Aileron reports results from Cohort 2 of its Phase 1b clinical trial of LTI-03

Aileron Therapeutics (ALRN) announced positive topline data from Cohort 2 of its Phase 1b clinical trial evaluating the safety and tolerability of inhaled LTI-03 in patients diagnosed with idiopathic pulmonary fibrosis, or IPF. LTI-03 is a novel, Caveolin-1-related peptide that modulates both pro-fibrotic activity and sustain critical alveolar epithelial cells. Following inhaled administration of high dose LTI-03, a positive trend was observed in seven out of eight biomarkers, with evidence of reduced expression among profibrotic proteins produced by basal-like cells and fibroblasts that contribute to the progression of IPF, including data from four biomarkers that were statistically significant in the combined data set of Cohort 1 and Cohort 2, and data from five biomarkers that showed dose dependence relative to the data from those biomarkers in Cohort 1. Overall, the collective findings from this Phase 1b clinical trial provide the Company with strong confidence that LTI-03 has the potential to improve lung function and reverse the course of IPF. Twelve patients were enrolled in Cohort 2 of the ongoing Phase 1b clinical trial, three in the placebo arm and nine in the active arm. Patients had a bronchoscopy at baseline, received a high dose of LTI-03 twice a day for 14 days, followed by a bronchoscopy on day 14 and seven days of post-treatment follow-up. Cohort 2 findings include: educed expression of multiple profibrotic proteins active in both pathologic basal-like cells and fibroblasts, with four biomarkers showing statistically significant decreases in the combined data set supporting the potential of LTI-03 to reduce fibrosis, inflammation and associated functional changes in the lung. Dose dependent trends were observed in five biomarkers, including COL1A1, CXCL7, TSLP, GAL-7, and Surfactant protein D (SPD) which provide evidence of active LTI-03 pharmacodynamics. SPD, an indicator of epithelial cell health that is significantly linked to decline in lung function, decreased by 5% in Cohort 2 at 14 days of treatment, while current standard of care for IPF reduced SPD by 4% at 12-weeks in precedent trials. LTI-03 did not induce inflammation in peripheral blood mononuclear cells (PBMCs) in either Cohort, measured by pAKT, a safety marker for inflammation in this trial. LTI-03 was generally well-tolerated, and there were no drug-related adverse events that resulted in a discontinuation of the trial.