Moorestown, New Jersey, United States
biotecHOPE™ 2022
biotecHOPE™ 2022
CAR-T Candidate BNT211
CAR-T Candidate BNT211
CAR-T Candidate BNT211
CAR-T Candidate BNT211
Eplontersen, formerly known as IONIS-TTR-LRx and AKCEA-TTR-LRx
BioNTech Receives Priority Medicines (PRIME) Designation from EMA for Enhanced Regulatory Support of CAR-T Candidate BNT211 in Testicular Cancer
- First BioNTech product candidate to receive priority medicines (PRIME) designation by the European Medicines Agency for enhanced regulatory support facilitating the clinical development of the investigational cell therapy candidate BNT211 in the third- or later-line setting in patients with heavily pretreated testicular cancer
- Designation follows positive interim Phase 1/2 data for BNT211 demonstrating an encouraging safety profile and early signs of anti-tumor activity in testicular cancer patients
- BNT211 combines two innovative approaches in one regimen, an autologous chimeric antigen receptor (CAR) T cell therapy targeting the oncofetal antigen Claudin-6 (CLDN6) and a CLDN6-encoding CAR-T cell amplifying RNA vaccine (CARVac) to improve persistence and functionality of the adoptively transferred cells
- The European Medicines Agency’s priority medicines status is granted to drug candidates that may offer a major therapeutic advantage over existing treatments, or benefit patients without treatment options
MAINZ, Germany, June 23, 2022 (GLOBE NEWSWIRE) – BioNTech SE (Nasdaq: BNTX, “BioNTech” or “the Company”) today announced that that the European Medicines Agency (EMA) has granted Priority Medicines (PRIME) designation to BioNTech’s fully owned product candidate BNT211 for the third- or later-line treatment of testicular germ cell tumors. BNT211 is a potential first-in-class therapeutic approach which comprises a synergistic combination of two of the Company’s proprietary drug products, an autologous chimeric antigen receptor (CAR) T cell therapy targeting the oncofetal antigen Claudin-6 (CLDN6) and a CLDN6-encoding CAR-T cell amplifying RNA vaccine (CARVac). The product candidate is currently being investigated in an ongoing Phase 1/2 study (NCT04503278; 2019-004323-20) that aims to evaluate the safety and preliminary efficacy in heavily pretreated patients with relapsed or refractory advanced solid tumors.
“Patients with relapsed or treatment refractory testicular cancers have a poor prognosis with limited remaining treatment options. The PRIME designation underlines the potential of our approach in this high medical need setting. Our approach brings together several new elements: Firstly, targeting CLDN6, a pan-cancer cell surface marker, secondly, our CAR design, and thirdly, “remote control” of CAR T cells by our uridine RNA-lipoplex based vaccine. We believe that a combination of engineered T cells and mRNA vaccines in one treatment regimen can stimulate and expand T cells. This could enable us to develop truly powerful precision immunotherapies,” said Prof. Özlem Türeci, M.D., Co-Founder and Chief Medical Officer at BioNTech. “With the PRIME status and support by the EMA, we aim to expedite the further development of the BNT211 program to bring a novel therapeutic option for patients with life-threatening testicular cancer, and thus to extend the successes of CAR-T therapy also to hard-to-treat solid tumors.”
The designation is based on positive preliminary Phase 1/2 data from the ongoing study that was presented at the AACR Annual Meeting in April 2022. The results demonstrated that treatment with CLDN6 CAR-T alone or in combination with CARVac was well tolerated and showed encouraging signs of anti-tumor activity in testicular cancer patients at the first evaluated dose levels. In the study all six patients with heavily pretreated testicular cancer eligible for efficacy analysis showed clinical benefits highlighting the potential of this novel approach. One patient achieved a complete response 18 weeks after infusion. Three patients achieved a partial response and showed deepening and durability of responses (one of them in the lowest CAR-T dose level cohort in combination with CARVac). One patient had stable disease with shrinkage of target lesions.
The PRIME scheme is a regulatory mechanism introduced by the EMA that provides early and proactive support to developers of promising medicines, to optimize development plans and speed up evaluations so these medicines can reach patients faster. The goal is to help patients benefit as early as possible from innovative new therapies that have demonstrated the potential to significantly address an unmet medical need.
About BNT211
Aiming to harness the power of cell therapies for solid cancers and to overcoming hurdles to date, BioNTech has combined their CAR-T and FixVac platform technologies to develop a highly tumor-specific CAR-T cell therapy product which is consecutively enhanced by a CAR-T Cell Amplifying RNA Vaccine (CARVac) that is based on BioNTech`s mRNA-lipoplex technology and encodes for the respective CAR-T target antigen. The CARVac is based on BioNTech’s backbone-optimized uridine mRNA (uRNA)-lipoplex technology which through its inherent adjuvant function enables a potent T cell stimulation to improve persistence and functionality of the adoptively transferred CAR-T cells, thus enabling and maintaining a therapeutic effect even at low CAR-T doses. BNT211 is an investigational CAR-T cell therapy directed against the novel oncofetal antigen Claudin-6 (CLDN6), a target discovered by BioNTech founders and expressed on multiple solid tumors such as ovarian cancer, sarcoma, testicular cancer, endometrial cancer and gastric cancer. The program is currently being evaluated in a first-in-human Phase 1/2 trial as a monotherapy and in combination with a CLDN6-encoding CARVac, aiming to boost persistence and functionality of the CLDN6-CAR-T cells, in patients with CLDN6-positive relapsed or refractory advanced solid tumors.
