Bruce Leuchter, MD, Co-founder and CEO of Neurvati Neurosciences and GRIN Therapeutics, discusses the Breakthrough Therapy designation for radiprodil for treatment of patients with GRIN-related neurodevelopmental disorders.
GRIN-related neurodevelopmental disorders are a family of rare genetic conditions caused by pathogenic mutations in the GRIN genes. These disorders are characterized by childhood-onset epilepsy, developmental delay, movement disorders, and features of autism spectrum disorder. The symptoms a child experiences and the severity of the disorder can vary widely.
Radiprodil is a selective, potent negative allosteric modulator of the N-methyl-D-aspartate (NMDA) receptor subtype 2B (NR2B or GluN2B). The investigational drug has been shown to modulate GluN2B-NMDA and have an antiseizure effect.
Recently, the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy designation to radiprodil for the treatment of seizures associated with GRIN-related neurodevelopmental disorder with gain-of-function mutations. This designation grants access to the benefits of Fast Track designation alongside FDA guidance from senior management of drug development programs.
The Breakthrough Therapy designation followed positive data from the phase 1b Honeycomb clinical trial of radiprodil in patients with GRIN-related neurodevelopmental disorders. This included a 86% reduction in seizure frequency from baseline in patients with countable motor seizures treated with radiprodil versus anti-seizure medications. This frequency reduction was consistent across GRIN genotypes with gain-of-function variants.
Plans to initiate a phase 3 pivotal trial for radiprodil are set for early to mid-2025. The goal will be to evaluate the impact of targeted treatment on core aspects of the disease including seizures, behavioral abnormalities and functional outcomes.
To learn more about GRIN-related neurodevelopmental disorders and other rare neurological conditions, visit https://checkrare.com/diseases/neurology-nervous-system-diseases/