Roche announced positive long-term results from its phase III BALATON and COMINO studies evaluating Vabysmo (faricimab) in patients with macular edema due to retinal vein occlusion (RVO). The data show that patients maintained vision improvements while extending treatment intervals up to every four months.
Sustained Efficacy with Extended Treatment Intervals
From weeks 24 to 72, all participants in both studies received Vabysmo using a treat-and-extend dosing regimen, which allows physicians to tailor treatment intervals based on individual patient response. The results demonstrated that patients maintained the vision gains achieved in the first 24 weeks while extending the time between treatments.
Importantly, Vabysmo continued to show robust and sustained drying of retinal fluid from baseline up to week 72, as measured by reduction in central subfield thickness. This marks the first time that vision and anatomical improvements have been maintained for more than a year using a personalized treat-and-extend dosing regimen in global phase III studies for both branch retinal vein occlusion (BRVO) and central retinal vein occlusion (CRVO).
"These are the first retinal vein occlusion studies to show vision maintenance and anatomical improvements up to 72 weeks in both central and branch RVO," said Levi Garraway, M.D., Ph.D., Roche's Chief Medical Officer and Head of Global Product Development. "These data further support Vabysmo's potential as a new treatment for RVO, allowing people to preserve their vision while spending less time managing their condition."
The safety profile remained consistent with previous studies, with Vabysmo being generally well-tolerated.
Expanding Treatment Indications
RVO affects approximately 28 million people globally and, if approved, would become the third indication for Vabysmo, joining neovascular or 'wet' age-related macular degeneration (nAMD) and diabetic macular edema (DME). Together, these three conditions affect around 70 million people worldwide and are among the leading causes of vision loss.
Data from the first 24 weeks of the BALATON and COMINO studies, presented earlier this year, showed early and sustained vision improvement with Vabysmo, with both studies meeting their primary endpoints of non-inferior vision gains compared to aflibercept. Secondary endpoints demonstrated that Vabysmo achieved rapid and robust drying of retinal fluid.
Regulatory applications for Vabysmo in RVO are currently under review by health authorities globally, including the U.S. Food and Drug Administration and European Medicines Agency, with a decision from the FDA expected in late 2023.
Novel Mechanism of Action
Vabysmo stands out as the first and only bispecific antibody approved for ophthalmic use. Its unique mechanism targets and inhibits two distinct signaling pathways linked to vision-threatening retinal conditions by neutralizing both angiopoietin-2 (Ang-2) and vascular endothelial growth factor-A (VEGF-A).
The level of Ang-2 is elevated in RVO and is thought to drive disease progression. By blocking pathways involving both Ang-2 and VEGF-A, Vabysmo is designed to stabilize blood vessels, potentially offering advantages over treatments targeting VEGF-A alone.
Global Adoption and Availability
To date, Vabysmo has received approval in more than 80 countries for the treatment of nAMD and DME, including the United States, Japan, the United Kingdom, and the European Union. The treatment has achieved public reimbursement in over 25 markets, with more than 1.5 million doses distributed globally.
About Retinal Vein Occlusion
RVO is the second most common cause of vision loss due to retinal vascular diseases, affecting an estimated 28 million adults globally, primarily those aged 60 or older. The condition can lead to severe and sudden vision loss.
RVO typically results in sudden, painless vision loss in the affected eye because vein blockage restricts normal blood flow in the affected retina, resulting in ischemia, bleeding, fluid leakage, and retinal swelling called macular edema. Current standard treatment involves repeated intravitreal injections of anti-VEGF therapies.
There are two main types of RVO:
- Branch retinal vein occlusion (BRVO), which affects more than 23 million people globally and occurs when one of the four smaller 'branches' of the main central retinal vein becomes blocked
- Central retinal vein occlusion (CRVO), which affects more than four million people worldwide and occurs when the eye's central retinal vein becomes blocked
Study Design
The BALATON and COMINO studies are randomized, multicenter, double-masked, global phase III trials evaluating the efficacy and safety of Vabysmo compared to aflibercept.
For the first 20 weeks, patients were randomized 1:1 to receive six-monthly injections of either Vabysmo (6.0 mg) or aflibercept (2.0 mg). From weeks 24 to 72, all patients received Vabysmo (6.0 mg) up to every four months, using a treat-and-extend dosing regimen.
The BALATON study included 553 people with branch retinal vein occlusion, while the COMINO study involved 729 people with central retinal or hemiretinal vein occlusion. The primary endpoint of each study was the change in best-corrected visual acuity from baseline at 24 weeks.
Detailed results from weeks 24 to 72 of both studies will be presented at an upcoming medical meeting.
Broader Clinical Development Program
Roche continues to expand its clinical development program for Vabysmo, which includes extension studies evaluating long-term safety and efficacy in nAMD and DME, as well as phase IV studies in underrepresented patient populations and real-world settings.
The company is also supporting independent studies to further understand retinal conditions with high unmet needs, reinforcing its commitment to advancing ophthalmology care and preserving vision for patients worldwide.
