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.
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.
In a global deal worth up to $770.5 million in up-front and milestone payments, Stevenage, U.K.-based Autifony Therapeutics Ltd. signed with Dublin-based Jazz Pharmaceuticals plc for two ion channel-targeting programs aimed at finding and developing new drugs for neurological disorders. Autifony will take selected candidates through preclinical development, while Jazz will cover clinical development, manufacturing, regulatory activities and commercialization.
Harmony Biosciences Holdings Inc.’s agreement to acquire Zynerba Pharmaceuticals Inc. for $60 million cash plus as much as $140 million in contingent value rights (CVRs) didn’t register as one of the larger potential outlays recently, but Wall Street had questions about the term sheet just the same. Plymouth Meeting, Pa.-based Harmony is paying about $1.10 per share in cash for Zynerba, of Devon, Pa., plus one non-tradeable CVR tied to clinical, regulatory and sales milestones.
A drug that Novartis AG discontinued in fragile X syndrome in 2014 after it failed in two phase II/III trials has been in-licensed by neuroscience specialist Stalicla SA, which plans to revive the prospects of the glutamate receptor antagonist by applying its precision medicine technology to identify likely responders.
Swiss-American startup Opna Bio SA launched this week with a $38 million series A, a Science paper on one of its targets and a pipeline stretching from preclinical to phase II.
Investigators at the École Polytechnique Fédérale de Lausanne (EPFL; Swiss Federal Institute of Technology Lausanne) have identified a broad role for the fragile X mental retardation protein (FMRP) in suppressing antitumor immunity, they reported in the Nov. 18, 2022, issue of Science. The results could lead to new ways to boost antitumor immunity. Scientifically, they also provide new insights into the link between tumors and the nervous system.
Mutations in FMR1, the gene that codes for FMRP, cause fragile X syndrome, a neurodevelopmental syndrome that is characterized by mental retardation and autism-like symptoms.Previous work in the laboratory of Douglas Hanahan, who is the senior author of the Science paper, as well as by other teams had shown that FMRP levels were increased in several tumor types, and increased the chances that those tumors would metastasize.
Researchers from Astellas Pharma Inc. presented preclinical data for the novel 5-HT5A receptor antagonist, ASP-5736, being developed for the treatment of fragile X syndrome (FXS).
Four scientists have shared the 2022 Kavli Prize in neuroscience, "for pioneering the discovery of genes underlying a range of serious brain disorders," together and separately.
PERTH, Australia – Sydney-based Actinogen Medical Ltd. will advance its lead compound, Xanamem, into two proof-of-concept phase II studies in Alzheimer’s disease and fragile X syndrome.