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Wednesday, Sep 9, 2020

New Data Show Genentech’s Enspryng Significantly Reduces Severity and Risk of Relapse in Neuromyelitis Optica Spectrum DIsorder

  • Enspryng lowered relapse severity in double-blind periods of SAkura Phase III studies
  • Pooled data from SAkura open-label extension (OLE) studies support continued effect of Enspryng reducing risk of relapse in the longer term
  • Ongoing data continues to show a favorable safety profile for Enspryng
  • Enspryng was recently approved by the U.S. Food and Drug Administration (FDA) for adults with anti-aquaporin-4 (AQP4) antibody positive NMOSD

South San Francisco, CA -- September 9, 2020 --

SOUTH SAN FRANCISCO, Calif. – September 9, 2020 – Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), will present new Enspryng (satralizumab-mwge) data on reducing relapse severity in the treatment of neuromyelitis optica spectrum disorder (NMOSD), a rare disease of the central nervous system. These data are being presented at MSVirtual2020, the 8th Joint Americas Committee for Treatment and Research in Multiple Sclerosis (ACTRIMS) - European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) Meeting, in addition to longer-term efficacy data supporting the continued effect of Enspryng on reducing the risk of NMOSD relapse, as well as its favorable benefit:risk profile.
 

“The data for Enspryng at MSVirtual2020 are promising and suggest it significantly reduces relapse severity and frequency, which are important goals of the treatment for people with NMOSD,” said Anthony Traboulsee, M.D., neurologist and professor, University of British Columbia, and Research Chair of the MS Society of Canada. “Enspryng, the first approved treatment for NMOSD that can be taken at home, offers favorable efficacy and safety, which are important for improved long-term outcomes.’’ 

 

In a post-hoc analysis of the Enspryng-treated group, the risk of severe relapse was reduced by 79% compared to placebo (5 of 27 [19%] vs. 12 of 34 [35%]), for patients across the double-blind periods of the SAkura studies. Preventing relapses, the most severe of which cause cumulative, irreversible, neurological damage and disability, is the primary goal for NMOSD treatment management. The patients treated with Enspryng were also less likely to require rescue therapy for a relapse compared with placebo (OR 0.46; 95% CI, 0.25–0.86, p=0.015). A relapse was categorized as severe if it resulted in a change of ≥2 points on the Expanded Disability Status Scale.
 

In a separate pooled analysis, Enspryng reduced the risk of relapse in the combined double-blind period and open-label extension (OLE) by 51% (HR, 0.49; 95% CI, 0.31–0.79; p=0.002) compared to those originally in the placebo group. This effect was more pronounced in aquaporin-4 antibody (AQP4-IgG) seropositive patients, who tend to experience a more severe disease course, with 66% reduction in risk of relapse (HR, 0.34; 95% CI, 0.19–0.62; p<0.001) compared to those originally in the placebo group.  


“The longer-term data for Enspryng further reinforce the previously observed efficacy of this medicine for this debilitating disorder that is often mistaken for multiple sclerosis,” said Levi Garraway, M.D., Ph.D., chief medical officer and head of Global Product Development. “Enspryng is the first and only FDA-approved subcutaneous, self-administered medicine for NMOSD and the first medicine for NMOSD that is designed to target the interleukin-6 receptor, which is believed to play a key role in the inflammation associated with this disorder.”
 

In the double-blind periods, infection rates were lower in the Enspryng-treated group compared to placebo in the SAkuraStar study (99.8 vs. 162.6 events/100 patient years [PY]), whereas infection rates did not differ between groups in the SAkuraSky study. Serious infection rates were comparable between both groups in each of the studies (SAkuraSky: 2.6 vs. 5.0 events/100PY; SAkuraStar: 5.2 vs. 9.9 events/100PY). Infection and serious infection rates for Enspryng-treated patients in the combined double-blind and OLE periods were consistent with those for Enspryng-treated patients in the double-blind portion in terms of the nature and rate of adverse events and did not increase over time.

 

Enspryng is approved in Canada, Japan, Switzerland and the U.S. Additional applications are under review with numerous regulators, including in the EU and China. Enspryng has been designated as an orphan drug in the U.S., Europe and Japan. In addition, it was granted Breakthrough Therapy Designation for the treatment of NMOSD by the FDA in December 2018.
 

About SAkuraStar and SAkuraSky in NMOSD

SAkuraStar is a Phase III multicenter, randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of Enspryng monotherapy administered to patients with neuromyelitis optica spectrum disorder (NMOSD). The primary endpoint is the time to first protocol-defined relapse (PDR), adjudicated by an independent review committee in the double-blind period. Results from the SAkuraStar study were presented at the 35th Congress of the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS), September 11-13, 2019, and were published in the May 1, 2020 edition of The Lancet Neurology.
 

