Today at the 2024 meeting of the American Society of Retina Specialists in Stockholm, Dilsher S. Dhoot, MD, presented a late-breaking interim analysis from the HELIOS study of Axpaxli (OTK-TKI; Ocular Therapeutix), a sustained-release axitinib implant. The phase 1 HELIOS study is evaluating Axpaxli vs sham control in patients with moderately severe to severe nonproliferative diabetic retinopathy (NPDR) without diabetic macular edema.
Despite research in the Protocol W study that found anti-VEGF treatment of NPDR to reduce vision-threatening complications, less than 1% of patients are treated, said Dr. Dhoot. With an aim to address this unmet need, the phase 1 trial is evaluating Axpaxli in 21 patients over 52 weeks compared to sham control, and Dr. Dhoot presented 48-week interim data. The primary outcome was safety, and secondary outcomes were efficacy based on DRSS, changes in the BCVA, changes in central subfield thickness, and proportion of patients receiving rescue therapy. Patients receiving Axpaxli had sustained or improved DRSS to week 48, with 23.1% of patients experiencing a ≥2-step improvement and 46.2% of patients experiencing a 1-step or a ≥2-step improvement. There were no vision-threatening complications or rescue therapy needed, unlike patients in the sham control arm in which 37.5% of patients developed proliferative DR or central-involved DME.
“The 48-week HELIOS phase 1 data for NPDR are compelling,” Dr. Dhoot told Retinal Physician. “Axpaxli has the potential to be safe and effective, with sustained and durable activity in the retina. Current approved anti-VEGF agents for diabetic retinopathy have established level-1 data demonstrating their benefit; however, they require frequent injections, and for that reason the usage for this indication has been limited. In NPDR, maintaining or improving patients’ vision is ultimately the goal. If this can be achieved with a 6-month to 12-month treatment, I believe this would help to shift the paradigm toward treating NPDR patients in a more proactive manner. I look forward to seeing this program expand to a registration trial so we can continue to see the potential of Axpaxli for DR.”
Axpaxli is a sustained-release implant made of a proprietary bioresorbable hydrogel that contains axitinib, a tyrosine kinase inhibitor that has a high affinity for VEGFR-2 and is highly selective for all VEGF receptors. The implant enables a dosing interval of 6 to 12 months. Axitinib also is currently being evaluated in the pivotal phase 3 SOL-1 trial for the treatment of wet age-related macular degeneration.
