News Release

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ARTham Therapeutics again receives the rare disease drug development grant from AMED for its ART-001 program (development for the treatment of slow-flow vascular malformations)

ARTham’s clinical program ART-001, an oral selective PI3Kα inhibitor, for slow-flow vascular malformations in both pediatric and adult population was awarded additional three-year R&D grant from “the Japan Agency for Medical Research and Development (AMED) Program for Orphan Drugs prior to Designation”.




Slow-flow vascular malformations are benign (non-cancerous) lesions that are present at birth, and may become visible for weeks or months after birth. Unlike hemangiomas, vascular malformations continue to grow slowly throughout life. It has been reported that most patients with slow-flow vascular malformation have a gain-of-function mutation in PI3K pathway, which plays an important pathological role in vascular formation. ART-001 inhibits PI3Kα with high selectivity, being expected to exert clinically meaningful therapeutic effect.

ART-001 program had been supported by AMED for last 3 years and had made a significant progress including new formulation development, completion of Ph-1 clinical trial as well as multiple clinical research to better understand the disease background such as gene mutation and natural history. Ph-2 clinical trial is anticipated to start in third quarter of 2021. ARTham is striving to deliver medicines that matter for patients.