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), behavioral 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 behavioral 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.