News Release

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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 https://www.arthamther.com/en/

 

Contact

Tel: +81-45-225-8858

info@arthamther.com


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We are pleased to announce that Dr. Victor Stone from Takeda Ventures was elected and appointed as a new director at our Ordinary General Meeting of Shareholders on June 16, 2021.


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Website updated.


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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”.

 

Link: https://www.amed.go.jp/koubo/11/02/1102C_00004.html

 

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.


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ARTham Therapeutics Presents Phase I Results for both ART-001 and ART-648 at ASCPT 2021 Annual Meeting

YOKOHAMA, January 15, 2021 – ARTham Therapeutics, Inc. will present Phase I results for both ART-648, an investigational PDE4 inhibitor, and ART-001, an investigational PI3Kα inhibitor in a poster session at the American Society for Clinical Pharmacology and Therapeutics (ASCPT) 2021 Annual Meeting (March 8-17, 2021). ART-648 is an orally-available, selective and potent inhibitor of PDE4 in development for the treatment of bullous pemphigoid and COVID-19. ART-001 is an orally-available selective PI3Kα inhibitor in development for the treatment of slow-flow vascular malformations. Details of the poster presentations are as follows:

Abstract title: PII-040 – The novel PDE4 inhibitor ART-648 exhibits robust target engagement at clinically safe and tolerated doses identified in healthy subjects

Abstract title: PII-041 – Safety and pharmacokinetics of novel PI3Kα inhibitor ART-001 in oral pediatric formulation for the treatment of vascular malformations

Session title: Interactive Poster Hour II

Date & Time: Friday, March 12, 6:00 – 7:00 pm (EST)

 

About ART-648

ART-648 is an orally-available PDE4 inhibitor that is currently under development for bullous pemphigoid and COVID-19. ART-648 potently and selectively inhibits PDE4 enzyme and exhibits direct “anti-inflammatory,” “antimetabolite,” and “antifibrotic” effects in multiple disease relevant in vitro or in vivo models. In addition, ART-648 possesses superior physico-chemical properties, which contribute to tolerability profile similar or better to marketed PDE4 inhibitors. Phase IIa trials are scheduled to initiate in late 2021.

 

About ART-001

ART-001 is an orally-available PI3Kα inhibitor that is currently under development for low-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. The new formulation will also enable administration in pediatric patients. Phase IIa trials are scheduled to initiate in late 2021.

 

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 https://www.arthamther.com/en/

 

Contact

Tel: +81-45-225-8858

info@arthamther.com