Study Rationale: Parkinson’s disease (PD) is a complex neurodegenerative disorder marked by the loss of dopaminergic neurons, however the underlying exact reasons are still not fully understood. Current treatments focus on alleviating some of the symptoms of the disease, but do not stop disease progression. ACT-02, a new drug developed by Accure Therapeutics, offers a more comprehensive approach by addressing several key aspects of PD, such as alpha-synuclein aggregation, oxidative stress (cell respiration dysfunction) and inflammation. This multi-target strategy not only impacts on slowing disease progression but also improves motor and non-motor symptoms at the same time, offering new hope for managing PD more effectively.
Hypothesis: We hypothesize that inhibition of prolyl endopeptidase (PREP), an enzyme involved in protein aggregation and oxidative stress among other hallmarks of PD, will prevent neuronal death. ACT-02 treatment is expected to simultaneously improve motor and non-motor symptoms while slowing down disease progression, offering a comprehensive therapeutic approach for the treatment of PD.
Study Design: We aim to extend the tissue analysis and validate biomarkers in existing serum and brain tissue samples from transgenic animals (line 61; a reference model of PD), where we previously showed clear improvement of motor and non-motor symptoms, as well a decrease in α-synuclein aggregation and neuroinflammation, after short (1-month) ACT-02 treatment. These promising results support the design of a second efficacy study to assess the impact of longer/term ACT-02 treatment, to support phase II clinical trials. Simultaneously, we plan to complete the remaining experiments required for filing the Investigational New Drug (IND) to enable the Phase I clinical trial within the next 12 months.
Impact on Diagnosis/Treatment of Parkinson’s disease: The ACT-02 program offers a unique and distinct approach for PD treatment by targeting multiple pathways involved in the disease. ACT-02 treatment allows to slow disease progression and address both motor and non-motor symptoms, offering a broader strategy compared to therapies that focus on only one pathway.
Next Steps for Development: Having completed most of the regulatory and proof-of-concept pre-clinical studies required to reach first-in-human trials already, with support from the MJFF we aim to bring ACT-02 to the clinic within 12 months. Additional funding is needed to test the new formulation prototype and conduct longer efficacy study to support a Phase II clinical trial.