Roche to present new data at AAN highlighting extensive research for OCREVUS and expanding neuroscience pipeline

Transcription

1 Investor Update Basel 16 April 2018 Roche to present new data at AAN highlighting extensive research for OCREVUS and expanding neuroscience pipeline Breadth of data will reinforce efficacy and safety of OCREVUS in relapsing and primary progressive multiple sclerosis and demonstrate its benefits in slowing disability progression, including new cognitive and biomarker results Important research in Alzheimer s disease, Huntington s disease, Spinal Muscular Atrophy and Duchenne Muscular Dystrophy will highlight the strength of Roche s neuroscience pipeline Roche (SIX: RO, ROG; OTCQX: RHHBY) announced today that new data on its approved and investigational medicines for neurological conditions will be presented during the 70 th American Academy of Neurology (AAN) Annual Meeting from April in Los Angeles, California. These data will reinforce the efficacy and safety of OCREVUS (ocrelizumab) and expand the clinical understanding of disability progression in multiple sclerosis (MS). They will also represent investigational research from the Roche neuroscience pipeline in Alzheimer s disease, Huntington s disease, spinal muscular atrophy (SMA) and Duchenne muscular dystrophy (DMD). Our neuroscience pipeline is one of the deepest and most diverse in the industry, spanning both common and rare neurological conditions with the greatest unmet need, said Sandra Horning, MD, Roche s Chief Medical Officer and Head of Global Product Development. OCREVUS is now approved in over 55 countries with more than 40,000 people treated. We remain committed to continuing our research and development to understand MS progression further and help those living with MS and their physicians. OCREVUS data will show significant and sustained efficacy as well as benefits in cognition in people with relapsing MS (RMS). The early impact of OCREVUS on biomarkers of inflammation and neurodegeneration in people with RMS and primary progressive MS (PPMS) will be shared for the first time through the OBOE (Ocrelizumab Biomarker Outcome Evaluation) study. F. Hoffmann-La Roche Ltd 4070 Basel Switzerland Investor Relations investor.relations@roche.com Tel Fax /8

2 Additional notable MS presentations include updated safety analyses for OCREVUS, which will further inform and reinforce its continued favourable benefit-risk profile. New data from the FLOODLIGHT pilot study, which support mobile technology as a complement to in-clinic testing to provide a more complete and real-time picture of a patient s underlying disease activity, will also be presented. Encouraging data from other investigational medicines in Roche s neuroscience pipeline will also be presented: Alzheimer s disease is an important area of focus for Roche. Four presentations on investigational antiamyloid beta antibodies crenezumab and gantenerumab show promise in Roche s Alzheimer s disease pipeline and the findings have informed the design for both the CREAD and GRADUATE pivotal Phase III trial programmes, respectively. In a platform session, data on gantenerumab will be presented showing a significant reduction in brain amyloid beta plaques with a higher dosing regimen (1200mg) in two Phase III, open label extension studies. Data from these two studies guided the dose and titration regimen selection for the recently initiated Phase III GRADUATE pivotal programme investigating gantenerumab for the treatment of early Alzheimer s disease. Two posters on crenezumab will be presented; one will focus on preclinical data and will discuss its proposed mechanism of action, including data supporting its preferential binding to neurotoxic amyloid beta oligomers. A second poster will describe the results from a safety, tolerability and pharmacokinetics Phase Ib study in doses up to 120mg. Data from this study were used to determine an optimal dose now used in the ongoing CREAD pivotal programme investigating crenezumab for the treatment of early Alzheimer s disease. Data from a Phase I/IIa multiple-ascending dose study of RG6042 (formerly known as IONIS HTTRx) in Huntington s disease will be presented in a plenary session. These data will highlight the safety and tolerability of this investigational medicine over four monthly doses, demonstrate dose-dependent lowering of the mutant huntingtin protein (mhtt), and show additional exploratory analyses from this first-in-human study. These results for RG6042 are the first data demonstrating lowering of mhtt, the disease-causing protein in people with Huntington s disease. The SMA presentations include late-breaking interim data on the increase in survival of motor neuron (SMN) protein levels following treatment with RG7916 in infants with Type 1 SMA. SMA, the leading genetic cause of mortality in infants and toddlers, is a rare neuromuscular disease caused by a deficiency of SMN protein. RG7916 is an investigational oral SMN2 splicing modifier being developed in collaboration with PTC Therapeutics, Inc. and the SMA Foundation. 2/8

