–– Roche, Genentech and partners embark on global prevalence research, trend-spotting with digital analysis, and telehealth initiatives to assess & address deterrents to brain health to better understand holistic health journeys of patients with neurologic diseases
–– Long-term data to be presented demonstrate delayed MS disability with Ocrevus; reduced disease activity with Enspryng in NMOSD and with Evrysdi in SMA
–– Data from late-stage neuroscience pipeline to be presented, including efficacy and safety results from Phase II prasinezumab study in early Parkinson’s Disease
SOUTH SAN FRANCISCO, Calif.–(BUSINESS WIRE)–Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), is announcing three new partnerships focused on improving brain health outcomes. These industry-leading initiatives are designed to quantify and assess the complexity, economic impact and patient and care partner burden of brain health conditions, and generate robust data around the nuanced dynamics of neurologic care. Conditions affecting the brain and nervous system cause more death and illness globally than cardiovascular disease, cancers, and all other conditions.1 Genentech is proud to partake in this groundbreaking collaboration with the University of Washington, Institute for Health Metrics and Evaluation (IHME), PicnicHealth and Cleveland Clinic, and insights from these partnerships will be further discussed as part of Genentech’s presence at the 75th American Academy of Neurology (AAN) Annual Meeting being held April 22-27 in Boston.
Roche and Genentech will also present 33 data abstracts spanning Ocrevus® (ocrelizumab) in multiple sclerosis (MS), Enspryng® (satralizumab-mwge) in neuromyelitis optica spectrum disorder (NMOSD) and Evrysdi® (risdiplam) in spinal muscular atrophy (SMA), as well as from its broad pipeline of medicines being investigated in neuroimmunologic and neurodegenerative diseases, and neuroscience digital health and telehealth interventions.
“Diseases affecting the brain and central nervous system are associated with loss of more disability-adjusted life-years than almost any other condition,” said Levi Garraway, M.D., Ph.D., Genentech’s chief medical officer and head of Global Product Development. “With that in mind, we are committed to developing and delivering new medicines that address areas of highest need in neuroscience. Accordingly, we have more than a dozen investigational medicines that span a range of neurological conditions, with the ultimate goal of slowing progression and delaying disability for as long as possible.”
Commitment to Advancing Understanding of Brain Health and Neurological Disorders
Roche and Genentech are the number one investor in R&D among all healthcare companies, with $14.7 billion invested in 2022 alone, of which more than 20% was dedicated to evaluating therapeutic, diagnostic and digital technology interventions to discover and potentially address brain health disorders. As part of Roche and Genentech’s commitment to brain health, we have embarked on substantial new initiatives and expanded partnerships that aim to realize the full value of innovation to reduce disease burden, improve lives and help build more resilient and responsive health systems to meet current and future needs across the spectrum of brain health, including:
University of Washington, Institute for Health Metrics and Evaluation (IHME) Brain Health Initiative
Roche and Genentech are proud to support IHME as a founding member in their newly launched Brain Health Collaborative. The multi-year effort will develop a view of the current and future burden of brain health conditions (mental, neurological and cerebrovascular) and create publicly available, interactive research tools including the Brain Health Atlas, which illustrates the global burden of health conditions measured over time and geographies, exploring the full economic impact of brain disorders globally, including analysis on total economic and social costs. This initiative will be featured as part of AAN’s Practice and Policy Hub session, featuring experts from Roche, IHME and other leaders on the emerging science and technologies of brain health on Wednesday, April 26 from 7:45-8:45 a.m. ET. See here for additional details.
PicnicHealth “BrainPower” Pan-Neuroscience Patient Experience Research
Genentech is initiating a neuroscience research study called BrainPower (Brain Patient Outcomes With Experience Research), an expansion of our five-year partnership with a patient-centered medical records healthcare technology company, PicnicHealth, to analyze longitudinal, real-world clinical data of people living with neurologic diseases, such as Alzheimer’s disease (AD) and related dementias (EMPOWER AD), Myasthenia gravis (MG), and MS to identify common data trends and understand shared patient challenges across neurological disorders. This further builds on previously announced neuroscience partnerships in MS and Huntington’s disease. Details on the EMPOWER AD study with PicnicHealth will be shared in an oral presentation on Monday, April 24 at 8:00 a.m. ET.
