MOVES-PD was a global Phase 2 study to assess the efficacy and safety of a drug called venglustat in Parkinson’s patients who have a GBA-1 genetic mutation. The pharmaceutical company Sanofi conducted the trial to determine if venglustat could change the course of the disease. When the final data was collected, however, the results of the study indicated that the drug had no impact on slowing the progression of the condition.
Sanofi designed the MOVES-PD study, which involved 221 people with GBA-associated Parkinson’s being administered with either venglustat or a placebo treatment for one year, to determine if venglustat could improve performance of on clinical measures of disease progression (the Movement Disorder Society Unified Parkinson’s Disease Rating Scale, Parts II and III). Unfortunately, Sanofi have announced that the clinical trial has not demonstrated any improvements.
Significant insights have been learnt from the MOVES-PD study and these will be taken forward to help with future trials in GBA-associated Parkinson’s. Sanofi has pledged to learn as much as it can from this study and share their findings with the Parkinson’s research community for the benefit of the field and to inform future studies. They have planned to follow-up with participants over time and this data is crucial to better understand and explain the findings in more detail.
While the outcome of MOVES-PD was not what we had hoped, Sanofi remains committed to further analyses of the data and transparent communication with the Parkinson’s community. The support of researchers and contribution of patients worldwide demonstrates a strong spirit of collaboration, and we will continue to honor this in our work.Dr Pablo Sardi, R&D Director, Sanofi
Even though the venglustat trial has not been successful, the pipeline for GBA-associated Parkinson’s is rich with potential therapeutics targeting the GBA pathway, including:
Ambroxol, a repurposed drug, prioritised by Cure Parkinson’s International Linked Clinical Trials Committee (iLCT) in 2015 and commonly used in Europe as an anti-mucolytic respiratory medicine.
BIA 28-6156 is a GCase enzyme activator being developed by BIAL Biotech. After completing its Phase 1 safety clinical trial, it was prioritised by the iLCT committee in 2020 and will be moving into phase 2 trials in 2022.
PR001A is a gene therapy treatment being developed by Prevail Therapeutics. Gene therapy involves treating conditions with DNA rather than drugs, and the researchers are attempting to replace the faulty GBA-1 gene in the brains of people with GBA-associated Parkinson’s with a normal version of the gene. A five-year Phase 1/2a clinical trial is currently on-going in the U.S.
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