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Knopp Biosciences

Knopp Biosciences

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Knopp Biosciences Announces NIH Blueprint Grant Award

February 16, 2017

PITTSBURGH, PA, USA—February 16, 2017—Knopp Biosciences LLC today announced that the National Institutes of Health Blueprint Neurotherapeutics Network has awarded the company a grant under the Small Business Innovation Research (SBIR) program to advance novel treatments for epilepsy.

Terms of the grant provide the potential for up to $2.5 million of direct support based on milestone attainment.

The award supports Knopp's preclinical and clinical development of small-molecule drug candidates directed to a validated, anti-seizure pharmaceutical target known as Kv7.2. Knopp expects initially to advance novel Kv7 activators in neonatal epileptic encephalopathy associated with a rare mutation in the KCNQ2 gene. These mutations cause severe epilepsy and profound developmental disability in newborns and infants, for whom conventional anti-seizure medications are insufficient or ineffective.

Steven Dworetzky, PhD., Knopp's Chief Scientific Officer and the Principal Investigator in the Blueprint-funded project, is a longtime Kv7 researcher whose group first cloned the Kv7.2 gene during his former tenure with the Bristol-Myers Squibb Co. "This award comes not just with significant direct support but with access to top collaborators in the Blueprint Neurotherapeutics Network, who are committed to the discovery of disease-modifying treatments for the underlying causes of epilepsy," he said. "We already see evidence that our Blueprint collaboration will accelerate our move into human studies in challenging indications, including KCNQ2 encephalopathy and treatment-resistant, generalized epilepsy syndromes."

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 in clearly defined patient populations. Our clinical-stage small molecule, dexpramipexole, will be entering Phase 2/3 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.

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