NurExone Announces Clinical Trial Plans for Acute Spinal Cord Injury Therapy at Annual Meeting of American Spinal Injury Association

Rhea-AI Summary

NurExone Biologic announced its plans to initiate Phase 1/2a clinical trials in 2026 for ExoPTEN, their innovative exosome-based therapy for acute spinal cord injuries. Professor Nahshon Knoller, senior clinical advisor and former Director at Sheba Medical Center, will present the trial plans and preclinical results at the American Spinal Injury Association annual meeting. The Phase 1 trial will evaluate safety in up to 18 patients, followed by a Phase 2a randomized, double-blind, placebo-controlled trial with 10-15 patients. ExoPTEN, which has received Orphan Drug Designation from both FDA and EMA, uses mesenchymal stem cell-derived exosomes loaded with PTEN-targeting siRNA to promote neural regeneration. Preclinical studies have demonstrated significant recovery in motor and sensory function in rat models.

Positive

• ExoPTEN has received Orphan Drug Designation from both FDA and EMA
• Preclinical studies showed robust recovery of motor, sensory, and structural function in rat models
• Company advancing to Phase 1/2a clinical trials in 2026, marking transition from preclinical stage
• Therapy demonstrates effective targeting with exosomes homing to injury site for up to seven days

Negative

• Clinical trials won't begin until 2026, indicating a long path to potential commercialization
• Limited initial trial size with only up to 18 patients in Phase 1
• Early-stage company with no approved products or revenue yet

TORONTO and HAIFA, Israel, May 30, 2025 (GLOBE NEWSWIRE) -- NurExone Biologic Inc. (TSXV: NRX) (OTCQB: NRXBF) (FSE: J90) (“NurExone” or the “Company”) is pleased to announce that Professor Nahshon Knoller M.D., senior clinical advisor to the Company, will be presenting at the prestigious annual meeting of the American Spinal Injury Association (“ASIA”), where he will discuss the Company’s plans for future clinical trials in 2026 in the field of acute spinal cord injuries (“SCI”) for ExoPTEN, a first-in-class exosome-based therapy, as well as results from the Company’s preclinical studies.

NurExone expects to initiate a Phase 1/2a clinical trial in 2026 (“2026 SCI Trial”) marking a major milestone in the advancement of regenerative therapies for central nervous system injuries.

Adult patients with traumatic SCI between spinal levels C5 and T10, classified as ASIA Grade A or B, will be enrolled in the 2026 SCI Trial. These patients will be treated within 3 to 7 days post-injury. Phase 1 will be a dose-escalation study evaluating safety in up to 18 patients, followed by a Phase 2a randomized, double-blind, placebo-controlled trial measuring functional recovery in 10–15 patients.

At the 2025 ASIA meeting, Professor Knoller, a renowned neurosurgeon and former Director of the Spinal Trauma Unit at Sheba Medical Center, the Middle East’s largest hospital and ranked by Newsweek as one of the ten leading hospitals in the world, will provide updates on the 2026 SCI Trial, as well as results from the Company’s other preclinical studies.

Professor Knoller commented: “it is a real honor for a preclinical-stage company to be invited to present at ASIA. This is one of the most respected gatherings in the SCI field, and our inclusion highlights the strength and relevance of the ExoPTEN program and the impressive results we have obtained as a minimally invasive approach to treat SCI. Moreover, it reflects the scientific quality and the clinical need behind what NurExone is advancing.”.

Dr. Lior Shaltiel, CEO of NurExone, highlighting the significance of the Company’s transition into clinical trials stated that: “this is the first time we are publicly outlining our clinical plans for ExoPTEN, and it is an important step forward for the Company. We’ve made meaningful progress in validating our science and building the foundation for clinical readiness. As we prepare for first-in-human studies, we remain focused on advancing this program and with a clear view of its potential to address a critical unmet need in spinal cord injury.”

ExoPTEN is based on mesenchymal stem cell-derived exosomes loaded with siRNA targeting PTEN, a molecular inhibitor of neural regeneration through the mTOR pathway. Delivered via intranasal and intrathecal routes, ExoPTEN is designed to reduce cell death and promote axonal regrowth in the acute post-injury phase. The therapy has been granted Orphan Drug Designation by both the United States Food and Drug Administration and European Medicines Agency.

Preclinical studies to be presented by Professor Knoller at ASIA showed robust recovery of motor, sensory, and structural function in rat models of complete spinal cord transection and compression. MRI, BBB scoring, von Frey testing, and histology have confirmed the therapeutic effects of ExoPTEN. Fluorescent labeling has shown that exosomes effectively homed to the injury site for up to seven days post-injury. Being invited to present these findings at ASIA underscores the Company’s emergence as an innovator in neuroregeneration.

