Ultomiris Met Primary Endpoint in Phase 3 Trial in Adults with Rare Autoimmune Condition NMOSD

Rare Daily Staff

AstraZeneca’s rare disease subsidiary Alexion report that top line results from its open-label phase 3 CHAMPION-NMOSD trial showed that Ultomiris achieved a statistically significant and clinically meaningful reduction in the risk of relapse in adults with neuromyelitis optica spectrum disorder, a rare, autoimmune condition.

Photo: Marc Dunoyer, CEO of Alexion

The study involved participants with anti-aquaporin-4 (AQP4) antibody-positive (Ab+) neuromyelitis optica spectrum disorder and used an external placebo arm from the pivotal Soliris PREVENT clinical trial as a comparator.

Ultomiris met the primary endpoint of time to first on-trial relapse, as confirmed by an independent adjudication committee. The company noted that no relapse was observed in 58 patients over a median treatment duration of 73 weeks.

The safety and tolerability of Ultomiris in the Champion-NMOSD trial were consistent with previous clinical studies and other approved indications. Fifty-six patients are continuing to receive treatment in a long-term extension period, which is ongoing.

Neuromyelitis optica spectrum disorder (NMOSD) is a rare and devastating autoimmune disease that affects the central nervous system, including the spine and optic nerves. Most people living with NMOSD often experience unpredictable relapses, a new onset of neurologic symptoms or worsening of existing neurologic symptoms, also referred to as attacks, which tend to be severe and recurrent and may result in permanent disability.

Ultomiris is a long-acting C5 complement inhibitor that offers immediate, complete, and sustained complement inhibition. It works by inhibiting the C5 protein in the terminal complement cascade, a part of the body’s immune system. When activated in an uncontrolled manner, the complement cascade over-responds, leading the body to attack its own healthy cells. Ultomiris is administered intravenously every eight weeks or, for pediatric patients less than 20 kg, every four weeks, following a loading dose.

“Soliris established the role of complement inhibition in preventing relapses in NMOSD, and with Ultomiris, we continue to innovate for patients with a more convenient every eight-week dosing schedule,” said Marc Dunoyer, CEO of Alexion “These trial results show that ULTOMIRIS may help patients move towards eliminating relapses, which is an important advancement in the treatment of NMOSD.”

The data will be presented at a forthcoming medical meeting and submitted to global health authorities as rapidly as possible to bring forward ULTOMIRIS to the NMOSD community.