Treatment with givinostat alongside corticosteroids continues to delay disease progression — and maintain patients’ ability to walk — in boys with Duchenne muscular dystrophy (DMD), new data from a long-term Phase 2/3 clinical study show.
The new data, from an ongoing seven-year study, was presented by Paolo Bettica, MD, PhD, chief medical officer at Italfarmaco, givinostat’s maker, at the virtual 28th International Conference on Duchenne and Becker Muscular Dystrophy, held online Feb. 20.
“We are very encouraged to see that the long-term study with Givinostat continues to show a benefit in boys with DMD, which further supports its potential as a treatment,” Bettica said in a press release.
The study (NCT03373968) is an extension of an earlier Phase 2 trial (NCT01761292) and is testing givinostat’s long-term safety and tolerability when used with corticosteroids among boys who were ages 7 to 11 at the start of treatment. Givinostat is given as an oral suspension (10 mg/mL) twice daily, after meals.
After more than seven years, the mean (average) age at which boys treated with givinostat lose the ability to walk was 16. By comparison, boys on corticosteroids only for at least a year lost ambulation, or walking ability, at a mean age of 13.4, as found in an earlier study conducted by the Cooperative International Neuromuscular Research Group.
Respiratory function declined less per year among boys taking givinostat than comparable patients participating in prior natural history studies. Forced vital capacity and peak expiratory flow, which assess the amount of air and and the peak flow when a person forcefully exhales, fell by 1.7% to 0% compared with the 4% to 6% yearly decline seen in the other studies.
“Overall these results suggest a potential long-term beneficial effect of Givinostat in DMD boys,” said Eugenio Mercuri, PhD, professor of pediatric neurology at the Catholic University, in Rome.
“We look forward to the final results and remain hopeful that these results can support the registration of Givinostat for the treatment of DMD,” Mercuri said.
Givinostat is intended to promote muscle repair, reduce inflammation, and lessen fat and fibrosis (scarring). It works by blocking enzymes called histone deacetylases (HDACs), which limit genes’ accessibility. Overactive HDACs are thought to contribute to muscle degeneration in DMD and related conditions.
Last year, the medication received rare pediatric disease designation in the United States, according to information provided by Bettica in a webinar in September. The U.S. Food and Drug Adminstration also has awarded givinostat orphan drug and fast track status. Collectively, these designations aim to bring promising medicines targeting unmet needs to market more quickly.
The therapy also is being tested in a pivotal Phase 3 trial called EPYDIS (NCT02851797), expected to run through March 2022. In that trial, which has enrolled 179 boys ages 6 to 17, the therapy is being compared with a placebo on measures of motor function and of muscle replacement by fat.
“Our Phase 3 pivotal clinical study is on track and continues as planned,” Bettica said, “and we look forward to announcing the results in the second quarter of next year.”
“We have made significant progress despite the pandemic and instituted procedures to ensure the safety and well-being of all trial participants while being able to continuously provide access to the study drug, as well as to maintain the scientific validity and integrity of the trial,” he added.
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