“Social determinants of health impact quality of life, outcomes and overall health, and we are acutely aware of the disparities and inequities they perpetuate across neurological disorders — particularly in the United States. We are partnering with the leading minds in healthcare technology, telehealth, inclusive research and evidence generation to identify barriers and co-create solutions with the neurology community to facilitate equitable healthcare delivery,” said Gregory A. Rippon, M.D., M.S., F.A.A.N., Vice President and Chief Medical Partner, Neurology. “Our hope is to help people living with neurological disorders preserve their health, independence, and what makes them uniquely who they are – and that will only be possible when every person not only receives a transformative treatment, but also receives the same quality of care, regardless of their zip code, income or background.”
Cleveland Clinic Neurology Telehealth Pilot
Telehealth is an accessible option for a variety of patients, including those living in rural areas, facing barriers to healthcare access, who are immunocompromised or could face issues with an in-person setting (e.g., due to financial or mobility challenges). Neurology practices saw one of the highest uptake rates for ambulatory telemedicine (nearly half of total visits) during the early part of the COVID-19 pandemic. Cleveland Clinic and Genentech established a research collaboration in 2022 to better understand the utilization of telehealth in nearly 200,000 people living with neurological conditions (MS, Alzheimer’s, Parkinson’s), and impact on access to care, clinical outcomes, and healthcare resource utilization within Cleveland Clinic’s health system. We hope to understand patient barriers to facilitate more equitable healthcare delivery, particularly among underserved minority communities, older and lower socioeconomic status individuals and people living in rural areas. See AAN oral presentation details on Genentech’s telehealth pilot with Cleveland Clinic here, Tuesday, April 25 at 3:54 p.m. ET.
Longer-term data to be presented at AAN to reinforce importance of early treatment and slowing disease activity and progression in MS, NMOSD and SMA
Across neurological disorders the main goal of treatment is to prevent disease progression to ultimately preserve function. Genentech will present 33 abstracts, including new long-term data for market leading Ocrevus in MS and Evrysdi in SMA, including:
The Phase III OPERA I/II and Phase IIIb ENSEMBLE trials highlight the importance of early treatment with Ocrevus, with new 8.5- and 9-year follow-up data from the Phase III OPERA I/II trials in relapsing MS (RMS), which significantly slows disease activity and disability progression – an important goal in MS. Also presented for Ocrevus will be new four-year data from the Phase IIIb ENSEMBLE trial demonstrating minimal disease activity in newly diagnosed patients living with relapsing remitting MS (RRMS). Ocrevus is the #1 prescribed MS medicine in the US, and more than 300,000 patients globally have been treated with Ocrevus. See AAN oral session presentation details on Ocrevus sessions here.
Data from three studies reinforce the longer-term efficacy and safety of Evrysdi for people living with SMA across a broad range of ages, disease severities and treatment histories, including: three-year data from the FIREFISH study in infants with Type 1 SMA showing improvements in survival and reaching motor milestones, four-year data from the SUNFISH study in people aged 2 to 25 years with Types 2 or 3 SMA with sustained motor function and a decreased rate of adverse events, and two-year data from the JEWELFISH study in people with SMA aged 6 months to 60 years who initially received other investigational or approved SMA therapies, which showed maintenance of motor function. Without treatment, children with Type 1 SMA are never able to sit without support and natural history data show that patients with Type 2 or 3 SMA typically show a decline in motor function over time. More than 8,500 people globally — from newborns to those over 60 — have been treated with Evrysdi, which is now approved in more than 95 countries worldwide. See AAN oral session presentation details on Evrysdi sessions here.
