Opthea’s $37.6M Cash Fuels OPT-302 Push into LAM Treatment

Strategic Pivot to a Rare Disease

Opthea Limited, a biotechnology company listed on the ASX, has unveiled a decisive strategic refocus centered on OPT-302, a drug candidate designed to trap VEGF-C and VEGF-D proteins. These proteins play a key role in the progression of Lymphangioleiomyomatosis (LAM), a rare and debilitating lung disease primarily affecting women of reproductive age. This move marks a significant shift from Opthea’s previous broader drug development efforts, honing in on a niche with high unmet medical need and promising commercial potential.

The company’s leadership, including Executive Chairman Dr Jeremy Levin, highlighted the rationale behind this pivot – OPT-302’s mechanism aligns closely with the biology of LAM, where abnormal lymphatic vessel growth and leakage driven by VEGF-C/D contribute to lung tissue damage and respiratory decline. By sequestering these growth factors, OPT-302 aims to stabilize lung function and slow disease progression, potentially complementing existing mTOR inhibitor therapies that manage but do not cure LAM.

Robust Foundations and Financial Strength

Opthea enters this new chapter with a robust intellectual property portfolio, including patents that could extend protection until 2046, and a well-experienced management team. The company reported a pro-forma cash position of A$37.6 million, bolstered by a recent A$10.8 million R&D tax incentive rebate, providing a significant runway to fund the staged clinical development plan.

The strategic review considered multiple options, including asset sales, mergers, or capital returns, but concluded that repurposing OPT-302 for LAM offered the best balance of feasibility, shareholder value, and return on investment. This approach leverages existing data packages, manufacturing know-how, and safety profiles, reducing development risk and timelines compared to new drug discovery.

Clinical and Regulatory Pathway

Opthea’s development plan is methodical and stage-gated, beginning with preclinical studies in large animals to assess inhaled delivery methods, followed by early human pharmacodynamic and tolerability studies. The company aims to demonstrate meaningful clinical proof-of-concept in LAM patients, focusing on lung function and lymphatic outcomes.

Crucially, Opthea intends to pursue orphan drug designation across key markets including the US, Europe, Japan, and Australia. This status could unlock accelerated regulatory reviews, reduced filing fees, and extended market exclusivity of 7-10 years post-approval. Given the rarity of LAM and the high unmet need, orphan designation also supports premium pricing strategies, with annual treatment costs for orphan drugs often reaching up to $500,000 per patient.

Market Opportunity and Patient Impact

LAM is a rare genetic condition affecting an estimated 3 to 8 women per million worldwide, though recent data suggest underdiagnosis may mean higher prevalence. The disease is characterized by progressive lung cyst formation, respiratory decline, and lymphatic complications, with no current cure. Existing treatments like mTOR inhibitors can stabilize symptoms but do not halt disease progression, leaving a significant unmet clinical need.

Opthea’s focus on LAM taps into a concentrated patient population managed by approximately 70 global specialty clinics, facilitating efficient clinical trials and patient recruitment. The company is also building strong partnerships with LAM foundations and research networks to support development and eventual market access.

ASX Reinstatement and Forward Outlook

Following completion of its strategic review, Opthea plans to seek reinstatement of its securities on the ASX in the first half of 2026. The company aims to rebuild market confidence through transparent communication and disciplined capital management. Success in LAM could pave the way for OPT-302’s application in other VEGF-C/D–linked conditions, potentially expanding the commercial and therapeutic footprint of the drug.

While the path forward is promising, clinical proof of OPT-302’s efficacy and safety in LAM remains to be demonstrated. The company’s staged approach with clear stop criteria reflects a prudent balance of ambition and risk management.