Aileron Therapeutics, Inc. announced positive data from Cohort 1 of the ongoing Phase 1b clinical trial evaluating the safety and tolerability of inhaled LTI-03 in patients diagnosed with idiopathic pulmonary fibrosis (IPF). LTI-03 is a novel, Caveolin-1-related peptide that addresses both inhibition of pro-fibrotic signaling and survival of critical epithelial cells. Following inhaled administration of low dose LTI-03 (2.5 mg BID), 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 several biomarkers that were statistically significant, reinforcing the potential of LTI-03 to improve lung function and reverse the course of IPF.

Twelve patients were enrolled in Cohort 1 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 low dose of LTI-03 (2.5mg BID) twice a day for 14 days, followed by a bronchoscopy on day 14 and seven days of follow-up. Cohort 1 findings include: Reduced expression of multiple profibrotic proteins in both pathologic basal-like cells and fibroblasts, with statistically significant decreases observed in GAL-7, TSLP and Col-1a1 biomarkers, supporting the potential of LTI-03 to reduce fibrosis, inflammation and associated changes in the lung.

Stimulated production of solRAGE, a factor indicative of type I epithelial cell health that is a critically important aspect of IPF and has gone largely unaddressed. LTI-03 did not induce inflammation in peripheral blood mononuclear cells (PBMCs). Results show LTI-03 to be generally well-tolerated with no serious adverse events (SAEs) reported.

The Phase 1b study is ongoing, with topline results from the high-dose cohort expected in the third quarter of 2024.