Intracellular chloride levels are crucial for physiological brain development and correct functioning. On the other hand, aberrant expression of chloride importer NKCC1 (basolateral Na-K-Cl symporter, SLC12A2) and exporter KCC2 (K-Cl cotransporter 2, SLC12A5) is involved in neurodevelopmental and neurologic conditions, including mesial temporal lobe epilepsy (MTLE), among others, leading to an imbalance between excitatory and inhibitory signals.
Xenon Pharmaceuticals Inc.’s Kv7 potassium channel opener XEN-1101 remains very much alive despite the primary endpoint miss in top-line data from the phase II proof-of-concept X-Nova trial in moderate to severe major depressive disorder.
Saniona AB has initiated the candidate selection phase with a proprietary subtype selective frontrunner molecule from its Kv7 lead optimization program for epilepsy.
In a global deal worth up to $770.5 million in up-front and milestone payments, Stevenage, U.K.-based Autifony Therapeutics Ltd. signed with Dublin-based Jazz Pharmaceuticals plc for two ion channel-targeting programs aimed at finding and developing new drugs for neurological disorders. Autifony will take selected candidates through preclinical development, while Jazz will cover clinical development, manufacturing, regulatory activities and commercialization.
Researchers from the University of Michigan and affiliated organizations presented data from a study that identified a link between a new neurodevelopmental disorder and biallelic variants in the EPB41L3 gene, which encodes band 4.1-like protein 3 that plays important roles in neuronal development, myelination and cytoskeletal organization.
A large-scale genetic study found 26 risk loci for epilepsy, a chronic brain disease with multiple forms, not all of them heritable. The work, by more than 300 authors from the International League Against Epilepsy (ILAE), investigated seven different subtypes of this neurological condition. “There are over 100 genes that we know can harbor mutations that cause epilepsy,” the co-corresponding author Gianpiero Cavalleri told BioWorld. These genes have rare forms that cause that epilepsy. However, “this particular GWAS is focused more on common forms of epilepsy,” he said.
Rapport Therapeutics Inc. raised $150 million in a series B round, less than six months after unveiling its $100 million series A investment. It’s further evidence that its distinctive precision neuromedicine approach has gained traction with some deep-pocketed investors and adds further momentum both to its clinical development and its discovery research efforts.
Stoke Therapeutics Inc.’s stock tumbled 32% after the company reported phase I/IIa data of its antisense oligonucleotide, STK-001, in Dravet syndrome, showing it reduced convulsive seizures in children most consistently at the highest dose, with benefits increasing as time progressed.
Cannabidiol derivatives potentially useful for the treatment of Parkinson’s disease and epilepsy have been described in a Deyi Pharmaceutical Co. Ltd. patent.
