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ARTham announces first patient dosed in Phase 2 clinical trial of ART-001 in patients with slow-flow vascular malformations.

ARTham Therapeutics announced today that the first patient has been dosed in a Phase 2 clinical trial evaluating ART-001 as a potential treatment for slow flow vascular malformations (jRCT2071210027). ART-001 is a novel PI3Kα selective inhibitor with excellent pharmacokinetic and safety profile that have been demonstrated in its Phase 1 trial in healthy adults.

This Phase 2 trial is a multi-center, double-blind, randomized controlled study to evaluate the efficacy, safety, and pharmacokinetics of ART-001 in pediatric (≥ 2 years old) and adult patients with slow-flow vascular malformations including venous malformation, lymphatic malformation (lymphangioma) and Klippel-Trenaunay syndrome. The trial is enrolling a total of 34 patients. The primary endpoint of the trial is the proportion of subjects with response defined by MRI volumetric change of target lesion at Week 24. Secondary efficacy endpoints include the proportion of subjects with response at Week 12, changes from baseline in MRI volume of target lesion, pain VAS, QOL score and performance status.


About slow flow vascular malformations

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 malformation.


About ART-001

ART-001 is an orally-available PI3Kα inhibitor that is currently under development for slow-flow vascular malformations. ART-001 potently and selectively inhibited PI3Kα and exhibits “antitumor effect” and “anti-angiogenesis effect” in in vitro and in vivo preclinical models. In addition, the excellent pharmacokinetic and safety profile with the new formulation was demonstrated in the Phase I study in healthy adults.


About ARTham Therapeutics

ARTham Therapeutics is a clinical stage biopharmaceutical company that will deliver “medicines that matter” for patients, with a compelling portfolio developed through unraveling hidden value of existing pharma assets. ARTham is a virtual R&D company that seeks the best academic and business partners to rapidly develop high value clinical candidates. The company is headquartered in Yokohama, Japan. For more information, please visit



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