Phase 2 BETTER trial results shared at ARVO 2025

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Isarna Therapeutics shared final positive results from the Phase 2 BETTER trial, which evaluated the candidate ISTH0036 to address retinal fibrosis. 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.¹

This data was presented at the Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting on May 6, 2025 by Isarna’s Chief Medical Officer, Prof. Marion R. Munk.

The BETTER study evaluated ISTH0036 in both treatment-naïve and anti-VEGF–pretreated but inactive patients across sites in Austria and India. Patients received intravitreal injections of ISTH0036 every eight weeks (Q8W). These patients 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).¹

The company noted¹ that 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.

In addition to presenting this data at the ARVO 2025 meeting in Salt Lake City, Utah, Prof. Marion R. Munk, CMO of Isarna Therapeutics is also quoted in the company’s press release¹ on this news, saying, “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. 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.”

Reference:

1. Isarna Therapeutics Presents Positive Phase 2 BETTER Trial Final Results at ARVO 2025. Isarna Therapeutics. May 7, 2025. Accessed May 7, 2025. https://www.streetinsider.com/Globe+Newswire/Isarna+Therapeutics+Presents+Positive+Phase+2+BETTER+Trial+Final+Results+at+ARVO+2025/24752465.html