Opthea (ASX:OPT) has announced its reinstatement to the ASX and unveiled a sharply refocused strategy centred on advancing OPT-302 for lymphangioleiomyomatosis, or LAM, a rare pulmonary disease with significant unmet need.
The company said the decision follows a comprehensive strategic review and will lean on its existing development, manufacturing, and clinical infrastructure to pursue the new direction.
In 2025, the company announced negative results from its pivotal Phase 3 trials of OPT-302 in combination with Bayer's EYLEA and Novartis' LUCENTIS. These trials did not meet their primary endpoints, leading to their discontinuation and significant financial and operational repercussions for the company.
The company's share price fell significantly following its reinstatement to the ASX.
Executive Chair Dr Jeremy Levin framed the relaunch as a focused effort to evaluate a mechanism-driven therapeutic opportunity in LAM and to leverage the company’s assets.
He said, “Opthea is relaunching with a focused strategy centred on OPT-302 and a clear objective: to evaluate a differentiated, mechanism-driven therapeutic opportunity in LAM while leveraging the Company’s substantial existing development, manufacturing and clinical infrastructure.”
He continued, “We believe this approach provides a highly capital-efficient pathway into an area of significant unmet medical need. LAM is a debilitating rare disease with limited treatment innovation and no approved therapies directly targeting aberrant VEGF-C and VEGF-D signalling, biological pathways strongly associated with disease progression and directly targetable by OPT-302.”
Dr Levin added, “With important translational work already underway alongside leading scientific collaborators, and a disciplined stage-gated development plan funded from existing cash reserves, the Company believes it is positioned to pursue meaningful long-term shareholder value creation.”
OPT-302 is designed to inhibit VEGF-C and VEGF-D to dampen lymphatic signalling that fuels LAM, thereby stabilising lung function and slowing disease progression. The company emphasised that the only currently approved treatment for LAM is an immunosuppressant that does not directly target VEGF biology, can produce systemic side effects, and is often discontinued by patients.
Opthea confirmed it held $31.2 million in cash and cash equivalents as at March 31 2026, and that the Board believes these funds are sufficient to support the three-stage LAM program.