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Endeavor BioMedicines to Present New Data From Phase 2a Clinical Trial of ENV-101 at American Thoracic Society 2024 International Conference

Data from Phase 2a trial of novel Hedgehog (Hh) signaling pathway inhibitor in patients with idiopathic pulmonary fibrosis selected for featured late-breaking oral presentation on May 19

SAN DIEGO – May 1, 2024Endeavor BioMedicines (“Endeavor”), a clinical-stage biotechnology company developing medicines with the potential to deliver transformational clinical benefits to patients with life-threatening diseases, announced that data from a completed Phase 2a clinical trial evaluating the company’s lead investigational candidate ENV-101 in patients with idiopathic pulmonary fibrosis (IPF) will be presented for the first time in a late-breaking oral session at the American Thoracic Society 2024 (ATS 2024) International Conference. ATS 2024 will take place May 17-22, 2024, in San Diego.

The Phase 2a, randomized, double-blind, placebo-controlled clinical trial (NCT04968574) evaluated the safety and efficacy of ENV-101 vs. placebo in 41 patients with confirmed IPF who were treated for 12 weeks.

Following are details about the ENV-101 oral presentation at ATS 2024:

  • Title: ENV-101, A Novel Hedgehog Inhibitor, Increases Lung Function, and Reduces Lung Fibrosis in Patients With Idiopathic Pulmonary Fibrosis: Results From a Randomized, Double-blind, Placebo-controlled Phase 2 Trial
  • Presenter: Toby M. Maher, M.D., Ph.D., Professor of Medicine and Director of Interstitial Lung Disease at Keck School of Medicine, University of Southern California, Los Angeles
  • Session: A-18 – Fixing What’s Broken: Novel Therapeutics for Lung Remodeling
  • Date and Time: May 19, 2024, 10:51-11:03 a.m. PT
  • Location: San Diego Convention Center, Room 8 (Upper Level)

“We look forward to sharing the results from the Phase 2a trial of ENV-101 with the pulmonology community for the first time at ATS 2024,” said Paul A. Frohna, M.D., Ph.D., Pharm.D., Chief Medical Officer, Endeavor BioMedicines. “There is a tremendous need for new therapies that have the potential to reverse lung fibrosis and improve lung function in patients with IPF, and these data suggest ENV-101 could be an important new option for patients.”

ENV-101 blocks a cellular wound-healing pathway known as Hedgehog (Hh) that is abnormally activated in fibrotic lung diseases such as IPF and causes the buildup of scar tissue in the lungs. Current standard-of-care therapies do not address the underlying cause of IPF. They slow the decline of lung function, but do not stop or reverse it, and they have tolerability issues that limit their long-term use in most patients.

Based on results from the Phase 2a trial of ENV-101, Endeavor BioMedicines intends to initiate a Phase 2 trial in patients with IPF and in a parallel cohort, patients with progressive pulmonary fibrosis (PPF), in 2024.

About Idiopathic Pulmonary Fibrosis

IPF is a chronic, progressive lung disease that affects more than 100,000 adults in the United States. Although the exact cause of IPF is unknown, various environmental factors can deliver repeated injuries to lung cells that trigger abnormal wound-healing processes and life-threatening lung scarring. IPF is a chronic disease with limited treatment options and a very poor prognosis: the average life expectancy is only three to five years after diagnosis.

About ENV-101

Endeavor BioMedicines’ investigational medicine ENV-101 is a Hedgehog signaling pathway inhibitor. By binding to and inhibiting a key receptor in the Hedgehog pathway, ENV-101 stops the abnormal accumulation of the myofibroblasts that cause fibrosis. This may resolve the excessive wound-healing process seen in IPF and PPF, creating the potential to reverse fibrosis and improve lung volume and function.

About Endeavor BioMedicines

Endeavor BioMedicines is a clinical-stage biotechnology company developing medicines with the potential to deliver transformational clinical benefits to patients with life-threatening diseases. Endeavor’s lead candidate, ENV-101, is an inhibitor of the Hedgehog (Hh) signaling pathway in clinical development for the treatment of idiopathic pulmonary fibrosis (IPF) and progressive pulmonary fibrosis (PPF). Their second candidate, ENV-501, is a HER3 antibody-drug conjugate (ADC) for the treatment of HER3-positive solid tumors. More information is available at and on LinkedIn or X.