“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,” Catherine Moukheibir, CEO of MedDay Pharmaceuticals, said in a press release.
MD1003 is a highly concentrated oral formulation of biotin. According to the company, the investigational therapy acts on two fronts, targeting both neurodegeneration and demyelination — loss of myelin, the protective layer of nerve fibers. (Myelin loss is a hallmark of MS.)
The therapy has the potential to increase the production of myelin through activation of cellular processes in cells that produce myelin, called oligodendrocytes. Moreover, MD1003 also activates the Krebs cycle (a cycle that is the major source of energy in organisms), supporting the energy demands of demyelinated nerve fibers.
The previous MS-SPI Phase 3 trial (NCT02220933) assessed MD1003’s ability to improve disability of patients with progressive MS, with special focus on their walking ability. Patients were chosen randomly to take either the therapy or a placebo. After 36 months, 133 people had received MD1003.
Follow-up results showed that MD1003 significantly improved patients’ disability level (measured by the Expanded Disability Status Scale, or EDSS), and in their exercise capacity (assessed by the time it takes to walk 25 feet, TW25 test) at nine months, compared with placebo-treated patients. These benefits were maintained at 18 and 30 months.
After patients in the initial placebo group switched to the treatment group (at nine months), no differences were observed in EDSS scores between the treated and control groups at months 24 and 36.
However, those who received MD1003 in the first nine months had overall better cumulative results, suggesting that earlier treatment leads to lower disability, the researchers said.
At one year and follow-up, TW25 scores also were better in those who were initially treated with MD1003. In contrast, the performance of the group initially treated with a placebo worsened in the final year.
The SPI2 study, which was intended to confirm the results of MS-SPI trial, recruited a larger group of non-active progressive MS patients. A total of 642 adults, who were relapse-free but showed evidence of disease progression, were assigned randomly to take either an oral 100 mg capsule of MD1003 or a placebo, three times per day.
Primary endpoints (goals), once again, were the proportion of patients with an improvement in either the EDSS or TW25 — an overall measure of reversal of functional disability. Secondary endpoints included reduction in the risk of disability progression, clinical global impression scale of change – improvement (CGI-I score) evaluated by both the clinician and patient, and mean change in TW25.
MedDay’s announcement confirmed that both the primary and secondary endpoints of the trial were not achieved. No treatment-related safety adverse events (side effects) were reported.
Detailed results of the trial will be presented at the upcoming 2020 American Academy of Neurology (AAN) Annual Meeting, to be held in April in Toronto, Canada.
“We will review the findings in detail to understand these outcomes to help inform future clinical research in progressive MS and other neurological diseases,” said Frédéric Sedel, MD, PhD, chief scientific officer and co-founder of MedDay Pharmaceuticals. “I remain confident of the importance of the neurometabolic approach to neurodegenerative diseases with high unmet medical need.”
“We would like to thank our collaborators including the participating clinicians, medical staff and, most importantly, the patients for all of their efforts and participation in the trial,” Moukheibir said. “All were invaluable partners throughout the process of completing the SPI2 trial.”
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