Alexion starts phase 3 trials for two ultra-rare diseases

Alexion Pharmaceuticals has announced the initiation of two Phase 3 trials of ALXN1210, a highly innovative, longer-acting anti-C5 antibody that inhibits terminal complement.

The first trial is a Phase 3 open-label, multinational, active-controlled study of ALXN1210 compared to eculizumab (Soliris) in complement inhibitor treatment-naïve patients with paroxysmal nocturnal hemoglobinuria (PNH), said the company in its press release.

Furthermore, Alexion has accelerated the initiation of a registration trial of ALXN1210 in patients with atypical hemolytic uremic syndrome (aHUS). This second trial is a Phase 3, open-label, single arm, multinational trial to evaluate the safety and efficacy of ALXN1210 in complement inhibitor treatment-naïve adolescent and adult patients with aHUS. Both studies will evaluate ALXN1210 administered intravenously every eight weeks. Alexion expects to begin enrolling patients into these trials later this year, and plans to initiate a Phase 3 trial of ALXN1210 in pediatric patients with aHUS in 2017.

Alexion has also commenced dosing of a new formulation of ALXN1210 administered subcutaneously in healthy volunteers in a Phase I study.

PNH is a debilitating, ultra-rare and life-threatening blood disorder characterized by complement-mediated hemolysis (destruction of red blood cells). aHUS is a genetic, chronic, ultra-rare disease associated with vital organ failure and premature death. Both PNH and aHUS are caused by chronic uncontrolled complement activation.

“With more than 20 years of expertise in the discovery and development of complement inhibitors, our ongoing commitment is to bring even higher levels of innovation to patients with devastating ultra-rare diseases,” said Martin Mackay, Ph.D., Executive Vice President and Global Head of R&D at Alexion. “We are very pleased with our agreement with global regulators to progress ALXN1210 into Phase 3 studies in patients with PNH and aHUS and are now working with investigators to enroll patients into the registration studies with urgency.”