Endeavor BioMedicines has reported that its Phase IIb WHISTLE-PF clinical trial assessing taladegib (ENV-101) achieved its primary and several key secondary endpoints in idiopathic pulmonary fibrosis (IPF) patients.
The double-blind, randomised, dose-ranging, placebo-controlled, multi-centre trial evaluated 213 participants with IPF over a 24-week period across 74 medical centres located in 14 countries.
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The programme permitted baseline antifibrotic treatment, which 73% of enrolled patients were receiving.
According to the clinical findings, taladegib produced a statistically significant increase in percent predicted forced vital capacity (ppFVC) relative to placebo at 24 weeks.
A pre-specified analysis restricted to on-treatment participants also confirmed sustained ppFVC improvements from baseline over the 24-week timeframe.
Patients receiving the therapy exhibited gains over placebo in absolute forced vital capacity (mL) as well as quantitative high-resolution computed tomography measures, including total lung capacity, percent quantitative total interstitial lung disease, and percent quantitative lung fibrosis.
The investigation additionally recorded a numerical, non-statistically powered improvement over placebo in the Living with Pulmonary Fibrosis questionnaire.
Endeavor BioMedicines co-founder, CEO and chairman John Hood said: “The unprecedented results from WHISTLE-PF reinforce our confidence in the potential of taladegib to be the first treatment to reduce fibrosis and improve symptoms for IPF patients.
“The totality of these findings, including statistically significant reductions in fibrosis and improvements in lung function, further distinguish taladegib in the IPF treatment landscape and validate our approach to targeting Hedgehog signalling.
“We are deeply grateful to the investigators, study coordinators, and patients worldwide whose participation enabled WHISTLE-PF to enrol ahead of expectations and achieve these important results.”
Taladegib is an inhibitor targeting the Hedgehog signalling pathway to suppress abnormal cellular scarring mechanisms in fibrotic pulmonary conditions.
Throughout the trial, the therapy exhibited a tolerability profile consistent with earlier research, while serious adverse events occurred at similar rates between the active and placebo groups.
The compound holds orphan drug designation from both the European Medicines Agency’s Committee for orphan medicinal products and the US Food and Drug Administration (FDA), as well as PRIority MEdicines status from the European Medicines Agency.
In November 2024, Endeavor BioMedicines dosed the first subject in the randomised Phase IIb WHISTLE-PF trial in Australia, assessing the efficacy and safety of taladegib for treating IPF.
