A Phase 3 clinical trial intending to confirm the potential of MD1003, a high-dose biotin, in treating progressive multiple sclerosis (MS) patients is completely enrolled, MedDay Pharmaceuticals, the investigational therapy’s developer, announced.
The randomized, double-blind, and placebo-controlled study (NCT02936037), called SPI2, follows the previous Phase 3 trial (NCT02220933), called MS-SPI, that demonstrated MD1003 (100 mg capsule, three times a day) could slow or prevent further disease progression in patients with progressive MS.
MD1003 is a highly concentrated oral formulation of biotin that acts on different aspects of neurons’ metabolism to minimize the loss and promote the repair of the protective myelin sheath that is progressively destroyed in the course of MS. Like its predecessor, the SPI2 trial aims to determine whether MD1003 is able to slow or stop disease progression, particularly in gait and walking.
“The mechanism of action of our investigational product MD1003 has significant potential in the treatment of progressive multiple sclerosis. MedDay is fully committed to advance its clinical program as initially planned with a view of offering a new therapeutic alternative to patients,” Christian Chavy, chief executive officer of MedDay, said in a press release.
SPI2 enrolled a larger group of primary and secondary progressive MS patients (642 adults in total), who are relapse-free but show evidence of disease progression, the company reported. They will again take either MD1003 as a 100 mg capsule, or a placebo, three times a day.
The study runs for at least 15 months, then patients have the option of moving to a one-year and open-label extension, where all will be treated.
A primary trial goal is a treatment-induced slowing or stopping of disease progression, measured by changes from initial scores in either the Expanded Disability Status Scale (EDSS) or the time to walk 25 feet (TW25) test at one year, and confirmed at 15 months.
Secondary goals include time to disease progression — as measured by changes in EDSS, a clinical global impression of improvement assessed by the patient and an assigned physician, and mean changes in the TW25 test — between the study’s start and its conclusion at 15 months.
The trial is currently active in 90 sites worldwide — 48 in the U.S. and Canada, 39 in Europe, and three in Australia. MedDay expects first results to be available in early 2020.
The company is planning to file a New Drug Application (NDA) for MD1003 with the U.S. Food and Drug Administration (FDA) and a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) later that same year.