Phase III REGAIN trial data of Soliris in myasthenia gravis published in Muscle & Nerve journal

Alexion Pharmaceuticals announced the publication of data from an interim analysis of the Phase III open-label extension study (ECU-MG-302) of REGAIN (ECU-MG-301) evaluating the long-term efficacy and safety of Soliris (eculizumab) for the treatment of adult patients with anti-acetylcholine receptor (AChR) antibody-positive refractory generalized myasthenia gravis (gMG). Published in Muscle & Nerve, the results indicate that the improvements demonstrated during the initial six-month duration of the double-blinded Phase III REGAIN trial, were sustained over a treatment period of three years. Additionally, patients who had previously been treated with placebo in REGAIN showed rapid and significant improvement upon starting treatment with Soliris in the open-label extension (OLE). At the time of data analysis more than 55% of patients enrolled in the OLE showed clinically meaningful response, based on the MG-Activities of Daily Living scale (MG-ADL), and the majority of patients (56%) achieved minimal manifestations or pharmacological remission.

The data from the OLE also confirm that the safety profile of Soliris , when used to treat patients with anti-AChR antibody-positive gMG, is consistent with its safety profile seen in REGAIN. No cases of meningococcal infection were reported by the interim data cut-off [one case, which was resolved with antibiotic treatment, occurred after this date], and the most common adverse events were headache (37.6% of patients) and nasopharyngitis (31.6%). The most common serious adverse event was myasthenia gravis worsening (12.8% of patients). When compared with data from the year prior to the start of REGAIN, patients in the OLE experienced significant reductions in rates of exacerbations (75% reduction) and hospitalization (83% reduction).