Abstract
Purpose :
While intravitreal (IVT) therapy remains the standard-of-care (SOC) for retinal diseases, such as neovascular macular degeneration (nAMD), diabetic macular edema (DME), heavy treatment burden limits their real-world utility. TO-O-1002, our novel topical formulation designed to deliver a small-molecule tyrosine kinase inhibitor as a noninvasive option for the treatment of retinal diseases, was investigated in a Phase IIa study in patients with nAMD.
Methods :
The Phase IIa study (NCT05390840) is open-label, monotherapy design that enrolled 24 subjects. Naïve or washed-out (at least 2 months after last IVT injection) patients received TO-O-1002 (0.8%) eyedrop TID for 12 weeks. Central macular thickness (CST) and best-corrected visual acuity (BCVA) were measured for study eyes at pre-dose and every month post-dose. Rescue-free proportion and time-to-rescue were also recorded. In addition, a compassionate program (CP) was available for patients with options of receiving either SOC or TO-O-1002 treatment for up to an additional 6 months.
Results :
TO-O-1002 was safe and well-tolerated with no severe drug-related ocular or systemic adverse event (AE) observed. Only 14% patients required additional IVT therapy at the end of 12 weeks. At the end of study, 33% of patients opted to stay on TO-O-1002 treatment for additional 6 months. During the 6-month extended use, 75% of patients did not receive additional IVT. No severe drug-related ocular or systemic AE were reported during the CP.
Conclusions :
This study demonstrated the potential of TO-O-1002 as a noninvasive option for the treatment of nAMD. Rescue-free data indicated that 86% patients unnecessary to receive IVT within 12 weeks of IP treatment, and most patients express more willingness to keep using study eye drops for long-term treatment. TO-O-1002 also allows patients to self-administer at home, which greatly reduces the burden placed on care-givers. Additional studies are being planned to investigate TO-O-1002 in nAMD, DME, and diabetic retinopathy (DR).
This abstract was presented at the 2024 ARVO Annual Meeting, held in Seattle, WA, May 5-9, 2024.