Knopp Biosciences Receives Year 4 Renewal of NIH Blueprint Grant Award for Advancing the Kv7 Activator KB-3061 in a Rare Neonatal Epilepsy

Knopp’s lead Kv7 platform candidate advancing toward clinical studies in KCNQ2 epileptic encephalopathy

PITTSBURGH, PA, SEPTEMBER 25, 2019—Knopp Biosciences LLC today announced the renewal of its grant award for Year 4 from the National Institutes of Health Blueprint Neurotherapeutics Network (BPN) to advance its lead Kv7 potassium channel activator, KB-3061, in rare neonatal epilepsy.

Based on milestone attainment, Knopp was awarded an additional $679,492 in direct support. The grant has a total potential direct award value of $2.4 million over four years. Beyond the direct award, the NIH BPN may use BPN-sponsored contract research organizations to underwrite the cost of toxicology and safety pharmacology studies and drug manufacturing required to submit an Investigational New Drug application (IND) for KB-3061 to the U.S. Food and Drug Administration.

Knopp is advancing the development of KB-3061 (BPN-25203) as a potential precision medicine treatment for KCNQ2 epileptic encephalopathy (KCNQ2-EE), a genetically defined disease associated with seizures beginning in the first days of life and profound developmental delay. The disease is caused by dominant-negative mutations in the KCNQ2 gene, which produces a potassium channel, Kv7.2, critical to early brain development.

The BPN, part of the NIH Blueprint for Neuroscience Research, is a collaboration of NIH Institutes and Centers that supports innovative research on the nervous system with the goal of developing new neurotherapeutic drugs.

“We are very pleased to secure the ongoing support of the NIH Blueprint Program as we advance KB-3061 toward clinical development in a rare and profoundly disabling brain disease in newborns,” said Knopp’s CEO, Michael Bozik, M.D., a neurologist with clinical development expertise in potassium channel modulators.

Knopp has initiated pre-clinical studies to support the filing of an IND with the goal to assess the clinical effects of KB-3061. As previously announced, KB-3061 has demonstrated potent seizure control and a wide therapeutic index in an in vivo model of epilepsy, and the restoration of function in cells transfected with gene variants that cause KCNQ2-EE.

Knopp is collaborating with key clinical investigators in the development of KB-3061, as well as with the KCNQ2 Cure Alliance, whose 2020 Family and Professional Summit will be held May 1-3, 2020, in Philadelphia, PA.


Knopp Biosciences is a privately held drug discovery and development company focused on delivering breakthrough treatments for inflammatory and neurological diseases of high unmet need. In addition to developing oral dexpramipexole for eosinophil-associated diseases, Knopp’s preclinical Kv7 platform is directed to small molecule treatments for KCNQ2 epileptic encephalopathy, non-opioid treatments for pain, and other CNS hyperexcitability disorders. Please visit


The Blueprint Neurotherapeutics Network provides non-dilutive support for small molecule drug discovery and development, from hit-to-lead chemistry through Phase I clinical testing. Please visit BPN.


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

Knopp’s Kv7 research is supported under Award Number U44NS093160 of the National Institute of Neurological Disorders and Stroke of the National Institutes of Health (NIH). The content of this announcement is solely the responsibility of Knopp and does not necessarily represent the views of the NIH.


This press release contains “forward-looking statements,” including statements relating to planned regulatory filings and clinical development programs. All forward-looking statements are based on management’s current assumptions and expectations and involve risks, uncertainties and other important factors, specifically including the uncertainties inherent in clinical trials and product development programs, the availability of funding to support continued research and studies, the availability or potential availability of alternative therapies or treatments, the availability of patent protection for the discoveries and strategic alliances, as well as additional factors that may cause Knopp’s actual results to differ from our expectations. There can be no assurance that any investigational drug product will be successfully developed or manufactured or that final results of clinical studies will be supportive of regulatory approvals required to market a product. Knopp undertakes no obligation to update or revise any such forward-looking statements, whether as a result of new information, future events or otherwise.

Knopp’s pipeline consists of investigational drug products that have not been approved by the U.S. Food and Drug Administration. These investigational drug products are still undergoing clinical study to verify their safety and effectiveness.