Phase 2 study of potential DME oral therapy meets primary endpoints
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Key takeaways:
- RZ402 reduced central subfield thickness at all dose levels.
- The safety profile was good, with generally mild adverse events.
A phase 2 clinical study of RZ402, a potential oral therapy for diabetic macular edema, met its primary endpoints of safety and reduction in central subfield thickness, according to a press release from Rezolute.
“As a field, we have been evaluating noninvasive treatment options (eye drops and oral pills) to address DME early and reduce treatment burden,” primary investigator Arshad M. Khanani, MD, MA, FASRS, told Healio. “Importantly, an oral therapy would afford us the opportunity to address both eyes and intervene much earlier, potentially altering the long-term prognosis for our patients with DME.”
The multicenter, randomized, double-masked, placebo-controlled, parallel-arm, proof-of-concept study enrolled 94 DME participants with mild to moderate nonproliferative diabetic retinopathy who had received no more than three anti-VEGF injections previously. Central subfield thickness (CST) was 320 µm or greater in men and 305 µm or greater in women. All participants had best corrected visual acuity of 78 letters or less on ETDRS assessment.
Image: Adobe Stock
Participants were randomly assigned equally to receive active treatment with RZ402 50 mg, 200 mg or 400 mg or placebo once daily for 12 weeks.
There was an improvement in CST up to approximately 50 µm at all RZ402 dose levels compared with placebo (P = .02). The 200 mg dose had the largest response, but there was no significant difference between the three dose levels. More than 20% of participants who received the 200 mg dose had clinically significant improvements from baseline compared with none who received placebo.
The safety profile of RZ402 was good, with generally mild adverse events at rates comparable with placebo. Three serious adverse events were deemed to be unrelated to RZ402.
In other endpoints, 20% of participants who received the 200 mg dose experienced a one-step improvement in Diabetic Retinopathy Severity Scale compared with one participant who received placebo. There were no significant improvements in BCVA in treated participants compared with placebo.
“The recent results from the phase 2 proof-of-concept study evaluating safety and anatomic benefit of RZ402 in DME are encouraging,” Khanani said. “The study met both the primary endpoints of change in macular edema (CST) and safety, as treatment with RZ402 was safe and resulted in an anatomic benefit as seen by a decrease in CST. This was an early phase 2 study and was conducted with patients in the study receiving the drug for 3 months. We need a longer-term study to see the effect of RZ402 on CST and especially best corrected visual acuity, since the latter is a registration endpoint.”
Editor’s note: This article was updated on May 28, 2024, to add comments from Arshad M. Khanani, MD, MA, FASRS.
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