Week of October 2

Oncology

• Myovant Sciences’s Phase 3 trial for relugolix failed to achieve clinically significant improvements compared to standard of care on time to resistance to castration medications in advanced prostate cancer patients. Link

• FDA approved Bristol Myers Squibb’s Opdivo and Yervoy as a first-line treatment of patients with unresectable malignant pleural mesothelioma (MPM). In the first-line setting, this is the combination’s third approved indication to treat patients with a form of thoracic cancer. However, the combination of Opdivo and Yervoy did not meet clinical significance in treating patients who have had a complete surgical removal of stage IIIb/c/d or stage IV melanoma. Link 1, Link 2

• Positive results from a topline Phase 2 trial demonstrated that Amgen‘s sotorasib had a ORR consistent with its previously reported Phase 1 data in treating patients with KRAS G12C-mutant advanced NSCLC who had failed a median of two prior lines of therapies. Link

CNS Disorders and Neurodegeneration

• EIP Pharma, a CNS-focused therapeutics company, announced positive Phase 2 results for neflamapimod in treating patients with mild-to-moderate dementia with Lewy bodies (DLB). Compared to placebo and neflamapimod BID, neflamapimod TID met its primary endpoint of an improvement in cognition and secondary endpoints. Results will be presented at the upcoming 13th Clinical Trials in Alzheimer’s Disease (CTAD) meeting. Link

• In collaboration with the NIH, Neurotrope dosed the first patient in their long-term Phase 2 study of Bryostatin-1 for the treatment of Alzheimer's disease. The trial will evaluate the PKCε activator over a six-month period and will measure patient’s sustained cognitive function. Link

Technologies

• While the FDA voted 9:1 that the data for Mesoblast Limited’s lead allogeneic cell therapy, remestemcel-L, demonstrates efficacy in treating pediatric steroid-refractory acute graft versus host disease (SR-aGVHD), the FDA recommends an additional randomized, controlled clinical study before approving Mesoblast’s BLA. Link

• The FDA lifted its clinical hold on Solid Biosciences’s Phase 1/2 trial for their Duchenne Muscular Dystrophy experimental gene therapy trial. Link

• Pfizer received Fast Track designation from the FDA for their investigational gene therapy candidate (PF-06939926) to treat Duchenne muscular dystrophy (DMD).PF-06939926 is a recombinant adeno-associated virus serotype 9 (rAAV9) capsid carrying a shortened version of the human dystrophin gene (mini-dystrophin). Link

• Sanofi published Phase 1/2a results in the New England Journal of Medicine for their investigational recombinant factor VIII therapy, BIVV001, having promising safety and tolerability in patients with severe hemophilia A. Link

Industry and Corporate Developments

• Bristol Myers Squibb announced their acquisition of MyoKardia for $13.1B. The key driver of this deal is MyoKardia’s mavacamten, a potential first-in-class myosin inhibitor to treat patients with obstructive hypertrophic cardiomyopathy (“HCM”). An NDA submission for mavacamtenis is expected in the first quarter of 2021. Link

• Johnson & Johnson announced the completion of its acquisition of Momenta Pharmaceuticals for $6.5B. This expands Johnson & Johnson drug development into treating patients with autoantibody-driven and immune-mediated diseases. Link

• Inventisbio, a Shanghai-based clinical stage biotech company, announced the closing of a $147M Series D financing to advance products into Phase 2 trials for HR+ breast cancer and gout. Link

• AZTherapies, a Boston-based biotech company, announced the closing of a $33.6M Series C-1 financing to advance their Phase 3 COGNITE trial in Alzheimer’s disease. Link

• Orgenesis announced the acquisition of Koligo Therapeutics, a regenerative medicine company, for $16.3M. Once completed, Orgenesis plans to utilize Koligo’s 3D-V bioprinting technology on their POCare platform which identifies promising cell and gene therapies and provides an avenue to reach patients more quickly and more efficiently. Link

• Covis Group agreed to pay $650M to acquire AMAG therapeutics, a commercial-stage biopharmaceutical company developing therapeutics to a variety of under-treated disease indications. Link

• Pfizer and CStone Pharmaceuticals announced a strategic collaboration to treat lung, gastric, and esophageal cancers. Pfizer will invest $200M into the Hong Kong company in exchange for the development and commercialization of CStone’s PD-L1 antibody, sugemalimab, in mainland China. Link

• A US Congressional House Oversight Committee released staff reporters accusing Celgene, Bristol Myers Squibb, and Teva with increasing drug prices as the Committee is hearing from CEOs from 6 drug companies. Link