New ExoPTEN Preclinical Study Indicates Significant Improvement in Walking Quality in Spinal Cord Injury Model

Medium and high doses improved movement quality in up to 100% of the animals in a dose-dependent manner

(“NurExone” or the “Company”) is pleased to announce new preclinical results demonstrating that 100% of small animals treated with a higher dose of ExoPTEN regained motor function after spinal cord injury. The results of the preliminary, dose-ranging study were confirmed using precise measurements using the CatWalk XT system.

Using the CatWalk XT system, researchers assessed ExoPTEN’s effect on the animals’ ability to walk. All animals (100%) in the higher-dose group demonstrated measurable gait recovery, in contrast to one animal in the untreated group which exhibited minimal stepping.

“This is a significant milestone for our program,” said Dr. Tali Kizhner, Director of Research and Development at NurExone. “Seeing the animals regain the ability to walk, with measurable improvement in locomotion function, is incredibly exciting. The CatWalk XT provided us with objective data that strengthens the scientific foundation for ExoPTEN’s potential to restore function after an acute spinal cord injury.”

In the study, researchers compared medium and high single doses of ExoPTEN, administered minimally-invasively on the day of spinal cord compression surgery, to a control group that received injection of the vehicle only. Medium and high doses used in this study refer to escalating dose levels used to explore potential therapeutic effects and tolerability in animals.

The treatment demonstrated a dose-dependent effect, with 100% of animals in the high-dose group regaining walking ability in both hind limbs, compared to 50% in the medium-dose group, and only 1 out of 6 rats in the untreated control group (Figure1 A-B).

The gait analysis data also showed dose-dependent improvement in walking function. Animals treated with higher dose of ExoPTEN displayed larger paw print areas (Fig. 1C), greater maximal contact area of their hind paws (Fig. 1D), a wider base of support (Fig. 1E), and an extended duration of the paw contact with the walkway (Fig. 1F). These indicators reflect improved balance, strength, coordination and weight bearing during walking.

Evaluation of additional study parameters is ongoing. Notably, the high dose was well tolerated, with no observed side effects. As part of this ongoing work, the Company plans to initiate additional studies to explore alternative dosing regimens, while also advancing the optimization of ExoPTEN’s manufacturing processes and analytical methods. These efforts aim to refine the drug’s therapeutic profile and facilitate engagement with regulatory authorities.

The CatWalk XT system, developed by Noldus Information Technology, is widely considered a leading tool for studying animal movement1. It uses an illuminated glass walkway to capture footprints and movement patterns, allowing researchers to collect precise, objective data on an animal’s motor function.

NurExone continues to advance its research and development efforts, optimizing ExoPTEN’s dosing strategies and manufacturing processes, and preparing for regulatory submissions as it aims to launch first-in-human clinical trials. The Company remains committed to developing treatments that bring new hope to people who suffer nervous system injuries.