The Eylea franchise was in need of a win after an appellate court last week denied the pharma’s bid to block Amgen’s biosimilar from the U.S.
Two investigational eye therapies, from Opthea Limited and UNITY Biotechnology, were unable to unseat Regeneron’s blockbuster anti-VEGF therapy Eylea on Monday as the standard of care in two different ophthalmic indications.
The news comes as Regeneron last week lost its court appeal to prevent Amgen’s biosimilar Pavblu from entering the U.S. market. In a March 15 note to investors, BMO Capital Markets analysts said the failed appeal “reads as a modest negative to Regeneron” and compounds the existing risk to its Eylea franchise.
Presenting topline data from the Phase III COAST study, Australian biotech Opthea revealed that its investigational VEGF blocker sozinibercept failed to significantly outperform Eylea at improving vision in patients with wet age-related macular degeneration (AMD).
At 52 weeks, best-corrected visual acuity (BCVA) in those treated with sozinibercept improved by 12.8 letters on average from baseline, while mean improvement in Eylea counterparts was 13.7 letters.
Like Eylea, sozinibercept is designed to be injected into the eye and also works by binding VEGF, a protein involved in the formation of new blood vessels. Uniquely, however, sozinibercept targets the VEGF-C and VEGF-D subtypes (Eylea binds to VEGF-A and placental growth factor), which according to Opthea is highly expressed in patients receiving the standard VEGF-A therapies, in turn leading to leaky blood vessels that could ultimately also harm vision.
Sozinibercept is Opthea’s only clinical candidate, and wet AMD is its most advanced indication. Monday’s Phase III stumble thus presents a financial problem for the biotech, which in its news release revealed that it “has been assessing its rights and obligations” to investors. Opthea could be on the hook for certain payments that “would have a material adverse impact on the solvency of the company,” it wrote.
The biotech is in “active discussions” with its investors, trying to determine “whether there is a pathway that represents the best outcome for the company and its shareholders,” according to Monday’s release. Opthea has yet to reach a decision but is mulling over terminating the COAST trial or unmasking a separate wet AMD trial.
Also on Monday, California-based UNITY Biotechnology announced that its investigational eye injection UBX1325 likewise fell 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 versus baseline.
These gains, according to UNITY, were “non-inferior” to Eylea at almost all time points through 36 weeks except at the 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 missing its target, UNITY CEO Anirvan Ghosh sang an optimistic tune in a statement on Monday, noting that UBX1325 “showed robust vision improvements in a difficult to treat patient population.” The biotech will continue to advance the candidate into late-stage studies.
