Developing a potentially best-in-class Hh inhibitor for pulmonary fibrosis.

Hedgehog Pathway Inhibitor

VEN-001 is an inhibitor of the hedgehog pathway initially developed by Takeda and known as Tak-441.

Preclinical evidence of VEN-001 in lung fibrosis

Several preclinical studies demonstrate how treatment with VEN-001 reduces lung fibrosis in Bleomycin induced models.

Safety and Efficacy

VEN-001 has been administered to 60 patients in two clinical trials, demonstrating a good safety profile as well as efficacy.

About VEN-001

A Potential Best-in-Class Hedgehog Inhibitor

At Ventalis Pharma, we are focused on leveraging our expertise to target Pulmonary Fibrosis. Our innovative approach and research drive our pursuit of impactful therapies for this unmet clinical need.

PK Profile as Differentiator

VEN-001 PK profile is linear and dose proportional with rapid absorption and fast elimination providing us with more flexible treatment regimens that reduce toxicity.

ABOUT US

Ventalis Pharma is a clinical-stage biopharmaceutical company developing the potentially best-in-class Hedgehog Inhibitor VEN-001 for pulmonary fibrosis.

Our team comprises highly experienced senior executives with decades of industry leadership in drug development, with a focused and proven track record in pulmonary diseases. Together, we bring deep scientific, clinical, and regulatory expertise in lung disease therapeutics, enabling informed decision-making, efficient execution, and risk-aware development.

About Pulmonary Fibrosis

Pulmonary Fibrosis is a progressive, chronic fibrosing disease. With the only curative option being a lung transplant which is limited to a low number of patients.

1. An Unmet Clinical Need

Approved therapies slow down the disease progression but don't reduce the fibrosis or increase lung capacity.

Patients diagnosed with IPF have a median survival of 3 to 5 years.

2. An Orphan Disease

In the United States, approximately 50,000 cases are newly diagnosed and 40,000 patients die each year.

The prevalence is estimated at 2.4 cases per 10,000 people and the incidence is 0.75 new cases per 10,000 people.

Step 1:

Chronic Lung Injury

Step 2:

Activation of Hedgehog Pathway

Fibroblast activation and myofibroblast differentiation:

Produce excessive ECM proteins like collagen. This contributes to the fibrotic scar formation that characterizes pulmonary fibrosis.

Fibroblast proliferation:

Hedgehog signaling enhances fibroblast proliferation, which exacerbates fibrosis by increasing the number of fibroblasts in the lung.

Inflammation and epithelial-to-mesenchymal transition (EMT):

They lose their epithelial characteristics and gain mesenchymal properties, further fueling fibrosis.

The Role of the Hedgehog Pathway in Fibrosis

In the context of pulmonary fibrosis, the Hedgehog pathway has been implicated in the pathogenesis and progression of the disease, influencing key aspects such as fibroblast activation, myofibroblast differentiation, and extracellular matrix (ECM) deposition.

MEET THE TEAM

Management Team

Serial entrepreneur
Founder of Nelum Pharma, PH Clinical Research, Personalized Oncology Services, PX Source and Science Tools

Evelio Perea

CEO and Chairman
of the Board

20 years of experience in global clinical operations, spanning both large pharmaceutical companies and emerging biotech.

Daniele Tompkins

Director Clinical Execution

Over 20 years of experience leading business development, clinical operations, and large-scale program execution across global organizations. PPD (Thermo Fisher Scientific) and Syneos Health.