JERSEY CITY, N.J.—Mitsubishi Tanabe Pharma Corporation, Inc. reported today that Mitsubishi Tanabe Pharma Development America, Inc. has initiated a Phase 3 clinical trial in the U.S. for MT-7117 (dersimelagon). The investigational oral therapy is being studied as a possible treatment option to increase pain-free light exposure in adult and adolescent patients with a history of phototoxicity — including severe pain on exposure to sunlight — from erythropoietic protoporphyria (EPP) or X-linked protoporphyria (XLP).
MT-7117 is a synthetic, non-peptide small molecule which acts as a selective agonist of melanocortin-1 receptor (MC1R). Mitsubishi Tanabe Pharma is developing MT-7117 as a potential treatment option for EPP or XLP. The company was also recently granted Orphan Drug Designation for MT-7117 by the U.S. Food and Drug Administration on June 8.
“Mitsubishi Tanabe Pharma group companies are working hard to advance research and development activities to deliver new therapy options that address the needs of patients fighting serious diseases,” said Atsushi Fujimoto, president of Mitsubishi Tanabe Pharma America. “Through further clinical development and potential regulatory approval, this investigative oral treatment could provide another option for patients with EPP or XLP.”
EPP is an inherited disorder of the heme biosynthetic pathway that results from mutations of the ferrochelatase (FECH) gene. Less common is XLP, which results from mutations in the aminolevulinic acid synthase-2 gene. Both diseases are characterized by accumulation of protoporphyrin in blood, erythrocytes and tissues, and by cutaneous photosensitivity.
EPP and XLP usually present in early childhood, with extremely painful phototoxic reactions which are preceded by a prodrome of tingling, stinging and/or burning of sun-exposed skin. The onset of prodromal symptoms after direct sun exposure varies but may occur in less than 10 minutes. And continued exposure to sunlight following the onset of prodromal symptoms will lead to phototoxicity-induced pain.
The global Phase 3 study is a multicenter, randomized, double-blind, placebo-controlled study to evaluate efficacy, safety and tolerability of MT-7117 in approximately 159 people with EPP or XLP over a 26-week treatment period. The primary objective of the study is to investigate efficacy of MT-7117 on time to first onset of prodromal symptoms associated with sunlight exposure in adults and adolescents with EPP or XLP.
The Phase 3 study design will leverage experience and knowledge obtained from an earlier Phase 2 study, which assessed the efficacy, safety and tolerability of MT-7117 in 102 people with EPP. The study consisted of a two-week screening period, a 16-week double-blind treatment period and a six-week follow-up period at Week 22.