLIS, March 16, 2021 /PRNewswire/ -- Eli Lilly and Company (NYSE: LLY) announced today that mirikizumab met the primary and all key secondary endpoints in LUCENT-1, a 12-week Phase 3 induction study evaluating the efficacy and safety of mirikizumab for the treatment of patients with moderate to severe ulcerative colitis (UC). LUCENT-2, a multicenter, randomized, double-blind, placebo-controlled maintenance study of mirikizumab in patients who have completed the 12-week LUCENT-1 induction study is ongoing.
Mirikizumab is a humanized IgG4 monoclonal antibody that binds to the p19 subunit of interleukin 23. Mirikizumab is being studied for the treatment of immune diseases, including psoriasis, ulcerative colitis and Crohn's disease.
About the LUCENT Clinical Trial Program
The LUCENT Phase 3 clinical development program for mirikizumab includes LUCENT-1, LUCENT-2 and LUCENT-3. LUCENT-1 (NCT03518086) is a multicenter, randomized, double-blind, placebo-controlled, Phase 3 induction study of mirikizumab in patients with moderate to severe UC who had failed conventional and/or biologic treatments. LUCENT-2 (NCT03524092) is a multicenter, randomized, double-blind, placebo-controlled maintenance study of mirikizumab in patients who have completed the 12-week LUCENT-1 induction study. LUCENT-3 (NCT03519945) is an open label extension study for eligible patients who have participated in mirikizumab UC trials.
The program began in 2018, with full results from the induction and maintenance studies anticipated in early 2022. To learn more about Lilly, please visit us at lilly.com
View original content to download multimedia:
SOURCE Eli Lilly and Company
Mar. 16, 2021 8:35 AM ET
By: Mamta Mayani, SA News Editor
AC Immune Announces New Clinical Results in Down Syndrome and Plans for Future Development of Anti-Amyloid-Beta Vaccine
March 16, 2021
Topline ACI-24 Phase 1b immunogenicity and safety results reported today at a global Down syndrome symposium
Reported new data in non-human primates for optimized vaccine formulation which shows strong response against key pathological Abeta species, including oligomeric and pyroglutamate Abeta
LAUSANNE, Switzerland, March 16, 2021 (GLOBE NEWSWIRE) -- AC Immune SA (NASDAQ: ACIU), a Swiss-based, clinical-stage biopharmaceutical company with a broad pipeline focused on neurodegenerative diseases, today announced plans to advance its novel anti-amyloid-beta (Abeta) vaccine into mid-stage clinical testing to treat and prevent the progression of Down syndrome (DS)-related Alzheimer’s disease (AD). Topline results reported today by AC Immune’s Chief Scientific Officer, Dr. Marie Kosco-Vilbois, at a global DS symposium co-sponsored by AC Immune, showed that ACI-24 demonstrated encouraging immunogenicity and safety in Phase 1b clinical testing in people with DS. The Company also disclosed new non-human primate data for an optimized formulation of the vaccine, which shows broad potential for the treatment and prevention of Abeta-driven diseases based on its superior efficacy in non-human primates.
Highlights from the Phase 1b study in DS-related AD
Due to the high vulnerability of people with DS to severe COVID-19 sequelae, initiation of the next clinical trial will be delayed to ensure the safety of study participants. In the interim, AC Immune is taking advantage of this time to accelerate development of its optimized anti-Abeta vaccine formulation, which demonstrated encouraging safety and superior immunogenicity results in mouse and non-human primate (NHP) studies. Dr. Kosco-Vilbois presented some of these key findings during her presentation today:
Key preclinical results for the optimized anti-Abeta vaccine formulation in NHPs
ACI-24 is also currently being tested in a Phase 2 clinical trial in patients with mild AD. In this study, there have been no safety concerns nor evidence for CNS inflammation or ARIA related to ACI-24 in any subject. The Phase 2 study is progressing toward an 18-month interim analysis, which is planned for Q2 2021.
Mar. 16, 2021 8:22 AM ET
By: Aakash Babu, SA News Editor
March 16, 2021 at 6:59 AM EDT
Phase 2/3 study expected to enroll 6,750 healthy pediatric participants less than 12 years of age
Study will enroll in the U.S. and Canada
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Mar. 16, 2021-- Moderna Inc. (Nasdaq: MRNA), a biotechnology company pioneering messenger RNA (mRNA) therapeutics and vaccines, today announced that the first participants have been dosed in the Phase 2/3 study, called the KidCOVE study, of mRNA-1273, the Company’s vaccine candidate against COVID-19, in children ages 6 months to less than 12 years. The study is being conducted in collaboration with the National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health (NIH) and the Biomedical Advanced Research and Development Authority (BARDA), part of the Office of the Assistant Secretary for Preparedness and Response at the U.S. Department of Health and Human Services.
About the Moderna COVID-19 Vaccine
The Moderna COVID-19 Vaccine is an mRNA vaccine against COVID-19 encoding for a prefusion stabilized form of the Spike (S) protein, which was co-developed by Moderna and investigators from the NIAID’s Vaccine Research Center. The first clinical batch, which was funded by the Coalition for Epidemic Preparedness Innovations, was completed on February 7, 2020 and underwent analytical testing; it was shipped to the NIH on February 24, 2020, 42 days from sequence selection. The first participant in the NIAID-led Phase 1 study of the Moderna COVID-19 Vaccine was dosed on March 16, 2020, 63 days from sequence selection to Phase 1 study dosing. On May 12, 2020, the U.S. Food and Drug Administration granted the Moderna COVID-19 Vaccine Fast Track designation. On May 29, 2020, the first participants in each age cohort: adults ages 18-55 years (n=300) and older adults ages 55 years and above (n=300) were dosed in the Phase 2 study of the vaccine. On July 8, 2020, the Phase 2 study completed enrolment. Results from the second interim analysis of the NIH-led Phase 1 study of the Moderna COVID-19 Vaccine in the 56-70 and 71+ age groups were published on September 29, 2020 in The New England Journal of Medicine. On July 28, 2020, results from a non-human primate preclinical viral challenge study evaluating the vaccine were published in The New England Journal of Medicine. On July 14, 2020, an interim analysis of the original cohorts in the NIH-led Phase 1 study of the vaccine was published in The New England Journal of Medicine. On November 30, 2020, Moderna announced the primary efficacy analysis of the Phase 3 study of the vaccine conducted on 196 cases. On November 30, 2020, the Company also announced that it filed for Emergency Use Authorization with the U.S. FDA and a Conditional Marketing Authorization (CMA) application with the European Medicines Agency. On December 3, 2020, a letter to the editor was published in The New England Journal of Medicine reporting that participants in the Phase 1 study of the Moderna COVID-19 Vaccine retained high levels of neutralizing antibodies through 119 days following first vaccination (90 days following second vaccination). On December 18, 2020, the U.S. FDA authorized the emergency use of the Moderna COVID-19 Vaccine in individuals 18 years of age or older. Moderna has also received authorization for its COVID-19 vaccine from health agencies in Canada, Israel, the European Union, the United Kingdom, Switzerland, Singapore and Qatar. Additional authorizations are currently under review in other countries and by the World Health Organization.
The Biomedical Advanced Research and Development Authority (BARDA), part of the Office of the Assistant Secretary for Preparedness and Response (ASPR) within the U.S. Department of Health and Human Services (HHS) is supporting the continued research and development of the Company’s COVID-19 vaccine development efforts with federal funding under contract no. 75A50120C00034. BARDA is reimbursing Moderna for 100 percent of the allowable costs incurred by the Company for conducting the program described in the BARDA contract. The U.S. government has agreed to purchase supply of mRNA-1273 under U.S. Department of Defense contract no. W911QY-20-C-0100.
Moderna COVID-19 Vaccine is authorized for use under an Emergency Use Authorization (EUA) for active immunization to prevent coronavirus disease 2019 (COVID-19) caused by severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) in individuals 18 years of age and older. Moderna COVID-19 Vaccine is investigational and not approved by FDA.
To learn more, visit www.modernatx.com.
View source version on businesswire.com: https://www.businesswire.com/news/home/20210316005514/en/
Mar. 16, 2021 7:07 AM ET
March 16, 2021 6:45 am EST
Application Based on Objective Response Rate From Phase 2 Trial Evaluating Belzutifan in Patients With Von Hippel-Lindau Disease-Associated Renal Cell Carcinoma
New Filing Further Strengthens Merck’s Expanding and Diverse Oncology Portfolio
KENILWORTH, N.J.--(BUSINESS WIRE)-- Merck (NYSE: MRK), known as MSD outside the United States and Canada, today announced that the U.S. Food and Drug Administration (FDA) has accepted and granted priority review for a New Drug Application (NDA) for the hypoxia-inducible factor-2 alpha (HIF-2α) inhibitor belzutifan (pronounced bell-ZOO-ti-fan), a novel investigational candidate in Merck’s oncology pipeline, for the potential treatment of patients with von Hippel-Lindau (VHL) disease-associated renal cell carcinoma (RCC), not requiring immediate surgery. This NDA is based on data from the Phase 2 Study-004 trial, in which belzutifan showed a confirmed overall response rate of 36.1% (n=22/61) (95% CI: 24.2-49.4) in patients with VHL disease-associated RCC. The FDA has set a Prescription Drug User Fee Act (PDUFA), or target action, date of September 15, 2021.
About the Phase 2 Study-004 Trial
This application is based on data from Study-004 (ClinicalTrials.gov, NCT03401788 ), which is a Phase 2, open-label trial evaluating belzutifan for the potential treatment of patients with VHL disease who had at least one measurable solid tumor localized to the kidney and who did not require immediate surgery. The study enrolled 61 patients who received belzutifan 120 mg orally once daily until disease progression or unacceptable toxicity. The primary endpoint was objective response rate in VHL disease-associated RCC. Secondary endpoints in RCC tumors include disease control rate, duration of response, time to response, progression-free survival, time to surgery and safety.
Additionally, this study evaluated response rates in other common VHL disease-associated tumors, including pancreatic cysts, pancreatic neuroendocrine tumors, central nervous system (CNS) hemangioblastomas, and retinal hemangioblastomas.
Belzutifan (MK-6482) is a novel, potent and selective inhibitor of HIF-2α. Proteins known as hypoxia-inducible factors, including HIF-2α, can accumulate in patients when VHL, a tumor-suppressor protein, is inactivated. If not properly regulated, the accumulation of HIF-2α can stimulate several oncogenes associated with cellular proliferation, angiogenesis and tumor growth, leading to the growth of both benign and malignant tumors. This inactivation of VHL has been observed in more than 90% of clear cell RCC tumors. Research into VHL biology that led to the discovery of HIF-2α was awarded the Nobel Prize in Physiology or Medicine in 2019. For more information, visit www.merck.com View source version on businesswire.com: https://www.businesswire.com/news/home/20210316005293/en/
Mar. 16, 2021 7:01 AM ET
By: Gaurav Batavia, SA News Editor
bluebird bio Presents Long-Term Data for elivaldogene autotemcel (eli-cel, Lenti-D™) Gene Therapy for Cerebral Adrenoleukodystrophy (CALD)
90% of evaluable patients (27/30) alive and free of major functional disabilities (MFDs) at two years follow-up in Phase 2/3 Starbeam study (ALD-102)
Patients in long-term follow-up study (LTF-304) continue to remain alive and MFD-free through up to nearly seven years of follow-up, suggesting eli-cel stabilizes the progression of disease
No reports of graft failure, graft rejection, graft-versus-host disease, replication competent lentivirus or insertional oncogenesis in the 51 patients treated with eli-cel in clinical studies (ALD-102/LTF-304 and ALD-104)
Data presented in oral session during Presidential Symposium at the 47th Annual Meeting of the EBMT
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Mar. 15, 2021-- bluebird bio, Inc. (Nasdaq: BLUE) announced new data from the clinical development program for its investigational elivaldogene autotemcel (eli-cel, Lenti-D™) gene therapy in patients with cerebral adrenoleukodystrophy (CALD), including updated results from the pivotal Phase 2/3 Starbeam study (ALD-102) and the long-term follow-up study LTF-304, as well as safety outcomes from the Phase 3 ALD-104 study. Data were presented today in an oral presentation during the Presidential Symposium at the 47th Annual Meeting of the European Society for Blood and Marrow Transplantation (EBMT 2021), taking place virtually from March 14 - 17, 2021.
elivaldogene autotemcel (eli-cel, Lenti-D™) Gene Therapy
Eli-cel is a one-time investigational gene therapy designed to add functional copies of the ABCD1 gene into a patient’s own hematopoietic (blood) stem cells (HSCs) that have been transduced ex vivo with the Lenti-D lentiviral vector (LVV). The addition of the functional ABCD1 gene allows patients to produce the adrenoleukodystrophy protein (ALDP), which is thought to activate the breakdown of VLCFAs. The goal of treatment with eli-cel is to stabilize the progression of CALD and consequently preserve as much neurological function as possible. Importantly, with eli-cel, there is no need for donor HSCs from another person.
