Pepgen Inc. seems to have gained a leg up on competitors in early data with PGN-EDODM1 in myotonic dystrophy type 1 (DM1), and shares of the Boston-based firm (NASDAQ:PEPG) closed Feb. 24 at $2.29, up 92 cents, or about 67%. The company unveiled initial positive data from the 5- and 10-mg/kg dose cohorts in the ongoing Freedom-DM1 phase I study with PGN-EDODM1, which deploys Boston-based Pepgen’s Enhanced Delivery Oligonucleotide technology to deliver a therapeutic oligonucleotide that is designed to restore the normal function of MBNL1, a key RNA splicing protein.
Solid Biosciences Inc. is preparing for a sit-down with the U.S. FDA this year to discuss the firm’s results with the next-generation gene therapy SGT-003 for Duchenne muscular dystrophy (DMD).
F. Hoffmann-La Roche Ltd. and Hoffmann-La Roche Inc. have identified new benzimidazoles acting as serine/threonine-protein kinase SIK inhibitors reported to be useful for the treatment of rheumatoid arthritis, metabolic dysfunction-associated steatohepatitis (MASH), giant cell arteritis, primary sclerosing cholangitis, inflammatory bowel disease, atherosclerosis, type 2 diabetes and glomerulonephritis, among others.
Bristol Myers Squibb Co. has patented new protein-arginine deiminase type-4 (PADI4; PAD4) inhibitors reported to be useful for the treatment of rheumatoid arthritis, among others.
Alterity Therapeutics Ltd. reported positive top-line phase II results for lead candidate ATH-434 for treating multiple system atrophy, a rare neurological disorder similar to Parkinson's disease.
The first successful phase II trial in Duchenne muscular dystrophy (DMD) heart disease rolled out from Cumberland Pharmaceuticals Inc., which said top-line findings from the experiment called Fight DMD showed promising results in the indication that represents the main cause of death for such patients.
Glucocorticoid-induced osteoporosis, the leading cause of secondary osteoporosis, is characterized by diminished bone density and compromised osteoblast function. As current treatment options often have significant side effects, researchers are actively seeking new drug candidates to improve patient outcomes.
Alterity Therapeutics Ltd. reported positive top-line phase II results for lead candidate ATH-434 for treating multiple system atrophy, a rare neurological disorder similar to Parkinson's disease.
Positive data for Veru Inc.’s enobosarm lend more weight to the potential progress of the company’s body-mass preservation program in patients taking Wegovy (semaglutide). The side effect of lean mass loss has dogged those taking GLP-1s. The study results didn’t support the company’s stock on the day of the data release as it had the previous four weeks.
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