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Ipsen update on Phase II FALKON trial in patients with ultra-rare bone disease, fibrodysplasia ossificans progressiva (FOP)
PARIS, FRANCE, 19 December 2025 - Ipsen (Euronext: IPN; ADR: IPSEY) today announced that the pivotal Phase II FALKON trial did not meet its primary endpoint of reducing new heterotopic ossification (HO) in adults and children living with fibrodysplasia ossificans progressiva (FOP) vs. placebo, as a result the study will be closed. The investigational medicinal product (fidrisertib) was generally well tolerated, with no safety concerns in the trial. “These results are disappointing for the FOP co
About this update from Ipsen Sa
[{"type":"image","alt":"Ipsen Pharma","displaySize":"","headline":null,"caption":"Ipsen Pharma","className":"","disableSlideshowImg":false,"size":{"original":{"width":300,"height":157,"url":"https://media.zenfs.com/en/globenewswire.com/e6eb628cc0e353381e6b738c556a0e31"},"resized":{"url":"https://s.yimg.com/ny/api/res/1.2/89RavcsKEHEdDt270X336Q--/YXBwaWQ9aGlnaGxhbmRlcjt3PTQyMDtoPTIyMDtjZj13ZWJw/https://media.zenfs.com/en/globenewswire.com/e6eb628cc0e353381e6b738c556a0e31","width":300,"height":157}},"lazy":false},{"type":"text","content":"PARIS, FRANCE, 19 December 2025 - Ipsen (Euronext: IPN; ADR: IPSEY) today announced that the pivotal Phase II FALKON trial did not meet its primary endpoint of reducing new heterotopic ossification (HO) in adults and children living with fibrodysplasia ossificans progressiva (FOP) vs. placebo, as a result the study will be closed. The investigational medicinal product (fidrisertib) was generally well tolerated, with no safety concerns in the trial.","length":457,"tagName":"p"},{"type":"text","content":"“These results are disappointing for the FOP community and patients living with this devastating disease,” said Christelle Huguet, PhD, EVP, 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.”","length":727,"tagName":"p"},{"type":"text","content":"About FOP","length":9,"tagName":"p"},{"type":"text","content":"FOP is a genetic condition caused by pathogenic variants of the ALK2 kinase that leads to bone formation in soft and connective tissues, like muscles, tendons and ligaments, a process known as HO. Once formed, HO is irreversible. There are limited treatment options for patients with FOP. The average/median age of diagnosis is 5 years old and ultimately FOP shortens the life expectancy to a median of 56 years, with untimely death caused by bone formation around the ribcage, leading to breath...