Broad Neuroscience Pipeline & Areas of Focus
As global populations continue to grow and age, their impact is set to increase, creating new challenges for families, carers, health systems, employers, and economies. To meet the needs of people living with brain health conditions, Genentech is continuing to grow and advance its pipeline, with key areas of focus in neuroimmunologic, neuromuscular, neurodegenerative and neurodevelopmental diseases. In addition to the Phase II PASADENA trial, evaluating prasinezumab in people with early Parkinson’s, data being featured at AAN, Genentech’s later-stage neuroscience programs include tenecteplase for acute ischemic stroke, fenebrutinib for MS, prasinezumab in Parkinson’s, tominerson in Huntington’s disease, rugonersen in angelman syndrome, satralizumab in MG and trontinemab, utilizing Roche’s Brain Shuttle technology for Alzheimer’s. Genentech’s late-stage pipeline is available here.
The full range of data from Genentech’s clinical development program in neuroscience being presented at AAN 2023 can be found at https://aan.com/events/annual-meeting.
About Genentech in Neuroscience
Neuroscience is a major focus of research and development at Genentech. 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 MS, SMA, NMOSD, Alzheimer’s, Huntington’s, Parkinson’s, Acute ischemic stroke, Duchenne muscular dystrophy and Angelman syndrome. 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 Ocrevus® (ocrelizumab)
Ocrevus is the first and only therapy approved for both RMS (including RRMS and active, or relapsing, secondary progressive MS [SPMS], in addition to clinically isolated syndrome [CIS] in the U.S.) and PPMS. Ocrevus is a humanized monoclonal antibody designed to target CD20-positive B cells, a specific type of immune cell thought to be a key contributor to myelin (nerve cell insulation and support) and axonal (nerve cell) damage. This nerve cell damage can lead to disability in people with MS. Based on preclinical studies, Ocrevus binds to CD20 cell surface proteins expressed on certain B cells, but not on stem cells or plasma cells, suggesting that important functions of the immune system may be preserved. Ocrevus is administered by intravenous infusion every six months. The initial dose is given as two 300 mg infusions given two weeks apart. Subsequent doses are given as single 600 mg infusions.
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 disease processes, triggering the inflammation cascade and leading to damage and disability. Enspryng was designed using novel recycling antibody technology. When compared to conventional antibodies, Enspryng’s recycling antibody technology enables the medicine to remain in the bloodstream for a longer period of time and bind repeatedly to its target (the IL-6 receptor) – maximally sustaining IL-6 suppression in a chronic disease like NMOSD and enabling subcutaneous dosing every four weeks.
Positive Phase III results for Enspryng, as both monotherapy and in combination with baseline immunosuppressive therapy, suggest that IL-6 inhibition is an effective therapeutic approach for NMOSD who are AQP4-IgG seropositive. The Phase III clinical development program for Enspryng includes two studies: SAkuraStar and SAkuraSky.
Enspryng is currently approved in 80 countries, including the United States, Canada, Japan, South Korea and the European Union.
Enspryng has been designated as an orphan drug in the United States, Europe, Japan and Russia. In addition, it was granted Breakthrough Therapy Designation for the treatment of NMOSD by the FDA in December 2018, which is given to treatments that may demonstrate substantial improvement over other available options.
About Evrysdi® (risdiplam)
Evrysdi is a survival motor neuron 2 (SMN2) splicing modifier designed to treat SMA caused by mutations in chromosome 5q that lead to survival motor neuron (SMN) protein deficiency. Evrysdi is administered daily at home in liquid form by mouth or by feeding tube.
Evrysdi is designed to treat SMA by increasing and sustaining the production of SMN protein in the central nervous system (CNS) and peripheral tissues as demonstrated in animal models. SMN protein is found throughout the body and is critical for maintaining healthy motor neurons and movement.
