U.S. Food and Drug Administration (FDA) Requests Additional Non-Clinical Data to Support Dose Justification Before Initiation of a Phase II Clinical Trial with NBI-921352 in Pediatric SCN8A-DEE Patients
FDA Grants Rare Pediatric Disease Designation for NBI-921352 for the Treatment of SCN8A-DEE
SAN DIEGO and BURNABY, British Columbia, Oct. 08, 2020 (GLOBE NEWSWIRE) -- Neurocrine Biosciences, Inc. (NASDAQ:NBIX) and Xenon Pharmaceuticals Inc. (NASDAQ:XENE) today provided an update on the ongoing collaboration for the clinical development of NBI-921352, previously known as XEN901. Neurocrine Biosciences has an exclusive license to NBI-921352, a clinical stage selective Nav1.6 sodium channel inhibitor with potential in SCN8A developmental and epileptic encephalopathy syndrome (SCN8A-DEE), a rare pediatric epilepsy, and other forms of epilepsy, including focal epilepsy. The U.S. Food and Drug Administration (FDA) provided feedback on an Investigational New Drug (IND) application submitted by Neurocrine Biosciences in support of a Phase II clinical trial in pediatric SCN8A-DEE patients. As part of its review of the IND, the FDA is requesting additional non-clinical data to support dose justification in this pediatric study. Neurocrine Biosciences and Xenon will engage with the FDA to address the feedback received with the goal of initiating a Phase II clinical trial in 2021. In parallel with this interaction, Neurocrine Biosciences is advancing clinical plans to develop NBI-921352 for the treatment of adult focal epilepsy. In addition, the FDA recently granted Rare Pediatric Disease Designation for NBI-921352 for the treatment of SCN8A-DEE.
"We expect to engage with the FDA in the near term to discuss their request for additional non-clinical data to enable a pediatric trial in SCN8A-DEE patients. In parallel, we are continuing to develop plans to study NBI-921352 in patients with adult focal epilepsy. We are committed to working with the FDA to address their feedback in a timely manner, with the goal of initiating a Phase II pediatric clinical trial in 2021," said Eiry W. Roberts, M.D., Chief Medical Officer at Neurocrine Biosciences.
"The recent Rare Pediatric Disease Designation from the FDA underscores that SCN8A-DEE is a devastating pediatric epilepsy, with a lack of approved treatments, that results in serious, life-threatening seizures and neurodevelopmental impairment, further validating our ‘precision medicine' approach to develop treatments for pediatric epilepsies. We are now working with the team at Neurocrine Biosciences to respond to the FDA's request for information and also to support the clinical development plans for NBI-921352 in adult focal epilepsy," said Dr. Simon Pimstone, Xenon's Chief Executive Officer.
Pursuant to the Collaboration Agreement, upon FDA acceptance of an IND for NBI-921352 in either SCN8A-DEE or another major indication, Xenon is eligible to receive a milestone payment of either $25 million or $10 million, respectively, with 55% of the amount in the form of an equity investment in Xenon at a 15% premium to Xenon's 30-day trailing volume weighted average price at that time.