Vaderis Therapeutics' Engasertib Studies Published in NEJM

Vaderis Therapeutics has announced the publication of its Engasertib studies in The New England Journal of Medicine. The studies focus on Engasertib's potential benefits for patients with Hereditary Hemorrhagic Telangiectasia (HHT).

The 12-week Proof-of-Concept study demonstrated dose-dependent improvements, particularly in reducing epistaxis duration and frequency. The 40 mg dose showed a 41% reduction in bleeding duration and a 28% reduction in frequency, compared to 24% and 18% respectively for placebo. Patient-reported outcomes were also favorable.

The long-term Open-Label Extension study indicated sustained benefits over 12 months. Engasertib maintained a favorable safety and tolerability profile.

Dr. Hanny Al-Samkari emphasized the potential of Engasertib as a promising treatment option for HHT, highlighting the lack of approved therapies. Vaderis plans to initiate the Phase 3 HEROIC study in 2026.