Sydnexis, Inc. has received FDA acceptance for its new drug application (NDA) of SYD-101, a proprietary formulation under clinical development for the treatment of pediatric myopia progression.
First up: Sydnexis.
The San Diego, California-based pre-commercial stage biopharmaceutical company was launched in 2014 with a single purpose: to decrease myopia progression in pediatric patients.
Key to this mission: SYD-101, its patented eye drop formulation.
Talk about this eye drop.
As Sydnexis’s lead asset, SYD-101 is a patented low-dose atropine sulfate ophthalmic solution intended to decrease pediatric myopia progression.
Why develop a low-dose (versus a high-dose) atropine formulation?
The company has addressed this reasoning.
Sydnexis stated that while atropine 1.0% and 0.5% (already commercially available) have undergone clinical testing and demonstrated effectiveness in treating myopia progression, high doses of this formulation have also been associated with a high incidence of adverse events (AEs)—often resulting in treatment discontinuation.
So what’s the argument in favor of low-dose atropine?
It’s a little complicated, with both pros and cons to this lower dosage.
While research on low-dosage atropine (0.01% and 0.05%) in recent years has found such drops to delay and potentially even prevent the onset of myopia, the only form of the dosage currently available on the U.S. market must be obtained directly from a compounded pharmacy.
- To note: A compound pharmacy customizes “compounded drug” medications (that are not FDA-approved) for specific patient needs when either a marketed (and approved) drug isn’t appropriate to treat them or there is a drug shortage.
And the issue with compounded low-dose atropine?
With no federal guidelines to clinically test and verify the safety and efficacy of compounded low-dose atropine—including its potency, pH, and sterility—a number of AEs can result in patients.
Such examples include significant variability in physiologic response as well as reported atropine degradation after just one month of storage at room temperature.
Keep in mind: This degradation not only reduces the formulation’s concentration but also its efficacy.
So what makes SYD-101 unique?
The eye drop is a “specifically engineered formulation” with a “unique stabilizing excipient” that prevents the rapid atropine degradation occurring in most drops made in water-based solutions.
To be more specific: Sydnexis CEO Perry J. Sternberg previously shared that the SYD-101 formulation “could be the first highly stable form of atropine at a physiologically neutral pH, thereby potentially providing additional benefits, including improved penetration and comfort.”
- Additionally: It is “also possibly the first eye drop with a 3-year shelf life.”
And what is this acceptance based on?
That would be 3-year data from the primary and secondary endpoints of the pivotal phase 3 STAR study (NCT03918915).
About the study: The trial is analyzing two doses of SYD-01 (0.01% and 0.03%) versus a placebo dose to determine its ability to slow pediatric myopia progression as well as its risk for associated comorbidities.
The details:
- Design: 5-arm, multicentered, randomized, double-masked, vehicle-controlled trial
- Participants: 852 patients (aged 3 to 14) diagnosed with myopia (0.5 D to 5.00 D in both eyes)
- Setup: Trial split into two parts, with patients receiving one drop in each eye of one (of two possible) SYD-101 doses or a vehicle solution (placebo)
- Outcome measures (measured at or up to 36 months):
- Primary: myopic progression of >0.75 D
- Secondary: see here
- Duration: 4 years (in total)
Any data yet … or next steps for the study?
No data—but we do have an update on its next stage.
Since the study recently completed its third year, participants are being re-randomized into a 1-year washout (with placebo-receiving patients to receive 0.03% atropine drops for the fourth year).
Notably: “Our landmark STAR Study is the largest clinical study ever completed for the treatment of progression of pediatric myopia,” stated Sydnexis President Patrick Johnson, PhD.
And the potential for the U.S. market?
Sternberg also previously stated that SYD-101 could become the “foundation of treatment as the first and only pharmacological option to slow the progression of pediatric myopia” as well as the “only treatment option approved for patients under the age of 8.”
Lastly: When can we expect an FDA decision on this NDA?
The agency assigned a Prescription Drug User Fee Act (PDUFA) target action date of Oct. 23, 2025.