NurExone Biologic Receives FDA Orphan-Drug Designation, Accelerating Development of ExoPTEN therapy for Acute Spinal Cord Injury Treatment
The orphan drug designation provides significant benefits to pharmaceutical companies developing drugs for rare diseases, i.e.
TORONTO and HAIFA, Israel, Oct. 30, 2023 (GLOBE NEWSWIRE) -- NurExone Biologic Inc. (TSXV: NRX) (FSE: J90) (NRX.V) (the “Company” or “NurExone”), a pioneering biotechnology company, is thrilled to announce that the U.S. Food and Drug Administration (FDA) has granted Orphan-Drug Designation (ODD) for its ExoPTEN therapy, recognizing the potential of this groundbreaking regenerative therapy for acute spinal cord injury, a condition where effective treatments are limitedi.
The orphan drug designation provides significant benefits to pharmaceutical companies developing drugs for rare diseases, i.e. those impacting fewer than 200,000 people in the United Statesii. These benefits include market exclusivity, financial incentives, regulatory assistance, and support with drug development. Overall, the designation incentivizes and supports the development of certain treatments, increasing access to therapies for patients.
Earning orphan-drug designation is a significant milestone for the Company. The designation covers the use of mesenchymal stem cell (MSC) derived small extracellular vesicles (EVs) loaded with short and modified interfering RNA (siRNA) targeting the phosphatase and tensin homolog (PTEN) protein for acute spinal cord injury, as implemented in the Company’s ExoPTEN drug under development.
“Orphan-drug designation is expected to streamline our go-to-market, shorten our regulatory process saving the Company millions of dollars, and provide valuable market exclusivity. We appreciate the formal recognition of the potential impact of our therapy on the lives of patients suffering from acute spinal cord injuries,” said Dr. Shaltiel, CEO of NurExone Biologic, Ltd.
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. Via its fully-owned subsidiary, NurExone Biologic Ltd, 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: [email protected]
Thesis Capital Inc.
Investment Relation - Canada
Phone: +1 905-347-5569
Email: [email protected]
Dr. Eva Reuter
Investment Relation - Germany
Phone: +49-69-1532-5857
Email: [email protected]
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’s ExoTherapy drug, ExoPTEN and the potential benefits to the Company of the orphan-drug designation of ExoPTEN. 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 our ability to retain key personnel, our ability to continue investing in research and development, our ability to secure available funding and to continue as a going concern, our ability to meet the post-approval requirements in respect of the orphan-drug designation, the general business and economic conditions of the industries and countries in which we operate, that the Company’s operations in Israel will not be material disrupted by unrest in Israel and the Israel-Hamas War, our ability to execute on our business strategy, that there will be certain amount of demand for the Company’s potential product, inflation will remain stable, and that the results of our studies reflect results that can be extrapolated.
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 not being able to demonstrate clinical superiority of its ExoTherapy drug, ExoPTEN, 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, dependence on the Company’s strategic partners, the effect of the Israel-Hamas War on the Company’s operations and the risks discussed under the heading “Risk Factors” on pages 29 to 36 of the Company’s Annual Information Form dated March 30, 2023, 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 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.
i https://www.accessdata.fda.gov/scripts/opdlisting/oopd/detailedIndex.cfm?cfgridkey=940823
ii https://www.fda.gov/patients/rare-diseases-fda
