Scirhom GmbH has submitted a clinical trial application (CTA) for its lead candidate, SR-878, an antibody designed to target inactive rhomboid protein 2 (iRhom2) as a therapeutic strategy for numerous autoimmune disorders.
There is increasing evidence on the involvement of Toll-like receptor 7 (TLR7) in the pathogenesis of systemic lupus erythematosus (SLE). At the recent American College of Rheumatology meeting, researchers from Daiichi Sankyo Co. Ltd. presented preclinical data on DS-7011a, a TLR7 antagonist antibody with for the potential treatment of SLE.
Reneo Pharmaceuticals Inc.’s last patient visit Oct. 9 in the pivotal study with mavodelpar called Stride – due to yield top-line data in December 2023 – kept interest strong in primary mitochondrial myopathy (PMM), where parties are pursuing development bids by way of assorted strategies.
Electrical stimulation of the spinal cord has restored the ability to walk normally to a patient whose chronic Parkinson’s disease (PD) had led to severe motor deficits, researchers reported in the Nov. 6, 2023, online issue of Nature Medicine.
Angitia Biopharmaceuticals raised $46 million in a series B round extension, bringing the total raised under the series B to $170 million led by Morningside Group and will enable the company to advance its pipeline of musculoskeletal therapies.
Failing to meet the primary endpoint in its confirmatory phase III Embark trial, Sarepta Therapeutics Inc.’s gene therapy, Elevidys (delandistrogene moxeparvovec), which received accelerated approval in June and was priced at $3.2 million, has one of three fates in its future, all of which are dependent on how the U.S. FDA perceives the data. Based on secondary endpoints showing statistical significance and a recent positive meeting with the agency, Sarepta could continue to market Elevidys under its current label for 4- and 5-year-old ambulatory Duchenne muscular dystrophy (DMD) patients; Sarepta is filing the postmarketing requirement needed to transition from accelerated to full approval.
Vyne Therapeutics Inc. has released promising new preclinical data on its oral small-molecule BD2-selective BET inhibitor, VYN-202, in preclinical models of psoriasis and rheumatoid arthritis. In a well-established model, a psoriasis phenotype was induced in BALB-C mice, and treatment was administered intraperitoneally with either VYN-202, the allosteric TYK2 inhibitor Sotykto (deucravacitinib, Bristol Myers Squibb Co.) or placebo.