Week of September 21
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
Novartis announced positive Phase 3 results for Piqray to treat HR+/HER2- advanced breast cancer with a PIK3CA mutation. In combination with fulvestrant, Piqray demonstrated clinically relevant improvement in OS of eight months. However, this improvement did not reach the prespecified threshold of statistical significance set for the secondary objective OS of more than 14 months. Link
Eli Lily announced positive interim Phase 3 results for Verzenio to treat HR+/HER2- high-risk early breast cancer. Verzenio in combination with standard adjuvant endocrine therapy improved the invasive disease-free survival (IDFS) at two years. Link
At the European Society of Medical Oncology (ESMO) Virtual Congress, AstraZeneca, and Merck announced positive Phase 3 results for Lynparza to treat metastatic castration-resistant prostate cancer patients with BRCA1/2 or ATM gene mutations. Compared to Johnson & Johnson’s Zytiga or Pfizer and Astellas’ Xtandi, Lynparza reduced the risk of death by 31%. Link
Alkermes announced positive Phase 1/2 results for ALKS 4230 as a monotherapy and in combination with Merck’s Keytruda. ALKS 4230 demonstrated encouraging single-agent activity in melanoma and durable responses in multiple tumor types. Link
Bristol Myers Squibb and Exelixis announced positive Phase 3 results for Opdivo in combination with Cabometyx to treat previously untreated advanced renal cell carcinoma. Compared to sunitinib, the combination demonstrated a greater median PFS (8.3 months vs. 16.6 months). Link
Sanofi announced positive Phase 3 results for Libtayo to treat first-line locally advanced or metastatic NSCLC with tumors confirmed PD-L1 expression of ≥50%. Compared to platinum-doublet chemotherapy, Libtayo monotherapy demonstrated a 39% ORR, reduced the risk of death by 43%, and reduced the risk of disease progression by 46%. However, the median OS of 14 months was not significantly different. Link
Sanofi announced positive Phase 2 for Libtayo to locally advanced basal cell carcinoma (BCC) who had progressed or were intolerant to hedgehog inhibitor (HHI) therapy. Libtayo demonstrated a 31% ORR however median duration of response and median overall survival had not reached statistical significance. Link
Bristol Myers Squibb announced initial positive Phase 3 results for Opdivo to treat esophageal or gastroesophageal junction (GEJ) cancer following neoadjuvant chemoradiation therapy (CRT) and tumor resection. Compared to patients receiving placebo after surgery, the median DFS doubled in Opdivo treated patients after surgery. Link
Chi-Med announced positive Phase 3 results for Surufatinib to treat pancreatic neuroendocrine tumors. Patients receiving Surufatinib had a median PFS of 10.9 months compared to 3.7 months in patients receiving a placebo. Link
CNS Disorders and Neurodegeneration
Satsuma Pharmaceuticals announced negative Phase 3 results for STS101 to treat acute migraines. Compared to the placebo, STS101 failed to meet the statistical significance of its co-primary endpoints of freedom from pain and the most bothersome symptom. Link
Technologies
Autolus Therapeutics announced positive Phase 1/2 for AUTO3 to treat relapsed/refractory diffuse large B cell lymphoma. AUTO3, a CD19 and CD22 dual targeting CAR-T therapy, demonstrated a favorable safety profile with no patients suffering grade 3 or higher cytokine release syndrome compared to approved CD19 CAR-T therapies. Link
Industry and Corporate Developments
Bristol Myers Squibb announced the completion of their acquisition of Forbius. This deal provides Bristol Myers Squibb access to Forbius’ TGF-β program, especially its lead candidate AVID200 currently in Phase 1 trials. Link
Merck and Seattle Genetics announced a two-prong strategic collaboration worth up to $4.2B. Both companies will co-develop and commercialize an investigational ADC which is currently in phase 2 clinical trials for breast cancer and other solid tumors. Additionally, Seattle Genetics granted Merck an exclusive license to commercialize Tukysa outside the US, Canada, and Europe. Link
Athira Pharma, a late clinical-stage biopharmaceutical company aimed to restore neuronal health for those suffering from neurological diseases, raised $204M for its IPO. Link
Moderna and Vertex Pharmaceuticals announced a strategic collaboration to discover and develop lipid nanoparticles and mRNAs gene-editing therapies to treat cystic fibrosis. These gene therapies will be delivered to lung cells to encourage the production of functional cystic fibrosis transmembrane conductance regulator (CFTR) protein. Moderna is eligible for up to $450M in payments. Link
Roche announced the acquisition of Inflazome for €380M. Inflazome’s portfolio is composed of clinical and preclinical NLRP3 inhibitors to be tested across a wide variety of indications with a high unmet medical need. Link