Aileron Therapeutics, a biopharmaceutical company focusing on orphan pulmonary and fibrosis indications, presented encouraging pre-clinical and Phase 1b trial results for its investigational drug LTI-03 at the 22nd International Colloquium on Lung and Airway Fibrosis (ICLAF). LTI-03 is being studied as a potential treatment for idiopathic pulmonary fibrosis (IPF), a progressive and fatal lung disease that affects approximately 100,000 people in the U.S.

In the ongoing Phase 1b clinical trial, early data from the first cohort of patients with IPF showed promising results. After 14 days of treatment with low-dose LTI-03 (2.5 mg BID), a positive trend was observed in seven out of eight biomarkers associated with the progression of IPF. Significant reductions were noted in collagen synthesis, inflammation, and fibrogenesis biomarkers, suggesting that LTI-03 may help improve lung function and potentially reverse the course of the disease.

LTI-03 targets profibrotic proteins produced by basal-like cells and fibroblasts, which play a key role in IPF development. The results of these trials indicate that LTI-03 could offer a novel therapeutic approach to managing IPF, a condition known for its poor prognosis and limited treatment options. The company also showcased pre-clinical findings that support LTI-03’s ability to attenuate fibrosis in precision-cut lung slices, further validating its potential effectiveness.

Advancing Clinical Trials and New Data Expected

Aileron recently completed enrollment for Cohort 2 of its Phase 1b trial, where patients are being treated with a higher dose (5 mg BID) of LTI-03. In this double-blind, placebo-controlled, multi-center trial, 24 participants were randomly assigned to receive either LTI-03 or a placebo. The primary goal of the study is to assess the safety and tolerability of LTI-03, while exploratory endpoints include the measurement of protein biomarkers related to IPF. Topline results from this cohort are expected soon.

LTI-03: A Peptide-Based Therapy with a Dual Mechanism

LTI-03 is a synthetic peptide derived from the caveolin scaffolding domain (CSD), a crucial region of the Cav1 protein. Cav1 plays an essential role in regulating lung repair and cell movement by maintaining a balance between pro-fibrotic and anti-fibrotic signals. In IPF patients, Cav1 expression is often reduced, contributing to fibrosis. By restoring this balance, LTI-03 aims to slow disease progression and possibly restore healthy lung function by protecting epithelial cells.

About IPF and Aileron Therapeutics

IPF is a chronic lung condition that leads to scarring of lung tissue, impairing lung function over time. It primarily affects adults aged 65 and older and is often fatal within two to five years of diagnosis. With limited treatment options, there is a significant unmet need for new therapies to manage and treat IPF.

Aileron Therapeutics is at the forefront of developing first-in-class medicines for orphan pulmonary and fibrosis conditions. LTI-03 is currently the company’s lead product candidate, and Aileron is optimistic that ongoing clinical trials will further demonstrate its potential as a breakthrough therapy for IPF.

As the company continues to advance its clinical trials, it remains focused on addressing the unmet medical needs of patients with life-threatening pulmonary diseases, aiming to bring novel treatments to market that could significantly improve patient outcomes.