– ENV-101, a novel investigational Hedgehog signaling pathway inhibitor, has demonstrated clinical potential to improve lung function and total lung capacity, and reverse multiple key quantitative measures of lung fibrosis
SAN DIEGO – November 20, 2024 – Endeavor BioMedicines, a clinical-stage biotechnology company developing medicines with the potential to deliver transformational clinical benefits to patients with life-threatening diseases, announced that the first patient has been dosed in the Phase 2b WHISTLE-PF trial evaluating the safety and efficacy of ENV-101 (taladegib), the company’s lead investigational medicine, in individuals with idiopathic pulmonary fibrosis (IPF). ENV-101 is a novel Hedgehog signaling pathway inhibitor that demonstrated potential in a Phase 2a trial to be the first disease-modifying treatment for IPF.
“Initiating the WHISTLE-PF trial is an important milestone for our company as we continue to develop ENV-101 for the treatment of IPF, a relentless disease that has a worse prognosis than most cancers,” said John Hood, Ph.D., Co-founder, CEO and Chairman, Endeavor BioMedicines. “Following the positive results from our Phase 2a trial of ENV-101, Endeavor BioMedicines is now intensely focused on efficiently advancing the WHISTLE-PF trial to get this potentially transformative drug to patients as quickly as possible.”
IPF is a chronic, progressive lung disease with limited treatment options that affects approximately 150,000 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.
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. ENV-101 is designed to block a cellular wound-healing pathway known as Hedgehog (Hh) that is abnormally activated in fibrotic lung diseases such as IPF and drives the continuous pathophysiologic buildup of scar tissue in the lungs.
Results from the randomized, double-blind, placebo-controlled Phase 2a trial of ENV-101 showed that patients who received the investigational therapy experienced statistically significant improvements in lung function and total lung capacity, and reversal of multiple key quantitative measures of lung fibrosis through 12 weeks of treatment. There were no treatment-related serious adverse events, treatment-related grade 3 or 4 adverse events, or clinically meaningful safety findings on laboratory analyses, vital signs, electrocardiograms, or physical examination reported for ENV-101-treated patients.
The Phase 2b WHISTLE-PF (Wound-remodeling Hedgehog-Inhibitor ILD Study Testing Lung Function Endpoints-PF) clinical trial is a global, randomized, placebo-controlled study evaluating the therapeutic potential of ENV-101 in individuals with IPF (NCT06422884). Endeavor BioMedicines intends to conduct the trial in 14 countries globally, including Australia, where the first patient was dosed.
The WHISTLE-PF trial will evaluate the efficacy of a range of ENV-101 doses through 24 weeks of treatment, characterize the investigational compound’s safety, assess its effect on patient reported outcomes and its effects on lung capacity and lung fibrosis as measured by chest high-resolution computed tomography (HRCT).
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 (taladegib), is an inhibitor of the Hedgehog signaling pathway in development for fibrotic lung diseases, including idiopathic pulmonary fibrosis (IPF). The company’s second candidate, ENV-501, is a HER3 antibody-drug conjugate (ADC) in development for the treatment of HER3-positive solid tumors. More information is available at www.endeavorbiomedicines.com and on LinkedIn or X.