Potential Fragile X Treatment Earns Rare Pediatric Disease Status in U.S.

The United States Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation to Neuronascent’s investigational treatment NNI-351 for the treatment of fragile X syndrome (FXS).

“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.” , said Judith Kelleher-Andersson, PhD, founder and CEO of Neuronascent, said in a press release.

NNI-351 is an oral small molecule that, in preclinical models, has been shown to promote neurogenesis – the process by which immature progenitor cells develop into mature neurons (nerve cells). The therapy is specifically thought to work by simultaneously activating the p70S6kinase protein while blocking another protein, DYRK1A.

According to Neuronascent, experiments in preclinical animal models of Fragile X have shown that NNI-351 treatment promotes neurogenesis in the hippocampus, a brain region important for memory. This neurogenesis was associated with less anxious behavior, better learning and reduced hyperactivity in young animals.

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Neuronascent is developing NNI-351 to treat fragile X and other conditions marked by developmental delays and intellectual disabilities, such as Down syndrome.

The FDA’s Rare Pediatric Disease Priority Voucher Program is designed to help encourage companies to develop new treatments for rare diseases, defined as those affecting fewer than 200,000 people in the United States, which primarily affect children under 18 years old.

With the new designation, Neuronascent, as the developer of NNI-351, will receive a voucher that the company can redeem for priority review of this drug or another; the voucher can also be exchanged or sold to other companies. Priority review is a process in which the FDA allocates additional resources to review an application, shortening the review process from the usual 10 months to approximately six months.

“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,” Kelleher said. -Andersson.

Fragile X, the most common cause of inherited intellectual disability and autism spectrum disorder, is estimated to affect 1 in 4,000 men and 1 in 8,000 women in the United States. He currently has no targeted treatment, with symptoms being managed through programs such as talk, occupational therapy and behavioral therapy.

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