The second pivotal phase 3 trial (SPI2) (NCT02936037) of investigational high-dose biotin (MD1003, MedDay Pharmaceuticals, Boston, MA) did not improve disability for individuals with active secondary progressive MS (SPMS). There were no treatment-emergent safety signals. The SPI2 trial was designed to confirm the results of the first positive phase 3 study, MS-SPI, in progressive multiple sclerosis (MS).
“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,” commented Catherine Moukheibir, chief executive officer of MedDay Pharmaceuticals. “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. All were invaluable partners throughout the process of completing the SPI2 trial.”
“We will review the findings in detail to understand these outcomes to help inform future clinical research in progressive MS and other neurologic diseases,” commented Frédéric Sedel, MD, PhD, chief scientific officer and cofounder of MedDay Pharmaceuticals. “I remain confident of the importance of the neurometabolic approach to neurodegenerative diseases with high unmet medical need.”
The randomized, double-blind, and placebo controlled SPI2 trial evaluated safety and efficacy of t3 daily doses of 100 mg of MD1003 versus placebo in 642 participants with progressive MS without recent relapses, also called nonactive progressive MS. The primary endpoint for the study was reversal of functional disability as measured by the proportion of patients with an improvement in either the Expanded Disability Status Scale (EDSS) or in the time needed to walk 25 feet (TW25) over a 12-month time frame and confirmed at 15 months.
Divakar Mithal, MD, PhD, and Tracy Gertler, MD, PhD
Vishnu Anand Cuddapah, MD, PhD; Susan Matesanz, MD; Sabrina W. Yum, MD; Brenda L. Banwell, MD; and Donna Stephenson, MD