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.
CEO Donald McCaffrey told BioWorld that Resverlogix Corp.'s unpaid loan of $68.8 million is "something we don't want, and our shareholders don't want it, and we are very pleased to have that circumstance on the way out."
Acelrx Pharmaceuticals Inc. CEO Vincent Angotti said he was “hesitant to put a timeline on this until we get further clarification from the FDA” regarding what’s required to satisfy the agency’s issues raised in a complete response letter (CRL) related to its NDA for 30-mg sublingual tablet Dsuvia (sufentanil), an opioid therapy that’s intended to reduce moderate to severe acute pain as well as dosing errors that can happen with intravenous administration.