“CALD is a terrible disease that occurs in early childhood and, if left untreated, often leads to eventual death for these boys, a difficult fact for any clinician to bear. These data from the Phase 2/3 Starbeam study show some potentially promising evidence, with up to almost seven years of follow-up and nearly all patients have a stable neurologic function score (n=31/32), indicating that minimal neurologic function was lost following eli-cel infusion. In addition there were no reports of graft failure, graft rejection, or graft-versus-host disease,” said Dr. Jörn-Sven Kühl, Department of Pediatric Oncology, Hematology and Hemostaseology, Center for Women’s and Children’s Medicine, University Hospital Leipzig. “These long-term results therefore suggest treatment with eli-cel may stabilize disease progression and consequently preserve as much neurological function as possible in boys with CALD.”
eli-cel Presentation at EBMT21
Elivaldogene autotemcel (eli-cel; Lenti-D) gene therapy for cerebral adrenoleukodystrophy: Updated results from the Phase 2/3 study and safety outcomes report from the Phase 3 study
Presenting Author: Jörn-Sven Kühl, M.D., Department of Pediatric Oncology, Hematology and Hemostaseology, Center for Women’s and Children’s Medicine, University Hospital Leipzig
Oral Session & Number: GS2-8 Presidential Symposium
Date & Time: Monday, March 15, 3:33-3:42 PM CET/10:33-10:42 AM EDT; Auditorium 1
All EBMT sessions will be available to registered attendees on-demand on the Annual Meeting website after they are aired live; content will be accessible online for two months following the close of the meeting.
About elivaldogene autotemcel (eli-cel, formerly Lenti-D™ gene therapy)
In October 2020, the European Medicines Agency (EMA) accepted bluebird bio’s marketing authorization application (MAA) for its investigational eli-cel gene therapy for the treatment of patients with cerebral adrenoleukodystrophy (CALD). The EMA accepted eli-cel gene therapy for the treatment of CALD into its Priorities Medicines scheme (PRIME) in July 2018, and previously granted Orphan Medicinal Product designation to eli-cel.
The U.S. Food and Drug Administration (FDA) granted eli-cel Orphan Drug status, Rare Pediatric Disease designation, and Breakthrough Therapy designation for the treatment of CALD. bluebird bio is currently on track to submit the Biologics License Application (BLA) in the U.S. in mid-2021.
Eli-cel is not approved for any indication in any geography.
bluebird bio is currently enrolling patients for a Phase 3 study (ALD-104) designed to assess the efficacy and safety of eli-cel after myeloablative conditioning using busulfan and fludarabine in patients with CALD. Contact email@example.com for more information and a list of study sites.
Additionally, bluebird bio is conducting a long-term safety and efficacy follow-up study (LTF-304) for patients who have been treated with eli-cel for CALD and completed two years of follow-up in bluebird bio-sponsored studies.
The Phase 2/3 Starbeam study (ALD-102) has completed enrollment. For more information about the ALD-102 study visit: www.bluebirdbio.com/our-science/clinical-trials or clinicaltrials.gov and use identifier NCT01896102.
More information about newborn screening is available at https://www.bluebirdbio.com/patients-and-advocacy/newborn-screening-toolkit-for-ALD.
For more information, visit bluebirdbio.com.
View source version on businesswire.com: https://www.businesswire.com/news/home/20210315005105/en/
Mar. 15, 2021 6:31 AM ET
By Bojan Pancevski
March 13, 2021 8:00 am ET
BioNTech SE, a German company that joined with Pfizer Inc. to manufacture and distribute its vaccine, has marshaled an alliance of 13 companies, including Novartis AG , Merck KGaA and Sanofi SA, in an effort to meet—and perhaps exceed—an ambitious target of making two billion doses of vaccine this year.
The European Union has been struggling with a shortage of vaccines as manufacturers, including British-Swedish pharmaceutical firm AstraZeneca PLC, have fallen behind on their delivery pledges to the bloc.
Mar. 13, 2021 11:59 PM ET
March 13, 2021Download PDF
Findings from the primary endpoint supported by consistency of all secondary outcome measures assessing cognition and function
TRAILBLAZER-ALZ is the first study to screen and enroll patients based on their tau pathology
Biomarker results suggest potential for long-term disease modification following fixed duration treatment with slowing of tau accumulation across key brain regions
INDIANAPOLIS, March 13, 2021 /PRNewswire/ -- Phase 2 TRAILBLAZER-ALZ results presented today by Eli Lilly and Company (NYSE: LLY) at the 15th International Conference on Alzheimer's & Parkinson Diseases™ 2021 (AD/PD™ 2021) held virtually March 9-14, 2021 and published simultaneously in the New England Journal of Medicine (NEJM) expand on previously reported top-line data that found donanemab met its primary endpoint and showed significant slowing of decline on the integrated Alzheimer's Disease Rating Scale (iADRS), a composite measure of cognition and daily function, in patients with early symptomatic Alzheimer's disease compared to placebo1,2.
About TRAILBLAZER-ALZ Study
TRAILBLAZER-ALZ (NCT03367403) is a randomized, placebo-controlled, double-blind, multi-center Phase 2 study to assess the safety, tolerability and efficacy of donanemab in patients with early symptomatic Alzheimer's disease. The trial enrolled 272 patients who were selected based on cognitive assessments in conjunction with amyloid plaque imaging and tau staging by PET imaging. The study's primary endpoint is change from baseline until 76 weeks in the Integrated Alzheimer's Disease Rating Scale (iADRS), a composite tool combining the Alzheimer's Disease Assessment Scale-Cognitive subscale (ADAS-Cog13) and the Alzheimer's Disease Cooperative Study - instrumental Activities of Daily Living (ADCS-iADL) for function. Key secondary endpoints include changes between baseline and 76 weeks in the Alzheimer's Disease Assessment Scale-Cognitive Subscale (ADAS-Cog13), ADCS-iADL, MMSE, and Clinical Dementia Rating Scale Sum of Boxes (CDR-SB) scores. Other secondary biomarker endpoints include changes from baseline to week 76 in brain amyloid deposition and brain tau deposition and volumetric MRI. The safety, tolerability and efficacy of donanemab are also being evaluated in the ongoing randomized, placebo-controlled, double-blind, multi-center Phase 2 study TRAILBLAZER-ALZ 2 (NCT04437511).
Donanemab is a monoclonal antibody that was designed to bind a specific form of post-translationally modified Aß, N-terminal pyroglutamate, and thereby yield rapid and complete clearance of
"We are confident in the results of the TRAILBLAZER-ALZ study," said Daniel Skovronsky, M.D., Ph.D., Lilly's chief scientific officer and president of Lilly Research Laboratories. "This is the first late-stage study in Alzheimer's disease to meet its primary endpoint at the primary analysis. Donanemab has the potential to become a very important treatment for Alzheimer's disease. We were pleased to see not only slowing of cognitive and functional decline, but also very substantial clearance of amyloid plaques and slowing of spread of tau pathology. The constellation of clinical and biomarker results indicates the potential for long-term disease modification. We are grateful to the patients, caregivers, and investigators who participated in this landmark study."
Specifically, at 76 weeks compared to baseline, treatment with donanemab slowed decline by 32 percent compared to placebo as measured by the iADRS, which was statistically significant. As early as nine months (36 weeks) after initiation of treatment, a significant difference in decline by iADRS was observed.
In addition, 40 percent of participants treated with donanemab achieved amyloid negativity as early as six months after starting treatment and 68 percent achieved this target by 18 months. Donanemab is a monoclonal antibody that was designed to bind a specific form of post-translationally modified Aß, N-terminal pyroglutamate, and thereby yield rapid and complete clearance of amyloid plaques.
March 13, 2021
AC Immune Presents New Preclinical Data for Therapeutic and Diagnostic Candidates Addressing Novel Targets for Neurodegenerative Diseases
March 09, 2021
Four presentations at AD/PD™ 2021 feature latest findings from wholly owned therapeutic and diagnostic programs targeting pathological forms of alpha-synuclein and TDP-43
First biologically active small molecule inhibitors of intracellular alpha-synuclein aggregation advancing toward in vivo proof-of-concept studies
Anti-TDP-43 therapeutic antibody candidates demonstrate dual mechanism of action against pathological TDP-43 in vivo
LAUSANNE, Switzerland, March 09, 2021 (GLOBE NEWSWIRE) -- AC Immune SA (NASDAQ: ACIU), a Swiss-based, clinical-stage biopharmaceutical company with a broad pipeline focused on neurodegenerative diseases, today outlined new preclinical data that will be presented at the 15th International Conference on Alzheimer’s & Parkinson’s Diseases (AD/PD™), taking place virtually from March 9–14, 2021. Data from the Company’s wholly owned, first-in-class therapeutic and diagnostic programs targeting pathological forms of alpha-synuclein and TAR DNA-binding protein 43 (TDP-43) are described during four oral and e-poster presentations. Together the presentations further illustrate the synergy between AC Immune’s SupraAntigen™ and Morphomer™ technology platforms to deliver a precision medicine approach to treating neurodegenerative diseases (NDD).
Morphomer™ TDP-43 anti-TDP-43 antibody
Details of AC Immune’s AD/PD™ 2021 presentations
Morphomer™ TDP-43 imaging
First-in-class TDP-43 PET tracers were characterized using a newly optimized radiobinding assay, which enabled the identification of several distinct chemical series of promising Morphomers™ that bind to recombinant and brain-derived TDP-43 aggregates. Selected compounds also demonstrate direct target engagement on patient-derived brain tissue, as assessed by a positive signal in a proprietary high-resolution autoradiography assay that co-localized with pathological TDP-43. Medicinal chemistry is ongoing to further optimize the properties of hit compounds, and investigational new drug (IND)-enabling studies are expected to begin in Q3 2021.
Title: Discovery of PET tracers for TDP-43 proteinopathies
Date: Thursday, March 11, 2021 | 10:45 – 11:00 am CET
Presenter: Oral presentation by Tamara Seredenina
Data to be presented show that AC Immune’s lead anti-TDP-43 antibody, the first with reported in vivo activity, significantly reduced levels of pathological (phosphorylated or insoluble) forms of TDP-43 in the brain in a murine neurodegenerative disease model. New data also demonstrate the antibody’s dual mechanism of action, showing that it inhibits TDP-43 aggregation and promotes the uptake and clearance of pre-existing TDP-43 aggregates by microglia. The lead candidate is currently in IND-enabling studies and is expected to start preclinical toxicology studies by year end.
Title: TDP-43 antibody directed microglial clearance and inhibition of seeded aggregation mitigates neuropathology in models of TDP-43 proteinopathy
Date: Thursday, March 11, 2021 | 11:15 – 11:30 am CET
Presenter: Oral presentation by Tariq Afroz
Morphomer™ alpha-synuclein imaging
New preclinical data for ACI-12589, a next-generation alpha-synuclein PET tracer being developed as a first-in-class diagnostic imaging agent for Parkinson’s disease and other alpha-synucleinopathies, confirm that the Morphomer™-derived candidate has a desirable brain-PET ligand pharmacokinetic profile in non-human primates. ACI-12589 has previously shown excellent target engagement and signal specificity on tissue samples from patients with alpha-synucleinopathies, including Parkinson’s disease, multiple system atrophy (MSA) and dementia with Lewy bodies (DLB). ACI-12589 is currently being evaluated in a first-in-human study.
