Cytokinetics Inc. was regrouping after an unfavorable vote by the U.S. FDA’s Cardiovascular and Renal Drugs Advisory Committee CRDAC on the heart failure (HF) drug, omecamtiv mecarbil. Only three CRDAC members voted that the drug’s benefits outweighed its risks for the treatment of HF in patients with reduced ejection fraction. The other eight panelists turned thumbs down on the profile, with some pointing out that findings available seem to work for and against the drug. CEO Robert Blum cited the already “long and winding road” with omecamtiv, and said during a conference call with investors the day after the adcom that “the next few months leading into the [Feb. 28] PDUFA data will be telling.” Shares of the firm (NASDAQ:CYTK) were trading midday at $40.70, up $2.37.

Avidity muscles into positive phase I/II data with lead candidate AOC-1001

While a partial clinical hold remains on new participant enrollment in Avidity Biosciences Inc.’s phase I/II study of AOC-1001 in myotonic dystrophy type 1 (DM1), the company has released positive data from the trial. A preliminary assessment showed the first-ever targeted delivery of RNA into muscle, an area that has been untreatable with existing RNA therapeutics, according to the company. In September, a serious adverse event (SAE) in one participant led the U.S. FDA to place the partial clinical hold on the study of the antibody oligonucleotide conjugate in treating DM1, the most common form of muscular dystrophy in adults. Remaining participants continue in their current dosing cohort as the company investigates the SAE. Avidity’s stock (NASDAQ:RNA) had risen a powerful 55% to about $17 per share in midday trading.

UK newborn genome project targeting rare disease backed by £105M

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. The project will screen for 200 conditions, which are all very hard to diagnose, but for which there are treatments. The aim is to end the diagnostic odyssey which families endure and, by treating pre-emptively, prevent the developmental damage caused during the average five years it takes to diagnose rare diseases.

Pediatrix in-licenses atopic dermatitis candidate from Aclaris 

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. The deal gives Shanghai-based Pediatrix rights to develop, manufacture and commercialize ATI-1777 in greater China, which includes mainland China, Hong Kong, Macau and Taiwan. “As a ‘soft’ JAK 1/3 inhibitor, ATI-1777 could provide patients with atopic dermatitis with a new therapeutic option that minimizes the potential for systemic toxicities,” said Cheng Zhang, CEO of Pediatrix.

Path to scarless healing could be among the gifts reindeer bring

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. Biernaskie and his colleagues have taken advantage of the difference between antler skin, also called velvet, and the skin on their bodies, which forms typical mammalian scars after an injury, to gain new insights into how velvet retains its regenerative capacity.

US FDA starts afresh with TB drug guidance

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 guidance, the agency cited advancements made in nonclinical models, streamlined clinical development programs and growing interest in treatment-shortening combination regimens.

Also in the news

1st Biotherapeutics, 89bio, Abbvie, Aldeyra, Alpha-9, ALX Oncology, Arbutus, Ascentage, Ascletis, Astex, Astraxeneca, Atara, Avencell, Bioxytran, Boehringer Ingelheim, Carina, Carocell, Cellpoint, Coimmune, Corvus, Crispr, Curis, Disc Medicine, Eli Lilly, Elixirgen, Erasca, Etcembly, Eva, Exicure, Eyenovia, Fuji Yakuhin, Galapagos, Genentech, Geron, Glycomimetics, Gracell, GRI, GSK, Harpoon, Harrow, Imago, Immix, Immunogen, In8bio, Incyte, Indaptus, Inhibikase, Innate, Innovent, Ipsen, Janssen, Junshi, JW, Kaleibe, Karolinska, Karyopharm, Keros, Kite, Kura, Lava, Maat, Magenta, Maia, MD Anderson, Meiragtx, Mendus, Merck, Metafora, Mirati, Mirum, Moderna, Montai Health, Nektar, Nexcella, Nimble, NLS, Nurix, Oncopeptides, Oncternal, Orca, Orchard, Oryzon, Pfizer, Phoremost, Poolbeg, Praxis, Precigen, Prothena, Psyence, Qrons, Qu, Regeneron, Remegen, Replay, Roche, Rocket, Ryvu, Salarius, Seagen, Selecta, Sellas, Servier, Starpharma, Storm, Sumitomo, Sutro, Syndax, Syntrix, Syros, Taiho, Tessa, Thinkcyte, Treadwell, Tris, Tscan, UCB, Urica, Vallon, Vanda, Vincerx, Wugen , X4, Xencor