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Week of October 12

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.


  • A systematic review published in JAMA Oncology found that acute myeloid leukemia patients who have a negative measurable residual disease (MRD) status were associated with improved survival outcomes. MRD detection could be implemented as a clinical trial endpoint to rapidly ensure the efficacy of investigational therapies. Link

  • While beneficial in metastatic breast cancer, Pfizer’s Ibrance (palbociclib) failed to meet its primary endpoint of improved invasive disease-free survival (iDFS) as second adjuvant therapy in HR+/HER2- early breast cancer study. Link

  • In a Phase 3 trial, Bristol-Myers Squibb’s Opdivo reported improved pathologic complete response as a neoadjuvant treatment for patients with resectable non-metastatic NSCLC. This is the first time an immune checkpoint inhibitor-based combination demonstrated efficacy over SOC in this indication. Link

  • The National Medical Products Administration (NMPA) of China approved Innovent Biologics and Eli Lilly’s Halpryza (rituximab injection) to treat patients with B-cell lymphoma (DLBCL), follicular lymphoma (FL), and chronic lymphocytic leukemia (CLL). Halpryza is the second monoclonal antibody approved by NMPA co-developed by Innovent BIologics and Eli Lilly. Link

  • The European Patent Office granted Targovax ASA’s patent application for their development of oncolytic viruses to target hard-to-treat solid tumors in combination with anti-PD1 checkpoint inhibitors. By the end of the year, Targovax will present data from a clinical trial determining the efficacy of their ONCOS-102 therapy in refractory melanoma who have progressed on checkpoint inhibitor treatment. Link

  • Seagen, formally known as Seattle Genetics, and Astellas Pharma announced positive Phase 2 results for Padcev (enfortumab vedotin-ejfv) to treat locally advanced or metastatic urothelial cancer. Patients receiving Padcev, an antibody-drug conjugate (ADC) targeting Nectin-4, had a 52% ORR and a median duration of response of 10.9 months. Link

CNS Disorders and Neurodegeneration

  • At the upcoming Child Neurology Society/International Child Neurology Association (CNS/ICNA) 2020 Virtual Congress, Ovid Therapeutics will present 4 abstracts for OV101 (gaboxadol) to treat neurodevelopmental conditions. Abstracts will include Phase 2 results to treat patients with Fragile X Syndrome and Angelman Syndrome. Link


  • The University of Manchester developed an investigational gene therapy to treat patients with Hunter Syndrome. The lentiviral gene therapy works by transducing a patient’s hematopoietic stem cells with a therapeutic transgene containing the functional lysosomal enzyme iduronate-2-sulfatase (IDS). AVROBIO recently announced an exclusive licensing agreement for this gene therapy, called AVR-RD-05, and is expecting to open a Phase 1/2 trial in the second half of 2021. Link

  • Takeda Pharmaceutical Company and Arrowhead Pharmaceuticals announced a collaboration and licensing agreement to develop an RNA interference (RNAi) therapy to treat alpha-1 antitrypsin-associated liver disease (AATLD). ARO-AAT is a therapy that reduces the production of mutant alpha-1 antitrypsin protein which contributes to AATLD progression. Link

Industry and Corporate Developments

  • Earlier this year, protein degraders received a lot of attention with companies receiving almost $230M in funding. Recently, Janpix announced the closing of its $10M Series B funding to advance its pipeline of STAT protein degrades. Link

  • A2 Therapeutics raised $71.5M in Series B financing for their Tmod platform and clinical development of 3 near-term cell therapies. The design and concept behind their cell therapies was recently published in Molecular Immunology. Link

  • BridgeBio Pharma announced the acquisition of one of its formally owned subsidiaries, Eidos Therapeutics, for up to $175M. This merger will give BridgeBio rights to a transthyretin stabilizer to treat patients with transthyretin (TTR) amyloidosis (ATTR) cardiomyopathy and polyneuropathy. Link

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