Jazz Pharmaceuticals plc has disclosed macrocyclic orexin OX2 receptor agonists reported to be useful for the treatment of hypersomnia, narcolepsy and sleep disorders.
Sudo Biosciences Inc. has closed a $116 million series B financing, with the funding raised to be used to advance two investigational TYK2 candidates into the clinic in 2024.
Iama Therapeutics Srl has obtained clinical trial application (CTA) clearance from the Italian Medicines Agency (AIFA) to initiate a first-in-human phase I study of IAMA-6, an orally administered small-molecule therapeutic targeting NKCC1 for the treatment of autism and epilepsy.
NZP UK Ltd. has divulged 3-azasteroid compounds reported to be useful for the treatment of myalgic encephalomyelitis/chronic fatigue syndrome, acute radiation syndrome and neurodegenerative disorders.
Innovstone Therapeutics Ltd. has synthesized aromatic heterocyclic compounds acting as leucine-rich repeat kinase 2 (LRRK2; Dardarin) inhibitors reported to be useful for the treatment of Parkinson’s disease.
On-again, off-again investor enthusiasm for Uniqure NV’s Huntington’s disease (HD) gene therapy AMT-130 got another boost as the company followed this summer’s news from phase I/II trials with additional interim data. Shares of Uniqure (NASDAQ:QURE) closed Dec. 19 at $6.64, down $1.34, or 17%, as the company offered results on up to 30 months of follow-up from 39 patients enrolled in the ongoing U.S. and European experiment.
Neuren Pharmaceuticals Ltd. plans to progress NNZ-2591 to phase III trials following positive top-line phase II results in children with Phelan-McDermid syndrome, a genetic neurodevelopmental disorder for which there are no approved treatments.
Researchers from Engrail Therapeutics Inc. presented preclinical data for the novel D2/3 receptor antagonist ENX-104, being developed for the treatment of depression.
Researchers from Shandong University reported on the preclinical characterization of A3B3C-1, a multifunctional agent for the treatment of Alzheimer’s disease (AD), taking into account its complex nature. With multiple processes involved in its pathogenesis, the exploration of multi-target drugs may fit to address the combination of pathological factors in AD.
Neuren Pharmaceuticals Ltd. plans to progress NNZ-2591 to phase III trials following positive top-line phase II results in children with Phelan-McDermid syndrome, a genetic neurodevelopmental disorder for which there are no approved treatments.
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