Dersimelagon(Dersimelagon) - 药物靶点：MC1R_在研适应症：红细胞生成性卟啉病，X-连锁红细胞生成性原卟啉病，X-连锁淋巴增生症_专利_临床

概要

基本信息

药物类型

小分子化药

别名

Dersimelagon Phosphate、MT-7117、MT-7117-MB

+ [1]

靶点

MC1R

作用方式

激动剂

作用机制

MC1R激动剂(黑素皮质素受体1激动剂)

治疗领域

免疫系统疾病遗传病与畸形消化系统疾病+ [4]

在研适应症

红细胞生成性卟啉病X-连锁红细胞生成性原卟啉病X-连锁淋巴增生症+ [1]

非在研适应症

紫绀和肝病弥漫性硬皮病

原研机构

Tanabe Pharma Corp.

在研机构

University of Michigan

Tanabe Pharma America, Inc.

Mitsubishi Tanabe Pharma Development America, Inc.

+ [1]

非在研机构-

权益机构

LEO Pharma A/S

Tanabe Pharma Corp.

最高研发阶段临床3期

首次获批日期-

最高研发阶段(中国)临床1期

特殊审评快速通道 (美国)、孤儿药 (美国)

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结构/序列

分子式C36H45F4N3O5

InChIKeyMUNWOYRHJPWQNE-GMFUQMJFSA-N

CAS号1835256-48-8

关联

13

项与 Dersimelagon 相关的临床试验

NCT06994286

/ Completed临床1期

A Phase 1, Randomized, Double-Blind, Placebo-Controlled Study to Investigate the Pharmacokinetics, Safety, Tolerability and Pharmacodynamics of Single and Multiple Doses of MT-7117 in Chinese Healthy Subjects

The purpose of this study is to investigate the safety, tolerability and pharmacokinetics of MT-7117 in Chinese healthy subjects.

开始日期2025-06-24

申办/合作机构

Tanabe Pharma Corp.

NCT06144840

/ Completed临床3期

A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Efficacy, Safety, and Tolerability of MT-7117 in Adults and Adolescents With Erythropoietic Protoporphyria or X-Linked Protoporphyria

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.

开始日期2023-12-11

申办/合作机构

Tanabe Pharma America, Inc.

NCT05241535

/ Completed临床1期

A Phase 1, Randomized, Partial Double-Blind, Placebo- and Positive-Controlled, Three-way Crossover Study to Evaluate the Effect of MT-7117 on the QT/QTc Interval in Healthy Subjects

The purpose of this study is to evaluate the effect of a single oral dose of MT-7117 on the QT/QTc interval in healthy subjects.

开始日期2022-01-12

申办/合作机构

Tanabe Pharma America, Inc.

100 项与 Dersimelagon 相关的临床结果

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100 项与 Dersimelagon 相关的转化医学

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100 项与 Dersimelagon 相关的专利（医药）

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13

项与 Dersimelagon 相关的文献（医药）

2026-03-04·EXPERT OPINION ON PHARMACOTHERAPY

New and currently investigated pharmacotherapies for the erythropoietic protoporphyrias: spotlight on dersimelagon and bitopertin

Review

作者: Falchetto, Rocco ; Dechant, Cornelia ; Barman-Aksözen, Jasmin ; Wäscher, Sebastian ; Granata, Francesca

INTRODUCTION:

The erythropoietic protoporphyrias (EPP) are ultra-rare inborn errors of the heme biosynthesis characterized by painful and debilitating phototoxic reactions in the blood vessels upon exposure to visible light. Afamelanotide is the only approved treatment for EPP and effectively prevents pain and prolongs the time patients can spend in sunlight. However, afamelanotide does not address the underlying disease mechanism and is currently only approved for use in adult patients, leaving children and adolescents without a treatment option. The two investigational pharmacotherapies, dersimelagon and bitopertin, could offer benefits such as treatment options for children and prevention of some of the associated disease complications.

AREAS COVERED:

This narrative review (using Pubmed and trial databases) aims to provide an overview of the status of development of dersimelagon and bitopertin, with a focus on the prevention of phototoxicity.

EXPERT OPINION:

The currently available trial data on dersimelagon and bitopertin suggests treatment effects in EPP as compared to placebo control groups. However, safety and efficacy of dersimelagon and bitopertin need to be further characterized. Moreover, to ensure benefit for patients and access to therapy after regulatory approval, it also would be important to generate data on the relative safety and efficacy as compared to afamelanotide.

