Portola Pharmaceuticals Inc.'s chief financial officer, Mardi Dier, told BioWorld Today the deal with Healthcare Royalty Partners (HCRP) was "exactly what we planned to do" – add "part two" to the "part one" of nondilutive funding late last year, when the company signed a $50 million loan agreement with longtime partners Bristol-Myers Squibb Co. (BMS) and Pfizer Inc. for more resources to develop factor Xa inhibitor antidote Andexxa (andexanet alfa).
The two-month clinical hold that threw a wrench into Cytrx Corp.'s aldoxorubicin (aldox) phase III trial for soft-tissue sarcoma (STS) last July dimmed hopes, but by late November the company had dug deep enough into the results that it could plan for an NDA filing late this year. As the Los Angeles-based firm gears up for a type B pre-NDA meeting in March with U.S. regulators, investors are weighing odds. "I think our chances are reasonably good," the firm's chief medical officer, Daniel Levitt, told BioWorld Insight. "We're certainly going in optimistic," since data suggest that the compound is as safe and effective "or better than any treatment that was being used at the time we did the study."
Cytokinetics Inc. kept mum on the eventual sales force for omecamtiv mecarbil, possible changes in the Royalty Pharma deal's assigned percentage, and an additional safety trial with the phase III candidate, as well as potential reimbursement from Amgen Inc. for "certain activities" in co-promoting.
"We're doing the bold thing" by going for phase III superiority [rather than non-inferiority] vs. standard of care with an antibiotic in Clostridium difficile (C. diff) infection, Summit Therapeutics plc CEO Glyn Edwards told BioWorld Today, and it's going to cost about $100 million.
Science stands "at the very beginning of understanding the complexity" of the gut microbiome, Synthetic Biologics Inc. CEO Jeffrey Riley told BioWorld Today, noting that his company engages in "more traditional drug discovery and development vs. what Seres [Therapeutics Inc.] is doing, which is more on the cutting edge" – an edge where complexity can take its toll. And did.
After one analyst earlier this month referred to Calithera Biosciences Inc.'s benefit from "pairing promiscuously" with the lead candidate in its pipeline, the company matched efforts in a different way with Incyte Corp. on an early stage prospect, and stands to collect not only $45 million up front and an $8 million equity investment but also as much as $430 million in milestone payments.
Novan Inc.'s effort to market the first-ever topical nitric oxide (NO) macromolecule – one that would be the first new chemical entity for acne in decades – stumbled on word that topical SB2014 met with success in one phase III trial but only a partial win in the second, identical study.
"Everything we're seeing now leads us to believe that this is going to be a sustainable effect," Asterias Biotherapeutics Inc. CEO Steve Cartt told BioWorld Today, talking about positive results from the ongoing SCiStar phase I/IIa trial with AST-OPC1 (oligodendrocyte progenitor cells) in patients rated to have complete cervical spinal cord injury (SCI) according to the American Spinal Injury Association Impairment Scale, referred to as the AIS-A category.
Missing the secondary endpoint in the phase III trial with NBI-98854 did little to dampen enthusiasm for Neurocrine Biosciences Inc.'s small-molecule vesicular monoamine transporter 2 (VMAT2) inhibitor against tardive dyskinesia (TD), which achieved a resounding tackle of the primary endpoint: change from baseline in the Abnormal Involuntary Movement Scale (AIMS) at the sixth week in the 80 mg once-daily dosing group compared to placebo, as judged by blinded video raters.