About BioNTech
Biopharmaceutical New Technologies (BioNTech) is a next generation immunotherapy company pioneering novel therapies for cancer and other serious diseases. The Company exploits a wide array of computational discovery and therapeutic drug platforms for the rapid development of novel biopharmaceuticals. Its broad portfolio of oncology product candidates includes individualized and off-the-shelf mRNA-based therapies, innovative chimeric antigen receptor T cells, bispecific checkpoint immuno-modulators, targeted cancer antibodies and small molecules. Based on its deep expertise in mRNA vaccine development and in-house manufacturing capabilities, BioNTech and its collaborators are developing multiple mRNA vaccine candidates for a range of infectious diseases alongside its diverse oncology pipeline. BioNTech has established a broad set of relationships with multiple global pharmaceutical collaborators, including Genmab, Sanofi, Genentech, a member of the Roche Group, Regeneron, Genevant, Fosun Pharma and Pfizer.
For more information, please visit www.BioNTech.com
BioNTech testicular cancer candidate receives priority designation from EMA
Jun. 23, 2022 11:23 AM ET
By: Jonathan Block, SA News Editor1 Comment
- The European Medicines Agency has awarded priority medicines designation (PRIME) to BioNTech's (NASDAQ:BNTX) CAR-T therapy BNT211 for testicular cancer.
- https://seekingalpha.com/news/3851282-biontech-testicular-cancer-candidate-receives-priority-designation-from-ema
- https://seekingalpha.com/symbol/BNTX
- https://www.biontech.com/int/en/home.html
- https://www.nasdaq.com/market-activity/stocks/bntx/dividend-history
Eplontersen, formerly known as IONIS-TTR-LRx and AKCEA-TTR-LRx
Eplontersen, formerly known as IONIS-TTR-LRx and AKCEA-TTR-LRx
Eplontersen, formerly known as IONIS-TTR-LRx and AKCEA-TTR-LRx
Ionis announces eplontersen met co-primary and secondary endpoints in interim analysis of the Phase 3 NEURO-TTRansform study for hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN)
June 21, 2022 at 2:00 AM EDT
- Eplontersen showed statistically significant and clinically meaningful improvements in mNIS+7 and Norfolk QoL-DN
- Eplontersen demonstrated a favorable safety profile
- Ionis and AstraZeneca expect to file a New Drug Application this year
CARLSBAD, Calif., June 21, 2022 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced positive topline results from a 35-week interim analysis of the Phase 3 NEURO-TTRansform study of Ionis and AstraZeneca's eplontersen in patients with hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN). Eplontersen demonstrated a statistically significant and clinically meaningful change from baseline for the co-primary endpoints of percent change in serum transthyretin (TTR) concentration and the modified Neuropathy Impairment Score +7 (mNIS+7), a measure of neuropathic disease progression, versus the historical placebo group. Eplontersen also met its key secondary endpoint of change from baseline in the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QoL-DN), showing treatment with eplontersen significantly improved patient-reported quality of life versus the historical placebo group.
In the study, eplontersen demonstrated a favorable safety and tolerability profile with no specific concerns. The study data are consistent with the clinical profile seen across Ionis' other LICA programs, further validating how advancements in the company's LIgand-Conjugated Antisense technology position Ionis to deliver potentially transformative treatments for a range of unmet medical needs.
Based on these study results, the companies will seek regulatory approval for eplontersen for ATTRv-PN and plan to file a new drug application with the U.S. Food and Drug Administration this year.
"These encouraging data reinforce the safety profile of eplontersen and demonstrate clear evidence of its potential to provide much needed therapeutic benefit to patients living with hereditary transthyretin-mediated amyloid polyneuropathy," said Teresa Coelho, M.D., a neurologist and neurophysiologist at Hospital Santo António, Centro Hospitalar Universitário do Porto, Portugal and an investigator for the NEURO-TTRansform study.
"We are pleased that the data in the NEURO-TTRansform study demonstrate eplontersen had a positive impact on disease progression, including improvement in neuropathy impairment and quality of life in a substantial number of patients. These highly statistically significant and clinically meaningful results put us on the cusp of providing a new therapeutic option for polyneuropathy patients living with this debilitating and fatal disease," said Eugene Schneider, M.D., Ionis' executive vice president and chief clinical development officer. "We are grateful to the patients, families and clinicians who are participating in NEURO-TTRansform. Without their commitment the eplontersen program would not have progressed as successfully as it has."
The data from the 35-week interim analysis will be presented at an upcoming medical meeting later this year.
As part of a global development and commercialization agreement between Ionis and AstraZeneca, eplontersen is being jointly developed and commercialized by both companies in the U.S. and will be developed and commercialized in the rest of the world by AstraZeneca (with the exception of Latin America).
Eplontersen was granted Orphan Drug Designation in the U.S. It is also currently being evaluated in the Phase 3 CARDIO-TTRansform study for amyloid transthyretin cardiomyopathy (ATTR-CM), a systemic, progressive and fatal condition that leads to progressive heart failure and death within four years from diagnosis.
For more information on the NEURO-TTRansform study, please visit: https://clinicaltrials.gov/ct2/show/NCT04136184.
About the NEURO-TTRansform Study
NEURO-TTRansform is a global, open-label, randomized study evaluating the efficacy and safety of eplontersen in patients with hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN). The study has enrolled adult patients with stage 1 or stage 2 polyneuropathy and will compare efficacy of eplontersen to the historical placebo arm from the TEGSEDI® (inotersen) NEURO-TTR registrational study that Ionis completed in 2017. The final primary endpoint analysis will be completed at week 66 and all patients will be followed until week 85 when they will have the option to transition into the open label extension study.
