Almac Discovery Originated Molecule <br> Advances to Global Phase 3 Study Following Significant Development Milestone<br> for Vaderis Therapeutics 

Almac Discovery, a research-driven drug discovery company and member of the Almac Group, is pleased to congratulate Vaderis Therapeutics on the initiation of the global Phase 3 HEROIC study of engasertib (VAD044), a molecule originally discovered by Almac Discovery. The milestone marks a significant step in the development of a potential first approved treatment for hereditary hemorrhagic telangiectasia (HHT), a rare genetic vascular disorder for which there are currently no approved treatments worldwide. 

Engasertib is a novel, allosteric subtype-selective AKT kinase inhibitor discovered and advanced through early preclinical development by Almac Discovery before being out-licensed worldwide to Vaderis Therapeutics in 2020.  

The global Phase 3 HEROIC study will evaluate the once-daily oral therapy in patients with moderate-to-severe HHT. It will build on positive proof-of-concept and long-term extension data published in the New England Journal of Medicine, demonstrating clinically meaningful and sustained improvements across multiple measures of disease.  

In addition to the launch of the Phase 3 study, Vaderis announced the completion of an oversubscribed $152 million Series B financing, expected to support operations through regulatory submissions and potential U.S. approval of engasertib. 

Professor Tim Harrison, Vice President Drug Discovery, commented: 

“The advancement of engasertib into a global Phase 3 study represents a significant milestone for Vaderis and an extremely promising potential to address a serious unmet medical need for people living with HHT. 

“To see a molecule discovered within Almac progress into pivotal-stage clinical development is tremendously rewarding and reflects the strength of our approach to discovering and advancing innovative drug candidates through strategic partnerships. We congratulate the Vaderis team on this important achievement.”