Welcome to The BioPharm Brief, your daily snapshot of developments shaping the biopharmaceutical industry. Here’s what we have for today.
First, the FDA has approved daraxonrasib for previously treated metastatic pancreatic ductal adenocarcinoma, making it the first approved RAS(ON) inhibitor and the first therapy to validate a tri-complex approach to targeting active RAS. The once-daily oral drug, developed by Revolution Medicines, was supported by Phase 3 RASolute 302 data showing median overall survival of 13.2 months versus 6.7 months with chemotherapy. The approval came about six and a half months ahead of the FDA’s action date after the application received Breakthrough Therapy, Orphan Drug, Priority Review, and Commissioner’s National Priority Voucher designations.
In multiple myeloma, HaemaLogiX has dosed the first patient in its Phase 1 KOALA trial of KMCAR T-cell therapy. The autologous CAR-T is designed to target the Kappa Myeloma Antigen on kappa-restricted malignant plasma cells while sparing healthy immune cells. The approach differs from BCMA-directed CAR-T therapies by focusing on a more selective target. The first patient was reported to have tolerated the initial infusion without serious adverse events, and a second patient has entered the dose-escalation study.
And in manufacturing, 4basebio and Genezen are expanding a collaboration around cell-free synthetic DNA. The non-exclusive agreement will give developers access to 4basebio’s research-use, high-quality, and GMP-grade synthetic DNA for viral vector programs. The cell-free process eliminates bacterial backbone sequences and antibiotic resistance genes from the DNA starting material. In AAV production, the companies say the technology has produced comparable titers to plasmid DNA while using about 30% less DNA mass and transfection reagent.
That’s The BioPharm Brief for today. For analysis and expert insights, please visit BioPharmInternational.com.
Key takeaways:
- Daraxonrasib becomes the first approved RAS(ON) inhibitor in metastatic pancreatic cancer.
- HaemaLogiX moves its kappa-targeted CAR-T into first-in-human clinical testing.
- 4basebio and Genezen are bringing cell-free synthetic DNA into viral vector manufacturing.
