Pharma Focus America

Phase 2a Clinical Trial Enrollment for ENV-101 (Taladegib) in the Treatment of Idiopathic Pulmonary Fibrosis (IPF) Successfully Concluded

Thursday, September 07, 2023

Endeavor BioMedicines, a clinical-stage company with a focus on addressing fibrosis and oncology, has announced the successful completion of patient enrollment in its Phase 2a clinical trial for assessing ENV-101 (taladegib) in the treatment of idiopathic pulmonary fibrosis (IPF). ENV-101 is a small-molecule inhibitor designed to target the Hedgehog (Hh) signaling pathway, a pivotal factor in the development of IPF.

John Hood, Ph.D., Co-Founder, CEO, and Chairman of Endeavor BioMedicines, expressed his optimism, stating, "The achievement of full enrollment in the ENV-101 IPF study brings us closer to delivering a critically needed therapy that has the potential to address the fundamental mechanisms of the disease and potentially halt the progression of fibrosis. This enrollment milestone represents a significant accomplishment, and we are looking forward to reporting top-line results in Q1 of 2024. I extend my gratitude to the participating patients and clinical trial sites for their dedicated efforts in advancing the study to this stage."

The Phase 2a clinical trial is designed as a randomized, placebo-controlled, multi-center study aimed at assessing the safety and effectiveness of ENV-101 as a standalone treatment for individuals with mild to moderate IPF. The trial encompasses multiple regions, including the Asia-Pacific area, Canada, and Mexico, and includes a total of 41 participants. Primary trial endpoints include the evaluation of changes from baseline in the frequency and severity of adverse events, as well as vital sign measurements. Secondary endpoints focus on assessing changes in forced vital capacity (FVC) and other indicators of lung function.

ENV-101 was originally developed to target Hedgehog-driven cancers and has undergone evaluation in around 200 subjects, demonstrating outstanding safety and effective target inhibition in human trials. Unlike its typical inactivity in adults, the Hedgehog pathway is upregulated in Idiopathic Pulmonary Fibrosis (IPF) as part of the tissue remodeling process. This upregulation triggers the emergence of myofibroblasts through a process called epithelial-mesenchymal transition.

Myofibroblasts are the primary contributors to the deposition of fibrotic matrix and lung tissue contraction, leading to the characteristic inelastic, contracting, and poorly perfused lungs responsible for IPF patients' suffering and mortality. By selectively inhibiting this pathway within lung tissue, ENV-101 induces apoptosis in myofibroblasts, effectively eliminating the key cellular driver of IPF progression. This targeted approach shows significant promise in addressing the root causes of IPF and potentially improving the outlook and quality of life for individuals affected by the disease.

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