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.

The best of times, the worst of times, for palovarotene
Feb 2019 Ipsen buys Clementia for $1bn
Dec 2019 FDA partial hold on palovarotene studies, in patients under 14 years
Dec 2019 Ipsen's chief executive, David Meek, departs
Jan 2020 Move trial stopped for futility
Mar 2020 Ipsen restarts dosing in palovarotene studies in patients aged 14 and older
Aug 2020 Ipsen reports data from post-hoc analysis of Move
Source: company releases.

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