Get to Know ExoTherapy and ExoPTEN One of the key assets of NurExone's technology is its drug delivery system, branded ExoTherapy. ExoTherapy is a play on the combination of exosomes with therapeutic compounds. The first ExoTherapy product, which uses modified siRNA as its therapeutic molecule, is called ExoPTEN, which is designed to treat acute spinal cord injuries and has shown promising initial results in preclinical studies for neuron regeneration in rat models.
While very early in development, the data from an internal preclinical study of ExoPTEN were very positive and inspiring. In short, the company used the ExoTherapy platform to produce and load exosomes with pharmaceutical cargo targeting damaged tissue at the core of SCI. Healthy rats with full mobility underwent a complete transection of the spinal cord, rendering their rear legs paralyzed. The rats were then treated with exosomes only (control group) or with intranasal administration of ExoPTEN. NurExone reported positive imaging results visualizing successful nerve regeneration following traumatic full transection of the spinal cord in rats.
These imaging results align with previous functional results showing a remarkable recovery of motor function in 75% of paralyzed rats suffering from spinal cord injuries that were treated with the new therapy. In other words, rats treated with ExoPTEN experienced restored functionality of their legs including sensorial recovery. These results align with previous Proof of Concept experiments (see video here: https://nurexone.com/study-results/)
The study indicates that ExoPTEN therapy enables new nerve-connections to be made, effectively repairing the transected spinal cord, at least partially. For humans, the research suggests that ExoTherapy may facilitate even a partial recovery, providing patients with a better outcome than had been possible to date. NurExone is aiming for filing an investigational new drug (IND) application during 2024 to initiate clinical trials.
ExoPTEN is differentiated with some competitive advantages. First, it can be administered non-invasively (intranasal). Next, the treatment is off-the-shelf and not personalized, which conquers some of the logistic headaches involved with autologous (derived from patient) therapies. Further, being a novel therapeutic targeting a patient population under 200,000 people, ExoPTEN may be an ideal candidate for an Orphan Drug designation, which expedites development and provides financial incentives to develop a new drug.
According to Grand View Research, the global market for acute spinal cord injury will reach $2.9 billion by 2027.
As for the other business silo, licensing the proprietary exosome production and loading platform to biopharmaceutical companies for a litany of diseases and conditions, Grand View Research estimates the global market for exosome-based diagnostics and therapeutics to reach $2.3 billion by 2030. Broadly, Grand View’s analysts see the global exosomes market size as valued at $112.25 million in 2022 and anticipate it to expand at a compound annual growth rate (CAGR) of 32.75% by 2030. | |