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NEW YORK – Thousands of failed cancer trials and only a smattering of cancer drug approvals prompted the FDA to adopt draft guidance in 2010 to allow for adaptive study designs and, while a number of studies such as the public-private Lung-MAP study are ongoing, still more work needs to be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.
Vertex Pharmaceuticals Inc.'s fourth quarter earnings boasted few surprises – the company had already given investors a taste at the J.P. Morgan Healthcare Conference earlier this month – but that didn't stop the Wednesday evening earnings call from running well over an hour as analysts persisted in teasing out management predictions for uptake of cystic fibrosis (CF) drug Orkambi.
The good news is that Biogen Inc.'s efforts last year to regroup after slumping sales of multiple sclerosis drug Tecfidera (dimethyl fumarate) resulted in a dismal first half of 2015 appear to be working.
Medivation Inc. hit a little snag on the way to its pivotal phase II study testing supposed PD-1 inhibitor MDV9300 (pidilizumab) in relapsed or refractory diffuse large B-cell lymphoma (DLBCL) when the company concluded the drug does not actually inhibit PD-1 after all.
SAN FRANCISCO – As he began his J.P. Morgan Healthcare Conference presentation, Bluebird Bio Inc. CEO Nick Leschly added to the usual disclosures by priming attendees for "a tremendous amount" of forward-looking statements, something that has probably become standard operating procedure in the gene therapy space in recent years, a lesson Bluebird knows well.
SAN FRANCISCO – Though a decision on Duchenne muscular dystrophy (DMD) drug Kyndrisa (drisapersen) is due any day now, Biomarin Pharmaceutical Inc. executives played down the U.S. market opportunity during their J.P. Morgan Healthcare Conference presentation, focusing instead on the market potential of the drug in Europe and highlighting the remainder of the pipeline, including presenting new phase II data for Pompe disease drug reveglucosidase alfa.