2025-02-01·Pharmacology Research & Perspectives

Assessment of Potential Drug–Drug Interactions for Novel Oral Melanocortin‐1 Receptor Agonist Dersimelagon

Article

作者: Ogasawara, Akihito ; Inoue, Shinsuke ; Teng, Renli ; Ide, Ryosuke ; Tsuda, Minoru

ABSTRACT:

Dersimelagon is a novel investigational orally administered selective agonist of the melanocortin‐1 receptor. The drug–drug interaction (DDI) potential of dersimelagon was investigated in both nonclinical (in vitro) and clinical studies. The in vitro inhibition of CYP/UGT isoforms and efflux/uptake transporters by dersimelagon was assessed. The impact of 300‐mg dersimelagon on the pharmacokinetics (PK) of substrate drugs and the effect of co‐administering verapamil on 100‐mg dersimelagon PK (as substrate drug) were investigated in healthy participants in a Phase 1 study. DDIs were assessed based on ratios of Cmax and AUC0‐∞ of substrate drug administered alone and with dersimelagon (or verapamil). Relatively potent in vitro inhibition of CYP2C9, CYP3A, UGT1A1, BCRP, P‐gp, and OATPs by dersimelagon was observed. In the clinical study, exposures of atorvastatin (CYP3A, P‐gp, BCRP, OATP substrate) rosuvastatin (BCRP and OATP substrate), and β‐hydroxy simvastatin (metabolite of simvastatin) increased 2‐ to 3‐fold (atorvastatin: Cmax LS mean ratio = 198.0%; AUC0‐∞ ratio = 196.6%; rosuvastatin: Cmax ratio = 316.5%, AUC0‐∞ ratio = 206.0%) when co‐administered with dersimelagon. Midazolam (CYP3A substrate), digoxin (P‐gp), pravastatin (OATP), and simvastatin (CYP3A) did not show any clinically relevant DDI effects when co‐administered with dersimelagon. Dersimelagon exposure increased ~25% when co‐administered with verapamil, an effect not considered clinically relevant. Dersimelagon 300 mg did not elicit major DDIs involving CYP/UGT enzymes and drug transporters; however, dersimelagon may have potential for clinically relevant DDIs with drugs that are substrates for BCRP, such as atorvastatin and rosuvastatin, and caution should be exercised when co‐administering 300‐mg dersimelagon with these statin drugs.Trial Registration:ClinicalTrials.gov: NCT04793295, NCT04402489, NCT04440592, NCT02834442, NCT03520036, NCT03503266

2024-12-26·JOURNAL OF MEDICINAL CHEMISTRY

Discovery of MT-7117 (Dersimelagon Phosphoric Acid): A Novel, Potent, Selective, and Nonpeptidic Orally Available Melanocortin 1 Receptor Agonist

Article

作者: Adachi, Takashi ; Andou, Junki ; Suzuki, Tsuyoshi ; Morokuma, Kenji ; Sato, Atsushi ; Ogasawara, Akihito ; Kawano, Yuko ; Kondo, Masahiro ; Miyashiro, Masahiko ; Yamamoto, Yasuo ; Ide, Mika

Activation of the melanocortin 1 receptor (MC1R) mediates melanogenesis in melanocytes, anti-inflammatory effects in inflammatory cells, and antifibrotic effects in fibroblasts. Thus, MC1R agonists are expected to be beneficial for treating skin, autoimmune, inflammatory, and fibrotic diseases. Afamelanotide, an α-melanocyte-stimulating hormone (α-MSH) analogue MC1R agonist, is used clinically for treating erythropoietic protoporphyria (EPP) as a subcutaneous implant formulation. We explored nonpeptidic small-molecule MC1R agonists with the aim of identifying more convenient oral drugs. By exploring the structure of previously reported compound 5, we discovered compound 11 (MT-7117: dersimelagon phosphoric acid). This compound exhibited strong MC1R agonistic activity, good pharmacokinetic properties, and excellent safety profiles. Furthermore, compound 11 was effective in animal pigmentation evaluation and skin fibrosis model studies. Compound 11 is currently in clinical trials for the treatment of EPP, X-linked protoporphyria (XLP), and systemic sclerosis (SSc). Proof of concept was obtained in phase 2 clinical studies on EPP and XLP.

