CuroNZ is aiming to change the scope of therapeutic treatment possibilities for refractory epilepsies and difficult to treat encephalopathies by putting emphasis on targeting seizures and co-morbidities (like memory impairment and anxiety). Our neurologically active peptide drug candidates have been identified by phenotypic drug discovery screens using rodent brain tissue and have been named Neuronal Regeneration Peptides (NRPs). Our clinical lead peptidomimetic NRP2945 is readied now for clinical phase 2 in adults diagnosed with drug-resistant Temporal Lobe Epilepsy (TLE). Subsequently, we will target a severe paediatric encephalopathy (Lennox-Gastaut Syndrome) where NRP2945 has been awarded orphan drug destination by CDER back in April 2021. NRPs have the potential to be a high impact drug as they work on a unique MOA and are neuroregenerative as well as neuroprotective and is enhancing memory pconsolidation and retrieval. By enhancing phasic inhibition via differential protein expression regulation of subunits located inside the GABA A receptors, NRP2945 is breaking oscillatory activity profiles (seen in epilepsy) leading to seizure freedom and memory improvement while also downregulating anxiety and hyperactivity profiles known for both aforementioned patient populations. Furthermore, NRP2945 has good prospects to be developed for orphan motor diseases like ALS or other childhood encephalopathies where specifically the synaptic GABA A protein subunit GABRB3 is downregulated in these syndromes. CuroNZ is poised that after achieving proof-of-concept in the clinical phase I/II studies, a follow-up compound currently at start of preclinical development can be readied for large neurological diseases like Alzheimer, progressive MS and dementia where the GABAergic system is dysfunctional and is in need of re-calibration to alleviate disease symptoms and provide true regeneration.
