Pressed for details about timing of next steps with gemcabene in dyslipidemia – still on partial clinical hold by the FDA – Gemphire Therapeutics Inc. CEO Steve Gullans said the regulators' demand for more toxicology data is "very recent news, so we haven't had time, working with all the third-party vendors that will be involved, to figure out the exact timeline. We would love to get the 13-week study up and running immediately, but these PPAR alpha knockout mice require some work to get a breeding colony up and rolling. That's why we're targeting the end of the year, beginning of next year. Right now, it's the best estimate we're operating from. That's in keeping with what we've seen in prior work we've done around breeding colonies. Roughly speaking, we would see a nine to 12-month delay over where we were before" the agency made known its decree.
Not all investors caught the drift of Ovid Therapeutics Inc.'s phase II data with OV-101 (gaboxadol) in Angelman syndrome (AS), but CEO Jeremy Levin told BioWorld that insiders rejoiced and the company is moving ahead with the delta-selective GABAA receptor agonist.
After the Centers for Medicare & Medicaid Services (CMS) brought up reimbursement concerns about Giapreza (angiotensin II), La Jolla Pharmaceutical Co.'s intravenous therapy to increase blood pressure in adults with septic or other distributive shock, investors had cause to worry that the compound wouldn't qualify for a new technology add-on payment (NTAP) under the inpatient prospective payment system (IPPS).
Readying for a phase II in the third quarter with Karxt, its combo drug for schizophrenia, Karuna Pharmaceuticals Inc. aims to capitalize on data gained previously by licensor Eli Lilly and Co. with the muscarinic acetylcholine receptor agonist xanomeline as well as historic findings with trospium chloride, a muscarinic receptor antagonist approved by the FDA in 2004 for overactive bladder.
With about $77 million in hand from a public offering and a phase II trial in progress with its farnesyl transferase inhibitor, tipifarnib (tipi), Kura Oncology Inc. is gearing up for a 59-patient pivotal study in squamous head and neck cancer patients with mutations in the HRAS oncogene. Enrollment could take two years, but the experiment puts the company "at the cutting edge" of research into a notoriously tough disease, CEO Troy Wilson told BioWorld.
Biogen Inc. and Eisai Co. Ltd. raised the curtain on phase II data from Study 201 in early Alzheimer's disease (AD) with anti-amyloid beta protofibril antibody BAN-2401, offering promise at last against the scourge that has foiled so many candidates.
Glaxosmithkline plc (GSK) and the direct-to-consumer genetic testing firm 23andme Inc. kicked off a four-year collaboration – with an option to extend into a fifth year – that includes a $300 million equity investment by the pharma player.
Sutro Biopharma Inc.'s deal with Merck & Co. Inc. puts $60 million in the bank immediately and could garner as much as $1.6 billion more in milestone payments as the companies put Sutro's cell-free protein synthesis and site-specific conjugation platforms to work in a bid for immune-modulating cytokine derivatives targeting oncology and autoimmune indications.
Data from Irving, Texas-based Reata Pharmaceuticals Inc.'s phase II Cardinal study with bardoxolone in patients with chronic kidney disease (CKD) due to Alport syndrome whetted investor appetite for the phase III portion of the study, which includes a placebo arm and looks likely to win.
This month's guidance from the FDA regarding gene therapy development put even more oomph behind PTC Therapeutics Inc.'s buyout of Agilis Biotherapeutics Inc., a move that brings aboard four programs, including one due for a BLA next year.
