Study to Evaluate Efficacy, Safety, and Tolerability of MT-7117 in Subjects With Erythropoietic Protoporphyria

NCT03520036 ↗

Phase 2 Completed 102 enrolled Tanabe Pharma America, Inc.

RandomizedParallel-groupQuadruple-blindTreatment

Summary

The purpose of this study is to investigate the efficacy and safety of MT-7117 on sunlight exposure duration without symptoms and tolerance in subjects with EPP.

Timeline

Start: 2018-07-05
Primary completion: 2019-09-28
Completion: 2019-09-28

Outcome

Met primary endpoint

paper the least-squares mean difference from placebo in the change from baseline to week 16 was 53.8 minutes in the 100-mg dersimelagon group (P = 0.008) PMID 37043653 ↗

Publications

Results Balwani M, Bonkovsky HL, Levy C, Anderson KE, Bissell DM, Parker C, Takahashi F, Desnick RJ, Belongie K; Endeavor Investigators. Dersimelagon in Erythropoietic Protoporphyrias. N Engl J Med. 2023 Apr 13;388(15):1376-1385. doi: 10.1056/NEJMoa2208754.
Ogasawara A, Ide R, Inoue S, Tsuda M, Teng R. Assessment of Potential Drug-Drug Interactions for Novel Oral Melanocortin-1 Receptor Agonist Dersimelagon. Pharmacol Res Perspect. 2025 Feb;13(1):e70069. doi: 10.1002/prp2.70069.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Dersimelagon	Small molecule	100 mg	Oral
Subject	Dersimelagon	Small molecule	300 mg	Oral