Vaderis Therapeutics Announces Oversubscribed $152 Million Series B Financing and Initiation of the Global Phase 3 HEROIC Study of Engasertib for Hereditary Hemorrhagic Telangiectasia

Together, these milestones mark a significant step forward in the development of engasertib, an investigational oral allosteric AKT inhibitor. Engasertib is positioned to become the first approved therapy specifically developed for people living with HHT, a rare genetic vascular disorder that currently has no approved treatment options worldwide.

The Series B financing, supported by both new and existing investors, reflects strong confidence in the potential of engasertib to address significant unmet needs in HHT.  The proceeds from the financing are expected to fund the company's planned operations through regulatory submissions and potential U.S. regulatory approval.

Following the closing, Vaderis' Board of Directors comprises Giovanni Mariggi of Medicxi, Colin Walsh of Goldman Sachs Alternatives, Giuliano Marostica of TCGX, Francesco Draetta of Omega Funds, Nick Williams of Medicxi, Azmi Nabulsi, President and Chief Executive Officer of Vaderis, and Rahul Ballal, who serves as an independent director.

The financing and initiation of HEROIC follows publication of positive proof-of-concept and long-term extension data for engasertib in The New England Journal of Medicine, which demonstrated clinically meaningful and sustained improvements across multiple measures of disease in patients with HHT. These data established the scientific foundation for advancing engasertib into pivotal development and its potential to address the significant unmet needs of patients living with HHT.

"Today represents a defining moment for HHT patients," said Azmi Nabulsi, MD, MPH, President and Chief Executive Officer of Vaderis Therapeutics. "Closing this financing and initiating HEROIC as the first Phase 3 study utilizing a molecule specifically developed for HHT marks an exciting new chapter. This milestone reflects the dedication of our patients, investigators, study teams, and advocacy organizations, to whom we extend our deepest gratitude. We are also thankful to our investors for their confidence and support, which have been essential in bringing us to this point."

"Having partnered with Vaderis since its inception, Medicxi has seen the company consistently translate cutting-edge science into meaningful clinical progress," said Giovanni Mariggi, co-founder and Partner, Medicxi and Chairman of Vaderis Therapeutics. "The advancement of engasertib into Phase 3 represents the culmination of years of disciplined execution, scientific innovation and close collaboration with the HHT community. Building on the important contributions of clinicians and scientists that have advanced the field, we are proud to continue supporting Vaderis as it pioneers a regulatory pathway for therapies specifically developed for HHT, while working to bring the first such treatment to patients.

"Vaderis has generated compelling clinical evidence supporting targeted AKT inhibition as a novel treatment approach for HHT," said Colin Walsh, PhD, Managing Director, Life Sciences at Goldman Sachs Alternatives. "The company's strong scientific foundation, disciplined execution and clear focus on addressing a significant unmet medical need gave us conviction in both the financing and the Phase 3 program."

"We are pleased to partner with Vaderis and a high-quality investor syndicate to advance engasertib through this important stage of development," added Giuliano Marostica, Managing Partner, TCGX.

Phase 3 HEROIC Study Now Underway

HEROIC is a global, randomized, double-blind, placebo-controlled Phase 3 clinical study designed to evaluate the efficacy and safety of once-daily oral engasertib in patients with moderate-to-severe HHT. The study is expected to enroll patients across sites in North America, South America and Europe.

"HHT remains a serious, lifelong disease that places a substantial burden on patients, yet there are still no approved therapies," said Hanny Al-Samkari, MD, Associate Professor of Medicine at Harvard Medical School, The Peggy S. Blitz Endowed Chair in Hematology/Oncology at Mass General Brigham Cancer Institute, and Principal Investigator of the HEROIC study. "As the Principal Investigator of HEROIC, I believe this study has been thoughtfully designed to rigorously evaluate engasertib in a larger patient population and confirm the encouraging findings from the earlier proof-of-concept study."

About Engasertib (VAD044)

Engasertib is an investigational oral selective allosteric inhibitor of AKT1/2 being developed for the treatment of hereditary hemorrhagic telangiectasia (HHT), a rare genetic vascular disorder characterized by recurrent bleeding and arteriovenous malformations. By targeting dysregulated signaling pathways implicated in vascular malformations, engasertib is designed to address the underlying pathophysiology of disease.

Engasertib has not been approved for use in any country for any indication.

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 science-driven 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. Vaderis is headquartered in Basel, Switzerland with a U.S. subsidiary in Lincolnshire, Illinois. For more information, visit www.vaderis.com.

Forward-Looking Statement

This press release contains forward-looking statements regarding the development, regulatory review, and potential approval of engasertib and related programs, the anticipated initiation, timing, and execution of clinical trials, the expected use of proceeds from the financing, and the company's anticipated operational runway. By their nature, forward-looking statements involve a number of risks, uncertainties and assumptions that could cause actual results or events to differ materially from those expressed or implied by the forward-looking statements, including due to risks, uncertainties, and the inherent complexities of clinical development, regulatory review, financing activities, manufacturing, and other factors.

This press release does not constitute an offer or solicitation for the purchase or disposal of, trading or any transaction in any securities of Vaderis Therapeutics in any jurisdiction.

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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SOURCE Vaderis Therapeutics AG