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.”