The 2025 ASIA meeting will be held on June 2-4, 2025 in Scottsdale, Arizona. This meeting is the leading clinical and academic conference dedicated to SCI care and research.

Investor Summit Virtual – June 10, 2025

NurExone Biologic Inc. will be participating in the Q2 Investor Summit Virtual on June 10, 2025, with a presentation scheduled for 2:00 PM EDT. The Investor Summit is an exclusive event focused on connecting investors with promising microcap companies that are poised for growth. Investors will have the opportunity to engage directly with company leadership and hear from industry experts. To register for the complementary event, please visit the website https://investorsummitgroup.com and click on “Registration.”

About NurExone

NurExone Biologic Inc. is a TSX Venture Exchange (“TSXV”), OTCQB, and Frankfurt-listed biotech company focused on developing regenerative exosome-based therapies for central nervous system injuries. Its lead product, ExoPTEN, has demonstrated strong preclinical data supporting clinical potential in treating acute spinal cord and optic nerve injury, both multi-billion-dollar markets ⁱ . Regulatory milestones, including obtaining the Orphan Drug Designation, facilitates the roadmap towards clinical trials in the U.S. and Europe. Commercially, the Company is expected to offer solutions to companies interested in quality exosomes and minimally invasive targeted delivery systems for other indications. NurExone has established Exo-Top Inc., a U.S. subsidiary, to anchor its North American activity and growth strategy.

For additional information and a brief interview, please watch Who is NurExone?, 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: info@nurexone.com

Dr. Eva Reuter
Investor Relations – Germany
Phone: +49-69-1532-5857
Email: e.reuter@dr-reuter.eu

Allele Capital Partners
Investor Relations – U.S.
Phone: +1 978-857-5075
Email: aeriksen@allelecapital.com

FORWARD-LOOKING STATEMENTS

This press release contains certain “forward-looking statements” that reflect the Company’s current expectations and projections about its future results. 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. Forward-looking statements in this press release include, but are not limited to, statements relating to: the Company receiving all regulatory approvals; the Company advancing towards clinical and commercial breakthroughs in regenerative medicine; the Company enhancing its presence in key markets; the advancement of the Company’s therapeutic programs and clinical milestones; the results of the Company’s preclinical trials and its suggestion of a promising treatment pathway for SCI; the results of the Company’s preclinical trials and its suggestion of a promising treatment pathway for SCI; the Company developing groundbreaking therapies for regenerative medicine in several indications; ExoPTEN having the potential to address SCI and improve patient lives; the Company is advancing toward clinical translation in several high-impact indications; Professor Knoller will provide updates pertaining to the 2026 SCI Trial and results from the Company’s other preclinical studies at the 2025 ASIA meeting; types of adult patients and the treatment they will receive as part of the 2026 SCI Trial as discussed herein; preclinical studies as previously published and presented at ASIA will show robust recovery of motor, sensory, and structural function in rat models of complete spinal cord transection and compression; and the NurExone platform technology offering novel solutions to drug companies interested in minimally invasive targeted drug delivery for other indications, including recovery of optic nerve function and overall visual health.

These statements reflect management’s current beliefs and are based on information currently available to management as at the date hereof. In developing the forward-looking statements in this press release, we have applied several material assumptions, including: the Company will realize on the benefits of exosome loaded drugs in regenerating or repairing damaged nerves; the ability of the Company’s products to be used for patient treatment; the Company will fulfill its intended future plans and expectations; there being growing clinical demand for innovative treatments in spinal cord, optic nerve, and other therapeutic areas; the Company will carry out its pre-clinical trials and realize upon the benefits of the pre-clinical trials; the Company having the ability to maintain its ongoing commitment to using its ExoTherapy platform to advance the field of regenerative medicine and cell therapy applications; the Company will receive all regulatory approvals; the Company will have clinical and commercial breakthroughs in regenerative medicine;  the Company will be able to realize its future development plans, operational initiatives, and strategic objectives; the Company’s ability to advance its therapeutic programs and clinical milestones; the results of the Company’s preclinical trials and its ability to be a promising treatment pathway for SCI; the Company’s ability in advancing toward clinical translation in several high-impact indications; the results of the Company’s preclinical trials and its suggestion of a promising treatment pathway for SCI; the Company developing groundbreaking therapies for regenerative medicine in several indications; ExoPTEN having the ability to address vision loss and improve patient lives; the Company having the ability to advance toward clinical translation in several high-impact indications; Professor Knoller will have the ability to provide updates pertaining to the 2026 SCI Trial and results from the Company’s other preclinical studies at the 2025 ASIA meeting; the Company will be able to treat the adult patients and with the treatments they will receive as part of the 2026 SCI Trial as discussed herein; preclinical studies presented at ASIA will show robust recovery of motor, sensory, and structural function in rat models of complete spinal cord transection and compression; and the NurExone platform technology offering novel solutions to drug companies interested in minimally invasive targeted drug delivery for other indications, including recovery of optic nerve function and overall visual health

