Topline results from investigational nAMD drop SYL1801 (Sylentis)

Sylentis presented results from the phase 2a study in a poster at the Association for Research in Vision and Ophthalmology meeting. Image credit: ©Gorodenkoff – stock.adobe.com

Sylentis recently announced topline results from the SYL1801_II phase 2a study investigating SYL1801 for the treatment of neovascular age-related macular degeneration (nAMD).¹ SYL1801 is an investigational small interfering RNA (siRNA) therapy administered via eye drops, rather than intravitreal injection, to treat nAMD. Sylentis, based in Madrid, Spain, said the primary endpoint was reached and more than 71% of patients in the mid-dose level maintained visual acuity over the 6-week study period.

"With these data from this explorative study of SYL1801, we are encouraged to put efforts into the subsequent clinical research program with proper guidance from the patient outcomes in the concluded study,” said Andreas Segerros, CEO of Sylentis.

Three parallel dose level cohorts of SYL1801 were evaluated in a double-blind study. A total of 99 subjects with treatment-naïve eyes of nAMD were followed for 42 days. The primary endpoint was to evaluate the effect of SYL1801 on the chance from baseline to the end of treatment in best corrected visual acuity (BCVA) score, according to the company’s press release. Throughout the study’s duration, it was found that all dose-levels of SYL1801 maintained visual acuity similarly, with the middle concentration of 25mg/mh achieving a statistically significant improvement of visual acuity at 42 days.

The company presented these results in a poster titled “AMD, Clinical Research,” at the 2025 Association for Research in Vision and Ophthalmology (ARVO) annual meeting held 4-8 May in Salt Lake City.

Reference

1. Sylentis, a PharmaMar Group company, announces the positive results of the Phase IIa dose-ranging study for SYL1801 in patients with Age-Related Macular Degeneration (AMD) – Sylentis. Sylentis.com. Published 2025. Accessed May 15, 2025. https://sylentis.com/sylentis-a-pharmamar-group-company-announces-the-positive-results-of-the-phase-iia-dose-ranging-study-for-syl1801-in-patients-with-age-related-macular-degeneration-amd/