Date: 2015-05-11
Type of information: Presentation of results at a congress
phase: preclinical
Announcement: presentation of results at the Annual Meeting of the Association for Research in Vision and Ophthalmology (ARVO) held May 3-7 in Denver, Colorado.
Company: Isarna Therapeutics (Germany)
Product: ISTH0036 - antisense oligonucleotide directed against TGF-ß2 mRNA
Action
mechanism: antisense drug/oligonucleotide. ISTH0036 is a locked nucleic acid-modified antisense oligonucleotide selectively targeting the messenger ribonucleic acid (mRNA) of TGF-β2. TGF-ß (transforming growth factor beta) plays an important role in key pathways such as cell proliferation, cell differentiation, immune response and tissue modeling. Preclinical studies have demonstrated that ISTH0036 is highly potent and shows selective target engagement (TGF-ß2 mRNA and protein downregulation) consistent with long-lasting tissue uptake and pharmacodynamic effects.
Disease: glaucoma
Therapeutic area: Ophtalmological diseases
Country:
Trial details:
Latest
news: * On May 11, 2015, Isarna Therapeutics announced the presentation of preclinical data for its lead candidate ISTH0036, a locked nucleic acid-modified antisense oligonucleotide, currently in phase I development for the treatment of advanced-stage glaucoma. Isarna presented supporting preclinical results in one podium presentation and two poster publications at the Annual Meeting of the Association for Research in Vision and Ophthalmology (ARVO) held May 3-7 in Denver, Colorado. As described in the podium presentation, ISTH0036 was administered to evaluate its therapeutic potential in murine models of glaucoma filtration surgery (GFS) and laser-induced choroidal neovascularization (CNV). In the murine GFS model, upon intraocular administration, ISTH0036 was able to significantly prolong bleb survival, as compared to control oligonucleotide- and saline-treated eyes. In addition, ISTH0036 was able to significantly decrease the extent of fibrosis in the bleb area in a sequence-specific manner. Furthermore, in a murine CNV model, intravitreal administration of ISTH0036 was able to significantly reduce (40%) the process of angiogenesis, as compared to saline- and control oligonucleotide-treated eyes. This observation may open up development opportunities beyond glaucoma. Also presented at the conference, two posters described the overall preclinical profile and the testing of ISTH0036 in which it successfully demonstrated excellent cellular uptake, potent TGF-ß2 mRNA downregulation (target engagement) in cell-based assays and in relevant tissues of the eye. Long-lasting tissue distribution was consistent with the observed target engagement.
