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Knopp Biosciences Receives NIH HEAL Grant to Discover and Advance Non-Opioid Treatment for Chronic Pain

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Knopp Biosciences LLC today announced that the National Institutes of Health (NIH) has awarded the company a grant of as much as $8 million to utilize its novel Kv7 platform to discover and develop innovative non-opioid therapies for the treatment for chronic pain. The multi-year award comes through the NIH’s Helping to End Addiction Long-term, or the NIH HEAL Initiative, which aims to improve treatments for chronic pain, curb the rates of opioid use disorder and overdose, and achieve long-term recovery from opioid addiction.

Knopp has developed a platform of molecules and assays directed to a non-narcotic biological target linked to neuropathic pain, or pain caused by damage to nerves. The target is a cellular membrane potassium channel called Kv7, which regulates the flow of electrically charged ions required to modulate the excitability of cells. Growing scientific evidence suggests that selectively activating key Kv7 channel subtypes can control nerve-cell hyperexcitability associated with chronic neuropathic pain.

“Knopp is excited to participate in the HEAL initiative and to discover novel solutions to this public health emergency,” said Michael Bozik, M.D., Chief Executive Officer of Knopp. “Our platform has already demonstrated the potential therapeutic benefit of targeted Kv7 channel activation in preclinical models of epilepsy, a disease, like pain, driven by abnormal firing of nerve cells. We are eager to extend Kv7 pharmacology to models of pain with the hope of developing effective oral medicines.”

Under the grant award, Knopp will identify targeted Kv7 activators for preclinical development with the goal of advancing effective treatments for chronic neuropathic pain, creating the potential for a new class of non-opioid medicines. Knopp’s research grant of as much as $8 million is based upon the attainment of milestones over five years.

Knopp’s award is one of 375 grant awards across 41 states made by the NIH in fiscal year 2019 to apply scientific solutions to reverse the national opioid crisis. “It’s clear that a multi-pronged scientific approach is needed to reduce the risks of opioids, accelerate development of effective non-opioid therapies for pain, and provide more flexible and effective options for treating addiction to opioids,” said NIH Director Francis S. Collins, M.D., Ph.D., who launched the initiative in early 2018. “This unprecedented investment in the NIH HEAL Initiative demonstrates the commitment to reversing this devastating crisis.”

Knopp’s lead Kv7 modulator, KB‐3061, is in preclinical development for KCNQ2 epileptic encephalopathy (KCNQ2-EE), a rare neonatal disease characterized by seizures beginning in the first days of life and by significant developmental delay in cognitive and motor function. As previously announced, KB-3061 has demonstrated potent seizure control and a wide therapeutic index in an in vivo model of epilepsy, and the restoration of mutated Kv7 channel function in cells transfected with gene variants that cause KCNQ2-EE.

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. 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, non-opioid treatments for pain, 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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