Home / Press Releases / Neuralstem Announces NSI-189 Granted Orphan Drug Designation from the U.S. FDA for the Treatment of Angelman Syndrome

Neuralstem Announces NSI-189 Granted Orphan Drug Designation from the U.S. FDA for the Treatment of Angelman Syndrome

GERMANTOWN, Md., Aug. 09, 2018 (GLOBE NEWSWIRE) — Neuralstem, Inc. (Nasdaq:CUR), a biopharmaceutical company focused on the development of nervous system therapies based on its neural stem cell technology, today announced that the U.S. Food and Drug Administration has granted orphan drug designation to NSI-189 for the treatment of Angelman syndrome.

“Angelman Syndrome is a rare disease with significant unmet medical need, and for which there are no FDA-approved therapies,” said Jim Scully, Chief Executive Officer of Neuralstem. “Orphan drug designation is an important regulatory milestone in the development of NSI-189 and we are committed to evaluating NSI-189’s role as a treatment to improve the lives of patients with Angelman syndrome.”

In pre-clinical models, NSI-189 has demonstrated the ability to restore long term potentiation (LTP), a measure of synaptic plasticity and an in vitro biomarker of memory.

The FDA’s orphan-drug designation program provides special status and incentives to encourage the development of drugs for diseases affecting fewer than 200,000 people in the U.S. Orphan drug designation confers seven years of market exclusivity upon FDA approval, as well as other development incentives, such as tax credits related to clinical trial expenses, an exemption from the FDA-user fee and FDA assistance in clinical trial design.

About Angelman Syndrome

Angelman Syndrome (AS) is a rare congenital genetic disorder caused by a lack of function in the UBE3A gene on the maternal 15th chromosome.  It affects approximately one in 15,000 people – about 500,000 individuals globally.  Symptoms of AS include developmental delay, lack of speech, seizures, and walking and balance disorders. Patients with AS may never walk or speak and require life-long care. Life expectancy is normal which places a significant burden on patients and caregivers. There are currently no FDA-approved therapies for the treatment of Angelman syndrome.

About Neuralstem

Neuralstem is a clinical-stage biopharmaceutical company developing novel treatments for nervous system diseases of high unmet medical need. The Company has two lead development candidates:

  • NSI-189 is a small molecule in clinical development for major depressive disorder and in preclinical development for Angelman syndrome, irradiation-induced cognitive impairment, Type 1 and Type 2 diabetes, and stroke.
  • NSI-566 is a stem cell therapy being tested for treatment of paralysis in stroke, Amyotrophic Lateral Sclerosis (ALS), chronic spinal cord injury (cSCI), and traumatic brain injury (TBI). NSI-566 was shown to have restorative function in a primate paralysis model. The study was published in the peer reviewed journal Nature Medicine, and the full manuscript can be found here.

Neuralstem’s diversified portfolio of product candidates is based on its proprietary neural stem cell technology.

Cautionary Statement Regarding Forward Looking Information

This news release contains “forward-looking statements” made pursuant to the “safe harbor” provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements relate to future, not past, events and may often be identified by words such as “expect,” “anticipate,” “intend,” “plan,” “believe,” “seek” or “will.” Forward-looking statements by their nature address matters that are, to different degrees, uncertain. Specific risks and uncertainties that could cause our actual results to differ materially from those expressed in our forward-looking statements include risks inherent in the development and commercialization of potential products, uncertainty of clinical trial results or regulatory approvals or clearances, need for future capital, dependence upon collaborators and maintenance of our intellectual property rights. Actual results may differ materially from the results anticipated in these forward-looking statements. Additional information on potential factors that could affect our results and other risks and uncertainties are detailed from time to time in Neuralstem’s periodic reports, including the Annual Report on Form 10-K for the year ended December 31, 2017 filed with the Securities and Exchange Commission (SEC), and in other reports filed with the SEC. We do not assume any obligation to update any forward-looking statements.

