Week of August 17

Oncology

• Bristol Myers Squibb announced positive results for two Phase 3 trials for Opdivo as first-line therapy and as adjuvant therapy for metastatic gastric cancer, gastroesophageal junction cancer, and esophageal adenocarcinoma. As first-line therapy, Opdivo plus chemotherapy met its primary endpoints of OS at a pre-specified interim analysis and PFS at the final analysis. As an adjuvant therapy, Opdivo following neoadjuvant chemoradiation therapy (CRT) met its primary endpoint of DFS at a prespecified interim analysis. Bristol Myers Squibb plans to share the results at an upcoming medical conference. Link

• FDA approved Guardant Health’s comprehensive genomic profiling diagnostic tool, Guardant360, to identify EGFR alterations in NSCLC patients. Guardant360 would help identify NSCLC patients who would benefit from Tagrisso treatment. Link

• CARMA Cell Therapies, a subsidiary of MaxCyte, announced the expansion of their ongoing Phase 1 trial for MCY-M11. The expanded cohort will evaluate relapsed/refractory ovarian cancer and malignant peritoneal mesothelioma patients that were preconditioned with cyclophosphamide prior to the MCY-M11 infusion. Preliminary results from the no-preconditioning cohort are expected later this year. Link

• AbbVie published positive Phase 3 data in the New England Journal of Medicine in treating acute myeloid leukemia (AML) patients with venetoclax plus azacytidine. The combination compared to azacitidine plus placebo increased median OS rates by 5 months reducing the risk of death by 34%. Link

CNS Disorders and Neurodegeneration

• FDA approved Roche’s Enspryng to treat anti-aquaporin-4 (AQP4) antibody-positive neuromyelitis optica spectrum disorder (NMOSD). This is the first approved therapy designed to treat NMOSD which inhibits IL-6 receptor activity. Link

• Triplet Therapeutics announced their participation in the large international natural history study (END-DM1) looking to advance understanding of myotonic dystrophy type 1 (DM1). Triplet Therapeutics is providing financial support for patient enrollment and investigation of additional potential biomarkers for DM1. Link

Technologies

• Oncologic Drugs Advisory Committee (ODAC), an FDA advisory group, voted in favor of Mesoblast Limited’s Ryoncil to treat pediatric patients with steroid-refractory acute graft versus host disease (SR-aGVHD). The FDA will take ODAC’s recommendation into account for its final decision by September 30, 2020. Ryoncil is a cell therapy composed of culture-expanded mesenchymal stem cells from unrelated donors. Link

• With the FDA maintaining their clinical hold on Passage Bio’s gene therapy PBGM01, Passage Bio announced delaying the dosing of its Phase 1/2 trial into late 2020 or early 2021. The clinical hold was placed due to concerns about PBGM01’s delivery mechanism, direct injection into the cisterna magna of the brain. Link

• FDA approved Roche’s supplemental Biologics License Application (sBLA) for a self-administration option for Xolair across all approved US indications. If approved Xolair prefilled syringes would become available to select patients to self-administer or their caregivers to administer. A final FDA approval is expected early next year. Link

Industry and Corporate Developments

• JW Therapeutics submitted its IPO filing on the Hong Kong stock exchange. This comes after JW Therapeutics and Lyell Immunopharma announced a strategic collaboration where JW has exclusive rights in China for two of Lyell’s CAR-T programs. Link

• Cellular Biomedicine Group announced it has signed a merger agreement to be acquired by three Chinese wealth management companies and the company will become a private company again. Cellular Biomedicine develops CAR-T cell and stem cell therapies. Link

• Sanofi announced the acquisition of Principia Biopharma for $3.7B. A key driver for this deal was Principia’s brain-penetrant BTK inhibitor SAR442168 to treat multiple sclerosis (MS) which Sanofi wants to expand into other CNS diseases. Link

• Bayer announced the acquisition of KaNDy Therapeutics for $875M. The key driver for this deal was KaNDy Therapeutics’s NT-814, a first in class, non-hormonal neurokinin-1,3 receptor antagonist to treat menopause symptoms. It is estimated that if approved, NT-814 could generate up to 1 billion euros globally. Link

• Bristol Myers Squibb and Dragonfly Therapeutics announced a global exclusive license agreement for Dragonfly’s interleukin-12 (IL-12) investigational immunotherapy program and its extended half-life cytokine DF6002. Bristol Myers Squibb will focus on the development and global commercialization of DF6002 and Dragonfly will receive $475M in upfront payments. Link

• Innovent Biologics and Eli Lilly announced a global expansion of their strategic partnership of TYVYT, an anti-PD-1 monoclonal antibody. Eli Lilly will obtain an exclusive license for TYVYT outside of China and pursue TYVYT registration in the US and other markets. TYVYT is approved in China to treat relapsed or refractory classic Hodgkin's lymphoma as third-line therapy and both companies are looking to expand into squamous NSCLC after the National Medical Products Administration (NMPA) of China approved the companies’ supplemental NDA. Link

• Tango Therapeutics closed $50M in equity financing from Casdin Capital, Boxer Capital, Cormorant Asset Management, and Gilead Sciences. The funding will help initiate clinical studies for its lead program and help other programs that are in late-stage drug discovery. Link

• Novo Nordisk and Evotec announced a partnership to develop pre-clinical candidates to help treat all modalities of chronic kidney disease (CKD). Evotec can receive up to $179M per CKD candidate that meets certain milestones. Link