Harmony Biosciences Acquires Epygenix Therapeutics

Harmony Biosciences (Nasdaq: HRMY), today announced the acquisition of Epygenix Therapeutics, Inc., accelerating its growth strategy by adding a rare epilepsy franchise to its expanding late-stage pipeline of innovative CNS assets.

“The acquisition of Epygenix gives us three distinct CNS franchises in late-stage development, each with a potential US peak sales opportunity of $1B – $2B,” said Jeffrey M. Dayno, M.D., President and Chief Executive Officer at Harmony Biosciences. “We have been strategically building a robust and diverse pipeline of innovative CNS assets for rare diseases that has transformed our business. This acquisition builds on our leadership position in sleep/wake and our franchise in neurobehavioral disorders, allowing us to leverage our expertise in CNS and internal synergies to deliver new therapies to patients with unmet medical needs.”

“Harmony shares our team’s core values, including a commitment to patients and families living with Dravet syndrome and Lennox-Gastaut syndrome, where a significant unmet need for effective therapies remains,” said Alex Yang, Chair and CEO of Epygenix Therapeutics. “Harmony’s demonstrated expertise in drug development and commercialization will accelerate the full potential of these treatments and make a real difference in the lives of Dravet and LGS families.”

The acquisition includes clemizole hydrochloride (EPX-100), a potent, oral, centrally acting serotonin (5HT2) agonist. It is currently in a pivotal registrational clinical trial for the treatment of Dravet syndrome in children and adults and is poised to enter Phase 3 for the treatment of Lennox-Gastaut syndrome. The proven mechanism of action of clemizole could potentially offer an improved product profile over currently available treatment options and improve daily functioning in patients living with Dravet syndrome and Lennox-Gastaut syndrome.

EPX-100 has received Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) from the FDA for both Dravet syndrome and Lennox-Gastaut syndrome. A second investigational product, EPX-200, is a potent, oral, centrally active and selective 5HT2C agonist, and is currently in IND-enabling studies. EPX-200 also received ODD from FDA for Dravet syndrome and Lennox-Gastaut syndrome as well as RPDD for Lennox-Gastaut syndrome.

Under the terms of the definitive agreement, Harmony paid $35 million in cash with the potential for payments of up to $130 million based on development and regulatory milestones. In addition, there are potential payments of up to $515 million if certain sales milestones are achieved.

About Clemizole hydrochloride (EPX-100)

EPX-100, clemizole hydrochloride, is under development for the treatment of Dravet syndrome (DS) and Lennox-Gastaut syndrome (LGS). EPX-100 acts by targeting central 5-hydroxytryptamine receptors to modulate serotonin signaling. The drug candidate is administered orally twice a day in a liquid formulation and has been developed based on a proprietary phenotype-based zebrafish drug screening platform.1 DS is caused by a loss of function mutation in the SCN1A gene, and scn1 mutant zebrafish replicate the genetic etiology and phenotype observed in the majority of DS patients. The scn1Lab mutant zebrafish model that expresses voltage gated sodium channels has been used for high-throughput screening of compounds that modulate Nav1.1 in the central nervous system.

About Dravet syndrome

Dravet syndrome (DS) is a severe and progressive epileptic encephalopathy that begins in infancy and causes significant impact on patient functioning. DS begins in the first year of life and is characterized by high seizure frequency and severity, intellectual disability, and a risk of sudden unexpected death in epilepsy.Approximately 85% of Dravet syndrome cases are caused by de novo loss-of-function (LOF) mutations in a voltage-gated sodium channel gene, SCN1A1.2 DS has an estimated incidence rate of 1:15,7003.

About Lennox-Gastaut syndrome

Lennox-Gastaut syndrome (LGS) is a rare and drug-resistant epileptic encephalopathy characterized by onset in children between 3-5 years of age. The underlying cause of LGS is unknown and can be related to a wide range of factors including genetic differences and structural differences in the brain.2,4 As a result, patients experience multiple seizure types, including atonic seizures, and developmental, cognitive, and behavioral issues.3 LGS affects approximately 48,000 patients in the U.S5.

About Harmony Biosciences

At Harmony Biosciences, we specialize in developing and delivering treatments for rare neurological diseases that others often overlook. We believe that where empathy and innovation meet, a better life can begin for people living with neurological diseases. Established by Paragon Biosciences, LLC, in 2017 and headquartered in Plymouth Meeting, PA, our team of experts from a wide variety of disciplines and experiences is driven by our shared conviction that innovative science translates into therapeutic possibilities for our patients, who are at the heart of everything we do. For more information, please visit www.harmonybiosciences.com.

About Epygenix Therapeutics, Inc.

Epygenix Therapeutics, Inc. is a precision medicine-based biopharmaceutical company focused on genetically screening, discovering, and developing drugs to treat rare and intractable forms of genetic epilepsy in childhood, such as Dravet syndrome (DS). Epygenix is currently focused on developing EPX-100 and EPX-200, drug candidates that were discovered in a zebrafish model of DS which replicates the genetic mutation that causes DS and mimics the human pathology. This mechanism of action has been shown to eliminate both clinical and electrographic seizure activity which could translate into improved patient functioning.