Saniona AB has presented preclinical data and its clinical development strategy for its lead clinical candidate, SAN-2668, which is a GABA-A receptor positive allosteric modulator under development for the treatment of severe pediatric epilepsies.
Italian family-owned Angelini Pharma SpA is making its first move into the U.S. market, acquiring rare diseases specialist Catalyst Pharma Inc. in an all-cash deal worth $4.1 billion. The acquisition gives Angelini ownership of three marketed drugs for treating epilepsy and neuromuscular diseases that had combined sales of $589 million in 2025, a 19.8% increase over 2024.
Shanghai Unixell Biotechnology Co. Ltd. has obtained IND clearance from the FDA for UX-GIP001, its iPSC-derived allogeneic cell therapy for focal epilepsy. A phase I study will evaluate UX-GIP001 in patients with drug-resistant epilepsy.
Any lingering disappointment in the delay for Xenon Pharmaceuticals Inc.’s readout of the phase III X-Tole2 study testing azetukalner in focal onset seizures (FOS) appeared thoroughly extinguished as the company’s KV7 potassium channel opener yielded better-than-expected data, even besting earlier phase IIb findings and positioning the drug for an NDA submission later this year.
Angelini Pharma SpA and Quiver Bioscience Inc. have entered into a collaboration and licensing agreement to advance novel therapeutics for genetic epilepsies. Under the multiyear collaboration, Angelini Pharma will leverage Quiver’s technology platform to gain deeper understanding of a broad set of developmental and epileptic encephalopathies.
The SCN2A Foundation has entered into a research collaboration with Unravel Biosciences Inc. to advance preclinical research for SCN2A-related disorders caused by loss-of-function mutations, a subset of SCN2A conditions driven by insufficient functional protein.
Scientists at Osaka University and Tokyo University of Science have described compounds targeting Claudin-5 (CLDN5) acting as blood-brain barrier (BBB) permeability regulators reported to be useful for the treatment of sepsis, cerebral edema, infections, epilepsy, multiple sclerosis, psychiatric disorders, Alzheimer’s disease and Parkinson’s disease, among others.
Epilepsygtx Ltd. has raised a $33 million series A to fund a phase I/IIa trial of EPY-201, a gene therapy for treating drug-resistant focal epilepsy. EPY-201 uses an adeno-associated viral vector to deliver KCNA1, the gene encoding Kv1.1, a potassium ion channel that modulates neuronal excitability.
Researchers from Neumirna Therapeutics ApS have presented an anti-miR-134 ASO approach named NMT.001 for the potential treatment of drug-resistant epilepsy.
Mutations in the KCNT1 gene produce gain-of-function effects that lead to overactivation of the potassium channel and consequent disruption of normal neuronal electrical signaling. These alterations give rise to a severe, early-onset developmental and epileptic encephalopathy that is typically associated with a high seizure burden and resistance to standard antiseizure medications.
