Ipsen throws itself a bone with palovarotene data dredge

At the beginning of this year Ipsen’s rare bone disease project palovarotene looked dead and buried. The phase III Move study, in fibrodysplasia ossificans progressiva (FOP), was stopped in January after a futility analysis found it was unlikely to demonstrate a benefit with palovarotene versus untreated natural history patients on its primary endpoint, change in new heterotopic ossification (HO) – the abnormal bone growth seen in FOP. Since then the company has been pursuing a post-hoc analysis for a way forward, and details were released today. On a slightly different endpoint, mean annualised new HO volume, palovarotene generated a 62% reduction versus untreated patients. The company plans to file the project in the US, and says that discussions with EU regulators are ongoing. The FDA might be in a forgiving mood given that there is nothing else approved for FOP. But toxicity worries could yet scupper palovarotene: the early growth plate closure that led to a partial clinical hold last year was seen in 27% of skeletally immature patients, Ipsen said today. It is understandable that the French group wants to recoup something from its $1bn acquisition of palovarotene’s developer, Clementia, but it could be throwing good money after bad.