Denali Therapeutics Inc. has divulged NAD(+) hydrolase SARM1 (SAMD2; MyD88-5) inhibitors reported to be useful for the treatment of glaucoma, spinal cord Injury, multiple sclerosis, Niemann-Pick disease, stroke, Alzheimer’s disease, amyotrophic lateral sclerosis and diabetic neuropathy, among others.
Targeting human glutaminyl-peptide cyclotransferase (QPCT) has emerged as a potential strategy in Alzheimer’s disease (AD). As the main substrates of this enzyme are involved in biological processes associated with neurodegenerative diseases, Parkinson’s disease (PD) and others may be other options to explore as well.
Beijing DP Technology Co. Ltd. has nominated DPT-0415, a novel small molecule targeting lipoprotein‐associated phospholipase A2 (Lp-PLA2), as a preclinical candidate for the treatment of diabetic retinopathy (DR) and diabetic macular edema (DME).
Therapeutics for peripheral neuropathies, such as Charcot-Marie-Tooth (CMT) disease, are still quite limited and focus on managing symptoms. Augustine Therapeutics NV has presented an orally available histone deacetylase 6 (HDAC6) inhibitor, AGT-100216, for the treatment of CMT. AGT-100216 was designed to work through improving axonal regeneration and nerve functional restoration.
Cannabidiol (CBD) is a nonpsychoactive cannabinoid with potential therapeutic use in several diseases such as epilepsy or anxiety, but shows poor solubility, erratic absorption and structural polymorphisms that limit its bioavailability. Researchers from Artelo Biosciences Inc. presented the pharmacokinetic profile of ART12.11, a proprietary co-crystallization of CBD and co-former tetramethylpyrazine (TMP), formulated to achieve improved bioavailability and stability and to overcome polymorphism of CBD.
Scientists at the University of Copenhagen have demonstrated that the trigeminal nerve, a cranial nerve whose activation underlies migraine pain, has direct access to cerebrospinal fluid (CSF) transported by the glymph system. Furthermore, in the run-up to a migraine, levels of multiple proteins in the CSF changed. One of them was calcitonin gene-related peptide (CGRP), a driver of migraine pain and target of several approved drugs for both treatment and prevention of migraine.
NMD Pharma ApS has described chloride channel protein 1 (CLCN1; ClC-1) channel blockers reported to be useful for the treatment of myasthenia gravis, Lambert-Eaton syndrome, critical illness myopathy, amyotrophic lateral sclerosis, spinal muscular atrophy, Guillain-Barré syndrome, post-poliomyelitic and chronic fatigue syndrome, among others.
Less than a month after the U.S. FDA’s Peripheral and Central Nervous System Drugs Advisory Committee handed down a unanimous vote in favor of Eli Lilly and Co.’s Alzheimer’s disease candidate (AD), donanemab, the agency approved the drug as a once-monthly injection for adults with early symptomatic disease. Branded Kisunla, the beta-amyloid antagonist marks the second approved AD drug that has demonstrated in clinical trials an ability to slow cognitive decline, going up against Leqembi (lecanemab) from Biogen Inc. and Eisai Co. Ltd., which won full approval in July 2023, only six months after nabbing an accelerated nod.
At the Congress of the European Academy of Neurology, researchers from Dianthus Therapeutics Inc. presented preclinical data on DNTH-103, a monoclonal antibody engineered to selectively target the active form of complement C1s.
Entheogenix Biosciences Inc. has described 5-HT2A receptor agonists reported to be useful for the treatment of post-traumatic stress, depression and anxiety disorders.
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