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.
In an alternative to small molecules, researchers at the University of Ferrara and Università degli Studi di Padova sought to create an NOP agonist based on peptides, specifically dimers of peptides derived from native nociceptin.
Epiminder Ltd. raised AU$125 million (US$82.99 million) in its initial public offering on the Australian Securities Exchange to commercialize its Minder system, a long-term ambulatory electroencephalography monitoring device for epilepsy.
People with drug-resistant epilepsy have had few other options, but Neuropace Inc. appears on target to provide an alternative. The Nautilus trial of its responsive neurostimulation system for individuals with drug-resistant idiopathic generalized epilepsy showed a 77% reduction in generalized tonic-clonic seizures sustained over 18 months.
Positive efficacy results led to Praxis Precision Medicines Inc.’s phase II Embold study in developmental and epileptic encephalopathies (DEEs) being halted early, propelling the company’s shares dramatically upward.
The ALS Association has announced the recipients of its 2025 Hoffman ALS Clinical Trial Awards. These research grants, worth up to $1 million each, support early-phase trials that generate critical data to accelerate the development of new amyotrophic lateral sclerosis (ALS) treatments.
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