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
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
About
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For more information, please contact:
Dr.
Chief Executive Officer and Director
Phone: +972-52-4803034
Email: info@nurexone.com
Investment Relation -
Phone: +1 905-347-5569
Email: IR@nurexone.com
Dr.
Investment Relation -
Phone: +49-69-1532-5857
Email: e.reuter@dr-reuter.eu
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
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
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i https://www.accessdata.fda.gov/scripts/opdlisting/oopd/detailedIndex.cfm?cfgridkey=940823
ii https://www.fda.gov/patients/rare-diseases-fda
Source:
2023 GlobeNewswire, Inc., source