Genentech: Pivotal Phase 3 Data for Neuromyelitis Optica Spectrum Disorder (Devic’s Disease)
Genentech presented new pivotal phase 3 data for the rare autoimmune disease neuromyelitis optica spectrum disorder (NMOSD), also known as Devic’s Disease, for which there are no approved treatments.
NMOSD is a rare, lifelong and debilitating autoimmune disease of the central nervous system, which primarily damages the optic nerve and spinal cord, causing blindness, muscle weakness and paralysis. People with NMOSD experience unpredictable, severe relapses directly causing accumulating, permanent, neurological damage and disability. In some cases, relapses can result in death. Due to the involvement of the optic nerve and the central nervous system, NMOSD patients are often under the care of both neurologists and ophthalmologists. Approximately 15,000 people in the U.S. are living with the condition.
The SAkuraSky data being presented today (abstract can be found here) show Genentech’s investigational medicine satralizumab demonstrated efficacy across a broad NMOSD patient population compared to placebo, as an add-on to baseline immunosuppressants and/or corticosteroids, according to a Genentech statement.
Key highlights from the study presented include:
Satralizumab demonstrated a 62 percent reduction in the risk of relapses, compared to placebo, in patients with NMOSD including AQP4-IgG seropositive and AQP4-IgG seronegative patients, when used as an add-on to baseline therapy;
Satralizumab showed a 79 percent reduction in the risk of relapses in AQP4-IgG seropositive patients compared to placebo plus baseline therapy in a pre-defined sub-group analysis.
Regarding safety, the proportion of patients experiencing adverse events and serious adverse events, including serious infections and neoplasms, was similar in patients treated with satralizumab or placebo, with or without concomitant immunosuppressants and steroids.
The SAkuraSky study adds to a growing body of evidence that satralizumab could be efficacious across a broad NMOSD patient population, including adolescents over the age of 12, whether given as an add-on treatment or monotherapy. The company is planning to present additional monotherapy data later in the year and continues to work closely with regulators to bring this medicine to patients as quickly as possible. Satralizumab was granted Breakthrough Therapy Designation by the FDA in December 2018.
Genentech medicines are currently approved for two other rare, ophthalmic conditions. Actemra was approved to treat giant cell arteritis (GCA) is 2017 and Lucentis was approved for the treatment of myopic choroidal neovascularization (mCNV) that same year.