With the release by the FDA of briefing documents Friday, any lingering investor frets eased about the fate of Acadia Pharmaceuticals Inc.'s Nuplazid (pimavanserin) for psychosis associated with Parkinson's disease (PDP) at Tuesday's advisory panel. U.S. regulators seemed happy with the 5HT2a inverse agonist's efficacy as well as safety, making no mention of a black-box warning, though the truth won't be known until members of the Psychopharmacologic Drugs Advisory Committee cast their votes on three questions and the full agency decides.
With sales of vascular endothelial growth factor (VEGF) trap Eylea (aflibercept) as a monotherapy possibly slowing down, Regeneron Pharmaceuticals Inc. and Bayer AG have broadened their work in eye disease with a new $130 million agreement to test the treatment in combination with the angiopoietin2 (Ang2) antibody nesvacumab, and two phase II trials are enrolling patients with wet age-related macular degeneration (AMD) or diabetic macular edema (DME).
The study of arginine residue on proteins at Scripps Research Institute – a "pretty arcane" and "obscure" research area – that led to the forming of Padlock Therapeutics Inc. laid the foundation for Bristol-Myers Squibb Co.'s (BMS) $600 million buyout, Padlock CEO Michael Gilman told BioWorld Today.
"We're not done," Zymeworks Inc. CEO Ali Tehrani told BioWorld Today as the firm disclosed its takeover of Kairos Therapeutics Inc. to bring aboard a next-generation antibody-drug conjugate (ADC) technology.
The slip-up on the secondary neurocognitive endpoint in Biomarin Pharmaceutical Inc.'s pivotal phase III PRISM-2 study in phenylketonuria (PKU) with pegvaliase, also known as Peg-Pal, probably matters less than the win with regard to phenylalanine (Phe) levels, analysts figure – and may not have been much of a miss anyway.
Intrigued by results from a 13-patient, phase II pilot study with peroxisome proliferator-activated receptor (PPAR) delta agonist MBX-8025 for homozygous familial hypercholesterolemia (HoFH), Cymabay Therapeutics Inc. has begun exploring the prospect of combining it with a proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitor to make the drug work even better.
Axsome Therapeutics Inc. is hoping the fixed-dose marriage of dextromethorphan (DM) with established depression player bupropion in oral AXS-05 will do the trick against treatment-resistant depression (TRD) in the newly launched phase III trial called STRIDE-1 (Symptom Treatment in Resistant Depression 1).
Bioblast Pharma Ltd. CEO Colin Foster told BioWorld Today "it would not be unreasonable to think" that regulators could deem pivotal the upcoming phase IIb trial with trehalose in oculopharyngeal muscular dystrophy (OPMD), but the company doesn't count on that. "We're looking forward, recognizing that we [probably] have to do a phase III study," he said.
Celator Pharmaceuticals Inc. CEO Scott Jackson told BioWorld Today his firm has taken “the first step – it’s a huge step, but it’s the first step – toward becoming a new foundation of care” in high-risk (secondary) acute myeloid leukemia with Vyxeos, a liposomal form of cytarabine and daunorubicin.
Caladrius Biosciences Inc.'s worldwide, three-pronged cell-therapy technology deal through a subsidiary with Tokyo-based Hitachi Chemical Co. Ltd. not only brings money aboard but also could help snag a partner in Japan for the firm's CD34 ischemic-repair bid, communications director Eric Powers told BioWorld Today.
