New interim analysis from a phase 2 study reveals promising outcomes for progressive multiple sclerosis patients treated with glatiramer acetate (GA) depot, developed by Mapi Pharma. The three-year data demonstrates disease stabilization and a favorable safety profile, marking a potential breakthrough for progressive MS treatment.
The study, presented at the 2025 ACTRIMS Forum in West Palm Beach, Florida, focused on patients with primary progressive MS (PPMS) aged 18-65 years. Participants showed remarkable stability in their disease progression, with mean Expanded Disability Status Scale (EDSS) scores remaining nearly unchanged from baseline (5.1) to three years (4.9). Notably, only one case of 12-week confirmed disability progression was reported during the entire study period.
Functional Outcomes and Disease Stability
The treatment demonstrated impressive results across multiple functional measures. According to Ehud Marom, president and CEO of Mapi Pharma, 89.7% of patients maintained stability in the 9-Hole Peg Test, while 79.3% showed stable results in the Timed 25-Foot Walk test over three years. Significantly, 69.0% of patients demonstrated no evidence of progression, showing neither clinical nor radiological signs of worsening disability.
Brain Volume Changes and MRI Findings
MRI volumetric analyses revealed progressive brain volume changes over the treatment period:
- Year 1: -0.77%
- Year 2: -1.46%
- Year 3: -2.14%
Neocortical brain volume changes were also tracked, showing:
- Year 1: -1.97%
- Year 2: -2.09%
- Year 3: -3.30%
Safety Profile and Dosing Considerations
The study evaluated two dosing regimens, with the 25mg dose showing a more favorable safety profile compared to the 40mg dose. Key safety findings include:
- 83% of adverse events were mild
- Lower adverse event rates in the 25mg group (0.20 vs 0.49)
- Reduced injection site reactions in the 25mg group (0.01 vs 0.15)
- No serious adverse events in the 25mg group
Development Status and Recent Regulatory Action
GA Depot, designed as a monthly intramuscular injection, represents an evolution of the approved glatiramer acetate (Copaxone). While the therapy recently received a Complete Response Letter from the FDA for relapsing MS, these new findings in progressive MS support continued development.
The treatment's potential is particularly significant given the limited therapeutic options for progressive MS patients. These results support the advancement to a global, phase 3 double-blinded study, potentially offering a new treatment avenue for this challenging form of MS.
