Opthea and UNITY Fall Short in Challenging Regeneron's Eylea Dominance

Opthea's Sozinibercept Fails to Outperform Eylea in Wet AMD Trial

Australian biotech Opthea Limited announced disappointing results from its Phase III COAST study, revealing that its investigational VEGF blocker sozinibercept failed to significantly outperform Regeneron's Eylea in improving vision for patients with wet age-related macular degeneration (AMD).

At the 52-week mark, patients treated with sozinibercept showed an average improvement in best-corrected visual acuity (BCVA) of 12.8 letters from baseline, compared to a mean improvement of 13.7 letters in the Eylea group. This outcome falls short of demonstrating superiority over the current standard of care.

Sozinibercept, Opthea's sole clinical candidate, targets VEGF-C and VEGF-D subtypes, which are reportedly highly expressed in patients receiving standard VEGF-A therapies. The company had hoped this unique mechanism of action would provide an advantage over existing treatments.

The Phase III setback presents significant financial challenges for Opthea, which disclosed that it is currently assessing its obligations to investors. The company warned that certain payments "would have a material adverse impact on the solvency of the company." Opthea is now in "active discussions" with investors to determine the best path forward, including the possibility of terminating the COAST trial or unmasking a separate wet AMD trial.

UNITY Biotechnology's UBX1325 Narrowly Misses Primary Endpoint in Diabetic Macular Edema Study

California-based UNITY Biotechnology also faced disappointment as its investigational eye injection UBX1325 fell short of demonstrating non-inferiority to Eylea in patients with diabetic macular edema. Topline data from the Phase IIb ASPIRE study showed that UBX1325 improved visual acuity by more than five letters at 24 and 36 weeks compared to baseline.

While UNITY claimed these gains were "non-inferior" to Eylea at almost all time points through 36 weeks, the treatment missed the mark at the critical average of weeks 20 and 24. At this time point, UBX1325 showed non-inferiority at an 88% confidence interval, just shy of the 90% prespecified threshold for the study's primary endpoint.

Despite the setback, UNITY CEO Anirvan Ghosh remained optimistic, highlighting that UBX1325 "showed robust vision improvements in a difficult to treat patient population." The company plans to continue advancing the candidate into late-stage studies.

Regeneron's Eylea Faces New Challenges Amid Recent Court Decision

These clinical trial results come at a crucial time for Regeneron Pharmaceuticals, as the company recently lost its court appeal to prevent Amgen's biosimilar Pavblu from entering the U.S. market. The failed appeal, described by BMO Capital Markets analysts as a "modest negative to Regeneron," adds to the existing pressures on the Eylea franchise.

The ability of Eylea to maintain its position as the standard of care in both wet AMD and diabetic macular edema, despite challenges from these investigational therapies, underscores its continued importance in the ophthalmology market. However, the looming threat of biosimilar competition and ongoing efforts to develop superior treatments suggest that Regeneron may face increasing pressure to defend its market share in the coming years.