With positive initial phase I/II data in hand from two trials, Dyne Therapeutics Inc. plans to report more findings and start enrolling registrational cohorts in both studies by the end of this year for DYNE-101 in myotonic dystrophy type 1 (DM1) and a study called Deliver with DYNE-251 in Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping.
Jiangsu Atom Bioscience and Pharmaceutical Co. Ltd. has received FDA clearance of its IND application for a phase I trial in the U.S. of ABP-745, an anti-inflammatory oral small-molecule drug, for the treatment of acute gout.
Aurinia Pharmaceuticals Inc. has submitted an IND application to the FDA for AUR-200, a potential next-generation therapy for B-cell-mediated autoimmune diseases.
Dice Alpha Inc. has disclosed IL-17A inhibitors reported to be useful for the treatment of psoriasis, radiographic axial spondyloarthritis (ankylosing spondylitis), hidradenitis suppurativa, spondyloarthritis, psoriatic and rheumatoid arthritis.
Rheumatoid arthritis (RA) is a systemic autoimmune disorder where Bruton tyrosine kinase (BTK) and Janus kinase 3 (JAK3) are among the most relevant signaling pathways involved.
Wall Street’s measure of how Cogent Biosciences Inc.’s KIT D816V inhibitor bezuclastinib (often shortened to bezu) might fare in mastocytosis against U.S. FDA cleared Ayvakit (avapritinib), the tyrosine kinase inhibitor from Blueprint Medicine Corp., caused the former’s stock (NASDAQ:COGT) to tumble, closing Dec. 11 at $4.06, down $4.58, or 53%. Data from the ongoing phase II Summit trial testing bezu in non-advanced systemic mastocytosis rolled out during the American Society of Hematology (ASH) meeting in San Diego. Waltham, Mass.-based Cogent’s prospect turned up a rapid and continuing improvement in patient symptoms, with a 57% median best improvement on Mast Cell Quality-of-Life.
Shanghai Meiyue Biotech Development Co. Ltd. has identified MAPK p38 inhibitors reported to be useful for the treatment of cancer, arthritis, psoriasis, systemic lupus erythematosus, diabetes, atherosclerosis and Alzheimer’s disease.
Regenerative medicine company Mesoblast Ltd. plans to raise AU$97 million (US$64.5 million) to conduct additional phase III registration trials for its allogeneic stem cell treatment for steroid-refractory acute graft-vs.-host disease and for chronic back pain, as required by the U.S. FDA.
A multi-institutional research team has suggested that aberrant TDP-43 processing of the pre-mRNA of a microtubule-associated protein, stathmin-2 (STMN2), may be the primary contributor to amyotrophic lateral sclerosis (ALS).
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