Vaderis Therapeutics
A Basel-based clinical-stage biotech advancing the first disease-specific therapeutic candidate for hereditary hemorrhagic telangiectasia into pivotal development
Vaderis Therapeutics AG is a clinical-stage biopharmaceutical company founded in 2018–2019 in Basel, Switzerland, with a mandate to develop therapeutics for rare and orphan diseases caused by vascular malformations. The company is headquartered at Building WSJ-350, Lichtstrasse 35, CH-4056 Basel, with a U.S. subsidiary in Lincolnshire, Illinois.
A Harvard Business School alumnus (2003–2005), Benedict built a two-decade operating track record across European pharma and biotech prior to founding Vaderis. He served as Chief Operating Officer at Novartis Corp, Chief Business Officer at Basilea Pharmaceutica, and Executive Vice President, Head of Europe at Archimedes Pharma, followed by a CEO stint at Lumavita AG (2008–2009) and, in 2013, the founding of antibiotics developer Allecra Therapeutics, which he led as CEO through 2018. Per BioCentury reporting, the genesis of Vaderis traces to 2018, when Benedict and two former Novartis colleagues — Pierre Saint-Mezard and Damien Picard — identified a link between AKT signaling and vascular-malformation disease biology after reviewing a paper published in Nature Communications, which became the founding scientific thesis for the company. Leadership has since transitioned: the company’s current President & CEO is Azmi Nabulsi, MD, MPH.
A former Novartis colleague of Benedict and Picard, Saint-Mezard co-founded Vaderis and led the scientific case for repurposing AKT inhibition — an oncology-derived mechanism — toward a genetically validated, non-oncology rare-disease indication.
A physician-executive with more than 30 years of drug development and commercialization experience, Nabulsi co-founded Phathom Pharmaceuticals and helped steer it from private financing through FDA approval and its transition to a publicly traded company. His background also includes senior global R&D leadership roles at Takeda and Abbott, plus time at Frazier Healthcare Partners and ReAlta Life Sciences.
Data caveat: The precise timing and circumstances of the CEO transition from Nicholas Benedict to Azmi Nabulsi have not been explicitly disclosed in company press releases. This account is reconstructed by cross-referencing publicly available materials at different points in time — Benedict was identified as CEO in the company’s 2022 stealth-exit announcement, while Nabulsi is identified as President & CEO in 2025–2026 materials.
Vaderis is a clinical-stage biopharmaceutical company targeting rare diseases caused by vascular malformations, with its lead asset engasertib (VAD044) in development for hereditary hemorrhagic telangiectasia (HHT). HHT is the second most common inherited bleeding disorder globally, with a prevalence of roughly 1 in 3,800 — yet no approved therapy currently exists anywhere in the world.
A once-daily, oral, selective allosteric inhibitor of AKT1/2. In HHT, mutations in the ALK1 pathway drive AKT overactivation, resulting in aberrant, fragile vascular growth; engasertib is designed to inhibit AKT signaling and restore vascular stability. The compound was originally discovered by Almac Discovery and in-licensed by Vaderis in 2020.
A randomized, double-blind, placebo-controlled, multicenter trial enrolling 75 adults with moderate-to-severe HHT across U.S. and European sites. Both the 30mg and 40mg dose arms demonstrated dose-dependent reductions in epistaxis frequency and duration relative to placebo, with benefit sustaining and deepening through a 12-month open-label extension. Results were published in the New England Journal of Medicine in December 2025.
A global, randomized, double-blind, placebo-controlled Phase 3 study evaluating oral engasertib across sites in North America, South America, and Europe in patients with moderate-to-severe HHT. The Principal Investigator is Dr. Hanny Al-Samkari of Harvard Medical School / Mass General Brigham. HEROIC is described as the first Phase 3 study of a molecule developed specifically for HHT.
Proof-of-concept data detail: Mean reduction in epistaxis frequency from baseline was 26.5% in the 30mg arm, 27.8% in the 40mg arm, and 18.0% on placebo; corresponding reductions in epistaxis duration were 29.9%, 41.4%, and 23.8%, respectively. The most common adverse event was mild-to-moderate rash, observed in 21% (30mg), 42% (40mg), and 8% (placebo) of patients, with serious adverse event rates broadly similar across arms.
Data caveat: Certain secondary pharma databases tag engasertib as an “Orphan Drug,” but this framing frequently conflates the underlying disease’s rare/orphan classification with a formal regulatory designation for the compound itself. The only regulatory designation explicitly confirmed in primary company communications is FDA Fast Track (Nov 2024); a distinct, formal Orphan Drug Designation from FDA or EMA is not clearly substantiated by primary sources and would require independent verification.
Vaderis was established in 2019 under Medicxi’s venture-creation model and has since progressed through an initial 2020 financing to a substantial 2026 Series B, bringing disclosed cumulative funding to an estimated ~$170M (subject to currency and reporting-basis differences across sources — see caveat below). The August 2026 $152M Series B was co-led by Goldman Sachs Alternatives and TCGX and closed oversubscribed, indicating institutional demand exceeded the round’s allocated capacity.
