Axpaxli Shows Promise vs. Aflibercept for Wet AMD in Phase 3 Trial
- A new tyrosine kinase inhibitor, Axpaxli, has demonstrated superiority to the standard of care, aflibercept, in the treatment of wet age-related macular degeneration (AMD), according to results from...
- Age-related macular degeneration is a common condition that affects the central part of the retina, known as the macula.
- Axpaxli is an intravitreal implant – meaning it’s injected directly into the eye – that delivers axitinib, a tyrosine kinase inhibitor.
A new tyrosine kinase inhibitor, Axpaxli, has demonstrated superiority to the standard of care, aflibercept, in the treatment of wet age-related macular degeneration (AMD), according to results from a phase 3 clinical trial. The findings, released on , offer a potential new option for patients with this leading cause of vision loss.
What is Wet AMD?
Age-related macular degeneration is a common condition that affects the central part of the retina, known as the macula. The “wet” form of AMD occurs when abnormal blood vessels grow under the retina, leaking fluid and blood, which can cause rapid vision loss. Current treatment typically involves regular injections of anti-VEGF (vascular endothelial growth factor) medications, like aflibercept, to inhibit the growth of these blood vessels. However, these injections are frequent and place a significant burden on patients.
Axpaxli: A Novel Approach
Axpaxli is an intravitreal implant – meaning it’s injected directly into the eye – that delivers axitinib, a tyrosine kinase inhibitor. Tyrosine kinases play a role in the growth of new blood vessels, and inhibiting them can help control the progression of wet AMD. Unlike aflibercept, which requires repeated injections, Axpaxli is designed to provide a more sustained release of the medication, potentially reducing the frequency of treatment.
The SOL-1 Trial Results
The phase 3 SOL-1 trial enrolled treatment-naive patients with wet AMD. Participants received either a single injection of Axpaxli or aflibercept after an initial loading phase with aflibercept. The primary endpoint of the trial was the proportion of patients who maintained visual acuity – defined as a loss of fewer than 15 ETDRS letters on a standard eye chart – at .
The results showed that of patients treated with Axpaxli maintained their vision at , compared to of those treated with aflibercept. This difference was statistically significant, with a risk difference of (). Further analysis at demonstrated continued benefit, with of Axpaxli-treated patients maintaining vision compared to in the aflibercept group ().
Rescue-Free Rates and Fluid Control
Beyond visual acuity, the trial also assessed “rescue-free” rates – the proportion of patients who did not require additional treatment (rescue injections) to control their disease. A higher percentage of patients in the Axpaxli group remained rescue-free at compared to those receiving aflibercept. Axpaxli demonstrated superior control of central subfield thickness (CST), a measure of fluid buildup in the macula, at .
Safety Profile
According to the release, Axpaxli was generally well-tolerated in the SOL-1 trial, with no treatment-related ocular or systemic serious adverse events reported. Patients are continuing to be monitored for safety through , and have received a second injection at , with another planned for .
What This Means for Patients
“Despite advances in [neovascular age-related macular degeneration] treatment, there remains unpredictability around patient response to treatment, resulting in unsustainable burden and lack of adherence,” said Pravin U. Dugel, MD, executive chairman, president and CEO of Ocular Therapeutix. “fluid fluctuations and increased fibrosis lead to poor long-term outcomes. We believe the implications of the SOL-1 trial results provide compelling evidence of the potential to dramatically reduce treatment burden while maintaining vision, which should translate into improved quality of life for patients and caregivers.”
Ocular Therapeutix plans to submit a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) based on the SOL-1 data. The company is also continuing a complementary phase 3 trial, SOL-R, which is evaluating Axpaxli’s non-inferiority to aflibercept, with topline data expected in the first quarter of .
These findings represent a significant step forward in the treatment of wet AMD, offering the potential for a more convenient and effective therapy for patients facing this debilitating condition. Further research and regulatory review will be necessary before Axpaxli becomes widely available.