- Innovative antisense therapy blocking production of TGF-β2 shows promise in reducing retinal fibrosis and improving outcomes in patients with wet AMD and DME
Gräfelfing, Germany, May 7th, 2025 – Isarna Therapeutics presented final positive results from its Phase 2 BETTER trial at the Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting on May 6th. Isarna’s Chief Medical Officer, Prof. Marion R. Munk, shared data on ISTH0036, a selective TGF-β2-blocking antisense oligonucleotide, in patients with wet age-related macular degeneration (nAMD) and diabetic macular edema (DME). The study evaluated the potential of ISTH0036 to address retinal fibrosis, an unmet medical need not targeted by current anti-VEGF therapies.
In the international multicenter study, patients who received intravitreal injections of ISTH0036 every eight weeks (Q8W) experienced stable or improved best-corrected visual acuity (BCVA), along with meaningful anatomical improvements in the retina. All patient groups showed a reduction in central retinal thickness (CRT). Notably, eyes with nAMD and fibrosis-associated hyperreflective material (HRM) exhibited a significant decrease in HRM volume following ISTH0036 treatment, contrasting sharply with increases seen in fellow eyes receiving standard anti-VEGF therapy. In DME patients, ISTH0036 reduced intraretinal fluid volume in treatment-naïve and previously anti-VEGF–treated eyes. Intraocular pressure remained, and the treatment was well tolerated.
“The results from the BETTER trial underscore the potential of ISTH0036 as a first-in-class antifibrotic agent that directly targets TGF–β2–driven fibrosis—a key driver of disease progression in nAMD and DME,” said Prof. Marion R. Munk, CMO of Isarna Therapeutics. “This is a promising advance toward transforming care for patients facing progressive vision loss despite optimal standard therapy. Isarna’s next step will be to discuss the results and our development strategy with regulators in the US and EU to move ISTH0036 into Phase 2b/ Phase 3 pivotal clinical studies.”
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About ISTH0036 and the BETTER Trial
ISTH0036 is an investigational antisense oligonucleotide designed to selectively suppress the production of transforming growth factor beta 2 (TGF-β2), a key cytokine involved in fibrosis and disease progression in retinal pathologies. The BETTER study evaluated ISTH0036 in both treatment-naïve and anti-VEGF–pretreated but inactive patients across sites in Austria and India.
About Isarna Therapeutics
Isarna Therapeutics was built on profound expertise in antisense oligonucleotide design and the therapeutic targeting of the TGF-β signaling axis, a critical driver in a wide range of diseases. The company is advancing a pipeline of antisense compounds, with ISTH0036 in late-stage clinical development for retinal indications including wet AMD and DME.
Isarna Contact
Email: info@isarna-therapeutics.com
Media Contact
Trophic Communications
Gretchen Schweitzer or Desmond James
Phone: +49 151 678 59086
Email: isarna@trophic.de
