Manufacturing & Production

REGENXBIO, a clinical-stage biotech company, has announced positive phase 2 data for its drug RXG-314, which was developed by the company’s NAVXpress bioreactor platform process.

RXG-314 is a potentially one-time treatment for wet AMD, diabetic retinopathy and other chronic retinal conditions. It contains the NAV AAV8 vector, which encodes an antibody fragment designed to inhibit vascular endothelial growth factor (VEGF), and is believed to inhibit the VEGF pathway, where new, leaky blood vessels contribute to the accumulation of f luid in the retina.

The pharmacodynamics, safety and efficacy of RXG-314 is being tested in the phase 2 bridging study, where 60 patients with wet AMD are given subretinal doses in two different measures (6.4x1010 GC/eye and 1.311 GC/eye). At each dose level, half the patients are given RXG-314 produced by the NAVXpress platform process, and the other half are given RXG-314 produced by the adherent cell culture manufacturing process that was used in phase 1/2a of the trial.

As of November 2022, RGX-314 was well-tolerated across 46 patients. The two high dose cohorts have completed six-month visit assessments, where it was found that target protein concentrations in the eye were similar between the manufacturing processes.

Curran Simpson, chief operating officer of REGENXBIO said, “The interim results observed in the phase 2 bridging study show a similar clinical profile between our manufacturing processes. We believe our approach, focused on early product quality and process control, allows us to efficiently transition from clinical trials to commercial readiness. This update provides validation of our plans for the NAVXpress platform process to support the production of RGX-314 in anticipation of future commercialisation.”

“There is a significant need for treatment options that can reduce the burden of frequent injections for wet AMD patients while maintaining optimal function and anatomic outcomes. The clinical profile of RGX-314 manufactured using the commercial-scale process is encouraging, as is the potential of a one-time therapy for the treatment of wet AMD,” said Dr Charles C Wykoff MD PhD, director of Research at Retina Consultants of Texas, chairman of Research, Retina Consultants of America and deputy chair of Ophthalmology for the Blanton Eye Institute, Houston Methodist Hospital.