The co-primary efficacy endpoints at week 66 are:
- Percent change from baseline in serum TTR concentration
- Change from baseline in the modified Neuropathy Impairment Score +7 (mNIS+7), a measure of neuropathic disease progression
- Change from baseline in the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QoL-DN)
About Eplontersen
Eplontersen is an investigational antisense medicine that uses Ionis' advanced LIgand-Conjugated Antisense, or LICA, technology designed to inhibit the production of the transthyretin (TTR) protein at its source. Eplontersen, which is planned to be delivered to patients via an autoinjector, is in development to treat all types of ATTR, a systemic, progressive and fatal disease.
About Ionis Pharmaceuticals, Inc.
For more than 30 years, Ionis has been the leader in RNA-targeted therapy, pioneering new markets and changing standards of care with its novel antisense technology. Ionis currently has three marketed medicines and a premier late-stage pipeline highlighted by industry-leading cardiovascular and neurological franchises. Our scientific innovation began and continues with the knowledge that sick people depend on us, which fuels our vision of becoming a leading, fully integrated biotechnology company.
To learn more about Ionis, visit www.ionispharma.com and follow us on Twitter @ionispharma.
View original content to download multimedia:https://www.prnewswire.com/news-releases/ionis-announces-eplontersen-met-co-primary-and-secondary-endpoints-in-interim-analysis-of-the-phase-3-neuro-ttransform-study-for-hereditary-transthyretin-mediated-amyloid-polyneuropathy-attrv-pn-301571475.html
SOURCE Ionis Pharmaceuticals, Inc.
AstraZeneca/Ionis rare disease drug shows positive data in late stage study
Jun. 21, 2022 5:01 AM ET
Ionis Pharmaceuticals, Inc. (IONS), AZN
By: Ravikash, SA News Editor4 Comments
AstraZeneca (NASDAQ:AZN) and Ionis Pharmaceuticals (NASDAQ:IONS) said they would seek regulatory approval for eplontersen in the U.S. after the drug showed clinically meaningful changes on the co-primary goals of a phase 3 trial in patients with hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN).
https://seekingalpha.com/symbol/IONS
https://seekingalpha.com/symbol/AZN
https://www.ionispharma.com/medicines/akcea-ttr-l/
elivaldogene autotemcel (eli-cel)
Eplontersen, formerly known as IONIS-TTR-LRx and AKCEA-TTR-LRx
elivaldogene autotemcel (eli-cel)
FDA Advisory Committee Unanimously Endorses eli-cel Gene Therapy for Cerebral Adrenoleukodystrophy
If approved, eli-cel will be the first and only gene therapy for the treatment of early active CALD, a rare neurodegenerative disease that primarily affects young children and leads to irreversible loss of neurologic function and death
PDUFA goal date is set for September 16, 2022
SOMERVILLE, Mass.--(BUSINESS WIRE)--Jun. 9, 2022--
bluebird bio, Inc. (Nasdaq: BLUE) today announced the outcome of the U.S. Food and Drug Administration’s (FDA) Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC) discussion of elivaldogene autotemcel (eli-cel) for the treatment of early active cerebral adrenoleukodystrophy (CALD) in patients less than 18 years of age who do not have an available and willing human leukocyte antigen (HLA)-matched sibling hematopoietic stem cell (HSC) donor.
On the question “Do the benefits of eli-cel outweigh the risks, for the treatment of any sub-population of children with early active cerebral adrenoleukodystrophy (CALD)?” the CTGTAC voted 15 (yes) to 0 (no).
“For decades, the CALD community has fought for the opportunity to stave off the rapid, irreversible decline associated with this devastating disease,” said Andrew Obenshain, chief executive officer, bluebird bio. “Today we are one step closer to delivering a potentially lifesaving therapy for CALD. We are grateful to the families, clinicians and committee members who participated in today’s advisory committee discussion and remain committed to working with the FDA as it completes its review of the eli-cel Biologics License Application.”
CALD is a rare, progressive, neurodegenerative disease that primarily affects young boys and causes behavioral, cognitive, and neurological deficits. Nearly half of patients who do not receive treatment die within five years of symptom onset. Allogeneic hematopoietic stem cell transplant (allo-HSCT) is currently the only effective treatment option but is associated with serious potential complications and mortality that increase in patients without a matched sibling donor. If approved, eli-cel will be the first approved gene therapy to address the underlying genetic cause of disease for patients living with CALD in the U.S. – offering the more than 70% of patients diagnosed with CALD who do not have a matched sibling donor an alternative to allo-HSCT.
The committee’s recommendation is based on the Biologics License Application (BLA) currently under priority review by the FDA with a PDUFA goal date set for September 16, 2022. The BLA for eli-cel is supported by efficacy and safety data from the completed Phase 2/3 Starbeam study (ALD-102) (N=32). Additionally, the BLA contains data for 35 subjects dosed in the Phase 3 ALD-104 study. In clinical studies, patients treated with eli-cel were more likely to achieve both overall and event-free survival than allo-HSCT patients without a matched sibling donor, with the clearest benefit for patients without a matched donor of any type.
The eli-cel clinical program was placed on a clinical hold following a Suspected Unexpected Serious Adverse Reaction (SUSAR) of myelodysplastic syndrome (MDS) in August 2021. Consistent with this known risk, two additional cases of MDS have subsequently been reported. All patients who received eli-cel in the clinical program continue to be closely monitored, per study protocols.
