German biotechnology company Isarna Therapeutics has reported favorable results from its phase 2 BETTER trial of ISTH0036, an experimental antisense therapy targeting transforming growth factor beta-2 (TGF-β2), in patients with neovascular age-related macular degeneration (nAMD) and diabetic macular edema (DME). The data were presented May 6 at the Association for Research in Vision and Ophthalmology (ARVO) annual meeting in Salt Lake City, Utah.

In the international trial, conducted in Austria and India, patients receiving ISTH0036 injections every 8 weeks showed stable or improved vision and reductions in central retinal thickness. Eyes with nAMD and fibrosis-associated hyperreflective material demonstrated a decrease in fibrotic tissue volume, whereas fellow eyes on standard anti-VEGF therapy showed an increase. In DME patients, the drug reduced intraretinal fluid in both treatment-naïve and previously treated eyes.

The treatment was well tolerated, with no elevation in intraocular pressure reported.

ISTH0036 is designed to suppress production of TGF-β2, a cytokine associated with fibrosis in retinal disease. Current therapies for nAMD and DME do not target this pathway directly. Isarna plans to discuss regulatory pathways for phase 2b or phase 3 studies in the United States and Europe. RP