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Clementia rare disease drug cuts abnormal bone growth in phase 2 study

Montreal-based Clementia Pharmaceuticals Inc. said the phase 2 trial of palovarotene showed that patients with a certain rare bone disorder saw meaningful improvements when given the therapy.

In part B of the ongoing phase 2 study, dubbed Move, Clementia said palovarotene-treated patients with fibrodysplasia ossificans progressiva, or FOP — a condition where bone forms outside the normal skeleton — saw a meaningful reduction in abnormal bone growth.

Statistically significant results showed a 91% reduction in new bone growth in abnormal places at flare-up sites after 12 weeks compared to untreated flare-up sites from patients in Clementia's natural history study and the placebo group.

Further, evaluable patients who had no new abnormal bone growth at 12 weeks also saw no new bone growth in abnormal places at 12 months. Clementia said the data suggests that flare-up data could potentially predict longer-term outcomes.

Palovarotene was well-tolerated by patients under the study. The part B portion of the Move study enrolled 53 patients.

FOP progressively restricts a sufferer's movement by causing joints to lock, leading to function loss, progressive disability and risk of early death.