12 October 2020 - - Italy-based specialty pharmaceutical company The Italfarmaco Group has provided an update on the development of Givinostat, its proprietary histone deacetylase inhibitor, in boys with Duchenne Muscular Dystrophy.

The US Food and Drug Administration granted a Rare Pediatric Disease designation to Givinostat for the treatment of DMD, which allows an expedited review process for new treatment modalities.

The company also announced the completion of patient enrollment in the EPIDYS Phase 3 trial on September 25th, 2020 and remains on track with its proposed timelines for reporting topline data in June 2022.

The Rare Pediatric Disease designation is granted by the FDA to encourage development of new drugs for serious or life-threatening manifestations of a disease or condition that primarily affect children 18 years of age and younger.

Subject to FDA approval of Givinostat for the treatment of DMD, Italfarmaco would be eligible to receive a voucher, which may be redeemed to receive priority review for a subsequent marketing application for a different product candidate or which could be sold or transferred.

In April 2020, Italfarmaco announced that interim data from the EPIDYS Phase 3 clinical trial was reviewed by an Independent Data Monitoring Committee and that the trial was recommended to continue based on the lower limb muscle fat infiltration seen in the Magnetic Resonance Spectroscopy images of DMD patients with Givinostat treatment compared to the placebo group after 12 months of treatment.

These MRS results corroborate the histological results of the company's Phase 2 study, which revealed slower disease progression in boys treated with Givinostat and on a stable regimen of steroids.

The Independent Data Monitoring Committee, which regularly monitors and reviews the study, confirmed the safety of the treatment with Givinostat.

With trial enrollment completed, the last patient is expected to complete the 72-week treatment period in the first quarter of 2022. Italfarmaco is expected to report topline data from the Phase 3 EPIDYS trial in June 2022.

Givinostat is an investigational drug discovered through Italfarmaco's internal research and development efforts in collaboration with Lorenzo Puri (Sanford Burnham Prebys Medical Research Institute, San Diego, formerly Santa Lucia Foundation, Rome) and his team, and partnerships with Telethon and Parent Project aps.

It is being evaluated for safety and efficacy for the treatment of Duchenne- and Becker- Muscular Dystrophy.

Givinostat inhibits histone deacetylases (HDACs). HDACs are enzymes that prevent gene translation by changing the three-dimensional folding of DNA in the cell.

Studies show that Duchenne patients have higher than normal HDACs activity, which may prevent muscle regeneration, and also trigger inflammation.

In the company's clinical study in DMD, boys aged 7 to less than 11 years, Givinostat was observed to slow disease progression, significantly increase muscle mass and reduce the amount of fibrotic tissue.

Givinostat treatment also significantly reduced muscle tissue necrosis and fatty replacement, two additional parameters related to disease progression (Bettica et al., Neuromuscular Disorder 2016).

Italfarmaco is a specialty pharmaceutical company engaged in the discovery, development, manufacturing and marketing of branded prescription and nonprescription products in more than 60 countries on 5 continents.

Italfarmaco's research and development expertise is best demonstrated through its HDAC inhibitor development programs, addressing new therapeutic treatments of specialty and rare diseases.

Through both marketed drugs and compounds in development, Italfarmaco is dedicated to serving patients whose needs remain largely unmet.