Knopp Biosciences Presents Positive Preclinical Data for Lead Neonatal Epilepsy Drug Candidate at 2019 Antiepileptic Drug and Device Trials XV Conference

Knopp Biosciences LLC, a privately held drug discovery and development company focused on delivering breakthrough treatments for inflammatory and neurological diseases of high unmet need, today presented in vitro and in vivo data for the company’s lead molecule for the targeted treatment of KCNQ2-neonatal epileptic encephalopathy (KCNQ2-NEE) at the Antiepileptic Drug and Device Trials XV Conference in Miami, Florida. KCNQ2-NEE, a rare infant genetic epilepsy, results from mutations in genes encoding the Kv7.2/Kv7.3 ion channel, leading to frequent seizures and profound developmental delay.

The preclinical data were presented by Steven Dworetzky, Ph.D., Knopp’s Chief Scientific Officer, a longtime Kv7 researcher who led the group that first cloned the Kv7.2 and Kv7.3 genes during his former tenure with the Bristol-Myers Squibb Co. Dr. Dworetzky presented results demonstrating the in vitro nanomolar activity of KB-3061 and its significant seizure protection in the maximal electroshock model (MES) of epilepsy. In addition, in vivo tolerability was demonstrated by the absence of an effect on neurological score at doses that conferred full protection. KB-3061 is differentiated from other molecules in its class, including ezogabine, which was also tested in the in vitro and in vivo models. Among the highlights:

• In the MES model, KB-3061 protected rats from seizures at a dose more than 20 times lower than the dose of ezogabine required to achieve equivalent seizure protection and also showed significantly greater tolerability.
• KB-3061 did not increase the activity of GABA in an allosteric GABA_A receptor assay.
• KB-3061 was chemically stable in a standard photooxidation assay.

“Knopp is very encouraged by the in vitro profile and in vivo profile of KB-3061,” Dr. Dworetzky said. “Children with KCNQ2-NEE and their families urgently need precision medicines with the potential to restore ion channel function and change the course of this disease.”

Knopp is conducting preclinical toxicology studies of KB-3061 in advance of submitting an Investigational New Drug Application to the Food and Drug Administration to initiate clinical trials.

Ion channels are proteins that regulate the excitability of cells. Drugs that modulate ion channels play an important role in treating a variety of neurological, cardiovascular, and smooth muscle conditions. Among ion channel targets, potassium channels play a key role in the pathophysiology of epilepsy and other diseases of high unmet need in the nervous system. Knopp is advancing a library of Kv7.2/7.3 channel activators to modulate this key ion channel in excitable cells, targeting not only the devastating genetic mutation associated with neonatal encephalopathy, but also other rare epilepsies and neuropathic pain.

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.

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. Knopp’s clinical-stage small molecule, dexpramipexole, is entering Phase 3 development in hypereosinophilic syndrome and Phase 2 clinical studies in 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.

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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