Sanofi Genzyme is now recruiting participants for a Phase 2 trial to study the safety and efficacy of GZ/SAR402671 for treating Parkinson’s disease (PD) in patients carrying a mutation in the GBA gene — the most common genetic risk factor for PD.
Such mutations interfere with the production of the encoded protein, called glucocerebrosidase, which leads to the accumulation of fat molecules (glycosphingolipids) within cells.
Mutations in both copies of the GBA gene cause Gaucher disease, an illness with multiple symptoms that may include severe neurological deficits. But a mutation in only one of the gene’s copies may predispose individuals for PD. Between 5 and 10 percent of PD patients carry a mutation in this gene.
“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,” Anthony Schapira, MD, the trial’s principal researcher, said in a news release. “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.”
The MOVES-PD trial (NCT02906020) was designed to test whether GZ/SAR402671 can reduce the production and consequent accumulation of glycosphingolipids in patients with a single mutated copy of the GBA gene.
The study will enroll more than 200 patients worldwide. In its first part, researchers will assess the safety and tolerability of oral GZ/SAR402671 compared to a placebo. The second part will determine the efficacy of the treatment.
Researchers will then assess the change in score from baseline to week 52 (the study’s primary point) in the Movement Disorder Society Unified Parkinson’s Disease Rating Scale Part II and III, a measure of PD progression. This measure includes the patients’ self-report 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 Parkinson’s disease,” said Tanya Fischer, MD, PhD, Sanofi’s global project head of early development for PD and movement disorders. “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.”