Drug Profile

EPF 001

Alternative Names: EPF-001; RK-701

Latest Information Update: 28 Apr 2026

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At a glance

Originator RIKEN; Tokyo University of Pharmacy and Life Sciences
Class Antianaemics; Small molecules
Mechanism of Action EHMT2 protein inhibitors

Orphan Drug Status

Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

No
New Molecular Entity Yes

Highest Development Phases

Preclinical Beta-thalassaemia; Sickle cell anaemia

Most Recent Events

24 Mar 2026 EpiFrontier Therapeutics plans a phase-II trial for Sickle cell anaemia and Beta thalassemia in Japan and other countries
24 Mar 2026 Preclinical trials in Beta-thalassaemia in Japan (PO)
24 Mar 2026 Preclinical trials in Sickle cell anaemia in Japan (PO)

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