The treatment showed improved lung function and reversed lung fibrosis in idiopathic pulmonary fibrosis (IPF) patients over 12 weeks.
The results, which also showed that ENV-101 had an acceptable tolerability profile, were presented for the first time at the American Thoracic Society 2024 (ATS 2024) International Conference.
“There is a tremendous need for IPF treatments that do more than slow the inevitable decline of lung function,” said Toby Maher, professor of medicine and director of interstitial lung disease at Keck School of Medicine.
“These preliminary signs of clinical and antifibrotic activity suggest that ENV-101 has the potential to change the trajectory of this otherwise relentless disease.”
IPF is a chronic, progressive lung disease that affects more than 100,000 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.
IPF is a chronic disease with limited treatment options and a very poor prognosis: the average life expectancy is only three to five years after diagnosis.
According to Endeavor, current standard-of-care therapies do not address the underlying cause of IPF. Rather, they slow the decline of lung function, but do not stop or reverse it, and have tolerability issues that limit their long-term use in most patients.
However, 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 causes the continuous pathophysiologic buildup of scar tissue in the lungs.
Based on the phase 2a trial results, the company will now initiate the Phase 2 WHISTLE-PF trial.
This study is expected to include a global phase 2b cohort in individuals with IPF and a parallel phase 2 cohort in individuals with progressive pulmonary fibrosis (PPF).
In addition, Endeavor recently announced the closing of a $132.5 million Series C financing that will support clinical development of ENV-101 in IPF and PPF.
“These trial results suggest ENV-101 could have transformational clinical benefits for individuals with IPF, who are in need of therapies that change treatment expectations - from slowing disease progression to potentially reversing it,” said Paul Frohna, chief medical officer at Endeavor BioMedicines.
“We look forward to further evaluating ENV-101’s potential in the upcoming WHISTLE-PF clinical trial.”