JERSEY CITY, N.J., May 21, 2025 – Mitsubishi Tanabe Pharma America, Inc. (MTPA) today announced that patient enrollment has been completed for the INSPIRE (Increased Sun Exposure Without Pain In Research Subjects With EPP Or XLP) clinical study (NCT06144840). INSPIRE is a global, randomized, double-blind, placebo-controlled phase 3 study investigating the efficacy, safety, and tolerability of dersimelagon (MT-7117) in adults and adolescents with erythropoietic protoporphyria (EPP) or X-linked protoporphyria (XLP). Dersimelagon is a novel orally-administered, small molecule under study for its potential to increase the time in sunlight before patients experience prodromal symptoms (i.e., the appearance of initial symptoms such as burning, tingling, itching, or stinging). Topline data from the INSPIRE study is expected this fall.
EPP and XLP are rare inherited disorders of the heme biosynthetic pathway that are characterized by severe pain in the skin with exposure to sunlight and some forms of artificial light. It’s estimated that approximately 1 in 75,000 to 1 in 200,000 individuals have EPP.
“There are limited options for adults and no approved options for adolescents with EPP or XLP beyond sun protection or avoidance, resulting in a critical gap in care for many who are left to endure significant disruption in their lives,” said Kristen Wheeden, United Porphyrias Association (UPA) President. “The completion of this study enrollment is an encouraging step forward in the effort to improve care and explore innovative therapies for the porphyria community.”
The phase 3 INSPIRE study will evaluate the efficacy of dersimelagon on time to onset and severity of first prodromal symptoms associated with sunlight exposure in adults and adolescents with EPP or XLP over the course of 16 weeks of treatment. Following this treatment period, participants will have the option to continue in the open-label period to receive dersimelagon 200 mg once daily for 36 weeks. Another open-label extension study will continue to evaluate the long-term safety and tolerability of dersimelagon and enroll participants from the INSPIRE study and participants who have completed the study MT-7117-G01, if interested.
MTPA is committed to bringing innovative products like dersimelagon to market for patient populations with unmet needs. Dersimelagon is an investigational medication that received Fast Track Designation from the U.S. FDA in June 2018 and was granted Orphan Drug Designation in June 2020. Dersimelagon has not been approved by the FDA or any other regulatory agency.
About INSPIRE Study
INSPIRE is a global, randomized, double-blind, placebo-controlled phase 3 study investigating the efficacy, safety, and tolerability of dersimelagon (MT-7117) in approximately 150 adults and adolescents with erythropoietic protoporphyria (EPP) or X-linked protoporphyria (XLP). After a screening, study participants (ranging from 12 to 75 years of age) received either dersimelagon 200 mg or placebo once daily. The primary endpoint was change from baseline in average daily sunlight exposure time (in minutes) to first prodromal symptom (burning, tingling, itching, stinging) associated with sunlight exposure between 1-hour post-sunrise and 1-hour pre-sunset at week 16. Secondary endpoints were patient Global Impression of Change (PGIC) at week 16, the total number of sunlight-induced pain events defined as prodromal symptoms with pain rating of 1-10 on the Likert scale during the 16-week double-blind treatment period, and the total number of sunlight-induced non-prodrome, phototoxic reactions during the 16-week doubleblind treatment period.
About Dersimelagon (MT-7117)
Dersimelagon is a novel synthetic, orally-administered, non-peptide small molecule, which acts as a selective agonist of melanocortin 1 receptor (MC1R) with a potential for being effective to increase pain free light exposure in patients with a history of phototoxicity from erythropoietic protoporphyria (EPP) or X-linked protoporphyria (XLP). Mitsubishi Tanabe Pharma Corporation (MTPC) is developing dersimelagon as a potential treatment option for EPP or XLP.
About Mitsubishi Tanabe Pharma America, Inc.
Based in Jersey City, N.J., Mitsubishi Tanabe Pharma America, Inc. (MTPA) is a wholly-owned subsidiary of Mitsubishi Tanabe Pharma Corporation (MTPC). It was established by MTPC to develop and advance our pipeline as well as commercialize approved pharmaceutical products in North America. For more information, please visit www.mt-pharma-america.com or follow us on X (formerly Twitter), Facebook and LinkedIn.
About Mitsubishi Tanabe Pharma Corporation
Mitsubishi Tanabe Pharma Corporation (MTPC) is one of the oldest pharmaceutical companies in the world, founded in 1678, and focusing on ethical pharmaceuticals. MTPC is headquartered in Doshomachi, Osaka, the birthplace of Japan’s pharmaceutical industry. MTPC sets the MISSION of “Creating hope for all facing illness”. To that end, MTPC is working on the disease areas of central nervous system, immuno-inflammation, diabetes and kidney, and cancer. MTPC is focusing on “precision medicine” to provide drugs with high treatment satisfaction by identifying patient populations with high potential for efficacy and safety. In addition, MTPC is working to develop “around the pill solutions” to address specific patient concerns based on therapeutic medicine, including prevention of diseases, pre-symptomatic disease care, prevention of aggravation and prognosis. For more information, go to https://www.mt-pharma.co.jp/e/.
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