Week of July 9

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


  • Immunomedics announced positive results from their Phase 3 ASCENT study of Trodelvy in previously treated relapsed or refractory advanced mTNBC. Trodelvy, an antibody-drug conjugate, met its primary endpoints of progression-free survival and overall survival. Link


  • FDA announced the creation of an online platform, Project Patient Voice, for compiling patient-reported symptoms from cancer clinical trials. The FDA hopes this information, usually not listed on drug labels, can help healthcare providers make more informed treatment choices. Link


  • The Helsinn Group and MEI Pharma decided to discontinue their Phase 3 study of Pracinostat in combination with azacitidine in AML. The decision was made after an interim analysis indicated that the overall survival endpoint would not be met. Link


  • FDA approved Pfizer’s Bavencio as first-line maintenance for treating locally advanced or metastatic urothelial carcinoma. In the first-line setting, Bavencio is the first immunotherapy to significantly improve overall survival in urothelial carcinoma. Link


CNS Disorders and Neurodegeneration


  • AstraZeneca’s and Merck & Co.’s selumetinib received orphan drug designation in Japan to treat neurofibromatosis type 1 (NF1). In the US, Selumetinib is approved to treat NF1 in pediatric patients since April 2020. Link


  • BrainStorm Cell Therapeutics announced dosing completion of their Phase 3 study of NurOwn in treating ALS. This adult stem cell gene therapy enrolled approximately 200 ALS patients receiving three doses of NurOwn or a placebo. The company expects the data to be available by the fourth quarter of 2020. Link


Technologies


  • Verve Therapeutics presented two studies suggesting that an adenine base editing (ABE) technology, licensed from Beam Therapeutics, has promise in controlling cholesterol levels and coronary heart disease. Targeting two heart disease-related genes in non-human primates showed significant drops in LDL cholesterol and blood triglyceride levels. Link


Industry & Corporate Developments


  • Amylyx Pharmaceuticals raised $30 Million in series B funding to advance its leading candidate, AMX0035, into Phase 2 trials for Alzheimer’s disease. Link


  • While healthcare M&A activity decreased in Q2 2020, Kaufman Hall’s recent report indicated that two key deals in June signaled that the overall M&A activity decline was not as dramatic as expected. Furthermore, Kaufman Hall anticipates an increase in activity due to the pandemic demonstrating the importance of strategic partnerships and an increase in distressed healthcare systems. Link 1, Link 2

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