Q&A: Justis P. Ehlers, MD, on the Phase 1 HELIOS NPDR trial

(Image Credit: AdobeStock/noon@photo)

Non-proliferative diabetic retinopathy (NPDR) continues to be an area of focus for pharmaceutical innovations as patients and ophthalmologists hope for better treatment solutions for this condition. Among those treatments currently in development is OTX-TKI, a tyrosine kinase inhibitor from Ocular Therapeutix.

Justis P. Ehlers, MD, spoke to a recent post hoc analysis which looked at the impact of OTX-TKI on macular fluid metrics in patients with NPDR without center-involved diabetic macular edema. For this research, Ehlers served as the director for advanced image analysis.

Ophthalmology Times caught up with Ehlers following the meeting. In this conversation he shared the following answers to our questions on his presentation and the HELIOS trial.

Ophthalmology Times: Can you summarize the key takeaway from the presentation on the HELIOS trial at ARVO?

Justis P. Ehlers, MD: This was a post-hoc analysis of multiple imaging biomarkers from the Phase 1 HELIOS NPDR clinical trial. The key takeaways were that a single OTX-TKI injection provided sustained reduction in intraretinal fluid and quantitative leakage over the course of one year compared to sham.

OT: What is the value in having a treatment that could consistently and continuously maintain fluid for patients with DR?

Ehlers: The biggest advantage is significantly reducing the treatment burden compared to current therapies. The sustained therapeutic response over the course of one year with a single injection provides a potential new opportunity for managing DR with intravitreal pharmacotherapy.

OT: Were there any surprises or unexpected events that were seen in the trial data?

Ehlers: Previous studies have demonstrated that quantitative leakage in DR is a leading indicator of disease progression or waning therapeutic effect. The demonstration of sustained reduction of quantitative leakage on ultra-widefield imaging is an important indicator of persistent therapeutic activity at one year.

OT: What are the next steps for OTX-TKI?

Ehlers: The Phase 3 pivotal programs for OTX-TKI for wet AMD are currently fully enrolled. The next phase clinical trial for DR is currently being planned.

Previously, Ophthalmology Times reported on the HELIOS trial data that were presented at the Hawaiian Eye and Retina 2025 Meeting, in Kauai, HI in January of 2025. Diana V. Do, MD, Professor of Ophthalmology and Vice Chair for Clinical Affairs at the Byers Eye Institute, Stanford University, Los Altos, CA, and Clinic Chief of Ophthalmology at Stanford Health Care, Stanford, CA, spoke on behalf of the HELIOS investigators at this event.

Our team also spoke with Dilsher S. Dhoot, MD, with California Retina Consultants, Retina Consultants of America, in Santa Barbara, California, at the Retina World Congress 2025 in Lauderdale, Florida. At this meeting, Dhoot shared research on the pharmaceutical candidate, OTX-TKI which is being evaluated as a potential treatment for diabetic retinopathy. The presentation focused on the HELIOS trial, which demonstrated promising results for a potential new treatment approach.