Researchers have defined an amyotrophic lateral sclerosis (ALS) reversal phenotype as having an initial diagnosis of ALS but subsequently showing a progressive and sustained clinical improvement, based on an unusual case they found.
Voyager Therapeutics Inc. has selected a lead development candidate for its superoxide dismutase 1 (SOD1)-mutated amyotrophic lateral sclerosis (ALS) gene therapy program. The company anticipates filing an IND application for the candidate with the FDA in mid-2025.
Leukotriene B4 (LTB4) stimulates the release of cytokines and mediators that participate in tissue inflammation and acts as a chemoattractant for neutrophils, eosinophils and monocytes at the inflammation site.
Bsense Bio Therapeutics Ltd. has nominated BSEN-760, a dual Kv7.2/3 and TRPV1 ion channel modulator, as a clinical candidate for the treatment of chronic pain.
Rhythm Pharmaceuticals Inc. has presented preclinical data on its new drug candidate, RM-718, a next-generation melanocortin MC4 receptor (MC4R) agonist for weekly administration with a potential development path for hypothalamic obesity.
At the recent American College of Neuropsychopharmacology meeting, Trevena Inc. presented preclinical data for the novel selective sphingosine 1-phosphate S1P1 receptor modulator TRV-045, being developed for the treatment of chemotherapy-induced peripheral neuropathy (CIPN).
Simcere Pharmaceutical Group Ltd.’s subsidiary Simcere Zaiming Pharmaceutical Co. Ltd. has received FDA clearance of an IND application for SIM-0501, an oral small-molecule inhibitor of ubiquitin-specific peptidase 1 (USP1), for advanced solid tumors.
Voronoi and Voronoi Bio Inc. have patented heteroaryl derivative compounds acting as serine/threonine-protein kinase B-raf (BRAF) inhibitors and reported to be useful for the treatment of cancer.
Phosphatidylinositol 3-kinase α (PI3Kα) (H1047R mutant) inhibitors are described in a Relay Therapeutics Inc. patent as potentially useful for the treatment of cancer, particularly, breast and ovarian cancer.