ARTham Therapeutics presents data from the phase 2 clinical trial of its investigational oral PI3Kα inhibitor ART-001 in patients with slow-flow vascular malformations at International Conference on Vascular Anomalies (VAC2023) in Brussels, Belgium. The abstract was selected as an oral presentation on February 1st, 2023.

This was a multi-center, double-blind, randomized controlled proof-of-concept study in patients with slow-flow vascular malformations. Patients received once-daily doses of either 50 mg or 100 mg of ART-001 over 24 weeks. ART-001 met the primary endpoint (the response rate based on the change in target lesion volume measured by MRI volumetry at week 24). The details of study results including efficacy, safety and pharmacokinetics will be presented at the conference.

Conference website: https://vacure-conference.net/

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, which had been presented at the American Society for Clinical Pharmacology and Therapeutics (ASCPT) 2021 Annual Meeting. ART-001 is being developed for refractory intractable vascular malformations, diseases with high unmet medical need without no effective treatment.