Phase-3 Clinical Trial of MT-7117 INITIATED IN THE U.S. for Rare DiseaseS, Erythropoietic Protoporphyria (EPP) and X-Linked Protoporphyria (XLP)

MT-7117 Receives Orphan Drug Designation from FDA

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Mitsubishi Tanabe Pharma America, Inc.

Jun 19, 2020, 13:41 ET

JERSEY CITY, N.J., June 19, 2020 /PRNewswire/ -- Mitsubishi Tanabe Pharma America, Inc. (MTPA) today announced that Mitsubishi Tanabe Pharma Development America, Inc. (MTDA) has initiated a Phase-3 clinical trial in the U.S. of the selective melanocortin 1 receptor (MC1R) agonist), MT-7117 (dersimelagon), an investigational oral therapy 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).¹

MTDA was granted Orphan Drug Designation for MT-7117 by the U.S. Food and Drug Administration on June 8, 2020², and received Fast Track Designation in June 2018.³

"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, Mitsubishi Tanabe Pharma America, Inc. "Through further clinical development and potential regulatory approval, this investigative oral treatment could provide another option for patients with EPP or XLP."

The global Phase-3 study (NCT04402489) is a multicenter, randomized, double-blind, placebo-controlled study to evaluate efficacy, safety, and tolerability of MT-7117 in approximately 159 people (age 12-75 years) with EPP or XLP over a 26-week treatment period.⁴ 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.⁴ The Phase-3 study design will leverage experience and knowledge obtained from an earlier Phase-2 study.

The Phase-2 study (NCT03520036) was a randomized, double-blind, placebo-controlled study to assess the efficacy, safety, and tolerability of MT-7117 in 102 people (age 18-75 years) with EPP.⁵ 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 22.⁵

About MT-7117 (Dersimelagon)
MT-7117 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) and X-Linked Protoporphyria (XLP). Mitsubishi Tanabe Pharma Corporation (MTPC) is developing MT-7117 as a potential treatment option for EPP or XLP. MT-7117 is an investigational medication and not approved by FDA or any other regulatory authority.

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) gene or, less commonly X-Linked Protoporphyria (XLP) that results from mutations in the aminolevulinic acid synthase-2 (ALAS2) gene.¹ Both EPP and XLP are characterized by accumulation of protoporphyrin in blood, erythrocytes and tissues and cutaneous photosensitivity.¹ 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.¹ The onset of prodromal symptoms after direct sun exposure varies but may occur in less than 10 minutes.⁶^,7 Importantly, continued exposure to sunlight following the onset of prodromal symptoms will lead to phototoxicity-induced pain.⁸

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's (MTPC) 100 percent owned U.S. holding company, Mitsubishi Tanabe Pharma Holdings America, Inc. MTPA is dedicated to delivering innovative products that address the unmet medical needs of patients in North America. It was established by MTPC to commercialize approved pharmaceutical products in North America with plans to expand its product line through collaborations with partners. For more information, please visit www.mt-pharma-america.com or follow us on Twitter, Facebook and LinkedIn.

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 is dedicated to research and develop innovative pharmaceutical products that address the unmet medical needs of patients. For more information, please visit https://mt-pharma-development-america.com/

Overview of Mitsubishi Tanabe Pharma Corporation
Mitsubishi Tanabe Pharma, which was founded in 1678, has its headquarters in Doshomachi, Osaka, which is the birthplace of Japan's pharmaceutical industry. With business centered on ethical pharmaceuticals, Mitsubishi Tanabe Pharma is a well-established company with one of the longest histories of pharmaceutical companies in Japan.⁹ In accordance with the corporate philosophy of "contributing to the healthier lives of people around the world through the creation of pharmaceuticals," the Company formulated the key concept of Open Up the Future under the Medium-Term Management Plan 2016-2020. Through the discovery of drugs that address unmet medical needs, centered on its priority disease areas — immune-inflammation diseases, diabetes and kidney, central nervous system, and vaccines — Mitsubishi Tanabe Pharma will strive to contribute to the health of patients around the world. MTPC is the parent company of MTPA and MTDA. For more information, go to http://www.mt-pharma.co.jp/.

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¹ NIH website: https://rarediseases.info.nih.gov/diseases/4527/erythropoietic-protoporphyria
² FDA website: https://www.accessdata.fda.gov/scripts/opdlisting/oopd/detailedIndex.cfm?cfgridkey=645518
³ MTPC Press Release: https://www.mt-pharma.co.jp/e/release/nr/2018/pdf/e_MTPC180727.pdf
⁴ ClinicalTrials.gov. U.S. National Library of Medicine: https://clinicaltrials.gov/ct2/show/NCT04402489
⁵ ClinicalTrials.gov. U.S. National Library of Medicine: https://www.clinicaltrials.gov/ct2/show/NCT03520036
⁶ Balwani M, Naik H, Anderson KE, Bissell DM, Bloomer J, Bonkovsky HL, et al. Clinical, biochemical, and genetic characterization of north american patients with erythropoietic protoporphyria and X-linked protoporphyria. JAMA Dermatol. 2017 Aug 1;153(8):789-96.
⁷ de Bataille S, Dutartre H, Puy H, Deybach JC, Gouya L, Raffray E, et al. Influence of meteorological data on sun tolerance in patients with erythropoietic protoporphyria in France. Br J Dermatol. 2016 Oct;175(4):768-75.
⁸American Porphyria Foundation: https://porphyriafoundation.org/for-patients/types-of-porphyria/epp-xlp/
⁹Research by TOKYO SHOKO RESEARCH, LTD.