NurExone Biologic Inc. has successfully completed a pivotal preclinical study of its lead therapeutic candidate ExoPTEN, demonstrating significant improvements in both motor function recovery and blood flow at spinal cord injury sites. The study represents an important milestone as the company advances toward Investigational New Drug (IND) submission and first-in-human clinical trials.
The research evaluated different dosing regimens of ExoPTEN, an exosome-based therapy designed to enhance the body's natural repair mechanisms following spinal cord injury. Both tested approaches—a single high dose administered on the day of surgery and a lower dose given over five consecutive days—showed marked therapeutic benefits.
"This preclinical study evaluated dosing regimens to provide efficacy data in support of our IND submission," said Dr. Tali Kizhner, Director of R&D at NurExone. "The results reinforce ExoPTEN's potential to enhance the body's natural repair mechanisms following spinal cord injury. Notably, the increased blood vessel size observed in treated subjects indicated improved circulation, which is crucial for oxygen and nutrient delivery to damaged tissues."
Significant Improvements in Motor Function and Vascular Recovery
The study measured functional recovery using the modified Basso, Beattie, and Bresnahan (BBB) locomotor rating scale, a standardized assessment tool for evaluating motor function in animal models of spinal cord injury. Both ExoPTEN treatment groups demonstrated significant improvements compared to the control group.
Particularly noteworthy was the histological analysis revealing that ExoPTEN treatment significantly increased the average blood vessel size at the injury site. This finding is especially important as scientific literature has established that post-injury angiogenesis and vascular remodeling strongly correlate with improved functional recovery in spinal cord injury models.
Improved circulation is considered a critical factor in post-injury healing, as it enhances oxygen and nutrient delivery to damaged tissues—essential components for neural regeneration and functional restoration.
Addressing a Significant Unmet Medical Need
Spinal cord injury represents a substantial unmet medical need with limited effective treatment options currently available. According to industry estimates, spinal cord injury constitutes a multi-billion-dollar market opportunity, alongside optic nerve injury, which is another potential indication for ExoPTEN.
The therapy leverages exosomes—naturally occurring extracellular vesicles that play crucial roles in cell-to-cell communication—loaded with PTEN (phosphatase and tensin homolog) siRNA. This approach aims to inhibit the PTEN protein, which has been identified as a significant barrier to nerve regeneration following injury.
Pathway to Clinical Development
NurExone has indicated that it will continue refining ExoPTEN's therapeutic profile as part of its ongoing preclinical program. These efforts are designed to support the company's IND submission and obtain regulatory approval for first-in-human clinical trials.
The company has already achieved important regulatory milestones, including Orphan Drug Designation, which facilitates a streamlined pathway toward clinical trials in both the United States and Europe.
"These findings suggest that ExoPTEN has the potential to become a transformative therapeutic candidate, and we are eager to advance toward clinical trials," Dr. Kizhner added.
About NurExone's Technology Platform
NurExone Biologic Inc. is developing regenerative exosome-based therapies specifically targeting central nervous system injuries. The company's proprietary technology platform enables the production of exosomes loaded with therapeutic cargo, such as siRNA, which can be delivered to injury sites using minimally invasive techniques.
Beyond spinal cord injury applications, NurExone's platform technology may offer solutions to companies interested in quality exosomes and targeted delivery systems for other indications. The company has established Exo-Top Inc., a U.S. subsidiary, to anchor its North American activities and growth strategy.
As NurExone advances ExoPTEN toward clinical development, the scientific and medical communities will be watching closely to see if this innovative approach can translate its promising preclinical results into effective treatments for patients suffering from spinal cord injuries—a condition for which new therapeutic options are urgently needed.
