For initial indication of Rett Syndrome
BHV-5000 is an orally available, first-in-class, low-trapping, NMDA receptor antagonist prodrug that we are initially developing for the treatment of symptoms associated with Rett syndrome, including breathing irregularities. As a low-trapping NMDA receptor antagonist, BHV-5000 has differentiating pharmacologic properties compared to other agents that have been in development within this drug class. The unique property of a low-trapping NMDA receptor antagonist is its ability to uncouple from the target receptor more freely, a property that is thought to mitigate the dissociative or psychotic-like effects that have been observed with other NMDA antagonists. Rett syndrome is a rare and severe genetic neurodevelopmental disorder. After six to 18 months of apparently normal post-natal development, patients with Rett syndrome develop global deceleration of psychomotor function, loss of acquired cognitive skills and brain-mediated episodes of transient respiratory suppression. With intensive care, patients may survive into adulthood, yet they are severely physically and cognitively impaired. Rett syndrome affects approximately 15,000 individuals in the United States. No approved drug therapies for Rett syndrome are currently available and care is supportive.
