Neuronascent Receives Rare Pediatric Drug Designation from FDA for NNI-351 Treatment of Fragile X Syndrome

Neuronascent, Inc.

ROCKVILLE, Md., May 24, 2022 (GLOBE NEWSWIRE) — Neuronascent Inc., a clinical-stage biopharmaceutical company discovering and developing neural-generating therapies for rare developmental disorders, today announced that the Food and Drug Administration (FDA) of the United States had granted rare pediatric designation for NNI-351 for fragile X syndrome.

Fragile X syndrome (FXS) is an inherited monogenic disorder and is a major cause of intellectual disability and autism. Currently, there are no effective therapies to treat hippocampus-related deficits such as social anxiety, learning disabilities and hyperactivity as well as impaired neurogenesis.

Evaluation of NNI-351 in preclinical models of FXS (supported in part by the FRAXA organization, https://www.fraxa.org) by selectively inducing neural progenitor cells to become neurons, i.e. neurogenesis, NNI-351 reverses behavioral deficits to normal levels. Neuronascent’s oral therapy could therefore slow and potentially reverse the behavioral deficits of FXS in young people.

With a marketing application for NNI-351 for young FXS patients, Neuronascent as a sponsor will request the award of a good priority rare pediatric disease. The FDA’s Rare Pediatric Disease Priority Voucher Program is designed to incentivize companies to develop new therapies to treat rare disorders with fewer than 200,000 people and that primarily affect those under the age of 18. The priority review voucher could then be used for a subsequent drug, or can be sold to another pharmaceutical company, which speeds time to market and provides greater value for rare pediatric treatment, even with a small pool of population.

“Receiving this rare pediatric designation from the FDA allows Neuronascent to become eligible for the Pediatric Priority Review Voucher, which brings significant value to our first-in-class therapeutic NNI-351 for FXS in young people,” said the founder and CEO, Judith Kelleher-Andersson, PhD. “With a large unmet need for new therapies that can directly address pediatric behavioral deficits in FXS, for example to improve the education and social outcomes of these young patients, this designation highlights the true potential of NNI-351. “

About NNI-351

NNI-351 is Neuronascent’s patented new lead chemical entity aimed at reversing developmental disorders by producing new neurons after birth to enhance neurogenesis that is slowed during the early development of diseases such as FXS. The ability of NNI-351 to reverse behaviors in a number of models of developmental disorders, including the rare pediatric disorder, FXS, suggests this new therapy may be the first therapy to show true clinical benefit in young patients. with FXS.

About Neuronascent, Inc.

Neuronascent, Inc., a private, clinical-stage pharmaceutical company, was founded to discover and develop new therapies that treat CNS disorders with high unmet needs by replacing and enhancing the number of neurons, not just connections. Through its proprietary phenotypic discovery platform, Neuronascent has discovered a pipeline of patented small molecule neuron regeneration candidates, including clinical therapy NNI-362 for age-related disorders, particularly Alzheimer’s and of Parkinson’s.

Neuronascent website: https://www.neuronascent.com

Contact information :

Judith Kelleher-Andersson, PhD,

[email protected],

Such. 1-240-876-7496

Nick Veronico

[email protected]

Safe Harbor Statement
This release contains forward-looking statements, which are made pursuant to the safe harbor provisions of the United States Private Securities Litigation Reform Act of 1995. Forward-looking statements are generally identified by words such as “would”, “may”, “will, “expects” and other terms with a similar meaning. Forward-looking statements are based on current beliefs, assumptions and expectations and speak only as of the date of this release and involve risks and uncertainties that could cause actual results to differ materially from current expectations.

About Antoine L. Cassell

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