Evrysdi was granted PRIME designation by the European Medicines Agency (EMA) in 2018 and Orphan Drug Designation by the U.S. Food and Drug Administration in 2017. In 2021, Evrysdi was awarded Drug Discovery of the Year by the British Pharmacological Society as well as the Society for Medicines Research award for Drug Discovery. Evrysdi is currently approved in more than 90 countries and the dossier is under review in a further 16 countries.
Evrysdi is currently being evaluated in five multicenter trials in people with SMA:
FIREFISH (NCT02913482) – an open-label, two-part pivotal clinical trial in infants with Type 1 SMA. Part 1 was a dose-escalation study in 21 infants with the primary objective of assessing the safety profile of risdiplam in infants and determining the dose for Part 2. Part 2 is a pivotal, single-arm study of risdiplam in 41 infants with Type 1 SMA treated for two years, followed by an open-label extension. Enrollment for Part 2 was completed in November 2018. The primary objective of Part 2 was to assess efficacy as measured by the proportion of infants sitting without support for 5 seconds after 12 months of treatment, as assessed by the Gross Motor Scale of the Bayley Scales of Infant and Toddler Development – Third Edition (BSID-III) (defined as sitting without support for 5 seconds). The study met its primary endpoint.
SUNFISH (NCT02908685) – a two-part, double-blind, placebo-controlled pivotal study in people aged 2-25 years with Types 2 or 3 SMA. Part 1 (n=51) determined the dose for the confirmatory Part 2. Part 2 (n=180) evaluated motor function using the total score of Motor Function Measure 32 (MFM-32) at 12 months. MFM-32 is a validated scale used to evaluate fine and gross motor function in people with neurological disorders, including SMA. The study met its primary endpoint.
JEWELFISH (NCT03032172) – an open-label exploratory trial designed to assess the safety, tolerability, pharmacokinetics and pharmacodynamics in people with SMA aged 6 months to 60 years who received other investigational or approved SMA therapies for at least 90 days prior to receiving Evrysdi. The study has completed recruitment (n=174).
RAINBOWFISH (NCT03779334) – an open-label, single-arm, multicenter study, investigating the efficacy, safety, pharmacokinetics, and pharmacodynamics of risdiplam in babies (~n=25), from birth to six weeks of age (at first dose) with genetically diagnosed SMA who are not yet presenting with symptoms. The study is fully enrolled.
MANATEE (NCT05115110) – a global Phase II/III clinical study to evaluate the safety and efficacy of GYM329 (RG6237), an anti-myostatin molecule targeting muscle growth, in combination with Evrysdi for the treatment of SMA in patients 2-25 years of age. The FDA Office of Orphan Products Development granted GYM329 Orphan Drug Designation for the treatment of patients with SMA in December 2021. The study is currently recruiting.
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.
Indications and Important Safety Information
What is Ocrevus?
Ocrevus is a prescription medicine used to treat:
Relapsing forms of multiple sclerosis (MS), to include clinically isolated syndrome, relapsing-remitting disease, and active secondary progressive disease, in adults
Primary progressive MS, in adults.
It is not known if Ocrevus is safe or effective in children.
Who should not receive Ocrevus?
Do not receive Ocrevus if you have an active hepatitis B virus (HBV) infection.
Do not receive Ocrevus if you have had a life threatening allergic reaction to Ocrevus. Tell your healthcare provider if you have had an allergic reaction to Ocrevus or any of its ingredients in the past.
What is the most important information I should know about Ocrevus?
Ocrevus can cause serious side effects, including:
Infusion reactions: Infusion reactions are a common side effect of Ocrevus, which can be serious and may require you to be hospitalized. You will be monitored during your infusion and for at least 1 hour after each infusion of Ocrevus for signs and symptoms of an infusion reaction. Tell your healthcare provider or nurse if you get any of these symptoms:
coughing or wheezing
throat irritation or pain
redness on your face (flushing)
swelling of the throat
shortness of breath
fast heart beat
These infusion reactions can happen for up to 24 hours after your infusion. It is important that you call your healthcare provider right away if you get any of the signs or symptoms listed above after each infusion.
