Soliris does not reach statistical significance in REGAIN study for refractory generalized myasthenia gravis- Alexion Pharma

Alexion Pharmaceuticals, Inc.announced topline results from the REGAIN study, a Phase III registration trial of eculizumab (Soliris) in patients with refractory generalized myasthenia gravis (gMG). Refractory gMG is an ultra-rare segment of MG�a debilitating, complement-mediated neuromuscular disease�in which patients have largely exhausted conventional therapy and continue to suffer profound muscle weakness throughout the body, resulting in slurred speech, impaired swallowing and choking, double vision, upper and lower extremity weakness, disabling fatigue, shortness of breath due to respiratory muscle weakness, and episodes of respiratory failure.

In the 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 (p=0.0698) as measured by a worst-rank analysis. The first prospectively defined secondary efficacy endpoint of change from baseline in Quantitative Myasthenia Gravis (QMG) total score, a physician-administered assessment of MG clinical severity, with eculizumab treatment compared to placebo at week 26, achieved a p-value of 0.0129 as measured by a worst-rank analysis. In addition, the second and third prospectively defined secondary efficacy endpoints of responder status in MG-ADL and QMG achieved p-values <0.05: the proportion of patients with at least a 3-point reduction in mg-adl total score and no rescue therapy from baseline to week 26 with eculizumab treatment compared to placebo achieved a p-value of 0.0229 and the proportion of patients with at least a 5-point reduction in qmg total score and no rescue therapy from baseline to week 26 with eculizumab treatment compared to placebo achieved a p-value of 0.0018.>

Alexion continues to analyze the data from the REGAIN study, and results will be presented on July 7, 2016, during the Hot Topics session at the 14th International Congress on Neuromuscular Diseases (ICNMD) in Toronto.

Comment; While the REGAIN study missed its primary endpoint, the results showed clinically meaningful improvements in MG-ADL ( Myasthenia Gravis-Activities of Daily Living Profile) and QMG (Quantitative Myasthenia Gravis ) measures in patients treated with eculizumab compared with placebo.