Isarna Therapeutics has announced positive data from its international multicenter study evaluating ISTH0036, a selective TGF-β2-blocking antisense oligonucleotide, in patients with wet age-related macular degeneration (nAMD) and diabetic macular edema (DME). The study, led by Chief Medical Officer Prof. Marion R. Munk, demonstrated ISTH0036’s potential to address retinal fibrosis—an unmet medical need not effectively targeted by existing anti-VEGF therapies.
The study investigated the safety and efficacy of ISTH0036, administered through intravitreal injections every eight weeks (Q8W). Patients treated with ISTH0036 experienced stable or improved best-corrected visual acuity (BCVA), alongside meaningful anatomical improvements in the retina. All patient groups showed a reduction in central retinal thickness (CRT), indicating a positive therapeutic effect.
A key finding was observed in nAMD patients with fibrosis-associated hyperreflective material (HRM). These patients demonstrated a significant reduction in HRM volume following ISTH0036 treatment, contrasting with the increase in HRM volume in fellow eyes receiving standard anti-VEGF therapy. This outcome suggests that ISTH0036 directly targets fibrosis, offering a new therapeutic avenue for patients whose condition is not adequately managed with existing treatments.
In DME patients, ISTH0036 showed consistent benefits, reducing intraretinal fluid volume in both treatment-naïve and previously anti-VEGF-treated eyes. The results further highlighted the treatment’s favorable safety profile, with intraocular pressure remaining stable and the therapy being well tolerated across patient groups.
Commenting on the findings, Prof. Marion R. Munk stated, “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 Therapeutics plans to engage with regulators in the United States and the European Union to discuss the results and define the path forward for ISTH0036’s clinical development. The company aims to advance ISTH0036 into Phase 2b or Phase 3 pivotal clinical trials, bringing it closer to potential market approval.
If successful, ISTH0036 could become a groundbreaking therapy for nAMD and DME patients, particularly those experiencing fibrosis-related complications who do not fully benefit from conventional anti-VEGF treatments. The company’s continued focus on antifibrotic therapies reflects a strategic commitment to addressing significant unmet medical needs in ophthalmology and beyond.
Isarna Therapeutics is dedicated to developing innovative RNA-based therapies that modulate key molecular pathways involved in disease progression. The advancement of ISTH0036 highlights the company’s leadership in the field and its potential to make a meaningful impact on patient care.
