Harmony Biosciences Jumps on Positive Epilepsy Drug Data
Shares rise after company's EPX-100 drug candidate shows a 50% reduction in seizure frequency for patients with severe, treatment-resistant Dravet syndrome.
Shares of Harmony Biosciences (NASDAQ: HRMY) surged in morning trading after the pharmaceutical company presented encouraging new data for its experimental drug, EPX-100, in patients with Dravet syndrome, a rare and severe form of epilepsy.
The stock climbed 4.13% to $39.83 as investors reacted to the news, which positions the company to address a significant unmet need in pediatric neurology. The data, from an open-label extension of its Phase 3 ARGUS trial, showed the drug achieved a median reduction of approximately 50% in the frequency of countable motor seizures.
Harmony Biosciences presented the findings at a medical conference, highlighting that 50% of patients in the study experienced at least a 50% reduction in seizures. Dravet syndrome is a notoriously difficult-to-treat condition that begins in infancy and is characterized by frequent, prolonged, and treatment-resistant seizures, alongside developmental delays.
"These are clinically meaningful results for a patient population that continues to face a significant burden from seizures," said Dr. Kumar Budur, Chief Medical Officer of Harmony Biosciences. "The sustained effectiveness and safety profile observed in this study are highly encouraging as we work to bring a new treatment option to individuals living with Dravet syndrome."
The positive data serves as a crucial "de-risking event" for a key asset in Harmony's pipeline, according to analysts at H.C. Wainwright & Co. The results bolster confidence in EPX-100's potential as Harmony, with a market capitalization of $2.29 billion, seeks to diversify its portfolio beyond its primary commercial product, WAKIX, which is approved for treating excessive daytime sleepiness in adults with narcolepsy.
The market for Dravet syndrome treatments is substantial and growing, with some market analyses projecting it could reach over $1 billion by 2035. Success in this area would represent a significant new revenue stream for the Pennsylvania-based company.
Wall Street has maintained a bullish outlook on the company, with a consensus "Strong Buy" rating from analysts covering the stock. The average 12-month price target sits at approximately $46.18, suggesting further upside even after the recent gains. The stock is now trading near its 52-week high of $40.93.
The open-label extension study followed patients who had completed the initial 16-week double-blind portion of the Phase 3 trial. The sustained reduction in seizure frequency over a longer period is a key indicator of the drug's potential long-term efficacy and durability. The company also reported a favorable safety and tolerability profile, a critical factor for any therapy intended for a pediatric population with a chronic condition.
Moving forward, Harmony is expected to continue its analysis of the full data set and engage with regulatory authorities, including the U.S. Food and Drug Administration (FDA), to determine the next steps for a potential new drug application. The progress of EPX-100 will be closely watched by investors as a primary catalyst for the company's future growth.