Knopp Biosciences Receives Rare Pediatric Disease Designation for Kv7 Activator KB-3061 for Treatment of KCNQ2 Epileptic Encephalopathy

PITTSBURGH, July 15, 2020 (GLOBE NEWSWIRE) -- Knopp Biosciences LLC, a privately held drug discovery and development company focused on delivering breakthrough treatments for immunological and neurological diseases with high unmet needs, announced today that it has received Rare Pediatric Disease Designation from the U.S. Food and Drug Administration (FDA) for its therapeutic candidate KB-3061, an activator of voltage-gated Kv7.2/7.3 potassium channels, for the treatment of KCNQ2 epileptic encephalopathy (KCNQ2-EE).

Knopp is advancing the development of KB-3061 as a potential precision medicine for KCNQ2-EE, a rare genetic disease associated with seizures beginning in the first days of life and profound neurodevelopmental delay. The disease is caused by dominant-negative mutations in the KCNQ2 gene, which encodes for Kv7.2, a potassium channel that plays a critical role in early brain development.

As previously announced, in cells transfected with gene variants that cause KCNQ2-EE, KB-3061 has demonstrated the ability to fully restore mutated Kv7.2 potassium channel function in in vitro experiments.

“The granting of this designation from the FDA represents a major milestone for Knopp Biosciences,” said Michael Bozik, M.D., Chief Executive Officer. “The Rare Pediatric Disease Designation highlights the significant unmet medical need facing patients with KCNQ2-EE. We look forward to working with the FDA to advance the development of KB-3061.”

“The Rare Pediatric Disease Designation by FDA for KB-3061 advances our mission of promoting research and supporting families of children with KCNQ2 developmental and epileptic encephalopathy,” added Jim Johnson, president of the KCNQ2 Cure Alliance. “We are excited that the KB-3061 compound represents a novel approach to the condition that is so devastating to our families." 

Rare Pediatric Disease Designation by the FDA is granted in the case of serious or life-threatening diseases in the U.S. affecting fewer than 200,000 people in which the serious or life-threatening manifestations are primarily in individuals 18 years of age and younger. The designation provides a specific regulatory incentive for companies to develop and market therapies that treat these rare pediatric conditions. The designation may be used alone or in combination with other programs, such as Orphan Drug Designation.

Knopp's Kv7 research is supported in part 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.

Knopp Biosciences, based in Pittsburgh, PA, USA, is a privately held drug discovery and development company focused on delivering breakthrough treatments for immunological and neurological diseases with high unmet need. Knopp’s clinical-stage small molecule, oral dexpramipexole, has entered Phase 2 clinical trials in eosinophilic asthma and is slated for Phase 3 development in hypereosinophilic syndrome. Knopp’s preclinical Kv7 platform is directed to small molecule treatments for neonatal epileptic encephalopathy, other rare epilepsies, tinnitus, and neuropathic pain. Please visit

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

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 pre-clinical or clinical study to verify their safety and effectiveness.

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