As the Feb. 28, 2023 PDUFA date for the compound nears, Cytokinetics Inc. CEO Robert Blum insisted that his firm is not mulling withdrawal of the marketing application for heart failure drug omecamtiv mecarbil, nor is the company now considering another study, after an unfavorable vote on the drug Dec. 13 by the U.S. FDA’s Cardiovascular and Renal Drugs Advisory Committee. Read More
A preliminary assessment of Avidity Biosciences Inc.’s phase I/II study of AOC-1001 in myotonic dystrophy type 1 (DM1) showed the first-ever targeted delivery of RNA into muscle, an area previously untreatable with existing RNA therapeutics. Sarah Boyce, Avidity’s CEO, said in a Dec. 14 call that the antibody oligonucleotide conjugate’s data were unprecedented in the RNA space and in myotonic dystrophy type 1 (DM1), labeling it a “revolutionary advancement.” Read More
The U.K. is taking the next step in applying genomics to health care with the launch of a £105 million (US$130 million) project that will sequence the whole genomes of 100,000 healthy newborn babies to detect rare genetic conditions. Read More
Pediatrix Therapeutics Inc. licensed exclusive China rights to Aclaris Therapeutics Inc.'s ATI-1777 for investigational “soft” JAK 1/3 inhibitor, for diseases including atopic dermatitis, in exchange for an up-front payment of $5 million and up to $91 million in milestones. Read More
Unlike amphibians, mammals do not regenerate appendages. Except when they do. “If you amputate one of the branches off of the antler [of a reindeer], it will also regenerate,” Jeff Biernaskie told BioWorld. Even without amputation, the antlers of both male and female reindeer regenerate annually, including their skin. That regeneration is “the only large mammal model of true skin regeneration,” he said. Read More
As largely expected, Mirati Therapeutics Inc.’s adagrasib gained U.S. FDA accelerated approval ahead of its Dec. 14 PDUFA date, cleared for second-line use in patients with non-small-cell lung cancer (NSCLC) harboring the KRAS G12C mutation, in which it will go up ahead Amgen Inc.’s Lumakras (sotorasib), which has the advantage of a year and a half head start. Read More
Given all the advances that have been made over the past decade, the U.S. FDA decided to issue a new draft guidance on developing antibacterial drugs to treat pulmonary tuberculosis (TB) rather than finalize the draft it issued in 2013. In releasing the new draft, the agency cited advancements made in nonclinical models, streamlined clinical development programs and growing interest in treatment-shortening combination regimens. Read More
Clinical updates, including trial initiations, enrollment status and data readouts and publications: 89bio, Aldeyra, Arbutus, Ascletis, Crispr, Curis, Immix, Indaptus, Inhibikase, Janssen, Maia, Meiragtx, Moderna, Precigen, Tessa. Read More
Biopharma happenings, including deals and partnerships, grants, preclinical data and other news in brief: Abbvie, Bioxytran, Eva, Exicure, Harrow Ipsen, Lilly. Read More
Regulatory snapshots, including global drug submissions and approvals, clinical trial approvals and other regulatory decisions and designations: 1st, AB, Boehringer, Carina, Cytokinetics, Erasca, Eyenovia, Mersana, Mirati, Mirum, Myrtelle, NMD, Panbela, Pharmajet, Remegen, Virpax. Read More
