Could a more selective treatment mean brighter days for people with PIK3CA-driven vascular malformations?

About the ReInspire Study

The ReInspire Study is evaluating an investigational study drug called RLY-2608 in individuals with PIK3CA-driven vascular malformations. PIK3CA-driven vascular malformations may include lymphatic malformations and PIK3CA-related overgrowth spectrum (PROS). PROS consists of syndromes such as Klippel-Trenaunay, CLOVES, FAVA, MCAP, and more. These types of malformations are caused by mutations or changes in the PIK3CA gene, which create a malfunctioning form of the protein PI3Kα. When mutated, PI3Kα can cause vascular malformations and overgrowths throughout the body, along with other symptoms.

The study aims to learn more about the safety and effectiveness of the study drug, RLY-2608, in the treatment of individuals with a PIK3CA-driven vascular malformation. RLY-2608 is taken orally and is designed to specifically hinder the mutated PI3Kα protein that can cause PIK3CA-driven vascular malformations. By selectively targeting the mutated proteins and limiting impact on the non-mutated proteins, the study drug aims to reduce side effects and provide more effective treatment over non-mutant-selective therapies.

Investigational study drugs have not yet been approved by the US Food and Drug Administration (FDA) or any other regulatory agency, such as the European Commission in the EU, and are not available outside of a clinical research study.

What Are PIK3CA-Driven Vascular Malformations?

PIK3CA-driven vascular malformations may include lymphatic malformations and PIK3CA-related overgrowth spectrum (PROS). PROS consists of syndromes such as Klippel-Trenaunay, CLOVES, FAVA, MCAP, and more.

This clinical research study is designed to learn about a potential new treatment option for PIK3CA-driven vascular malformations. These types of malformations are caused by mutations or changes in the PIK3CA gene, which create a malfunctioning form of the protein PI3Kα. When mutated, PI3Kα can cause vascular malformations and overgrowths throughout the body, along with other symptoms. Although everyone has a unique experience, common symptoms include pain, swelling, infections, and impaired physical functioning, which can significantly impact a person’s quality of life.