Aileron Therapeutics(ALRN) Prnewswire·2024-09-23 11:30Core Insights - Aileron Therapeutics has completed enrollment in Cohort 2 of its Phase 1b clinical trial for LTI-03, targeting idiopathic pulmonary fibrosis (IPF) patients [1][2] - The trial aims to evaluate the safety and tolerability of high-dose LTI-03 (5 mg BID) after positive trends were observed in Cohort 1 with low-dose LTI-03 (2.5 mg BID) [1][2] - The company expects to report topline data from Cohort 2 in the near term [2] Company Overview - Aileron Therapeutics is focused on developing first-in-class medicines for unmet medical needs in orphan pulmonary and fibrosis indications [6] - The lead product candidate, LTI-03, is a synthetic peptide designed to target alveolar epithelial cell survival and inhibit profibrotic signaling [6][7] - Aileron's second product candidate, LTI-01, has completed Phase 1b and Phase 2a trials for loculated pleural effusions and has received Orphan Drug Designation in the US and EU [7] Clinical Trial Details - The Phase 1b trial is a randomized, double-blind, placebo-controlled study involving 24 patients, with a 3:1 randomization to receive either LTI-03 or placebo [2][3] - The primary objective is to assess safety and tolerability over 14 days, with exploratory endpoints measuring multiple protein biomarkers [3] Disease Context - IPF is a chronic lung disease characterized by progressive tissue scarring, affecting approximately 100,000 people in the United States, typically presenting in adults aged 65 or older [4] - The disease is usually fatal within two to five years after diagnosis [4] Mechanism of Action - LTI-03 is derived from the caveolin scaffolding domain (CSD) of the Cav1 protein, which plays a critical role in preventing fibrosis [5] - Cav1 expression is decreased in IPF lung tissues, and restoring its balance may slow lung function decline and protect healthy epithelial cells [5]