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Subject: FDA

Epygenix Therapeutics Receives U.S. FDA Orphan Drug Designation for EPX-200 in Lennox-Gastaut Syndrome

PARAMUS, N.J., Aug. 2, 2021 /PRNewswire/ -- Epygenix Therapeutics, Inc., a clinical stage biopharmaceutical company focused on rare and intractable genetic epilepsy, today announced that the U.S. Food and Drug Administration (FDA) granted orphan drug designation to EPX-200 for the treatment of Lennox-Gastaut Syndrome (LGS). LGS is a rare epileptic encephalopathy presenting in childhood with intractable seizures. Approximately 48,000 children and adults in the United States currently suffer with LGS. Characterized by a triad of signs including multiple seizure types, slow spike-wave complexes on electroencephalographic (EEG) recordings, and impairment of cognitive function, therapeutic options for LGS are desperately needed as seizure control with currently available treatments is not adequate.

"Granting Orphan Drug Designation for LGS to EPX-200 is one of the company's significant development milestones, because the Company is expanding targeted indications to multiple refractory epilepsies, including LGS. The FDA's ODD grant to EPX-200 for LGS recognizes the significance of EPX-200 as a drug candidate for these patients. Building on discoveries made in zebrafish models for rare genetic epilepsies, we have several drug candidates moving through the clinical trial process, and will be able to test the safety and efficacy of EPX-200 in LGS patients soon," stated Hahn-Jun Lee, CEO of Epygenix Therapeutics.

Dr. Scott C. Baraban, Ph.D., Professor & William K. Bowes Jr. Endowed Chair in Neuroscience Research at UCSF and Chair of the Scientific Advisory Board at Epygenix Therapeutics, Inc. issued a joint comment that "This is a long-awaited step toward finding and developing new drugs to treat the devastating early childhood seizures seen in patients with LGS. We are quite hopeful that new therapeutic options will quickly get to this patient population."

Alex Yang, J.D., LLM, President and CEO of Mstone Partners and Chair of the Board at Epygenix Therapeutics also stated that "Epygenix is committed to provide multiple drug options that are safe, effective and patient-friendly for a broad category of refractory epilepsies. We are very excited with another milestone towards commercialization."

Under the U.S. Orphan Drug Act, the FDA's Office of Orphan Products Development provides sponsors with special status and incentives to facilitate the drug development for rare disease affecting fewer than 200,000 people in the U.S. Orphan Drug Designation provides seven years of market exclusivity if the drug candidate receives regulatory approval together with tax credits for qualified clinical trial cost, exemptions from certain FDA application fees, and assistance in clinical trial design.

About Epygenix Therapeutics, Inc.

Epygenix Therapeutics, Inc. is a clinical stage biopharmaceutical company focused on developing drugs to treat rare and intractable genetic epilepsy. Epygenix is currently focused on developing EPX-100, -200, and -300. These candidates abolish convulsive behavior and electrograhic seizure activity and were discovered in a zebrafish Dravet Syndrome model which mimics the human pathology and confirmed its validity by the human efficacy with EPX-200 and -300. For more information, please visit www.epygenix.com.

About EPX-200

EPX-200 is a weight gain management drug which acts via modulation of serotonin (5HT) signaling pathways. EPX-200 was firstly identified using a zebrafish disease model for Dravet Syndrome. EPX-200 also has been reported to have anti-seizure effects based on the result from small number of patients.

Media Contact
Hahn-Jun Lee, M.Sc., Ph.D.

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SOURCE Epygenix Therapeutics, Inc.

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