Encouraging data from the MS-SPI trial were presented on April 21 at the annual meeting of the American Academy of Neurology. The study tested the efficacy of MD1003, a pharmaceutical-grade biotin, administered at a dosage of 300 mg/day in patients with “not active” progressive multiple sclerosis (MS), for which there is no approved treatment.
According to the drug’s developer (Paris-based MedDay Pharmaceuticals), MD1003 offers a unique mechanism of action involving two MS targets: 1) it activates the Krebs cycle, the main route for energy production that protects against axonal degeneration, and 2) it potentially activates acetyl-CoA carboxylases (ACC1 and ACC2), the rate-limiting enzymes in the synthesis of fatty acids required for myelin repair.
The MS-SPI study was designed to assess the ability of MD1003 to reverse disease progression in patients with “not active” progressive MS. Patients included in this study had to demonstrate disability progression during the previous two years with no evidence of inflammatory activity. The patients were randomly assigned to receive either MD1003 (n = 103) or placebo (n = 51) for 12 months, followed by a 12-month extension phase, during which all of the patients received MD1003. The study’s primary endpoint was the proportion of patients demonstrating the reversal of disability on either the Extended Disability Status Scale or the timed 25-foot walking distance after nine months of treatment (M9), confirmed at 12 months (M12), which equated to confirmed reversal of disability progression.
The study’s primary endpoint was met, with 13% of the patients in the MD1003 arm showing confirmed reversal of progression at M9 (confirmed at M12), compared with none of the patients in the placebo arm (P = 0.0051). During the 12-month extension phase, patients initially treated with MD1003 exhibited sustained improvement versus baseline, with 13% of patients showing improvement at M18 (confirmed at M24) and 15% of patients showing improvement at M24. When patients in the placebo group were switched to MD1003 for the extension phase, the proportions of responders reached 7% at M18 (confirmed at M24) and 12% at M24, demonstrating that treatment with MD1003 reversed progression in some patients switched to MD1003.
In a separate study, MD1003 was investigated in MS patients who had experienced fixed visual loss (greater than or equal to six months) after acute optic neuritis during the previous three years (nonprogressive chronic optic neuropathy; n = 62), as well as in MS patients who showed progressive visual loss at two different visits during the previous three years (progressive optic neuropathy; n = 31). In the 24-week placebo-controlled phase, 65 patients received MD1003 and 28 received placebo. This was followed by a 24-week extension phase, during which all of the patients (n = 92) received MD1003. The study’s primary endpoint was the mean change from baseline in 100% contrast visual acuity in the selected eye (defined as the eye with the worst visual acuity and acute or progressive worsening during the three years before study entry) at 24 weeks.
The results showed that patients who received MD1003 improved slightly more than patients who received placebo (an average of 3.1 letters in the MD1003 arm versus 1.8 letters in the placebo arm), but this difference did not reach statistical significance. Patients in both the MD1003 and placebo groups continued to improve during the extension phase (a mean of 4.25 letters in the initial MD1003 arm versus 4.0 letters in the placebo-treated patients switched to MD1003).
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