Sydnexis Announces Topline Pivotal Data from Phase 3 STAR Trial of SYD-101 for Patients with Pediatric Progressive Myopia Presented at AMCP Nexus 2025

Largest global clinical trial in pediatric myopia met primary efficacy endpoint of proportion of patients with confirmed progression of -0.75D and key secondary endpoint of annual progression rate T reatment effects evident by month 12 and sustained through month 36; greatest treatment benefit observed in younger children, in participants with a history of myopia progression, or when administered earlier in progression SYD-101 well tolerated with no unexpected atropine-related adverse events DEL MAR, Calif.–( BUSINESS WIRE )–Sydnexis, Inc., ( www.sydnexis.com ), a biopharmaceutical company focused on pediatric progressive myopia (PPM), today announced topline results from the Phase 3 STAR trial of SYD-101, a proprietary 0.01% atropine formulation developed with enhanced ocular delivery characteristics and room-temperature stability to slow the progression of myopia in children. These findings were recently presented in a poster session at Academy of Managed Care Pharmacy (AMCP) Nexus 2025 in National Harbor, MD. “The data presented at AMCP Nexus 2025 show strong treatment effects in the full study population at 36 months but in younger patients, the magnitude of effect gets larger, and in patients with documented faster progression, the magnitude of treatment effect increases even more,” said Perry Sternberg, Chief Executive Officer of Sydnexis. “The STAR data demonstrate that younger kids who historically exhibit fast progression, which is the subpopulation of children at highest risk for severe disease, benefit the most from treatment with SYD-101 0.01%.” The Phase 3 STAR ( St udy of A tropine for the R eduction of Myopia Progression) trial is the largest global clinical program completed to date in pediatric myopia. It evaluated a broad population of 847 children aged 3-14 at treatment initiation. Participants with myopia -0.50 diopters (D) to ‑6.00D with a mean baseline progression of -2.65D were enrolled across the U.S. and Europe, and randomized (1:1:1) to vehicle (placebo), SYD-101 0.01%, or SYD-101 0.03%. The study’s primary efficacy endpoint was proportion of patients with confirmed progression of -0.75D, an endpoint proposed by FDA and a key secondary endpoint was annual progression rate. SYD-101 0.01% successfully met both the primary and key secondary endpoints. Additionally, SYD-101 was well tolerated with no unexpected atropine-related adverse events. “Findings from the STAR trial, which is the largest pediatric myopia study to date, reinforce the importance of early intervention, both in terms of patient age and disease progression,” said Christie Morse MD, Executive Vice President of the American Association for Pediatric Ophthalmology and Strabismus. “The results highlight the potential of low-dose atropine to help preserve children’s vision over time, a meaningful step forward in how we approach the management of this increasingly prevalent condition.” “Compounded low-dose atropine has posed challenges from both an access and a consistency standpoint. SYD-101’s unique formulation of atropine, which has been well-researched and supported by the results of the STAR trial, could be a valuable treatment option to help protect children’s vision and improve long-term outcomes,” said Kevin Chan, OD, MS, FAAO, Senior Clinical Director of Treehouse Eyes. SYD-101 is currently approved in the European Union, where it is licensed to Santen S.A. and marketed as Ryjunea ® . Summary of Key Topline Findings 1 : In the full study population, SYD-101 0.01% met its primary efficacy endpoint of proportion of patients with confirmed progression of -0.75D at 36 months versus vehicle [139 vs 111 patients (Vehicle vs. 0.01%); p=0.0226]. Additionally, SYD-101 0.01% met its key secondary endpoint, mean myopic annual progression rate, in the full study population at month 36 (Vehicle: -0.38 D/year vs 0.01% -0.30 D/year (p=0.0002). The primary efficacy and key secondary endpoints were met even though 36% of the total patients in the SYD-101 0.01% arm were between the ages 16 to 18 and exhibited very little progression by the end of the study. In the pre-specified analysis of change from baseline spherical equivalent (SE), SYD-101 0.01% and 0.03% had significantly smaller changes compared with vehicle in the full study population at month 36 [Vehicle ‑0.92 D; 0.01% -0.71 D (p-0.0022); 0.03% 0.76 D (p=0.0158)]. Data presented at AMCP also showed that the magnitude of