Work at Neumora Therapeutics Inc. has led to the identification of new casein kinase I isoform δ (CKI-δ) inhibitors reported to be useful for the treatment of neurological disorders.
EG 427 SAS has received IND clearance from the FDA for EG-110A, a gene therapy for the treatment of neurogenic detrusor overactivity in patients with spinal cord injury. A phase Ib/IIa study is being initiated.
Argenx SE gained U.S. FDA approval of subcutaneously given Vyvgart Hytrulo (efgartigimod alfa and hyaluronidase-qvfc) for adult patients with chronic inflammatory demyelinating polyneuropathy (CIDP). The company’s stock (NASDAQ:ARGX) was up 11.7% to $440.59 at the close of trading June 24. About 24,000 people in the U.S. are being treated for CIDP, and patients are generally diagnosed between 40 and 60 years of age.
Scientists spread across Europe and the U.S. filed for protection of an implantable electrode array including a plurality of microneedles, which may be implanted into the auditory nerve bundle and stimulate the auditory nerve in response to receiving electrical signals representative of observed sounds.
Vertex Pharmaceuticals Inc. has divulged orexin OX2 receptor modulators reported to be useful for the treatment of amyotrophic lateral sclerosis, obesity, hypertension, retinopathy, multiple sclerosis, narcolepsy, hypersomnia and Parkinson’s disease.
Sage Therapeutics Inc. has synthesized sterol derivatives acting as glutamate receptor ionotropic, NMDA 2A (GRIN2A; GluN2A) negative allosteric modulators reported to be useful for the treatment of neurological disorders.
Astellas Pharma US Inc., a U.S. affiliate of Astellas Pharma Inc., has entered into a sponsored research agreement with the University of Massachusetts Medical School (UMass Chan Medical School) to conduct research for an AAV vector-mediated gene therapy for the treatment of Alexander disease.
Alzheon Inc. has raised $100 million in a series E financing round to push its oral drug candidate for early Alzheimer’s disease (AD), ALZ-801 (valiltramiprosate), through a late-stage, Apolloe4 study.
A nearly two-year-old partial clinical hold has been lifted by the U.S. FDA on PTC Therapeutics Inc.’s pivotal phase II study in Huntington’s disease. The agency had paused enrollment in October 2022, saying it wanted more data on PTC-518, an orally bioavailable small-molecule splicing modifier, before enrollment could continue.
RSS