Contact:
Kimberly Minarovich
Argot Partners (Investor Relations)
212-600-1902
neuralstem@argotpartners.com

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Home / Press Releases / Neuralstem Announces NSI-189 Granted Orphan Drug Designation from the U.S. FDA for the Treatment of Angelman Syndrome

Neuralstem Announces NSI-189 Granted Orphan Drug Designation from the U.S. FDA for the Treatment of Angelman Syndrome

GERMANTOWN, Md., Aug. 09, 2018 (GLOBE NEWSWIRE) — Neuralstem, Inc. (Nasdaq:CUR), a biopharmaceutical company focused on the development of nervous system therapies based on its neural stem cell technology, today announced that the U.S. Food and Drug Administration has granted orphan drug designation to NSI-189 for the treatment of Angelman syndrome.

“Angelman Syndrome is a rare disease with significant unmet medical need, and for which there are no FDA-approved therapies,” said Jim Scully, Chief Executive Officer of Neuralstem. “Orphan drug designation is an important regulatory milestone in the development of NSI-189 and we are committed to evaluating NSI-189’s role as a treatment to improve the lives of patients with Angelman syndrome.”

In pre-clinical models, NSI-189 has demonstrated the ability to restore long term potentiation (LTP), a measure of synaptic plasticity and an in vitro biomarker of memory.

The FDA’s orphan-drug designation program provides special status and incentives to encourage the development of drugs for diseases affecting fewer than 200,000 people in the U.S. Orphan drug designation confers seven years of market exclusivity upon FDA approval, as well as other development incentives, such as tax credits related to clinical trial expenses, an exemption from the FDA-user fee and FDA assistance in clinical trial design.

About Angelman Syndrome

Angelman Syndrome (AS) is a rare congenital genetic disorder caused by a lack of function in the UBE3A gene on the maternal 15th chromosome.  It affects approximately one in 15,000 people – about 500,000 individuals globally.  Symptoms of AS include developmental delay, lack of speech, seizures, and walking and balance disorders. Patients with AS may never walk or speak and require life-long care. Life expectancy is normal which places a significant burden on patients and caregivers. There are currently no FDA-approved therapies for the treatment of Angelman syndrome.

About Neuralstem

Neuralstem is a clinical-stage biopharmaceutical company developing novel treatments for nervous system diseases of high unmet medical need. The Company has two lead development candidates:

  • NSI-189 is a small molecule in clinical development for major depressive disorder and in preclinical development for Angelman syndrome, irradiation-induced cognitive impairment, Type 1 and Type 2 diabetes, and stroke.
  • NSI-566 is a stem cell therapy being tested for treatment of paralysis in stroke, Amyotrophic Lateral Sclerosis (ALS), chronic spinal cord injury (cSCI), and traumatic brain injury (TBI). NSI-566 was shown to have restorative function in a primate paralysis model. The study was published in the peer reviewed journal Nature Medicine, and the full manuscript can be found here.

Neuralstem’s diversified portfolio of product candidates is based on its proprietary neural stem cell technology.

Cautionary Statement Regarding Forward Looking Information

This news release contains “forward-looking statements” made pursuant to the “safe harbor” provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements relate to future, not past, events and may often be identified by words such as “expect,” “anticipate,” “intend,” “plan,” “believe,” “seek” or “will.” Forward-looking statements by their nature address matters that are, to different degrees, uncertain. Specific risks and uncertainties that could cause our actual results to differ materially from those expressed in our forward-looking statements include risks inherent in the development and commercialization of potential products, uncertainty of clinical trial results or regulatory approvals or clearances, need for future capital, dependence upon collaborators and maintenance of our intellectual property rights. Actual results may differ materially from the results anticipated in these forward-looking statements. Additional information on potential factors that could affect our results and other risks and uncertainties are detailed from time to time in Neuralstem’s periodic reports, including the Annual Report on Form 10-K for the year ended December 31, 2017 filed with the Securities and Exchange Commission (SEC), and in other reports filed with the SEC. We do not assume any obligation to update any forward-looking statements.

Contact:
Kimberly Minarovich
Argot Partners (Investor Relations)
212-600-1902
neuralstem@argotpartners.com

Check Also

Open Society Names New Head of West Africa Foundation

New York, Aug. 15, 2018 (GLOBE NEWSWIRE) — The Open Society Foundations are pleased to announce the appointment of Ayisha Osori as executive director of the Open Society Initiative for West Africa (OSIWA), effective September 4. Osori is a lawyer, development consultant, and communications strategist with 18 years of experience in both the public and […]