Merck & Co. Inc. has tapped Skyhawk Therapeutics Inc. for its expertise in the discovery and development of small molecules that modulate RNA splicing, agreeing to pay it up to $600 million per program target plus royalties on sales of any commercialized products of the collaboration. The deal, focused on potential treatments for certain neurological diseases and cancer, was accompanied by news of an expansion of Skyhawk's collaboration with Biogen Inc., which originally signed on with the Waltham, Mass-based company in January.
Despite a February adcom meeting urging the agency to wait for more data, the FDA has approved Karyopharm Therapeutics Inc.'s selinexor, in combination with dexamethasone (dex), as a new treatment for certain adults with relapsed refractory multiple myeloma (MM). The approval covers patients who have received at least four prior therapies and whose disease is resistant to several other forms of treatment, including at least two proteasome inhibitors, at least two immunomodulatory agents and an anti-CD38 monoclonal antibody.
Adding I.V. sildenafil – the active ingredient in Viagra – to inhaled nitric oxide (iNO) therapy for newborns with persistent pulmonary hypertension (PPHN) failed to achieve a statistically significant reduction in treatment failure rates or time on iNO vs. iNO alone in the first part of a phase III study sponsored by Pfizer Inc.
Months sooner than expected, Zogenix Inc. said it's planning to resubmit an NDA for Fintepla (ZX-008, fenfluramine hydrochloride), a drug for seizures associated with Dravet syndrome (DS) that received a refusal to file (RTF) letter from the FDA in April.
Minovia Therapeutics Ltd., an Israeli company running the first ever cell therapy trial to treat a mitochondrial disease, has dosed the first participant in a phase I/II study of a treatment for the rare pediatric condition Pearson syndrome. A second patient will be dosed in three weeks. Five of the seven patients that will ultimately participate in the trial have already been recruited, fueling hope that the study will be completed by year-end or earlier.
SAN FRANCISCO – New research presented at the annual meeting of the American Society of Microbiology has shown that pure cannabidiol (CBD), the main nonpsychoactive chemical compound of cannabis and hemp, can rapidly kill gram-positive bacteria. Evidence of the effect has put clinical testing of BTX-1801, a potential therapy for serious skin infections under development at Philadelphia-based Botanix Pharmaceuticals Ltd., on track to enter the clinic later this year.
Shares of Acer Therapeutics Inc. (NASDAQ:ACER) fell 78.6% to $4.12 Tuesday after the FDA issued a complete response letter (CRL)calling for the company to conduct an "adequate and well-controlled trial" to determine whether the orphan-designated beta-blocker Edsivo (celiprolol) reduces the risk of clinical events in patients with vascular Ehlers-Danlos syndrome (vEDS), an inherited connective tissue disorder.
SAN FRANCISCO – New research presented at the annual meeting of the American Society of Microbiology has shown that pure cannabidiol (CBD), the main nonpsychoactive chemical compound of cannabis and hemp, can rapidly kill gram-positive bacteria.
New clinical data on Krystal Biotech Inc.'s investigational gene therapy for dystrophic epidermolysis bullosa (DEB), KB-103, found five of six wounds treated with the topical candidate closed completely during a phase II trial and, along with wounds investigators treated in phase I, have stayed closed so far.
A tumultuous stretch of active trading in Contravir Pharmaceuticals Inc. continued Friday as shares (NASDAQ:CTRV) climbed 21.2% to $5.20 on news of positive FDA feedback regarding a pre-IND package for CRV-431 in nonalcoholic steatohepatitis (NASH).