40

项与 Dersimelagon 相关的新闻（医药）

2026-08-24

·BioPharmaDive

Leo Pharma buys rare skin disease drug in $435M deal

Leo Pharma on Tuesday said it will acquire the rights to dersimelagon, an experimental medicine from that’s undergoing Food and Drug Administration review, in a deal potentially worth hundreds of millions of dollars. If approved, the medicine would be the first oral treatment for two skin conditions known as erythropoietic protoporphyria and X-linked protoporphyria, Leo said. The rare genetic diseases cause severe reactions to the sun; patients can experience burning sensations, rashes, swelling and in some cases even liver damage. Leo did not release exact details of the transaction, other than saying it includes as much as $435 million in upfront and near-term milestone payments “together with potential downstream milestones and tiered royalties on net sales.” The private company, co-owned by the Leo Foundation and Nordic Capital, separately said its first-half revenue rose 10% to 7.26 billion Danish kroner, or about $1.12 billion. The deal adds a late-stage asset to Leo’s pipeline as the dermatology company works toward a goal of launching a new medicine every two to three years and considers an initial public offering. The Danish drugmaker said the acquisition will build on its purchase of the gene therapy specialist Reply in April and a partnership with Boehringer Ingelheim announced last year that centers on the drug Spevigo. Tanabe, a Japanese pharmaceutical company now owned by Bain Capital, submitted its application for dersimelagon to the FDA in June. The agency has granted the drug Fast Track and Orphan Drug designations, designed to reward makers of therapies that fill an unmet need by speeding up development and offering benefits such as tax credits and exemptions from user fees. “Dersimelagon represents a compelling opportunity to expand our rare dermatology pipeline with a late-stage oral therapy candidate,” Leo CEO Christophe Bourdon said in the company’s statement. “This acquisition is closely aligned with our strategy of identifying and investing in high-impact innovation in medical dermatology.” A Phase 3 study released earlier this year showed dersimelagon could help patients spend more time in sunlight while experiencing fewer episodes of pain. Tanabe said the drug was generally well tolerated; the most common side effects were benign moles, headache, nausea, diarrhea and skin hyperpigmentation. The content above comes from the network. if any infringement, please contact us to modify.

孤儿药并购快速通道临床结果

2026-08-24

·PMLiVE

LEO Pharma acquires rights for dersimelagon from Tanabe Pharma

LEO Pharma has agreed to acquire worldwide rights to dersimelagon from Tanabe Pharma. Dersimelagon is an oral, once-daily small MC1R agonist being developed for EPP and XLP. EPP and XLP are rare and severe genetic diseases that cause severe sunlight-induced skin damage and can significantly limit patients’ ability to spend time outdoors. Symptoms may include skin rash, swelling, redness, burning sensations and, in some cases, liver damage. Dersimelagon is designed to increase skin melanin, helping reduce sunlight penetration and protect against phototoxic reactions. “Patients living with EPP or XLP face the devastating lifelong burden of severe reactions to sunlight, and there is a clear need for treatment options that can make a meaningful difference in their everyday lives,” said Christophe Bourdon, CEO of LEO Pharma. “By addressing a clear unmet need in a rare skin disease, dersimelagon represents a compelling opportunity to expand our rare dermatology pipeline with a late-stage oral therapy candidate.” If approved, dersimelagon would represent the first oral therapy for the treatment of EPP and XLP and help address an area of significant unmet need. Under the terms of the agreement, LEO Pharma will acquire worldwide rights to dersimelagon from Tanabe Pharma for up to $435m in up front and near-term milestone payments together with potential down- stream milestones and tiered royalties on net sales. Dersimelagon has been granted both US FDA Fast Track Designation and Orphan Drug Designation, and an NDA for the treatment of EPP and XLP was submitted to the US FDA for regulatory review in June 2026, but has not yet been approved by the FDA or any other regulatory agency. “EPP and XLP are devastating lifelong diseases that can severely limit patients’ ability to live everyday lives free from sunlight-induced pain, and dersimelagon has the potential to become an important new treatment option,” said Akihisa Harada, CEO of Tanabe Pharma. Earlier this year, Tanabe Pharma announced results from the global, randomised, double-blind, placebo-controlled phase 3 INSPIRE study of EPP and XLP that showed dersimelagon demonstrated statistically significant and clinically meaningful outcomes across primary and secondary endpoints, including key functional outcomes such as a significant prolongation of average daily sunlight exposure time to first prodromal symptoms. The phase 3 data was presented as a late breaker at the 2026 American Academy of Dermatology Annual Meeting (AAD).