Co-founded by former Novartis executives Nicholas Benedict, Pierre Saint-Mezard, and Damien Picard. Medicxi partner Giovanni Mariggi joined as Board Chairman, reflecting the asset-centric, venture-creation structure characteristic of Medicxi-backed companies.
Vaderis raised more than CHF 18 million from Medicxi and, in April 2020, acquired a portfolio of allosteric AKT inhibitors from Almac Discovery of Belfast. Proceeds funded the lead asset’s advancement into the clinic and completion of Phase 1a.
Vaderis publicly emerged from stealth mode and announced initiation of INSIGHT, a randomized, double-blind, placebo-controlled proof-of-concept trial in HHT patients.
Vaderis received FDA Fast Track Designation in November 2024, followed by positive topline PoC results in August 2025 and peer-reviewed publication of PoC and long-term extension data in the New England Journal of Medicine in December 2025, externally validating the scientific thesis.
Round characteristics: A $152M Series B financing via private placement, co-led by Life Sciences at Goldman Sachs Alternatives and TCGX, with new participation from Omega Funds, EQT Life Sciences, Perceptive Advisors, and Kalehua Capital, alongside existing investors Medicxi and Droia. The round closed oversubscribed.
Use of proceeds: The financing is intended to fund the company’s operations through potential U.S. regulatory approval of engasertib, and concurrently funds initiation of the global Phase 3 HEROIC study.
Board composition: Following the close, Vaderis’ Board comprises Giovanni Mariggi of Medicxi (Chairman), Colin Walsh of Goldman Sachs Alternatives, Giuliano Marostica of TCGX, Francesco Draetta of Omega Funds, Nick Williams of Medicxi, Azmi Nabulsi (CEO), and independent director Rahul Ballal.
Data caveat: The 2020 initial financing amount is reported inconsistently across sources — “over CHF 18 million” per official press materials versus “$19M” per Crunchbase/PitchBook — likely reflecting currency-conversion or aggregation differences. Cumulative funding figures should be treated as directional given these discrepancies.
The investment case for Vaderis rests on four pillars: scarcity value as effectively the sole clinical asset in Phase 3 development for a disease with zero approved therapies, peer-reviewed validation of the underlying science, secured FDA regulatory pathway status, and demonstrated institutional conviction reflected in an oversubscribed financing round.
HHT is the second most common inherited bleeding disorder worldwide, yet remains without an approved treatment. Engasertib, developed specifically for the HHT indication, is understood to be the first molecule of its kind to reach global Phase 3, positioning Vaderis for a potential first-in-disease designation upon approval.
Proof-of-concept and 12-month open-label extension data have cleared external academic scrutiny via publication in the New England Journal of Medicine. The observed dose-dependent efficacy, deepening benefit over time, and favorable tolerability profile provide an evidentiary basis for Phase 3 conviction.
FDA Fast Track Designation, granted in November 2024, provides scope for expedited regulatory review. The appointment of Dr. Hanny Al-Samkari of Harvard Medical School — a recognized HHT authority — as Principal Investigator for the Phase 3 HEROIC study further strengthens execution credibility.
An oversubscribed Series B co-led by Goldman Sachs Alternatives and TCGX, with participation from multiple large specialist life-sciences funds, signals strong institutional confidence. The company’s Medicxi-backed, asset-in-licensing origin — acquiring a validated compound portfolio rather than pursuing de novo discovery — has also supported a capital-efficient development path to date.
Vaderis’ enterprise value is effectively concentrated in a single asset and single indication — a structure that amplifies both upside optionality and downside risk. From a hedge fund analyst’s perspective, the following factors warrant explicit consideration in any investment assessment.
The disclosed pipeline is effectively limited to engasertib in HHT. No meaningfully diversified follow-on pipeline has been publicly disclosed, meaning enterprise value is highly correlated with a single Phase 3 outcome.
The proof-of-concept study enrolled 75 patients; whether the observed effect size replicates in a larger, multi-region Phase 3 population remains unproven. Bleeding-related clinical endpoints are subject to meaningful patient-to-patient variability.
Because no therapy has ever been approved for HHT globally, there is no established regulatory precedent regarding FDA/EMA endpoint acceptance or approval requirements for this indication. Fast Track status does not guarantee eventual approval.
Per Endpoints News reporting, headcount stands at roughly 13 employees. Running a global Phase 3 trial across North America, South America, and Europe at this scale implies significant reliance on CROs and external partners to manage operational load.
Management states that the $152M is expected to fund operations “through potential U.S. regulatory approval.” Rare-disease Phase 3 programs and regulatory submissions typically span multiple years, and the sufficiency of current capital relative to total costs — including any ex-U.S. registration efforts — cannot be independently confirmed from public disclosures.
Co-founder and original CEO Nicholas Benedict has been succeeded by current CEO Azmi Nabulsi, but the timing and rationale behind this transition have not been formally detailed in public materials. Continuity between the founding scientific leadership and the current commercialization-stage leadership warrants ongoing monitoring.