Dear MO community,
My name is Clarissa Desjardins, and I founded Clementia in order to determine whether our drug candidate, palovarotene, could help patients with fibrodysplasia ossificans progressiva (FOP), an ultra-rare genetic bone disorder. Due to new scientific insights on the cause of multiple osteochondromas (MO, also known as MHE or HME), we believe palovarotene may also be of potential benefit in MO patients.
Both of these conditions appear to be due to an increase in bone morphogenetic protein (BMP) signaling, an intracellular communications process that tells the body to produce bone, and palovarotene has shown reduced BMP signaling in preclinical studies. So we started laboratory work with our collaborators to evaluate the biologic activity of palovarotene in MO/MHE mouse models. In a recently published scientific article, we reported that palovarotene reduced the number of osteochondromas in an animal model of MO and did so in a dose-dependent manner. Subsequently, we hosted discussions with physicians and scientific advisors to explore appropriate next steps, and decided to initiate a clinical trial with palovarotene in MO.
The MO-Ped Trial (Multiple Osteochondromas Pediatric Trial) will soon begin and will recruit up to 240 patients in 29 sites across 12 countries and four continents, testing two different doses of palovarotene versus placebo. This is the first clinical trial to be conducted in MO and we are thrilled to pioneer these efforts.
At a recent MO/MHE community advisory panel we held in Brussels, I heard firsthand how this disorder touches you and your loved ones and invades every part of your lives. We listened and learned about the pain, the deformities, the potential trauma of surgery and the fact that many people do not understand the seriousness of this condition. Most importantly, we committed to working to improve awareness while trying to develop a medical treatment intended to prevent the osteochondromas.
This is the beginning of a journey. The answers to our questions are uncertain. The drug development process is long and we commit to working as efficiently as possible and bringing our best efforts to you. As the first company to ever conduct a clinical trial in MO/MHE, we look forward to sharing what we learn, and sharing our hope that some day there may be an effective treatment for MO.