Less than a year after landing the first gene therapy approval in the U.S. and just months after licensing certain rights to another, Novartis AG is making yet further bets on the field with an $8.7 billion offer to buy Chicago-based Avexis Inc., a company developing a gene therapy for spinal muscular atrophy (SMA). Read More
Despite two positive phase II trials showing serlopitant helped patients with pruritus –itchy skin – Menlo Therapeutics Inc. couldn't go three for three at the proof-of-concept plate, failing to show an effect on pruritus in patients with a history of atopic dermatitis. Read More
Verastem Inc. CEO Robert Forrester told BioWorld that the company seeks to share the upper atmosphere's "rarefied air" with small-to-mid cap biotechs that have reached the market with new oncology drugs to fly the same skies as big pharma. "These things don't happen very often," he noted. "If we are fortunate, we will be the 11th" such smaller firm since 2010 to win clearance for a promising cancer compound. Read More
LONDON – AMO Pharma Ltd. is laying plans, both clinical and financial, for a phase III study of its lead program, AMO-02 (tideglusib), after reporting final data from a phase IIb proof-of-concept trial in the treatment of congenital and childhood onset myotonic dystrophy type 1. Read More
HONG KONG – The Trump administration has proposed placing a 25 percent tariff on more than 1,300 imported goods from China, such as biotech materials and medical devices, a move some say could eventually hurt U.S. drugmakers. Read More
Shares of Therapix Biosciences Ltd. gained 24 percent Monday on top-line results from a phase IIa study showing that THX-110 significantly improved symptoms over time in adults with Tourette syndrome. The Tel Aviv, Israel-based firm plans to launch a larger, randomized, double-blind trial to test the cannabinoid product, which combines dronabinol and palmitoylethanolamide. Read More
Rxi Pharmaceuticals Corp., of Marlborough, Mass., entered agreements with investors for the purchase of about 1.5 million shares of common stock at $3.24375 apiece for gross proceeds of about $4.9 million in a registered direct offering priced at the market. Read More
U.S. Rep. Frank Pallone (D-N.J.), ranking member of the House Energy and Commerce Committee, is taking the Centers for Medicare & Medicaid Services (CMS) to task for not updating its drug spending dashboards since Donald Trump became president. Read More
Ionis Pharmaceuticals Inc., of Carlsbad, Calif., said it licensed IONIS-AZ6-2.5-LRx, or AZD-2693, to Cambridge, U.K.-based Astrazeneca plc now that the drug has advanced into development. Read More
