Aileron Therapeutics Announces Positive Data from Cohort 1 of the Phase 1b Clinical Trial of LTI-03 in Idiopathic Pulmonary Fibrosis (IPF)

Low-dose LTI-03 (2.5 mg BID), a Caveolin-1 related peptide, reduced expression of multiple profibrotic proteins in both pathological basal-like cells and fibroblasts and increased the expression of a biomarker indicative of epithelial health, suggesting potential therapeutic effect

Positive trend was observed in seven of eight IPF biomarkers evaluated

Low-dose LTI-03 was well-tolerated, with no safety signal observed

Data from Cohort 2 evaluating high-dose LTI-03 (5 mg BID) is expected in the third quarter of 2024

Company to host conference call on Wednesday, May 1^st at 9:00 am ET

WALTHAM, Mass., May 01, 2024 (GLOBE NEWSWIRE) -- Aileron Therapeutics, Inc. (“Aileron”) (NASDAQ: ALRN), a biopharmaceutical company advancing a novel pipeline of first-in-class medicines to address significant unmet medical needs in orphan pulmonary and fibrosis indications, today 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.

“We find it encouraging that low dose LTI-03 achieved statistical significance in three out of eight biomarkers evaluated in the trial” said Brian Windsor, Ph.D., President and Chief Executive Officer of Aileron. “This, paired with the positive trends observed in several of the other biomarkers, strengthens our belief that LTI-03 has the potential for disease stabilization or even reversal. We look forward to continuing to evaluate LTI-03 in the ongoing Phase 1b study and sharing results from the high-dose cohort later this year.”

Summary of Cohort 1 Analysis

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: