It was a busy week for the biotech sector with several drug approvals along with regulatory and other pipeline updates. While coronavirus-related news continues to grab headlines, other regulatory events were also in the spotlight. BioMarin BMRN plunged on the FDA issuance of a Complete Response Letter (CRL) for its hemophilia A drug. Updates from quite a few biotech biggies were also in focus.
Recap of the Week’s Most Important Stories:
Bristol-Myers Updates on Leukemia Study, To Acquire Oncology Company: Bristol Myers Squibb BMY announced that the late-stage study on Idhifa (enasidenib) did not meet the primary endpoint. The phase III IDHENTIFY study evaluating Idhifa plus best supportive care (BSC) versus conventional care regimens, which include best supportive care (BSC) only — azacitidine plus BSC, low-dose cytarabine plus BSC or intermediate-dose cytarabine plus BSC — did not meet the primary endpoint of overall survival (OS) in patients with relapsed or refractory acute myeloid leukemia (R/R AML) with an isocitrate dehydrogenase-2 (IDH2) mutation. The drug is already approved in the United States for the treatment of adult patients with R/R AML with an IDH2 mutation, as detected by an FDA-approved test.
Earlier, Bristol Myers announced that it will acquire a privately-held, clinical-stage protein engineering company, Forbius, that designs and develops biotherapeutics for the treatment of cancer and fibrotic diseases. Forbius has developed a portfolio of highly selective and potent inhibitors of TGF-beta 1 & 3, which are key mediators of immunosuppression and fibrosis. The transaction includes an upfront payment and future success-based milestone payments. The transaction is expected to close in the fourth quarter of 2020.
BioMarin Submits NDA, Plunges on CRL for Hemophilia Drug: BioMarin Pharmaceutical Inc. submitted a New Drug Application (NDA) to the FDA for pipeline candidate, vosoritide. The company is seeking approval of vosoritide, an investigational, once-daily injection, analog of C-type Natriuretic Peptide (CNP) for children with achondroplasia, the most common form of disproportionate short stature in humans.
Earlier, shares of the company plummeted significantly after it announced the receipt of a CRL from the FDA for its Biologics License Application (BLA) for valoctocogene roxaparvovec gene therapy for severe hemophilia A. The BLA application was based on interim data from an ongoing phase III study and the updated three-year results from a long-term phase I/II study.
However, the FDA was not satisfied with the available data and needs more information before it can approve the product. Per the regulatory agency, the differences between the phase I/II and the phase III analyses limited its ability to rely on the results from the phase I/II alone to support the durability of effect. According to management, the FDA introduced a recommendation in the CRL.
The agency requires BioMarin to provide substantial proof of a durable effect from the ongoing phase III study (270-301), using Annualized Bleeding Rate (ABR) as the primary endpoint. The FDA authorities asked BioMarin to complete the phase III study and submit two-year follow-up safety and efficacy data on all participants from the program.
Vertex’s Kaftrio Approved in Europe: Vertex Pharmaceuticals Incorporated VRTX announced that the European Commission has approved its triple combination cystic fibrosis (CF) regimen, Kaftrio (ivacaftor/tezacaftor/elexacaftor). The drug is now approved in combination with Kalydeco (ivacaftor) for treating CF in patients aged 12 years and above with one F508del mutation and one minimal function mutation (F/MF) or two F508del mutations (F/F) in their CFTR gene. The combination is already marketed in the United States as Trikafta, which was approved by the FDA in October 2019.
Amgen Gets FDA Nod for Kyprolis: Amgen AMGN announced that the FDA has approved the label expansion of its multiple myeloma drug, Kyprolis. The drug is now approved for use in combination with Darzalex (daratumumab) plus dexamethasone (DKd) in two dosing regimens — once weekly and twice weekly — for the treatment of patients with relapsed or refractory multiple myeloma (R/R MM) who have received one to three previous lines of therapy. The approval was based on results from the CANDOR and EQUULEUS studies in patients with relapsed/refractory multiple myeloma.
Novavax Initiates COVID-19 Vaccine Study: Novavax, Inc. NVAX announced that it has enrolled the first volunteer in the phase II portion of its ongoing phase I/II study, which is evaluating the immunogenicity and safety of its COVID-19 vaccine candidate, NVX-CoV2373. The phase II portion will include adult patients aged between 60 years and 84 years, comprising almost 50% of the current patient population in the study. NVX-CoV2373 is a stable, prefusion protein made using Novavax’s proprietary nanoparticle technology and includes its proprietary Matrix M adjuvant.
The phase II portion of the study is looking for robust immune responses in older adults and is an expansion of the phase I study following encouraging safety and immunogenicity data on NVX-CoV2373 from the same. The study will enroll up to 1,500 healthy volunteers in the United States and Australia, with approximately 50% between 60 years and 84 years of age. It will assess two dose sizes (5 and 25 µg), each with 50 µg of Matrix M.
Novavax currently carries a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
Moderna Gains on Positive Data for Coronavirus Vaccine Candidate: Shares of Moderna MRNA gained after it announced data on its experimental coronavirus vaccine mRNA-1273 from a phase I interim analysis of older adult cohorts (ages 56-70 and 71+). Data showed that at the 100 µg dose, mRNA-1273 was generally safe and well-tolerated in all age cohorts. At the 100 µg dose, mRNA-1273 induced consistently high levels of neutralizing antibody titers in all participants in the 56-70 (n=10) and 71+ age cohorts (n=10). Neutralizing antibody titers and T cell responses in these age cohorts were consistent with those reported in younger adults.
Medical - Biomedical and Genetics Industry 5YR % Return
Medical - Biomedical and Genetics Industry 5YR % Return
The Nasdaq Biotechnology index lost 0.67% in the last five trading sessions. Among the biotech giants, Amgen gained 4.03% during this period. Over the past six months, shares of Regeneron have gained 33.65%. (See the last biotech stock roundup here: Biotech Stock Roundup: GILD Gets CRL, REGN Collaborates With RHHBY & More)
What's Next in Biotech?
Stay tuned for more pipeline updates.
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