ISTH-0036 / Isarna Therap - LARVOL DELTA

Final results of the phase 2 BETTER trial using intravitreal ISTH0036, a selective TGF-β2 blocking antisense in nAMD and DME (ARVO 2025) - "Layman Abstract (optional): Provide a 50-200 word description of your work that non-scientists can understand. Describe the big picture and the implications of your findings, not the study itself and the associated details."

Clinical • P2 data • Cataract • Ophthalmology • TGFB1 • TGFB2

First Results of the Phase 2 BETTER Trial Using Intravitreal ISTH0036, a Selective TGF-β2 Blocking Antisense in AMD and DME (AAO 2024) - "Sixteen percent of treated eyes showed worsening of cataract. Conclusions ISTH0036 offers the potential to be a first-in-class antifibrotic agent, addressing the high unmet medical need of suppressing and treating fibrosis development in retinal and macular pathologies."

P2 data • Age-related Macular Degeneration • Cataract • Diabetic Macular Edema • Ophthalmology • Wet Age-related Macular Degeneration • TGFB1

First results of the phase 2 BETTER trial using intravitreal ISTH0036, a selective TGF-β2 blocking antisense in AMD and DME (ARVO 2024) - "ISTH0036 offers the potential to be a first in class antifibrotic agent, addressing the high unmet medical need of suppressing and treating fibrosis development in retinal and macular pathologies."

P2 data • Age-related Macular Degeneration • Cataract • Ophthalmology • TGFB1 • TGFB2

[VIRTUAL] Long-term safety and efficacy of ISTH0036 – a selective TGF-β2 blocking antisense oligonucleotide in preclinical and Phase 1 clinical studies. (ARVO 2021) - "Compared tests of ISTH0036 in rabbits and NHPs resulted in similar toxicity profile, although with higher dose-dependent sensitivity observed in Rabbits. Pronounced and long-lasting time- and dose-dependent ocular tissue distribution and target engagement in retina and VH was observed, with longer effects observed in NHPs. Aligned with biological efficacy demonstrated in preclinical models, and preliminary evidence of safety in a first-in-human study, these results strongly support the planned Phase 2 development of ISTH0036 in wAMD and DME."

P1 data • Preclinical • Cataract • Ophthalmology • Retinal Disorders • TGFB1 • TGFB2

RNA therapeutics in ophthalmology - translation to clinical trials. (PubMed, Exp Eye Res) - "This review provides a detailed insight into the recent developments and clinical trials that have been conducted for several gene-silencing therapies, including ISTH0036, SYL040012, SYL1001, PF-04523655, Sirna-027, QR-110, QR-1123, QR-421a and IONIS-FB-L in glaucoma, dry eye disease, age-related macular degeneration, diabetic macular oedema and various inherited retinal diseases. Our aim is to explore the potential of these drugs whilst evaluating their associated advantages and disadvantages, and to discuss the future translation of RNA therapeutics in ophthalmology."

Clinical • Journal • Review • Age-related Macular Degeneration • Complement-mediated Rare Disorders • Diabetic Macular Edema • Dry Age-related Macular Degeneration • Dry Eye Disease • Glaucoma • Macular Degeneration • Macular Edema • Ocular Infections • Ophthalmology • Retinal Disorders