How does HER-096 work in the body?

The complex brain pathology underlying degeneration and death of dopamine neurons involves:

 

These different mechanistic aspects of the disease are intimately interlinked and may feed forward each other.

While the root cause of Parkinson’s disease still remains poorly understood, the therapeutic hypothesis of HER-096 is based on breaking this vicious cycle to protect dopamine neurons from further degeneration – and to promote their functional recovery.

HER-096 was developed based on the active site of cerebral dopamine neurotrophic factor (CDNF), an endogenous human protein that

  • Regulates the UPR pathway.
  • Protects neurons from cell death induced by, e.g., chronically elevated endoplasmic reticulum (ER) stress

 

HER-096 development is built on 15 years of research and development work related to CDNF’s role in neurodegeneration both in the academia and in the industry. This work has generated many important insights and data to help us understand how HER-096 works, and how cells and tissues respond to exposure to HER-096.

  • HER-096 is different from CDNF as it can effectively pass the blood-brain barrier (BBB) as demonstrated in several animal species. This allows easy peripheral administration of HER-096 as opposed to the complicated intracranial delivery that is required for the CDNF protein.
  • The combination of the disease-modifying potential of HER-096 with its capacity of BBB penetration makes it a compelling candidate to become a ground-breaking novel treatment for Parkinson’s disease, with potential for slowing down or stopping the disease process – not just treating the symptoms of the disease.

With the clinical trial application (CTA) regulatory approval of the Phase 1a in February 2023, an important milestone in HER-096 development was reached. Several years of intense preclinical development work had reached a point which allows moving forward to clinical development. To reach this point, we have demonstrated that HER-096 is safe and well tolerated in two animal species with the intended subcutaneous route of administration. Moreover, HER-096 has robust therapeutic effects and is modulating its target pathway in the brain in an animal model of Parkinson’s disease. We aim at conducting the Phase 1a clinical study during 2023 to demonstrate i) the safety and tolerability of a single subcutaneous dose of HER-096 in healthy volunteers, and ii) the BBB penetration with HER-096. Successful completion of the study would represent a significant milestone for the company.

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