June 15, 2020

Start of a Phase 3 Clinical Trial of MT-7117
for the Ultra-Rare Disease, Erythropoietic Protoporphyria (EPP)and X-Linked Protoporphyria (XLP)
Mitsubishi Tanabe Pharma Corporation (MTPC, Head Office: Chuo-ku, Osaka;
President & Representative Director, CEO: Hiroaki Ueno) announced today that its
research and development subsidiary in U.S., Mitsubishi Tanabe Pharma
Development America, Inc. (MTDA, President: Hideki Kuki), has started a global
Phase 3 clinical trial of MT-7117 (dersimelagon – selective melanocortin 1 receptor(MC1R) agonist), an investigational oral treatment for the increased pain free light
exposure in adult and adolescent patients with a history of phototoxicity (includingsevere pain on exposure to sunlight) from erythropoietic protoporphyria (EPP) 1and
X-Linked Protoporphyria (XLP).
MTPC received Fast Track Designation for dersimelagon by the U.S. Food and Drug
Administration (FDA) in June 2018 and was granted Orphan Drug Designation by
FDA on June 8th 20202.
The global phase 3 trial leveraged the experience and knowledge obtained from thesuccessfully completed ENDEAVOR study. ENDEAVOR was a phase 2 proof ofconcept study for dersimelagon. Analysis of the primary endpoint showed asignificant improvement in average daily time to first prodromal symptom
associated with sunlight exposure between 1 hour post sunrise and 1 hour presunset in dersimelagon subjects compared to placebo subjects for the 100 mg (53.8min/day, p<0.008) and 300 mg (62.5 min/day, p<0.003) cohorts at Week 16.
Dersimelagon was generally well tolerated with an acceptable safety profile. These
results were presented in the late-breaking clinical session at the annual AAD 2020conference.
MTPC believes that further development and potential regulatory approval of MT7117, an oral treatment, could provide another option for healthcare providers
treating EPP.
Mitsubishi Tanabe Pharma Group will continue to advance R&D activities so that
we can deliver new therapy options that address the needs of patients around theworld fighting serious diseases as quickly as possible.
1NIH website: https://rarediseases.info.nih.gov/diseases/4527/erythropoietic-protoporphyria
https://www.accessdata.fda.gov/scripts/opdlisting/oopd/detailedIndex.cfm?cfgridkey=645518

2Mitsubishi Tanabe Pharma Corporation
Corporate Communications Department
Media contacts: TEL:+81 6 6205 5119

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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 ofphototoxicity from erythropoietic protoporphyria (EPP) and X-Linked Protoporphyria(XLP). Mitsubishi Tanabe Pharma Corporation (MTPC) is developing dersimelagon
for the treatment of EPP or XLP.
Dersimelagon is an investigational medication and not approved by FDA or any otherregulatory authority. Mitsubishi Tanabe Pharma Corporation received Fast Track
Designation for dersimelagon by the U.S. Food and Drug Administration in June
2018.
About Erythropoietic Protoporphyria and X-Linked Protoporphyria
Erythropoietic Protoporphyria (EPP) is an inherited disorder of the heme
biosynthetic pathway that results from mutations of the ferrochelatase (FECH) geneor, less commonly X-Linked Protoporphyria (XLP) that results from mutations in theaminolevulinic acid synthase-2 (ALAS2) gene. Both EPP and XLP are characterized
by accumulation of protoporphyrin in blood, erythrocytes and tissues and cutaneous
photosensitivity.

EPP and XLP usually present early in 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.
Importantly, continued exposure to sunlight following the onset of prodromal
symptoms will lead to phototoxicity-induced pain.
About Mitsubishi Tanabe Pharma Development America, Inc.
The U.S. headquarters of Mitsubishi Tanabe Pharma Development America, Inc.
(MTDA) is located in Jersey City, New Jersey. MTDA is a wholly-owned subsidiary of
Mitsubishi Tanabe Pharma Corporation’s 100 percent-owned U.S. holding company,
Mitsubishi Tanabe Pharma Holdings America, Inc. MTDA has obtained the approvalof Radicava® the new treatment option for ALS in more than 20 years in the ALS
therapeutic area in the United States. MTDA is dedicated to research and develop
innovative pharmaceutical products that address the unmet medical needs ofpatients.
https://mt-pharma-development-america.com/

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