NurExone Announces Scientific Survey Results of Proprietary Exosome-based Technology

Results show a significant increase in effectiveness of NurExone's therapeutic compounds in inhibition of PTEN expression

October 04, 2022 9:00 AM EDT | Source: NurExone Biologic Inc.

Calgary, Alberta--(Newsfile Corp. - October 4, 2022) - NurExone Biologic Inc. (TSXV: NRX) (FSE: J90) (the "Company" or "NurExone"), a biopharmaceutical company developing biologically-guided exosome therapy ("ExoTherapy") for patients with traumatic spinal cord injuries, announced today results from a controlled survey in the Company's lab showing the effectiveness of the Company's proprietary small interfering RNA (siRNA) in inhibiting the expression of the phosphatase and tensin homolog (PTEN). Inhibition of PTEN promotes axon regeneration and neural repair[1], key elements for healing after a traumatic spinal cord injury.

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Figure 1

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Figure 1: qPCR analysis of PTEN in HEK 293 cells 72 hours post-transfection with siRNA molecules (10 nM). 

  • POC siRNA (n=5), is a sequence commercially purchased that contains a loading moiety that was used in POC experiments of the ExoTherapy (Guo et al., 2019). 
  • NUR001, NUR002, and NUR003 are proprietary siRNA sequences of NurExone with different chemical modifications and moieties. They significantly reduce PTEN gene expression at the RNA level (n=4).
  • NTC- No template control. Negative control
    **** Significance was determined by one-way ANOVA (p<0.0001)
    *** Significance was determined by one-way ANOVA (p<0.001)

A survey of multiple samples of siRNA from various sources with different chemical compositions (Fig. 1) was carried out in 2 separate experiments in the Company's labs to assess the effect of the siRNA on inhibition of PTEN in a cell culture (HEK 293). The survey results indicated that transfection of the cells with the Company's proprietary siRNA sequences achieved statistically significant PTEN inhibition in RNA (Fig. 1) and protein level compared to a commercial siRNA sequence that was used in the academic pre-clinical proof of concept (POC) studies on animals.

"Our proprietary siRNA sequences were shown to achieve knock-down of the relative expression of PTEN to a level of only 20-29% compared to the untreated negative control (cells only) and compared to 66% with a commercial sequence," said Dr. Lyora Aharonov, Director of Research and Development at NurExone. "This indicates that our proprietary siRNA caused an absolute reduction in PTEN expression of 80% and, moreover, was 2x as effective in PTEN inhibition than the commercial sequence that was used in the landmark pre-clinical study of ExoTherapy in which motor function was restored to rats."

The Company's proprietary siRNA sequences (NUR001, NUR002, and NUR003) were developed by NurExone's expert team of biologists and chemists as therapeutic "cargo" to be carried by exosomes to inflamed tissues to promote a healing environment. The compositions and the chemical modifications of these particular sequences are among more than 20 siRNA sequences that inhibit PTEN, described in a provisional patent filed in May 2022, as part of the Company's expanded IP assets.

For traumatic spinal cord injury patients, NurExone's ExoTherapy uses proprietary exosomes as biologically-guided nanocarriers to deliver specialized siRNA therapeutic compounds to target areas. The delivered molecules promote an environment that induces a healing process at the target location.

Lior Shaltiel, CEO of Nurexone, added, "Congratulations to our talented Research and Development team for successfully achieving effective siRNA sequences that are expected to serve as part of our ExoTherapy treatment for a market of thousands of patients who suffer traumatic spinal cord injuries each year[2]. Developing effective therapeutic compounds is a core technological building block for us, and I am very pleased to note this advance."

About NurExone Biologic Inc.

NurExone Biologic Inc. is a TSXV listed pharmaceutical company that is developing a platform for biologically-guided ExoTherapy to be delivered, non-invasively, to patients who suffered traumatic spinal cord injuries. ExoTherapy was conceptually demonstrated in animal studies at the Technion, Israel Institute of Technology. NurExone is translating the treatment to humans, and the company holds an exclusive worldwide license from the Technion and Tel Aviv University for the development and commercialization of the technology.

For additional information, please visit www.nurexone.com or follow NurExone on LinkedIn, Twitter, Facebook, or YouTube.

For more information, please contact:

Dr. Lior Shaltiel
Chief Executive Officer and Director
Phone: +972-52-4803034
Email: lior@nurexone.com

For investors:
Investor Relations
IR@nurexone.com
+1 905-347-5569

FORWARD-LOOKING STATEMENTS

This press release contains certain forward-looking statements, including statements about the Company's future plans and intellectual property, the scientific and development activities to be carried out by the company, future potential manufacturing and marketing activities and the treatment of certain conditions. Wherever possible, words such as "may", "will", "should", "could", "expect", "plan", "intend", "anticipate", "believe", "estimate", "predict" or "potential" or the negative or other variations of these words, or similar words or phrases, have been used to identify these forward-looking statements. These statements reflect management's current beliefs and are based on information currently available to management as at the date hereof. Forward-looking statements involve significant risk, uncertainties and assumptions. Many factors could cause actual results, performance or achievements to differ materially from the results discussed or implied in the forward-looking statements. These risks and uncertainties include, but are not limited to, risks related to the Company's early stage of development, lack of revenues to date, government regulation, market acceptance for its products, rapid technological change, dependence on key personnel, protection of the Company's intellectual property and dependence on the Company's strategic partners. These factors should be considered carefully and readers should not place undue reliance on the forward-looking statements. Although the forward-looking statements contained in this press release are based upon what management believes to be reasonable assumptions, the Company cannot assure readers that actual results will be consistent with these forward-looking statements. These forward-looking statements are made as of the date of this press release, and the Company assumes no obligation to update or revise them to reflect new events or circumstances, except as required by law.

Neither TSX Venture Exchange nor its Regulation Services Provider (as that term is defined in the policies of the TSX Venture Exchange) accepts responsibility for the adequacy or accuracy of this release.

  1. Yosuke Ohtake, Umar Hayat, and Shuxin Li, M.D., Ph.D., PTEN Inhibition and Axon Regeneration and Neural Repair, Neural Regen Res. 2015 Sep; 10(9): 1363-1368, NIH Library of Medicine
  2. World Health Organization, Spinal Injury Fact Sheet, Incidence of spinal cord injuries

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