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Knopp Biosciences Announces Renewal of NIH Blueprint Grant Award for Advancing KCNQ2 Modulators in Epilepsy

October 23, 2017

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.

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 for dexpramipexole. 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 dexpramipexole will be successfully developed or manufactured or that final results of clinical studies will be supportive of regulatory approvals required to market the 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.

Media Contact:
Thomas Petzinger Jr.
412-488-1776
tom@knoppbio.com