Title: [18F]ACI-12589, a novel alpha-synuclein radiotracer as a biomarker in patients with Parkinson’s disease and other synucleinopathies
E-poster ID: P531 / #868
Presenter: E-poster presentation by Efthymia Vokali
Morphomer™ alpha-synuclein small molecule aggregation inhibitor
The first biologically active small molecule inhibitors targeting intracellular alpha-synuclein aggregates have been identified using the Morphomer™ platform. Data to be presented for the first time show that these initial compounds, from several distinct chemical series, significantly decrease alpha-synuclein aggregate formation in cellular assays by interfering with the fibrillation process. Iterative medicinal chemistry optimization led to the identification of compounds with favorable CNS-penetrant pharmacokinetic properties, which will be progressed into in vivo proof-of-concept studies in models of alpha-synucleinopathies, expected to begin in Q3 2021.
Zurich-Schlieren, Switzerland, March 09, 2021.
Molecular Partners AG (SIX: MOLN), a clinical-stage biotech company that is developing a new class of custom-built protein drugs known as DARPin® therapeutics, and its collaborator Novartis, today announced initial results from its ongoing phase 1 study of its first tri-specific COVID-19 antiviral treatment, ensovibep (MP0420), in healthy volunteers. DARPin® candidates, MP0420 and MP0423,
Molecular Partners two antiviral DARPin® candidates, MP0420 and MP0423, are designed to target multiple different sites on the SARS-CoV-2 virus simultaneously for enhanced antiviral effects and potential use as both prophylactics and treatments. The benefits of this multi-specificity include cooperative binding, extremely high potencies and potential prevention of viral ‘escape’ via mutations. The candidates are formatted with a half-life extending DARPin® domain that binds to human serum albumin (HSA) to support long-acting activity. All DARPin® candidates are constructed to benefit from high-yield and low-cost microbial manufacturing. Molecular Partners is investigating whether the high thermal stability of DARPin® molecules can be used to overcome cold-chain requirements.
Following strong preclinical data supporting the anti-COVID-19 program candidates, in October 2020 the Company entered into a collaboration with Novartis in the form of an option and license agreement to develop, manufacture and commercialize Molecular Partners’ anti-COVID-19 DARPin® program. Per the terms of the agreement, Molecular Partners is conducting Phase 1 clinical trials for ensovibep and performing all remaining preclinical work for MP0423; Novartis will conduct Phase 2 and Phase 3 clinical trials, with Molecular Partners as sponsor of these trials. Upon option exercise, Novartis would be responsible for all further development and commercialization activities. Molecular Partners is also collaborating with AGC Biologics, Baccinex, and Ivers-Lee Clinical Supply Management (IL-CSM) to support development of its anti-COVID-19 program, and has reached an agreement with the Swiss Government regarding rights to purchase up to 3.2 million doses of MP0420, if it is approved in Switzerland.
For more information see www.molecularpartners.com
Find out more at https://www.novartis.com.
Tue March 9, 2021 1:00 AM| Accesswire
Molecular Partners (OTCPK:MLLCF) and its collaborator Novartis (NYSE:NVS) announced initial results from their ongoing phase 1 study of first tri-specific COVID-19 antiviral treatment, ensovibep (MP0420), in healthy volunteers.
March 8, 2021 11:40 am EST
Company to Proceed with Phase 2 Development Program for Islatravir Subdermal Implant
KENILWORTH, N.J.--(BUSINESS WIRE)-- Merck (NYSE: MRK), known as MSD outside the United States and Canada, today announced results from a Phase 1 study evaluating the safety, tolerability and pharmacokinetics (PK) of the company’s investigational subdermal drug-eluting implant with potential for extended administration of islatravir for pre-exposure prophylaxis (PrEP) of HIV-1 infection. Islatravir is an investigational nucleoside reverse transcriptase translocation inhibitor (NRTTI) currently being evaluated across a variety of doses, formulations and frequencies for both the treatment of HIV-1 infection in combination with other antiretroviral agents and for the prevention of HIV-1 infection as a single agent. Study results, presented as a late-breaking oral presentation [Presentation 88] at the 2021 Conference on Retroviruses and Opportunistic Infections (CROI 2021), demonstrate that the implant achieved active drug concentrations above the pre-specified PK threshold at 12 weeks across the three doses of islatravir studied (48 mg, 52 mg and 56 mg), and is projected to provide drug concentrations likely above threshold for one year at the 56 mg dose. Based on these findings, Merck plans to initiate a Phase 2 trial to further explore the potential of a subdermal implant containing islatravir as a long-acting option for PrEP for up to 12 months.
Islatravir (formerly MK-8591)
About Islatravir (MK-8591)
Islatravir (formerly MK-8591) is Merck’s investigational nucleoside reverse transcriptase translocation inhibitor (NRTTI) under evaluation in clinical trials for the treatment of HIV-1 infection in combination with other antiretrovirals, including the ILLUMINATE clinical trials program for once-daily treatment. Islatravir is also being studied for pre-exposure prophylaxis (PrEP) of HIV-1 infection as a single agent across a variety of formulations, including the IMPOWER clinical trials evaluating an oral once-monthly regimen.
View source version on businesswire.com: https://www.businesswire.com/news/home/20210308005050/en/
For more information, visit www.merck.com
Mar 8, 2021PDF Version
NOVATO, Calif., March 08, 2021 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare genetic diseases, today announced that the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application for UX053, an investigational mRNA therapy being evaluated for the treatment of Glycogen Storage Disease Type III (GSDIII). Enrollment in a Phase 1/2 study is expected to begin in the second half of 2021.
UX053 is an investigational mRNA-based biologic therapy encoding full-length, glycogen debranching enzyme encapsulated in a lipid nanoparticle (LNP) designed to provide the deficient protein in GSDIII. For more information on Ultragenyx, please visit the company's website at: www.ultragenyx.com.
Monday, March 08, 2021 - 08:30amEST
Saint-Herblain (France) and New York, NY, March 8, 2021 – Valneva SE (“Valneva”), a specialty vaccine company focused on prevention of diseases with major unmet needs, and Pfizer Inc. (NYSE: PFE) today announced initiation of study VLA15-221. The VLA15-221 study builds on previous positive Phase 2 studies, incorporates new dose regimens and is anticipated to be the final Phase 2 study readout before a decision to progress into pivotal Phase 3 studies.
About VLA 15
VLA15 is the only active Lyme disease vaccine candidate in clinical development today, and covers six serotypes that are prevalent in North America and Europe. This investigational multivalent protein subunit vaccine targets the outer surface protein A (OspA) of Borrelia, an established mechanism of action for a Lyme disease vaccine. OspA is one of the most dominant surface proteins expressed by the bacteria when present in a tick. VLA15 has demonstrated strong immunogenicity and safety data in pre-clinical and clinical studies so far. The program was granted Fast Track designation by the U.S. Food and Drug Administration (FDA) in July 20173.
Valneva and Pfizer announced a collaboration for VLA15’s development and commercialization at the end of April 2020. The two companies are working closely together on the next development steps.
About Clinical Study VLA15-221
VLA15-221 is a randomized, observer-blind, placebo-controlled Phase 2 study. It is the first clinical study with VLA15 that enrolls a pediatric population aged 5 years and older.
A total of approximately 600 participants will receive VLA15 at two different immunization schedules (Month 0-2-6 or Month 0-6, 200 volunteers each) or placebo (Month 0-2-6, 200 volunteers). Vaccinees will receive VLA15 at a dose of 180µg, which was selected based on data generated in the two previous Phase 2 studies. The main safety and immunogenicity readout (Primary Endpoint analysis) will be at Month 7, where peak antibody titers are expected. A subset of participants will receive a booster dose of VLA15 or placebo at Month 18 (Booster Phase) and will be followed up for further three years to monitor antibody persistence.
VLA15 will be tested as an alum-adjuvanted formulation and administered intramuscularly. The study will be conducted at sites which are located in areas where Lyme disease is endemic and will enroll volunteers with a cleared past infection with Borrelia burgdorferi, the bacteria that cause Lyme disease, as well as B. burgdorferi naïve volunteers. In addition, to learn more, please visit us on www.Pfizer.com https://valneva.com/
Provention Bio Announces Publication of Extended Follow-up Data from the Pivotal "At-Risk" TN-10 Study of Teplizumab in Science Translational Medicine-One course of teplizumab delayed insulin dependence by approximately three years and improved beta cell function in at-risk (Stage 2) type 1 diabetes patients-March 03, 2021
RED BANK, N.J., March 3, 2021 /PRNewswire/ -- Provention Bio, Inc. (Nasdaq: PRVB), a biopharmaceutical company dedicated to intercepting and preventing immune-mediated disease, today announced that extended follow-up data from the pivotal "At-Risk" TN-10 Study were published in Science Translational Medicine. Results show that a single 14-day infusion course of teplizumab (PRV-031) delayed the onset of clinical disease and insulin dependence in at-risk type 1 diabetes (T1D) patients by approximately three years (median of 32.5 months), adding one year to previously reported results. The TN-10 Study was conducted through the Type 1 Diabetes TrialNet, an international research collaboration aimed at discovering ways to delay or prevent type 1 diabetes.
About Teplizumab (PRV-031):
Teplizumab is an investigational anti-CD3 monoclonal antibody (mAb) with a filed Biologics License Application (BLA) under Priority Review by the U.S. Food and Drug Administration (FDA) for the delay or prevention of clinical type 1 diabetes (T1D) in at-risk individuals. More than 800 patients have received teplizumab in multiple clinical studies involving more than 1,000 subjects. In previous studies of newly diagnosed patients, teplizumab consistently demonstrated the ability to preserve beta-cell function, a measure of endogenous insulin production. It correspondingly reduced the need for exogenous insulin use. Teplizumab has been granted Breakthrough Therapy Designation by the FDA and PRIME designation by the European Medicines Administration. Provention is currently also evaluating teplizumab in patients with newly diagnosed insulin-dependent T1D (the Phase 3 PROTECT study).
Teplizumab, Provention's lead drug candidate, is an anti-CD3 monoclonal antibody currently under review by the U.S. Food and Drug Administration (FDA) for the delay or prevention of clinical T1D in at-risk individuals, defined as having two or more T1D-related autoantibodies and dysglycemia (Stage 2 T1D). The lifetime risk of insulin-dependent clinical disease (Stage 3 T1D) approaches 100% in these pre-symptomatic Stage 2 patients.
"Teplizumab is the first disease-modifying investigational drug with data showing an ongoing delay to insulin-dependent T1D, now by approximately three years after a single course," said Dr. Kevan Herold, M.D., Professor of Immunology and Medicine at Yale University, lead author of the study. "These data build on existing clinical evidence demonstrating the potential for teplizumab to change the course of the disease and advance the treatment paradigm. We are continuing to observe patients in the TN-10 Study to determine whether the observed delay will extend even further over time."
Visit www.ProventionBio.com for more information
Mar. 03, 2021 5:02 PM ET
News March 2, 2021argenx Announces FDA Acceptance of BLA Filing for Efgartigimod for the Treatment of Generalized Myasthenia Gravis
Breda, the Netherlands
Efgartigimod is an investigational antibody fragment designed to reduce disease-causing immunoglobulin G (IgG) antibodies and block the IgG recycling process. Efgartigimod binds to the neonatal Fc receptor (FcRn), which is widely expressed throughout the body and plays a central role in rescuing IgG antibodies from degradation. Blocking FcRn reduces IgG antibody levels representing a logical potential therapeutic approach for several autoimmune diseases known to be driven by disease-causing IgG antibodies, including: myasthenia gravis (MG), a chronic disease that causes muscle weakness; pemphigus vulgaris (PV), a chronic disease characterized by severe blistering of the skin; immune thrombocytopenia (ITP), a chronic bruising and bleeding disease; and chronic inflammatory demyelinating polyneuropathy (CIDP), a neurological disease leading to impaired motor function.
