What Vaderis Therapeutics does

Vaderis Therapeutics AG is a privately held, clinical-stage biotechnology company founded in 2019 and headquartered in Basel, Switzerland, with an additional operating office in Lincolnshire, Illinois. The company focuses exclusively on developing therapeutics for rare and orphan diseases characterized by vascular malformations, with its lead and only clinical candidate, VAD044 (also known as engasertib), being developed as the first potential disease-modifying therapy for Hereditary Hemorrhagic Telangiectasia (HHT, also called Osler-Weber-Rendu syndrome) — a rare autosomal dominant disorder for which no approved treatment exists anywhere in the world.

The company's core technology is a portfolio of allosteric AKT serine kinase inhibitors in-licensed from Almac Discovery Ltd (Belfast, UK) in April 2020. Engasertib is an oral, once-daily, selective allosteric inhibitor of AKT1 and AKT2 that targets the underlying molecular pathology of HHT rather than merely managing symptoms. The mechanism normalizes abnormal angiogenesis by restoring physiological endothelial migration (AKT2-driven) and proliferation (AKT1-driven) and corrects defective ALK5-Smad3 signaling, with preclinical data showing complete suppression of arteriovenous malformation (AVM) development at doses that do not induce hyperglycemia — a key safety differentiator versus first-generation AKT inhibitors. The asset has FDA Fast Track designation (November 2024), completed a positive Phase 2 proof-of-concept trial (INSIGHT, 75–80 patients across USA, Canada, and Europe with positive dose-dependent reductions in epistaxis frequency and duration), long-term extension data through 12 months, and results published in The New England Journal of Medicine in December 2025. The company is preparing to initiate the pivotal Phase 3 HEROIC study in the first half of 2026.

Vaderis is pre-revenue with no approved product and no expanded access or commercial pricing in place. Its commercial model is a traditional clinical-stage biotech pathway: regulatory approval followed by launch of a single-asset orphan therapy into a niche specialty distribution channel via HHT specialist centers, with potential future expansion into other rare vascular diseases sharing the same BMP9/BMP10/AKT pathway biology. The company is venture-backed, primarily by Medicxi (founding investor, with partner Giovanni Mariggi as Board Chair) and DROIA Ventures (Managing Partner Luc Dochez on the Board). Operations are run by a leadership team recruited largely from Abbott, Takeda, Amgen, Novartis, and Phathom Pharmaceuticals, providing late-stage development and regulatory expertise that is atypical for a biotech of this size.

Name

Vaderis Therapeutics

Legal name

Vaderis Therapeutics AG

Website

https://vaderis.com

Company type

Private

Founded year

2019

Operating status

Operating

Headcount range

11–50 employees

Short description

Vaderis Therapeutics is a privately held, clinical-stage Swiss biotech developing VAD044 (engasertib), an oral allosteric AKT1/2 inhibitor, as the first potential disease-modifying therapy for Hereditary Hemorrhagic Telangiectasia (HHT), a rare orphan vascular disease with no approved treatment.

Ownership category

akta.pro rank

Product category

Biopharmaceuticals

NAICS

Research and Development in Biotechnology (except Nanobiotechnology) (541714)

SIC

Biological Products, (No Disgnostic Substances) (2836)

akta.pro primary industry

Rare Cardiovascular & Vascular Disorder Therapies (HLAIAIAH)

akta.pro secondary industry

Pulmonary Hypertension & Rare Pulmonary Disease Therapies (HLAIAIAG)

Keywords

• Rare disease therapeutics
• Allosteric AKT inhibitors
• Vascular malformation treatment
• Clinical stage biotechnology
• Orphan drug development

Headquarters

HQ city

Basel

HQ country

Switzerland

HQ region

Europe

Offices2 records

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

Personnel, Technology or R&D, Operations, Marketing or Sales

Revenue model

1. Pharmaceutical commercialization: Pre-revenue clinical-stage biotech. Once VAD044/engasertib receives regulatory approval, the company plans to commercialize the therapy for HHT and potentially other rare vascular diseases. No approved therapies exist globally for HHT, representing a potential first-to-market opportunity.

Go-to-market motion1 record

Distribution channels1 record

Marketing channels4 records

Core offering

Vaderis Therapeutics is developing VAD044 (engasertib), an oral, once-daily, selective allosteric inhibitor of AKT1 and AKT2, as a first-of-class therapy for Hereditary Hemorrhagic Telangiectasia (HHT) and other rare vascular diseases. The drug is being advanced through clinical development (proof-of-concept completed; Phase 3 HEROIC study planned for H1 2026) and targets the underlying molecular dysregulation of vascular malformations rather than symptom management.

Product overview

Vaderis Therapeutics is a clinical-stage biopharmaceutical company developing a single lead product candidate: VAD044 (also known as engasertib), an oral, once-daily allosteric AKT1/2 inhibitor. This first-of-its-kind therapy targets the underlying molecular pathway dysregulation in Hereditary Hemorrhagic Telangiectasia (HHT), aiming to restore vascular balance rather than just manage symptoms. The company is advancing VAD044 through clinical development, with completed proof-of-concept trial and planned Phase 3 HEROIC study.

