Juno Therapeutics Inc."invested to learn deeply" from last year's deaths in clinical trials, Sunil Agarwal, president of research and development, told BioWorld, and "having put both feet in at the beginning" is what led to the success with a chimeric antigen receptor (CAR) T-cell therapy disclosed Wednesday.
Voyager Therapeutics Inc. CEO Steven Paul said his firm is "pleased to retain – and gain, if you will – worldwide rights to this [Parkinson's disease (PD)] program" from Sanofi SA. "We believe it's a winner."
With her company "in the hunt for a series B" that could "unfold in the coming few months," X4 Pharmaceuticals Inc. CEO Paula Ragan told BioWorld that the latest data with X4P-001-IO, a C-X-C receptor type 4 (CXCR4) inhibitor, should give clear-cell renal cell carcinoma cancer patients new hope.
In 2010, when Sanofi SA signed a potential $560 million deal to get its hands on JAK2 inhibitor fedratinib (then known as TG-101348) via the buyout of Targegen Inc., the latter's director of research, John Hood, had high hopes for patients with myelofibrosis (MF) and polycythemia vera (PV). Several years later, though, safety problems turned up at the phase III stage, the FDA put fedratinib on clinical hold, and Sanofi dropped fedratinib like a hot potato.
A degree of investor fretting over Immune Design Corp.'s interaction with the FDA on the subject of phase III plans with CMB-305 in synovial sarcoma didn't stop the Seattle-based firm from pricing a public offering of 19.5 million shares at $4.10 each.
Arnon Rosenthal, CEO of Alector LLC, told BioWorld that his firm is "opening a new modality, a new concept in therapeutics for Alzheimer's disease [AD]," and made a believer out of Abbvie Inc., which put down $205 million up front for the option rights to two candidates.
"Nobody ever thought that you could make a modification to the transgene and not start over," Uniqure NV CEO Matt Kapusta told BioWorld, but that's what the company did with its gene therapy for hemophilia B, and Wall Street was delighted.
The FDA's Endocrinologic and Metabolic Drugs Advisory Committee gave its blessing to semaglutide, voting 16-0 with one abstention to recommend that the candidate from Novo Nordisk S/A be approved for type 2 diabetes.
Gemini Therapeutics Inc.'s $42.5 million series A round will "take us into the clinic with multiple programs," CEO James McLaughlin told BioWorld. The Cambridge, Mass.-based firm is working on genetically defined dry age-related macular degeneration (AMD) and associated rare genetic diseases, with "about 18 months of R&D under our belt," he said.
Whether and to what extent the risk of diabetic retinopathy will figure into deliberations of Wednesday’s FDA panel on Novo Nordisk A/S’ semaglutide stood as one of the main questions as investors scanned briefing documents made public Monday.
