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Letter from the CEO – 2018

By Matthew Dennis,

Dear FOP community,

In my last letter to you, I quoted a passage saying that the tiniest thread can be twisted into an unbreakable cord. I feel that this is what we are achieving together!

The hope we have all shared over the last few years is now supported by our Phase 2 clinical results. Through the collective efforts of the community, including FOP families, researchers, and clinicians and their teams, we have collected data on over 100 flare-ups, including after treatment with 4 distinct regimens of palovarotene. We now have important data on 114 patients in the Natural History Study (NHS) which, through a ground-breaking agreement with IFOPA, we will share with the patient community so that the knowledge gained will be available to others coming after us.

We have observed that treating chronically with palovarotene 5 mg once daily, followed by treatment with higher doses at the earliest signs of a flare-up, appears to be the best way to reduce new bone formation after flare-ups. In fact, for those on this regimen,we detected a greater than 70% reduction in new bone volume at 12 weeks as compared to untreated flare-ups. The FDA granted us Breakthrough Therapy Designation in July in recognition of this clinical evidence indicating that palovarotene may provide substantial improvement over existing therapies for FOP. Now is the time to take everything learned and focus our energies on the efficient completion of the final step in our clinical development program for palovarotene in FOP: the Phase 3 MOVE Trial.

We are thrilled with the recruitment of the first several patients in the MOVE Trial last year and plan to continue enrolling up to 80 patients at 20 sites in 16 countries across 6 continents. Everyone who enrols in this final clinical trial will receive palovarotene, as regulatory authorities have agreed that the NHS could be used as the external control. We believe that if the trends in bone reduction observed to date continue, it is possible that palovarotene will extend the time patients retain mobility.

Less than 5 years ago, I was an individual who had never heard of FOP. I read a Nature Medicine paper and decided to change my life. I quit my job and started Clementia. Building Clementia has been a life-changing experience. I don’t believe it is possible to become aware of FOP and, in particular, to meet and interact with families who live with FOP and not be transformed. I know that I speak for the entire Clementia team when I say thank you to all of you for the opportunity to serve your community. You have provided us with the most laudable goal to direct our expertise and work and inspired us beyond what we knew was possible.

Finally, while we applaud our progress, we must also acknowledge the recent loss and deep grief within the FOP community. We realize that every day poses risks for those living with FOP and are sincerely grateful for those who, having lived with FOP for decades, have courageousely kept hope alive and given of their time and energy so that others would benefit. You are the heroes of this story.

Thank you and best wishes for a transformative 2018!

– Clarissa Desjardins

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Letter to the Community – 2017

By Matthew Dennis,

Hello everyone,

We hope that you have all had a fantastic FOP Awareness Day celebration! This past Sunday, April 23rd, marked the day on which the FOP gene was discovered 11 years ago and which the community has established as FOP Awareness Day. This discovery was the culmination of years of effort from a successful collaboration in which the global patient community played a crucial role and opened the door to increased disease understanding, the development of therapeutic approaches and potential therapeutic candidates.

We marked this special day at Clementia as a group on Friday April 21st with great pleasure and fun! We have indeed participated at the ‘Fun Feet for FOP Day’ initiative proposed by the IFOPA and came to work with wacky shoes, socks or toes. We shared lunch across our various offices and spoke of our joy and urgency in serving the FOP community. Your strength, your determination, and your thoughtfulness are a source of inspiration for each of us in our daily work.

The big highlights at Clementia, as you might have read in the last Clementia press release dated March 28th, 2017 -preliminary data of palovarotene Phase 2 Part A OLE (open label extension) trial, are the preparations for the upcoming Phase 3 trials: MOVE and REMOVE. The MOVE trial is a treatment trial that will evaluate the effect of palovarotene on the course of FOP and the REMOVE trial will evaluate the effect of palovarotene following surgical excision of HO (heterotopic ossification). Our medical and clinical teams are preparing furiously for these two imminent trials, so lots of early mornings, late evenings, and short weekends in the Clementia offices.