For more information, visit www.argenx.com
Mar. 02, 2021 2:32 AM ET
By: Mamta Mayani, SA News Editor
February 27, 2021 at 6:25 PM EST
TARRYTOWN, N.Y., Feb. 27, 2021 /PRNewswire/ --
Single administration of novel antibody cocktail controlled patients' allergic response to cat allergen, preventing early asthma reactions for the duration of the 3-month trial
Patients experienced significant improvements in lung function and cat allergen tolerance from the first assessment at week 1
Results presented at the virtual 2021 AAAAI Annual Meeting
Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced detailed results from a Phase 2 proof-of-concept trial evaluating the investigational antibody cocktail REGN1908-1909 in cat-allergic patients with mild asthma. The trial met the primary endpoint of preventing early asthma reactions (EAR, defined as a ≥20% decline in forced expiratory volume over one second [FEV1]). The trial also met key secondary endpoints, including improved lung function and an increased amount of cat allergen that patients could tolerate following a single dose of treatment, from as early as the first assessment conducted at week 1. The results were shared in an oral presentation at the virtual 2021 American Academy of Allergy, Asthma and Immunology (AAAAI) Annual Meeting.
About the Phase 2 Trial
The randomized, double-blind, parallel-group, single-dose proof-of-concept trial enrolled 56 cat-allergic patients with mild asthma who were not living with a cat. The trial consisted of up to a 12-week screening period followed by 1:1 randomization on day 1 to receive 600 mg REGN1908-1909 or placebo administered subcutaneously, followed by a 12-week assessment period and a 4-week safety follow-up period. During the screening period, patients underwent a 2-hour CAC in an EEU after initially being evaluated in a placebo challenge. Patients performed spirometry every 10 minutes during the EEU CAC until they experienced an EAR, or bronchoconstriction when their FEV1 was reduced by ≥20%. Following the CAC, patients were monitored for 6 hours and those meeting eligibility requirements were then randomized to receive a single 600 mg subcutaneous dose of REGN1908-1909 or placebo, and returned to the trial site to undergo a 4-hour CAC in the EEU (plus a 6-hour observation period) at weeks 1, 4, 8 and 12 following treatment administration.
The primary endpoint was prevention of EAR, as measured by FEV1, compared to placebo 1 week after treatment, following the CAC. Key secondary endpoints included the prevention of EAR on weeks 4, 8 and 12; change in FEV1 on weeks 1, 4, 8 and 12; and change in cat allergen quantity, as experienced by patients during exposure on weeks 1, 4, 8 and 12.
About Regeneron's VelocImmune® Technology
REGN1908-1909 was invented using Regeneron's VelocImmune technology that utilizes a proprietary genetically-engineered mouse platform endowed with a genetically-humanized immune system to produce optimized fully-human antibodies. When Regeneron's co-Founder, President and Chief Scientific Officer George D. Yancopoulos was a graduate student with his mentor Frederick W. Alt in 1985, they were the first to envision making such a genetically-humanized mouse, and Regeneron has spent decades inventing and developing VelocImmune and related VelociSuite® technologies. Dr. Yancopoulos and his team have used VelocImmune technology to create multiple antibodies including Dupixent® (dupilumab), Libtayo®, Praluent® (alirocumab), Kevzara® (sarilumab), Evkeeza™ (evinacumab-dgnb), Inmazeb™ (atoltivimab, maftivimab, and odesivimab-ebgn) and Regeneron's antibody cocktail for COVID-19, which was recently granted Emergency Use Authorization (EUA) in the U.S.
For additional information about the company, please visit www.regeneron.com
Feb. 27, 2021 9:04 PM ET
February 24, 2021 at 7:00 AM EST
SAN DIEGO, Feb. 24, 2021 (GLOBE NEWSWIRE) -- Kura Oncology, Inc. (Nasdaq: KURA), a clinical-stage biopharmaceutical company committed to realizing the promise of precision medicines for the treatment of cancer, today announced that its investigational drug, tipifarnib, has been granted Breakthrough Therapy Designation by the U.S. Food and Drug Administration (FDA) for the treatment of patients with recurrent or metastatic HRAS mutant head and neck squamous cell carcinoma (HNSCC) with variant allele frequency ≥ 20% after disease progression on platinum-based chemotherapy. Tipifarnib is currently being evaluated in an ongoing registration-directed clinical trial (AIM-HN) in this indication of high unmet need.
Tipifarnib’s Breakthrough Therapy Designation is based on data from RUN-HN, a Phase 2 clinical trial evaluating tipifarnib in patients with recurrent or metastatic HRAS mutant HNSCC. Data from this trial, presented at the American Society of Clinical Oncology Virtual Scientific Program in May 2020, showed an ORR of 50%, median PFS of 5.9 months and a median OS of 15.4 months among the 18 evaluable patients. HRAS represents approximately 4-8% of HNSCC patients. The HRAS biomarker can be found on most commercially available genomic panels.
Tipifarnib, is a potent, selective and orally bioavailable inhibitor of farnesyl transferase in-licensed from Janssen. Previously, tipifarnib was studied in more than 5,000 cancer patients and showed compelling and durable anti-cancer activity in certain patient subsets; however, no molecular mechanism of action had been determined that could explain its clinical activity across a range of solid tumor and hematologic indications. Leveraging advances in next generation sequencing as well as emerging information about cancer genetics and tumor biology, the Company is seeking to identify those patients most likely to benefit from tipifarnib. In addition to Breakthrough Therapy Designation, tipifarnib has been granted Fast Track designation by the FDA for the treatment of patients with HRAS mutant HNSCC. In addition to HNSCC, tipifarnib has demonstrated encouraging clinical activity in a number of additional genetically defined tumor types. Kura has received multiple issued patents for tipifarnib, providing patent exclusivity in the U.S. and foreign countries.
For additional information about Kura, please visit the Company’s website at www.kuraoncology.com.
Feb. 24, 2021 7:27 AM ET
By: Dulan Lokuwithana, SA News Editor
Feb 22, 2021
Celsion’s Commitment to Promoting Immune System Solutions to Fight Life-Threatening Diseases
Granted an Accelerated Development Pathway
Designation Provides Potential for an Expedited Regulatory Review.
LAWRENCEVILLE, N.J., Feb. 22, 2021 (GLOBE NEWSWIRE) -- Celsion Corporation (NASDAQ: CLSN), a clinical stage development company focused on DNA based immunotherapy and next generation vaccines, today announced that it has received Fast Track designation from the U.S. Food and Drug Administration (FDA) for GEN-1, its DNA-mediated interleukin-12 (IL-12) immunotherapy currently in Phase II development for the treatment of advanced ovarian cancer. GEN-1 was designed using TheraPlas, Celsion's proprietary, synthetic, non-viral nanoparticle delivery system platform.
About GEN-1 Immunotherapy
GEN-1, designed using Celsion's proprietary TheraPlas platform technology, is an IL-12 DNA plasmid vector encased in a nanoparticle delivery system, which enables cell transfection followed by persistent, local secretion of the IL-12 protein. IL-12 is one of the most active cytokines for the induction of potent anti-cancer immunity acting through the induction of T-lymphocyte and natural killer (NK) cell proliferation. The Company has previously reported positive safety and encouraging Phase I results with GEN-1 given as monotherapy or a combination therapy in patients with advanced peritoneally metastasized primary or recurrent ovarian cancer, and recently completed a Phase Ib dose-escalation trial (OVATION I Study) of GEN-1 in combination with carboplatin and paclitaxel in patients with newly diagnosed ovarian cancer.
GEN-1 is the subject of Celsion’s Phase II OVATION 2 Study, which combines GEN-1 with standard-of-care neoadjuvant chemotherapy (NACT) in patients newly diagnosed with Stage III/IV ovarian cancer. NACT is designed to shrink the cancer as much as possible for optimal surgical removal after three cycles of chemotherapy. Following NACT, patients undergo interval debulking surgery, followed by three adjuvant cycles of chemotherapy and up to nine additional weekly GEN-1 treatments, the goal of which is to delay progression and improve overall survival. The OVATION 2 Study is an open-label, 1-to-1 randomized trial, 80% powered to show the equivalent of a 33% improvement in progression-free survival (PFS) (HR=0.75), the primary endpoint, when comparing the treatment arm (standard of care + GEN-1) with the control arm (standard of care alone).
For more information on Celsion, visit www.celsion.com.
February 16, 2021 at 7:05 AM EST
ROCKVILLE, Md., Feb. 16, 2021 /PRNewswire/ --
REGENXBIO Inc. (Nasdaq: RGNX) reported at the Angiogenesis, Exudation, and Degeneration 2021 conference additional positive interim data from Cohorts 4 and 5 of its RGX-314 Phase I/IIa trial for the treatment of wet age-related macular degeneration (wet AMD), and Cohort 3 of its Long-Term Follow-Up (LTFU) study. RGX-314 is a potential best-in-class, one-time gene therapy for the treatment of wet AMD.
RGX-314 is being developed as a potential one-time treatment for wet AMD, diabetic retinopathy, and other chronic retinal conditions. RGX-314 consists of the NAV® AAV8 vector, which encodes an antibody fragment designed to inhibit vascular endothelial growth factor (VEGF). RGX-314 is believed to inhibit the VEGF pathway by which new, leaky blood vessels grow and contribute to the accumulation of fluid in the retina.
REGENXBIO is advancing two separate routes of administration of RGX-314 to the eye, through a standardized subretinal delivery procedure as well as delivery to the suprachoroidal space. REGENXBIO has licensed certain exclusive rights to the SCS Microinjector® from Clearside Biomedical, Inc. to deliver gene therapy treatments to the suprachoroidal space of the eye.
View original content to download multimedia:http://www.prnewswire.com/news-releases/regenxbio-announces-additional-positive-interim-phase-iiia-and-long-term-follow-up-data-of-rgx-314-for-the-treatment-of-wet-amd-301228344.html
Feb. 16, 2021 7:36 AM ET
ESSA Pharma Presents Favorable Initial Phase 1 Clinical Pharmacology Data of EPI-7386 for Advanced Forms of Prostate Cancer at the 2021 ASCO Genitourinary Cancers Symposium Vancouver, Canada and Houston, Texas, February 11, 2021 - ESSA Pharma Inc. (“ESSA”, or the “Company”) (NASDAQ: EPIX), a clinical-stage pharmaceutical company focused on developing novel therapies for the treatment of prostate cancer, today will present preclinical and clinical pharmacology data from ESSA’s Phase 1 clinical trial of EPI-7386 for the treatment of patients with metastatic castration-resistant prostate cancer (“mCRPC”) at the 2021 American Society of Clinical Oncology Genitourinary (“ASCO GU”) Cancers Symposium. EPI-7386, ESSA’s lead product candidate, is an investigational, highly-selective, oral, small molecule inhibitor of the androgen receptor’s Nterminal domain. ASCO GU is being held virtually from Thursday, February 11 to Saturday, February 13, 2021. The oral poster presentation titled, “Preclinical and clinical pharmacology of EPI-7386, an androgen receptor Nterminal domain inhibitor for castration-resistant prostate cancer,” will be presented and available for viewing starting February 11 at 8:00am ET.
The poster is available on the ASCO GU Virtual Symposium and on the “Events & Presentations” section of the Company’s website at www.essapharma.com.
About EPI-7386 EPI-7386 is an investigational, highly-selective, oral, small molecule inhibitor of the N-terminal domain of the androgen receptor. EPI-7386 is currently being studied in a Phase 1 clinical trial (NCT04421222) in men with metastatic castration-resistant prostate cancer (“mCRPC”) whose tumors have progressed on current standard-ofcare therapies. The Phase I clinical trial of EPI-7386 began in calendar Q3 of 2020 following FDA allowance of the IND and Health Canada acceptance. The U.S. FDA has granted Fast Track designation to EPI-7386 for the treatment of adult male patients with mCRPC resistant to standard-of-care treatment. ESSA retains all rights to EPI-7386 worldwide.
r. For more information, please visit www.essapharma.com
At ESSA, we are focused on the development of small molecule drugs for the treatment of cancer, with an initial focus on advanced prostate cancer
Feb. 11, 2021 2:19 PM ET ESSA Pharma Inc. (EPIX)
By: Dulan Lokuwithana, SA News Editor
February 9, 2021PDF Version
Lille, France; Cambridge, MA; February 09, 2021 - GENFIT (Nasdaq and Euronext: GNFT), a late-stage biopharmaceutical company dedicated to improving the lives of patients with metabolic and liver diseases, today announced that the positive results from the Phase 2 clinical trial evaluating elafibranor in patients with Primary Biliary Cholangitis (PBC) with incomplete response to ursodeoxycholic acid (UDCA) have been published in the Journal of Hepatology.
