MT-7117 Poised for Phase 3 Trial in Two Types of Porphyria

MT-7117 Poised for Phase 3 Trial in Two Types of Porphyria
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A Phase 3 clinical trial of Mitsubishi Tanabe’s investigational therapy MT-7117 (dersimelagon) for people with erythropoietic protoporphyria (EPP) or X-linked protoporphyria (XLP) has been launched.

The study (NCT04402489) will take place at 12 clinical sites in the U.S. and will test the therapy in adults and adolescents. Locations in Columbus, Ohio, and Boston are now active, and will be followed by additional sites globally, according to the American Porphyria Foundation.

For more information, potential participants can contact the foundation to be connected with the nearest study site. Contact information is [email protected] or [email protected] Also, 866-APF-3635 or 301-347-7166.

MT-7117 is a man-made (synthetic) small molecule that binds to and activates the melanocortin-1 receptor (MC1R) protein. Given orally, the experimental therapy is intended to increase the production of a pigment called melanin to induce photoprotection (protection against sunlight) of the skin.

A prior Phase 2 trial, ENDEAVOR (NCT03520036), evaluated the effectiveness, tolerability and safety of MT-7117 in adults with EPP.

Participants underwent a two-week screening period, followed by a 16-week treatment, where they received either one of two daily doses (high and low) of MT-7117 or a placebo. A six-week follow-up period ended the trial at week 24.

Results showed that the study met its primary goal, with MT-7117 extending the average daily time to the first early symptom associated with sunlight exposure relative to the study’s start. MT-7117 also was generally well-tolerated.

In the double-blind Phase 3 study, participants with either EPP — the most common porphyria of childhood — or XLP will be assigned randomly to a low or high-dose of MT-7117,  or a placebo, given once daily in the morning with or without food for 26 weeks. Enrolled patients may join an optional 26-week extension phase.

The study’s primary goal is to assess the effectiveness of MT-7117 in a similar way as in Phase 2. The team will evaluate whether the therapy extends the daily sunlight exposure time before participants develop the first early symptoms — burning, tingling, or stinging — associated with sun exposure in the periods between one hour post-sunrise and one hour pre-sunset.

Additional goals include assessing patients’ global impression of change and the total number of sunlight-induced pain events during the treatment period.  Pain will be scored from 1 to 10 according to the Likert scale, in which increasing scores represent greater pain.

MT-7117 received fast track designation from the U.S. Food and Drug Administration for the treatment of EPP in 2018.

Patricia holds her Ph.D. in Cell Biology from University Nova de Lisboa, and has served as an author on several research projects and fellowships, as well as major grant applications for European Agencies. She also served as a PhD student research assistant in the Laboratory of Doctor David A. Fidock, Department of Microbiology & Immunology, Columbia University, New York.
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José is a science news writer with a PhD in Neuroscience from Universidade of Porto, in Portugal. He has also studied Biochemistry at Universidade do Porto and was a postdoctoral associate at Weill Cornell Medicine, in New York, and at The University of Western Ontario in London, Ontario, Canada. His work has ranged from the association of central cardiovascular and pain control to the neurobiological basis of hypertension, and the molecular pathways driving Alzheimer’s disease.
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Patricia holds her Ph.D. in Cell Biology from University Nova de Lisboa, and has served as an author on several research projects and fellowships, as well as major grant applications for European Agencies. She also served as a PhD student research assistant in the Laboratory of Doctor David A. Fidock, Department of Microbiology & Immunology, Columbia University, New York.
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