In parallel to the planning of the next phase of the palovarotene clinical development program in FOP we are advancing – with the support of the FOP community – on other key initiatives that aim to improve the diagnosis and care of FOP patients and to ultimately prepare local health systems around the world to enable access to potential therapeutic innovations such as palovarotene for FOP patients.

In January, we reviewed the FOP Narrative project with our Phase 2 investigators at Penn, Mayo, and UCSF. If you attended the IFOPA Drug Development Forum in October of last year, you may recall this project. If not, our purpose for this project is to understand the experience of FOP affected individuals with their local health systems. We visited Michelle Davis from IFOPA in Kansas City too and exchanged with her on planned initiatives in 2017 as well as on key implications of the FOP Narrative project. Another very productive and enjoyable meeting!

In February, we celebrated Rare Disease Day (RDD) with several FOP patient group leaders in Brussels at EURORDIS’ (European Rare Diseases Organisation) annual Awards & Black Pearl evening and workshop. EURORDIS founded RDD several years ago to focus the research and policy communities’ attention on rare diseases, and the event, which occurs the last day of February every year has become very popular. While there, we hosted an advisory panel meeting with the FOP attendees. The insights gathered at that meeting were precious and will guide us in the implementation of future projects. This was both a productive and fun time for all!

In March, we were in Brazil with the FOP Narrative project – in close collaboration with Dr. Patricia Delai- who is the current President of FOP Brasil. This initiative helped us to better understand the experience of FOP patients with the Brazilian health system as well as more generally the challenges, the needs, and the expectations of FOP families in Brazil. Rich exchanges and precious moments full of emotion! Thanks to all the FOP families interviewed! Multo obrigada!

Later in March we had the opportunity to meet with Adam Sherman – the new Research Development & Partnerships Director at IFOPA – together with Betsy Bogard at our offices in Newton, MA, US. It was a very nice introductory meeting and we are looking forward to collaborating closely with him!

To close the month of March we attended the 2017 CORD (Canadian Organization for Rare Disorders) Rare Disease Day event that took place in Vancouver, Canada, with 3 FOP mother advocates. The main theme of the conference was the development of Rare Disease Networks in Canada– a key topic and very successful meeting! We have not only had a lot of fun but we have also had the opportunity to highlight FOP among other rare diseases. At that conference Carrie Connell- President of CFOPN- gave a vibrant testimonial on FOP and Clementia presented on the FOP Narrative project. All in all a lot of positive awareness on FOP and relevant themes debated!

These past months we have also had some memorable trips to Japan, Poland, Spain and Sweden meeting with FOP experts and patient advocates, receiving a warm welcome and kind hospitality in every location! Thanks so much!

We ended April by celebrating FOP Awareness Day – marking this unique day honoring all of you around the world- you are so special to us!

And now back to work…we have a lot to do!

Stay tuned on upcoming Clementia news and take good care of yourselves as well as your loved ones…

Warm regards, Stéphanie

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Letter from the CEO – 2016

By Matthew Dennis,

Dear FOP community,

I am very pleased to share with you the top-line results of our Phase 2 clinical trial. Overall, our study has exhibited positive trends in bone reduction and symptom reduction using palovarotene though these observations did not reach statistical significance. In the 40 subject placebo-controlled Phase 2 dose-ranging trial:

  • No subject required a reduction in dose due to tolerability issues nor was discontinued from the trial.
  • Those on placebo were at 2.6 times greater risk of forming heterotopic ossification (HO) than those on the palovarotene 10/5 mg treatment. In addition, those on either palovarotene regimen with new HO formed less HO than those on placebo.
  • We observed palovarotene-related reductions in patient-reported pain associated with flare-ups and reductions in the time to resolution of flare-ups.

