Soliris eculizumab: Phase II data

An investigator-initiated, single-arm, open-label, U.S. Phase II trial in 14 women with severe, relapsing neuromyelitis optica showed that Soliris significantly reduced the median annualized attack rate, the primary endpoint, from 3 attacks/patient pre-treatment to 0 attacks/patient after 12 months of treatment (p<0.0001). At 12 months, 12 patients were completely attack-free, while the other 2 patients had single possible relapses as reported by investigators - 1 patient had back pain without clinical or radiological evidence of inflammation of the spinal cord, while the other patient had new onset of visual blurring with a reduction in visual acuity. The visual blurring returned to baseline after IV immunoglobulin ( IVIG) treatment.

Soliris also significantly improved median Expanded Disability Severity Scale (EDSS) scores, a secondary endpoint, from 4.3 points pre-treatment to 3.5 points at 12 months (p<0.01). All patients experienced either improvement or stability in EDSS scores, ambulatory function and visual function. Soliris was well tolerated with headache, nausea and dizziness reported as the most common adverse events. During the first 3 months post-Soliris treatment, 2 patients experienced 3 severe relapses while continuing to receive immunosuppressive treatment. The trial enrolled patients who had experienced >=2 disease relapses in the preceding 6 months or 3 attacks in the preceding year. Patients received once-weekly 600 mg IV Soliris for the first 4 weeks, followed by a 900 mg dose at week 5 and a 900 mg maintenance dose every 2 weeks for 48 weeks. Data were presented at the American Neurological Association meeting in Boston. ...