Quentis Therapeutics Inc.'s $48 million in series A money will help, among other things, put into the clinic next year a small-molecule IRE1 alpha inhibitor for proof-of-concept work. Going after cancer immunotherapies that modulate endoplasmic reticulum (ER) stress response pathways in the tumor microenvironment (TME), Quentis will use the funds to push the leading prospect through clinical proof of concept, develop a pipeline of preclinical programs, and beef up the company's team.
Wall Street took a separate view, but Arca Biopharma Inc. CEO Michael Bristow said he is "very pleased with the outcome of this trial," a phase IIb experiment testing beta blocker and mild vasodilator Gencaro (bucindolol hydrochloride) compared with Toprol XL (metoprolol succinate, Astrazeneca plc).
"Pairing solid commercial execution with a pipeline that has been consistently and rapidly replenished, and now increasing profitability," Raymond James analyst Laura Chico found "much to like" in the latest earnings report from Biomarin Pharmaceutical Inc.
Vice President of Research Michael Holmes said Sangamo Therapeutics Inc.'s potential $3 billion-plus deal with Gilead Sciences Inc.'s Kite Pharma unit "leaves open a variety of areas for us to move into," with details to come later.
"People were surprised [by the deal] because we had been making a lot of progress and moving quickly [on our own]," Wave Life Sciences Ltd. CEO Paul Bolno told BioWorld, talking about the arrangement disclosed this week with Takeda Pharmaceutical Co. Ltd., a pact "very much aligned with our strategy, which is growth to becoming a fully integrated company."
A two-part webinar on drug pricing by sponsor USC-Brookings Schaeffer Initiative for Health Policy dragged somewhat during the first half, which dealt with the subject of whether and in what ways to retool Medicare Part D. But the livelier second half – on strategies to reduce cost sharing for commercially insured patients – included a talk by Fiona Scott Morton, professor of economics at the Yale School of Management, who raised some eyebrows on the panel.
At the WORLD Symposium on lysosomal diseases in San Diego this month, Ultragenyx Pharmaceutical Inc. made a splash with data related to Mepsevii (vestronidase alfa-vjbk, previously UX-003), the company's first drug to win approval.
Inadequately concentrated drug may have caused Tetraphase Pharmaceuticals Inc.'s topline phase III blowup with intravenous (I.V.) eravacycline in complicated urinary tract infections (cUTI), and the company said the results bear no read-through to the same compound's value in complicated intra-abdominal infections (cIAI), for which it's under review in the U.S. and Europe.
Pundits had more to debate than the U.S. reimbursement prospects for Vertex Pharmaceuticals Inc.'s cystic fibrosis (CF) drug Symdeko (tezacaftor/ivacaftor and ivacaftor), cleared by regulators just as the company halted phase III trials in France with a triple-combo CF prospect because a deal couldn't be reached there on reimbursement for another Vertex therapy, Orkambi (lumacaftor/ivacaftor).
Having chalked up another failure in the casualty-strewn Alzheimer's disease (AD) space, Boehringer Ingelheim GmbH (BI) is giving up on BI-409306, a phosphodiesterase 9 (PDE9) inhibitor, but not on its mechanism of action, and will keep pursuing work on the same compound in schizophrenia.
