Joins Global Rare Disease Community in Recognizing Rare Disease Day 2016
MONTREAL, Feb. 29, 2016 /CNW/ -- Clementia Pharmaceuticals Inc. today announced that the 40th and last study participant has enrolled into its Phase 2 clinical trial that is investigating palovarotene for the treatment of fibrodysplasia ossificans progressiva (FOP), an extremely rare disease that causes progressively disabling heterotopic bone formation (heterotopic ossification) in muscle and soft tissues.
The randomized, double-blind, placebo-controlled Phase 2 trial is a dose-ranging study designed to evaluate the effect of different doses of palovarotene on new bone formation during and after a flare-up, an inflammatory event of varying severity and duration that often precedes heterotopic ossification. Multiple clinical endpoints are evaluating the efficacy and safety of palovarotene, including imaging endpoints for new bone formation, clinical assessments of physical function, and patient-reported outcomes. Top-line data is expected by year's end.
"I'm pleased to be participating in this important study, which represents an achievement not only for Clementia but for the whole FOP community," said Frederick Kaplan, M.D., The Isaac & Rose Nassau Professor of Orthopaedic Molecular Medicine and Chief of the Division of Molecular Orthopaedic Medicine at the Perelman School of Medicine at the University of Pennsylvania. "The data from this trial will be instrumental in designing the next steps in Clementia's clinical program for FOP, as well as furthering our understanding of FOP and the impact that flare-ups have on the course of the disease."
"Completing enrollment in the Phase 2 trial represents another significant milestone in our clinical program and moves us closer to our goal of potentially bringing a much-needed treatment to people with FOP," said Clarissa Desjardins, Ph.D., CEO and Founder. "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."
Clementia recognizes the unique, profound effect that rare diseases, such as FOP, have on affected families. As such, in support of International Rare Disease Day (Feb. 29), its employees will participate in events in North America and Europe that will raise awareness of rare diseases and provide support to families affected by them. Across the world, hundreds of local events will bring together legislators, medical professionals, patients and caregivers and will recognize the powerful role that the patient community plays in instigating change.
United States: Employees will join families affected by rare disease and health care advocates at a Rare Disease Day 2016 Event and Forum (Feb. 29) to be held at the Massachusetts State House and sponsored by the Massachusetts Biotechnology Council (MassBio) and other local groups.
Canada: Employees along with advocates from the Canadian FOP community will participate in the Canadian Organization for Rare Disorders (CORD) two day conference and Rarity Awards Dinner Gala (March 9-10) in Ottawa, Canada.
Europe: Employees along with advocates from the European FOP community attended the EURORDIS Awards and Black Pearl Evening (February 23) in Brussels, Belgium.
To learn more about Clementia, visit www.clementiapharma.com.
Dr. Frederick Kaplan is the Global Principal Investigator for the Phase 2 Study. He is not a paid consultant to Clementia.
About Rare Disease Day
Rare Disease Day takes place every year on the last day of February (February 28 or February 29 in a leap year), the rarest date on the calendar, to underscore the nature of rare diseases and the challenges patients face. The worldwide awareness day was established in Europe in 2008 by EURORDIS, the organization representing rare disease patients in Europe, and is now observed in more than 80 nations, including Canada and the United States. For more information, visit www.rarediseaseday.org, www.rarediseaseday.us or http://www.raredisorders.ca.
About Fibrodysplasia Ossificans Progressiva (FOP)
FOP is a rare, severely disabling myopathy characterized by heterotopic ossification (HO) of muscle and soft tissues. Heterotopic bone formation progressively restricts movement by locking joints and leads to cumulative loss of function, disability, and increased risk of early death. Virtually all newborns with FOP have a hallmark toe malformation in which both big toes are shortened and bent inwards. FOP is caused by a mutation in the ACVR1 gene resulting in increased activity of the activin receptor type I (ACVR1/ALK2) involved in the bone morphogenic protein (BMP) pathway, a key pathway controlling bone growth and development. There are currently no approved treatments for FOP.
Palovarotene is a retinoic acid receptor gamma agonist being investigated as a treatment for FOP. Preclinical studies demonstrated that palovarotene blocked abnormal bone formation in animal models. Clementia licensed palovarotene from Roche Pharmaceuticals, which previously evaluated the compound in more than 800 subjects. Palovarotene received Fast Track designation from the U.S. Food and Drug Administration (FDA) and orphan designations for the treatment of FOP from both the FDA and the European Medicines Agency (EMA).
About Clementia Pharmaceuticals Inc.
Clementia is a clinical stage biopharmaceutical company committed to delivering treatments to people who have none. The company is developing its lead candidate palovarotene, a novel retinoic acid receptor gamma agonist, to treat fibrodysplasia ossificans progressiva (FOP) and other diseases. For more information, please visit www.clementiapharma.com.
SOURCE Clementia Pharmaceuticals Inc.