Global biopharmaceutical company Ipsen has announced that its FALKON trial failed to hit primary endpoint in rare bone disorder study. As a result, the study will be closed.

 

The investigational drug, fidrisertib, was generally well tolerated in patients with fibrodysplasia ossificans progressiva (FOP), with no safety concerns reported.

 

“These results are disappointing for the FOP community and patients living with this devastating disease,” said Christelle Huguet, Head of Research and Development. 

 

“However, we do believe that these data will contribute to the growing body of research on FOP, giving new insights into managing this disease for patients and their care providers. FALKON was a tremendous undertaking by Ipsen, continuing our commitment to FOP. FALKON enrolled 113 patients globally, taking over 5 years to reach this critical milestone. 

 

"We would like to thank the patients, caregivers, the FOP community and KOLs who dedicated their time to the FALKON trial, it has been a serious commitment to help advance our understanding of FOP.”

 

FOP is a rare genetic disorder caused by mutations in the ALK2 kinase, leading to irreversible bone growth in muscles, tendons, and ligaments. Diagnosis typically occurs around age five, and the disease shortens life expectancy to a median of 56 years due to complications like ribcage bone formation and breathing difficulties. Worldwide, roughly 900 people are diagnosed, though numbers vary by country.

 

Fidrisertib, an oral, highly selective inhibitor of the ALK2 kinase, was developed to target both flare-up and non-flare-up based HO formation in FOP. 

 

The FALKON trial, the largest Phase II study in this rare disease, enrolled 113 patients aged five and older with FOP-associated mutations. The trial evaluated two dose levels of fidrisertib against placebo and included a multi-part design with extension periods for responding patients.

 

Despite the setback, Ipsen emphasized the value of the data for future FOP research and patient care.