RESEARCH

Clementia Announces Top-line Results from Phase 2 Trial of Palovarotene for Treatment of Patients with Fibrodysplasia Ossificans Progressiva

MONTREAL, CANADA, October, 14, 2016 – Clementia Pharmaceuticals Inc. today announced top-line results from its Phase 2 clinical trial investigating palovarotene for the treatment of fibrodysplasia ossificans progressiva (FOP). “The results of this landmark clinical trial are encouraging and closely mirror what was observed in previously reported animal studies with palovarotene,” said principal investigator, Frederick Kaplan, MD, the Isaac & Rose Nassau Professor of Orthopaedic Molecular Medicine and Chief of the Division of Molecular Orthopaedic Medicine in the Perelman School of Medicine at the University of Pennsylvania. “This study has considerably enhanced our knowledge of FOP and is a significant step forward for the entire FOP community.” To read more...

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FOP Physical Function Questionnaire

Clementia Pharmaceuticals Inc. is developing a new tool to measure the impact of FOP on physical functioning. The tool will be called the FOP Physical Function Questionnaire (FOP-PFQ) and will be a custom-designed questionnaire to capture the unique and very challenging ways that FOP affects physical function and activities of daily living. These types of tools are used commonly to document the status of physical function and general well being in many diseases. This knowledge is important for understanding the degree of limitations imposed by a disease, which makes it very useful in the drug development process. 

Developing this tool requires input from individuals living with FOP. Clementia requested help from the IFOPA in obtaining this input, beginning with one-on-one interviews at the 25th Anniversary Celebration. Twenty adults participated in these interviews at the meeting and two others participated by phone afterwards.  A second round of interviews is expected to begin in a different group of approximately ten adults beginning in March. The first round of interviews was designed to elicit concepts relevant to FOP physical functioning while the second round is intended to assess cognitive understanding of these concepts and refine the draft questionnaire. Each round is conducted in a different group of individuals. In the next few months a similar process will be used to develop a questionnaire for the pediatric population.

When the questionnaire is ready for both adults and pediatrics, Clementia will use it in clinical studies to support the evaluation of palovarotene as a potential therapy for FOP, and also to evaluate the natural history of FOP in a physician-directed, non-interventional longitudinal study. Clementia plans to make the final questionnaire available to the IFOPA and to publish the questionnaire for use in other FOP research.

Clementia’s project is being conducted by a team of specialists from Evidera who have expertise in designing these types of patient-reported outcome measures. You can read more about both Clementia and Evidera here:

Gene Discovery and Focus of Research at U of Penn

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In 2006, Dr. Frederick Kaplan, Dr Eileen Shore and their research team at the University of Pennsylvania made the first step in finding a treatment when they discovered the gene that causes FOP. “The discovery of the FOP gene is relevant to every condition that affects the formation of bone and every condition that affects the formation of the skeleton,” Dr. Kaplan explains.  This discovery launch a growth in interest to study FOP.  Today there are approximately 20 research initiatives across the globe including efforts at Harvard, Oxford and work being done in Japan and Germany, biotech's and more.

Today the main focus of the Cali's efforts is to fund a treatment with the founding fathers of FOP research at the University of PA.  Currently, 80% of all research dollars for FOP at the University of PA come from FOP family fundraisers, endowments and donations.  The balance comes from the National Institutes of Health and other institution grants.  “The burden to keep research funded by families is a constant challenge but we feel that the discovery of the FOP gene and the prospect of a clinical trial looming on the horizon has given new hope to us all."  The discoveries in FOP research are sure to shed light on other more common conditions such as osteoporosis, hip fractures, heterotopic bone formation in amputees and heart valve disease.

Novartis Institutes Biomarker Study

Novartis Institutes for Biomedical Research is sponsoring a biomarkers study to evaluate whether certain changes in urine may be indicative of an FOP flare-up. Drs. Fred Kaplan and Robert Pignolo at the University of Pennsylvania are the investigators for the study. Approximately 25 patients have enrolled in the study, for which recruitment began at the 25th Anniversary Celebration. The study requires each participant to collect and ship urine samples on a regular basis for approximately 3 months for analysis by Novartis.

Once the 3-month period of sample collection has been completed for all participants, Novartis will start analyzing urine samples. Together with Drs. Kaplan and Pignolo, the data will then be reviewed and analyzed and key findings will be published. If any biomarkers are identified as predictors of an FOP flare-up, these will be published and will be useful for any future efforts to develop a therapy for FOP. 

The study is listed on clinicaltrials.gov under the identifier NCT02066324.

Two technical publications on biomarkers and their relevance to FOP are available here.

Most Recent Research FOP Report

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22nd Annual Report of the Fibrodysplasia Ossificans Progressiva (FOP) Collaborative Research Report

Oxford University Spring FOP Report

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Spring Report from the University of Oxford FOP Research Team

 FOP Lab at University of Pennsylvania

  • Researchers at the University of Pennsylvania School of Medicine, the only laboratory in the US dedicated to FOP research, announced the Discovery of the FOP Gene in Nature Genetics in April 2006.
  • 10,000 sq. ft. of shared research space in the Department of Orthopaedic Surgery
  • 3 principal investigators with 15 post-doctoral fellows, students, scientists, and staff
  • Funds spent on research - Approx. $1.5 million/year
  • 75% from FOP family fundraising and donations
  • 25% from institutional support (NIH/NIAMS, Orthopaedic Research and Education Foundation)
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Substance P Signalling

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I am a neurologist and stem cell biologist. We constructed a transgenic animal to study development of the nervous system, but we found that the animals developed heterotopic bone formation–a very striking phenotype. The transgene was for bone morphogenetic protein 4 (BMP4), and genetic data from humans suggested that BMP signaling is involved in the human disorder. That sparked our interest in FOP since we seemed to have an excellent model of the genetic disease.

FOP Update at University of Pennsylvania

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The Cali and Weldon families and friends have funded FOP research for many years, through major gifts and grassroots fundraising and have spearheaded both national and international awareness building for the condition and the research team at Penn. Their support has been crucial in making the Penn Medicine Center one of the world’s foremost sites devoted to the investigation of FOP’s molecular and genetic causes.

Potential Treatment Found for Debilitating Bone Disease in Wounded Soldiers and Children

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PHILADELPHIA, April 3, 2011 /PRNewswire-USNewswire/ -- Promising new research reveals a potentially highly effective treatment for heterotopic ossification (HO), a painful and often debilitating abnormal buildup of bone tissue. HO comes in two main forms—one that appears in children and is congenital, another that strikes wounded military personnel and surgery patients and is triggered by severe injuries and wounds.