Tyrosine Kinase Inhibitors and Gene Therapies Show Promise for Wet AMD Treatment
Key Insights
Tyrosine kinase inhibitors EYP-1901 and OTX-TKI have demonstrated preliminary results similar to aflibercept 2 mg in treating wet age-related macular degeneration (search).
Multiple gene therapy programs including ABBV-RGX-314, 4D-150, and ADVM-022 are currently under investigation for neovascular AMD treatment.
Vorolanib is designed for 6-month dosing intervals, potentially reducing treatment burden for wet AMD patients.
Emerging therapeutic approaches for wet age-related macular degeneration (search) (AMD) are showing promising results, with tyrosine kinase inhibitors and gene therapies advancing through clinical development to potentially transform treatment paradigms for this vision-threatening condition.
Tyrosine Kinase Inhibitors Demonstrate Comparable Efficacy
Two tyrosine kinase inhibitors have emerged as leading candidates in the wet AMD treatment landscape. EYP-1901 (vorolanib intravitreal insert, EyePoint Pharmaceuticals) and OTX-TKI, also known as Axpaxli (axitinib intravitreal implant, Ocular Therapeutix), have shown preliminary results similar to aflibercept 2 mg in current studies.
The SOL-1 and SOL-R trials are evaluating OTX-TKI in neovascular AMD, while the LUCIA and LUGANO studies are investigating vorolanib. Notably, vorolanib is designed to be delivered every 6 months to treat neovascular AMD, potentially offering a significant advantage in reducing treatment burden for patients.
"Current studies of the intravitreal implants demonstrated preliminary results similar to Eylea (aflibercept 2 mg, Regeneron)," said Dr. Esther M. Bowie, professor in the department of ophthalmology at Penn State Health, speaking at the American Academy of Ophthalmology meeting.
Gene Therapy Programs Advance Through Development
Multiple gene therapy programs are currently under investigation for wet AMD treatment, representing a potentially transformative approach to managing this condition. Three key programs are advancing through clinical development:
ABBV-RGX-314 (AbbVie (search), Regenxbio) is a subretinal gene therapy specifically designed for neovascular AMD. Additionally, two intravitreal gene therapies are being developed: 4D-150 (4D Molecular Therapeutics) and ADVM-022 (Adverum Biotechnologies), both targeting neovascular AMD through intravitreal delivery.
Clinical Outlook and Future Prospects
The convergence of these innovative therapeutic approaches suggests a potentially robust pipeline for wet AMD treatment. Dr. Arshad M. Khanani of Sierra Eye Associates in Reno, Nevada, expressed optimism about the near-term clinical landscape.
"I am very excited about the next year or two where we are going to see numerous readouts, and hopefully, many of these programs can achieve successful primary endpoints so we can have them available for our patients so we can optimize long-term vision for our patients with neovascular AMD," Khanani told Healio.
The development of these therapies comes at a time when the field already has established effective treatments. As Dr. Bowie noted, "At AAO 2025, we have great drugs that are durable, safe and effective, and we have drugs that are in the pipeline to extend and improve in what we already have."
These emerging therapies represent potential advances in both efficacy and convenience, with extended dosing intervals and novel mechanisms of action that could address current limitations in wet AMD management. The anticipated clinical readouts in the coming years will be crucial in determining which of these approaches will successfully transition from investigational status to clinical practice.