Summary from the IFOPA Research Committee

The FOP research landscape is evolving at a rapid pace in many wonderful ways. Global efforts to develop therapies for FOP are stronger than ever among academic and biopharmaceutical teams. The IFOPA works continuously to monitor and support these efforts, serve as an advocate for the FOP community, and inform you of progress whenever possible.

The annual FOP Italia meeting has become one of the key places to hear the latest news in FOP drug development. It is always an extraordinary gathering, to the credit of the entire FOP Italia team, and the 2016 meeting, held on April 15 and 16 in Livorno, Italy, was no exception. This year’s meeting was marked by an increase in participation from the biopharmaceutical industry, with representatives from Clementia Pharmaceuticals, Regeneron Pharmaceuticals, Alexion Pharmaceuticals, Blueprint Medicines, and Novartis in attendance. Chris Bedford-Gay, Betsy Bogard, Michelle Davis-Wingate, Moira Liljesthröm, Neal Mantick, and Eric Otto all participated in this year’s meeting on behalf of the IFOPA. You can see the full agenda online, and here are some of the highlights.

• Clementia Pharmaceuticals will be analyzing data from their Phase 2 clinical trial of palovarotene and completing enrollment in their FOP natural history study in the coming months. They expect to share the results from their Phase 2 program by the end of this year. If data are supportive, they expect their Phase 3 program will include two trials, one of which will be a surgical trial.
• Following their publication in 2015 about the role of activin A in FOP, Regeneron Pharmaceuticals has continued to evaluate a potential therapy for FOP, an anti-activin A antibody, in an FOP mouse model. At the FOP Italia meeting, they presented new data from this mouse model showing potential benefit of the antibody in early heterotopic bone lesions and, separately, in conjunction with surgical removal of heterotopic bone. Regeneron is preparing to publish these findings.
• Blueprint Medicines and Alexion Pharmaceuticals disclosed a partnership to develop a custom-designed drug for FOP in the class of compounds known as kinase inhibitors. Blueprint and Alexion are both publicly-owned biotechnology companies based in the northeastern United States. In the partnership, Blueprint is responsible for creating the molecule and conducting all preclinical research and Alexion will be responsible for clinical trials.
• Harvard and Oxford are collaborating to explore the potential use of saracatinib for the treatment of FOP. Saracatinib is an experimental kinase inhibitor developed by AstraZeneca and previously tested in 28 clinical trials.
• Vanderbilt continues their ongoing efforts to develop a potential drug for FOP, a novel kinase inhibitor, in partnership with La Jolla Pharmaceutical Company. In case you missed the announcement about their partnership, you can read about it here.

In addition to this news from FOP Italia, the University of Pennsylvania team recently shared new findings in FOP mouse models about palovarotene and, separately, the potential benefit of HIF-1-alpha inhibitors.

While there is much work ahead before we can understand whether any of these possibilities might one day become an approved therapy for FOP, we are heartened by the progress so far. We are fortunate to have many different teams working on a therapy, and even more so, to have many different types of therapies being considered. We are grateful to the countless talented, bright, and hard-working professionals who give their time and expertise to finding a therapy for FOP. Their devotion, persistence, and determination give us all hope.

We are equally grateful to the community members who participate in clinical trials, the FOP Connection Registry, and other research projects. You are heroes for all of us, giving so much of yourselves — your time, energy, biological samples, images, and data — and participating on behalf of those who can’t. We would be nowhere without you.

As the FOP research landscape has become more complex, leaders of the IFOPA have discussed our philosophy for engaging drug developers. Our goal in any dialogue or partnership is to enable development of therapies while maintaining our independence as a patient organization. We collaborate with drug developers who are conducting ethical, high-quality research in a responsible manner according to industry and international regulatory standards. When we have dialogue, it is to seek insight into the developer’s objectives, plans, and the potential drug being evaluated and to provide community-wide insight and perspective as needed and appropriate. Our sole incentive in these interactions is the eventual treatment of our loved ones with a safe and transformative therapy.

We are developing the details around this philosophy into guidelines to be sure it is clear, for ourselves and any current or potential partners, how we wish to operate. We will make these available on our website when they are ready, and will welcome comments and feedback on them.