NurExone Announces Promising Preclinical Results in Restoring Vision After Optic Nerve Damage

NurExone Biologic Inc., a biopharmaceutical company developing exosome-based regenerative therapies, has 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, consistently ranked one of the top 10 hospitals in the world, the study builds on previously announced preliminary results on June 28, 2024, and strengthens the suggestion of a promising treatment pathway for glaucoma, the leading cause of irreversible blindness globally.

The Optic Nerve Disorders treatment market is expected to grow from 5.54 (USD Billion) in 2023 to 11.5 (USD Billion) by 2032, at a compound annual growth rate (CAGR) of ~8.46% during the forecast period.

Researchers utilized a rodent model of optic nerve crush (ONC) to simulate the damage associated with conditions like glaucoma. After inducing injury, ExoPTEN was administrated via direct injection into the eyes. The study expanded on earlier findings which indicated that 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. Additionally, imaging results by optical coherence tomography (OCT) 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, said “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.”

NurExone Biologic Inc. is a TSX Venture Exchange (TSXV) and OTCQB listed pharmaceutical company that is developing a platform for biologically guided exosome-based therapies to be delivered, minimally invasively, to patients who have suffered Central Nervous System injuries. The Company’s first product, ExoPTEN for acute spinal cord injury, was proven to recover motor function in 75% of laboratory rats when administered intranasally. ExoPTEN has been granted Orphan Drug Designation by the US FDA and by the European Medicines Agency (EMA). The NurExone platform technology is expected to offer novel solutions to drug companies interested in minimally invasive targeted drug delivery for other indications.