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– MRI data showed that although the primary endpoint was not reached, Givinostat counteracted muscle degeneration, supporting its continued development as a treatment for Becker muscular dystrophy (BMD)-
– The company will meet with regulatory authorities to discuss Givinostat’s future development plan in BMD –
– The safety of Givinostat is consistent with previous observations –
Famaco Group Today announced the top-line data of its Phase 2 proof-of-concept trial using Givinostat in 51 adult men with Becker Muscular Dystrophy (BMD). Givinostat is the company’s proprietary histone deacetylase (HDAC). ) Inhibitors. This study aims to evaluate the effect of Givinostat on BMD and is based on the experience of Duchenne muscular dystrophy. Based on this experience, changes in total fibrosis in muscle biopsy were selected as the primary endpoint of the study, and changes in fat fraction and contractile CSA from quantitative magnetic resonance imaging (MRI) were used as key secondary endpoints. Compared with placebo, Givinostat did not show a significant difference in the primary endpoint. However, the significant difference between MRI of the entire thigh and quadriceps muscles and placebo confirms the ability of Givinostat to counteract muscle degeneration in BMD patients receiving treatment. The safety of Givinostat in BMD is consistent with previous studies, and no serious safety problems have been observed. Based on the overall results, the company plans to meet with US and EU regulators to discuss the next steps in the development of Givinostat in adult BMD.The top line data was presented by Paolo Bettica on June 26, 2021 on the virtual Parent Project Muscular Dystrophy (PPMD) Annual Meeting.
“Becker muscular dystrophy is a rare and debilitating disease. There is currently no treatment. The significant difference in muscle fat infiltration between the two groups after 12 months is very encouraging to us, which shows that Givinostat is slowing down muscle Degradation has a beneficial effect. These, and as other measures, support the further development of Givinostat in the BMD field, and we will evaluate the best way forward in discussions with regulators,” Paolo Bettica, MD and PhD, Chief Medical Officer of Italfarmaco Group said“We will continue to analyze the data to better understand the failure to reach the primary endpoint, which may be due to the high variability and imbalance of baseline histological parameters between the cohorts and the lack of progress within 12 months of the study duration. ”
Dr. Bettica added: “Although this BMD clinical trial is an independent study, the study further confirms the ability of Givinostat to prevent downstream pathogenic effects caused by defects in the dystrophin gene. Seen in young boys with Duchenne muscular dystrophy.”
The Phase 2 trial of Givinostat in BMD patients is a randomized, double-blind, placebo-controlled study (ClinicalTrials.gov: NCT03238235). A total of 51 patients between the ages of 19 and 61 were randomly divided into groups at a 2:1 ratio and were given Givinostat or placebo oral suspension twice a day for 12 months. Among the patients who participated in the study, 30 out of 34…
The full story on Benzinga.com
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