HELIOS-3: Investigating Axpaxli for Diabetic Retinopathy

Peter K. Kaiser, MD, FASRS, discusses the phase 3 HELIOS-3 trial of AXPAXLI for diabetic retinopathy, which is enrolling patients in Argentina, the United States, and other countries.

By: Patricio G. Schlottmann, MD, Peter K. Kaiser, MD

Retinal Physician November 10, 2026

Clinical Scorecard: HELIOS-3: Investigating Axpaxli for Diabetic Retinopathy

At a Glance

Category	Detail
Condition	Diabetic retinopathy (DR); the interview identifies proliferative DR and center-involved diabetic macular edema (DME) as vision-threatening complications in an earlier study.
Key Mechanisms	The interview does not specify OTX-TKI (Axpaxli)'s mechanism of action.
Target Population	Patients with diabetic retinopathy enrolled in the HELIOS-3 phase 3 trial; specific eligibility criteria are not provided in the interview.
Care Setting	The interview says HELIOS-3 is recruiting in Argentina, the United States, and other countries.

Key Highlights

HELIOS-3 is evaluating OTX-TKI (Axpaxli) for diabetic retinopathy.
The described study design includes injections at baseline and 48 weeks, with comparison against sham.
The trial will follow patients for development of complications and changes in retinopathy, including improvement or worsening.
Dr. Kaiser reports that in earlier studies, patients in a clinical study were stable or improved over one year; the interview does not clearly specify the treatment arm for this statement.
Dr. Kaiser reports that 40% of sham-treated patients in an earlier study developed vision-threatening complications—proliferative DR or center-involved DME—compared with none in the OTX-TKI arm.

Guideline-Based Recommendations

Diagnosis

Management

The interview describes OTX-TKI as being investigated in HELIOS-3 and does not provide a recommendation for routine clinical care.

Monitoring & Follow-up

In HELIOS-3, participants are followed for development of complications and changes in retinopathy.

Risks

Dr. Kaiser identifies proliferative DR and center-involved DME as vision-threatening complications observed in the earlier study's sham group.

Patient & Prescribing Data

Patients with diabetic retinopathy enrolled in the HELIOS-3 clinical trial; specific eligibility criteria are not provided in the interview.

The described trial design includes OTX-TKI injections at baseline and 48 weeks, with a sham comparison. The interview does not provide prescribing information or state that OTX-TKI is approved for DR.