Differentiator

Problem solved

Functional benefit

Products and services

• VAD044 (Engasertib) Oral, once-daily, selective allosteric inhibitor of AKT1 and AKT2 in development as a first-of-class therapy for Hereditary Hemorrhagic Telangiectasia (HHT) and potentially other rare vascular diseases. The drug normalizes abnormal angiogenesis by restoring physiological endothelial migration (AKT2-driven) and proliferation (AKT1-driven) and corrects defective ALK5-Smad3 signaling. Target users are HHT patients and the specialist centers that treat them.

Quantifiable outcome

• 41% reduction in mean epistaxis duration (40mg dose) vs 24% with placebo
• +4 more outcomes

Segments1 record

Ideal customer profiles2 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature2 records

Partnerships

Four partnerships are on record, tiered core and major.

• Almac Discovery LtdcoreTechnology or Integration · 1 April 2020In April 2020, Vaderis acquired a portfolio of allosteric AKT inhibitors from Almac Discovery Ltd of Belfast, UK. This acquisition provided the foundational intellectual property for VAD044/engasertib, a selective allosteric AKT inhibitor for treating vascular malformations.
• CureHHTcoreStrategic or Co-development PartnerCureHHT is the patient advocacy organization for HHT. Vaderis collaboration with patient and physician organizations including CureHHT has been a cornerstone of successful clinical trial implementation. The company works closely with the HHT community including caregivers and patient associations.
• Massachusetts General HospitalmajorStrategic or Co-development PartnerDr. Hanny Al-Samkari from Massachusetts General Hospital serves as co-lead principal investigator in the VAD044 proof-of-concept trial. MGH is a major clinical trial site and key opinion leader institution in HHT research.
• Netherlands Reference Centre for HHTmajorStrategic or Co-development PartnerDr. Hans-Jurgen Mager from St. Antonius Hospital Utrecht leads the Netherlands Reference Centre for HHT and serves as co-primary investigator. The center is part of the HHT International Foundation.

Scale indicators5 records

Recent moves6 records

Expansion highlights5 records

Direct peers

• Phathom Pharmaceuticals: Late-stage rare-disease biotech founded and led by multiple Vaderis executives (Nabulsi, Harris, Saint-Mezard, Zylberman, Buckley). Both companies share the same playbook: orphan indication focus, experienced repeat operators, and clinical-to-commercial transition — making Phathom the single most directly comparable peer.

Broad incumbents

• BioMarin Pharmaceutical: Established rare-disease commercial-stage biopharma with multiple orphan approvals (e.g., Vimizim, Naglazyme, Voxzogo). Comparable in targeting small genetically defined patient populations with high-priced biologics/small molecules and pursuing label expansions.
• Ultragenyx Pharmaceutical: Commercial-stage rare-disease biotech with a portfolio of approved and clinical-stage orphan assets across metabolic, neurologic, and skeletal disorders. Directly relevant as a comparably sized rare-disease commercial model and a likely strategic acquirer of late-stage orphan assets like Vaderis'.
• Alnylam Pharmaceuticals: Commercial-stage rare-disease biotech built on a novel platform modality (RNAi), with multiple orphan approvals (Onpattro, Givlaari, Oxlumo). Highly comparable in platform-driven rare-disease strategy, although it has reached commercial scale beyond Vaderis' current stage.
• Vertex Pharmaceuticals: Large-cap biopharma with a deep rare-disease franchise (cystic fibrosis, sickle cell) and emerging rare-pain and kidney programs. Relevant as a benchmark for the rare-disease commercial model and a potential strategic acquirer of late-stage orphan assets.
• Alexion (AstraZeneca Rare Disease): Established rare-disease franchise within AstraZeneca with multiple orphan approvals (Soliris, Ultomiris, Strensiq). Comparable in focusing on rare hematologic and complement-mediated disorders, and a likely strategic acquirer of late-stage orphan assets like Vaderis.

Emerging players

• BridgeBio Pharma: Clinical-to-commercial rare-disease biotech with a portfolio of genetic-disease programs (e.g., Acoramidis for ATTR-CM). Comparable in pursuing genetically defined rare cardiovascular and metabolic indications with platform-style optionality and similar late-stage clinical positioning.
• Ionis Pharmaceuticals: Clinical-stage antisense-platform rare-disease biotech with multiple partnered and wholly owned orphan programs (e.g., Spinraza, Tegsedi, Wainua). Comparable in platform-based approach to rare diseases and reliance on partnership economics for commercialization.
• Travere Therapeutics: Mid-cap rare-disease biotech (Filspari approval, late-stage programs in rare kidney and metabolic disease). Closely comparable in size, stage, and orphan-indication commercial trajectory to where Vaderis could be post-approval.
• Disc Medicine: Clinical-stage rare-hematology biotech with multiple programs in rare anemias and hematologic disorders. Highly relevant as a comparable in rare vascular/hematologic indications, late-Phase 2 to Phase 3 positioning, and orphan disease strategy.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat3 records

Key risks6 records

Key highlights7 records

Customer concentration

• LinkedInlinkedin.com/company/vaderis-therapeutics-ag

Revenue estimate

Valuation estimate

Number of profiles

Profiles8 records

Funding overview

Funding rounds2 records

Investors8 records

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M&A

Investments

M&A and investment is available on the Subscription and Enterprise plan.Contact sales →

Frequently asked questions about Vaderis Therapeutics