GENFIT is a publicly traded company listed on the Nasdaq Global Select Market and on compartment B of Euronext’s regulated market in Paris (Nasdaq and Euronext: GNFT). www.genfit.com
Dr. Jörn Schattenberg, Director Metabolic Liver Research Program, University Medical Center, Mainz, Germany added: “These promising findings along with existing safety data derived from past clinical trials suggest elafibranor is a promising development candidate as a potential novel treatment for patients with PBC. Regulatory authorities know the disease well and there remains an important unmet need to be addressed as many patients at present remain without a long-term therapeutic option.”
Based upon the Phase 2 data, elafibranor was granted Breakthrough Therapy designation by the Food and Drug Administration (FDA), as well as Orphan Drug designation by the FDA and the European Medicines Agency (EMA). In September 2020, GENFIT initiated enrollment of patients in ELATIVE™, a global pivotal Phase 3 clinical trial to evaluate the efficacy and safety of elafibranor in patients with PBC and an inadequate response to UDCA. The randomized study (2:1, elafibranor: placebo) will evaluate approximately 150 patients following 52 weeks of treatment. Topline data are expected in Q1 2023.
Dr. Kris V. Kowdley, Director, Liver Institute Northwest, Clinical Professor Elson S. Floyd College of Medicine, Washington State University added: “These encouraging Phase 2 data are particularly exciting as they highlight a favorable trend in pruritus, which is a debilitating symptom of PBC and one that significantly affects patients’ quality of life. These data suggest that elafibranor is a promising drug candidate, and I’m eager to see whether this trend becomes more significant following longer-term administration, while maintaining the favorable safety/tolerability profile we have seen in the Phase 2 trial.”
January 26, 2021 9:06 am EST
Clinical Trial is Part of the Company’s HIV-1 Prevention Clinical Program Studying Islatravir as a Long-Acting PrEP Agent
KENILWORTH, N.J.--(BUSINESS WIRE)-- Merck (NYSE: MRK), known as MSD outside the United States and Canada, today announced new interim data from the Phase 2a trial (NCT04003103) in adults evaluating the safety, tolerability and pharmacokinetics (PK) of the once-monthly oral islatravir tablet -- the company’s investigational oral nucleoside reverse transcriptase translocation inhibitor (NRTTI) -- for pre-exposure prophylaxis (PrEP). Interim findings demonstrate that once-monthly oral islatravir achieved the pre-specified efficacy PK threshold for PrEP at both of the two doses studied (60 mg and 120 mg). In the interim analysis using blinded data, both monthly doses of islatravir were found to have an acceptable tolerability profile. These data are shared as a late-breaking oral presentation during the virtual 2021 HIV Research for Prevention Conference (HIVR4P 2021) and featured in the official press conference of HIVR4P 2021.
Islatravir (formerly MK-8591) is Merck’s investigational nucleoside reverse transcriptase translocation inhibitor (NRTTI) under evaluation in clinical trials for the treatment of HIV-1 infection in combination with other antiretrovirals, including the ILLUMINATE clinical trials program for once-daily treatment, as well as for pre-exposure prophylaxis (PrEP) of HIV-1 infection as a single agent, across a variety of formulations.
To learn more about Merck’s infectious diseases pipeline, visit www.merck.com.
View source version on businesswire.com: https://www.businesswire.com/news/home/20210126005705/en/
- 77% of Bunionectomy Patients Receiving HTX-011 Required No Opioids to Manage Their Postoperative Pain Through 72 Hours After Surgery and Continued To Be Opioid-Free Through 28 Days of Recovery -
SAN DIEGO, Jan. 21, 2021 /PRNewswire/ -- Heron Therapeutics, Inc. (Nasdaq: HRTX
About HTX-011 for Postoperative Pain (ZYNRELEF in the European Union and European Economic Area)
HTX-011, an investigational non-opioid analgesic, is a dual-acting, fixed-dose combination of the local anesthetic bupivacaine with a low dose of the nonsteroidal anti-inflammatory drug meloxicam. It is the first and only extended-release local anesthetic to demonstrate in Phase 3 studies significantly reduced pain and opioid use through 72 hours compared to bupivacaine solution, the current standard-of-care local anesthetic for postoperative pain control. The U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy designation to HTX-011 and the New Drug Application (NDA) received Priority Review designation. A complete response letter (CRL) was received from the FDA regarding the NDA for HTX-011 in June 2020 relating to non‑clinical information. No clinical safety or efficacy issues and no chemistry, manufacturing and controls (CMC) issues were identified. Heron resubmitted an NDA to the FDA for HTX-011 in November 2020 and the FDA set a Prescription Drug User Fee Act goal date of May 12, 2021. Heron is working to respond to a list of questions received from Health Canada in July 2020. In September 2020, the European Commission (EC) granted a marketing authorization for ZYNRELEF (also known as HTX-011) for the treatment of somatic postoperative pain from small- to medium-sized surgical wounds in adults. The EC's centralized marketing authorization is valid for the 27 countries that are members of the European Union, and the other countries in the European Economic Area, including the United Kingdom.
For more information, visit www.herontx.com.
View original content:http://www.prnewswire.com/news-releases/heron-therapeutics-announces-publication-of-results-from-epoch-1-follow-on-study-of-htx-011-in-patients-undergoing-bunionectomy-surgery-301212178.html
Thu January 21, 2021 8:30 AM|PR Newswire| About: HRTXPR Newswire
SAN DIEGO, Jan. 21, 2021 /PRNewswire/ -- Heron Therapeutics, Inc. (HRTX),
Jan. 21, 2021 12:19 PM ET Heron Therapeutics, Inc. (HRTX)
16 January 2021
Issued: London, UK
GlaxoSmithKline (GSK) plc announced updated data from GARNET cohort F evaluating dostarlimab in mismatch repair-deficient (dMMR) non-endometrial advanced solid cancers being presented today at the 2021 American Society of Clinical Oncology Gastrointestinal Cancers Symposium (ASCO GI). Study results (abstract #9) showed a 38.7% objective response rate (ORR) (N=106, 95% CI; 29.4–48.6) in patients with dMMR advanced solid cancers who received dostarlimab, an investigational anti-programmed death-1 (PD-1) monoclonal antibody. Additionally, after a median follow-up of 12.4 months, the median duration of response (DoR) had not yet been reached, and responses were durable across tumour types.
Dostarlimab is a humanised PD-1 monoclonal antibody that binds with high affinity to the PD-1 receptor and blocks its interaction with the ligands PD-L1 and PD-L2.[ii] In addition to GARNET, dostarlimab is being investigated in other registrational enabling studies, including the phase 3 RUBY study for patients with recurrent or primary advanced endometrial cancer in combination with standard of care (SOC) chemotherapy[iii] and the phase 3 FIRST study of platinum-based therapy with dostarlimab and niraparib versus SOC platinum-based therapy as first-line treatment of stage III or IV non-mucinous epithelial ovarian cancer. Dostarlimab is also being evaluated in combination with other therapeutic agents for patients with advanced solid tumours or metastatic cancer.
Dostarlimab was discovered by AnaptysBio and TESARO, Inc. under a Collaboration and Exclusive License Agreement signed in March 2014. The collaboration has resulted in three monospecific antibody drugs that have progressed into the clinic. These are: dostarlimab (GSK4057190), a PD-1 antagonist; cobolimab, (GSK4069889), a TIM-3 antagonist; and GSK4074386, a LAG-3 antagonist. GSK is responsible for the ongoing research, development, commercialisation, and manufacture of each of these products under the Agreement.
A Study of Dostarlimab (TSR-042) Plus Carboplatin-paclitaxel Versus Placebo Plus Carboplatin-paclitaxel in Patients with Recurrent or Primary Advanced Endometrial Cancer (RUBY). https://clinicaltrials.gov/ct2/show/NCT03981796. Accessed December 2020.
For further information please visit www.gsk.com/about-us.
Fri January 15, 2021 8:01 AM|GlobeNewswire|About: NCNA
Promising Efficacy Signals Including a 62% Disease Control Rate and a Partial Response in Patients who had Progressed on Prior Fluoropyrimidine Therapy
NuCana Presents Encouraging Data at ASCO GI for NUC-3373 in Heavily Pre-Treated Patients with Metastatic Colorectal Cancer
Safety Profile Continues to be Favorable
EDINBURGH, United Kingdom, Jan. 15, 2021 (GLOBE NEWSWIRE) -- NuCana plc (NCNA) today announced interim data from the ongoing NuTide:302 study at the ASCO GI Conference, being held virtually January 15-17, 2021.
NuTide:302 is a three-part study investigating NUC-3373, NuCana’s targeted thymidylate synthase inhibitor, in heavily pre-treated patients with metastatic colorectal cancer. The study is evaluating NUC-3373’s optimal dose and schedule in combination with agents commonly used to treat patients with colorectal cancer and is assessing safety, pharmacokinetics and anti-cancer activity. NUC-3373 has been designed to overcome the main challenges associated with 5-FU and capecitabine, including cancer-resistance mechanisms which limit efficacy, off-target toxicity and administration burdens.
Jan. 15, 2021 8:32 AM ET NuCana plc (NCNA)
By: Mamta Mayani, SA News Editor
LOS ANGELES, Calif., Jan. 15, 2021 - Puma Biotechnology, Inc. (NASDAQ: PBYI), a biopharmaceutical company, announced interim results from the biliary tract cancers cohort of the ongoing SUMMIT trial of neratinib at the virtual 2021 Gastrointestinal Cancers Symposium hosted by the American Society of Clinical Oncology (ASCO GI) that is currently taking place. The presentation, entitled, “Targeting HER2 (ERBB2) mutation-positive advanced biliary tract cancers with neratinib: Results from the phase II SUMMIT “basket” trial,” is being presented at a Poster Session by James J. Harding, MD, Regional Director, Early Drug Development, Memorial Sloan Kettering Cancer Center, an investigator of the trial. A copy of this poster presentation is available on the Puma website.
Further information about Puma Biotechnology may be found at www.pumabiotechnology.com.
NERLYNX® (neratinib) tablets, for oral use
INDICATIONS AND USAGE: NERLYNX is a kinase inhibitor indicated:
Please see Full Prescribing Information for additional safety information.
Fri January 15, 2021 8:03 AM| Business Wire|About: PBYI
LOS ANGELES--(BUSINESS WIRE)-- Puma Biotechnology, Inc. (PBYI)
Jan 15 | 2021Download PDF
– Data Presented at the 2021 ASCO GI Cancers Symposium –
BOSTON--(BUSINESS WIRE)--Jan. 15, 2021-- AVEO Oncology (Nasdaq: AVEO) today announced the presentation of results from the Phase 1b portion of the Phase 1b/2 DEDUCTIVE clinical trial of tivozanib (FOTIVDA®), AVEO’s vascular endothelial growth factor receptor (VEGFR) tyrosine kinase inhibitor (TKI) drug candidate, in combination with IMFINZI® (durvalumab), AstraZeneca’s (LSE/STO/Nasdaq: AZN) human monoclonal antibody directed against programmed death-ligand 1 (PD-L1), in patients with advanced or metastatic hepatocellular carcinoma (HCC). The results are being presented today in a poster session at the 2021 American Society of Clinical Oncology Gastrointestinal (ASCO GI) Cancers Symposium being held virtually.