Ninety-five adult patients were randomized to either of the following two treatment groups in a 2:1 ratio: Enspryng (120 mg) or placebo. Both treatments were administered subcutaneously at week 0, 2, and 4. The subsequent treatment was continued at 4-week intervals. The double-blind treatment period ended at 1.5 years after the enrollment of the last patient. After experiencing a PDR or upon completion of the study, patients in both groups were offered treatment with Enspryng in an open label extension (OLE) period. Patients with aquaporin-4 (AQP4) antibody positive or negative neuromyelitis optica (NMO, as defined by the diagnostic criteria in 2006) and those with AQP4 antibody positive NMOSD were enrolled. The number of AQP4 antibody negative patients was limited to approximately 33% of the total population of the study. Data have shown that AQP4 antibody positive patients may experience a greater likelihood of relapse and poorer long-term outcomes than AQP4 antibody negative patients.

 

SAkuraSky is a Phase III multicenter, randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of Enspryng added to baseline immunosuppressant therapy in patients with NMOSD. The primary endpoint was the time to first PDR as adjudicated by an independent review committee in the double-blind period. Results from the SAkuraSky study were published in the November 28, 2019 edition of the New England Journal of Medicine (NEJM).

 

Seventy-six adult patients were randomized to either of the following two treatment groups in a 1:1 ratio: Enspryng (120 mg) or placebo added to baseline therapy (azathioprine, mycophenolate mofetil and/or corticosteroids). Both treatments were administered subcutaneously at week 0, 2, and 4. The subsequent treatment was continued at 4-week intervals. The double-blind treatment period ended when patients experienced a PDR; the study ended when the total number of PDRs reached 26. After experiencing a PDR or upon completion of the study, patients in both groups were offered treatment with Enspryng in an OLE period. Patients with AQP4 antibody positive or negative neuromyelitis optica (NMO, as defined by diagnostic criteria in 2006) and those with AQP4 antibody positive NMOSD were enrolled. AQP4 antibody negative patients represented approximately 30% of the SAkuraSky study population.

 

About neuromyelitis optica spectrum disorder (NMOSD)

NMOSD is a rare, lifelong and debilitating autoimmune condition of the central nervous system that primarily damages the optic nerve(s) and spinal cord, causing blindness, muscle weakness and paralysis. People with NMOSD experience unpredictable, severe relapses directly causing cumulative, permanent, neurological damage and disability. In some cases, relapse can result in death. NMOSD affects over 10,000 people in Europe, up to 15,000 people in the United States and approximately 200,000 people worldwide. NMOSD can affect individuals of any age, race and gender, but is most common among women in their 30s and 40s, and appears to occur at higher rates in people of African or Asian background. There is some evidence that people of African or Asian descent may also experience a more severe disease course. 

 

NMOSD is commonly associated with pathogenic antibodies (AQP4) that target and damage a specific cell type, called astrocytes, resulting in inflammatory lesions of the optic nerve(s), spinal cord and brain. AQP4 antibodies are detectable in the blood serum of around 70-80% of NMOSD patients.

 

Although most cases of NMOSD can be confirmed through diagnostic tests, people living with the condition are still frequently misdiagnosed with multiple sclerosis. This is due to overlapping characteristics of the two disorders, including a higher prevalence in women, similar symptoms and the fact that both are relapse-based conditions.

 

About Enspryng™ (satralizumab-mwge)

Enspryng, which was designed by Chugai, a member of the Roche Group, is a humanized monoclonal antibody that targets interleukin-6 (IL-6) receptor activity. The cytokine IL-6 is believed to be a key driver in NMOSD, triggering the inflammation cascade and leading to damage and disability. Enspryng was designed using novel recycling antibody technology, which compared to conventional technology, allows for longer duration of the antibody and subcutaneous dosing every four weeks. 

 

Positive Phase III results for Enspryng, as both monotherapy and used concurrently with baseline immunosuppressant therapy, suggest that IL-6 inhibition is an effective therapeutic approach for NMOSD. The Phase III clinical development program for Enspryng includes two studies: SAkuraStar and SAkuraSky.

 

Enspryng is approved in the U.S., Canada, Japan and Switzerland. Applications are under review with numerous regulators, including in the EU and China. 

 

Enspryng has been designated as an orphan drug in the U.S., Europe and Japan. In addition, it was granted Breakthrough Therapy Designation for the treatment of NMOSD by the FDA in December 2018.

 

What is Enspryng?

Enspryng is a prescription medicine used to treat neuromyelitis optica spectrum disorder (NMOSD) in adults who are aquaporin-4 (AQP4) antibody positive.

