The trial results showed that ENV-101 treatment reversed key fibrosis markers over the trial period. Credit: Chinnapong / Shutterstock.com.

Endeavor BioMedicines has revealed results from the Phase IIa clinical trial where ENV-101 demonstrated to improve lung function in idiopathic pulmonary fibrosis (IPF) patients.

ENV-101 is engineered to inhibit the Hedgehog (Hh) pathway, which is overactive in fibrotic lung diseases and leads to the progressive accumulation of scar tissue.

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The double-blind, randomised, placebo-controlled study assessed the safety and efficacy of ENV-101 compared to a placebo in 41 IPF patients.

Conducted across 16 sites in five countries, participants were assigned to receive either a 200mg dose of ENV-101 or a placebo daily for 12 weeks.

In September last year, the company concluded subject enrolment in the Phase IIa trial of ENV-101 (taladegib) to treat IPF.

The trial’s primary goal was to evaluate the safety of ENV-101.

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Measuring changes in lung function from the trial’s start to week 12 alongside patient-reported outcomes from the UCSD shortness-of-breath questionnaire were the trial’s secondary endpoints.

The results showed that ENV-101 led to significant improvements in lung function and reversed key fibrosis markers over the trial period.

Patients treated with the therapy exhibited a statistically significant mean improvement in per cent predicted FVC (ppFVC) of 1.9% from baseline, compared to a mean decline of 1.3% in the placebo group.

Additionally, ENV-101 recipients saw a significant increase in total lung capacity (TLC) by a mean increase of 200mL at 12 weeks, against a mean decrease of 56mL for those on placebo.

In the ENV-101 group, 80% of patients experienced a TLC increase while 70% of placebo recipients saw a decrease.

The treatment also significantly reduced the absolute percent of quantitative interstitial lung disease (QILD) by 9.4% from baseline, compared to a 1.1% increase in the placebo group (P<0.05).

ENV-101 was found to be well-tolerated in the trial without any serious adverse events, grade 3 or 4 adverse events, or significant safety concerns.

Dysgeusia, alopecia and muscle spasms were reported to be the most frequently seen mild to moderate adverse events linked to ENV-101.

Endeavor BioMedicines chief medical officer Paul Frohna said: “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. “We look forward to further evaluating ENV-101’s potential in the upcoming WHISTLE-PF clinical trial.”