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Knopp Biosciences Announces Year 3 Renewal of NIH Blueprint Grant Award for Advancing KCNQ2 Activators in Rare Neonatal Epilepsy

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Knopp Biosciences LLC today announced the renewal of its grant award for Year 3 from the National Institutes of Health Blueprint Neurotherapeutics Network (BPN) to advance novel treatments for epilepsy.

Based on milestone attainment, Knopp was awarded an additional $693,000 in direct support. The grant has a total potential value of $2.3 million over four years. Beyond direct support, the NIH BPN may underwrite the cost of key external studies at BPN-sponsored-contract research organizations.

As previously reported, Knopp is directing its Kv7 potassium channel activator program to the discovery and development of best-in-class small-molecule drug candidates, consistent with the goals of the NIH BPN. The BPN, part of the NIH Blueprint for Neuroscience Research, is a collaboration of NIH Institutes and Centers that supports research on the nervous system with the goal of developing new neurotherapeutic drugs.

These novel molecules have been synthesized to modulate an anti-seizure pharmaceutical target encoded by the KCNQ2 gene, which contributes significantly to early brain development. Knopp intends to first advance its novel KCNQ2 activators in neonatal epileptic encephalopathy, a rare disorder caused by sporadic 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 current 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 NIH BPN grant. He said, "We’re very pleased to receive this latest Blueprint award, which provides ongoing support that will help us advance promising clinical drug candidates to treat a rare and devastating disease without approved treatment options.” He said Knopp expects its first KCNQ2 candidate to enter Phase 1 testing in 2019.

Knopp is collaborating with key clinical investigators in the development of its first candidate, as well as with the KCNQ2 Cure Alliance, whose 2018 Family and Professional Summit will be held Sept. 14-16, 2018, in Anaheim, CA.

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. Knopp’s clinical-stage small molecule, dexpramipexole, is entering Phase 3 clinical studies in hypereosinophilic syndrome and Phase 2 clinical studies in eosinophilic asthma. Knopp’s preclinical Kv7 platform is directed to small molecule treatments for neonatal epileptic encephalopathy, other rare epilepsies, tinnitus, and neuropathic pain. Please visit knoppbio.com.

ABOUT BPN

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.

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. 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.

Contact information

Knopp Biosciences LLC
Tom Petzinger, +1-412-488-1776
tom@knoppbio.com

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