On the regulatory front Friday, one pain drug company, Trevena Inc., lost and another, Acelrx Pharmaceuticals Inc., won, as the FDA – just for good measure – also cleared a biosimilar product from Coherus Biosciences Inc. in a busy afternoon of notifications.
As expected, Dublin-based Alkermes plc encountered rough waters in the joint meeting of the FDA's Psychopharmacologic Drugs Advisory Committee and Drug Safety and Risk Management Advisory Committee, which deliberated over ALKS-5461 sublingual tablets for the adjunctive treatment of major depressive disorder (MDD).
Tango Therapeutics Inc.'s immuno-oncology (I-O) deal with Gilead Sciences Inc. brings "substantial preclinical milestone [payments], substantial enough that they move the needle for us on our financing timeline," CEO Barbara Weber told BioWorld.
Alkermes plc's decision – against the advice of the FDA – to use an abbreviated, six-item version of the 10-item Montgomery-Asberg Depression Rating Scale (MADRS) in Study 207, one of the main trials with ALKS-5461 (buprenorphine/samidorphan), became a bone of contention in briefing documents made public ahead of Thursday's adcom meeting.
Although a few skeptics remain, Esperion Inc.'s top-line data from its pivotal phase III study known as Study 2, or 1002-047, with bempedoic acid (BA) for lowering LDL-C seemed to lay mostly to rest the worries brought on by five neoplasm (lung cancer) deaths in Study 1. "When we think back to May [when data from Study 1 with BA were disclosed], the issue was really: What is the perceived meaningfulness of what we called and still call the spurious result?" CEO Timothy Mayleben told BioWorld.
In the company's second program with UX-007 (triheptanoin, or trihep), Ultragenyx Pharmaceutical Inc.'s data are "much more robust" – enough so that the FDA recently said the firm could file an NDA based on phase II findings, spokesperson Danielle Keatley told BioWorld.
Clementia Pharmaceuticals Inc. delighted Wall Street with news that the company would file an NDA in the second half of 2019 – a full year earlier than previously planned – based on data from the completed phase II trial with its lead candidate, oral retinoic acid receptor gamma agonist palovarotene in the ultra-rare bone disease fibrodysplasia ossificans progressiva (FOP).
Stoke Therapeutics Inc. CEO Edward Kaye told BioWorld that the company cranked its quest "into turbocharge mode, once we had the animal data [in June] that suggest we were having some pretty significant efficacy results," and clinical work is expected to begin by 2020.
When The Medicines Co. (MDCO) gained FDA approval in August 2017 for the antibiotic Vabomere (meropenem and vaborbactam), the win provided clear validation of the approach taken by new company Qpex Biopharma Inc., which has pulled down $33 million in series A money.
A spokesperson for Merrimack Pharmaceuticals Inc. said the company had little to add to its press release disclosing the halt of the phase II study with MM-121, but a pipeline review was promised during the third-quarter earnings call next month.
