Objective:
To evaluate the efficacy and safety of ISTH0036, an antisense therapy targeting TGF-β2, in patients with neovascular age-related macular degeneration (nAMD) and diabetic macular edema (DME).
Key Findings:
- Patients showed stable or improved vision and reductions in central retinal thickness.
- Decrease in fibrotic tissue volume in nAMD patients compared to increased volume in eyes on standard anti-VEGF therapy.
- Reduction of intraretinal fluid in DME patients, both treatment-naïve and previously treated.
- Treatment was well tolerated with no reported elevation in intraocular pressure.
Interpretation:
ISTH0036 effectively targets TGF-β2, a cytokine linked to fibrosis in retinal diseases, showing promise for treating nAMD and DME.
Limitations:
- Phase 2 trial results need further validation in larger phase 2b or phase 3 studies.
- Limited geographic scope of trial may affect generalizability.
Conclusion:
The BETTER trial results support further testing of ISTH0036 in retinal diseases, with plans for regulatory discussions in the U.S. and Europe.
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