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.

"What we've accomplished from then to now is what we dreamt of," Ardelyx Inc. CEO Mike Raab told BioWorld, referring to stellar phase III data with Na-hydrogen exchanger 3 (NHE3) transporter blocker tenapanor in irritable bowel syndrome (IBS-C), unveiled less than a week before the 10-year anniversary of the firm's founding. "Companies like ours don't get the attention at times from the world out there," he said, because they lack headline-grabbing approaches such as immuno-oncology and often face "a tougher, more extensive path to success."

Anaptysbio Inc.'s phase IIa interim data win with intravenous (I.V.) ANB-020, the antibody targeting interleukin-33 (IL-33) for eczema, touched off speculation about competitors working in the same pathway, as the firm's shares (NASDAQ:ANAB) climbed 101 percent, or $35.41, to close Tuesday at $70.41.