Ocular Therapeutix announced positive top-line results from the phase 1 HELIOS study evaluating Axpaxli vs a sham control in patients with moderately severe to severe nonproliferative DR without DME. Overall, Axpaxli demonstrated promising safety and efficacy outcomes. Axpaxli exhibited good tolerability without any observed inflammation, including no instances of iritis, vitritis, or vasculitis. In terms of efficacy, 46.2% of patients in the Axpaxli group experienced a 1-step or 2-step improvement in the Diabetic Retinopathy Severity Scale (DRSS) at the 40-week mark, with 2 of those patients showing a 2-step improvement. Conversely, none of the control group patients demonstrated such improvements. Moreover, no patients in the Axpaxli group experienced any worsening in DRSS, while only 1 patient in the control group exhibited worsening at the same time point. Notably, the effect of a single Axpaxli injection endured up to 40 weeks without the need for rescue medication. These encouraging findings have prompted plans to progress directly to a phase 3 study for Axpaxli in patients with NPDR, pending discussions with the FDA. 

“In an effort to get Axpaxli to patients as quickly as possible, we unmasked this study, and based on the observed safety, efficacy, and durable treatment effect, we have decided to accelerate the DR program to phase 3,” Jeffrey S. Heier, MD, chief scientific officer of Ocular Therapeutix, said in a news release.