MONTREAL, June 5, 2017 /PRNewswire/-- Clementia Pharmaceuticals, Inc. today announced that it will host a symposium entitled "Novel Perspectives in Two Rare Bone Disorders" on Monday, June 12th, 2017, at the 8th International Conference on Children's Bone Health (ICCBH) in Würzburg, Germany. This session, to be chaired by Dr. Geneviève Baujat of the Institut Imagine, Hôpital Necker (AP-HP), Paris, France will provide updates on fibrodysplasia ossificans progressiva (FOP) and multiple osteochondromas (MO), both rare bone conditions in which Clementia is conducting investigational studies for palovarotene, its lead compound.
"We continue to work with patients, researchers and the appropriate regulatory bodies towards the start of a Phase 3 registration trial in our lead program in FOP, later this year," said Clarissa Desjardins, Ph.D., Chief Executive Officer of Clementia. "Now, supported by encouraging preclinical data and our deep knowledge of RARγ and palovarotene, we look to expand our development efforts to another unmet medical need. We expect to begin a clinical study in patients with MO later this year."
"While we move forward with other programs, our commitment to FOP is unwavering," added Donna Grogan, M.D., Chief Medical Officer of Clementia. "Clementia's team continues to grow to support these expanded efforts, recently adding Dr. Fei Shih as Executive Medical Director, Clinical Development, whose primary responsibility will be the MO program. A pediatric rheumatologist and PhD immunologist by training, her clinical development experience will help Clementia drive multiple clinical programs forward."
Palovarotene is a retinoic acid receptor gamma agonist (RARγ) being investigated as a treatment for patients with debilitating bone and other diseases with high unmet medical need. Preliminary Phase 2 data in subjects with FOP mirrors the decrease in HO volume observed in lab studies in mouse models of FOP, and support the initiation of a confirmatory Phase 3 program. Palovarotene also inhibits the formation of osteochondromas (OCs) in mouse models of MO, supporting development in this indication. Palovarotene has received Fast Track and Orphan Drug Designations for FOP from the U.S. Food and Drug Administration (FDA), and was granted orphan status for the treatment of FOP in the EU.
About Fibrodysplasia Ossificans Progressiva (FOP)
FOP is a rare, severely disabling congenital myopathy characterized by heterotopic ossification (HO), or bone that forms outside the normal skeleton. In FOP, HO progressively restricts movement by locking joints leading to a cumulative loss of function, disability, and risk of early death. FOP is caused by a mutation in the ACVR1 gene, resulting in excess BMP signaling in the bone morphogenetic (BMP) pathway, a key pathway controlling bone growth and development. There are currently no approved treatments for FOP.
About Multiple Osteochondroma (MO)
MO, also called hereditary multiple exostoses, is a rare genetic musculoskeletal condition in which multiple benign bone tumors, also known as osteochondromas (OCs), develop on bones. MO is associated with skeletal abnormalities including short stature, joint deformity, limb length discrepancies, and early onset osteoarthritis. Of patients with MO, 70% often undergo surgeries, sometimes in excess of 20, to remove painful OCs and/or address deformities.
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 RARγ agonist, to treat Fibrodysplasia Ossificans Progressiva (FOP), Multiple Osteochondroma (MO), and other diseases. For more information, please visit www.clementiapharma.com.
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SOURCE Clementia Pharmaceuticals Inc.