Clementia Pharmaceuticals (NASDAQ:CMTA), a clinical-stage company innovating new treatments for people with ultra-rare bone disorders and other diseases, today announced the start of the MO-Ped Trial, its Phase 2 study evaluating the safety and efficacy of palovarotene for the treatment of pediatric patients with multiple osteochondromas (MO), also known as multiple hereditary exostoses (MHE). Palovarotene, an RARγ agonist, is Clementia’s lead product candidate and is also being developed in a pivotal trial for patients with fibrodysplasia ossificans progressiva (FOP), as well as other diseases.

As quoted in the press release:

In a joint statement, Dror Paley, M.D., FRCSC, and David Feldman, M.D. of the Paley Orthopedic and Spine Institute at St. Mary’s Medical Center, co-principal investigators in the MO-Ped Trial, commented, “MO is a rare, disabling disease, for which there are no approved treatments other than surgery. MO causes benign tumors called osteochondromas to form on bones throughout the body, resulting in pain, deformity, limb length discrepancy, disability and eventually, arthritis and possible malignancy. The majority of those affected undergo multiple bone surgeries, throughout childhood and even in the adult years. Palovarotene has the potential to inhibit new tumor growth in patients. This could significantly change the prognosis and quality of life for MO individuals. We are excited to be part of this clinical trial and helping advance this potential treatment for individuals with this debilitating condition.”
“The initiation of the MO-Ped Trial marks the second global, late-stage study now underway for palovarotene. To our knowledge, this trial is the first-ever clinical trial of an investigational agent for the treatment of MO, a debilitating and life-altering disorder,” said Clarissa Desjardins, Ph.D., chief executive officer of Clementia. “Palovarotene’s mechanism of action, which is to inhibit BMP signaling, along with its promising preclinical data and favorable tolerability profile in clinical studies, give us hope that this could be the first safe and effective treatment for MO. We are thankful to the patients and families, caregivers, physicians and support groups around the world with whom we are collaborating to make important studies such as MO-Ped possible.”

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