Week of August 24
This weekly post looks to identify important advancements in the areas of oncology, CNS disorders and neurodegeneration, technologies, and industry and corporate developments. More in-depth reading and analysis can be found in the attached links.
Oncology
FDA announced a clinical hold on Poseida Therapeutics’ CAR T-cell candidate P-PSMA-101 to treat patients with metastatic castrate resistant prostate cancer after a third patient died. Link
England’s National Institute for Health and Care Excellence (NICE) approved Roche’s Polivy in B-cell lymphoma after an initial rejection earlier this year. The decision comes after Roche provided updated survival data and offered a discount on the therapy. Link
FDA approved Amgen’s and Johnson and Johnson’s Kyprolis and Darzalex combinational therapy to treat relapsed and refractory multiple myeloma. Link
Japanese Ministry of Health, Labor and Welfare approved AstraZeneca’s Imfinzi to treat extensive-stage small cell lung cancer (ES-SCLC). Approval was based on positive Phase 3 results demonstrating Imfinzi plus chemotherapy demonstrated an improved overall survival (OS) versus chemotherapy alone. Link
Bristol Myers Squibb announced negative Phase 3 results for IDHIFA in combination with best supportive care (BSC) to treat relapsed or refractory acute myeloid leukemia with an isocitrate dehydrogenase-2 (IDH2) mutation. The combination failed meet its overall survival primary endpoint compared to best supportive care BSC only, azacitidine plus BSC, low-dose cytarabine plus BSC or intermediate-dose cytarabine plus BSC. Link
CNS Disorders and Neurodegeneration
Takeda Pharmaceutical and Ovid Therapeutics announced positive topline Phase 2 results for soticlestat to treat children with Dravet syndrome (DS) or Lennox-Gastaut syndrome (LGS). Soticlestat, a highly selective cholesterol 24-hydroxylase (CH24H) inhibitor, met its primary endpoint of reducing convulsive seizure (DS) and drop seizure (LGS) frequency compared to placebo. Link
AbbVie submitted an application to the FDA for their selective and reversible JAK inhibitor, RINVOQ, to treat active ankylosing spondylitis. Earlier this year, AbbVie submitted an application to the European Medicines Agency (EMA). Link
Technologies
Pfizer and BioNTech published positive Phase 1 results for their lead mRNA vaccine candidate BNT162B2 against COVID-19. The preprint manuscript demonstrates that 7 days after the second dose elicit a strong immunogenic response in both younger (18-55 years of age) and older adults (65-85 years of age). The vaccine was well tolerated with less than 20% of participants having a mild to moderate fever. Link
FDA announced a new primary endpoint for BioMarin Pharmaceutical’s ongoing Phase 3 hemophilia A gene therapy trial. The FDA recommends a 2-year annualized bleeding rate (ABR) from all patients enrolled in the trial. BioMarin plans to meet with the FDA to discuss next steps in order to obtain approval. Link
Poseida Therapeutics will be filing an IND in late 2020 or early 2021 for their allogeneic CAR-T candidate, P-BCMA-ALLO1, to treat relapsed/refractory multiple myeloma with genetic edits to reduce both host-vs-graft and graft-vs-host alloreactivity. Link
GSK announced the first patient dosed for their Phase 3 trial investigating its 5-in-1 meningitis vaccine (MenABCWY) compared to licensed vaccines, Bexsero and Menveo. MenABCWY hopes to immunize patients from the five serogroups of meningitis: A, B, C, W, and Y. Link
Audentes Therapeutics announced that a third patient has died in their Phase 1/2 trial for AT132 to treat X-linked myotubular myopathy. Link
Industry and Corporate Developments
Sinopharm announced on Chinese state media that its coronavirus vaccine candidate would be priced at less than 1000 yuan ($145) for two doses. It is unclear whether the price is referring to the out-of-pocket cost or list price. Link
Johnson & Johnson announced the acquisition of Momenta Pharmaceuticals for $6.5B. The key driver for this deal was Momenta Pharmaceuticals’ potentially best-in-class anti-FcRn antibody, nipocalimab. Johnson & Johnson hopes to pursue additional autoimmune indications with nipocalimab. Link
Bayer and Informed Data Systems Inc. (One Drop) announced an agreement to jointly develop digital health products to help patients manage their conditions. Bayer increased its stake in Informed Data Systems as its lead Series C financing investor. Link
Bristol Myers Squibb announced the acquisition of Forbius. Forbius has a portfolio of highly selective and potent TGF-β 1 & 3 inhibitors to treat cancer and fibrotic diseases. Link
Roche and Regeneron announced a collaboration to help develop, manufacture, and distribute Regeneron’s investigational antiviral antibody combination REGN-COV2. To met global demand, Regeneron will focus its distribution in the US while Roche will focus outside the US. Link
Teva Pharmaceutical is responding to criminal indictments of antitrust violations under the Sherman Act. Link