Tivozanib (FOTIVDA®) in Combination with IMFINZI® (durvalumab)
About Tivozanib (FOTIVDA®)
Tivozanib is an oral, once-daily, next-generation VEGFR TKI discovered by Kyowa Kirin Co. and approved as FOTIVDA® for the treatment of adult patients with advanced RCC in the European Union and other countries in the territory of the Company’s partner, EUSA Pharma (UK) Limited (EUSA territory). It is a potent, selective and long half-life inhibitor of all three VEGF receptors and is designed to optimize VEGF blockade while minimizing off-target toxicities, potentially resulting in improved efficacy and minimal dose modifications.1,2 Tivozanib is being studied in the TIVO-3 trial, which is supporting a regulatory submission of tivozanib in the U.S. seeking marketing approval as a treatment for adult patients with relapsed or refractory advanced RCC. Tivozanib has been shown to significantly reduce regulatory T-cell production in preclinical models3 and has demonstrated synergy in combination with nivolumab (anti PD-1) in a Phase 2 study in RCC.4 Tivozanib has been investigated in several tumor types, including renal cell, hepatocellular, colorectal, ovarian and breast cancers. Tivozanib is also being studied by partner Kyowa Kirin Co. in non-oncology indications.
View source version on businesswire.com: https://www.businesswire.com/news/home/20210115005128/en/
Fri January 15, 2021 8:00 AM| Business Wire|
– Data Presented at the 2021 ASCO GI Cancers Symposium –
BOSTON--(BUSINESS WIRE)-- AVEO Oncology (Nasdaq: AVEO)
Cardiff Oncology Presents Data that Continues to Demonstrate the Clinical Benefit of Onvansertib in KRAS-Mutated mCRC and Initial Findings from its Expanded Access Program- Of the 12 Phase 1b patients evaluable for efficacy as of the ASCO-GI data cut-off date (November 1, 2020), 5 (42%) achieved a partial response (PR) and 8 (67%) have shown a durable response ranging from 6.1 to 13.7 months- Since the ASCO-GI data cut-off date, 2 additional Phase 1b patients have had their initial 8-week scan showing stable disease (SD) and both remain on treatment to-date- Time to achieving a PR ranges from 2 to 6 months in patients on treatment- The recommended Phase 2 dose (RP2D) of onvansertib has been established at 15mg/m2 and the Phase 2 segment of the ongoing trial is open to full enrollment of 26 patients across 6 sites in the U.S.- In the Expanded Access Program (EAP), 6 (66%) of the initial 9 patients treated have shown tumor shrinkage and remain on treatment to-date with durable responses lasting an average of 6 months; 5 different KRAS mutation subtypes are represented (G12A, G12C, G12V, G13D, A146T); all patients had received prior treatment with FOLFIRI- Decreases in the KRAS mutational burden in patients after the first cycle of treatment have been predictive of subsequent tumor shrinkage in both the clinical trial and EAP
SAN DIEGO, Jan. 15, 2021 /PRNewswire/ -- Cardiff Oncology, Inc. (Nasdaq: CRDF)
For more information, please visit https://www.cardiffoncology.com.
About the Phase 1b/2 Trial of Onvansertib in KRAS-mutated mCRC
This is a multi-center, open-label Phase 1b/2 trial of onvansertib in combination with standard-of-care FOLFIRI and Avastin® (bevacizumab) to evaluate the safety and preliminary efficacy of the combination regimen in the second-line treatment of patients with KRAS-mutated mCRC. The trial, A Phase 1b/2 Study of Onvansertib (PCM-075) in Combination with FOLFIRI and Bevacizumab for Second–Line Treatment of Metastatic Colorectal Cancer in Patients with a KRAS Mutation, will enroll up to 44 patients with a KRAS mutation and histologically confirmed metastatic and unresectable disease. In addition, eligible patients must have failed treatment with, or be intolerant to, FOLFOX (fluoropyrimidine and oxaliplatin) with or without bevacizumab. The trial is being conducted at six cancer centers across the U.S.: USC Norris Comprehensive Cancer Center, The Mayo Clinic (Arizona, Rochester and Jacksonville), Kansas University Medical Center (KUMC) and CARTI Cancer Center. For more information on the trial, please visit https://clinicaltrials.gov/ct2/show/NCT03829410.
Company Release - 01/15/2021
VANCOUVER, British Columbia--(BUSINESS WIRE)-- Zymeworks Inc. (NYSE: ZYME), a clinical-stage biopharmaceutical company developing multifunctional biotherapeutics, today announced new and updated clinical data for the HER2‑targeted bispecific antibody zanidatamab, in both HER2-expressing biliary tract cancer (BTC) and gastroesophageal adenocarcinoma (GEA). The data are being presented today at the American Society of Clinical Oncology (ASCO) Gastrointestinal Cancers Symposium, taking place virtually January 15 – 17, 2021.
This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20210115005163/en/
Zanidatamab is a bispecific antibody, based on Zymeworks’ Azymetric™ platform, that can simultaneously bind two non-overlapping epitopes of HER2, known as biparatopic binding. This unique design results in multiple mechanisms of action including dual HER2 signal blockade, increased binding, and removal of HER2 protein from the cell surface, and potent effector function leading to encouraging antitumor activity in patients. Zymeworks is developing zanidatamab in multiple Phase 1, Phase 2, and pivotal clinical trials globally as a targeted treatment option for patients with solid tumors that express HER2. The FDA has granted Breakthrough Therapy designation for zanidatamab in patients with previously treated HER2 gene-amplified BTC, and two Fast Track designations to zanidatamab, one as a single agent for refractory BTC and one in combination with standard of care chemotherapy, for first-line gastroesophageal adenocarcinoma (GEA). These designations mean zanidatamab is eligible for Accelerated Approval, Priority Review and Rolling Review, as well as intensive FDA guidance on an efficient drug development program. Zanidatamab has also received Orphan Drug designations for the treatment of biliary tract, gastric and ovarian cancers, as well as Orphan Drug designation for the treatment of gastric cancer from the European Medicines Agency.
For additional information about Zymeworks, visit www.zymeworks.com
Fri January 15, 2021 8:30 AM|Business Wire|About: ZYME
VANCOUVER, British Columbia--(BUSINESS WIRE)-- Zymeworks Inc. (ZYME)
January 15, 2021 at 6:55 AM ESTPDF Version
SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Jan. 15, 2021-- Five Prime Therapeutics, Inc. (NASDAQ: FPRX)
FGFR2b Overexpression and Reinforce Potential of Bemarituzumab Plus Chemotherapy
The fibroblast growth factor (FGF)/fibroblast growth factor receptor (FGFR) pathway is implicated in the development and growth of cancer cells. FGFR2b is a splice form of FGFR which can be found in tumors of epithelial origin. Data from the FIGHT trial suggests that approximately 30 percent of patients with non HER2 positive gastroesophageal cancers overexpress FGFR2b.1 Five Prime and Roche Tissue Diagnostics have also found that FGFR2b is overexpressed in numerous other cancers, including squamous non-small cell lung cancer (NSCLC), triple negative breast cancer (TNBC), ovarian, pancreatic and intrahepatic cholangiocarcinoma.
Bemarituzumab (anti-FGFR2b) is a first-in-class targeted antibody that blocks fibroblast growth factors (FGFs) from binding and activating FGFR2b, inhibiting several downstream pro-tumor signaling pathways and potentially slowing cancer progression. Bemarituzumab is being developed in gastric and GEJ cancer as a targeted therapy for tumors that overexpress FGFR2b. The company is also evaluating the potential for bemarituzumab in other cancers that overexpress FGFR2b.
Five Prime granted an exclusive license to Zai Lab to develop and commercialize bemarituzumab in Greater China, and Zai Lab collaborated with Five Prime on the Phase 2 FIGHT trial in Greater China.
For more information, please visit www.fiveprime.com.
View source version on businesswire.com: https://www.businesswire.com/news/home/20210115005136/en/
Fri January 15, 2021 6:55 AM|Business Wire|About: FPRX
NIDA-Funded Study Evaluating Extended-Release Injectable Naltrexone Plus Bupropion for the Treatment of Methamphetamine Use Disorder Published in New England Journal of Medicine
DUBLIN, Jan. 14, 2021 /PRNewswire/ -- Results from a National Institute on Drug Abuse (NIDA)-funded study evaluating the efficacy and safety of naltrexone for extended-release injectable suspension (XR-NTX) administered once every three weeks plus oral extended-release bupropion administered daily as a combination treatment for adults with moderate or severe methamphetamine use disorder (MUD) were published today by Dr. Madhukar H. Trivedi et al. in the New England Journal of Medicine (NEJM).1 This is the second published study evaluating this combination regimen for the treatment of MUD.2
VIVITROL® (naltrexone for extended-release injectable suspension)
VIVITROL® (naltrexone for extended-release injectable suspension) is a once-monthly medication for the treatment of alcohol dependence and for the prevention of relapse to opioid dependence, following opioid detoxification. Treatment with VIVITROL should be part of a comprehensive management program that includes psychosocial support. For more information, visit www.vivitrol.com.
For more information, please visit Alkermes' website at www.alkermes.com.
Cision View original content to download multimedia:http://www.prnewswire.com/news-releases/nida-funded-study-evaluating-extended-release-injectable-naltrexone-plus-bupropion-for-the-treatment-of-methamphetamine-use-disorder-published-in-new-england-journal-of-medicine-301208061.html
Thu January 14, 2021 7:00 AM|PR Newswire|About: ALKS
DUBLIN, Jan. 14, 2021 /PRNewswire/ -
Jan. 14, 2021 8:38 AM ET Alkermes plc (ALKS)
By: Mamta Mayani, SA News Editor
VIVITROL® (naltrexone for extended-release injectable suspension) is a once-monthly medication for the treatment of alcohol dependence as well as for the prevention of relapse to opioid dependence, following opioid detoxification. Treatment with VIVITROL should be part of a comprehensive management program that includes psychosocial support.
For more information, visit www.vivitrol.com.
January 13, 2021
SOUTH SAN FRANCISCO, Calif., Jan. 13, 2021 (GLOBE NEWSWIRE) -- Harpoon Therapeutics, Inc. (NASDAQ: HARP), a clinical-stage immunotherapy company developing a novel class of T cell engagers, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for HPN217 for the treatment of multiple myeloma. HPN217, a tri-specific T cell activating recombinant protein construct (TriTAC®) targets B-cell maturation antigen (BCMA), a well-validated antigen expressed on malignant multiple myeloma cells. Harpoon has four drug product candidates in clinical development for the treatment of solid and hematologic malignancies based on its proprietary TriTAC platform.
HPN217 FOR TREATMENT OF MULTIPLE MYELOMA
About the Phase 1/2 Trial for HPN217
In April 2020, Harpoon announced dosing of the first patient with HPN217 (BCMA TriTAC) in a Phase 1/2 clinical trial focused on relapsed/refractory multiple myeloma (RRMM). HPN217 is covered by a global development and option agreement with AbbVie Inc. (NYSE: ABBV). Dose escalation for HPN217 in the Phase 1/2 clinical trial is progressing rapidly. HPN217 has been well tolerated, and no DLTs had been observed as of the most recent December 1, 2020 data cutoff date. A presentation of interim data is anticipated in 2021, with initiation of a dose expansion cohort in the second half of 2021.
For additional information about the trial, please visit clinicaltrials.gov using the identifier NCT04184050.
For additional information about Harpoon Therapeutics, please visit www.harpoontx.com.
SOUTH SAN FRANCISCO, Calif., Jan. 13, 2021 (GLOBE NEWSWIRE) -- Harpoon Therapeutics, Inc. (HARP),
-TransConTM C-Type Natriuretic Peptide (CNP), an investigational once-weekly long-acting prodrug of CNP, is ready for Phase 2 Clinical Trial in China-
SHANGHAI, China, January 7, 2021 - VISEN Pharmaceuticals, a biotech company committed to the treatment of endocrine-related diseases, introducing the world’s leading treatment methods and drugs into the China market and hoping to provide more Chinese patients quick access to the world's most advanced and reliable treatment solutions, today announced that the China Center for Drug Evaluation (CDE) of National Medical Products Administration (NMPA) has approved the IND application to conduct a phase 2 clinical trial of TransConTM C-Type Natriuretic Peptide (CNP) for patients with achondroplasia (ACH). The trial is ready to be launched in China as“the ACcomplisH China trial”, in coordination with the ACcomplisH trial, an ongoing phase 2 global clinical trial of TransCon CNP being conducted globally outside of China by Ascendis Pharma.
