Aileron Therapeutics is focused on developing innovative therapies for orphan pulmonary and fibrosis conditions, with a particular emphasis on addressing significant unmet medical needs. The company is advancing two key compounds: LTI-03 and LTI-01. LTI-03 is designed to treat idiopathic pulmonary fibrosis (IPF), a chronic lung disease characterized by progressive tissue scarring. This compound operates through a novel mechanism that targets healthy epithelial cells and profibrotic proteins, promoting lung epithelium protection while inhibiting fibroblast growth. Currently, LTI-03 is undergoing evaluation in a Phase 1b clinical trial for patients recently diagnosed with IPF.

LTI-01 represents a first-in-class treatment for loculated pleural effusion (LPE), a condition where fluid accumulates in the pleural cavity due to scar tissue formation. This proenzyme is engineered to activate slowly within the pleural space, effectively breaking down scar tissue and facilitating fluid drainage. Aileron believes that this unique mechanism enhances efficacy and dosing convenience while minimizing the risk of significant bleeding side effects associated with traditional fibrinolytics. LTI-01 has already been evaluated in Phase 1b and Phase 2 clinical trials and has received Orphan Drug Designation in both the US and EU for treating empyemas, along with Fast Track Designation in the US.

United States

Following the acquisition of Lung Therapeutics in October 2023, Aileron is shifting its disease focus to advancing a pipeline of first-in-class medicines to address significant unmet medical needs in orphan pulmonary and fibrosis diseases, including LTI-03 for idiopathic pulmonary fibrosis (IPF) and LTI-01 for loculated pleural effusion (LPE).

Lung Diseases

Aileron Therapeutics 

Idiopathic pulmonary fibrosis

LTI-03 is a seven amino acid peptide, the sequence of which is derived from the caveolin scaffolding domain (CSD), an important binding region of the Cav1 protein. Cav1 normally serves a critical function in the prevention of fibrosis by maintaining a balance between pathways that both initiate and arrest lung repair and cell movement. Through the CSD, caveolin interacts with a large number of signaling molecules, all of which possess a caveolin binding domain region. Cav1 expression is decreased in IPF lung tissues and has been demonstrated to decrease during the fibrotic phase of bleomycin, or BLM, lung injury in mice. Restoring the balance of important biological signals in the lung may not only slow lung function decline but could also restore healthy lung function through the protection of healthy epithelial cells.

LTI-03

Loculated pleural effusions

LTI-01 a PAI-1 resistant plasmin activated proenzyme for loculated pleural effusions

LTI-01

Cancer

Non-small cell lung carcinoma

ALRN-6924, our first-in-class MDM2/MDMX dual inhibitor activating p53, &nbsp;is the only reported chemoprotective agent in clinical development to &nbsp;employ a biomarker strategy, in which we exclusively focus on treating &nbsp;patients with p53-mutated cancers. Our targeted strategy is designed to &nbsp;selectively protect multiple healthy cell types throughout the body from &nbsp;chemotherapy while ensuring we do not protect cancer cells. As a &nbsp;result, healthy cells are spared from chemotherapeutic destruction while &nbsp;chemotherapy continues to kill cancer cells. By reducing or eliminating &nbsp;multiple chemotherapy-induced side effects, ALRN-6924 may improve &nbsp;patients’ quality of life and help them better tolerate chemotherapy. &nbsp;Enhanced tolerability may result in fewer dose reductions or delays of &nbsp;chemotherapy and the potential for improved efficacy.
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Aileron Therapeutics

Company Details