Neurocrine Biosciences presents data on the demographic and clinical characteristics of pediatric patients with SCN8A-related epilepsy at AES 2022

Neurocrine Biosciences presents data on the demographic and clinical characteristics of pediatric patients with SCN8A-related epilepsy at AES 2022

SAN DIEGO, December 2, 2022 /PRNewswire/ — Neurocrine Biosciences, Inc. (Nasdaq: NBIX), a leading neuroscience-focused biopharmaceutical company, today released demographic and clinical data from studies based on genetic screening of children with SCN8A– related epilepsies. Data was collected from over 17,000 patients through Invitee Corporation’s Behind the Seizure® Program, of which Neurocrine Biosciences is a sponsor. The clinical characteristics of patients with a pathogenic or presumably pathogenic disease SCN8A variants were assessed to assess the heterogeneity of seizures and development of children with SCN8A– related epilepsies. These data (Poster #2.099: Demographic and Clinical Characteristics of Pediatric Patients with SCN8A-related epilepsies: results of a no-cost epilepsy gene panel) will be shared at the AES 2022 Annual Meeting at Nashville, TNfrom December 2 to 6, 2022. (link)

“These analyzes reveal the phenotypic spectrum of SCN8A variants and the high burden of seizures experienced by children with these rare and previously undiagnosed epilepsies,” said Eiry W. Roberts, MD, Chief Medical Officer.® program, which provides free genetic testing to children under the age of eight who have had one or more unprovoked seizures, including those who have SCN8A– related epilepsies. We are conducting a Phase 2 clinical study of NBI-921352, an investigational selective sodium channel blocker as a potential adjunctive therapy in children and young adults with SCN8A developmental and epileptic encephalopathy (SCN8A-DEE). »

Poster features analyzes of genetic testing data from 17,843 children younger than 8 and with ≥ 1 unprovoked seizure who participated in Behind the Crisis® program. Data analysis showed that 36 (0.2%) of the patients had a SCN8A variant classified as pathogenic (P) or probably pathogenic (LP). From 24 unique P/LPs SCN8A variants identified, 14 were classified as pathogenic and 10 were probably pathogenic. In patients with P/LP SCN8A variant, the mean (± SD) age at seizure onset was 1.4 (± 1.4) years and the mean age at testing was 2.1 (± 2.4) years. The most frequently reported seizure type was generalized motor seizures (50.0% of patients), followed by focal seizures (30.6%) and generalized non-motor (absence) seizures (13.9%). The most commonly reported developmental delays were language delays (30.6%), limited or absent speech (22.2%) and intellectual disability/motor developmental delays (16.7%). More than half (58.3%) of patients had ≥1 seizure in the last month and 44.5% of patients had ≥1 prolonged seizure (>5 min) in the last six months. At screening, 77.8% of patients were taking 1 to 3 anticonvulsant medications (ASMs) and almost a fifth (19.4%) had previously discontinued 1 to 3 MSAs.

Neurocrine Biosciences is a Sponsor and Participant of Behind Seizures® The program’s science exhibit at AES 2022, where 7 companies will showcase 30 science posters that highlight how the Behind Foreclosures® The program is transforming genetic epilepsy diagnoses and improving care pathways through cutting-edge research. Behind the scenes of crises® The scientific exhibition of the program will take place on Sunday, December 4 at the Music City Center, (Room 207 C/D, 2n/a floor) from 8:00 a.m. to 11:00 a.m. CT.

About NBI-921352 and the KAYAK™ Phase 2 Study
NBI-921352 is an investigational selective sodium channel 1.6 (Nav1.6) currently under development for the potential treatment of SCN8A developmental and epileptic encephalopathy (SCN8A-DEE) in children and adults. Neurocrine Biosciences is conducting the Phase 2 KAYAK™ study to evaluate the efficacy, safety, tolerability and pharmacokinetics of NBI-921352 as an adjunctive therapy in children and young adults with SCN8A-DEE. For more information about this study, please visit Where

After completion of the KAYAK™ study, eligible participants may have the option of enrolling in an open-label extension study to evaluate the long-term safety and tolerability of NBI-921352 in patients with SCN8A -DEE. For more information on this open-label study, please see

Neurocrine Biosciences has received Orphan Drug Designation and Rare Pediatric Disease Designation from the United States Food and Drug Administration (FDA) for NBI-921352 in SCN8A-DEE.

