Four years ago, MedDay Pharmaceuticals’ multiple sclerosis drug reversed the course of disease in a 150-patient pivotal study. The biotech set out to confirm those results in a much bigger phase 3 study, but it fell short. The treatment missed its endpoints in the study, which pitted it against placebo in 642 patients with non-active progressive multiple sclerosis—that is, patients who had not suffered recent relapses.
Patients received three 100-mg doses of the drug or placebo each day throughout the study. The investigators used the same tests as the earlier trial to assess the treatment: a measure of disability stemming from MS called the Expanded Disability Status Scale (EDSS) and the time it takes for a patient to walk 25 feet (TW25). The drug, MD1003, failed to beat placebo on both endpoints.
MD1003 also missed its secondary and exploratory endpoints, including the risk of patients’ disability getting worse, quality of life measures, and an assessment of how the treatment was working, done by each patient and his or her evaluating doctor.
“We are clearly disappointed that SPI2 did not meet its primary and secondary endpoints. Going forward, we will continue to evaluate the trial data and confer with regulators,” MedDay CEO Catherine Moukheibir said in a statement on Tuesday.
The plan is to review the data to improve its future R&D efforts in MS and other neurological diseases, MedDay Chief Scientific Officer Frédéric Sedel, M.D., Ph.D., added in the statement.
In patients with MS, the immune system attacks myelin, the fatty substance that coats and protects nerve fibers in the brain and spinal cord. Unlike other approaches that tweak the immune response, MD1003 targets metabolic pathways. It is designed to ramp up the Krebs cycle to provide more energy to demyelinated nerve fibers and boost the development of an insulating layer around those fibers.
“I remain confident of the importance of the neurometabolic approach to neurodegenerative diseases with high unmet medical need,” Sedel said.
MedDay is also developing MD1003 for Charcot-Marie-Tooth disease, a group of inherited conditions that damage the peripheral nerves, and hepatic encephalopathy, a loss of brain function stemming from severe liver disease.