Clementia is developing treatments for patients suffering from debilitating bone and other diseases with high unmet medical need
Our lead product candidate, palovarotene, is an oral small molecule that has shown potent activity in preventing abnormal new bone formation as well as fibrosis in a variety of tissues.
In 2016 we completed the first, randomized, placebo-controlled, adaptive design Phase 2 study in FOP, which enrolled 40 patients. This study showed encouraging safety and efficacy results, as well as unique insights regarding the disease in general and how to better dose patients and measure disease impact. Our Phase 2 trial and open label extensions, as well as additional insights, have led us to design our registration trial, the MOVE trial, for palovarotene in FOP. Our Phase 3 trial, MOVE, for the treatment of FOP in adults and children, will begin in 2017 and enroll up to 80 patients who will be treated chronically with palovarotene and with increased doses during flare-ups.
We are also advancing palovarotene for the treatment of Multiple Osteochondromas (MO), also called multiple hereditary exostoses (MHE), an ultra-rare genetic disease of new bone formation in children which is mediated by excess BMP signaling. Since it is believed that the mutations which cause MO also result in excess BMP signaling, we believe palovarotene can also inhibit this pathway in MO. Based on our knowledge of the safety and tolerability profile of palovarotene and our pre-clinical animal model data, we are planning to initiate a placebo-controlled Phase 2/3 study of palovarotene in MO.
We also believe that RARγ agonists have great potential as inhibitors of BMP signaling in other indications. Palovarotene has been shown to exert multiple effects in various tissues including in ocular tissues, where RARγ agonists generally demonstrate anti-fibrotic properties. Following the completion of pre-clinical studies, we plan to begin human clinical trials in 2018.