EpiFrontier Therapeutics Secures $32M AMED Grant to Advance First-in-Class G9a Inhibitor for Beta Globin Disorders
核心洞察
EpiFrontier Therapeutics (搜索) received up to $32 million in non-dilutive funding from Japan's AMED to advance EPF-001 (搜索), a novel G9a (搜索) inhibitor targeting sickle cell disease (搜索) and beta thalassemia (搜索).
EPF-001 (搜索) emerged from over a decade of research at RIKEN (搜索) and Tokyo University, involving screening of 140,000 compounds to identify a highly specific G9a (搜索) inhibitor that increases fetal hemoglobin expression.
The funding will support Phase 2 clinical development with trials planned primarily at overseas institutions, while patient enrollment in Japan will focus on severe beta thalassemia (搜索) cases.
EpiFrontier Therapeutics (搜索) announced it has secured up to $32 million in non-dilutive funding from the Japan Agency for Medical Research and Development (AMED) to advance clinical development of EPF-001 (搜索), a first-in-class small molecule G9a (搜索) inhibitor designed to increase fetal hemoglobin expression in patients with sickle cell disease (搜索) and beta thalassemia (搜索).
Novel Mechanism Targets Epigenetic Regulation
EPF-001 (搜索) represents the culmination of over a decade of pioneering drug discovery research originally conducted through a collaborative effort between RIKEN (搜索) Program for Drug Discovery and Medical Technology Platforms (DMP) and Tokyo University of Pharmacy and Life Sciences. The compound emerged from a comprehensive screening campaign of approximately 140,000 compounds, followed by sophisticated molecular design using X-ray crystallographic data and the synthesis and evaluation of more than 1,000 compounds.
This integrated approach leveraged RIKEN (搜索)'s advanced drug discovery infrastructure to enable the identification of a highly specific G9a (搜索) inhibitor with demonstrated ability to increase fetal hemoglobin expression in human cells and multiple preclinical animal models. EPF-001 (搜索) has demonstrated significant advantages over standard treatments in preclinical studies in terms of both efficacy and safety, positioning it as a potentially transformative therapy for these patient populations.
Addressing Critical Unmet Medical Need
Beta globin disorders (搜索), including sickle cell disease (搜索) and beta thalassemia (搜索), affect millions of patients globally and represent significant unmet medical needs. Current treatment options remain limited, and many patients continue to experience severe complications and reduced quality of life.
"We are honored to build upon the groundbreaking research conducted by our scientific founders at RIKEN (搜索) and Tokyo University of Pharmacy and Life Sciences," said Bruce Goldsmith, Chief Executive Officer of EpiFrontier Therapeutics (搜索). "Their innovative approach to targeting G9a (搜索) has yielded a clinical candidate with the potential to meaningfully improve outcomes for patients suffering from beta globin disorders (搜索)."
Clinical Development Strategy
The AMED funding will support EPF-001 (搜索)'s advancement through Phase 2 clinical development, with trials planned to be conducted primarily at overseas medical institutions with large patient populations. Patient enrollment in Japan is specifically planned for severe beta thalassemia (搜索). The funding will enable comprehensive clinical evaluation to demonstrate the drug's efficacy and safety in humans, supporting the company's goal of achieving key commercialization milestones.
Strong Scientific Foundation and Support
Dr. Minoru Yoshida, Executive Vice President, RIKEN (搜索), emphasized the significance of the collaboration: "The development of EPF-001 (搜索) exemplifies RIKEN's commitment to translating fundamental scientific discoveries into therapies that address critical unmet medical needs through such collaborative efforts. We are pleased to see this research transition from RIKEN to EpiFrontier Therapeutics (搜索) for clinical development, and we are grateful for AMED's continued support of this project—first through the Research Project for Intractable Diseases and now through the Strengthening Program for Pharmaceutical Startup Ecosystem."
EpiFrontier Therapeutics (搜索) was founded in July 2025 as a U.S.-based corporation with a Japanese subsidiary, following extensive business feasibility discussions with The University of Tokyo Edge Capital Partners (UTEC (搜索)), which began in spring 2024. The company has secured an exclusive license to the core intellectual property arising from research conducted at RIKEN (搜索) and Tokyo University of Pharmacy and Life Sciences and will pursue additional patent applications covering formulation, dosage and administration, and combination therapies to establish a comprehensive intellectual property portfolio.
Azusa Shiohara, Principal at UTEC (搜索) and Board Member of EpiFrontier Therapeutics (搜索), stated: "EpiFrontier exemplifies the powerful combination of world-class Japanese science and global drug development expertise. The pioneering work by RIKEN (搜索) and Tokyo University of Pharmacy and Life Sciences has created a truly differentiated clinical candidate with the potential to transform treatment for patients with beta globin disorders (搜索)."