In a significant development for the treatment of wet age-related macular degeneration (AMD), Ocular Therapeutix announced Tuesday that its investigational drug, Axpaxli, demonstrated superior efficacy compared to aflibercept (Eylea) in a Phase 3 clinical trial. The results, eagerly anticipated by the ophthalmology community, suggest a potential for reduced injection frequency for patients battling this leading cause of vision loss. Wet AMD affects millions worldwide and current treatment regimens often require frequent intravitreal injections to maintain vision, posing a burden for both patients and healthcare systems.
The trial focused on evaluating Axpaxli’s ability to maintain vision while potentially decreasing the number of injections needed. Currently, patients with wet AMD typically require injections every one to two months, depending on their individual response to treatment. Axpaxli, utilizing a sustained-release hydrogel technology, aims to deliver the therapeutic agent over an extended period, reducing the necessitate for frequent visits to the clinic. This novel approach to treatment could significantly improve the quality of life for those affected by this debilitating condition.
Axpaxli Demonstrates Vision Maintenance in Phase 3 Trial
The Phase 3 SOL-1 trial revealed that 74.1% of participants receiving a single eye injection of Axpaxli maintained their vision at week 36, representing a 17.5% risk difference (p=0.0006) compared to those treated with aflibercept (2 mg). Even at week 52, Axpaxli continued to demonstrate a benefit, with 65.9% of subjects maintaining vision, a 21.1% risk difference (p<0.0001) compared to the aflibercept group, according to a press release from Ocular Therapeutix. Rescue-free rates – the percentage of patients who did not require additional treatment during the study period – were also notably higher in the Axpaxli arm, reaching 80.6%, 74.7%, and 68.8% at weeks 24, 36, and 52, respectively.
The study’s findings indicate that Axpaxli’s unique delivery system, targeting the vascular endothelial growth factor (VEGF) pathway, effectively suppresses the abnormal blood vessel growth characteristic of wet AMD. VEGF plays a crucial role in the development and progression of the disease, and inhibiting its activity is a cornerstone of current treatment strategies. Axpaxli’s sustained-release mechanism appears to provide a more consistent and prolonged suppression of VEGF, potentially leading to improved outcomes and reduced treatment burden. The company reports that 77.1% of AXPAXLI subjects randomized in SOL-1 would have been rescue-free at Week 24 using SOL-R rescue criteria which align closely with clinical practice.
Commercial Potential and Regulatory Pathway
While the results are promising, analysts note that the margin of superiority over aflibercept was somewhat smaller than anticipated, potentially sparking debate about Axpaxli’s commercial viability in a competitive market. STAT News reports that investors were expecting a more pronounced difference. But, the demonstrated efficacy and potential for reduced injection frequency still position Axpaxli as a potentially valuable addition to the treatment landscape.
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, following planned discussions with the agency. The company anticipates that the positive trial results will support its application for approval. The drug is also being investigated for the treatment of diabetic retinopathy, another leading cause of vision loss. Ocular Therapeutix’s website details the ongoing research and development efforts for Axpaxli, highlighting its potential to transform the treatment of retinal diseases.
The safety profile of Axpaxli in the SOL-1 trial was also encouraging, with no treatment-related ocular serious adverse events reported. This is a critical factor for any new ophthalmic treatment, as patient safety is paramount.
What’s Next for Axpaxli and Wet AMD Treatment?
The coming months will be crucial as Ocular Therapeutix prepares its NDA submission and engages with the FDA. The agency’s review will determine whether Axpaxli will be approved for clinical use. Further data from ongoing studies, including long-term follow-up of SOL-1 participants, will also provide valuable insights into the drug’s durability and long-term safety. The potential approval of Axpaxli could offer a new treatment option for patients with wet AMD, potentially reducing the burden of frequent injections and improving visual outcomes. The detailed data from the SOL-1 trial are scheduled to be presented at the 49th Macula Society Annual Meeting.
This research represents a step forward in the ongoing quest to develop more effective and convenient treatments for age-related macular degeneration.
Disclaimer: This article is for informational purposes only and should not be considered medical advice. Always consult with a qualified healthcare professional for diagnosis and treatment of any medical condition.
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