The Royal Veterinary College has presented drug conjugates comprising hyaluronan (HA) synthesis inhibitors covalently bonded to betaine reported to be useful for the treatment of chondrosarcoma and osteoarthritis.
Following a research program to identify inhibitors of protein kinases TBK1 and IKK-ε, Domainex Ltd. has nominated DMXD-011 as a preclinical drug candidate.
Wellstat Therapeutics Corp. has described compounds for co-delivery of uridine and ketoleucine with high bioavailability reported to be useful for the treatment of muscle atrophy, sarcopenia and cachexia.
Gilead Sciences Inc. has described thienopyrrole compounds acting as Toll-like receptor 7 (TLR7) and/or TLR8 antagonists reported to be useful for the treatment of systemic lupus erythematosus, cutaneous lupus erythematosus, lupus nephritis and inflammatory disorders, among other disorders.
What a difference a U.S. FDA advisory committee meeting can make. In the wake of the Peripheral and Central Nervous System Drugs Advisory Committee voting 7-2 Sept. 7 to recommend approval of Amylyx Pharmaceuticals Inc.’s amyotrophic lateral sclerosis (ALS) candidate, shares of the Cambridge, Mass.-based company (NASDAQ:AMLX) more than regained the value they lost in March when the same committee voted against approval of AMX-0035.
Amylyx Pharmaceuticals Inc.’s amyotrophic lateral sclerosis candidate, AMX-0035, will get a rare second bite at the adcom apple Sept. 7. This time around, the Cambridge, Mass.-based company is looking to improve on its first performance by stressing the survival benefit of its drug.
Structure-guided design for carbazole antagonists of Toll-like receptors 7 and 8 (TLR7/8) for the potential treatment of autoimmune disorders was reported by Bristol Myers Squibb Co. TLR activation can induce proinflammatory pathway activation, which has been identified in patients with lupus.
Researchers from BeiGene presented data on the discovery of highly selective covalent tyrosine-protein kinase BTK inhibitors for the potential treatment of B-cell malignancies and autoimmune disorders.
“No good data goes unpunished in this market,” H.C. Wainwright analyst Andrew Fein wryly noted in an Aug. 17 research report highlighting Wall Street’s dismal response to Blueprint Medicines Corp.’s positive top-line readout of the registrational Pioneer study, in which KIT inhibitor Ayvakit (avapritinib) met the primary and all key secondary endpoints in patients with non-advanced systemic mastocytosis.
Kringle Pharma Inc.’s phase II trial evaluating its recombinant human hepatocyte growth factor ligand, oremepermin alfa, failed to meet both primary and secondary endpoints in a study of its potential to help people with amyotrophic lateral sclerosis (ALS).
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