Canada-based Clementia Pharmaceuticals has completed patient enrolment in its Phase II clinical trial of palovarotene, a retinoic acid receptor gamma agonist, to treat fibrodysplasia ossificans progressiva (FOP).
FOP is a rare disease that causes progressively disabling heterotopic bone formation (heterotopic ossification) in muscle and soft tissues.
The Phase II trial is to evaluate the effect of palovarotene on new bone formations during and after a 'flare-up', an inflammatory event of varying severity and duration that often precedes heterotopic ossification.
The company noted the trial's multiple clinical endpoints, including imaging endpoints for new bone formation, and clinical assessments of physical function and patient-reported outcomes.
Top-line data from the trial is expected to be reported by the end of this year.
Perelman School of Medicine Molecular Orthopaedic Medicine Division chief Dr Frederick Kaplan said: "The data from this trial will be instrumental in designing the next steps in Clementia's clinical programme for FOP, as well as furthering our understanding of FOP and the impact that flare-ups have on the course of the disease."
Preclinical trials of palovarotene show that it blocked abnormal bone formation in animal models.
The company had licensed the treatment from Roche Pharmaceuticals, which previously evaluated it in more than 800 subjects.
Palovarotene also secured fast-track designation from the US Food and Drug Administration (FDA), as well as orphan designations to treat FOP from both the FDA and the European Medicines Agency (EMA).
Clementia founder and CEO Clarissa Desjardins said: "Completing enrolment in the Phase II trial represents another significant milestone in our clinical programme and moves us closer to our goal of potentially bringing a much-needed treatment to people with FOP.
"We are grateful to the study participants and their families, the FOP community, and the clinical trial site teams for their commitment to this effort."