ENV-101 has potential to be the best-in-class treatment for idiopathic pulmonary fibrosis (IPF)
Endeavor expects to initiate Phase 2b study of ENV-101 for the treatment of IPF and progressive fibrosing-interstitial lung disease (PF-ILD) in 2024
SAN DIEGO – January 24, 2024 – Endeavor BioMedicines, a clinical-stage biotechnology company developing medicines with the potential to deliver transformational clinical benefits to patients with life-threatening diseases, today announced successful completion of its Phase 2a clinical trial evaluating the company’s lead investigational compound, ENV-101, in patients with idiopathic pulmonary fibrosis (IPF). ENV-101 is an oral small molecule inhibitor of the Hedgehog (Hh) signaling pathway that offers a novel approach to treating the underlying pathologic mechanisms that cause fibrotic lung diseases.
“Preliminary results from our Phase 2a trial reinforce our belief that ENV-101 has the potential to be the first ever disease-modifying therapy for IPF,” said John Hood, Ph.D., co-founder, CEO and chairman, Endeavor BioMedicines. “We are hopeful that the therapeutic capability of ENV-101 will offer treatment outcomes that go beyond slowing disease progression for patients with lung fibrosis.”
The completed Phase 2a, randomized, double-blind, placebo-controlled clinical trial evaluated the safety and efficacy of ENV-101 vs. placebo in 41 patients with confirmed IPF who were treated for three months.
“We plan to present the full data from this study at an upcoming medical conference,” said Paul Frohna, M.D., Ph.D., chief medical officer, Endeavor BioMedicines. “Additionally, we look forward to expanding ENV-101 clinical development activity by initiating a randomized, double-blind, dose-ranging Phase 2b trial in patients with IPF and in a parallel cohort of patients with progressive fibrosing-interstitial lung disease in 2024 to evaluate its broader therapeutic potential.”
ENV-101 binds to and inhibits a key receptor in the Hh cellular pathway, blocking Hh signaling and inhibiting the pathway. This resolves the excessive wound healing process seen in IPF and progressive fibrosing-interstitial lung disease (PF-ILD), potentially stopping or reversing fibrosis and lung tissue remodeling and, as a result, potentially reversing the disease trajectory.
About Idiopathic Pulmonary Fibrosis
Idiopathic pulmonary fibrosis (IPF) is a chronic, progressive disease that causes lung tissue to become thickened, stiff and scarred, eventually reducing the lungs’ ability to transfer oxygen into the bloodstream. There are more than 100,000 people affected by IPF in the U.S. alone, and average life expectancy is only three to five years after diagnosis. The limited treatment options currently available for IPF don’t address the underlying cause of the disease and only slow the decline of lung function.
About ENV-101
ENV-101 is an oral small molecule inhibitor of the Hedgehog (Hh) signaling pathway that offers an entirely new mechanism of action for treating IPF. The Hh pathway is rarely active in adults except in wound healing and scar formation. Aberrant activation of the Hh pathway through repeated environmental injury to specific lung cells causes excessive fibrotic matrix to accumulate, forming scar tissue and resulting in an inelastic, contracted lung and loss of lung 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 fibrosing-interstitial lung disease (PF-ILD). Their second candidate, ENV-501, is a HER3-antibody drug conjugate (ADC) for the treatment of HER3-positive solid tumors. Endeavor has assembled a highly credentialed leadership team with deep experience identifying promising drug candidates and developing them into leading medicines. More information is available at www.endeavorbiomedicines.com and on LinkedIn or X.