A clinical trial now underway is testing whether retinopathy of prematurity (ROP) can be prevented before the disease ever reaches the stage where screening identifies infants for treatment. If this approach is successful, it could shift management of the disease from detection to prevention, observed Darius Moshfeghi, MD, during the Clinical Trials at the Summit meeting in Las Vegas.

ROP, the leading cause of pediatric blindness in the United States, remains a significant clinical and logistical challenge, observed Dr. Moshfeghi. Approximately 20,000 premature infants are born each year in the United States, and 30% to 40% develop ROP. Current management relies on identifying infants who develop treatment-warranted disease, then intervening with laser photocoagulation or intravitreal anti-VEGF therapy. However, this approach is challenged by the geographic distribution of at-risk infants as well as a limited pool of experienced screeners. “There’s a large unmet need because we have limited access to these screening physicians,” said Dr. Moshfeghi, chief of the Retina Division at the Byers Eye Institute, Stanford University School of Medicine.

The ongoing phase 1b tROPhy-1 study is evaluating FLQ-101 (Feliqs), an investigational therapy designed to address the earliest phase of ROP development, preventing the onset of proliferative disease, explained Dr. Moshfeghi. ROP begins with a hyperoxic vaso-obliterative phase before progressing to a VEGF-driven vasoproliferative phase, which is typically when screening identifies disease requiring treatment. “FLQ-101 and other preventive strategies work by going in during the first phase, prior to screening, and preventing that from ever occurring,” explained Dr. Moshfeghi, a consultant to Feliqs.

The investigational therapy is a small-molecule lipid peroxidation inhibitor designed to interrupt oxidative stress pathways in the retina. According to the presentation, the drug blocks lipid peroxidation, supports antioxidant defenses, and protects mitochondrial function. Preclinical studies in oxygen-induced and light-induced retinopathy models have demonstrated preservation of retinal structure and function.

The ongoing phase 1b study is enrolling infants in a dose-escalation design consisting of low-, medium-, and high-dose cohorts. Investigators plan to evaluate safety as well as signals of reduced ROP development.

“We anticipate a rapid readout because the disease occurs in the 8- to 12-week window,” Dr. Moshfeghi said. The study is expected to complete enrollment later this year, with data anticipated in the fourth quarter of 2026, he said. Feliqs has already received orphan drug and fast track designations from the FDA and could advance into phase 2 testing in 2027, with a potential NDA submission projected for 2028. RP