Together, we have achieved the first and likely hardest goal of our development program, which is to determine how we can measure palovarotene’s effect and how we could potentially demonstrate efficacy of palovarotene in a Phase 3 clinical trial. We are extremely eager to move on to this next step in our clinical development program to hopefully achieve our goal of providing a therapy for those living with FOP.

The Phase 3 clinical trial is scheduled to begin in 2017 and will recruit patients from all over the world. We are currently planning and designing the features of this Phase 3 clinical trial as well as a surgical excision trial and will meet with regulatory authorities to ensure that the design of these trials meets with their expectations.

In most jurisdictions around the world, a sponsor, such as Clementia, is required to perform adequate and well-controlled studies that demonstrate a statistically significant impact of the drug on how a patient “feels, functions or survives.” This is a high bar to achieve even when positive trends are detected such as in our initial Phase 2 clinical trial. However, we are determined to meet this challenge.

All of us here at Clementia continue to be inspired everyday by your stories, your collaboration, and your grit. We thank all of you who participated in this trial and our other clinical trials. You have provided answers and hope for the entire FOP community for many years to come. You are our heroes. We also want to thank all the dedicated clinical teams around the world who worked tirelessly to generate these results.

Let me leave you with these words from an unknown author, “Whatever you do, hold on to hope. The tiniest thread will twist into an unbreakable cord. Let hope anchor you in the possibility that this is not the end of your story, that change will bring you to peaceful shores.”

Warm regards,

Clarissa

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Letter from the CEO – 2015

By Matthew Dennis,

Dear members of the FOP community,

In November 2013, I first introduced myself to you as the founder of a company whose goal was to develop a treatment for FOP. At that time, we had recently licensed the rights to palovarotene and were preparing to initiate clinical trials. As we move forward in these trials, we increasingly understand the gravity of FOP and what an approved treatment would mean for affected families.

The importance of potentially treating children before their mobility is significantly affected is something we all appreciate. That is why I am delighted to share that children age six and older may now participate in the Phase 2 study.

The inclusion of children between six and 14 years old will allow for the preliminary assessment of safety in this age group, bringing us to the last step needed to conclude the Phase 2 study. We anticipate that if enrollment continues as planned, we will complete the Phase 2 study by the end of 2015 and will formally announce when this occurs. Once complete, we pledge to efficiently analyze the data so that we may announce the findings in 2016 and finalize the design of the Phase 3 study. Our goal is that children even younger than six years old may be able to be enrolled in the Phase 3 study.

In addition to the milestone of children enrollment, our clinical program is proceeding well. Individuals who complete the Phase 2 study can enroll in the open-label extension study and receive palovarotene for any subsequent, eligible flare-ups. To date, all individuals who have completed the Phase 2 study have chosen to participate in the open-label extension study.

Clementia’s Natural History Study has been progressing at an extraordinary pace with 33 subjects enrolled and several others scheduled to be enrolled. Because of this progress, we have decided to increase enrollment from 50 to up to 100 participants. Through our data sharing agreement with the IFOPA, this crucial disease progression information will deepen the entire community’s understanding of FOP.

It has been an exciting year at Clementia and for the FOP community. More than ever, there is interest in developing a treatment for FOP. I cannot express enough our gratitude to all of the individuals who have volunteered to participate in our trials and to all of the loved ones who have supported them. We are amazed by your resolve, inspired by your courage and appreciative of your willingness to embrace us into your community. We are hopeful for a brighter future for all those affected by FOP and will continue to work hand-in-hand with you to move our clinical program forward.

Best regards,

Clarissa

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Letter from the CEO – 2014

By Matthew Dennis,

Dear members of the FOP community,

I wrote to you in November that Clementia’s goal was to develop a treatment for FOP, and I am thrilled to write today that palovarotene is being tested in a clinical trial for FOP. This would not be possible without the hard-won gains of the FOP community over the last several decades. Your strength, heart, courage, and resolve compelled researchers to test the retinoids in animal models and enabled Clementia to take palovarotene, one of the drugs tested, into this clinical trial, the first of what we hope will be many and a step toward the ultimate goal of delivering a treatment for patients with FOP.

