The race to best treat a disease long confused with MS heats up with Roche's PhIII results
With inflamed myelin and frequent optic neuritis episodes, NMOSD patients were for years confused with MS patients. Now multiple treatments for the rare autoimmune disease, officially called neuromyelitis optica spectrum disorder, are out or nearing approval.
Roche just published Phase III data on its NMOSD drug satralizumab in the New England Journal of Medicine, the second positive round of results. Of the 41 NMOSD patients who received the IL-6 antibody, 89% didn’t experience a relapse after 48 weeks. Meanwhile, 66% of the 42 patients in the placebo were relapse-free at that point.
Unlike multiple sclerosis, where disability is caused by overall disease progression, NMOSD strikes through attacks or relapses that cumulatively disable patients. Enough relapses can eventually cause blindness or paraplegia. If you prevent the relapses, you can halt the disease.
After 96 and 144 weeks, 78% and 74% of the treatment arm were relapse-free. That compared with 59% and 49% in the control arm. These were similar to the numbers shown in a separate study unveiled in September.
The drug also showed a heightened response in patients with a specific biomarker, AQP4-IgG or aquaporin-4, which often appears in patients with the harshest symptoms. Nearly 4x as many AQP4 patients relapsed in the placebo arm as in the treatment arm.
Combined with the September results and an FDA breakthrough tag from 2018, the latest data should help usher Roche’s drug past US regulators.
It would join Alexion’s Soliris, which became the first drug specifically approved for NMOSD, also known as Devic’s Disease, in June. And it may follow another candidate in Viela Bio’s inebilizumab. The AstraZeneca spinout submitted a BLA in late August for inebilizumab based off of a pivotal showing 88% in the drug arm went without relapse.
Most NMOSD patients have used off-label Rituxan — a drug not specifically approved for the indication but one that’s staved off symptoms to a degree that some analysts doubt whether new therapies will be able to penetrate the market.
The question is whether the benefit of new treatments justifies the cost, SVB Leerink’s Geoffrey Porges wrote after the Soliris approval. For Soliris, the price in the first year is $700,000, compared to $60,000 for off-label Rituxan.
For Roche and Viela Bio, the pressure to price well will be even steeper. Over 96% of patients in the Soliris trial hadn’t relapsed after 144 weeks.