Through its BRIDGES programme, the Luxembourg National Research Fund (FNR) is supporting Transcend-PD1, a collaborative project between the Luxembourg Centre for Systems Biomedicine (LCSB) and Accure Therapeutics, a translational R&D biotech in neuroscience based in Barcelona. Successfully launched in 2024, Transcend-PD aims to assess the efficacy of ACT-02, a drug candidate from Accure Therapeutics’ portfolio, in models developed by the Translational Neuroscience group at the LCSB. The 480.000€ grant, including monetary and in kind contributions, awarded to this public-private partnership will help evaluate the potential of this new compound in addressing critical unmet needs in Parkinson’s disease.
ACT-02 is a novel compound with disease-modifying potential. It inhibits the enzyme Prolyl Endopeptidase (PREP), a promising but previously overlooked target in Parkinson’s disease. This drug candidate has demonstrated properties to improve cognition while reducing motor symptoms, neuroinflammation and alpha-synuclein aggregation in preclinical models of brain disorders. The ACT-02 program is currently at an advanced preclinical stage, supported by comprehensive positive efficacy data in in vivo Parkinson’s models and by toxicology and safety pharmacology data sets.
“This collaboration will allow us to get further insights into Parkinson’s pathophysiology by understanding the mechanisms of action of ACT-02, which targets pathways involved in mitochondrial dysfunction and alpha-synuclein accumulation, two cardinal features of the disease,” details Dr Giuseppe Arena, who was a senior research scientist in the Translational Neuroscience group and has now taken on a scientific advisor role in Transcend-PD.
The LCSB offers an ideal setting to assess the efficacy of ACT-02, thanks to a range of different cellular models of Parkinson’s disease developed for translational studies. These serve as a robust, clinically-relevant platform for drug testing in patient-derived cells. Using neuronal models from carefully selected Parkinson’s patients will strengthen patient selection and stratification, enhancing the translational potential of ACT-02. “These cellular models will allow us to narrow down the subset of patients most likely to respond positively to the treatment in future clinical trials,” continues Dr Arena. A hurdle that many previous treatments have failed to overcome due to the heterogeneity of the disease.
“The goal of our research is ultimately to help develop new disease-modifying therapeutic strategies for Parkinson’s disease, so it is great to see our expertise with human induced pluripotent stem cells (iPSC)-derived neurons being used to test a new orally active drug candidate,” says Prof. Rejko Krüger, head of the Translational Neuroscience group. “We are looking forward to extending our highly translational iPSC work to guide the next clinical trial phases for ACT-02.”
“We are grateful to the FNR for their support of our ACT-02 asset. This grant is a significant step forward in the development of our first-in-class drug candidate,” concludes Dr Laurent Nguyen, co-founder and CEO at Accure Therapeutics. “With ACT-02, we aim to develop a game changer drug to treat patients suffering from Parkinson’s disease.”
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1 Pharmacological targeting of Prolyl Endopeptidase in human induced pluripotent stem cells-derived neurons for the development of precision medicine therapeutic strategies in Parkinson’s disease (Transcend-PD)