Rein Therapeutics (RNTX) investor relations material

Strategic focus and pipeline

• Advancing multi-pathway therapies for orphan pulmonary and fibrosis indications, with LTI-03 as a lead candidate showing antifibrotic and regenerative properties and a favorable safety profile to date.

• LTI-03 targets idiopathic pulmonary fibrosis (IPF), while LTI-01 is developed for loculated pleural effusions; LTI-05 addresses cystic fibrosis and other fibrotic indications.

• Phase 2 clinical trial for LTI-03 in IPF is underway, with interim data expected in the second half of 2026.

• LTI-01 development is temporarily delayed to focus resources on LTI-03.

Market opportunity and competitive landscape

• IPF affects about 100,000 patients annually in the US, with a median survival of 3–5 years post-diagnosis.

• Only three drugs are approved for IPF as of 2025; the global IPF market is projected to reach $11.7B.

• LTI-03’s mechanism could address other fibrosis conditions beyond the lung, including heart, kidney, liver, skin, and eyes.

• Competitive landscape includes multi-pathway and single-pathway drugs, with LTI-03 positioned as a novel Caveolin-1 CSD mimic.

Scientific rationale and mechanism of action

• Caveolin-1 regulates multiple fibrosis-related pathways; its loss in fibrotic states disrupts cellular homeostasis in various organs.

• LTI-03 is a seven-amino-acid peptide mimicking the Caveolin-1 CSD, affecting phosphorylation of profibrotic proteins and delivered via dry powder inhaler.

• Demonstrated inhibition of profibrotic proteins and preservation of lung progenitor cells in preclinical and ex vivo studies.