临床3期临床结果快速通道孤儿药引进/卖出

2026-08-21

·TPC Health Biobrief

生物医药动态 2026年8月17日–8月21日

一、海外重磅动态 1. Moderna+默沙东：intismeran autogene（mRNA‑4157/V940）III期阳性（8.19） INTerpath‑001研究针对完全切除后IIB～IV期高危黑色素瘤术后辅助治疗，联合帕博利珠单抗同时达成RFS（无复发生存期）、DMFS（无远处转移生存期）双主要终点；总生存期OS仅作为次要终点，试验将继续长期随访。两家计划向FDA、EMA提交上市申请，为全球首个III期阳性的个体化mRNA新抗原肿瘤疫苗。消息推动Moderna股价单日大涨超176%。备注：定位为术后辅助防复发，并非用于晚期大块肿瘤治疗，完整疗程最多9剂、每3周给药一次。 2. 跨国药企BD/并购（8.17–8.20） - 8.17：强生与Sail Biomedicines达成CAR‑T合作，拥有最高25.8亿美元的收购选择权；同期强生将一款淋巴瘤双特异性CAR‑T资产返还AbelZeta。 - 8.18：LEO Pharma收购dersimelagon；BioMarin收购Alesta；礼来与OmniAb达成抗体研发合作。二、国内新药获批 & 临床进展 1. 恒瑞医药（8.20，双项公告） ① 1类新药富马酸立康可泮胶囊（HRS‑5965）获批上市，补体因子B抑制剂，用于初治阵发性睡眠性血红蛋白尿症（PNH）成人患者，可同时抑制血管内、血管外溶血，累计研发投入约2.78亿元； ② SHR‑8203注射液（全新抗肿瘤分子，国内外暂无同类上市）获批晚期实体瘤临床；SHR‑7012注射液获批皮肤型红斑狼疮临床。 2. 舒泰神（8.20）子公司注射用STSP‑0601获美国FDA临床许可，用于伴抑制物血友病A/B出血；该1类生物药已在国内获批上市。 3. 维立志博（8.20） PD‑L1/4‑1BB双抗维利信®联合化疗一线治疗非小细胞肺癌II期摘要公布：60例可评估患者ORR 65%、DCR 95%，鳞癌、非鳞癌亚组均有效，安全性可控；完整7个月以上随访数据将在WCLC大会口头报告披露。三、国内融资 & BD交易 1. 复宏汉霖 × 山德士（8.17）签署生物类似药框架合作，最多覆盖10款单抗/ADC类似药；首批3款（西妥昔单抗、依洛尤单抗、贝利尤单抗类似药）+选择权，总里程碑上限3.22亿美元，约定销售分成最高40%，面向全球市场推进商业化。 2. 时安生物（8.20）完成近4亿元A轮系列融资（Pre‑A/A/A+轮），聚焦创新蛋白/多肽药物研发。 3. 橙帆医药（8.21）完成超6000万美元Pre‑A+轮融资，推进多特异性抗体、ADC（FIC/BIC）管线进入全球临床，布局肿瘤、自身免疫病领域，老股东持续加注。四、行业小热点受intismeran阳性消息带动，国内个体化mRNA新抗原疫苗赛道（立康生命、新合生物、云顶新耀、恒瑞瑞宏迪等）受到资本市场集中关注；国内同类管线大多仍处于I期注册临床或IIT阶段，进度与海外存在明显代差，且普遍优先布局胰腺癌、胃癌等亚洲高发“冷肿瘤”，和黑色素瘤的海外路线形成差异化。 — 入群交流 — — 关于我们 — TPC Health 简介 Trust/Target-Platform-Collaboration Health Club (TPC Health)，起源于清华大学、北京大学和康奈尔大学，寓意着生物医药领域的 “信任灯塔” 与 “创新合作引擎”。在技术迭代瞬息万变的当下，我们以 “可信赖” 为立身之本，依托深厚的行业洞察，打破地域与学科的界限，整合全球优质资源，构建全链条和一个充满信任的合作生态，搭建起科研、产业、临床深度融合的桥梁。 — 往期精彩 — — 我们来自 — — 欢迎关注 —

