Opthea (OPT) investor relations material

Strategic transformation and rare disease focus

• Transitioned to a rare disease company leveraging VEGFR-3 biology and OPT-302, shifting focus from ophthalmology to pulmonary and lymphatic diseases, specifically targeting LAM (lymphangioleiomyomatosis), a rare lung disease with high unmet need.

• OPT-302 targets VEGF-C/D signaling to address abnormal lymphatic function in LAM, supported by strong scientific rationale, prior clinical experience, and high-quality clinical, manufacturing, and safety data.

• Adopted a disciplined, capital-efficient operational model leveraging prior investment, significant cost reductions, outsourcing, and Australian R&D tax incentives.

• Engaged leading scientific advisors, rare disease experts, and established a Scientific Advisory Board with global LAM specialists to guide development and clinical strategy.

• Reinstatement on ASX effective 3 June 2026, with an EGM scheduled to consider a name change to Ceryvyn Therapeutics.

Clinical and operational plan

• Implemented a rigorous, stage-gated development plan with three critical decision points, predefined stop criteria, and milestones to ensure scientific and financial discipline.

• Preclinical development of nebulised OPT-302 is on track, with Gate 1 focused on inhalation capability and safety in large animals, and third parties contracted for preclinical and nonclinical work.

• Phase I-B/II-A trial will assess safety, tolerability, pharmacokinetics, target engagement, and early efficacy in LAM patients, using advanced imaging and biomarkers.

• Trial design allows for rapid assessment, with interim analysis at three months and confirmation at six months.

• Recruitment will leverage established global rare disease networks and approximately 70 global LAM centers, focusing initially on Australia and the US.

Financial and corporate overview

• Cash and cash equivalents of AUD 31.2 million as of 31 March 2026, sufficient to fund all three stage gates and the 18-month LAM program, with no debt following settlement of a funding agreement in August 2025.

• Estimated spend for the 18-month program is AUD 13.1 million, leaving substantial capital for future trials.

• Market capitalisation stands at AUD 19.15 million, with 1,367,978,173 shares on issue.

• Accumulated tax losses will be retained and available for future profitability.