Del Mar, California, USA | March 23, 2026
Sydnexis, Inc. has announced new Phase 3 clinical data from its STAR trial evaluating SYD-101, a novel low-dose atropine formulation for pediatric progressive myopia (PPM), presented at the 2026 American Association for Pediatric Ophthalmology and Strabismus (AAPOS) Annual Meeting in Boston. The results highlight statistically significant efficacy, strong safety profile, and meaningful subgroup insights, reinforcing SYD-101’s potential as a first-in-class pharmaceutical treatment for pediatric myopia progression, an area with significant unmet medical need in the United States.
Phase 3 STAR Trial Demonstrates Strong Efficacy Outcomes
The Phase 3 STAR trial, the largest global pediatric myopia study to date, enrolled 847 children aged 3 to 14 years across the United States and Europe. Patients were randomized to receive either SYD-101 0.01% or placebo, with the primary endpoint defined as confirmed myopia progression of -0.75 diopters or worse. The study successfully met both its primary and key secondary endpoints, demonstrating statistically significant reductions in myopia progression (p<0.001) and meaningful improvements in annual progression rate over 12, 24, and 36 months.
At 36 months, SYD-101 achieved a notable reduction in annual progression rate compared to placebo, confirming its long-term therapeutic potential. These results position SYD-101 as a leading candidate for regulatory approval in the U.S., where currently no FDA-approved pharmaceutical therapies exist for pediatric myopia progression. The trial’s robust design and large patient population further strengthen the clinical validity and regulatory relevance of the findings.
Subgroup Analysis Identifies High-Response Patient Populations
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A key highlight of the AAPOS presentation was the comprehensive subgroup analysis, which identified children aged 3 to 12 years and fast progressors with mild to moderate myopia as the populations deriving the greatest therapeutic benefit. In younger children, SYD-101 demonstrated up to 47.9% reduction in myopia progression at 12 months, with sustained effects observed through 36 months.
Even more striking results were observed in fast progressors, where treatment reduced myopia progression by up to 76.3% at 12 months, with continued benefits at later time points. These findings provide clinically actionable insights, enabling physicians to better target early intervention strategies and optimize treatment outcomes. The data also reinforce the importance of early diagnosis and treatment initiation, particularly in younger patients who are at higher risk of rapid disease progression and long-term complications.
Favorable Safety Profile and Regulatory Implications
SYD-101 demonstrated a favorable safety and tolerability profile, with no unexpected atropine-related adverse events, supporting its potential for long-term use in pediatric populations. The formulation is designed with enhanced ocular permeability, stability, and near-neutral pH, which may contribute to improved patient comfort and compliance, key factors in chronic disease management.
Importantly, SYD-101 is already approved in the European Union and United Kingdom, where it is marketed as Ryjunea®, providing additional validation of its clinical utility and commercial potential. The newly presented Phase 3 data are expected to play a critical role in ongoing regulatory discussions with the U.S. FDA, potentially paving the way for approval in a market with millions of affected pediatric patients.
Pediatric progressive myopia is a rapidly growing global health concern, with prevalence projected to reach over 740 million cases by 2050. The condition is associated with serious complications, including retinal detachment, glaucoma, and vision loss, underscoring the urgent need for effective and early therapeutic intervention.
The latest Phase 3 STAR trial data position SYD-101 as a promising, first-in-class pharmaceutical solution for pediatric progressive myopia, supported by strong efficacy, targeted subgroup benefits, and a favorable safety profile. With robust clinical evidence and ongoing regulatory engagement, Sydnexis is advancing toward addressing a critical gap in pediatric eye care, potentially transforming the treatment paradigm for a rapidly growing global disease.
Source: Sydnexis press release
