BASEL, Switzerland and LINCOLNSHIRE, Il., Dec. 2, 2025 /PRNewswire/ -- Vaderis Therapeutics today announced that data from its randomized, double-blind, placebo-controlled, multi-center proof-of-concept study and long-term extension of engasertib (VAD044) in Hereditary Hemorrhagic Telangiectasia (HHT) have been published in The New England Journal of Medicine (NEJM).
This peer-reviewed publication marks an important milestone following initial release of study information in August 2024 (see "Vaderis Announces Positive Clinical Proof-of-Concept Trial in HHT"). The NEJM publication further reinforces the scientific rigor and potential clinical relevance of the data.
"These early data suggest that engasertib's targeted AKT inhibition has the potential to deliver clinically meaningful improvements in bleeding for patients with HHT. These findings provide encouraging signs that we may be on a promising path toward a much-needed treatment option for this community, which remains without approved therapies," said Hanny Al-Samkari, MD, The Peggy S. Blitz Endowed Chair in Hematology/Oncology at the Mass General Brigham Cancer Institute, Associate Professor of Medicine at Harvard Medical School and the co-lead principal investigator in the study.
Study Design and Objectives
The Proof-of-Concept study enrolled 75 adults with moderate-to-severe HHT across the United States and European sites. Participants were randomized to receive oral engasertib (30 mg, 40 mg) or placebo for 12 weeks, followed by a long-term open-label extension. (ClinicalTrials.gov Identifier: NCT05406362)
Highlights from Published Results
"The publication of our results in The New England Journal of Medicine strengthens our conviction in the potential of engasertib to address the significant unmet need in HHT," said Azmi Nabulsi, MD, MPH, President & CEO, Vaderis Therapeutics. "With this important milestone as our foundation, we are moving forward with confidence as we prepare to initiate the Phase 3 HEROIC study in the first half of 2026."
About HHT
Hereditary hemorrhagic telangiectasia (HHT) is a rare genetic vascular disorder (prevalence ~1 in 3,800) characterized by recurrent severe epistaxis, anemia, and visceral arteriovenous malformations (AVMs). Despite the significant disease burden, there are currently no approved therapies for HHT globally.
About Vaderis Therapeutics
Vaderis Therapeutics is a clinical-stage biopharmaceutical company focused on discovering and advancing transformative treatments for rare vascular diseases. By targeting the underlying pathophysiology, the company aims to bring first-in-class targeted therapies to patients in need. For more information, visit www.vaderis.com.
Forward-Looking Statement
This press release contains forward-looking statements regarding the development and potential approval of engasertib and related programs. Actual results may differ materially from those described due to risks, uncertainties, and the complexities of clinical development and regulatory review.
Medical Information
Engasertib has not been approved for use in any country for any indication. Information in this press release is for medical and scientific reference only and is not intended to promote, recommend, or suggest use of this product. The safety and efficacy of engasertib have not been established by any regulatory authority.
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