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.
Khalifa University and The University of Queensland presented preclinical data for a novel acid-sensing ion channel 1 (ASIC1) inhibitor, Hi1a, evaluated in models of multiple sclerosis (MS).
The adenosine A1 receptor (A1R) modulates ionic conductance in neuronal membrane and contributes to the anticonvulsant and neuroprotective role of the endogenous neuromodulator adenosine. Because A1Rs are present all throughout the body, the development of A1R modulation as antiseizure strategy may face difficulties such as side effects in the heart or other organs.
It has been previously demonstrated that intranasal treatment with COG-201, an AAV9-shRNA designed to target the down-regulation of the 5-HT2A receptor, significantly decreased anxiety and improved memory in mice and rats.
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