Rein Therapeutics (RNTX) Canaccord Genuity 44th Annual Growth Conference & Private Company Showcase 2024 summary

Strategic focus and pipeline overview

• Acquired Lung Therapeutics in October, gaining pulmonary assets LTI-01 and LTI-03, now focusing on orphan pulmonary and fibrotic diseases.

• LTI-03 targets idiopathic pulmonary fibrosis (IPF) with a dual mechanism: anti-fibrotic and regenerative, aiming to restore lung function.

• LTI-01 addresses loculated pleural effusion, a severe pneumonia complication, with prior phase IIb/II data and a larger trial pending funding.

• LTI-03 is in phase IIb for IPF, with safety data from healthy volunteers and phase Ib in patients; top-line high-dose data expected by next month.

• Team includes experienced leadership and scientific expertise, notably CSO Cory Hogaboam, a leading IPF researcher.

LTI-03 mechanism and preclinical data

• LTI-03 is a peptide mimicking caveolin-1, regulating multiple fibrosis pathways, unlike single-target drugs.

• Demonstrated broad anti-fibrotic effects in human IPF lung tissue, comparable to nintedanib but with better tolerability.

• Animal models show LTI-03 reverses established fibrosis, matching saline controls in key markers.

• Unique regenerative effect: preserves and restores type II epithelial cells, confirmed by increased surfactant protein C and ABCA3.

• No significant tolerability issues at efficacious doses; high-dose toxicity linked to peptide aggregation, not immune response.

Clinical development and biomarker results

• Phase Ib study in IPF patients uses 5 and 10 mg doses, with 2-week dosing and bronchoscopy for biomarker analysis.

• Low-dose cohort showed only mild, grade 1 adverse events and strong biomarker trends; 7 of 8 biomarkers moved in the desired direction, 3 reached statistical significance.

• Key biomarkers (collagen 1A1, IL-11, sRAGE) are linked to lung function, the primary endpoint for future trials.

• Placebo group showed biomarker elevation, while LTI-03 group remained flat or decreased, supporting drug activity.

• High-dose cohort data expected by end of the current quarter, with plans to advance to phase II.