• Trial met primary endpoint: givinostat demonstrated statistically and clinically meaningful treatment benefit in one of the largest DMD phase 3 trials to date.
• Givinostat was well tolerated with a well-documented safety profile consistent with previous givinostat trials.
• Italfarmaco anticipates U.S. Food and Drug Administration (FDA) PDUFA response scheduled for March 21, 2024.

March 19, 2024 12:25 PM Eastern Daylight Time

MILAN--(BUSINESS WIRE)--Italfarmaco S.p.A. announced today that the full results from the Company’s pivotal phase 3 EPIDYS clinical trial with givinostat in ambulant boys 6 years of age and older with Duchenne muscular dystrophy (DMD) have been published in The Lancet Neurology. The EPIDYS clinical trial met its primary endpoint: change in four-stair climb assessment from baseline to 72 weeks, which demonstrated the potential of givinostat to delay disease progression when added to corticosteroid treatment. Givinostat-treated boys also showed favorable outcomes on key secondary endpoints assessed in the study. The full manuscript, titled, “Safety and efficacy of givinostat in boys with Duchenne muscular dystrophy (EPIDYS): a multicentre, randomized, double-blind, placebo-controlled, phase 3 trial,” is published on The Lancet Neurology website.

“DMD is an unforgiving degenerative disease with significant unmet medical need and givinostat has the potential, if approved, to benefit a broad DMD patient population independent of the underlying gene mutation,” said Eugenio Mercuri, MD, Professor of Paediatric Neurology at the Catholic University, Rome, Italy. “The givinostat-treated group fared better than the control group in a range of muscle function and strength tests at 72 weeks.”

“When managing DMD, a primary goal is to maintain motor function for as long as possible. The results from EPIDYS provide robust evidence that givinostat has the potential to support this goal,” added Craig M. McDonald, MD, Professor at the Department of Pediatrics and Physical Medicine Rehabilitation at the University of California Davis Health. “In addition to the primary endpoint, analysis of the North Star Ambulatory Assessment (NSAA), which measures motor function skills based on a 17-item rating scale, showed that treatment with givinostat resulted in 40% less decline compared to the control group. Taken together, these data suggest givinostat could be an effective new treatment for DMD management.”

“The results of the phase 3 EPIDYS study are encouraging and highlight the dedication of Italfarmaco’s research and clinical teams to achieve this milestone for the company,”