Week of October 26
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
Turning Point Therapeutic’s Phase 1 study of TPX-0022, a MET inhibitor, demonstrated clinical efficacy and safety across multiple tumor types. 9 out of 15 evaluated advanced solid tumor patients with MET-dysregulation achieved clinical benefit, confirmed or unconfirmed partial response or stable disease. Link
Forma Therapeutics announced positive interim Phase 2 clinical results for olutasidenib to treat IDH1m relapsed/refractory acute myeloid leukemia (AML). While Olutasidenib achieved their primary endpoint of a composite complete remission (CR+CRh) rate of 33.3%, olutasidenib did not meet their CR+CRh median duration. Link
CNS Disorders and Neurodegeneration
Sanofi and the Parkinson's Foundation announced a collaboration in the US to advance the availability of genetic testing and counseling for Parkinson's disease patients. Over the next 2 years, Sanofi will fund the PD GENEration initiative which test patients for key Parkinson's-related genes, GBA, LRRK2, and SNCA, and identify if they qualify for certain clinical trials. Link
Technologies
CRISPR Therapeutics’ CTX110 utilizes CRISPR/Cas9 to develop an allogeneic CD19 CAR T-cell therapy from healthy donor samples to treat relapsed or refractory CD19+ B-cell malignancies. Early Phase 1 results found that 4 out of 11 patients achieved a CR. However, one patient who received the highest dose died, leading to a pause in enrollment for the high dose arm of the trial. Link
Intellia Therapeutics received authorization from the United Kingdom Medicines and Healthcare products Regulatory Agency (MHRA) to start their Phase 1 trial evaluating NTLA-2001 treatment of patients with hereditary transthyretin amyloidosis with polyneuropathy (hATTR-PN). NTLA-2001 also utilizes CRISPR/Cas9 to knockout and reduce the levels of mutant TTR. Link
Industry and Corporate Developments
Bayer agreed to acquire Asklepios Biopharmaceuticals (AskBio) for $2B allowing Bayer to become a player in gene therapy development. AskBio has several pre-clinical and clinical AAV-based gene therapy candidates to treat Pompe disease, Parkinson’s disease, and congestive heart failure. Link
Be Biopharma, a biotech company looking to develop cell therapies from B-cells, raised $52M in Series A funding from Atlas Venture and RA Capital Management. Funds will be used to develop B-cell therapies to treat a variety of diseases. Link
Sarepta Therapeutics raised $107M for its new startup, AavantiBio, which will focus on developing gene therapies for rare genetic diseases. Link
Ultragenyx and Solid Biosciences announced a strategic collaboration to develop and commercialize new gene therapies for Duchenne Muscular Dystrophy (DMD). Solid Biosciences received $40M upfront with $255M in cumulative milestone payments for an exclusive license for their AAV8 gene therapy to treat DMD and other diseases. Link
Roche announced a collaboration with Atea Pharmaceuticals worth $350M to jointly develop Atea’s orally administered direct-acting antiviral to treat COVID-19 outside the hospital. Link
Pfizer’s Hospital Business agreed to acquire Arixa Pharmaceuticals. The main driver of this deal is Arixa’s lead candidate, ARX-1796, an oral prodrug of a beta-lactamase inhibitor to treat Gram-negative bacterial infections. Link