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Clementia Newsletter to FOP Community- Spring 2017

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 discovery was announced 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