RARE Daily

Noveome and Neuronascent Each Receive Rare Pediatric Disease Designations

May 24, 2022

The U.S. Food and Drug Administration granted Rare Pediatric Disease designation to two experimental preclinical therapies: Noveome Biotherapeutics’ ST266 for the treatment of necrotizing enterocolitis; and Neuronascent’s NNI-351 for the treatment of Fragile X syndrome.

Photo: Christopher Velis, CEO of Noveome

Necrotizing enterocolitis (NEC) is a devastating disease caused by inflammation of the intestines observed primarily in premature and very low birth weight babies (VLBWB). The inflammation can result in an overwhelming infection which quickly becomes a medical emergency and often requires surgery as a life-saving measure. NEC affects between 4,000 – 6,000 VLBWB each year in the United States and carries a 30 percent mortality rate. Babies that do survive are often left with life-long intestinal complications and are also at increased risk for neurodevelopmental delay with cognitive, visual, and motor impairment. There is currently no FDA-approved treatment for NEC.

Noveome’s ST266 is a cell-free sterile biologic solution containing hundreds of proteins and other factors at physiologic levels. Extensive preclinical studies have shown that ST266’s multiple components result in a variety of anti-inflammatory and neuroprotective responses. A drug master file has been submitted to the FDA, supporting all ST266 investigational new drug applications.

“NEC is a devastating disease that affects the most vulnerable among us,” said Christopher Velis, CEO of Noveome. “We remain motivated by the prospect of providing the first major improvement in lifesaving therapy for infants affected by NEC in over 40 years.”

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.

Neuronascent’s lead compound NNI-351 is a 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 the rare pediatric disorder FXS, suggests this novel therapy could be the first therapy to show true clinical benefit in young FXS patients.

Evaluation of NNI-351 in preclinical models of FXS (supported in part by the FRAXA organization) by selectively prompting neural progenitor cells to become neurons, has shown that 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.

“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,” said Judith Kelleher-Andersson, founder and CEO of Neuronascent. “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.”

The FDA grants Rare Pediatric Disease designation for serious and life-threatening diseases affecting children aged 18 years or younger. Pediatric diseases recognized as rare affect fewer than 200,000 people in the United States.

Under the FDA’s Rare Pediatric Disease designation and Voucher programs, if these companies receive marketing approval for their therapies, they may be eligible to receive a Priority Review Voucher (PRV) from the FDA. The voucher can be used to reduce the time of an FDA new drug approval review to six months from ten months. The vouchers are potentially lucrative because they are transferable. Most recently, BridgeBio Pharma sold its voucher for $110 million.

Author: Rare Daily Staff

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