July 22, 2019
3 min read

New treatments emerging to prevent neuromyelitis optica relapse

Genentech and Alexion have both released promising data for drugs aimed at fighting the autoimmune inflammatory disorder.

You've successfully added to your alerts. You will receive an email when new content is published.

Click Here to Manage Email Alerts

We were unable to process your request. Please try again later. If you continue to have this issue please contact customerservice@slackinc.com.

Two new studies have shown promising data supporting the use of eculizumab or satralizumab to significantly reduce the risk for neuromyelitis optica spectrum disorder relapses, which can leave patients either blind or paralyzed.

Genentech recently presented data from its SAkuraSky phase 3 multicenter, randomized, double-masked, placebo-controlled study evaluating the efficacy and safety of satralizumab added to baseline immunosuppressant therapy in patients with neuromyelitis optica spectrum disorder (NMOSD).

In addition, researchers from the Mayo Clinic published the findings of the Prevention of Relapses and Evaluation of Eculizumab in NMOSD Treatment (PREVENT) study, supported by Alexion Pharmaceuticals, in the New England Journal of Medicine, which evaluated the safety and efficacy of eculizumab added to current immunosuppressive therapy to treat NMOSD.

Patients taking either medication experienced a significantly lower risk for NMOSD relapse compared with a placebo.

Alexion’s eculizumab product, Soliris, was FDA approved in June as an injection for intravenous use for treatment of NMOSD in adult patients who are anti-aquaporin-4 (AQP4) antibody positive.


The PREVENT study, a randomized, double-masked, time-to-event trial funded by Alexion, included 143 adult patients randomized in a 2:1 ratio to receive either eculizumab or a match placebo. Patients in the eculizumab cohort began at a dose of 900 mg weekly for the first four doses starting on day 1, followed by 1,200 mg every 2 weeks starting at week 4, according to the study.

The primary efficacy endpoint was the first adjudicated relapse. Ninety-six patients received eculizumab, and 47 received placebo. In the eculizumab group, three patients experienced an adjudicated relapse compared with 20 in the placebo group. The median time until the first adjudicated relapse in the eculizumab group was not reached, while those in the placebo group experienced a median adjudicated relapse at 103 weeks, according to the study.

“Compared with placebo, eculizumab showed a highly significant benefit in preventing attacks in patients with AQP4-IgG seropositive NMOSD, whether patients were on another immunosuppressive therapy or not. The magnitude of the therapeutic effect is very strong,” Dean M. Wingerchuk, MD, a Mayo Clinic neurologist and senior study author, told Ocular Surgery News.

Only 3% of patients in the eculizumab treatment arm had attacks during the study compared with 43% of those in the placebo group, Wingerchuk said.

The study is the first international, multicenter, randomized controlled therapeutic trial completed for NMOSD, he said.

“Its results confirm the importance of the complement system in AQP4-IgG seropositive NMOSD and provide hope for patients with this disease,” Wingerchuk said.


Satralizumab treatment showed a 62% reduction in the risk for relapse compared with placebo in patients with NMOSD, including AQP4-IgG seropositive and AQP4-IgG seronegative patients, Hideki Garren, MD, PhD, global head of multiple sclerosis and neuroimmunology at Genentech, told OSN.


“A single relapse can leave patients with devastating blindness or paralysis. These data are promising and add to our belief that, if approved, satralizumab could be efficacious across a broad NMOSD patient population, including adolescents over the age of 12, whether given as a monotherapy or add-on treatment,” he said.

Subjects randomly received satralizumab 120 mg subcutaneously or placebo administered at weeks 0, 2 and 4 and every 4 weeks thereafter as an add-on to their baseline treatments of immunosuppressants and/or corticosteroids. The primary endpoint was time to first protocol-defined relapse.

In a predefined subgroup analysis, satralizumab showed a 79% reduction in the risk for relapse in AQP4-IgG seropositive patients compared with placebo plus baseline therapy, Garren said. At weeks 48 and 96, 91.5% of patients in the satralizumab cohort remained relapse-free compared with 59.9% and 53.3% of the placebo cohort, respectively. – by Robert Linnehan

Disclosures: Garren reports he is the global head of multiple sclerosis and neuroimmunology at Genentech. Wingerchuk reports he receives grants from Alexion Pharmaceuticals and personal fees from MedImmune, Celgene, Novartis, Ono Pharmaceutical, BrainStorm Therapeutics, Caladrius and Biogen.