clinical effect of change in SE was even larger in younger patients (3-12 years old at treatment initiation), with both doses demonstrating nominal statistical significance. [Vehicle ‑1.07 D; 0.01% -0.77 D (p-0.0002); 0.03% -0.85 D (p=0.0065)]. Also presented at AMCP was data in a subgroup of fast progressors [more than -0.5D/yr progression prior to the study] at 36 months. In these patients with low to moderate myopia (-0.50 D to -3.00D) at study initiation, SYD-101 0.01% reduced the progression by more than 50%, [Vehicle -1.18 D; 0.01% -0.51D (p=0.0004); 0.03% -0.92 D (p-0.1505)] highlighting the importance of early disease modifying intervention in childhood myopia. “The STAR study is an extremely important contribution to the growing body of evidence supporting low-dose atropine as a therapeutic option for PPM and reinforces the unmet need eye care practitioners see in the field every day,” said Patrick Johnson, PhD, President of Sydnexis. “SYD-101 can meaningfully slow the trajectory of this disease and would be a welcome improvement to the compounded products that lack adequate quality oversight that are currently being used chronically in kids.” Table 1. Confirmed Myopia Progression Worse than -0.75D at Month 36 Confirmed Progression At or Before Month 36 Vehicle (N=282) 0.01% (N=282) 0.03% (N=282) Participants with progression -n (%) 138.9 (49.26) 111.4 (39.50) 122.0 (43.11) 95% CI percentage with progression 43.0, 55.5 33.4, 45.6 37.0, 49.2 CMH p-value, difference to Vehicle — 0.0226 0.1319 Table 2. Annual Progression Rate at Month 36 Full Analysis Set (3-14 year-olds) Sub-group 3-12 year olds Vehicle -0.38 D/year — Vehicle -0.41 D/year — 0.01% -0.30 D/year p=0.0002 0.01% -0.30 D/year P<0.0001 0.03% -0.32 D/year p=0.0018 0.03% -0.32 D/year p=0.0005 Table 3. Mean Change from Baseline Spherical Equivalent at Month 36 Full Analysis Set (3-14 year-olds) Sub-group 3-12 year olds Vehicle -0.92 D — Vehicle -1.07 D — 0.01% -0.71 D p=0.0022 0.01% -0.77 D P=0.0002 0.03% -0.76 D p=0.0158 0.03% -0.85 D p=0.0065 Poster Presentation Details Title: A Novel Low-dose Atropine Eye Drop Slows the Progression of Pediatric Myopia: Evidence from the STAR Study Poster Number : 170 For more information, please click here . About SYD-101 SYD-101 is a proprietary low-dose atropine formulation developed to slow the progression of pediatric progressive myopia. It features several novel attributes designed to optimize tolerability, stability, and clinical performance: Demonstrated enhanced ocular tissue permeability in preclinical animal models compared to other formulations; Stable for up to 3 years at room temperature; Near-neutral pH, which may contribute to a favorable ocular safety and comfort profile. About Pediatric Progressive Myopia (PPM) Pediatric Progressive Myopia (PPM) is the most common eye disease in children and a rapidly rising global health concern. Nearly one-third of children worldwide are already affected by this degenerative disease, with prevalence projected to exceed 740 million cases by 2050 (Liang et al., 2024). In North America, myopia prevalence is expected to reach nearly 60% by 2050, according to a landmark study published in Ophthalmology (Holden et al., 2016). The most rapid progression of the disease occurs in children ages 3 to 10 years (Hu et al., 2020), with patients who start progressing younger experiencing more severe outcomes and associated co-morbidities, including cataracts, glaucoma, retinal detachment, and myopic maculopathy. Despite its growing prevalence, there are currently no FDA-approved pharmaceutical options in the United States to slow the progression of PPM, representing a significant gap in available treatment options for patients. About Sydnexis, Inc. Sydnexis, Inc . is a biopharmaceutical company dedicated to advancing care for pediatric progressive myopia. The company’s lead compound, SYD-101, is currently approved in the European Union, where it is licensed to Santen S.A. and marketed as Ryjunea ® . Sydnexis is supported by leading life-science investors, including Visionary Ventures, RA Capital, Longitude Capital, and Bluestem Capital. For more information on Sydnexis, please visit www.sydnexis.com . 1 In accordance with standard statistical methodologies to control for multiplicity with comparison of vehicle to two doses, both doses of SYD-101 were tested simultaneously. If one dose did not meet the primary analysis threshold (p<0.05), the other dose was required to demonstrate more rigor (p<0.025) to be successful. 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