AUSTIN, Texas — Rein Therapeutics (“Rein”) (NASDAQ: RNTX), a biopharmaceutical company advancing a novel pipeline of first-in-class medicines to address significant unmet medical needs in orphan pulmonary and fibrosis indications, announced that it has received orphan drug designation from the European Medicines Agency (EMA) for LTI-03, its lead drug candidate aimed at preserving lung function in patients with idiopathic pulmonary fibrosis (IPF).
The designation follows a positive opinion from the EMA’s Committee for Orphan Medicinal Products (COMP) and subsequent adoption by the European Commission, recognizing both the seriousness of IPF and the need for new treatment options for patients living with this devastating disease.
IPF is a rare, progressive lung disease characterized by irreversible scarring of lung tissue, that leads to declining lung function and, ultimately, respiratory failure. Despite existing treatments, patients continue to face poor outcomes and limited therapeutic options.
Orphan drug designation in the European Union provides important regulatory incentives, including reduced development fees, potential market exclusivity following approval, and enhanced development efficiency, which may help streamline clinical development and regulatory interactions.
The EMA’s final decision was supported by preclinical data demonstrating improved survival and lung function, reinforcing the scientific rationale behind Rein’s approach to targeting fibrosis. The EMA also agreed with Rein’s justification that LTI-03 could be of significant benefit to those affected by the condition as compared to authorized products, which may constitute a clinically relevant advantage.
Brian Windsor, Ph.D., Chief Executive Officer of Rein Therapeutics, commented, “This designation represents an important regulatory milestone for Rein and a meaningful step forward for patients living with IPF. Receiving orphan drug designation in Europe provides external validation of our strategy that can do more than slow disease progression. We believe LTI-03 has the potential to address this unmet need.”
LTI-03 has been entered into the Community Register of Orphan Medicinal Products under designation number EU/3/25/3188.
About Rein Therapeutics
Rein Therapeutics is a clinical-stage biopharmaceutical company advancing a novel pipeline of first-in-class therapies to address significant unmet medical needs in orphan pulmonary and fibrosis indications. Rein’s lead product candidate, LTI-03, is a novel, synthetic peptide with a dual mechanism targeting alveolar epithelial cell survival as well as inhibition of profibrotic signaling. LTI-03 has received Orphan Drug Designation in the U.S. Rein’s second product candidate, LTI-01, is a proenzyme that has completed Phase 1b and Phase 2a clinical trials for the treatment of loculated pleural effusions. LTI-01 has received Orphan Drug Designation in the U.S. and E.U. and Fast Track Designation in the U.S. For more information visit https://www.reintx.com/ or follow the company on X.
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