Knopp Biosciences Reports Data Demonstrating Restoration of Function by KB-3061 in Cellular Model of Gene Variants Causing KCNQ2 Epileptic Encephalopathy

Knopp Biosciences LLC today reported the results of preclinical experiments demonstrating that KB-3061, the lead molecule in the Company’s ion channels platform, restored the function of Kv7 potassium channels in cells engineered to express gene variants that cause the rare, neonatal disease KCNQ2 epileptic encephalopathy (KCNQ2-EE).

The data were presented by Michael Bozik, M.D., Chief Executive Officer of Knopp, at the Epilepsy Precision Medicine Conference being held Sept. 16-17, in Washington, D.C. His presentation included preclinical findings from experiments conducted in collaboration with Edward C. Cooper, M.D., Ph.D., Associate Professor of Neurology, Neurosciences, and Molecular & Human Genetics at Baylor College of Medicine. These experiments extend the pharmacology previously reported for KB-3061, including its in vitro nanomolar activity in wild type Kv7.2/7.3 potassium channels.

Dr. Bozik reported that cells transfected to express three highly recurrent missense mutations in the KCNQ2 gene demonstrated strong suppression of function, as measured by significantly reduced potassium current through the mutated Kv7.2 channels. When treated with KB-3061, the function of the mutated channels was fully restored, producing normal Kv7.2 channel current density.

Knopp is advancing the development of KB-3061 as a potential precision medicine treatment for KCNQ2-EE, a genetically defined disease associated with seizures beginning in the first days of life and profound developmental delay. The disease is caused by dominant-negative mutations in the KCNQ2 gene, which normally produces a potassium channel, Kv7.2, critical to early brain development.

“Our data generated with Dr. Cooper support the precision medicine approach to KCNQ2-EE,” said Dr. Bozik. “The results suggest that restoring suppressed Kv7.2 channel activity may ameliorate the dysfunction caused by KCNQ2 gene mutations.”

Knopp has initiated studies to support the filing of an Investigational New Drug application with the goal to assess the clinical effects of KB-3061. As previously announced by Knopp, KB-3061 has also demonstrated potent seizure control in an in vivo model of epilepsy.

ABOUT KNOPP BIOSCIENCES LLC

Knopp Biosciences is a privately held drug discovery and development company focused on delivering breakthrough treatments for inflammatory and neurological diseases of high unmet need. In addition to developing oral dexpramipexole for eosinophil-associated diseases, Knopp’s preclinical Kv7 platform is directed to small molecule treatments for KCNQ2 epileptic encephalopathy and other CNS hyperexcitability disorders. Please visit www.knoppbio.com.

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.

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