Week of October 19

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


  • Keytruda received an expanded label from the FDA to treat adult patients with relapsed or refractory classical Hodgkin lymphoma (cHL) as a monotherapy. This is the first anti-PD-1 therapy approved in this indication. Link


  • The European Medicines Agency (EMA) recommended approval of Bristol Myers Squibb’s Opdivo to treat patients with unresectable advanced, recurrent or metastatic esophageal squamous cell carcinoma (ESCC) as a 2L therapy. Link


  • Roche’s Venclexta (venetoclax) received full FDA approval to treat newly diagnosed acute myeloid leukemia (AML) in combination with azacitidine, or decitabine, or low-dose cytarabine (LDAC). Venclexta had previously received provisional approval in November 2018. Link


  • FDA granted priority review for Bristol Myers Squibb’s and Exelixis’s supplemental Biologics License Application (sBLA) and supplemental New Drug Application (sNDA) for Opdivo (nivolumab) in combination with Cabometyx (cabozantinib) to treat advanced renal cell carcinoma (RCC). Link


  • The FDA accepted and granted Priority Review for AstraZeneca’s sNDA for Tagrisso to treat patients with early-stage (IB, II and IIIA) epidermal growth factor receptor-mutated (EGFRm) NSCLC. Link


CNS Disorders and Neurodegeneration


  • The FDA placed Voyager Therapeutics’ IND on hold pending the resolution of certain chemistry, manufacturing and controls (CMC) matters. The IND is for Voyager’s experimental gene therapy to treat Huntington’s disease. Link


  • Chugai Pharmaceutical and Roche announced an NDA filing to Japan’s Ministry of Health, Labour and Welfare (MHLW) for risdiplam, an oral survival motor neuron-2 (SMN2) splicing modifier, to treat spinal muscular atrophy (SMA). Link


  • Pharmather, focused on the research and development of psychedelic pharmaceuticals, submitted an FDA application for ketamine to receive Orphan Drug Designation to treat of levodopa-induced dyskinesia associated with Parkinson’s Disease. Link


  • Novartis’s branaplam (LMI070) received FDA’s Orphan Drug Designation to treat Huntington’s disease (HD). In preclinical models, branaplam has been shown to reduce levels of the mutant huntingtin protein. Link


Technologies


  • Dyno Therapeutics and Roche will collaborate on the development of gene therapy delivery tools to treat neurological and liver diseases. The two companies will utilize Dyno’s artificial intelligence approach to develop novel AAV viral vectors, optimizing tissue targeting and immune-evading properties. Link


Industry and Corporate Developments


  • Eli Lily acquired Disarm Therapeutics, an early stage biotech developing treatment for neurological diseases, for $135M upfront with $1B in potential milestone payments. Link


  • Priothera raised $35M in Series A funding for clinical trials of its orally administered sphingosine 1 phosphate (S1P) receptor modulators to treat AML. Link


  • EdiGene, a Beijing biotech, raised $67M in Series B funding, which will be used to push forward its pipeline including an allogeneic CAR-T treatment. Link


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