Novel treatment will be tested in patients with Parkinson’s disease who have a GBA mutation.
Sanofi Genzyme recently initiated a phase 2 clinical trial evaluating an investigational oral drug in patients with Parkinson’s disease. Included in the study are patients who carry a single copy of a genetic mutation known to increase the risk of Parkinson’s disease.
The clinical trial will examine the drug’s dynamics, efficacy, and safety, and is the first industry-sponsored phase 2 study that includes a genetically defined group of patients with Parkinson’s disease, according to a press release.
Parkinson’s disease is a degenerative disorder that affects more than 1 million patients in the United States alone. Approximately 5% to 10% of patients carry a mutated glucocerebrosidase (GBA) gene that allows glycosphingolipids to build up in cells, Sanofi reported.
In the MOVES-PD study, the authors aim to determine whether GZ/SAR402671 can effectively reduce the creation of glycosphingolipids in patients with Parkinson’s disease.
“Patients with Parkinson’s disease and a GBA gene mutation are predisposed to develop motor symptoms at a younger age, have a higher prevalence of cognitive impairment and undergo more rapid disease progression,” said lead principal investigator Anthony Schapira, MD, DSc, FRCP, FMedSci, head of Department of Clinical Neurosciences, UCL Institute of Neurology. “Investigating a targeted therapy for these patients is an important first step in addressing the serious unmet needs these patients and their families face in managing Parkinson’s disease.”
Sanofi reported that the clinical trial will be split in 2 phases: a dose escalation study followed by an efficacy and safety study. More than 200 patients worldwide will be enrolled in the MOVES-PD clinical trial.
The primary endpoint of the study is to determine the change from baseline in disease progression, as measured by the Movement Disorder Society Unified Parkinson’s Disease Rating Scale Part 2 and 3. This scale includes self-reporting about daily activities and motor experience, and a motor evaluation scored by a clinician, according to Sanofi.
The company has been studying the GBA mutation for more than 30 years. While patients who have a single mutated copy tend to develop Parkinson’s disease, those with 2 mutated copies are at risk of Gaucher disease.
Gaucher disease causes a buildup of lipid cells that is characterized by bruising, fatigue, anemia, low blood platelets, bone and joint pain, enlargement of liver and spleen, seizures, and severe coordination problems. Sanofi created the world’s first treatment for Gaucher disease, and remains committed to creating novel treatments for patients with GBA mutation-related diseases, including Parkinson’s disease, according to the press release.
“We are excited to be able to bring the results of our many years of research in GBA gene mutations to a new therapeutic area with the potential to benefit patients with Parkinson’s disease,” said Tanya Fischer, MD, PhD, global project head of Early Development for Parkinson’s Disease and Movement Disorders, Sanofi R&D. “We look forward to evaluating whether this molecule, which has been shown to cross into the brain in preclinical studies, may positively impact the devastating neurologic effects of this disease.”