Ocular Therapeutix, Inc. announced the release of interim 10-month data from its U.S. phase 1 clinical trial evaluating OTX-TKI for treating age-related macular degeneration (AMD), diabetic retinopathy, and other retinal diseases.
Tell me about OTX-TKI.
OTX-TKI is an investigational bioresorbable, intravitreal hydrogel implant that incorporates axitinib, which is a small-molecule tyrosine kinase inhibitor (TKI) with anti-angiogenic properties.
Talk about the trial.
The ongoing trial (NCT04989699), is a prospective, multi-center, randomized, controlled study with 21 patients who had previously been treated with anti-vascular endothelial growth factor (anti-VEGF) therapy.
Patients were randomized 3:1 to an arm receiving OTX-TKI as a 600 µg dose in a single implant followed by an administered injection of 2 mg aflibercept every four weeks, and an arm receiving only 2 mg aflibercept injected every eight weeks.
Did anything unusual happen?
Funny you should ask… one patient with the OTX-TKI arm did not receive treatment that went with the study protocol—they incorrectly received an aflibercept injection in lieu of sham at the Month 3 and 5 visits. Researchers have since removed the subject from their efficacy analysis.
What does the interim data show?
As of December 12, 2022, the 10-month data cutoff date, OTX-TKI was found to be generally well tolerated and had no drug-related ocular or system serious adverse events (SAEs).
Per previously reported data, endophthalmitis was observed as an SAE in one patient from the OTX-TKI arm, relating to the injection procedure and occuring after the Month 1 aflibercept injection.
Show me the numbers.
Patients treated with the single OTX-TKI implant exhibited stable and sustained best-corrected visual acuity (BCVA), with a mean change from baseline of -0.3 letters, and center subfield foveal thickness (CSFT), mean change from baseline of -1.3 µm, in the OTX-TKI arm at Month 10.
This was similar to the aflibercept arm, which had a mean change from BCVA baseline of -0.8 letters and a mean change from CSFT baseline of -4.5 µm.
Patients who were rescue-free at Month 7 (73%) continued to show OTX-TKI’s extended duration of action at Month 10. In totality, OTX-TKI-treated patients showed a 92% reduction in treatment burden up to 10 months.
If OTX-TKI’s data for wet AMD continues to show promising results, it could have the potential to fill the unmet need for a retinal disease treatment with an efficacy that extends beyond three to four months with a single injection.
Ocular Therapeutics expects to discuss potential future clinical trial requirements with the FDA in the near future. Pending discussion and securing financing, the company plans to initiate a wet AMD pivotal trial in Q3 of 2023.
It will also continue to follow subjects from phase 1 through their respective one-year anniversaries.