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

Phase 3 Completed 184 enrolled Tanabe Pharma America, Inc.

RandomizedParallel-groupTriple-blindTreatment

Summary

The primary objective of this study is to investigate the efficacy of MT-7117 on time to onset and severity of first prodromal symptoms (burning, tingling, itching, or stinging) associated with sunlight exposure in adults and adolescents with EPP or XLP aged 12-75.

Timeline

Start: 2020-06-01
Primary completion: 2021-12-14
Completion: 2022-07-26

Outcome

Missed primary endpoint

registry p = 0.496; Least Square Mean Difference vs Placebo 9.8 (95% CI -18.52 to 38.12) NCT04402489 ↗

registry p = 0.116; Least Square Mean Difference vs Placebo 22.7 (95% CI -5.68 to 51.09) NCT04402489 ↗

Publications

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	—	Oral