Aileron Therapeutics Completes Enrollment for Phase 1b LTI-03 IPF Trial

Aileron Therapeutics, Inc., a biopharmaceutical company focused on developing innovative treatments for pulmonary and fibrosis conditions, has announced the completion of patient enrollment in Cohort 2 of its ongoing Phase 1b clinical trial for LTI-03, targeting idiopathic pulmonary fibrosis (IPF). The enrollment achieved a milestone with 24 patients by mid-September. Eligible patients were randomly assigned in a 3:1 ratio to receive either the inhaled drug or a placebo.

The primary goal of this trial is to assess the safety and tolerability of LTI-03 in IPF patients after a 14-day treatment period. The study also measures multiple protein biomarkers as exploratory endpoints. Brian Windsor, Ph.D., President and CEO of Aileron, expressed his satisfaction with the enrollment progress and the forthcoming evaluations. With positive trends previously observed in the low-dose cohort, the company is eager to evaluate the high-dose administration of 5 mg twice daily.

In May 2024, Aileron reported promising results from Cohort 1 of the trial, which evaluated a low dose of 2.5 mg twice daily. Data from this cohort indicated positive trends in seven out of eight biomarkers and significant reductions in profibrotic proteins. This suggests that LTI-03 has the potential to improve lung function and counteract the progression of IPF.

The Phase 1b clinical trial employs a randomized, double-blind, placebo-controlled, multi-center, dose-escalation design. Participants are patients recently diagnosed with IPF who have not received anti-fibrotic treatments for at least two months. The primary objective is to assess the safety and tolerability of LTI-03, with the measurement of several protein biomarkers serving as exploratory endpoints.

IPF is a chronic lung disease characterized by progressive scarring of lung tissue, which impedes proper lung function. Affecting approximately 100,000 people in the United States, the disease typically manifests in adults aged 65 and older and has a high mortality rate within two to five years post-diagnosis.

LTI-03, derived from a seven amino acid peptide sequence, plays a significant role in maintaining lung function. The peptide is part of the caveolin scaffolding domain (CSD), an integral binding region of the Cav1 protein. Cav1 is crucial in preventing fibrosis by balancing pathways that initiate and halt lung repair and cell movement. Reduced expression of Cav1 is observed in IPF lung tissues and during the fibrotic phase of certain lung injuries. Restoring this balance could not only slow the decline in lung function but also potentially restore healthy lung function by protecting epithelial cells.

Aileron Therapeutics is committed to advancing first-in-class medicines for orphan pulmonary and fibrosis conditions. Their lead product, LTI-03, is being evaluated for its dual mechanism targeting alveolar epithelial cell survival and inhibition of profibrotic signaling. Additionally, Aileron has another product candidate, LTI-01, which has completed Phase 1b and Phase 2a clinical trials for treating loculated pleural effusions and has received Orphan Drug Designation in the US and EU, as well as Fast Track Designation in the US.

In summary, the completion of enrollment in Cohort 2 marks a pivotal moment in Aileron's clinical trial for LTI-03. With promising data from the first cohort, the high-dose evaluations hold significant potential for advancing the treatment of IPF. The company's commitment to addressing unmet medical needs in pulmonary and fibrosis conditions continues to drive its innovative research and development efforts.