TransConTM C-Type Natriuretic Peptide (CNP
About TransCon™ Technology
TransCon refers to “transient conjugation.” The proprietary TransCon platform is an innovative technology designed to create new therapies that potentially optimize therapeutic effect, including efficacy, safety and dosing frequency.
TransCon molecules have three components: an unmodified parent drug, an inert carrier that protects it, and a linker that temporarily binds the two. When bound, the carrier inactivates and shields the parent drug from clearance. When injected into the body, physiologic conditions (e.g., pH and temperature) initiate the release of the active, unmodified parent drug in a predictable manner. Because the parent drug is unmodified, its original mode of action may be maintained. TransCon technology is designed to be applied broadly to a protein, peptide or small molecule in multiple therapeutic areas, and to be used systemically or locally.
Phase 2 Clinical Trial of TransCon CNP Sets to Accelerate Research and Development
Preclinical and clinical data have shown that increased CNP can offset the effects of the FGFR3 mutation, thereby promoting bone growth5. However, the half-life of natural CNP in the human body is short, only 2-3 minutes6, requiring continuous intravenous infusion, posing significant difficulties for clinical application.
Thu January 7, 2021 5:45 AM|PR Newswire|About: ASNDPR Newswire
SHANGHAI, Jan. 7, 2021 /PRNewswire/ -- VISEN Pharmaceuticals
Jan 8, 2021 PDF Version
Durable and Clinically Meaningful Responses Reported from Phase 1/2 Studies of DTX401 for GSDIa and DTX301 for OTC
Phase 3 Studies for DTX401 and DTX301 to Begin in 2021
IND for UX701 for Wilson Disease Submitted; Expect to Enter Clinic in First Half 2021 using AAV Drug Product Made by HeLa PCL Platform
NOVATO, Calif. , Jan. 08, 2021 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE)
DTX401, an investigational adeno-associated virus (AAV)
DTX401 (GSDIa) Program
Phase 1/2 data update: All patients (n=9) responding and demonstrating continued improvement of glucose control while reducing or eliminating cornstarch therapy
All nine patients continue to demonstrate improved glucose control while tapering or discontinuing oral glucose replacement with cornstarch and improvements in energy metabolism pathways over the long term. Patients continue to taper the amount and frequency of cornstarch dosing with progress in eliminating overnight and daytime cornstarch doses. At the primary evaluation timepoint at Week 52, the overall mean reduction in cornstarch was 77% across all three cohorts, including two patients in Cohort 3 showing a reduction of greater than 75%. Longer term follow-up for more than two years for the three patients in Cohort 1 have shown sustained and continued cornstarch reductions with a mean reduction of 91% through weeks 104 and 120. Two patients (one each from Cohort 1 and 3) are completely off cornstarch therapy at weeks 127 and 60, respectively.
For more information on Ultragenyx, please visit the company’s website at www.ultragenyx.com.
PFIZER DOSES FIRST PARTICIPANT IN PHASE 3 STUDY FOR DUCHENNE MUSCULAR DYSTROPHY INVESTIGATIONAL GENE
CIFFREO is a Phase 3 global, multi-center, randomized, double-blind, placebo-controlled study to assess the safety and efficacy of PF-06939926 investigational gene therapy in 99 ambulatory male participants, ages 4 through 7 years, with a genetic diagnosis of DMD who are on a stable daily regimen of glucocorticoids. The participants are negative for neutralizing antibodies against AAV9, as measured by the test done for the study as part of screening.
For more information, visit: ciffreoduchennetrial.com.
PF-06939926 is an investigational recombinant adeno-associated virus serotype 9 (rAAV9) capsid carrying a shortened version of the human dystrophin gene (mini-dystrophin) under the control of a human muscle-specific promotor. The rAAV9 capsid was chosen as the delivery vector because of its potential to target muscle tissue.
to learn more, please visit us on www.pfizer.com
View source version on businesswire.com: https://www.businesswire.com/news/home/20210107005031/en/
ACCORD is the second pivotal trial of AXS-05 in Alzheimer’s disease agitation
No treatments are currently approved for Alzheimer’s disease agitation
NEW YORK, Dec. 31, 2020 (GLOBE NEWSWIRE) -- Axsome Therapeutics, Inc. (NASDAQ: AXSM), a biopharmaceutical company developing novel therapies for the management of central nervous system (CNS) disorders, announced the initiation of the ACCORD (Assessing Clinical Outcomes in Alzheimer’s Disease Agitation) study, a Phase 3, randomized, double-blind, placebo-controlled trial to evaluate the efficacy and safety of AXS-05 in the treatment of Alzheimer’s disease (AD) agitation. AXS-05 (45 mg dextromethorphan-105 mg bupropion modulated delivery tablet) is a novel, oral, investigational NMDA receptor antagonist with multimodal activity. There is currently no approved treatment for AD agitation.
AXS-05 (dextromethorphan-bupropion modulated delivery tablet)
AXS-05 (dextromethorphan-bupropion modulated delivery tablet) is a novel, oral, patent-protected, investigational NMDA receptor antagonist with multimodal activity under development for the treatment of major depressive disorder and other central nervous system (CNS) disorders. AXS-05 utilizes a proprietary formulation and dose of dextromethorphan and bupropion, and Axsome’s metabolic inhibition technology, to modulate the delivery of the components. The dextromethorphan component of AXS-05 is an uncompetitive N-methyl-D-aspartate (NMDA) receptor antagonist, also known as a glutamate receptor modulator, which is a novel mechanism of action, meaning it works differently than currently approved therapies for major depressive disorder. The dextromethorphan component of AXS-05 is also a sigma-1 receptor agonist, nicotinic acetylcholine receptor antagonist, and inhibitor of the serotonin and norepinephrine transporters. The bupropion component of AXS-05 serves to increase the bioavailability of dextromethorphan, and is a norepinephrine and dopamine reuptake inhibitor, and a nicotinic acetylcholine receptor antagonist. AXS-05 is covered by more than 93 issued U.S. and international patents which provide protection out to 2040. AXS-05 has been granted U.S. Food and Drug Administration Breakthrough Therapy and Fast Track designations for Alzheimer’s disease agitation. AXS-05 is not approved by the FDA.
AXS-05 is a novel, oral, investigational NMDA receptor antagonist with multimodal activity under development for the treatment of CNS disorders. AXS-05 consists of a proprietary formulation and dose of bupropion and dextromethorphan, or DM, and utilizes our metabolic inhibition technology. The DM component of AXS-05 is a non-competitive N-methyl-D-aspartate, or NMDA, receptor antagonist, also known as a glutamate receptor modulator. The DM component of AXS-05 is also a sigma-1 receptor agonist, nicotinic acetylcholine receptor antagonist, and inhibitor of the serotonin and norepinephrine transporters. The bupropion component of AXS-05 serves to increase the bioavailability of dextromethorphan, and is a norepinephrine and dopamine reuptake inhibitor, and a nicotinic acetylcholine receptor antagonist. AXS-05 is an investigational drug not yet approved by the FDA.
For more information, please visit the Company’s website at axsome.com.
Exelixis Announces Cabozantinib Significantly Improved Progression-Free Survival in COSMIC-311 Phase 3 Pivotal Trial in Patients with Previously Treated Radioiodine-Refractory Differentiated Thyroid CancerPDF Version
– Cabozantinib reduced the risk of disease progression or death by 78%; hazard ratio = 0.22, (p<0.0001) compared to placebo –
– Exelixis will discuss the trial results and regulatory filing plans with the U.S. Food and Drug Administration (FDA) –
ALAMEDA, Calif.--(BUSINESS WIRE)--Dec. 21, 2020-- Exelixis, Inc. (NASDAQ: EXEL)
About CABOMETYX® (cabozantinib)
In the U.S., CABOMETYX tablets are approved for the treatment of patients with advanced RCC and for the treatment of patients with HCC who have been previously treated with sorafenib. CABOMETYX tablets have also received regulatory approvals in the European Union and additional countries and regions worldwide. In 2016, Exelixis granted Ipsen exclusive rights for the commercialization and further clinical development of cabozantinib outside of the United States and Japan. In 2017, Exelixis granted exclusive rights to Takeda Pharmaceutical Company Limited for the commercialization and further clinical development of cabozantinib for all future indications in Japan. Exelixis holds the exclusive rights to develop and commercialize cabozantinib in the United States.
CABOMETYX is not indicated for radioiodine-refractory differentiated thyroid cancer.
CABOMETYX is a prescription medicine used to treat people with:
It is not known if CABOMETYX is safe and effective in children.
Mon December 21, 2020 7:30 AM|Business Wire|About: EXEL
– Cabozantinib reduced the risk of disease progression or death by 78%; hazard ratio = 0.22, (p<0.0001) compared to placebo –
– Exelixis (EXEL) will discuss the trial results and regulatory filing plans with the U.S. Food and Drug Administration (FDA) –
ALAMEDA, Calif.--(BUSINESS WIRE)-- Exelixis, Inc. (NASDAQ: EXEL)
View source version on businesswire.com: https://www.businesswire.com/news/home/20201221005168/en/
Proof-of-concept phase 1/2 trial to initiate in 2021 to treat severe respiratory viral infections in patients following hematopoietic stem cell transplantation
ALVR106 designed to target devastating diseases caused by four respiratory viruses: respiratory syncytial virus, influenza, parainfluenza virus, and human metapneumovirus
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Dec. 17, 2020-- AlloVir (Nasdaq: ALVR), a late clinical-stage cell therapy company, announced that the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application for ALVR106, an allogeneic, off-the-shelf virus-specific T cell therapy (VST) designed to target infections and diseases caused by respiratory syncytial virus (RSV), influenza, parainfluenza virus (PIV), and human metapneumovirus (hMPV). The IND enables AlloVir to initiate a Phase 1/2 proof-of-concept clinical study in allogeneic and autologous hematopoietic stem cell transplant (HSCT) patients with respiratory infections caused by RSV, influenza, PIV or hMPV.
ALVR106 is an allogeneic, off-the-shelf, multi-virus specific VST investigational therapy designed to target infections and diseases caused by the respiratory syncytial virus (RSV), influenza, parainfluenza virus (PIV), and human metapneumovirus (hMPV). In vitro data demonstrate that ALVR106 has antiviral activity against each of the targeted viruses with minimal or no activity against non-virus-infected cells. This preclinical data supports the potential for antiviral benefit and safety of ALVR106 when administered to patients.
For more information visit www.allovir.com.
12/16/2020- Rimegepant 75 mg, already approved for the acute treatment of migraine, demonstrated efficacy and safety for the preventive treatment of migraine in this pivotal Phase 3 trial in adults- Rimegepant resulted in a -4.3 day reduction in the mean number of migraine days per month- Approximately half of rimegepant-treated patients demonstrated a 50% or greater reduction in the number of moderate-to-severe migraine days per month- Rimegepant is the first and only CGRP targeting medication in development to show dual efficacy for both the acute and preventive treatment of migraine
NEW HAVEN, Conn., Dec. 16, 2020 /PRNewswire/ --- Biohaven Pharmaceutical Holding Company Ltd. (NYSE: BHVN) announced today that The Lancet published the company's positive results from a Phase 3 clinical trial of rimegepant for the preventive treatment of migraine in adults. These data show rimegepant was superior to placebo in the reduction of monthly migraine days. Rimegepant was approved by the U.S. Food and Drug Administration (FDA) for the acute treatment of migraine in February 2020. Rimegepant is marketed as NURTEC® ODT, and is the first and only calcitonin gene-related peptide (CGRP) receptor antagonist available in an orally disintegrating tablet (ODT) designed for rapid onset of action.
Nurtec ODT (rimegepant)
Biohaven's Nurtec ODT (rimegepant) Received FDA Approval in February 2020. Nurtec ODT is the first and only CGRP receptor antagonist available in an orally disintegrating tablet (ODT) for the acute treatment of migraine, and the only oral CGRP receptor antagonist with product label claims including return to normal functioning and sustained durability of efficacy up to 48 hours after a single dose. – Read the Full Press Release
If you are a patient, caregiver, or healthcare provider with a question regarding Nurtec ODT, please click here.
