Endeavor BioMedicines to Present Phase 2a ENV-101 Data at ATS 2024

Endeavor BioMedicines, a biotechnology company at the clinical-stage, is working on innovative treatments for serious diseases. They recently announced the upcoming presentation of data from their Phase 2a clinical trial of ENV-101, a novel investigational treatment for idiopathic pulmonary fibrosis (IPF). The presentation will occur at the American Thoracic Society 2024 (ATS 2024) International Conference, scheduled for May 17-22, 2024, in San Diego.

The Phase 2a clinical trial, identified by its registration number NCT04968574, was a randomized, double-blind, placebo-controlled study. It assessed the safety and efficacy of ENV-101 against a placebo in 41 patients diagnosed with IPF over a 12-week treatment period. The oral presentation titled "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" will be delivered by Dr. Toby M. Maher, professor and director at the Keck School of Medicine, University of Southern California, at the San Diego Convention Center on May 19, 2024.

Dr. Paul A. Frohna, Chief Medical Officer at Endeavor BioMedicines, expressed enthusiasm about presenting the trial results. He emphasized the critical need for new therapies that can reverse lung fibrosis and enhance lung function in IPF patients. Current treatments only slow the decline in lung function without addressing the root cause or reversing the disease, and their side effects often restrict long-term use. In contrast, ENV-101 targets the Hedgehog (Hh) signaling pathway, which is overactive in fibrotic lung diseases like IPF. The abnormal activation of this pathway leads to scar tissue accumulation in the lungs. ENV-101 inhibits this pathway, potentially stopping the excessive formation of myofibroblasts and the subsequent fibrosis, thereby improving lung function.

The encouraging results from the Phase 2a trial have prompted Endeavor BioMedicines to plan a more extensive Phase 2 trial in 2024. This trial will not only include patients with IPF but also those with progressive pulmonary fibrosis (PPF), aiming to explore further the efficacy and safety of ENV-101.

Idiopathic Pulmonary Fibrosis (IPF) is a severe, chronic lung disease affecting over 100,000 adults in the United States. Its exact cause remains unknown, but environmental factors are believed to repeatedly damage lung cells, initiating abnormal wound-healing processes that result in fatal lung scarring. The prognosis for IPF is generally poor, with an average life expectancy of just three to five years post-diagnosis. Treatment options are limited, highlighting the urgent need for new therapies like ENV-101.

ENV-101 is specifically designed to inhibit the Hedgehog signaling pathway, which is implicated in the development of fibrosis in IPF and PPF. By targeting a crucial receptor in this pathway, ENV-101 aims to halt the pathological buildup of myofibroblasts that leads to lung fibrosis. This mechanism holds the promise of not only arresting the disease's progression but potentially reversing it, thereby restoring lung function and improving patient outcomes.

In addition to ENV-101, Endeavor BioMedicines is also developing ENV-501, a HER3 antibody-drug conjugate (ADC) designed to treat HER3-positive solid tumors, demonstrating the company’s commitment to tackling various life-threatening diseases through innovative biotechnological approaches.