Week of September 27

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


  • Janssen Pharmaceuticals announced positive interim results for a Phase I study evaluating a human bispecific EGFR and cMet antibody to treat patients with treatment-naïve EGFR-mutated NSCLC. Link


  • Merck’s pivotal NSCLC Phase 3 results demonstrated that compared to chemotherapy, Keytruda doubled the five-year survival rate and median OS in PD-L1 expressing metastatic tumors with no EGFR or ALK alterations. Link


  • Resolution Bioscience and LabCorp released Resolution ctDx Lung, a non-invasive liquid biopsy diagnostic for NSCLC. This blood test, covered by Medicare, detects actionable gene mutations associated with NSCLC. Link


  • Amgen published promising Phase I results in the New England Journal of Medicine for sotorasib. Across all doses, the KRASG12C inhibitor demonstrated an ORR and disease control rate (DCR) of 32.2% and 88.1%, respectively, in NSCLC patients. Link


  • Japan’s Ministry of Health, Labor, and Welfare approved Roche’s and Chugai Pharmaceutical’s Tecentriq and Avastin intravenous infusion to treat unresectable hepatocellular carcinoma (HCC). The approval is based on positive Phase 3 results demonstrating that the combination therapy reduced the risk of death by 42% and reduced the risk of disease worsening by 41%. Link


  • Bristol Myers Squibb’s Opdivo met its primary endpoint of improving disease-free survival (DFS) in patients with high-risk, muscle-invasive urothelial carcinoma in a pivotal Phase 3 trial. This is the first Phase 3 trial where immunotherapy has reduced the risk of relapse in the adjuvant setting for this indication. Link


  • FDA granted priority review to the Pfizer’s supplemental New Drug Application for Xalkori to treat pediatric patients with ALK+ relapsed or refractory systemic anaplastic large cell lymphoma (ALCL). If approved, Xalkori would be the first biomarker-driven therapy for this type of pediatric lymphoma. Link


CNS Disorders and Neurodegeneration


  • Roche and AC Immune’s anti-tau antibody, developed for Alzheimer's disease, failed to meet either its primary endpoint or its two secondary endpoints compared to placebo in a Phase 2 study. Link1, Link2


  • FDA granted Orphan Drug and Fast Track designations to AbbVie’s elezanumab to treat spinal cord injuries in multiple sclerosis and acute ischemic stroke. The monoclonal antibody binds selectively to repulsive guidance molecule A (RGMa), leading to neuronal regeneration and functional recovery following CNS damage. Link


  • Roche announced positive results for their open-label, two-part clinical trial to treat infants aged 2-7 months with symptomatic Type 1 spinal muscular atrophy (SMA). After 2 years, 17 of the 21 infants treated with Evrysdi (risdiplam) did not require permanent ventilation and achieved motor skill milestones. Link


Technologies


  • The FDA accepted for Priority Review Bristol Myers Squibb’s and bluebird bio’s Biologics License Application (BLA) for idecabtagene vicleucel (ide-cel; bb2121) to treat multiple myeloma patients who have received at least three prior therapies. The FDA has set a PDUFA goal date of March 27, 2021 for the investigational B-cell maturation antigen (BCMA)-directed CAR T-cell immunotherapy. Link


  • Sarepta Therapeutics presented results from their Duchenne muscular dystrophy (DMD) clinical trial at the 25th International Annual Congress of the World Muscle Society. After two years, participants who received the investigational gene transfer therapy, SRP-9001, exhibited a sustained functional improvement compared to baseline. Link


Industry and Corporate Developments


  • Illumina agreed to pay $8B to acquire Grail, a healthcare company focused on multi-cancer early detection, in an effort to expand into the cancer detection space. Link


  • Kronos Bio filed for a Nasdaq IPO to raise up to $100M. The funds will be used to start a registrational Phase 2/3 trial for Kronos’ SYK inhibitor licensed from Gilead to treat acute myeloid leukemia (AML) patients with an NPM1 mutation. Link


  • Greenwich LifeSciences, a clinical-stage biopharmaceutical company focused on developing immunotherapy to prevent the recurrence of breast cancer following surgery, announced the pricing of its IPO of 1,260,870 shares at $5.75 per share, grossing around $7.25M. Link


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