|PITTSBURGH, PA, USA, OCTOBER 23, 2017—Knopp Biosciences LLC today announced the renewal and expansion of its grant award from the National Institutes of Health Blueprint Neurotherapeutics Network to advance novel treatments for epilepsy.
The Phase 2 award under the NIH Small Business Innovation Research (SBIR) program anticipates NIH support of up to $2 million over the next three years of the project, subject to satisfactory completion of milestones. The renewal follows the successful completion of all milestones under a previously awarded Phase 1 grant of $400,000.
Knopp is directing its potassium channel activator program to preclinical and clinical development of small-molecule drug candidates against a validated, anti-seizure pharmaceutical target encoded by the KCNQ2 gene. Knopp intends to advance novel, small-molecule KCNQ2 activators in neonatal epileptic encephalopathy, a rare disorder caused by inherited mutations in the KCNQ2 gene. Neonatal epileptic encephalopathy is characterized by persistent seizures from the beginning of life and profound developmental disability in children for whom conventional anti-seizure medications are insufficient or ineffective.
Knopp President and CEO Michael Bozik, M.D., a neurologist with clinical development expertise in potassium channel modulators, serves as Principal Investigator on the Blueprint grant. “This Phase 2 award recognizes the need to go beyond seizure control to treat the underlying causes of epilepsy,” he said. “We are very pleased with the renewal of the NIH Blueprint grant and its emphasis on innovative approaches in the treatment of severe epilepsies.”
The NIH Blueprint collaboration will support Knopp’s advance into human studies in KCNQ2 neonatal epileptic encephalopathy. “The KCNQ2 community appreciates the recognition of the NIH to support the need for innovation in uncommon conditions like KCNQ2 epilepsy,” said Jim Johnson, president of the KCNQ2 Cure Alliance. He noted that the annual KCNQ2 Cure Family and Professional Summit, underway this week in Boston, “is focused on partnership and innovation in the search for effective treatments.”
ABOUT KNOPP BIOSCIENCES LLC
Knopp Biosciences, based in Pittsburgh, PA, USA, is a privately held drug discovery and development company focused on delivering breakthrough treatments for inflammatory and neurological diseases of high unmet need. Our clinical-stage small molecule, dexpramipexole, will be entering late-stage clinical studies in hypereosinophilic syndromes and Phase 2 clinical studies in eosinophilic asthma. Our preclinical platform is directed to small molecule treatments for neonatal epileptic encephalopathy, a devastating brain disorder of infants caused by a rare mutation in the KCNQ2 gene. Please visit knoppbio.com.
ABOUT KCNQ2 CURE ALLIANCE
KCNQ2 Cure Alliance is a non-profit organization dedicated to raising research funds for KCNQ2 epileptic encephalopathy, a rare and catastrophic form of epilepsy beginning in the first days of life. Please visit kcnq2cure.org.