Opthea Limited (ASX: OPT) is a clinical-stage biopharmaceutical company headquartered in Melbourne, Australia. It focuses on developing novel therapies for retinal eye diseases, particularly wet age-related macular degeneration (wet AMD), a chronic condition that causes blurred vision and can lead to blindness. Opthea’s flagship investigational therapy, sozinibercept (formerly OPT-302), was being tested in combination with existing anti-VEGF treatments to improve visual outcomes.
Phase 3 Trial Results: Disappointment and Fallout
On 31 March 2025, Opetha terminated the COAST trial and accelerated topline results from its second Phase 3 trial, ShORe, following disappointing results across both studies.
The COAST trial, which combined sozinibercept with aflibercept (a commonly used anti-VEGF therapy), was halted after failing to demonstrate the anticipated visual improvement in wet AMD patients. In response, the company rushed the release of results from ShORe, another standard wet AMD therapy. Unfortunately, ShORe also failed to meet its primary endpoint—mean change in best corrected visual acuity (BCVA) from baseline to week 52.
In a subgroup of patients with minimally classic and occult lesions, the results were similarly underwhelming. Here, sozinibercept combination again underperformed the 14.2-letter gain seen with monotherapy. While the therapy was well tolerated, the failure to show clinical benefit ultimately led to the decision to discontinue development of sozinibercept for wet AMD.
Opthea Responds: Disappointment and Strategic Shift
CEO Dr. Frederic Guerard expressed disappointment, acknowledging the trials’ failure to deliver meaningful vision improvement and reiterated the company’s commitment to evaluating next steps in consultation with stakeholders.
Given the lack of efficacy, Opthea and its Development Funding Agreement (DFA) investors agreed to immediately cease development of sozinibercept in wet AMD. Fortunately, this did not trigger a termination event under the DFA that would immediately require repayment.
However, serious financial risks remain. Opthea warned that certain conditions—such as insolvency or termination of the DFA—could require the company to repay a multiple of the funding received, which would pose a significant threat to its solvency.
Financial Snapshot and Uncertainty
Opthea reported it expects to hold around US$100 million in cash and cash equivalents at the end of March 2025. While this provides some short-term runway, the company cautioned that material uncertainty remains regarding its ability to continue operating as a going concern.
To mitigate potential insolvency issues, Opthea is currently relying on protections under Section 588GA of the Corporations Act 2001 (Cth)—a “safe harbour” provision designed to shield directors from liability while the company attempts to trade through financial distress. In light of this precarious position, the Australian Securities Exchange (ASX) has suspended trading of Opthea’s securities under Listing Rule 17.3. The full details of Opthea’s Development Funding Agreement are publicly accessible via its Annual Report on Form 20-F, filed with the U.S. Securities and Exchange Commission (SEC) in August 2024.
Final Thoughts
Opthea’s once-promising bid to revolutionise wet AMD treatment with sozinibercept has ended in disappointment, as back-to-back Phase 3 trials failed to outperform existing therapies. While the safety profile was encouraging, efficacy—particularly in improving visual acuity—fell short of expectations.
The company now faces an uncertain future, with suspended trading, financial pressure, and high-stakes negotiations with investors. The next steps will depend heavily on the outcome of ongoing discussions with DFA stakeholders, Opthea’s ability to preserve capital, and whether it can pivot or restructure its operations for long-term survival.
Investors, patients, and industry observers alike will be watching closely as the company seeks to navigate these turbulent waters and determine what comes next in its corporate journey.