If you get infusion reactions, your healthcare provider may need to stop or slow down the rate of your infusion.
Ocrevus increases your risk of getting upper respiratory tract infections, lower respiratory tract infections, skin infections, and herpes infections. Infections are a common side effect, which can be serious. Tell your healthcare provider if you have an infection or have any of the following signs of infection including fever, chills, or a cough that does not go away. Signs of herpes include cold sores, shingles, genital sores, skin rash, pain, and itching. Signs of more serious herpes infection include: changes in vision, eye redness or eye pain, severe or persistent headache, stiff neck, and confusion. Signs of infection can happen during treatment or after you have received your last dose of Ocrevus. Tell your healthcare provider right away if you have an infection. Your healthcare provider should delay your treatment with Ocrevus until your infection is gone.
Progressive Multifocal Leukoencephalopathy (PML): Although no cases have been seen with Ocrevus treatment in clinical trials, PML may happen with Ocrevus. PML is a rare brain infection that usually leads to death or severe disability. Tell your healthcare provider right away if you have any new or worsening neurologic signs or symptoms. These may include problems with thinking, balance, eyesight, weakness on 1 side of your body, strength, or using your arms or legs.
Hepatitis B virus (HBV) reactivation: Before starting treatment with Ocrevus, your healthcare provider will do blood tests to check for hepatitis B viral infection. If you have ever had hepatitis B virus infection, the hepatitis B virus may become active again during or after treatment with Ocrevus. Hepatitis B virus becoming active again (called reactivation) may cause serious liver problems including liver failure or death. Your healthcare provider will monitor you if you are at risk for hepatitis B virus reactivation during treatment and after you stop receiving Ocrevus.
Weakened immune system: Ocrevus taken before or after other medicines that weaken the immune system could increase your risk of getting infections.
Low Immunoglobulins: Ocrevus may cause a decrease in some types of antibodies. Your healthcare provider will do blood tests to check your blood immunoglobulin levels.
Before receiving Ocrevus, tell your healthcare provider about all of your medical conditions, including if you:
have ever taken, take, or plan to take medicines that affect your immune system, or other treatments for MS.
have ever had hepatitis B or are a carrier of the hepatitis B virus.
have had a recent vaccination or are scheduled to receive any vaccinations.
You should receive any required ‘live’ or ‘live-attenuated’ vaccines at least 4 weeks before you start treatment with Ocrevus. You should not receive ‘live’ or ‘live-attenuated’ vaccines while you are being treated with Ocrevus and until your healthcare provider tells you that your immune system is no longer weakened.
When possible, you should receive any ‘non-live’ vaccines at least 2 weeks before you start treatment with Ocrevus. If you would like to receive any non-live (inactivated) vaccines, including the seasonal flu vaccine, while you are being treated with Ocrevus, talk to your healthcare provider.
If you are pregnant or planning to become pregnant talk to your doctor about vaccinations for your baby, as some precautions may be needed.
are pregnant, think that you might be pregnant, or plan to become pregnant. It is not known if Ocrevus will harm your unborn baby. You should use birth control (contraception) during treatment with Ocrevus and for 6 months after your last infusion of Ocrevus. Talk with your healthcare provider about what birth control method is right for you during this time.
If you become pregnant while taking Ocrevus, talk to your doctor about enrolling in the Ocrevus Pregnancy Registry. You can enroll in this registry by calling 1-833-872-4370 or visiting http://www.Ocrevuspregnancyregistry.com. The purpose of this registry is to monitor the health of you and your baby.
are breastfeeding or plan to breastfeed. It is not known if Ocrevus passes into your breast milk. Talk to your healthcare provider about the best way to feed your baby if you take Ocrevus.
Media Contact: Justin Hurdle, (650) 467-6800
Advocacy Contact: Alana Paull, (925) 528-1014
Investor Contact: Loren Kalm, (650) 225-3217