US drugmaker Alexion Pharmaceuticals (Nasdaq: ALXN) has missed its primary endpoint in a Phase III trial of Soliris (eculizumab) in patients with refractory generalized myasthenia gravis (gMG).
The news is a setback for the company and for those affected by this ultra-rare segment of MG, which is a debilitating, complement-mediated neuromuscular disease in which patients have largely exhausted conventional therapy and continue to suffer profound muscle weakness throughout the body.
In the REGAIN study, the primary efficacy endpoint of change from baseline in myasthenia gravis-activities of daily living profile (MG-ADL) total score, a patient-reported assessment, at week 26, did not reach statistical significance, as measured by a worst-rank analysis.
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