The CTGTAC also discussed the overall safety of lentiviral vector (LVV) gene therapies, concluding in a 13 to 1 vote that the safety data from lovo-cel for sickle cell disease is not relevant to the review of eli-cel. In addition to granting eli-cel BLA priority review, the FDA previously granted eli-cel Orphan Drug status, Rare Pediatric Disease designation, and Breakthrough Therapy designation. bluebird bio is eligible to receive a priority review voucher upon potential approval of eli-cel.
Tomorrow, June 10, 2022, the CTGTAC will convene to discuss the efficacy and safety of betibeglogene autotemcel (beti-cel), an investigational LVV gene therapy for patients with beta-thalassemia who require regular red blood cell transfusions.
About elivaldogene autotemcel (eli-cel) gene therapy
eli-cel (pronounced ELL ee cell) uses ex vivo transduction with the Lenti-D lentiviral vector (LVV) to add functional copies of the ABCD1 gene into a patient’s own hematopoietic stem cells (HSCs). The addition of the functional ABCD1 gene allows patients to produce the ALD protein (ALDP), which is thought to facilitate the breakdown of very long-chain fatty acids (VLCFAs). The expression of ALDP and effect of eli-cel is expected to be life-long. The goal of treatment with eli-cel is to stop the progression of CALD and, consequently, preserve as much neurological function as possible, including the preservation of motor function and communication ability. Importantly, with eli-cel, there is no need for donor HSCs from another person.
bluebird bio’s clinical development program for eli-cel includes the completed pivotal Phase 2/3 Starbeam study (ALD-102) and the ongoing Phase 3 ALD-104 study, which has completed enrollment and treatment of all patients. Additionally, bluebird bio is conducting a long-term safety and efficacy follow-up study (LTF-304) for patients who have received eli-cel for CALD and completed two years of follow-up in ALD-102 or ALD-104. Enrollment into studies of eli-cel is currently on hold with the FDA and follow-up of all patients continues, per protocol.
In ALD-102, 90.6% (29/32) of patients met the primary endpoint of Major Functional Disabilities (MFD)-free survival at 24 months. As previously reported, two patients withdrew from study ALD-102 at investigator discretion, and one additional subject experienced rapid disease progression early after treatment, resulting in MFDs and subsequent death. All patients who completed ALD-102 enrolled in a long-term follow-up study (LTF-304). The median duration of follow-up is approximately four years (49 months; 13.4, 88.1).
Adverse reactions attributed to eli-cel observed in clinical trials include myelodysplastic syndrome, viral cystitis, pancytopenia, and nausea and vomiting. There have been no reports of graft-versus-host-disease, graft failure or rejection, transplant-related mortality, or replication competent lentivirus in the 67 patients who received eli-cel in clinical studies (ALD-102, ALD-104, LTF-304).
About bluebird bio, Inc.
bluebird bio is pursuing curative gene therapies to give patients and their families more bluebird days.
With a dedicated focus on severe genetic diseases, bluebird has industry-leading clinical and research programs for sickle cell disease, beta-thalassemia and cerebral adrenoleukodystrophy and is advancing research to apply new technologies to these and other diseases. We custom design each of our therapies to address the underlying cause of disease and have developed in-depth and effective analytical methods to understand the safety of our lentiviral vector technologies and drive the field of gene therapy forward.
Founded in 2010, bluebird has the largest and deepest ex-vivo gene therapy data set in the world—setting the standard for industry. Today, bluebird continues to forge new paths, combining our real-world experience with a deep commitment to patient communities and a people-centric culture that attracts and grows a diverse flock of dedicated birds.
For more information, visit bluebirdbio.com or follow us on social media at @bluebirdbio, LinkedIn, Instagram and YouTube.
Lenti-D and bluebird bio are trademarks of bluebird bio, Inc.
View source version on businesswire.com: https://www.businesswire.com/news/home/20220609006060/en/
Source: bluebird bio, Inc.
Bluebird bio neuro disorder gene therapy gets FDA panel nod, awaits meeting on another
Jun. 10, 2022 5:35 AM ET
By: Ravikash, SA News Editor1 Comment
Bluebird bio (NASDAQ:BLUE) said a panel of the U.S. Food and Drug Administration (FDA) voted in favor of its gene therapy candidate elivaldogene autotemcel (eli-cel) for cerebral adrenoleukodystrophy (CALD).
The company's stock had been halted for trading by Nasdaq on June 9 for 2 days (including today) ahead of the FDA committee meeting.
Inaxaplin (VX-147)
Eplontersen, formerly known as IONIS-TTR-LRx and AKCEA-TTR-LRx
elivaldogene autotemcel (eli-cel)
BackPDF VersionJun 8, 2022
Vertex Announces Inaxaplin (VX-147) Granted Breakthrough Therapy Designation by U.S. FDA and Priority Medicines (PRIME) Designation by the EMA
- Vertex granted nine Breakthrough Therapy Designations and three PRIME designations across its pipeline programs to date –
BOSTON--(BUSINESS WIRE)--Jun. 8, 2022-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the U.S. Food and Drug Administration (FDA) has granted inaxaplin (VX-147) Breakthrough Therapy Designation for APOL1-mediated focal segmental glomerulosclerosis (FSGS) and the European Medicines Agency (EMA) has granted inaxaplin Priority Medicines (PRIME) designation for APOL1-mediated chronic kidney disease (AMKD). Inaxaplin is the first investigational therapy aimed at treating the underlying cause of AMKD.
The FDA's Breakthrough Therapy Designation is intended to expedite development and review of medicines that aim to address a serious condition with preliminary clinical evidence indicating that the drug may demonstrate substantial improvement over existing treatments on one or more clinically significant endpoints. The Breakthrough Therapy Designation was granted based on the Phase 2 clinical study of inaxaplin in patients with APOL1-mediated FSGS, a form of AMKD.
