Knopp Biosciences Presents Data Further Characterizing Its Lead Kv7.2 Activator Drug Candidate for the Treatment of KCNQ2 Epileptic Encephalopathy at the American Epilepsy Society’s 2019 Annual Meeting

Knopp Biosciences LLC, a privately held drug discovery and development company focused on delivering breakthrough treatments for immunological and neurological diseases with high unmet need, today presented data characterizing KB-3061, the lead molecule in the Company’s Kv7 ion channel platform, at the American Epilepsy Society’s (AES) 2019 Annual Meeting in Baltimore, Maryland.

Knopp is advancing the development of KB-3061 as a potential precision medicine treatment for the rare, neonatal disease KCNQ2 epileptic encephalopathy (KCNQ2-EE), a genetically defined 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 produces a potassium channel, Kv7.2, critical to early brain development.

Data presented at AES by Knopp scientists and its academic collaborators support the continued development of KB-3061 as a potential treatment for children with KCNQ2-EE. Key highlights of these and previously reported data include:

• KB-3061 fully restored the function of Kv7 potassium channels in cells engineered to express gene variants from three “hot spot” domains – the voltage sensor, the pore, and the C-terminus – that cause KCNQ2-EE.
• Nanomolar brain concentrations of KB-3061 conferred significant seizure protection in the maximal electroshock model (MES) of epilepsy with minimal impact on neurobehavior.
• KB-3061 is differentiated from other molecules in its class, including ezogabine, having demonstrated improved stability to photo-oxidation, reduced GABA_A receptor activity, and greater potency with increased tolerability in preclinical epilepsy models.
• KB-3061 has no significant activity against a broad range of off-targets, including GPCRs, ion channels, transporters, cardiac ion channels, and kinases.
• KB-3061 IND-enabling toxicology and drug manufacturing process development studies are ongoing with support from the National Institutes of Health Blueprint Neurotherapeutics Network (BPN).

“Our conversations with members of the pediatric epilepsy community have underscored the urgent need for novel medicines that not only treat seizures but address the underlying cause of KCNQ2-EE,” said Michael Bozik, M.D., Chief Executive Officer of Knopp Biosciences. “While the potential of KB-3061 must be assessed clinically, we are vigorously pursuing its development as a targeted treatment for children affected by this disease.”

Knopp has initiated studies to support the filing of an Investigational New Drug (IND) application with the goal to assess the clinical effects of KB-3061.

Follow Knopp Biosciences on Twitter via @KnoppBio and keep up to date with AES Annual Meeting news and updates using the hashtag #AES2019.

ABOUT KNOPP BIOSCIENCES LLC

Knopp Biosciences is a privately held drug discovery and development company focused on delivering breakthrough treatments for immunological and neurological diseases of high unmet need. Knopp’s clinical-stage small molecule, dexpramipexole, is in Phase 2 clinical trials in moderate-to-severe eosinophilic asthma. Knopp’s preclinical Kv7 platform is directed to small molecule treatments for neonatal epileptic encephalopathy, other rare epilepsies, and neuropathic pain. Please visit www.knoppbio.com.

Knopp's Kv7 research is supported under Award Number U44NS093160 and Award Number U44NS115732 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.

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