Eagerly awaited data from Verastem Inc.'s phase III DUO study should "more than satisfy the skeptics," CEO Robert Forrester told BioWorld as the firm unveiled positive top-line data with duvelisib for relapsed or refractory chronic lymphocytic leukemia (CLL)/small lymphocytic lymphoma (SLL). "This drug, as we saw from the [earlier] DYNAMO study, is clearly active," he added, referring to a phase II experiment in non-Hodgkin's lymphoma (NHL), results from which investors deemed less than thrilling.
Nightstar Therapeutics Ltd. filed to raise about $86.2 million in an IPO to advance its gene therapy candidate, NSR-REP1, for choroideremia (CHM), due to enter phase III trials in the first half of next year.
With Insmed Inc.’s phase III Convert trial expected to read out any day, onlookers are busying themselves with guesswork about success for amikacin liposome inhalation suspension, formerly called Arikayce but now known by the acronym ALIS, in non-tuberculous mycobacterium (NTM) infection.
Immunogen Inc. has enough cash – almost – to reach the phase III readout with antibody-drug conjugate (ADC) mirvetuximab soravtansine (mirv) in ovarian cancer, thanks to the option deal for earlier-stage ADCs with Jazz Pharmaceuticals plc, which paid $75 million up front.
Calimmune Inc. CEO Louis Breton told BioWorld “a wave is about to crash on the shoreline” for technologies such as those that lured CSL Behring to the table for a buyout deal that included $91 million up front plus milestone payments of as much as $325 million.
Adamas Pharmaceuticals Inc.’s chief medical officer (CMO) Rajiv Patni told BioWorld that a full development program “just like one would do for a new chemical entity [NCE]” gets credit for the strong label – touting efficacy even during patients’ “off” times – for Gocovri (amantadine extended-release capsules), cleared by the FDA last week for levodopa-induced dyskinesia (LID) associated with Parkinson’s disease (PD).
Right on time, the FDA granted approval to Adamas Pharmaceuticals Inc.'s amantadine extended-release capsules (previously known as ADS-5102) for levodopa-induced dyskinesia (LID) associated with Parkinson's disease (PD). Marketing clearance was a prospect in which most onlookers, but not all, were confident ahead of the agency's edict.
Intra-Cellular Therapies Inc. vice president Kimberly Vanover told BioWorld that the company "should have everything we need" to file an NDA for lumateperone, also known as ITI-007, in the middle of next year, and the company's shares (NASDAQ:ITCI) closed Wednesday at $14.39, up $3.12, or 27.7 percent, on word that the FDA found adequate the company's responses to a request for more information regarding toxicology experiments in dogs.
Celularity Inc. raised “roughly in the $50 million range” by way of its series A round to boost cell and tissue regenerative therapies in cancer and other disorders, said Sorrento Therapeutics Inc. CEO Henry Ji, whose company is one of three involved in what amounts to a new business model. “Basically, the whole cell-therapy division of Celgene is coming back together into Celularity” as the end result, he told BioWorld.
Having promised to let Cardiome Pharma Corp. know about its NDA resubmission before the end of September, the FDA surprised the company by responding a month ahead, declaring the data package proposed for Brinavess (vernakalant hydrochloride) insufficient.