 

It is not known if Enspryng is safe and effective in children.


Important Safety Information
 

Patients should not take Enspryng if they:

  • are allergic to satralizumab-mwge or any of the ingredients in Enspryng
  • have an active hepatitis B infection
  • have active or untreated inactive (latent) tuberculosis (TB)

 

Enspryng may cause serious side effects including:

  • Infections. Enspryng can increase risk of serious infections some of which can be life-threatening. Patients should speak with their healthcare provider if they are being treated for an infection and call right away if there are signs of an infection, with or without a fever, such as:
    • chills, feeling tired, muscle aches, cough that will not go away or a sore throat
    • skin redness, swelling, tenderness, pain or sores on the body
    • diarrhea, belly pain, or feeling sick
    • burning when urinating or urinating more often than usual

A healthcare provider will check for infection and treat it if needed before starting or continuing to take Enspryng

    • A healthcare provider should test for hepatitis and TB before initiating Enspryng
    • All required vaccinations should be completed before starting Enspryng. People using Enspryng should not be given 'live' or 'live-attenuated' vaccines. 'Live' or 'live-attenuated' vaccines should be given at least 4 weeks before a patient starts Enspryng. A healthcare provider may recommend that a patient receive a 'non-live' (inactivated) vaccine, such as some of the seasonal flu vaccines. If a patient plans to get a 'non-live' (inactivated) vaccine it should be given, whenever possible, at least 2 weeks before starting Enspryng
  • Increased liver enzymes. A healthcare provider should order blood tests to check patient liver enzymes before and while taking Enspryng. A healthcare provider will dictate how often these blood tests are needed. Patients should complete all follow-up blood tests as ordered by a healthcare provider. A healthcare provider may wait to start Enspryng if liver enzymes are increased
  • Low neutrophil count. Enspryng can cause a decrease in neutrophil counts in the blood. Neutrophils are white blood cells that help the body fight off bacterial infections. A healthcare provider should order blood tests to check neutrophil counts while a patient is taking Enspryng.
  • Serious allergic reactions that may be life-threatening have happened with other medicines like Enspryng. Patients should call their healthcare provider right away if they have any of these symptoms of an allergic reaction:
    • shortness of breath or trouble breathing
    • swelling of lips, face, or tongue
    • dizziness or feeling faint
    • moderate or severe stomach (abdominal) pain or vomiting
    • chest pain

 

Before taking Enspryng, patients should tell their healthcare provider about all of their medical conditions, including if they:

  • have or think they have an infection
  • have liver problems
  • have ever had hepatitis B or are a carrier of the hepatitis B virus
  • have had or have been in contact with someone with TB
  • have had a recent vaccination or are scheduled to receive any vaccination
  • are pregnant, think they might be pregnant, or plan to become pregnant. It is not known if Enspryng will harm one’s unborn baby
  • are breastfeeding or plan to breastfeed. It is not known if Enspryng passes into breast milk. Patients should speak with their healthcare provider about the best way to feed one’s baby while on treatment with Enspryng

 

Patients should tell their healthcare provider about all the medicines they take, including prescription and over-the-counter medicines, vitamins and herbal supplements.

 

The most common side effects of Enspryng include:

  • sore throat, runny nose (nasopharyngitis)
  • headache
  • upper respiratory tract infection
  • rash
  • fatigue
  • nausea
  • extremity pain
  • inflammation of the stomach lining
  • joint pain

 

For more information about the risk and benefit profile of Enspryng, patients should ask their healthcare provider.

 

Patients may report side effects to the FDA at 1-800-FDA-1088 or http://www.fda.gov/medwatch. Patients may also report side effects to Genentech at 1-888-835-2555.

 

Please see the full Prescribing Information for additional Important Safety Information.

 

About Genentech in neuroscience
 Neuroscience is a major focus of research and development at Genentech and Roche. Our goal is to pursue groundbreaking science to develop new treatments that help improve the lives of people with chronic and potentially devastating diseases.

 

Genentech and Roche are investigating more than a dozen medicines for neurological disorders, including multiple sclerosis, stroke, Alzheimer’s disease, Huntington’s disease, Parkinson’s disease, Duchenne muscular dystrophy and autism spectrum disorder. Together with our partners, we are committed to pushing the boundaries of scientific understanding to solve some of the most difficult challenges in neuroscience today.

 

About Genentech

Founded more than 40 years ago, Genentech is a leading biotechnology company that discovers, develops, manufactures and commercializes medicines to treat patients with serious and life-threatening medical conditions. The company, a member of the Roche Group, has headquarters in South San Francisco, California. For additional information about the company, please visit http://www.gene.com.   

 

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