The EMA’s PRIME designation is a regulatory mechanism that provides early and proactive support to developers of promising medicines, to optimize the generation of robust data and enable accelerated assessment so these medicines can potentially reach patients faster. The goal of PRIME is to help patients benefit as early as possible from innovative new therapies that have the potential to significantly address an unmet medical need. PRIME designation was granted based on clinical proof-of-concept data from Vertex’s Phase 2 study of inaxaplin in APOL1-mediated FSGS. Inaxaplin is only the second nephrology product to be granted PRIME designation.
Vertex now holds three of the approximately 70 non-oncological PRIME designations granted to date, including its two PRIME designations for exagamglogene autotemcel (exa-cel), formerly known as CTX001, one for transfusion-dependent beta thalassemia and one for sickle cell disease. In the US, this is the ninth breakthrough therapy designation granted to Vertex across its portfolio programs.
About the Inaxaplin (VX-147) Pivotal Program
A randomized, double-blind, placebo-controlled Phase 2/3 adaptive study is ongoing and will first evaluate two doses of inaxaplin for 12 weeks to select a dose for Phase 3 and subsequently evaluate the efficacy and safety of the single, selected dose in the Phase 3 portion of the study.
Patients aged 18 to 60 years, with two APOL1 mutations, urine protein to creatinine ratio (UPCR) ≥0.7 g/g to <10 g/g, estimated glomerular filtration rate (eGFR) ≥25 to <75 mL/min/1.73m2 and on stable doses of standard of care medications are eligible to enroll. Approximately 66 patients are planned to be enrolled in the Phase 2 dose-ranging portion of the study, and approximately 400 additional patients are planned to be enrolled in the Phase 3 portion of the study.
The primary efficacy endpoint for the final analysis is eGFR slope in patients receiving the inaxaplin selected dose compared to placebo. The secondary efficacy endpoint is time to composite clinical outcome, which will also be assessed at the final analysis and is defined as a sustained decline of ≥30% from baseline in eGFR, the onset of end-stage kidney disease (i.e., maintenance dialysis for ≥28 days, kidney transplantation, or a sustained eGFR of <15 mL/min/1.73 m2), or death. The final study analysis will occur when subjects have at least two years of eGFR data and when approximately 187 composite clinical outcomes have occurred.
The study is also designed to have a pre-planned interim analysis at Week 48 evaluating eGFR slope, supported by a percent change from baseline in UPCR in the inaxaplin arm versus placebo. If positive, the interim analysis may serve as the basis for Vertex to seek accelerated approval of inaxaplin in the U.S. for patients with AMKD.
About Vertex
Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has multiple approved medicines that treat the underlying cause of cystic fibrosis (CF) — a rare, life-threatening genetic disease — and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust pipeline of investigational small molecule, cell and genetic therapies in other serious diseases where it has deep insight into causal human biology, including sickle cell disease, beta thalassemia, APOL1-mediated kidney disease, pain, type 1 diabetes, alpha-1 antitrypsin deficiency and Duchenne muscular dystrophy.
Founded in 1989 in Cambridge, Mass., Vertex's global headquarters is now located in Boston's Innovation District and its international headquarters is in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the industry's top places to work, including 12 consecutive years on Science magazine's Top Employers list and one of the 2021 Seramount (formerly Working Mother Media) 100 Best Companies. For company updates and to learn more about Vertex's history of innovation, visit www.vrtx.com or follow us on Facebook, Twitter, LinkedIn, YouTube and Instagram.
View source version on businesswire.com: https://www.businesswire.com/news/home/20220608005181/en/
Source: Vertex Pharmaceuticals Incorporated
Vertex wins key FDA and EU designations for kidney disease drug
Jun. 08, 2022 8:26 AM ET
Vertex Pharmaceuticals Incorporated (VRTX)
By: Dulan Lokuwithana, SA News Editor2 Comments
- Vertex Pharmaceuticals (NASDAQ:VRTX) announced on Wednesday that U.S. and European regulators issued key designations for its kidney disease candidate inaxaplin (VX-147) that would enable the company to speed up its regulatory work.
- https://seekingalpha.com/news/3846657-vrtx-stock-gains-on-fda-and-eu-designations-for-kidney-disease-drug
- https://seekingalpha.com/symbol/VRTX
BCX9250
Rylaze® (asparaginase erwinia chrysanthemi (recombinant)-rywn)
Rylaze® (asparaginase erwinia chrysanthemi (recombinant)-rywn)
FDA Grants Fast Track Designation for BioCryst’s ALK-2 Inhibitor, BCX9250
RESEARCH TRIANGLE PARK, N.C., June 08, 2022 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for BCX9250 for the prevention of heterotopic ossification (HO) in patients with fibrodysplasia ossificans progressiva (FOP).
According to the FDA, the purpose of the Fast Track designation is to get important new drugs to patients earlier by facilitating the development, and expediting the review, of drugs to treat serious conditions and fill an unmet medical need. Companies that receive this designation are eligible for benefits including more frequent meetings with and written communication from the FDA, eligibility for accelerated approval and priority review, if relevant criteria are met, and rolling review of the new drug application (NDA).
“We are pleased with the FDA’s decision to grant Fast Track designation to BCX9250, as there is a significant unmet need among patients living with FOP. With this designation following the EMA’s recent decision to grant PRIME eligibility for BCX9250 in Europe, we believe the non-clinical data and the first-in-human Phase 1 safety, tolerability and pharmacokinetics study in healthy subjects support the potential of our ALK-2 inhibitor program to be a meaningful therapeutic advance for the FOP community,” said Dr. Helen Thackray, chief research and development officer of BioCryst.
