Lead asset HER-096 - In development for Parkinson's disease
HER-096 is an engineered derivative of the Cerebral Dopamine Neurotrophic Factor (CDNF) protein targeted at treating neurodegenerative diseases with systemic administration
- HER-096 is designed to reach the brain upon subcutaneous administration, unlike the CDNF protein that requires direct administration into the brain
- HER-096 retains CDNF’s biological activity
- HER-096 is manufactured cost efficiently via simple chemical synthesis and has improved metabolic stability compared to CDNF
- HER-096 first-in-human clinical study is expected to commence in 2023
HER-096 has a unique Mechanism of Action
While the root cause of Parkinson’s disease remains unknown, the brain pathology is marked by a vicious cycle where chronic cellular stress in the brain increases the build up of misfolded proteins (e.g. a-synuclein) and neuroinflammation, which in turn add to the stress cells experience and escalates the disease process. CDNF and HER-096 have been shown to act against these key mechanisms of PD.
HER-096 crosses the Blood-Brain Barrier
HER-096, an optimized peptidomimetic compound, has been shown to cross the blood brain barrier (BBB) in animals.
The key advantage of HER-096 over CDNF is the possibility to administer less invasively, for example by a subcutaneous injection (similar to, e.g., insulin injections) to reach the brain tissue. Because of the subcutaneous route of administration, HER-096 can be administered also to subjects with earlier stage of Parkinson’s disease – a major benefit as in earlier stage of disease there are more dopamine producing nerve cells in the brain to be rescued.
Herantis’ goal is to start clinical development with HER-096 in 2023. If successful, HER-096 would have potential to become a multi-billion-euro disease-modifying CNS drug.
While HER-096 is a novel drug candidate and requires a complete preclinical development program, its development has significantly benefitted from the methods and knowledge cumulated in the development of CDNF. The early-stage development of HER-096 is markedly faster than it was for CDNF; the objectives for year 2022 are to complete the non-clinical program for this compound (CTA filing) and prepare for a Phase 1 clinical study to start in 2023.