July 5, 2016
By Mark Terry, BioSpace.com Breaking News Staff
Drug development is not for the timid, and so far this year, biopharma’s big gambles are coming up empty. John Carroll, writing for Endpoints, discusses about 10 big failures or setbacks.
Boulder, Colorado-based Clovis Oncology is definitely embattled, and the biggest fault is the company’s lung cancer drug, rociletinib. On April 12, the U.S. Food and Drug Administration (FDA)’s independent panel voted 12 to 1 against accelerated approval for the drug, and told the FDA it should wait for late-stage trial results before making a decision. This pretty much trashed the company stock, which dropped as much as 18.7 percent on that date.
Carroll writes, “Currently under investigation by the feds, Clovis is now ramping up a new effort to back rucaparib, which finds itself in yet another late-stage horse race with serious competition. Failure here is not an option, but it’s a very real possibility.”
UK-based AstraZeneca probably has enough to worry about with the recent Brexit vote, but the problem it had this year was it paid $2.7 billion for ZS-9, then had the FDA issue a Complete Response Letter (CRL) because of problems with a pre-approval manufacturing inspection.
So that drug’s probably delayed until the end of this year or early 2018, while its competitor, Relypsa ’s Veltassa, was approved in October. Also, its CTLA-4 drug tremelimum failed a Phase IIb trial for mesothelioma recently.
San Rafael, California-based BioMarin Pharmaceutical has had a tough year. Most recently, on June 17, the company indicated it had temporarily suspended recruitment for a hemophilia A trial. That may or may not be critical, since it appears to be the result of a safety concerns related to a subset of the trial participants.
It was the January rejection by the FDA of Kyndrisa (drisasperson) for Duchenne muscular dystrophy (DMD) that was the biggest blow. After some soul-searching, the company not only ended that lead drug campaign, it killed three follow-up therapies it had in Phase II.
“The company’s investigators couldn’t get anywhere with their argument that there was a treatment strategy that could help boys affected by the lethal disease,” writes Carroll.
Dublin-based Alkermes had two trials for ALKS-5461 for the treatment of depression fail. Trials for behavioral and central nervous system disorders are notoriously difficult, which is why drug companies run so many of them for each drug.
Although the company may be down, it’s not quite out. Carroll writes, “The company came up with some post hoc reasons to believe they were still on to something and some analysts believe that a positive readout for the third study could yet salvage the situation.”
Infinity Pharmaceuticals , based in Cambridge, Massachusetts, announced on June 14 that its Phase II trial of develisib in refractory indolent non-Hodgkin lymphoma (iNHL) met its primary endpoint, but wasn’t strong enough to be better than current therapies. North Chicago-based AbbVie , as a result, terminated its collaboration deal to develop and commercialize duvelisib.
Hampton, New Jersey-based Celldex announced in March that its Rintega cancer vaccine failed to meet its primary endpoint and as a result the study was being terminated.
PTC Therapeutics , headquartered in South Plainfield, New Jersey, is in the odd position of actually having the FDA refuse to let them file an application for Translarna (ataluren) for DMD.
“Regulators have carved out a special status for experimental drugs that treat Duchenne, though,” writes Carroll. “That helps explain why the Europeans gave it a conditional approval two years ago and still have done nothing after the latest setback. … PTC may have thought that same generosity would extend to Washington, D.C. But that was wrong.”
