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Letter to the MO Community – 2019

By Zhizi Zhao,

Dear MO Community,

Last April we were just about to launch our MO-Ped Trial (Multiple Osteochondromas Pediatric Trial), the first ever study to evaluate a potential therapy for MO. I am pleased to report to you that MO-Ped is underway at 15 clinical trial sites in 4 countries, and we are well on our way to expanding enrolment to 12 countries around the world.

MO-Ped is the first clinical trial to study MO in a systematic manner. The primary endpoint of MO-Ped is the rate of new osteochondromas (OCs) as measured by whole body MRI. Several other clinical outcome measures include the volume of OCs, the number of surgeries, the number of new or worsening deformities and functional limitations as well as quality of life and pain measures. This first clinical trial in MO will therefore serve as a reference for generations to come and will likely facilitate further research and understanding of MO.

I want to convey my sincere thanks to those of you participating in MO-Ped. Your dedication will benefit the entire MO community. We know there are still many of you anxiously awaiting the opening of new clinical sites and we are working diligently with these sites to ensure that they are ready to begin and expand recruitment as soon as possible. With your assistance, we hope to complete enrollment in this important study around mid 2019.

We also want you to know that beyond MO-Ped, our upcoming filing to seek US FDA approval of palovarotene for FOP may have implications for the MO community. First, our studies and our discussions with regulators have resulted in many learnings that may also help rapidly advance investigational palovarotene as a potential treatment for MO. Second, all of our preparations for a potential FOP approval and eventual commercial launch, from building a global supply chain, scaling up manufacturing of palovarotene, to hiring people to help serve patients worldwide, will be the foundation for serving the MO community in the future. I am very proud of the team we are building, and I look forward to the day that they can assist you as well.

In a few weeks, on February 28th, communities around the world will recognize Rare Disease Day 2019 and reflect on just how many “rare” individuals there are, and how much need there is for new treatments for the thousands of rare disorders, like MO. Thank you for engaging with us on this pioneering journey as we help educate the world on MO, and as we resolutely advance with the MO-Ped trial.

All the best to you and your families in 2019,

Clarissa Desjardins

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Letter to the MO Community – 2018

By Matthew Dennis,

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.

-Clarissa Desjardins

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