This Phase 2 clinical trial is designed to show whether palovarotene will have an effect on new bone formation during and after a flare-up in patients with FOP. Patients will be followed closely through in depth imaging studies, biomarkers, and patient-reported measures, all of which will likely set standards for future clinical trials. So regardless of palovarotene’s effect, this trial represents significant progress that will improve understanding about FOP in meaningful ways.

Clementia is fully cognizant that emotions surrounding this disease are very personal and may be stirred by palovarotene’s entry into the clinic. Some may rejoice at the symbolism of progress while others may recall their history with the disease. Ian Cali said it well when he said, “I don’t even allow myself to believe a treatment may soon be on the horizon since I do not want it to affect my current happiness and acceptance.” Although we cannot truly put ourselves in your shoes, we try, and that motivates us to execute this trial with utmost professional rigor, so that the data can begin to answer the questions we all have.

We do not know what the results of this clinical trial will be, and it would be irresponsible for us to speculate. We operate under regulatory guidelines that are in place to ensure that manufacturers do not try to influence unduly members of the disease communities whom they serve and, importantly, do not misrepresent any information about their experimental drugs. More importantly though, we do not wish to speak casually of things that are so important to so many, including ourselves. We will share the results of our work as quickly as possible. We see this first clinical trial as the beginning of a journey with you, one that is assuredly leading us forward in the right direction.

Best regards,

Clarissa

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Letter from the CEO – 2013

By Matthew Dennis,

A message to 25th anniversary IFOPA celebration participants
Congratulations on all of your hard work! Twenty-five years of working together to help fund research, educate, support families and ultimately treat FOP is truly something to celebrate.

I am the founder and CEO of Clementia. Our goal is to develop a treatment for FOP. We are planning and preparing for clinical trials where we will be testing Clm-001, a drug that was originally in development by a large pharmaceutical company in another disease.

I’d like to tell you a little about how Clementia came to be. Basically, it all started with Nature Medicine paper by Drs. Iwamoto and Pacifici. As many of you know, this paper showed that RARγ agonists could prevent heterotopic bone formation in animal models of FOP. I reached to the company whose drug was tested in this paper, and through several key contacts, found out that they were prepared to out-license the drug if sufficient funds could be raised to develop it. Fortunately, raising money for biotechnology companies is something I had done before.

One of the first calls I made before Clementia was even born, was to the person who had by far published the most on FOP, Dr. Frederick Kaplan. I knew after speaking with him and hearing his heroic story that this would be a very special journey.
Through Dr. Kaplan, I was introduced to the Cali family. I’ll never forget my first conversation with John Cali (whose son, Ian has FOP) where, in hearing that I was about to start a company and raise money for a clinical trial, he told me that he wanted to help me in any way he could. He said “there is nothing that I would not do or give to help develop a treatment for my son, Ian, and for all those with FOP”. I’m sure he spoke for all of you when he said this. He then sent me a video of a speech by Ian, then 17, speaking about what is was like to live with FOP and what the prospects of new treatments meant to him.

Ian has become our inspiration. Everyone in our company has seen Ian’s speech, including our Board. Ian was the first to inspire us but as we get to know more of you from the FOP community, Nathalie, Mathew, Sienna and all of you here, we continue to be inspired and we’re rooting for you all.

I wish I could tell you that our drug or others in the pipeline will be successful in reducing bone formation, but no one knows this for sure. Only clinical trials can provide that answer. But I can tell you for certain, is that we are doing absolutely everything we can to initiate a safe and well-designed clinical trial which will answer this question.

Dr. Donna Grogan, our Chief Medical Officer and other members of our team are eagerly looking forward to working with you, the IFOPA, clinical experts and regulators to get these trials underway.

Best regards,

Clarissa Desjardins

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