Mitsubishi Phase 2 Clinical Trial Meets Endpoint for Treatment of Erythropoietic Protoporphyria (EPP)

  • Tuesday, November 12, 2019

Mitsubishi Tanabe Pharma Development America, Inc. today announced successful completion of the Phase 2 clinical trial of MT-7117, an investigational oral treatment under development for the prevention of phototoxicity (including severe pain on exposure to sunlight) in patients with Erythropoietic Protoporphyria (EPP).

In this Phase 2 proof of concept trial, MT-7117 met its primary endpoint and was generally well tolerated with an acceptable safety profile.

"The results of ENDEAVOR are very encouraging and will pave the way for a pivotal trial to evaluate the safety, efficacy and effectiveness of MT-7117 as an oral once a day treatment option for EPP” said Robert Desnick, MD PhD, Dean for Genetic and Genomic Medicine, Icahn School of Medicine at Mount Sinai, NY and Lead Investigator on the study. “If confirmed and approved by regulatory agencies, this could be the first oral treatment option for EPP and we believe could be a clinically meaningful alternative for patients.”

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