First Patient Dosed in Endeavor BioMedicines’ Phase 2b Trial of ENV-101 for Idiopathic Pulmonary Fibrosis

Endeavor BioMedicines, a biotechnology firm specializing in clinical-stage developments, has announced a significant milestone in its efforts to combat idiopathic pulmonary fibrosis (IPF). The company has commenced dosing patients in the Phase 2b WHISTLE-PF trial for its leading investigational medication, ENV-101 (taladegib). This drug, which operates as a Hedgehog signaling pathway inhibitor, had shown promising results in a Phase 2a trial, suggesting it might become the first treatment to potentially modify the course of IPF.

CEO and Co-founder of Endeavor BioMedicines, Dr. John Hood, emphasized the importance of the WHISTLE-PF trial. He highlighted that the trial's initiation represents a critical step toward developing a transformative treatment for IPF, a disease characterized by a worse prognosis than many cancers. The company's focus is now on rapidly advancing this trial to make the drug available to patients in need as swiftly as possible.

IPF is a debilitating lung condition that affects approximately 150,000 adults in the United States alone. The disease is chronic and progressive, with limited treatment options. The exact cause of IPF remains unknown, but various environmental factors are believed to cause repeated injuries to lung cells, leading to abnormal wound-healing processes and severe lung scarring.

Current treatments for IPF do not address the root cause of the disease. These treatments primarily aim to slow the decline in lung function rather than halt or reverse it. Moreover, they often come with considerable side effects that limit their long-term use. ENV-101 aims to disrupt the aberrant Hedgehog pathway that is implicated in the development of fibrotic lung diseases like IPF, potentially halting or reversing lung scarring.

Data from the Phase 2a trial of ENV-101, which was randomized, double-blind, and placebo-controlled, revealed that patients on the investigational drug experienced significant improvements in lung function and total lung capacity. Additionally, there was a reversal in several key quantitative measures of lung fibrosis within 12 weeks of treatment. Importantly, no serious adverse events related to the treatment were reported, nor were there any significant safety concerns noted in various health parameters of the patients treated with ENV-101.

The Phase 2b WHISTLE-PF trial is a global study that will further evaluate the safety and efficacy of ENV-101 in individuals with IPF. This trial is set to be conducted across 14 countries, including Australia, where the first patient has already been dosed. The trial will assess the impact of different doses of ENV-101 over a 24-week period, focusing on various endpoints such as lung capacity, fibrosis levels measured by high-resolution computed tomography (HRCT), and patient-reported outcomes.

Endeavor BioMedicines is committed to developing innovative treatments that can deliver significant clinical benefits to patients with severe diseases. Besides ENV-101, the company's pipeline includes ENV-501, a HER3 antibody-drug conjugate designed for treating HER3-positive solid tumors.