A phase 2 clinical trial of venglustat in Parkinson’s disease patients has failed, prompting Sanofi to stop work in the indication. Sanofi disclosed the setback alongside news that it is stopping work on a clutch of other midphase programs.
Venglustat represented a novel approach to the treatment of Parkinson’s. Sanofi advanced the oral CNS-penetrating glucosylceramide synthase inhibitor into phase 2 in Parkinson’s on the strength of evidence it may help the subset of patients with a mutation in the GBA gene. The mutation, which is seen in up to 10% of patients, is associated with earlier onset of disease and worse outcomes such as cognitive impairment and rapid disease progression.
Sanofi designed the two-part, 270-subject clinical trial to determine whether daily dosing with venglustat drives improvements on the Movement Disorder Society Unified Parkinson's Disease Rating Scale Part II and III. Friday, Sanofi said the clinical trial missed its primary endpoint and work in the indication was stopped.
The setback still leaves Sanofi with chances to get venglustat to market. Sanofi is enrolling, or set to start enrolling, patients in several phase 2/3 clinical trials. Those studies are targeting autosomal dominant polycystic kidney disease, late-onset GM2 gangliosidosis and Gaucher disease.
Sanofi disclosed the failure of venglustat in Parkinson’s in fourth-quarter results that featured several other updates to its midphase pipeline. The Big Pharma has decided against taking IL4xIL13 bispecific romilkimab forward as a treatment for systemic scleroderma. Sanofi originally took the monoclonal antibody into the clinic in idiopathic pulmonary fibrosis but pulled the plug in that indication after a phase 2 found it had no effect on lung function.
Itepekimab, an anti-IL-33 antibody Sanofi is developing with Regeneron, has also hit a setback. After running a phase 2 that gave itepekimab in combination with Dupixent, Sanofi has decided not to take the drug forward in asthma. Enrollment in a phase 3 chronic obstructive pulmonary disease trial got underway late last year.
Finally, Sanofi stopped an assessment of its anti-CD38 antibody Sarclisa in combination with PD-1 checkpoint inhibitor Libtayo in lymphoma. Sanofi initiated a clinical assessment of the combination in the indication in 2018, having previously begun trials targeting diseases including multiple myeloma, prostate cancer and non-small cell lung cancer.