Alexion Pharmaceuticals, Inc. (Nasdaq: ALXN) today announced that researchers have presented data from a single-arm, open-label, investigator-initiated Phase 2 study of eculizumab (Soliris ®) as an investigational therapy in 14 patients with severe, relapsing neuromyelitis optica (NMO), a life-threatening, ultra-rare neurological disorder. The study met its primary efficacy endpoint with high degrees of clinical and statistical significance. Clinically and statistically significant improvements were also observed in key secondary endpoints. Data were presented today at a Scientific Symposium Oral Session at the American Neurological Association annual meeting in Boston, Mass. 1
NMO is a devastating, life-threatening, ultra-rare neurological disease that leads to severe weakness, paralysis, respiratory failure, loss of bowel and bladder function, blindness and premature death. 2-4 In patients with NMO, uncontrolled complement activation causes destruction of myelin-producing cells, leading to severe damage to the central nervous system (CNS), including the spinal cord and optic nerve. 5-7 Patients with NMO have a life-long exposure to the uncontrolled complement activation due to chronic autoimmune attack, and most patients experience an unpredictable, relapsing course of disease with cumulative disability, as each attack adds to the neurologic disability. 3,8,9 Fifty percent of relapsing NMO patients have been reported to sustain permanent severe disability, including paralysis and blindness, within 5 years of disease onset. 10 Most NMO-related deaths result from respiratory complications from NMO attacks 10,11; in one report, 30% of patients died within 5 years of disease onset. 10
Eculizumab is approved in over 40 countries as a treatment for patients with paroxysmal nocturnal hemoglobinuria (PNH) and in the United States and European Union for patients with atypical hemolytic uremic syndrome (aHUS). PNH and aHUS are both debilitating and life-threatening ultra-rare disorders caused by chronic, uncontrolled complement activation. Eculizumab is not approved for the treatment of NMO in any country and was used in the reported study on an investigational basis.