Opthea Phase 3 Trial Fails To Meet Primary Endpoint, Faces Financial Uncertainty

On Monday, Opthea Limited OPT released results from its global Phase 3 clinical trial COAST in patients with wet age-related macular degeneration (wet AMD).

The trial evaluated the efficacy and safety of intravitreally administered 2 mg sozinibercept every four or eight weeks in combination with 2 mg Regeneron Pharmaceuticals Inc’s REGN Eylea (aflibercept), as per label, every eight weeks after a loading phase for wet AMD.

The trial did not meet its primary endpoint of mean change in best corrected visual acuity (BCVA) from baseline to week 52.

In wet AMD patients with minimally classic and occult lesions, participants receiving sozinibercept combination therapy with a dosing regimen of every four weeks (n=296) or every eight weeks (n=297) achieved a mean change in BCVA of 13.2 or 13.2 letters from baseline to week 52, respectively, versus 13.8 letters with aflibercept monotherapy (n=299, p-values of 0.59 and 0.62 respectively).

In the overall population, participants receiving sozinibercept combination therapy with a dosing regimen of every four weeks (n=333) or every eight weeks (n=330) achieved a mean change in BCVA of 13.5 and 12.8 letters from baseline to week 52, respectively, versus 13.7 letters with aflibercept monotherapy (n=330, p-values of 0.86 and 0.42 respectively).

No numerical difference was observed in the key secondary endpoints. Sozinibercept combination therapy was well tolerated.

After the negative results from the COAST trial, Opthea is reviewing its obligations under its Development Funding Agreement (DFA) with investors.

Based on the terms of the DFA, the company may be required to make payments that could significantly impact its financial stability. Opthea could owe investors between $0 and $680 million if the agreement is terminated, depending on the circumstances.

Opthea’s leadership is actively discussing with DFA investors to explore possible solutions.

The DFA investors also hold a lien on all of Opthea’s assets, meaning the company cannot take on additional debt or sell key assets without their approval.

No decisions have been made regarding the COAST trial or whether to accelerate and unmask the ShORe trial. Discussions with DFA investors are ongoing to determine the best path forward.

As of February 28, 2025, Opthea had $113.8 million in cash and equivalents. However, there is significant uncertainty about the company’s ability to continue operating.

Opthea has requested that trading be suspended on both ASX and Nasdaq until Opthea is in a position to provide an announcement to the market by Opthea providing more clarity on these issues or the commencement of trading on March 31, 2025.

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