Regenxbio Announces Dosing of First Patient in Phase 2 ALTITUDE Trial of RGX-314 for the Treatment of DR

Regenxbio announced that the first patient has been dosed in ALTITUDE, a phase 2 trial to evaluate the suprachoroidal delivery of RGX-314 using the SCS Microinjector for the treatment of diabetic retinopathy (DR).

“We are pleased to announce the first patient dosed in our ALTITUDE trial, an important milestone as we continue to evaluate the overall clinical profile of RGX-314 for the treatment of chronic retinal conditions. This is our second phase 2 trial using the in-office suprachoroidal delivery approach, which may allow physicians to treat patients with diseases like DR earlier in the disease course,” Steve Pakola, MD, Chief Medical Officer of Regenxbio, said in a company news release. “Long-term treatment with anti-VEGF injections has been shown to significantly reduce disease progression and severity of DR, even in patients who are asymptomatic, as well as prevent vision threatening complications. We believe that one-time treatment with anti-VEGF gene therapy can have a meaningful impact for patients with DR, and we look forward to providing additional updates from this trial next year.”

“The SCS Microinjector is designed to provide targeted delivery of the gene therapy to the suprachoroidal space, with broad distribution to the back of the eye and into the retina through a one-time, in-office procedure, which could be an important alternative to current standard of care,” said Charles Wykoff, MD, PhD, trial investigator, Retina Specialist and Director of Research with Retina Consultants of Houston.

“DR is the most common cause of vision loss among patients with diabetes, with an average age of disease onset between 45-50 years old. Anti-VEGF injections have been approved by the FDA for diabetic retinopathy, but treatment with anti-VEGF agents requires frequent clinic visits and injections. RGX-314 may provide sustainable, long-term anti-VEGF protein production in the eye, which could be a one-time treatment option for working-age adults,” said Arshad M. Khanani, MD, MA, trial investigator and director of clinical research at Sierra Eye Associates.

ALTITUDE is a multicenter, open label, randomized, controlled dose-escalation trial that will evaluate the efficacy, safety and tolerability of suprachoroidal delivery of RGX-314. The trial is expected to enroll approximately 40 patients with DR across two cohorts. Patients will be randomized to receive RGX-314 versus observational control at a 3:1 ratio, and two dose levels of RGX-314 will be evaluated: 2.5×10¹¹ GC/eye and 5.0×10¹¹ GC/eye. Patients will not receive prophylactic immune suppressive corticosteroid therapy before or after administration of RGX-314.

The primary endpoint of the trial is the proportion of patients that improve in DR severity based on the Early Treatment Diabetic Retinopathy Study-Diabetic Retinopathy Severity Scale (ETDRS-DRSS) at 48 weeks. Other endpoints include safety and development of DR-related ocular complications.

The company expects to report initial data from this trial in 2021.