EXOPTEN

ExoPTEN For Acute Spinal Cord Injury

Regenerate, Rewire, Recovery

The Product

Born in the labs of Israel’s Technion, ExoPTEN is NurExone’s first nanodrug. Being developed for patients who have suffered acute spinal cord injury. ExoPTEN uses exosomes loaded with a specific and proprietary sIRNA sequence as the active pharmaceutical ingredient. Studies have demonstrated that ExoPTEN facilitates nerve regeneration, regrowth, and functional recovery following a brief intranasal administration in laboratory animals.

Roadmap to Success

October 30, 2023
September 13, 2023
Expected in 2025
NurExone announced that the U.S. FDA has granted Orphan-Drug Designation (ODD) for its ExoPTEN therapy, which has great economic benefits, such as 7-12 years market protection, tax benefits, shorten approval process, average price per treatment is in range of $140,000 (!)
NurExone announced that it has completed a Pre-Investigational New Drug (Pre-IND) meeting with the U.S. FDA in connection with ExoPTEN, NurExone’s first ExoTherapy product.
In a full animal transection model, ExoPTEN was proven to recover motor function in 75% of laboratory rats when administered intranasally over a two week period. ExoPTEN has received Orphan Drug Designation from the FDA with first-in-human expected in 2025.
October 30, 2023
September 13, 2023
Expected in 2025
NurExone announced that the U.S. FDA has granted Orphan-Drug Designation (ODD) for its ExoPTEN therapy, which has great economic benefits, such as 7-12 years market protection, tax benefits, shorten approval process, average price per treatment is in range of $140,000 (!)
NurExone announced that it has completed a Pre-Investigational New Drug (Pre-IND) meeting with the U.S. FDA in connection with ExoPTEN, NurExone’s first ExoTherapy product.
In a full animal transection model, ExoPTEN was proven to recover motor function in 75% of laboratory rats when administered intranasally over a two week period. ExoPTEN has received Orphan Drug Designation from the FDA with first-in-human expected in 2025.

Control Group

Treated With ExoPTEN

Preclinical Studies

The  regeneration of neurons and motor restoration in rats has been successfully replicated across multiple experiments conducted in  NurExone’s laboratories.

These results hold significant importance particularly when considering the challenges associated with reproducibility in scientific research. They both validate our scientific approach and instill confidence in our ability to translate these findings into tangible benefits for human patients

Motor function, sensation and bladder control recovery occurred in 75% of animals following a short, intranasal ExoPTEN cycle.

See for yourself

Potential Benefits

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Competition

ExoPTEN v. Other Therapies Under Development for Acute Spinal Cord Injury

Healthcare Burden

Spinal Cord Injury

ExoPTEN is targeted at patients who have recently suffered an acute spinal cord injury, e.g., vehicle, work, sports-related injuries.

Globally, an estimated 250,000–500,000 people suffer from spinal cord injuries (SCIs) annually, with 90% of these injuries stemming from traumatic causes such as vehicle accidents, workplace incidents, or sports-related mishaps. In the United States alone, this accounts for approximately 17,000 new cases per year, while in Europe, there are around 10,000 new cases annually. This suggests a potential market for ExoPTEN of approximately 50,000 new cases per year.

The financial burden on both patients and the healthcare system is considerable. It involves immediate expenses, such as emergency surgeries, followed by prolonged rehabilitation periods. Additionally, many individuals with spinal cord injuries require ongoing financial support to accommodate various disabilities and cope with unemployment.

Spinal cord injury care – ~90% of new cases each year are from trauma

The Science of ExoPTEN

How does ExoPTEN Work?

siRNA for PTEN Inhibition

Axonal growth and functional recovery following SCI are limited, because of the poor innate regenerative capacity of adult central nervous system neurons and the hostile injury environment comprising inflammation, myelin- associated inhibitors, glial scar components and compromised blood supply.

PTEN is expressed in neurons and regenerating axons and plays a vital role in controlling the regeneration of corticospinal neurons via downregulating cytoplasmic mammalian target of rapamycin (mTOR) activity.

This mTOR pathway is inhibited in axotomized adult neurons, restricting protein synthesis to sustain axonal growth. This unique activity makes PTEN a major homeostatic regulator and tumor suppressor protein, which function is absent or defective in a wide variety of tumors as a result of somatic alterations. The important role of the PI3K/AKT/mTOR signaling pathway in cell growth, regeneration and survival supports the rationale for the therapeutic targeting of PTEN.

Among the suggested PTEN inhibition-based therapeutic targets are nerve growth and regeneration after injury or damage, treatment of cardiac ischemia/reperfusion and associated disease, wound repair, and infertility.

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