When Vitae Pharmaceuticals Inc. called a halt to enrollment in its phase IIa trial testing VTP-43742 in psoriasis with no explanation, investors naturally feared the worst, sending shares plummeting to a 52-week low earlier this month.
Regeneron Pharmaceuticals Inc. and partner Sanofi SA are hoping now to make their case in the Federal Circuit after a U.S. District Court jury ruled that patents covering Amgen Inc.'s competing PCSK9 antibody, Repatha (evolocumab), are valid, a decision that, if it stands, could put Regeneron and Sanofi on the hook for damages, including royalties from sales of Praluent (alirocumab).
Gensight Biologics SA launched a pair of phase III studies aimed at testing gene therapy candidate GS010's ability to preserve and restore vision in patients with blindness-causing Leber's hereditary optic neuropathy (LHON), a disease in which "time is of the essence," said Chairman and CEO Bernard Gilly.
Shares of Catalyst Pharmaceuticals Inc. tumbled 37.3 percent Wednesday after the Coral Gables, Fla-based firm disclosed a refusal to file (RTF) letter from the FDA for its new drug application (NDA) for rare disease drug Firdapse (amifampridine phosphate). While RTFs are infrequent and usually only delay – rather than derail – regulatory approvals, the timing of Catalyst's letter is interesting for a couple of reasons.
NEW YORK – Despite the appeal of shorter development timelines – and the potential of a multibillion-dollar market – ophthalmology drug development has lagged behind work in other disease areas in terms of new targets, biomarker development and even the number of players operating in the space.
NEW YORK – By the time the 18th annual BIO CEO & Investor Conference got under way on its second and last day, the issue of drug pricing vs. treatment value had already reared its head in nearly every panel. And, during a session convened Tuesday morning to tackle the topic head-on, industry experts said the debate is unlikely to fade away as it has in years past.
NEW YORK – Last year's big win by Biogen Inc. with Alzheimer's disease candidate aducanumab, the first potentially disease-modifying drug to hit its prespecified endpoint in a proof-of-concept study, restored optimism to a space that had been riddled with failure and reaffirmed faith in the amyloid beta (abeta) hypothesis – at least for some.
As the saga of Martin Shkreli continues to unfold in all its obnoxious glory, with every tweet and smirk signifying the former biopharma CEO’s apparent aspirations to a level of sheer jerkiness heretofore unseen on planet Earth, there’s one question that I – and probably a lot of other folks – can’t help but ask: Is this guy for real? I mean, seriously? From his gleefully unapologetic, 5,500-percent price hike for an in-licensed, generic drug, to his now-infamous gray-hoodied perp walk following his arrest on charges of securities fraud, to his juvenile Twitter taunts – not to mention his outlandish...
NEW YORK – Thousands of failed cancer trials and only a smattering of cancer drug approvals prompted the FDA to adopt draft guidance in 2010 to allow for adaptive study designs and, while a number of studies such as the public-private Lung-MAP study are ongoing, still more work needs to be done to improve the cost efficiency and success rate of oncology drug development. (See BioWorld Insight, July 28, 2014.)
Like travelers encountering an oasis after a spiritless trek through the desert, investors rushed Wednesday to shares of Chinese cancer drug developer Beigene Ltd. and Cambridge, Mass.-based genome-editing company Editas Medicine Inc. as they became the first two companies – in any sector – to price U.S. IPOs this year.
