Prism Biolab Co. Ltd. has identified new 7-membered ring-fused compounds acting as Notch signaling inhibitors and reported to be potentially useful for the treatment of neuronal injury and neurodegenerative diseases.

Paxmedica Inc.’s shares (NASDAQ:PXMD) surged by 179% in early trading Nov. 7 after reporting the publication of phase II autism spectrum disorder results for PAX-101 (suramin) intravenous infusions. Suramin, an anti-trypanosomal and anti-purinergic agent introduced in 1923 for East African sleeping sickness, showed a statistically significant improvement at the 10-mg/kg dose in a secondary endpoint of Clinical Global Impressions – Improvement scale vs. placebo.

At the recent American Society of Human Genetics meeting, researchers from Stanford University reported clinical and functional evidence of the involvement of ARHGAP1, a Rho GTPase-activating protein (GAP) gene, in a patient exhibiting a syndromic neurodevelopmental disorder.

Takeda Pharmaceutical Co. Ltd. has identified orexin OX2 receptor agonists reported to be useful for the treatment of narcolepsy.

The latest firm to brave the rough IPO market, Lexeo Therapeutics Inc. made its Nasdaq debut after pricing about 9.1 million shares at $11 per share, raising proceeds of $100 million to advance its early clinical work on gene therapies for cardiovascular and neurological diseases.

Brenig Therapeutics Inc. has disclosed leucine-rich repeat kinase 2 (LRRK2; dardarin) inhibitors reported to be useful for the treatment of Parkinson’s disease, among others.

One of the main issues encountered by therapeutic antibodies for the treatment of neurodegenerative disorders such as Alzheimer’s disease (AD) is the difficulty of crossing the blood-brain barrier (BBB) to access the central nervous system. To overcome these problems, several attempts have been made using endothelial transmembrane proteins where antibodies can bind. One of these efforts involves transferrin receptor 1 (TfR1).