FOP is an ultra-rare, severely disabling genetic disorder characterized by HO, or the irregular formation of bone outside the normal skeleton. HO can occur in muscles, tendons, ligaments and other connective tissues. Patients with FOP become bound by this irregular ossification over time, with restricted movement and fused joints, resulting in deformities, restricted mobility and premature mortality.
BCX9250 is designed to inhibit the ALK-2 enzyme, which is a part of the normal signaling pathway for bone formation and responds to binding its specific ligands (bone morphogenic proteins, BMPs) by stimulating normal bone growth and renewal in healthy children and adults. Specific activating mutations of the ALK-2 gene are seen in all cases of FOP. An activating mutation in ALK-2 is necessary for the disease to occur, making the ALK-2 enzyme an ideal drug target for treatment of FOP.
In a Phase 1 clinical trial in healthy subjects, BCX9250 was safe and well tolerated at all doses studied, with linear and dose-proportional exposure supporting the potential for once-daily dosing.
About BioCryst Pharmaceuticals
BioCryst Pharmaceuticals discovers novel, oral, small-molecule medicines that treat rare diseases in which significant unmet medical needs exist and an enzyme plays a key role in the biological pathway of the disease. Oral, once-daily ORLADEYO® (berotralstat) is approved in the United States and multiple global markets. BioCryst has several ongoing development programs including BCX9930, an oral Factor D inhibitor for the treatment of complement-mediated diseases, BCX9250, an ALK-2 inhibitor for the treatment of fibrodysplasia ossificans progressiva, and galidesivir, a potential treatment for Marburg virus disease and yellow fever. RAPIVAB® (peramivir injection) is approved in the U.S. and multiple global markets, with post-marketing commitments ongoing. For more information, please visit the company’s website at www.biocryst.com.
BioCryst nabs FDA’s Fast Track status for candidate targeting rare bone forming disorder
Jun. 08, 2022 10:01 AM ET
BioCryst Pharmaceuticals, Inc. (BCRX)
By: Dulan Lokuwithana, SA News Editor
- Commercial-stage biotech BioCryst Pharmaceuticals (NASDAQ:BCRX) announced on Wednesday that the FDA granted its Fast Track designation for BCX9250 for the prevention of heterotopic ossification (HO) in ultra-rare genetic disorder fibrodysplasia ossificans progressiva (FOP).
- https://seekingalpha.com/news/3846729-bcrx-stock-gains-on-fda-fast-track-status-for-drug-for-bone-forming-disorder
- https://seekingalpha.com/symbol/BCRX
- https://www.biocryst.com/our-pipeline/
Rylaze® (asparaginase erwinia chrysanthemi (recombinant)-rywn)
Rylaze® (asparaginase erwinia chrysanthemi (recombinant)-rywn)
Rylaze® (asparaginase erwinia chrysanthemi (recombinant)-rywn)
Jazz Pharmaceuticals Presents Positive Data from Phase 2/3 Trial of Rylaze® (asparaginase erwinia chrysanthemi (recombinant)-rywn) in Acute Lymphoblastic Leukemia or Lymphoblastic Lymphoma at the ASCO 2022 Annual Meeting
June 07, 2022
Oral presentation confirms patients achieved clinically meaningful nadir serum asparaginase activity throughout the course of Rylaze treatment with intramuscular dosing regimen administered 25/25/50 mg/m2 on Monday/Wednesday/Friday
DUBLIN, June 7, 2022 /PRNewswire/ -- Jazz Pharmaceuticals plc (Nasdaq: JAZZ) today announced positive results from a Phase 2/3 trial, developed and conducted in close collaboration with the Children's Oncology Group (COG), evaluating the intramuscular (IM) administration of Rylaze® (asparaginase erwinia chrysanthemi (recombinant)-rywn) in adult and pediatric patients with acute lymphoblastic leukemia (ALL) and lymphoblastic lymphoma (LBL) who have developed hypersensitivity to an E. coli-derived asparaginase. These results will be presented as an oral presentation today at the American Society of Clinical Oncology (ASCO) 2022 Annual Meeting.
These results confirm the interim trial analysis presented in December 2021 at the 63rd American Society of Hematology (ASH) Annual Meeting, and demonstrated that ≥90% of patients in Cohort 1c receiving the IM dosing regimen of 25/25/50 mg/m2 administered Monday/Wednesday/Friday (MWF) achieved nadir serum asparaginase activity (NSAA) levels ≥0.1 IU/mL at 48 and 72 hours, with a safety profile consistent with what has been described for other asparaginases.
"We are excited to share these results from the Phase 2/3 trial of Rylaze highlighting the clinically meaningful nadir serum asparaginase activity from the Monday/Wednesday/Friday dosing regimen, which supports a new dosing schedule that aligns with current clinical practice," said Rob Iannone, M.D., M.S.C.E., executive vice president, global head of research and development of Jazz Pharmaceuticals. "Rylaze is an example of how Jazz and the Children's Oncology Group have advanced a critically needed treatment from development through FDA approval, and then continue to explore additional dosing and administration options to address the needs of patients."
"Asparaginase-based therapies remain a cornerstone in ALL and LBL treatment, and Rylaze has been an important option for patients who develop a hypersensitivity to an E. coli-derived asparaginase since its approval last year," said trial primary investigator Dr. Luke Maese, associate professor of pediatric hematology-oncology at the University of Utah, Primary Children's Hospital and Huntsman Cancer Institute. "It's encouraging to see these data further support an IM Monday/Wednesday/Friday dosing schedule for Rylaze, which is more in line with clinical practice in the U.S., in addition to the currently approved schedule of every 48 hours."
