Miralinc Pharma Inc. has patented new heterocyclic selective histone deacetylase 6 (HDAC6) inhibitors reported to be useful for the treatment of type 2 Charcot-Marie-Tooth disease and chemotherapy-induced peripheral neuropathy, among others.
Humanwell Pharmaceutical US Inc. has synthesized new sigma non-opioid intracellular receptor 1 (SIGMAR1) antagonists reported to be useful for the treatment of cancer, pain, psychosis and substance abuse and dependence.
The central nervous system (CNS) in adult mammals has limited regeneration capacity, and traumatic injuries to the CNS usually lead to permanent functional impairment.
Centessa Pharmaceuticals plc recently detailed the discovery and preclinical characterization of a novel potent and selective orexin OX2 receptor agonist, ORX-142.
Bad news has buffeted Sage Therapeutics Inc. twice in the past few months. Now its placebo-controlled phase II Lightwave study of dalzanemdor in Alzheimer's disease has missed the primary outcome measure, prompting the company to stop development of the NMDA receptor positive allosteric modulator in the indication.
Aviadobio Ltd. has entered a potential $2.18 billion license and commercialization agreement for its frontotemporal dementia gene therapy, AVB-101, with Astellas Pharma Inc. Astellas is making a $20 million equity investment in London-based Aviadobio and will pay up to $30 million up front in advance of deciding whether or not to exercise the exclusive option to worldwide rights.
A digital cognitive assessment developed by Braincheck Inc., Braincheck Assess, proved comparable to the gold-standard Montreal Cognitive Assessment in identifying patients with cognitive impairment in a study published in Frontiers in Psychology.
μ-Opioid receptor agonists have been described in a Miralogx LLC patent as potentially useful for anesthesia and sedation and the treatment of anxiety disorders, asthma, depression and pain.
Shares of Scholar Rock Holding Corp. (NASDAQ:SRRK) soared $26.86, or 362%, to close Oct. 7 at $34.28, after the Cambridge, Mass.-based firm disclosed positive top-line data from the phase III Sapphire study testing apitegromab in patients with spinal muscular atrophy (SMA). Apitegromab, which Wainwright analyst Andres Maldonado said will “transform SMA” therapy, met the primary endpoint with statistically significant and clinically meaningful improvement in motor function as measured by the Hammersmith Functional Motor Scale Expanded.
Aquinnah Pharmaceuticals Inc. has identified new TAR DNA-binding protein 43 (TARDBP; TDP-43) aggregation and microtubule-associated protein tau (PHF-tau; MAPT) aggregation inhibitors reported to be useful for the treatment of amyotrophic lateral sclerosis (ALS) and Alzheimer’s disease.