SCN8A developmental and epileptic encephalopathy (SCN8A-DEE) is a rare pediatric epilepsy syndrome associated with genetic mutations in the SCN8A embarrassed. It is characterized by early onset seizures, developmental delay, cognitive impairment, and other medical issues. Seizures begin at a median age of four months and are highly refractory to currently available anticonvulsant medications. Over 90% of children with SCN8A-DEE are non-verbal and half are non-ambulatory. Children living with rare pediatric epilepsies, such as SCN8A-DEE, are at higher risk of sudden unexpected death in epilepsy (SUDEP). There is currently no approved treatment for this form of pediatric epilepsy.

Behind the input® Program
More than 50% of epilepsies have a genetic basis. Developmental and epileptic encephalopathies (DEE) can be mainly attributed to genetic causes, including pathogenic mutations in the SCN8A embarrassed. Identifying the potential genetic underpinnings of different forms of epilepsy could support the development of more specific treatments.

To support research on the genetics of epilepsy, Neurocrine Biosciences is a sponsor of the Invitae project Behind the input® program in the United States and Canada. This program provides free access to comprehensive testing for genetic variations linked to epilepsy to any child under the age of eight who has had an unprovoked seizure.

About Neurocrine Biosciences
Neurocrine Biosciences is a leading neuroscience-driven biopharmaceutical company with a simple goal: to alleviate the suffering of people with high needs but few options. We are committed to discovering and developing life-changing treatments for patients with undertreated neurological, neuroendocrine and neuropsychiatric disorders. The company’s diverse portfolio includes FDA-approved treatments for tardive dyskinesia, Parkinson’s disease, endometriosis* and uterine fibroids*, as well as more than a dozen mid- to late-stage clinical programs in multiple therapeutic areas. For three decades, we have applied our unique knowledge of neuroscience and the interconnections between brain and body systems to treat complex conditions. We relentlessly seek out medicines to ease the burden of debilitating diseases and disorders, because you deserve brave science. For more information, visit and follow the company on LinkedIn, Twitter, and Facebook. (*in collaboration with AbbVie).

Neurocrine Biosciences, the Neurocrine logo and KAYAK are registered trademarks of Neurocrine Biosciences, Inc.

Forward-looking statement
In addition to historical facts, this press release contains forward-looking statements that involve a number of risks and uncertainties. These statements include, but are not limited to, statements relating to: the benefits to be derived from our products and product candidates, and our collaboration with Invitae’s Behind the Seizure® program. Among the factors that could cause actual results to differ materially from those set forth in the forward-looking statements are: our future financial and operating performance; risks related to the development of our product candidates; risks that the FDA or other regulatory authorities make adverse decisions regarding our products or product candidates; the risks that our products and/or product candidates may be prevented from commercialization by the proprietary or regulatory rights of third parties, or that they may experience unintended side effects, adverse reactions or incidents of misuse; risks associated with potential generic entrants for our products; the risks that clinical development activities will not be completed on time or at all, or will be delayed for regulatory, manufacturing, COVID-19 or other reasons, fail or replicate clinical trial results precedents, fail to demonstrate that our product candidates are safe and effective, or may not be predictive of actual results or the results of subsequent clinical trials; the risks that the potential benefits of agreements with our collaboration partners may never be realized; the risks and uncertainties associated with the magnitude and duration of the COVID-19 pandemic and the resulting global, national and local economic and financial disruptions; and other risks described in our periodic reports filed with the SEC, including, without limitation, our quarterly report on Form 10-Q for the quarter ended September 30, 2022. Neurocrine Biosciences disclaims any obligation to update the statements contained in this press release after the date hereof.

SOURCE Neurocrine Biosciences, Inc.

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