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.
Less than two weeks after getting a thumbs-up from the EMA’s Committee for Medicinal Products for Human Use, Santhera Pharmaceutical AG’s vamorolone secured U.S. FDA approval for use in patients, 2 and older, with Duchenne muscular dystrophy (DMD). A first-in-class drug, vamorolone, branded Agamree, is expected to offer a safer alternative to the steroid therapy, which the company has said will remain a foundational treatment of DMD, even with the introduction of gene therapies.
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.
Silo Pharma Inc. has announced promising data from a preclinical study investigating the binding affinity and optimization of SPU-21 liposomal joint homing peptide in human synovial tissue surrounding joints and tendons.
The EMA is standing firm on its refusal to recommend approval of the amyotrophic lateral sclerosis (ALS) treatment Albrioza in Europe after re-examining Amylyx Pharmaceuticals Inc.’s marketing authorization application and remaining unconvinced that the main study demonstrated the drug effectively slows disease progression.
It has been previously demonstrated that therapeutic inhibition of the prostaglandin E2 (PGE2)-degrading enzyme, 15-prostaglandin dehydrogenase (15-PGDH), was able to improve muscle strength in aged mice. Researchers from Epirium Bio Inc. have now reported the discovery and preclinical characterization an orally bioavailable small molecule inhibitor of 15-PGDH – MF-300 – being developed for the treatment of neuromuscular dysfunction.
Researchers from Immunophage Biomedical Co. Ltd. have published details on the discovery and preclinical evaluation of a new CD38 inhibitor being developed for the treatment of mitochondrial myopathy.
Researchers from East China Normal University and Shanghai Jiao Tong University presented the discovery and preclinical evaluation of new potent antiosteoporosis agents. Synthesis and optimization of a series of heterocyclic ring-fused derivatives of 20(S)-protopanaxadiol (PPD) led to the identification of SH-491 as the lead candidate with the most potent inhibitory effects on RANKL-induced osteoclastogenesis (IC50=11.8 nM).
Brainstorm Cell Therapeutics Inc. said it’s exploring all its options in the wake of a Sept. 27 U.S. FDA advisory committee vote, in which the committee overwhelmingly disagreed with the company that the data it presented supported the effectiveness of Nurown (debamestrocel) for the treatment of mild to moderate amyotrophic lateral sclerosis.
Brainstorm Cell Therapeutics Inc.’s Nurown got a thumbs down from the U.S. FDA’s Cellular, Tissue and Gene Therapies Advisory Committee Sept. 27, as the committee voted 1-17, with one abstention, that the data presented demonstrated substantial evidence of effectiveness for treatment of mild to moderate amyotrophic lateral sclerosis.
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