Sydnexis, Inc., (www.sydnexis.com), a biopharmaceutical company focused on pediatric progressive myopia (PPM), today announced that the U.S. Food and Drug Administration (FDA) has issued a complete response letter (CRL) for the New Drug Application (NDA) for SYD-101, a proprietary 0.01% atropine formulation developed with enhanced ocular delivery characteristics and room-temperature stability to slow the progression of pediatric myopia in children.

Sydnexis’ NDA for SYD-101 was supported by the results of the Phase 3 STAR (Study of Atropine for the Reduction of Myopia Progression) trial, the largest global clinical program completed to date in pediatric myopia, which evaluated more than 800 children aged 3-14 at treatment initiation. The study met its primary efficacy endpoint of proportion of patients with confirmed progression of -0.75D, which FDA had encouraged the Company to use. A key secondary endpoint, annual progression rate, also met statistical significance at 12, 24, and 36 months. Furthermore, in a subgroup of fast progressors [more than -0.5D/yr] at 36 months, SYD-101 demonstrated statistically significant treatment effects of a reduction in progression of more than 50%.

The FDA acknowledged in the complete response letter (CRL) that this primary endpoint was met but stated its view that the data do not support the effectiveness of low-dose atropine in children with myopia. No deficiencies were noted related to safety or product quality.

“While we are surprised and disappointed with this decision, Sydnexis is committed to working with the FDA to address the items outlined in the CRL and determining the best path forward toward approval for SYD-101,” said Perry Sternberg, Chief Executive Officer of Sydnexis. “Our goal is to provide eye care professionals in the U.S. with the first FDA-approved option to help slow the progression of pediatric myopia, rather than continued reliance on compounded formulations of atropine. We remain confident in our data and the potential of SYD-101 to fill a critical innovation gap and treat the most common eye disease in children.”

SYD-101 is currently approved in the European Union, where it is licensed to Santen S.A. and marketed as Ryjunea®.

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;
• 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.

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