Rylaze was approved in the U.S. in 2021 for use as a component of a multi-agent chemotherapeutic regimen for the treatment of ALL and LBL in adult and pediatric patients one month or older who have developed hypersensitivity to E. coli-derived asparaginase. It was approved with an IM dosing schedule of 25 mg/m2 every 48 hours based on observed data from the Phase 2/3 trial, in conjunction with data produced by a population pharmacokinetic (PPK) model. Data from the Phase 2/3 trial supported additional regulatory filings for Rylaze, including two supplemental Biologics Licensing Applications (sBLA) to the U.S. FDA to support MWF IM dosing completed in January 2022 and IV administration options completed in April 2022.
Trial Results
Data presented at ASCO include efficacy and safety results from the Phase 2/3 open-label, multicenter, pharmacokenitic (PK) trial of Rylaze (also known as JZP458) in patients with ALL/LBL who developed hypersensitivity or silent inactivation to a long-acting E. coli-derived asparaginase. These results are from Part A of the trial, which investigated three cohorts via IM administration:
- Cohort 1a (n=33): studied a dose of 25 mg/m2 Monday/Wednesday/Friday
- Cohort 1b (n=83): studied a dose of 37.5 mg/m2 Monday/Wednesday/Friday
- Cohort 1c (n=51): studied a dose of 25 mg/m2 on Monday and Wednesday and 50 mg/m2 on Friday
Efficacy Findings
The primary efficacy endpoint of the trial was the proportion of patients with NSAA levels ≥0.1 IU/mL at the last 72-hours during the first treatment course.
The key secondary endpoint was the proportion of patients with NSAA levels ≥0.1 IU/mL at the last 48-hours during the first treatment course.
A population PK (PPK) model was developed based on SAA data from the clinical trial to characterize the PK of JZP458 when given IM and to inform dosing decisions. Simulated data from the PPK model matched the observed data well.
Based on a PPK modeling and simulation analysis, the proportion of patients predicted to achieve NSAA levels ≥0.1 IU/mL with a 95% CI in Cohort 1c at the last 72- and 48-hour in Course 1 were 92% (91%, 93%) and 94% (93%, 95%) respectively, based on modeled data.
About Rylaze® (asparaginase erwinia chrysanthemi (recombinant)-rywn)
Rylaze, also known as JZP458, is approved in the U.S. for use as a component of a multi-agent chemotherapeutic regimen for the treatment of acute lymphoblastic leukemia (ALL) and lymphoblastic lymphoma (LBL) in adult and pediatric patients one month or older who have developed hypersensitivity to E. coli-derived asparaginase. Rylaze has orphan drug designation for the treatment of ALL/LBL in the United States. Rylaze is a recombinant erwinia asparaginase that uses a novel Pseudomonas fluorescens expression platform. JZP458 was granted Fast Track designation by the U.S. Food and Drug Administration (FDA) in October 2019 for the treatment of this patient population. Rylaze was approved as part of the Real-Time Oncology Review (RTOR) program, an initiative of the FDA's Oncology Center of Excellence designed for efficient delivery of safe and effective cancer treatments to patients.
The full U.S. Prescribing Information for Rylaze is available at: <https://pp.jazzpharma.com/pi/rylaze.en.USPI.pdf>
About Jazz Pharmaceuticals plc
Jazz Pharmaceuticals plc (Nasdaq: JAZZ) is a global biopharmaceutical company whose purpose is to innovate to transform the lives of patients and their families. We are dedicated to developing life-changing medicines for people with serious diseases –often with limited or no therapeutic options. We have a diverse portfolio of marketed medicines and novel product candidates, from early-to late-stage development, in neuroscience and oncology. Within these therapeutic areas, we are identifying new options for patients by actively exploring small molecules and biologics, and through innovative delivery technologies and cannabinoid science. Jazz is headquartered in Dublin, Ireland and has employees around the globe, serving patients in nearly 75 countries. For more information, please visit www.jazzpharmaceuticals.com and follow @JazzPharma on Twitter.
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SOURCE Jazz Pharmaceuticals plc
Jazz's new dosing regimen of Rylaze as part of cancer chemotherapy shows promise in trial
Jun. 07, 2022 8:49 AM ET
Jazz Pharmaceuticals plc (JAZZ)
By: Ravikash, SA News Editor
Jazz Pharmaceuticals plc (NASDAQ:JAZZ) reported data from a phase 2/3 trial of a new dosing regimen of its therapy Rylaze (asparaginase erwinia chrysanthemi (recombinant)-rywn).
The results — which were presented at the American Society of Clinical Oncology (ASCO) meeting — were from Part A of the study, which included three groups.
https://seekingalpha.com/symbol/JAZZ
SEASONAL INFLUENZA VACCINE CANDIDATE (MRNA-1010)
Rylaze® (asparaginase erwinia chrysanthemi (recombinant)-rywn)
SEASONAL INFLUENZA VACCINE CANDIDATE (MRNA-1010)
MODERNA ANNOUNCES FIRST PARTICIPANTS DOSED IN PHASE 3 STUDY OF SEASONAL INFLUENZA VACCINE CANDIDATE (MRNA-1010)
mRNA-1010 is Moderna's first seasonal influenza vaccine candidate to enter a Phase 3 trial
mRNA-1010 is one of several influenza vaccine candidates being developed in Moderna's respiratory portfolio
Moderna now has four programs in Phase 3 studies: Omicron-containing bivalent COVID booster, influenza, RSV, CMV
CAMBRIDGE, MA / ACCESSWIRE / June 7, 2022 / Moderna, Inc. (NASDAQ:MRNA), a biotechnology company pioneering messenger RNA (mRNA) therapeutics and vaccines, today announced the first participants have been dosed in a Phase 3 study of the Company's seasonal influenza vaccine candidate (mRNA-1010). The trial is expected to enroll approximately 6,000 adults in Southern Hemisphere countries.
