PA-KNOPP-BIOSCIENCES-LLC

Knopp Biosciences LLC today reports data demonstrating that compound subgroups from its proprietary library of potassium-channel modulators selectively target Kv7 channel subtypes associated with a wide range of neurological and smooth muscle diseases. The data is being presented at the International Kv7 Channels Symposium 2019 in Naples, Italy.

Presentation Title: The Re-emergence of Kv7 Drug Discovery
Presenter: Michael Bozik, M.D., CEO, Knopp Biosciences
Date & Time: September 13, 4:35 pm CEST

Michael Bozik, M.D., Chief Executive Officer of Knopp, will present the data derived from the Company’s proprietary library of more than 3,000 novel compounds directed to modulating the Kv7 channel, which regulates the flow of potassium ions across cell membranes. The data demonstrate that specific subgroups of Kv7 compounds selectively modulate different Kv7 channel subtypes across ranges of channel activation and inhibition, enabling selection of compounds capable of targeting neurological disorders associated principally with Kv7.2/7.3 channels and others associated principally with Kv7.4 channels.

“By characterizing a custom library of Kv7 modulators for activity and specificity, we have shown evidence of the potential to develop targeted treatments for diverse diseases of the central nervous system and smooth muscle,” said Dr. Bozik. “Accordingly, we believe our Kv7 library represents a precision tool-set for investigating Kv7 pharmacology across a wide range of therapeutic areas with high unmet need.”

Engineered cell lines expressing the channel subtypes Kv7.2/7.3, Kv7.4, Kv7.3/7.5, and Kv7.1/KCNE1 were used in a screening format to characterize compound activity. A thallium flux assay was the primary screen to determine effective concentration values (EC50) for Kv7.2/7.3 activity and to rank-order compounds. Active compounds were then counter-screened against other Kv7 cell lines. The QPatch HTx automated patch-clamp system provided a confirmatory functional assay to further characterize the electrophysiology properties of these novel compounds.

Knopp’s lead Kv7 modulator, KB-3061, is in preclinical development for KCNQ2 epileptic encephalopathy, a rare neonatal disease characterized by seizures beginning in the first days of life and by significant developmental delay.

ABOUT KNOPP BIOSCIENCES LLC

Knopp Biosciences is a privately held drug discovery and development company focused on delivering breakthrough treatments for neurological and immunological 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 .

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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