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; dependence on the Company’s strategic partners; the fact that preclinical drug development is uncertain, and the drug product candidates of the Company may never advance to clinical trials; the fact that results of preclinical studies and early-stage clinical trials may not be predictive of the results of later stage clinical trials; the uncertain outcome, cost, and timing of product development activities, preclinical studies and clinical trials of the Company; the uncertain clinical development process, including the risk that clinical trials may not have an effective design or generate positive results; the inability to obtain or maintain regulatory approval of the drug product candidates of the Company; the introduction of competing drugs that are safer, more effective or less expensive than, or otherwise superior to, the drug product candidates of the Company; the initiation, conduct, and completion of preclinical studies and clinical trials may be delayed, adversely affected or impacted by unforeseen issues; the inability to obtain adequate financing; the inability to obtain or maintain intellectual property protection for the drug product candidates of the Company; risks that the Company’s intellectual property and technology won’t have the intended impact on the Company and/or its business; the Company’s inability to carry out its pre-clinical trials and realize upon the stated benefits of the pre-clinical trials; the inability of the Company to realize on the benefits of exosomes; the inability of the Company to produce and/or supply exosomes for a wide range of applications; the inability of the Company’s products to be used for patient treatment; there not being broader adoption in the field and/or cell therapy applications; the inability of the Company to fulfill its intended future plans and expectations; there not being growing clinical demand for innovative treatments in spinal cord, optic nerve, and/or other therapeutic areas; the inability of the Company to collaborate with pharma companies; the Company’s inability to realize upon the stated potential for exosome-loaded drugs in regenerating or repairing damaged nerves; the Company’s inability to maintain its ongoing commitment to using its ExoTherapy platform to advance the field of regenerative medicine and/or cell therapy applications; the Company’s inability to expand into further studies; the Company will not receive all required regulatory approvals; the Company will not have clinical and/or commercial breakthroughs in regenerative medicine; the Company will be unable to enhance its presence in key markets; the NurExone platform technology not offering novel solutions to drug companies interested in minimally invasive targeted drug delivery for other indications; the Company will not realize its future development plans, operational initiatives, and strategic objectives; the Company will not advance its therapeutic programs and clinical milestones; the Company will not engage with regulatory agencies; the results of the Company’s preclinical trials not being a promising treatment pathway for SCI; the Company not advancing toward clinical translation in several high-impact indications; the Company not developing groundbreaking therapies for regenerative medicine in several indications; ExoPTEN not addressing vision loss or improving patient lives; the Company does not advance toward clinical translation in several high-impact indications; the NurExone platform technology not offering novel solutions to drug companies interested in minimally invasive targeted drug delivery for other indications, including recovery of optic nerve function and overall visual health; Professor Knoller will not provide updates pertaining to the 2026 SCI Trial and results from the Company’s other preclinical studies at the 2025 ASIA meeting; the adult patients will not receive the treatments as part of the 2026 SCI Trial as discussed herein; preclinical studies presented at ASIA will not show robust recovery of motor, sensory, and structural function in rat models of complete spinal cord transection and compression; and the risks discussed under the heading “Risk Factors” on pages 44 to 51 of the Company’s Annual Information Form dated August 27, 2024, a copy of which is available under the Company’s SEDAR+ profile at www.sedarplus.ca. 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 TSXV nor its Regulation Services Provider (as that term is defined in the policies of the TSXV) accepts responsibility for the adequacy or accuracy of this release.

FAQ

What is NurExone's ExoPTEN therapy and how does it work?

ExoPTEN is a first-in-class exosome-based therapy that uses mesenchymal stem cell-derived exosomes loaded with siRNA targeting PTEN to reduce cell death and promote axonal regrowth in acute spinal cord injuries.

When will NRXBF begin clinical trials for ExoPTEN?

NurExone plans to initiate Phase 1/2a clinical trials for ExoPTEN in 2026, starting with a safety study in up to 18 patients followed by a randomized controlled trial in 10-15 patients.

What regulatory designations has ExoPTEN received?

ExoPTEN has received Orphan Drug Designation from both the United States Food and Drug Administration (FDA) and European Medicines Agency (EMA).

What were the results of ExoPTEN's preclinical studies?

Preclinical studies demonstrated robust recovery of motor, sensory, and structural function in rat models, with exosomes effectively targeting the injury site for up to seven days post-injury.

Who is eligible for NurExone's 2026 spinal cord injury trial?

The trial will enroll adult patients with traumatic spinal cord injuries between spinal levels C5 and T10, classified as ASIA Grade A or B, who can be treated within 3 to 7 days post-injury.