Oral NNI-351 Awarded Orphan Drug Status by FDA

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by Patricia Inácio, PhD |

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The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to NeuroNascent’s NNI-351 for the treatment of fragile X syndrome.

NNI-351, given orally, is able to penetrate the brain, where it has been shown to boost the proliferation and growth of immature progenitor cells into mature neurons (nerve cells) in the hippocampus, a process called neurogenesis. The hippocampus is an area of the brain that plays an important role in learning and memory.

The therapy is believed to work by simultaneously activating the protein p70S6 kinase while blocking another protein, DYRK1A.

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Experiments in preclinical animal models of fragile X, conducted with partial support by the FRAXA Research Foundation, showed that boosting neurogenesis reversed several of the behavior deficits seen in fragile X patients. These included less anxious behavior, improved learning, and decreased hyperactivity in young animals.

According to NeuroNascent, the benefits seen across different models of the disease suggest that NNI-351 could be one of the first therapies to clinically benefit young fragile X patients.

The FDA’s decision is preceded by a rare pediatric disease designation granted last month.

“Both the Orphan Drug Designation and the Rare Pediatric Drug Designation provide greater incentive for collaborations aimed at completing the Investigational New Drug Application and clinical stage evaluations of NNI-351 to effectively treat this developmental disorder,” Judith Kelleher-Andersson, PhD, founder and CEO of Neuronascent, said in a press release.

NNI-351 is being developed for fragile X as well as other conditions characterized by developmental delays and intellectual impairments, like Down syndrome.

Both rare pediatric disease and orphan drug designation are awarded by the FDA to encourage the development of therapies for rare and serious diseases.

Orphan drug status entitles companies to a series of benefits, such as seven years of market exclusivity upon approval and exemption from regulatory application fees.

Fragile X has no targeted treatments. Its symptoms mainly are managed via speech, occupational, and behavior therapy.