ISTH0036 is a locked nucleic acid-modified antisense oligonucleotide selectively targeting the messenger RNA (mRNA) of TGF-β2. Preclinical studies have demonstrated that ISTH0036 is highly potent and shows selective target engagement (TGF-β2 mRNA and protein downregulation) consistent with long-lasting tissue uptake and pharmacodynamic effects.
Isarna and its collaboration partners presented Phase I safety and efficacy data in advanced-stage glaucoma patients undergoing trabeculectomy for ISTH0036 at The Annual Meeting of the Association for Research in Vision and Ophthalmology (ARVO) in 2017. Overall, the treatment was safe and well tolerated at all dose levels and a trend in post-operative intraocular pressure (IOP) analysis provided preliminary evidence for a beneficial effect of ISTH0036 at the two highest dose levels evaluated.
Based on the promising clinical and preclinical data package obtained, Isarna is currently initiating a Phase 2a clinical trial in neovascular age-related macular degeneration (nAMD) and diabetic macular edema (DME). More information will be provided in due time on our website and on www.clinicaltrials.gov.
ISTH0036 is a compound undergoing clinical development. ISTH0036’s use is restricted to patients enrolled in Isarna-sponsored clinical studies. The compound is not available for any other usage.