Visit Nurtec.com for more information.
NURTEC ODT is indicated for the acute treatment of migraine with or without aura in adults.
Nurtec ODT (rimegepant) orally disintegrating tablets is a prescription medicine for the acute treatment of migraine attacks with or without aura in adults. Nurtec ODT is not used as a preventive treatment of migraine. It is not known if Nurtec ODT is safe and effective in children.
More information about Biohaven is available at www.biohavenpharma.com.
PDF Version- Substantial improvement observed across all study endpoints, including overall survival, progression free survival and overall response rate, in the most challenging PDAC patients -- Company plans to meet with regulatory authorities as it evaluates next development steps -- Company to host Key Opinion Leader (KOL) webinar to discuss these results today, December 16, at 8:00 am EST; registration link below -
TEL AVIV, Israel, Dec. 16, 2020 /PRNewswire/ -- BioLineRx Ltd. (NASDAQ: BLRX) (TASE: BLRX), a late clinical-stage biopharmaceutical Company focused on oncology, today announced results from the triple combination arm of the Company's COMBAT/KEYNOTE-202 clinical study evaluating motixafortide (BL-8040) in combination with KEYTRUDA® (pembrolizumab) and chemotherapy in patients with second-line stage IV pancreatic ductal adenocarcinoma (PDAC).
About Motixafortide in Cancer Immunotherapy
Motixafortide is targeting CXCR4, a chemokine receptor and a well validated therapeutic target that is over-expressed in many human cancers including PDAC. CXCR4 plays a key role in tumor growth, invasion, angiogenesis, metastasis and therapeutic resistance, and CXCR4 overexpression has been shown to be correlated with poor prognosis.
Motixafortide is a short synthetic peptide used as a platform for cancer immunotherapy with unique features allowing it to function as a best-in-class antagonist of CXCR4. It shows high-affinity, long receptor occupancy and acts as an inverse agonist.
In a number of clinical and preclinical studies, motixafortide has been shown to affect multiple modes of action in 'cold' tumors, including immune cell trafficking, tumor infiltration by immune effector T cells, and reduction in immunosuppressive cells (such as MDSCs) within the tumor microenvironment, turning 'cold' tumors, such as pancreatic cancer, into "hot" (i.e., sensitizing them to immune checkpoint inhibitors and chemotherapy).
For additional information on BioLineRx, please visit the Company's website at www.biolinerx.com
Wed December 16, 2020 6:01 AM|PR Newswire|About: BLRX
TEL AVIV, Israel, Dec. 16, 2020 /PRNewswire/ -- BioLineRx Ltd. (BLRX) (TASE: BLRX)
Dec. 16, 2020 6:30 AM ET
Download as PDFDecember 15, 2020
NEW YORK, Dec. 15, 2020 (GLOBE NEWSWIRE) -- Fortress Biotech, Inc. (Nasdaq: FBIO)
About CUTX-101 (Copper Histidinate)
CUTX-101 is in clinical development to treat patients with Menkes disease by replenishing Copper Histidinate, restoring copper homeostasis and maintaining serum copper levels in the normal age appropriate range. CUTX-101 is a subcutaneous injectable formulation of Copper Histidinate manufactured under current good manufacturing practice (“cGMP”) and physiological pH that bypasses the defect in absorption of orally administered copper in patients with Menkes disease. In a Phase 1/2 clinical trial conducted by Stephen G. Kaler, M.D., M.P.H., at the National Institutes of Health (“NIH”), early treatment of patients with Menkes disease with CUTX-101 led to an improvement in neurodevelopmental outcomes and survival. A Phase 3 trial of CUTX-101 in patients with Menkes disease also led by Dr. Kaler has completed enrollment. In August 2020, Cyprium reported positive topline clinical efficacy results for CUTX-101, demonstrating statistically significant improvement in overall survival for Menkes disease subjects who received early treatment (ET) with CUTX-101, compared to an untreated historical control (HC) cohort, with a nearly 80% reduction in the risk of death. A Cyprium-sponsored expanded access protocol for patients with Menkes disease is ongoing at Nationwide Children’s Hospital (https://www.nationwidechildrens.org/specialties/menkes-disease-clinic) and other US medical centers.
Cyprium was founded by Fortress Biotech, Inc. (Nasdaq: FBIO) and is based in New York City. For more information, visit www.cypriumtx.com.
For more information, visit www.fortressbiotech.com.
Tue December 15, 2020 8:00 AM|GlobeNewswire|About: FBIOGlobeNewswire
NEW YORK, Dec. 15, 2020 (GLOBE NEWSWIRE) -- Fortress Biotech, Inc. (FBIO)
Wed December 9, 2020 5:00 PM|GlobeNewswire|About: GTHXGlobeNewswire
G1 Therapeutics is based in Research Triangle Park, N.C. For additional information, please visit www.g1therapeutics.com
G1 is advancing two novel therapies for people living with cancer. Trilaciclib is a first-in-class investigational therapy designed to improve outcomes for people with cancer who are treated with chemotherapy. Rintodestrant is a potential best-in-class oral selective estrogen receptor degrader, or SERD, for the treatment of ER+ breast cancer. In 2020, G1 out-licensed global development and commercialization rights to its differentiated oral CDK4/6 inhibitor, lerociclib.
Thu December 10, 2020 1:00 AM|GlobeNewswire|About: OBSV
GENEVA, Switzerland and BOSTON, MA (December 10, 2020) – ObsEva SA (OBSV)
Yselty® (linzagolix) is a novel, once daily, oral GnRH receptor antagonist with a potentially best-in-class profile. Linzagolix is currently in late-stage clinical development for the treatment of heavy menstrual bleeding associated with uterine fibroids and pain associated with endometriosis. ObsEva licensed linzagolix from Kissei in late 2015 and retains worldwide commercial rights, excluding Asia, for the product. Linzagolix is not currently approved anywhere in the world.
For more information, please visit www.ObsEva.com.
December 10, 2020
PRIMROSE 1 52-week results confirm the sustained efficacy and continued safety of linzagolix (Yselty®), with a potential best-in-class high-dose option…
December 06, 2020
-- Similar Overall and Complete Response Rates Observed Across Frontline AML Patients Ineligible for Intensive Chemotherapy and in High Unmet Need Patients with TP53-Mutant AML --
FOSTER CITY, Calif.--(BUSINESS WIRE)-- Gilead Sciences, Inc. (Nasdaq: GILD) today announced updated results from the magrolimab Phase 1b trial. Magrolimab is an investigational, potential first-in-class, anti-CD47 monoclonal antibody being studied in previously untreated acute myeloid leukemia (AML) patients who are ineligible for intensive chemotherapy, including patients with TP53-mutant AML. The study continues to demonstrate high response rates with magrolimab in combination with azacitidine, with an overall response rate of 63% (n=27/43) among the total patient population and 69% (n=20/29) in TP53-mutant patients. The data were presented at the 62nd American Society of Hematology (ASH) Annual Meeting and Exposition (Abstract #330).
Magrolimab is a potential, first-in-class investigational monoclonal antibody against CD47 and a macrophage checkpoint inhibitor that is designed to interfere with recognition of CD47 by the SIRPα receptor on macrophages, thus blocking the "don't eat me" signal used by cancer cells to avoid being ingested by macrophages. Magrolimab is being developed in several hematologic cancers, including myelodysplastic syndrome (MDS), as well as solid tumor malignancies.
For more information on Gilead Sciences, please visit the company’s website at www.gilead.com.
View source version on businesswire.com: https://www.businesswire.com/news/home/20201206005027/en/
- Beta thalassemia: All seven patients were transfusion independent with 3 to 18 months of follow-up after CTX001 infusion -
- Sickle cell disease: All three patients were free of vaso-occlusive crises with 3 to 15 months of follow-up after CTX001 infusion -
- Nineteen patients have been dosed with CTX001 across both programs -
- The New England Journal of Medicine publishes CTX001 manuscript containing the first report of investigational use of CRISPR/Cas9-based gene editing to treat inherited diseases in humans -
CTX001 is an investigational, autologous, ex vivo CRISPR/Cas9 gene-edited therapy that is being evaluated for patients suffering from TDT or severe SCD, in which a patient’s hematopoietic stem cells are edited to produce high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is a form of the oxygen-carrying hemoglobin that is naturally present at birth, which then switches to the adult form of hemoglobin. The elevation of HbF by CTX001 has the potential to alleviate transfusion requirements for patients with TDT and reduce painful and debilitating sickle crises for patients with SCD.
CRISPR Therapeutics and Vertex Present New Data for Investigational CRISPR/Cas9 Gene-Editing Therapy, CTX001™ at American Society of Hematology Annual Meeting and Exposition, Together With Publication in the New England Journal of Medicine
For more information, please visit www.crisprtx.com.
For company updates and to learn more about Vertex's history of innovation, visit www.vrtx.com
We are investigating the use of genetic therapies aimed at the underlying cause of SCD. The cause of SCD has been known since Linus Pauling described the "first molecular disease" in 1949, yet many people still don't have a treatment to address the underlying cause of their disease. With the discovery of tools like CRISPR gene editing, we now potentially have an opportunity to address diseases at their root cause.
We are collaborating with CRISPR Therapeutics to investigate the use of gene-editing technology, known as CRISPR-Cas9, to discover and develop a new one-time treatment for SCD. CTX001 is an investigational ex-vivo CRISPR gene-edited therapy, which aims to edit a person’s hematopoietic stem cells to produce fetal hemoglobin (HbF; hemoglobin F) in red blood cells. The aim of using the body's own machinery to switch red blood cells back to fetal hemoglobin production is a significant reduction or elimination of symptoms associated with the disease.
Fri December 4, 2020 7:00 AM|Business Wire|About: CRTX
-- GAIN Trial passes futility analysis and will continue to 1-year endpoint following the independent Data Monitoring Committee recommendation; topline results expected on time in December 2021 --
-- Final study enrollment remains at 643; no sample size adjustment --
-- Conference call and webcast today, Friday, December 4, 2020, at 8:30 a.m. EST / 5:30 a.m. PST --
SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)-- Cortexyme, Inc. (CRTX),
To learn more about Cortexyme, visit www.cortexyme.com
View source version on businesswire.com: https://www.businesswire.com/news/home/20201204005235/en/
Thu December 3, 2020 7:00 AM|PR NewswirePR Newswire
RARITAN, N.J., Dec. 3, 2020 /PRNewswire/ -- The Janssen Pharmaceutical Companies of Johnson & Johnson
Tue December 1, 2020 7:00 AM|GlobeNewswire|About: AGIO
– 40 Percent of Patients Treated with Mitapivat Achieved a Sustained Hemoglobin Increase of ≥1.5 g/dL Compared to 0 Placebo Patients (p<0.0001) –
– Safety Profile Consistent with Previously Reported Data –
– Topline Data from the Mitapivat Phase 3 ACTIVATE-T Trial in Regularly Transfused PK Deficiency Expected in Q1 2021 –
CAMBRIDGE, Mass., Dec. 01, 2020 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (AGIO)
Mitapivat Clinical Development
ACTIVATE is one of two studies intended to support a marketing application for mitapivat in patients with PK deficiency. In addition to the ACTIVATE trial, Agios has fully enrolled the global, pivotal Phase 3 ACTIVATE-T trial in adults with PK deficiency who receive regular transfusions. The primary endpoint of this single-arm trial is the proportion of patients who achieve a reduction in transfusion burden compared to individual historical transfusion burden standardized to 24 weeks. Agios anticipates reporting topline ACTIVATE-T data in Q1 2021. Agios is also enrolling an extension study for adults with PK deficiency previously enrolled in ACTIVATE or ACTIVATE-T, which is designed to evaluate the long-term safety, tolerability and efficacy of treatment with mitapivat.
There are no currently approved therapies for PK deficiency. For more information, please visit www.knowpkdeficiency.com.
For more information, please visit the company's website at www.agios.com.
Dec 1, 2020
Mon November 30, 2020 7:00 AM | GlobeNewswire | About: PRVL
NOVEMBER 30, 2020