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Week of November 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.


  • FDA removed its clinical hold on Poseidon Therapeutics CAR-T therapy Phase 1 trial to treat metastatic castration-resistant prostate cancer after agreeing to protocol amendments to increase patient compliance and safety. Link

  • Results from Jounce Therapeutics’ Phase 2 trial evaluating their ICOS-activating vopratelimab in combination with ipilimumab (Yervoy) to treat PD-L1-resistant NSCLC failed to meet pre-specified interim criteria to continue patient enrollment. Link

  • Allogene Therapeutics announced that they will present clinical results at the upcoming Annual Meeting of the American Society of Hematology (ASH) of their three allogeneic CAR T-cell therapies. Of note, the Phase 1 dose-escalation trial for their anti-BCMA allogeneic CAR T-cell therapy found that 2 out of 15 multiple myeloma patients were MRD negative while over a quarter of patients had Grade 3 or higher adverse events. Link

  • Also presenting at ASH, Eli Lilly and Loxo Oncology announced they will present Phase 1/2 results of their highly selective, non-covalent Bruton's tyrosine kinase (BTK) inhibitor, LOXO-305, in different non-Hodgkin lymphomas. LOXO-305 specifically targets BTKs with C481 mutations, the most common mutation after acquiring resistance to currently available BTK inhibitors. Link

CNS Disorders and Neurodegeneration

  • Biogen announced that the FDA advisory committee voted against approval for aducanumab to treat patients with Alzheimer’s disease. Based on the results from two Phase 3 trials, the committee almost unanimously did not find strong evidence that aducanumab is efficacious or reduces Alzheimer’s disease pathophysiology. Link

  • In response to a written response from the Center for Biologics Evaluation and Research (CBER) at the FDA, Sarepta Therapeutics announced it will adapt their plans for a large, multi-center Duchenne muscular dystrophy (DMD) trial evaluating its investigational gene transfer therapy into an open-label study enrolling up to 10 DMD patients. The study will evaluate the safety and expression of commercial process material for SRP-9001. Link


  • While the industry focuses on developing lentivirus or AAV based gene therapies, Orchard Therapeutics developed a gammaretroviral vector-based gene therapy to treat patients. However, one of the known risks associated with gammaretroviral vectors is leukemia. Recently, Orchard Therapeutics came under investigation after a patient treated with the gammaretroviral vector-based gene therapy, administered through a compassionate use program, was diagnosed with leukemia. Link

Industry and Corporate Developments

  • Merck announced the acquisition of VelosBio, which is focused on developing receptor tyrosine kinase-like orphan receptor 1 (ROR1) antibody-drug conjugate (ADC), for $2.75 B. The main driver of this deal is VelosBio’s lead candidate, VLS-101, which is being evaluated in Phase 1 for hematological malignancies and a Phase 2 clinical trial for solid tumors. Link

  • Homology Medicines received $60M in investment from Pfizer for their lead gene therapy candidates to treat rare metabolism disorders. Homology’s HM-102 received Fast Track Designation and orphan drug designation from the FDA and EMA to treat adults with phenylketonuria (PKU). Link

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