• Opthea to repurpose OPT-302 to target Lymphangioleiomyomatosis (LAM), a rare lung disease with major unmet need
• OPT-302 mechanism well-suited to LAM biology, targeting VEGF-C/D pathway
• Regulatory incentives and premium pricing potential for orphan drug designation

Opthea has strong IP, an experienced team, and significant cash reserves to execute on its new strategic direction. The company has completed a disciplined strategic review, exploring various options including acquisitions, mergers, and returning capital to shareholders. Ultimately, Opthea has decided to repurpose its OPT-302 asset to target Lymphangioleiomyomatosis (LAM), a rare, chronic lung disease affecting women of reproductive age. LAM meets the criteria for promising repurposing of OPT-302, with clear biology and relevance of the mechanism, a significant market opportunity with limited competition, and high unmet medical need. OPT-302 is a VEGF-C/D 'trap' designed to bind and sequester these growth factors, preventing activation of the VEGFR-3-mediated lymphatic signaling that fuels LAM progression. Opthea believes OPT-302 can complement current mTOR inhibitor therapy by addressing the lymphatic biology of LAM. The company plans a stage-gated development program, starting with preclinical studies and progressing to early human trials to evaluate OPT-302's effects on lung function and lymphatic outcomes in LAM patients. Opthea intends to leverage regulatory incentives for orphan drugs, which can provide market exclusivity, reduced development costs, and premium pricing power. The company will also build strong relationships with the global network of LAM foundations, clinics, and patient communities. Opthea aims to seek reinstatement of its securities on the ASX in the first half of 2026, following the completion of its strategic review and communication of its plans.

Opthea is focused on executing a stage-gated development plan to evaluate the potential of OPT-302 in Lymphangioleiomyomatosis (LAM), a rare lung disease with significant unmet need. The company believes OPT-302's mechanism of action is well-suited to address the underlying biology of LAM and plans to leverage regulatory incentives for orphan drugs to support the program.