CAMBRIDGE, Mass.--(BUSINESS WIRE)--Sanofi Genzyme, the specialty care global business unit of Sanofi,today announced the start of a Phase 2 trial of an investigational oral therapy for patients with Parkinsons disease who carry a single copy of a gene mutation that is the most common genetic risk factor for the disease. The trial will assess the drugs dynamics, efficacy and safety. This is the first industry-sponsored Phase 2 clinical trial in a genetically defined population of Parkinsons disease.
Parkinsons disease is a chronic, degenerative neurological disorder affecting an estimated one million people in the United States and more than five million people worldwide.1 An estimated 5 10% of Parkinsons disease patients carry a mutation of the glucocerebrosidase (GBA) gene that allows lipids called glycosphingolipids to build up in cells. The molecule being studied, GZ/SAR402671, reduces the production of glycosphingolipids.
Patients with Parkinsons 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, explains Anthony Schapira, MD, DSc, FRCP, FMedSci, Head of Department of Clinical Neurosciences, UCL Institute of Neurology and Lead Principal Investigator for the study. 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 Parkinsons disease.
The clinical trial, known as MOVES-PD, will be run in two phases: a dose escalation study followed by a study of efficacy and safety. The randomized, double blind study will enroll more than 200 patients at trial sites around the world. The primary endpoint of the study is the change in score from baseline in a scale commonly used to measure Parkinsons disease progression known as the Movement Disorder Society Unified Parkinson's Disease Rating Scale Part II and III. This includes self-evaluation of daily life activities and motor experience, and a clinician-scored motor evaluation.
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 Parkinsons disease, said Tanya Fischer, MD, PhD, Global Project Head of Early Development for Parkinsons 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.
Sanofi has studied GBA gene mutations for more than 30 years. People with GBA mutations in both copies of the gene, as opposed to a single mutation in GBA Parkinsons disease, have Gaucher disease. Gaucher disease is a rare genetic disorder in which the buildup of a lipid in the cells leads to a broad spectrum of systemic manifestations including bruising, fatigue, anemia, low blood platelets, bone and joint pain, enlargement of liver and spleen, as well as neurological manifestations such as seizures and incoordination in severe forms.
Sanofi Genzyme introduced the worlds first treatment for Gaucher disease and Sanofi R&D remains committed to developing treatments for conditions associated with GBA mutations, including Gaucher disease and Parkinsons disease.
For more information on this trial, please visithttps://www.clinicaltrials.gov/ orhttps://www.clinicaltrialsregister.eu.
About SanofiSanofi, a global healthcare leader, discovers, develops and distributes therapeutic solutions focused on patients' needs. Sanofi is organized into five global business units: Diabetes and Cardiovascular, General Medicines and Emerging Markets, Sanofi Genzyme, Sanofi Pasteur and Consumer Healthcare.
Sanofi Genzyme focuses on developing specialty treatments for debilitating diseases that are often difficult to diagnose and treat, providing hope to patients and their families.
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1 Michael J. Fox Foundation. 2016. Understanding Parkinsons [ONLINE]. Available at: https://www.michaeljfox.org/understanding-parkinsons/i-have-got-what.php [Accessed 8 December 2016]
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