Opthea Limited: Pioneering Breakthrough Therapies for Retinal Diseases

About Opthea Limited

Opthea Limited (ASX: OPT) is a leading biopharmaceutical company pioneering innovative therapies for retinal diseases such as wet age-related macular degeneration (wet AMD) and diabetic macular oedema. The company’s flagship product, sozinibercept, is in Phase 3 clinical trials (COAST and ShORe) to enhance standard anti-VEGF-A therapies, aiming to deliver superior vision outcomes. With a mission to improve and protect vision globally, Opthea is driven by bold therapeutic innovation and a commitment to reducing the impact of retinal diseases. Backed by a world-class ophthalmology team, Opthea aspires to transform lives and advance retinal healthcare.

Advancements in Sozinibercept for Diabetic Macular Oedema

Opthea Limited continues to establish itself as a leader in biopharmaceutical innovation with the publication of its Phase 1b trial results for sozinibercept combination therapy in DME. The findings, recently featured in the peer-reviewed journal Translational Vision Science & Technology (TVST), highlight sozinibercept’s potential as a first-in-class VEGF-C/D inhibitor when used alongside standard anti-VEGF-A treatments. The trial demonstrated improved safety, enhanced visual acuity, and significant anatomical improvements, offering a promising outlook for treating patients with persistent DME, a condition known for its challenging management.

In the Phase 1b open-label, multicentre, dose-escalation study, nine patients were administered varying doses of sozinibercept (0.3, 1, or 2 mg) in combination with aflibercept (2 mg) over a 12-week period. The study’s primary endpoint focused on safety, while secondary endpoints evaluated best-corrected visual acuity (BCVA) and central subfield thickness (CST). Results indicated a dose-response relationship, with the highest dose of sozinibercept demonstrating the greatest BCVA gains and meaningful CST reduction. These findings underline the therapeutic potential of sozinibercept in addressing the unmet needs of DME patients.

Strategic Growth Through Focused Clinical Programs

Opthea’s immediate focus remains on advancing its flagship product, sozinibercept, through pivotal Phase 3 clinical trials for wet age-related macular degeneration. Two studies, COAST and ShORe, are fully enrolled and expected to deliver topline data in early and mid-2025, respectively. This strategic progression underscores the company’s commitment to developing therapies that significantly improve visual and anatomical outcomes compared to existing anti-VEGF-A monotherapies.

In parallel with its wet AMD programs, Opthea aims to expand the clinical applications of sozinibercept in DME. With the condition’s global prevalence projected to rise from 19 million to 29 million by 2045, the need for innovative treatments is more critical than ever. By leveraging the success of its Phase 1b trial, Opthea is poised to address this growing medical challenge, offering hope to millions affected by diabetes-related vision loss.

Meeting Unmet Medical Needs in Retinal Diseases

Diabetic macular oedema is a leading cause of central vision loss in individuals with diabetes, driven by fluid accumulation in the retina due to leaky blood vessels. While current anti-VEGF-A therapies are effective, they often yield suboptimal results, leaving significant room for improvement. Sozinibercept’s unique mechanism of action—targeting VEGF-C and VEGF-D—aims to complement existing treatments by preventing further vascular leakage and promoting better visual outcomes.

Opthea’s approach reflects a broader commitment to addressing unmet needs in retinal disease management. The Phase 1b trial results demonstrated that sozinibercept not only enhances vision gains but also provides meaningful anatomical improvements. This dual impact positions the therapy as a potentially transformative solution for patients who have not responded adequately to anti-VEGF-A monotherapy.

Opthea’s vision extends beyond therapeutic development to fostering global advancements in retinal healthcare. By combining cutting-edge science with a patient-focused ethos, the company aspires to reduce the burden of retinal diseases worldwide. Its progress in clinical trials exemplifies its dedication to improving the quality of life for individuals with vision-threatening conditions.

The publication of the Phase 1b DME trial results in TVST serves as a milestone in Opthea’s journey. It not only validates sozinibercept’s potential but also highlights the company’s role in shaping the future of retinal disease treatments. With a robust pipeline, strategic focus, and innovative approach, Opthea is well-positioned to transform the standard of care in retinal diseases and redefine what is possible in vision restoration.