100 项与 Dersimelagon 相关的药物交易

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研发状态

10 条进展最快的记录,

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适应症	最高研发状态	国家/地区	公司	日期
红细胞生成性卟啉病	临床3期	美国	Tanabe Pharma America, Inc.	2021-08-10
红细胞生成性卟啉病	临床3期	日本	Tanabe Pharma America, Inc.	2021-08-10
红细胞生成性卟啉病	临床3期	澳大利亚	Tanabe Pharma America, Inc.	2021-08-10
红细胞生成性卟啉病	临床3期	保加利亚	Tanabe Pharma America, Inc.	2021-08-10
红细胞生成性卟啉病	临床3期	加拿大	Tanabe Pharma America, Inc.	2021-08-10
红细胞生成性卟啉病	临床3期	捷克	Tanabe Pharma America, Inc.	2021-08-10
红细胞生成性卟啉病	临床3期	法国	Tanabe Pharma America, Inc.	2021-08-10
红细胞生成性卟啉病	临床3期	德国	Tanabe Pharma America, Inc.	2021-08-10
红细胞生成性卟啉病	临床3期	意大利	Tanabe Pharma America, Inc.	2021-08-10
红细胞生成性卟啉病	临床3期	荷兰	Tanabe Pharma America, Inc.	2021-08-10

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临床结果

适应症

分期

评价

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT04440592 (CTgov)	临床2期	弥漫性硬皮病	76	MT-7117 (MT-7117)	鏇範繭構鹽憲壓糧繭餘(憲廠鏇夢鏇艱齋簾壓鏇) = 夢選壓願壓範壓構壓蓋鹹衊膚構蓋夢膚鏇糧醖 (壓餘鏇遞鏇鑰顧壓餘製, 構窪願醖齋網獵遞簾製 ~ 觸鑰鏇衊廠製鹽蓋範鑰) 更多	-	2025-11-03
				Placebo (Placebo)	鏇範繭構鹽憲壓糧繭餘(憲廠鏇夢鏇艱齋簾壓鏇) = 遞網夢願膚繭衊遞鹽製鹹衊膚構蓋夢膚鏇糧醖 (壓餘鏇遞鏇鑰顧壓餘製, 窪鬱顧鑰願窪衊簾鬱範 ~ 鹹醖夢鹽選憲網繭淵憲) 更多
NCT04402489 (CTgov)	临床3期	X-连锁红细胞生成性原卟啉病 \| X-连锁淋巴增生症	184	placebo (DBT ITT1 Placebo)	蓋鹹壓鏇獵膚選襯繭蓋(淵積鬱襯蓋積壓鑰齋願) = 衊鏇餘築糧廠窪廠壓築鹽醖夢鹽願膚艱膚艱襯 (艱醖憲製網獵窪醖衊積, 10.29) 更多	-	2025-03-21
				MT-7117 (DBT ITT1 MT- 7117 Low Dose)	蓋鹹壓鏇獵膚選襯繭蓋(淵積鬱襯蓋積壓鑰齋願) = 窪網膚窪築觸壓齋繭遞鹽醖夢鹽願膚艱膚艱襯 (艱醖憲製網獵窪醖衊積, 10.04) 更多
NCT02834442 (Pubmed)	临床1期	-	144	Dersimelagon	糧顧範醖蓋糧範夢鏇壓(壓憲築襯憲鏇簾網鹽簾) = 齋簾築獵醖鹹窪糧觸範顧餘網鹽鹽糧願繭醖壓 (遞積廠製夢遞夢構餘醖 ) 更多	-	2023-04-15
				Placebo	糧顧範醖蓋糧範夢鏇壓(壓憲築襯憲鏇簾網鹽簾) = 網鹹網鬱顧鹽選願衊憲顧餘網鹽鹽糧願繭醖壓 (遞積廠製夢遞夢構餘醖 )
NCT03520036 (CTgov)	临床2期	红细胞生成性卟啉病 \| 红细胞生成性原卟啉症	102	MT-7117 low dose (MT-7117 Low Dose)	衊餘膚積餘範餘衊顧衊(範廠憲艱願積願鬱壓鑰) = 積膚齋襯糧廠鏇鏇鬱窪醖簾餘鏇鹽構壓膚遞願 (夢顧鹹網網齋壓壓廠醖, 76.8) 更多	-	2023-01-20
				MT-7117 high dose (MT-7117 High Dose)	衊餘膚積餘範餘衊顧衊(範廠憲艱願積願鬱壓鑰) = 鬱選窪製構襯簾夢構願醖簾餘鏇鹽構壓膚遞願 (夢顧鹹網網齋壓壓廠醖, 100) 更多