EpiFrontier Therapeutics, a biotechnology company focused on developing treatments for beta globin disorders, announced it has secured up to $32 million in non-dilutive funding from the Japan Agency for Medical Research and Development. The funding will support the clinical development of EPF-001, a novel small molecule G9a inhibitor designed to increase fetal hemoglobin expression in patients with sickle cell disease and beta thalassemia.
EPF-001 is a first-in-class therapy that emerged from more than a decade of research conducted through a collaboration between RIKEN and the Tokyo University of Pharmacy and Life Sciences. The discovery process involved screening approximately 140,000 compounds, followed by molecular design using X-ray crystallography and the synthesis and testing of over 1,000 compounds. The result is a highly selective G9a inhibitor that has demonstrated the ability to increase fetal hemoglobin expression in human cells and multiple preclinical animal models.
The company said that beta globin disorders, including sickle cell disease and beta thalassemia, affect millions of people worldwide and remain areas of significant unmet medical need. Existing treatments are limited, and many patients continue to face severe complications and reduced quality of life. In preclinical studies, EPF-001 has shown advantages in both efficacy and safety compared to standard therapies.
The AMED funding will support advancement of EPF-001 through Phase 2 clinical development. Trials are expected to take place primarily at international medical institutions with large patient populations, while patient enrollment in Japan will focus on individuals with severe beta thalassemia. The program is intended to demonstrate both efficacy and safety in humans and help move the therapy toward commercialization.
EpiFrontier Therapeutics was founded in July 2025 as a U.S.-based company with a Japanese subsidiary, following collaboration with The University of Tokyo Edge Capital Partners. The company holds exclusive rights to intellectual property stemming from research conducted at RIKEN and the Tokyo University of Pharmacy and Life Sciences and plans to expand its patent portfolio across formulation, dosage, and combination therapies.
EPF-001, also known as RK-701, targets the histone methyltransferase G9a and is designed to boost fetal hemoglobin levels, offering a potentially transformative approach for treating beta globin disorders. The therapy has demonstrated strong pharmacological activity along with a favorable safety and pharmacokinetic profile in early studies.
KEY QUOTES:
“At RIKEN, we are tackling challenges facing humanity by integrating the world-leading technological platforms possessed by each research center. One such challenge is the realization of groundbreaking drug discovery. The development of EPF-001 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 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. This partnership represents an important model for advancing Japanese innovation to benefit patients globally.”
Dr. Minoru Yoshida, Executive Vice President, RIKEN
“We are honored to build upon the groundbreaking research conducted by our scientific founders at RIKEN and Tokyo University of Pharmacy and Life Sciences. Their innovative approach to targeting G9a has yielded a clinical candidate with the potential to meaningfully improve outcomes for patients suffering from beta globin disorders. This AMED grant recognizes the potential of the underlying science and provides crucial support as we advance EPF-001 toward clinical proof-of-concept. We are committed to realizing the therapeutic promise of this molecule for patients worldwide.”
Bruce Goldsmith, Chief Executive Officer, EpiFrontier Therapeutics
“EpiFrontier exemplifies the powerful combination of world-class Japanese science and global drug development expertise. The pioneering work by RIKEN 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. We are proud to support this company as it brings this important Japanese innovation to patients worldwide, and we are grateful for AMED’s significant investment in validating and advancing this breakthrough therapy.”
Azusa Shiohara, Principal at UTEC and Board Member, EpiFrontier Therapeutics
