Aileron Therapeutics, Inc. (NASDAQ: ALRN) presented new data at the 22nd International Colloquium on Lung and Airway Fibrosis (ICLAF) highlighting the potential of LTI-03 in treating Idiopathic Pulmonary Fibrosis (IPF). The data includes results from the Phase 1b clinical trial and pre-clinical studies.
The Phase 1b trial (NCT05954988) is a randomized, double-blind, placebo-controlled, multi-center, dose escalation study. It investigates the safety, tolerability, and exploratory biomarker responses of LTI-03 in IPF patients who have not received prior anti-fibrotic treatment for at least two months.
Phase 1b Trial Results
Data from Cohort 1 of the Phase 1b trial, which evaluated low-dose LTI-03 (2.5 mg BID) administered via inhalation, showed positive trends in seven of eight biomarkers associated with IPF progression. Twelve patients received inhaled low-dose LTI-03 over 14 days. Statistically significant data was observed in three biomarkers related to collagen synthesis, inflammation, and fibrogenesis. These findings suggest LTI-03 could potentially improve lung function and reverse the course of IPF.
Pre-clinical Evidence
Pre-clinical data presented at ICLAF further supports LTI-03's therapeutic potential. Ex-vivo precision cut lung slices (PCLS) from IPF patients demonstrated molecular activity indicative of fibrosis over five days in culture. LTI-03 treatment broadly attenuated pro-fibrotic proteins and pathways in these PCLS explants.
Ongoing Clinical Development
Aileron Therapeutics recently completed enrollment in Cohort 2 of the Phase 1b trial, evaluating a higher dose of LTI-03 (5 mg BID) in 12 IPF patients. In the trial, eligible patients (n=24) are randomly assigned (3:1) to receive either inhaled LTI-03 or placebo. Topline data from this cohort is expected in the near term. The primary objective of the trial is to evaluate the safety and tolerability of LTI-03 in patients with IPF after treatment for 14 consecutive days, with measurement of multiple protein biomarkers as exploratory endpoints.
About LTI-03
LTI-03 is a seven amino acid peptide derived from the caveolin scaffolding domain (CSD) of the Cav1 protein. Cav1 plays a critical role in preventing fibrosis by balancing pathways involved in lung repair and cell movement. Decreased Cav1 expression is observed in IPF lung tissues. LTI-03 aims to restore the balance of biological signals in the lung, potentially slowing lung function decline and restoring healthy lung function by protecting epithelial cells.
The Need for New IPF Treatments
IPF is a chronic, progressive lung disease characterized by tissue scarring that impairs lung function. It affects approximately 100,000 people in the United States and is typically fatal within two to five years of diagnosis, highlighting the urgent need for effective therapies.
