Phase 3 Trial Testing MT-7117 for EPP, XLP Now Enrolling at 3 US Sites

Phase 3 Trial Testing MT-7117 for EPP, XLP Now Enrolling at 3 US Sites
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A Phase 3 clinical trial of MT-7117 (dersimelagon), Mitsubishi Tanabe Pharma‘s experimental treatment for erythropoietic protoporphyria (EPP) or X-linked protoporphyria (XLP), is now enrolling participants at three sites in the U.S.

The three locations are in Winston-Salem, N.C., Columbus, Ohio, and Boston. More information on the trial sites, including those yet to open, can be found here.

Future sites include locations in six additional U.S. states, including New York, Texas, and Washington, as well as Australia and Canada. For contacts at the American Porphyria Foundation that can help reach a study location, please click here.

The Mitsubishi Tanabe Pharma-sponsored trial (NCT04402489), which launched earlier this year, plans to recruit an estimated 159 participants, ages 12 to 75, to test the safety, tolerability and efficacy of MT-7117.

The objective of the double-blind study is to determine if the therapy protects against initial symptoms — such as pain, itching, and tingling in the skin — that emerge shortly after exposure to sunlight in people with EPP or XLP. During the trial, patients will be randomly assigned to receive one of two doses of MT-7117, or a placebo, both administered orally in the morning for 26 weeks (six months). Participants will have the option to join a 26-week extension phase.

Following positive results in a Phase 2 trial (NCT03520036), the team now will assess time to initial skin reactions following sun exposure — between one hour post-sunrise and one hour pre-sunset — comparing changes from baseline with MT-7117 to those seen with the placebo at week 26. In addition, the researchers will measure the number and severity of sunlight-induced pain events, and collect patient assessments of symptoms and changes in physical function over the duration of the trial.

MT-7117 is a novel synthetic small molecule that activates melanocortin-1 receptor (MC1R), a protein involved in pigmentation and the skin’s natural photoprotection (protection against sunlight). In healthy skin, activation of MC1R produces a type of melanin that protects the skin from UV-induced sun damage. MT-7117 is intended to boost this production and, thereby limit the symptoms of EPP or XLP following sun exposure.

The U.S. Food and Drug Administration granted MT-7117 fast track designation in 2018 for the treatment of EPP. That designation is intended to expedite the review process of experimental therapies for serious or life-threatening diseases. Earlier this year, the FDA also granted orphan drug designation to MT-7117, which confers financial and regulatory incentives for the development of rare disease therapies.

Aisha Abdullah received a B.S. in biology from the University of Houston and a Ph.D. in neuroscience from Weill Cornell Medical College, where she studied the role of microRNA in embryonic and early postnatal brain development. Since finishing graduate school, she has worked as a science communicator making science accessible to broad audiences.
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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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Aisha Abdullah received a B.S. in biology from the University of Houston and a Ph.D. in neuroscience from Weill Cornell Medical College, where she studied the role of microRNA in embryonic and early postnatal brain development. Since finishing graduate school, she has worked as a science communicator making science accessible to broad audiences.
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