After two decades of work with Translarna (ataluren) for nonsense mutation Duchenne muscular dystrophy, PTC Therapeutics Inc. withdrew its bid for approval of the compound upon hearing from the U.S. FDA. Regulators told Warren, N.J.-based PTC that the data package for Translarna is “unlikely to meet the agency's threshold of substantial evidence of effectiveness.”
Bridgebio Pharma Inc. kept the phase III wins coming, this time with positive top-line results from Propel 3, the global phase III pivotal study of oral infigratinib, designed to inhibit FGFR3 signaling in children with achondroplasia.
Upstream Bio Inc.’s favorable top-line results from the phase II Valiant trial testing verekitug in adults with severe asthma were not enough to charm Wall Street, and shares of the firm (NASDAQ:UPB) closed Feb. 11 at $14.69, down $13.12, or 47%.
Moderna Inc. blamed a switcheroo by the U.S. FDA for the refusal-to-file (RTF) letter on the seasonal influenza vaccine mRNA-1010. Shares of the firm (NASDAQ:MRNA) closed Feb. 11 at $40.51, down $1.49, having traded as low as $36.66 as investors learned of the RTF letter, which Moderna said is “inconsistent with feedback” the company was given by regulators during pre-phase III as well as pre-BLA-submission talks.
Wall Street pushback against relaxin player Tectonic Therapeutic Inc. was sharp, but a trial design that differs could save the drug from the fate of Astrazeneca plc’s long-acting relaxin-2 analogue, AZD-3427, which the pharma giant has “removed from phase II,” where it was being tested in a trial called Re-phire, because of less than ideal efficacy.
Iambic Therapeutics Inc.’s multiyear technology and discovery pact with Takeda Pharmaceutical Co. Ltd. could help the San Diego-based firm advance its own pipeline in a big way.
Iambic Therapeutics Inc.’s multiyear technology and discovery pact with Takeda Pharmaceutical Co. Ltd. could help the San Diego-based firm advance its own pipeline in a big way.
Trumprx.gov launched with much fanfare late Feb. 5, and the online tool brings promise for the “world’s lowest prices” on prescription drugs. Alongside the website hoopla came word from Hims & Hers Health of its own steep discounts on what’s to be the compounded version of a Trumprx-featured therapy: oral Wegovy (semaglutide) for obesity. Novo Nordisk A/S, Wegovy’s originator, vowed to fight.
Amgen Inc.’s head of R&D James Bradner said his firm was “surprised” by the request from the U.S. FDA Jan. 16 to take Tavneos (avacopan) off the market – and the company said no. “We're in discussions with FDA, and we'll answer questions as we talk with them,” he added.
Another data cut from Ultragenyx Pharmaceutical Inc. regarding UX-111 (rebisufligene etisparvovec), an AAV9 gene therapy for type A Sanfilippo syndrome, continued to brighten the picture for the compound, recently taken under review again by the U.S. FDA.
