ROCKVILLE, Md., May 24, 2022 (GLOBE NEWSWIRE) -- Neuronascent Inc., a clinical-stage biopharmaceutical company discovering and developing neuron-generating therapies for rare developmental disorders, today announced that the US Food and Drug Administration (FDA) granted rare pediatric designation for NNI-351 for Fragile X syndrome.
Fragile X syndrome (FXS) is a heritable monogenic disorder and is a major cause of intellectual disabilities and autism. Presently, there are no effective therapies to treat the hippocampal-related deficits, such as social anxiety, learning disability 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 prompting neural progenitor cells to become neurons, i.e., neurogenesis, NNI-351 reverses behavioral deficits back to normal levels. Neuronascent’s oral therapy could therefore slow and potentially reverse FXS behavioral deficits in young individuals.
With a marketing application for NNI-351 for young FXS patients, Neuronascent as sponsor will request an award of a rare pediatric disease priority voucher. The FDA’s Rare Pediatric Disease Priority Voucher Program is designed to incentivize companies to develop novel therapies to treat rare disorders of under 200,000 individuals and that primarily afflict 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, expediting time to market and providing greater value for the rare pediatric therapeutic even with a small population pool.
“The receipt of this rare pediatric designation from the FDA allows Neuronascent to become eligible for the pediatric priority review voucher, which provides significant value to our first-in-class therapeutic NNI-351 for FXS in young individuals,” stated founder and CEO, Judith Kelleher-Andersson, PhD. “With vast unmet need for novel therapies that can directly address pediatric FXS behavioral deficits, such as to improve education and social outcomes of these young patients, this designation puts a spotlight on the true potential of NNI-351.”
About NNI-351
NNI-351 is Neuronascent’s lead, patented, new chemical entity aimed at reversing developmental disorders by producing new neurons postnatally to enhance neurogenesis that is slowed during early development in diseases such as FXS. NNI-351’s ability to reverse behaviors in a number of models of developmental disorders, including rare pediatric disorder, FXS, suggests this novel therapy could be the first therapy to show true clinical benefit in young FXS patients.