Ipsen Terminates Clinical Trial on Progressive Fibrodysplasia Ossificans
French laboratory Ipsen has announced the discontinuation of its Phase II FALKON trial on fidrisertib, following the failure to meet the primary endpoint. The candidate drug was aimed at treating progressive fibrodysplasia ossificans, an ultra-rare bone disease.
Results of the Phase II FALKON Trial
The pivotal Phase II FALKON trial did not demonstrate a reduction in the volume of abnormal new bone formation, known as heterotopic ossification, compared to placebo, the group reports. The study involved adult and child patients with progressive fibrodysplasia ossificans. As a result of these findings, the trial will be closed, the statement specifies. However, the experimental drug fidrisertib was generally well tolerated, with no safety concerns identified during the trial, according to the company. Christelle Huguet, Director of Research and Development at Ipsen, described these results as disappointing for the patient community affected by this disease.
Scope and Structure of the FALKON Trial
The FALKON trial represents the largest Phase II trial ever conducted on progressive fibrodysplasia ossificans, according to the laboratory. Structured in three parts, it aimed to assess the efficacy, safety, and tolerability of fidrisertib as a first-line treatment. Part A of this global, multicenter trial recruited 113 patients aged 5 years or older, carrying the R206H mutation of the ACVR1 gene or other associated variants. Participants were randomized to receive either a high dose of fidrisertib, a low dose, or a placebo, the statement details. The primary endpoint focused on the annual change in the volume of heterotopic ossification compared to the baseline value.
About Progressive Fibrodysplasia Ossificans
Progressive fibrodysplasia ossificans is a genetic disease caused by pathogenic variants of the ALK2 kinase, leading to irreversible bone formation in soft and connective tissues. Treatment options for patients with this condition are currently limited, Ipsen states. The disease is typically diagnosed at an average age of 5 years and reduces life expectancy to a median age of 56 years, according to the statement. The prevalence of progressive fibrodysplasia ossificans is approximately 1.36 per million individuals worldwide, the group specifies. Fidrisertib is a selective small molecule inhibitor targeting pathogenic variants of the ALK2 kinase, administered orally in capsule form.
