NurExone Biologic announced significant findings from an expanded preclinical study of the potential of its portfolio drug, ExoPTEN, for repairing optic nerve damage. Conducted in collaboration with the Goldschleger Eye Institute at Sheba Medical Center, the study builds on preliminary results previously announced on June 28, 2024 and strengthens the suggestion of a promising treatment pathway for glaucoma, the company said in a press release.
Researchers utilized a rodent model of optic nerve crush (ONC) to simulate the damage associated with conditions such as glaucoma. After inducing injury, ExoPTEN was administrated via direct injection into the eyes. The study expanded on earlier findings that indicated eyes treated with ExoPTEN regained nearly normal retinal activity, as evidenced by electrical tests.
Expanded analyses of the study data showed clear recovery of signal transmission in treated eyes compared to untreated controls, which showed no significant response, the company said. Additionally, imaging results by optical coherence tomography scans indicates and validates that in all of treated eyes (naïve exosome treatment or ExoPTEN treatment) a successful ONC procedure has been performed (Figure 1).
The study also showed that ExoPTEN treatment significantly enhanced the survival of retinal ganglion cells — key neurons responsible for transmitting visual information to the brain. Detailed analysis of retinal flat-mounts confirmed this effect, with treated eyes exhibiting substantially higher counts of these cells compared to untreated or control-treated eyes (Figures 2A and 2B).
Dr. Ifat Sher, the lead investigator from the Goldschleger Eye Institute, commented, “The results from this expanded study are extremely encouraging. ExoPTEN demonstrates potential as a treatment that restores functionality and offers neuroprotection. The study shows clear signal recovery, healthier optic nerve structures and preserved retinal ganglion cells. These results suggest that ExoPTEN could fundamentally change how we approach conditions like glaucoma and optic nerve trauma. Encouraged by these results, we are advancing to a larger study with more animals to validate and expand upon these findings.”
Dr. Lior Shaltiel, CEO of NurExone, added, “these findings are an important step forward in our mission to develop groundbreaking therapies for regenerative medicine in several indications. ExoPTEN’s ability to repair both the structure and function of the optic nerve highlights its transformative potential for addressing vision loss and improving tens of millions of patient lives.”