"We are pleased to begin this Phase 3 study of our seasonal influenza vaccine candidate, mRNA-1010, our fourth mRNA vaccine candidate to begin a pivotal Phase 3 study. mRNA-1010 is the first of several influenza vaccine candidates we are developing with the aim of iteratively improving traditional vaccines by inducing broad and robust immune responses. We believe our mRNA platform, with the flexibility and speed of our manufacturing process, is well-positioned to address the significant unmet need in seasonal flu, said Stéphane Bancel, Chief Executive Officer of Moderna. "Influenza vaccines are a key pillar in our respiratory vaccine strategy that includes the development of combination candidates targeting multiple viruses in a single vaccine, including influenza with SARS-CoV-2 and respiratory syncytial virus. With the start of dosing for its mRNA-1010 program, Moderna now has four programs in late stage Phase 3 studies, including its SARS-CoV-2 booster, RSV, seasonal flu and CMV vaccine candidates. Beginning in the fall of 2022, the Company's Phase 3 pipeline could lead to three respiratory commercial launches over the next two to three years."
This Phase 3 randomized, observer-blind study is designed to evaluate the safety and immunological non-inferiority of mRNA-1010 to a licensed seasonal influenza vaccine in adults 18 years and older. Participants will be randomly assigned on a 1:1 ratio to receive either a single dose of mRNA-1010 or a single dose of a licensed seasonal influenza vaccine as a comparator.
About mRNA-1010 & Seasonal Influenza
mRNA-1010 is a vaccine candidate that encodes for hemagglutinin (HA) glycoproteins of the four influenza strains recommended by the World Health Organization (WHO) for the prevention of influenza, including influenza A/H1N1, A/H3N2, and influenza B/Yamagata- and B/Victoria-lineages. HA is a major influenza surface glycoprotein that is considered an important target to generate broad protection against influenza and is the primary target of currently available influenza vaccines. Moderna is proactively preparing for a confirmatory efficacy study for mRNA-1010 as early as the 2022/2023 Northern Hemisphere influenza season, if needed.
Influenza (influenza A and influenza B) epidemics occur seasonally and vary in severity each year, causing respiratory illnesses and placing a substantial burden on healthcare systems. Worldwide, influenza leads to 3-5 million severe cases of influenza and 290,000-650,000 influenza-related respiratory deaths annually, despite the availability of current influenza vaccines. Although both influenza A and B cause seasonal epidemics, it is the influenza A viruses that lead to >95% of influenza-related hospitalization in adults.
Moderna's Respiratory Vaccine Program
Moderna is advancing a portfolio of respiratory candidates, including five influenza mRNA vaccine candidates. In addition to mRNA-1010, Moderna is developing influenza vaccine candidates that include additional HA antigens for broader coverage of circulating influenza A strains (mRNA-1011 and mRNA-1012), and vaccine candidates that incorporate both HA and neuraminidase (NA) antigens to target multiple proteins involved in the influenza virus lifecycle to reduce the potential of viral antigenic escape (mRNA-1020 and mRNA-1030).
Moderna is also developing two combination vaccine candidates, including a vaccine candidate against influenza and SARS-CoV-2 (mRNA-1073), and another candidate against influenza, SARS-CoV-2, and RSV (mRNA-1230). The goal of Moderna's combination vaccine candidates is to provide protection against multiple respiratory pathogens in a single vaccine.
About Moderna
In over 10 years since its inception, Moderna has transformed from a research-stage company advancing programs in the field of messenger RNA (mRNA), to an enterprise with a diverse clinical portfolio of vaccines and therapeutics across seven modalities, a broad intellectual property portfolio in areas including mRNA and lipid nanoparticle formulation, and an integrated manufacturing plant that allows for rapid clinical and commercial production at scale. Moderna maintains alliances with a broad range of domestic and overseas government and commercial collaborators, which has allowed for the pursuit of both groundbreaking science and rapid scaling of manufacturing. Most recently, Moderna's capabilities have come together to allow the authorized use and approval of one of the earliest and most effective vaccines against the COVID-19 pandemic.
Moderna's mRNA platform builds on continuous advances in basic and applied mRNA science, delivery technology and manufacturing, and has allowed the development of therapeutics and vaccines for infectious diseases, immuno-oncology, rare diseases, cardiovascular diseases and auto-immune diseases. Moderna has been named a top biopharmaceutical employer by Science for the past seven years. To learn more, visit www.modernatx.com.
Moderna has been named a top biopharmaceutical employer by Science for the past seven years. To learn more, visit www.modernatx.com.
Moderna begins dosing in phase 3 trial of influenza vaccine mRNA-1010
Jun. 07, 2022 10:52 AM ET
By: Ravikash, SA News Editor5 Comments
- Moderna (NASDAQ:MRNA) said the first participants were dosed in a phase 3 trial of its seasonal influenza vaccine mRNA-1010.
- https://seekingalpha.com/news/3846333-moderna-begins-dosing-in-phase-3-trial-of-influenza-vaccine-mrna-1010
- https://seekingalpha.com/symbol/MRNA
- https://investors.modernatx.com/overview/default.aspx
- https://clinicaltrials.gov/ct2/show/NCT04956575