Knopp Biosciences Enters Research Collaboration with Baylor College of Medicine to Develop New Treatments for KCNQ2 Neonatal Encephalopathy

Knopp Biosciences LLC, a privately held drug discovery and development company focused on delivering breakthrough treatments for inflammatory and neurological diseases with a high unmet need, today announced a research collaboration with Baylor College of Medicine in Houston, TX, to advance the development of innovative treatments for a rare and serious form of neonatal epilepsy.

The principal investigator in the collaboration is Edward C. Cooper, M.D., Ph.D., Associate Professor of Neurology, Neuroscience, and Molecular & Human Genetics at Baylor. Dr. Cooper’s laboratory is focused on understanding and developing new treatments for epilepsy, including rare genetic epilepsies associated with dysfunction of the developing infant brain. These diseases include KCNQ2 neonatal epileptic encephalopathy, which is caused by mutations in the KCNQ2 gene and loss of function in nerve-cell potassium channels.

Knopp has developed a large library of drug molecules designed as Kv7.3/7.3 channel activators, with a lead candidate entering preclinical toxicology studies. Under the sponsored research agreement, the Cooper Lab will characterize the effects of Knopp’s drug candidates on normal and mutated Kv7.2/7.3 ion channel activity.

“We look forward to collaborating with Dr. Cooper, whose pioneering work has opened new possibilities for the treatment of devastating childhood epilepsies,” said Michael Bozik, M.D., Chief Executive Office of Knopp. “The Cooper Lab at Baylor will play a key role in characterizing how our molecules interact with Kv7 channels closely linked with epilepsy and abnormal brain development, which we anticipate will help us to identify and advance drug candidates for further development.”

“Variants in KCNQ2 and related genes are now appreciated to be among the most common genetic causes of neonatal onset-epilepsy and disorders where epilepsy is accompanied by severe global disability,” said Dr. Cooper. “This collaboration will allow us to move toward testing the hypothesis that drugs directly reversing the effects of the disease-causing mutations may be of benefit to patients.”

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 with a high unmet need. Knopp’s clinical-stage small molecule, dexpramipexole, is entering Phase 3 development in hypereosinophilic syndrome and Phase 2 clinical development 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 www.knoppbio.com.

Forward Looking Statement
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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