SAN DIEGO, Dec. 1, 2018 /PRNewswire/ — RogCon Biosciences (RogCon), a biotechnology company focused on discovering and developing novel therapeutics for children suffering from SCN2A mutations, today announced preclinical data in a late-breaking poster presentation at the 2018 American Epilepsy Society Annual Meeting (AES) being held in New Orleans, LA November 30-December 4, 2018. Mutations in SCN2A are the most common cause of neurodevelopmental diseases such as severe epilepsy and autism, with numerous associated comorbidities and no known cure.
RogCon and its collaborators from The Florey Institute of Neuroscience presented preclinical data on the company’s lead program, RC-222, an antisense oligonucleotide designed to down-regulate SCN2A, which is being developed with Ionis Pharmaceuticals, Inc. (Ionis), the leader in RNA-targeted drug discovery and development. In preclinical studies evaluating the antisense oligonucleotide in an SCN2A mouse model, efficacy was evaluated by survival, seizure number, electroencephalography (EEG), behavioural test batteries and whole cell recording in brain slices. Results demonstrated that:
- The therapeutic effect of a single peri-natal dose of the antisense oligonucleotide was significant and long lasting, with nearly 70% of treated mice surviving to 80 days compared to 0% survival at 30 days in untreated mice or those receiving current standard of care.
- Treatment with the antisense oligonucleotide mitigated spontaneous seizures and largely restored neuronal excitability, EEG activity and behavioural characteristics of treated mice to wildtype levels.
- These results demonstrate the remarkable efficacy of SCN2A down-regulation and has laid an important foundation for the accelerated clinical development of the RC-222 program.
“We are encouraged by the preclinical data presented, which demonstrate the potential of Ionis’ antisense technology in advancing a transformative treatment for children with SCN2A mutations. We believe that the work we are doing in this collaboration may have a positive impact on these children and their families,” said Dr. C. Frank Bennett, Ionis senior vice president of research and franchise leader for neurological programs.
“The focus on SCN2A mutations by the scientific community has substantially increased over the past decade, revealing that SCN2A is a critical gene in neuronal function. However, despite this increased attention, no therapeutic options exist for children and their families suffering from mutations in SCN2A,” said Alex Nemiroff, CEO and Co-Founder of RogCon. “Our mission at RogCon is to discover and develop novel, disease-modifying treatments for all those suffering from SCN2A mutations, including my son Roger and co-founder Kelley Dalby’s son Connor, among many others. We believe that our collaboration with Ionis, scientific approach and collaborative efforts in the space, along with our personal connection to this disease, uniquely enable us to accelerate novel SCN2A therapies to the clinic and to patients.”
Late-Breaking Poster Presentation:
- Title: “Antisense oligonucleotide therapy for SCN2A gain-of-function epilepsies”
- Date/Time: December 1, 2018, 12-6 PM
- Hall H, First Floor
- The abstract and late-breaking poster can be accessed on AES’ website https://meeting.aesnet.org/.
About SCN2A Mutations
Mutations in SCN2A (Nav1.2) are the most common cause of neurodevelopmental disease — however, at present there is no corrective therapy specifically for SCN2A patients. All current medications are focused primarily on symptomatic relief. Epilepsy and autism are the most prominent conditions but there are numerous comorbidities, including sensory dysfunction, movement/muscle disorders, intellectual disability, gastrointestinal complications, metabolic abnormalities and others.
About RogCon Biosciences
RogCon is a biotechnology company focused on discovering and developing novel therapeutics for children suffering from SCN2A mutations, where no solutions currently exist. Mutations in SCN2A are the most common cause of neurodevelopmental disease, epilepsy, and autism, and there are numerous associated comorbidities. RogCon has amassed a group of world experts in genetic epilepsy research and drug development, with a focus on SCN2A, including collaborations with The Florey Institute of Neuroscience and with Ionis Pharmaceuticals, Inc. RogCon is developing a potentially disease-modifying therapy designed to down-regulate SCN2A (RC-222). RogCon, through its sister company RogCon U.R., Inc., is actively pursuing development of additional novel therapeutics, including RCUR-313 and RCUR-SMP, that are designed to up-regulate SCN2A to potentially treat multiple CNS indications. To learn more, please visit www.rogcon.com
Investor and Media Contact:
SOURCE RogCon Biosciences