Harmony Bio Acquires Epygenix, Adding Late-Stage Rare Epilepsy Assets

Rare Daily Staff

Harmony Biosciences has acquired Epygenix Therapeutics, accelerating its growth strategy by adding a rare epilepsy franchise to its late-stage pipeline of central nervous system assets.

Under the terms of the 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.

“The acquisition of Epygenix gives us three distinct CNS franchises in late-stage development, each with a potential U.S. peak sales opportunity of $1 billion to $2 billion,” said Jeffrey Dayno, president and CEO at Harmony Biosciences. “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.”

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 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 and Rare Pediatric Disease designations from the U.S. Food and Drug Administration 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 FDA Orphan Drug designation for Dravet syndrome and Lennox-Gastaut syndrome as well as Rare Pediatric Disease designation for Lennox-Gastaut syndrome.

“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,” said Alex Yang, chairman and CEO of Epygenix Therapeutics.

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 percent of Dravet syndrome cases are caused by de novo loss-of-function mutations in a voltage-gated sodium channel gene, SCN1A1.

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. As a result, patients experience multiple seizure types, including atonic seizures, and developmental, cognitive, and behavioral issues.

Photo: Jeffrey Dayno, president and CEO at Harmony Biosciences