Rein Therapeutics (RNTX) 3rd Annual Guggenheim Healthcare Innovation Conference summary

Pipeline and mechanism

• Focused on pulmonary orphan conditions, with two respiratory programs phase II-ready and idiopathic pulmonary fibrosis (IPF) as the lead focus.

• LTI-03 supplies caveolin scaffolding-domain activity, aiming to restore cellular regulation across fibrosis pathways where caveolin-1 is missing.

• An off-the-shelf dry powder inhaler is designed for local lung coverage with limited systemic uptake; phase I-B patients had sufficient inspiratory capacity.

RENEW phase II trial

• Global RENEW study doses IPF patients for 24 weeks and assesses HRCT and forced vital capacity, with CT scans before and after treatment.

• Enrollment was reported 25% faster than expected; sites are active in the U.S., U.K., Australia, Germany and Poland, with Bulgaria and Romania recently opened.

• Eligibility requires confirmed IPF and sufficient lung capacity for disease progression; any approved background standard of care is allowed, with patients stratified by therapy.

• A blinded interim snapshot is planned before year-end 2026, covering safety, tolerability and lung-function data after 12 weeks for a subset of 20–40 patients; it will not unblind the trial or guide decisions.

• Last patient is expected in spring 2027, with trial data targeted before year-end 2027.

Clinical evidence and tolerability

• In the phase I-B study, deep-lung brushings before and after 14 days of dosing showed movement in targeted protein biomarkers; five were statistically significant for one or the other dose, with dose response in some.

• IL-11, galectin-7 and surfactant protein D were highlighted; surfactant protein D also showed movement.

• Mild cough was the predominant phase I-B adverse event, but was not severe or serious; LTI-03 is dosed BID at 2.5 mg or 5 mg using a high-resistance inhaler.