The first-in-human Phase I trial for a treatment against glaucoma core


Isarna Therapeutics has initiated Phase I clinical trial with ISTH0036, an antisense oligonucleotide selectively targeting transforming growth factor beta (TGF-ß2) to treat advanced glaucoma.

This first-in-human Phase I trial, conducted at the University Hospitals of Mainz and Tuebingen, Germany, is designed to evaluate the safety and long-term tolerability of ISTH0036 in patients with advanced glaucoma undergoing filtration surgery (trabeculectomy) due to uncontrollable elevated intraocular pressure (IOP). The trial will enroll approximately 24-30 patients, who will be treated with escalating doses of ISTH0036. In addition to safety, patients will be monitored for intraocular pressure and visual field preservation.

Eugen Leo

Prof. Eugen Leo, Isarna’s Head of Clinical Development, said: “Glaucoma is the leading cause of irreversible blindness in the world, and ISTH0036 with its expected three-directional activity, which includes blocking TGF-ß2-mediated trabecular meshwork alteration, and inhibiting both direct optic nerve toxicity and scarring post trabeculectomy, has the potential to substantially alter the course of this disease and protect the patient’s vision. Notably, ISTH0036 is currently the sole compound in clinical development worldwide that directly targets the core driver of the pathophysiology of glaucoma: TGF-ß2.

TGF-ß plays an important role in key pathways such as cell proliferation, cell differentiation, immune response and tissue modeling. Significantly elevated levels of TGF-ß have been identified in glaucomatous eyes in the anterior chamber, the vitreous, and optic nerve head. TGF-ß has been shown to directly cause increased intraocular pressure, a critical risk factor in the progression of glaucoma through complex interaction with the trabecular meshwork, leading to decreased aqueous humor outflow and has been linked to direct optic nerve toxicity.

Sir Peng Khaw
Sir Peng Khaw

Prof. Sir Peng Khaw, Professor of Glaucoma and Ocular Healing, University College London/Moorfields Eye Hospital and clinical advisor to Isarna, stated: “This novel oligonucleotide selectively targeting TGF-ß2, an important driver of glaucoma, seems to possess impressive PK-PD properties that could make it a valuable treatment for different aspects of glaucoma. I am very much looking forward to the clinical exploration of this compound.



We already met Isarna during our first Tour of Germany. Here is the video, which may give you a good idea of the company behind this trial:

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