Week of August 3

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


  • FDA gave clearance to Zebra Medical Vision’s AI-based software, which identifies mammograms suspected of showing the presence of breast cancer. This is the company’s sixth FDA clearance but the first one in oncology. Link


  • FDA approved Roche’s VENTANA HER2 Dual ISH DNA Probe Cocktail assay to detect HER2 in breast cancer. This assay is designed to be completed within the same day and will be used as a diagnostic to determine Herceptin therapy usage. Link


  • Spectrum Pharmaceuticals announced positive topline Phase 2 results for poziotinib in treating NSCLC patients with HER2 exon 20 insertion mutations. NSCLC patients receiving poziotinib had increased ORR compared to a pre-specified ORR. Link


  • FDA granted breakthrough therapy designation to AstraZeneca’s Tagrisso as adjuvant therapy for early-stage (IB, II and IIIA) EGFR-mutated NSCLC following complete tumor resection. This designation was given after a Phase 3 trial determined that the addition of Tagrisso reduced the risk of disease recurrence or death by 79%. Link


  • The European Commission granted approval for Roche’s Rozlytrek in two indications: treating adults and pediatric solid tumors expressing a neurotrophic tyrosine receptor kinase (NTRK) gene fusion and treating adults with ROS1+ advanced NSCLC. Link


  • Bristol Myers Squibb and bluebird bio, Inc. announced their Biologics License Application (BLA) submission for idecabtagene vicleucel in treating relapsed and refractory multiple myeloma. Idecabtagene vicleucel is an investigational B-cell maturation antigen (BCMA)-directed chimeric antigen receptor (CAR) T cell immunotherapy that was previously granted breakthrough therapy designation by the FDA and PRIority MEdicines (PRIME) designation by the EMA for relapsed and refractory multiple myeloma. Link


  • FDA granted breakthrough therapy designation to Takeda Pharmaceutical’s pevonedistat in treating higher-risk myelodysplastic syndromes (HR-MDS). Pevonedistat could be the first novel treatment in more than a decade with treatment options limited to hypomethylating agents. Link


  • FDA approved Roche’s Tecentriq in combination with Cotellic and Zelboraf in treating BRAF V600 mutation-positive advanced melanoma patients. This was based on positive Phase 3 results determining the addition of Tecentriq to Cotellic and Zelboraf significantly increased median PFS, with no change in adverse events compared to placebo plus Cotellic and Zelboraf. Link


CNS Disorders and Neurodegeneration


  • Eli Lilly published their findings in JAMA for their Alzheimer's disease blood-based assay, P-tau217 (phosphorylated tau at threonine-217). Compared to an MRI or competing blood-based biomarker assays, P-tau217 was significantly better at distinguishing Alzheimer's disease from other neurodegenerative diseases. Link


  • Teva Pharmaceutical Industries announced submitting a New Drug Application for AJOVY in preventing episodic and chronic migraines in Japan. This long-acting anti-CGRP subcutaneous injection is already approved in the US and the EU. Link


  • AbbVie announced positive Phase 3 results for atogepant to treat patients with migraines. Atogepant met its primary endpoint of significantly reducing the mean number of migraine days per month compared to placebo. With these positive results, AbbVie will move forward with regulatory submissions in the US and other countries. Link


Technologies


  • Biotech jCyte and Santen Pharmaceuticals announced positive Phase 2b results for jCyte’s stem cell therapy to treat retinitis pigmentosa. Patients given human retinal progenitor cells experienced improved functional vision compared to the sham or placebo group. Link


  • TCR² Therapeutics announced positive interim Phase I results for their T cell receptor (TCR) fusion construct T-cell therapy to treat mesothelin-expressing solid tumors. TC-210 treatment lead to two patients with stable disease and two patients with unconfirmed partial responses out of five patients. Link


Industry and Corporate Developments


  • Roche announced a collaboration with UCB for their anti-Tau antibody UCB0107. Contingent on positive proof-of-concept results in Alzheimer’s disease, UCB will receive up to $2B for exclusive, world-wide rights to UCB0107. Link


  • Sangamo and Novartis announced a collaboration to develop genomic therapies for neurodevelopmental disorders. Sangamo will receive up to $795M to advance their genome regulation technology, zinc finger protein transcription factors (ZFP-TFs), in exchange for exclusive rights to ZFP-TFs targeting three undisclosed genes. Link


  • Anonymous sources suggested that the Moderna’s COVID-19 vaccine price will be between $50-60 per dose, up to three times that of Pfizer’s and BioNTech’s vaccine price of $19.50 per dose reported last week. Link


  • In response to four executive orders from the Trump administration aimed at lowering drug prices, Pharma executives declined to have a meeting with the White House focusing on ways to lower drug prices for patients. Link


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