Clementia reports the top-line results of its Phase 2 clinical trial for palovarotene in fibrodysplasia ossificans progressiva.
We would like to thank the FOP patient and medical communities without whom our Phase 2 program could not have succeeded. The investigators, their clinical research teams, and the subjects, families and caregivers provided leadership, support, commitment, and participation throughout the program. We enrolled 40 subjects in the Phase 2 trial and approximately 100 in the natural history study, and both trials have produced learnings that serve to advance the goals of the FOP community to understand the disease and to develop treatments.
- What was the purpose of Clementia’s Phase 2 trial?
- The purpose of the FOP clinical program is to develop evidence that supports marketing authorization (MA) for palovarotene as a treatment for FOP patients. The objective of our Phase 2 was to inform the design of a Phase 3 registrational trial by investigating whether palovarotene prevents heterotopic ossification (HO) following a flare-up, at what dose(s), and with what side effects.
- What observations did you make from your Phase 2 trial?
- We observed several positive trends though none reached statistical significance in this study.
- We observed that in the 40 subject placebo-controlled Phase 2 dose-ranging trial, no subject required a reduction in dose due to tolerability issues nor was discontinued from the trial.
- We observed that those on placebo were at 2.6 times greater risk of forming HO than those on the palovarotene 10/5 mg treatment. In addition, those on either palovarotene regimen with new HO formed less HO than those on placebo.
- We observed palovarotene-related reductions in patient-reported pain associated with flare-ups and reductions in the time to resolution of flare-ups.
- What are the next steps for your FOP clinical program?
- We are designing and plan to implement a Phase 3 registrational trial and a surgical excision trial as soon as possible in 2017.
- We will continue to press forward with the collaboration of all stakeholders, including patients, physicians, and regulatory authorities, on our goal to deliver a much needed potential therapy for all FOP patients.