3 Results from a Phase Ib/II study of the investigational adnectin fusion protein RG6206 in young male adolescents with DMD will also be presented. These data will highlight the myostatin suppression levels achieved and its potential effect in increasing lean body mass volume. Investigators will present the following plenary, platform and poster presentations: Medicine Abstract Title Abstract Number (type), Presentation Date, Time OCREVUS (ocrelizumab) Brain MRI Activity and Atrophy Measures in Patients Receiving Continuous Ocrelizumab or S6.002 (platform), Sunday, 22 April, 1:12 1:24 p.m. PDT Switching from Interferon Beta-1a to Ocrelizumab Therapy in the Open-label Extension Period of the Phase III Trials of Ocrelizumab in Patients with Relapsing Multiple Sclerosis Annualized Relapse Rate and Confirmed Disability Progression in Patients Receiving Continuous P1.366 (poster), Sunday, 22 April, Ocrelizumab or Switching from Interferon Beta-1a to Ocrelizumab Therapy in the Open-label Extension Period of the Phase III Trials of Ocrelizumab in Patients with Relapsing Multiple Sclerosis Confirmed Disability Progression in Different Subgroups of Patients with Relapsing Multiple P1.371 (poster), Sunday, 22 April, Sclerosis Who Received Ocrelizumab or Interferon Beta-1a in the Phase III OPERA I and OPERA II Studies Establishment of Optimal Bioanalytical Parameters for Measuring Neurofilament Light Chain (Nf-L) in P1.413 (poster), Sunday, 22 April, Multiple Sclerosis (MS) Subjects from Clinical Trial Cohorts Impact of Ocrelizumab on Cognition in Patients at Increased Risk of Progressive Disease P1.420 (poster), Sunday, 22 April, 3/8

4 Interim Analysis of the OBOE (Ocrelizumab Biomarker Outcome Evaluation) Study in Multiple Sclerosis (MS) Ocrelizumab May Reduce Tissue Damage in Chronic Active Lesions as Measured by Change in T1 Hypo-intensity of Slowly Evolving Lesions in Patients with Primary Progressive Multiple Sclerosis Safety of Ocrelizumab in Multiple Sclerosis: Updated Analysis in Patients with Relapsing and Primary Progressive Multiple Sclerosis Effect of Ocrelizumab on Vaccine Responses in Patients with Multiple Sclerosis FLOODLIGHT: Remote Self-monitoring is Accepted by Patients and Provides Meaningful, Continuous Sensor-based Outcomes Consistent with and Augmenting Conventional In-clinic Measures Design of the Ocrelizumab Pregnancy Registry to Assess Maternal, Fetal and Infant Outcomes in Women with Multiple Sclerosis Who Were Exposed to Ocrelizumab During, or Within 6 Months Before, Pregnancy Design of a Multi-source Post-marketing Study to Evaluate Pregnancy and Infant Outcomes in Women with Multiple Sclerosis Who Were Exposed to Ocrelizumab During, or Within 6 Months Before, Pregnancy Time to Cognitive Worsening in Patients with Relapsing Multiple Sclerosis in Ocrelizumab Phase III Trials Routine Laboratory Measures in the Controlledtreatment Period of Phase III Ocrelizumab Trials in Relapsing and Progressive Multiple Sclerosis S (platform), Tuesday, 24 April, 3:42 3:54 p.m. PDT P3.376 (poster), Tuesday, 24 S (platform), Wednesday, 25 April, 3:30 3:42 p.m. PDT S (platform), Wednesday, 25 April, 3:42 3:54 p.m. PDT P4.382 (poster), Wednesday, 25 P4.367 (poster), Wednesday, 25 P4.372 (poster), Wednesday, 25 S (platform), Thursday, 26 April, 4:18 4:30 p.m. PDT P5.425 (poster), Thursday, 26 4/8

5 Crenezumab Gantenerumab RG7916 Baseline Characteristics of the CHORDS Study Population: A Phase III Trial to Evaluate the Effectiveness and Safety of Ocrelizumab in Patients with RRMS who had Disease Activity with Prior Disease-modifying Therapies Real-World Experience with Ocrelizumab Characterization of the Selective In Vivo and In Vitro Binding Properties of Crenezumab: Insights into Crenezumab s Unique Mechanism of Action Safety, Tolerability and Pharmacokinetics of Crenezumab in Mild-to-Moderate AD Patients Treated with Escalating Doses for up to 32.3 Months Higher Dose Gantenerumab Leads to Significant Reduction in Amyloid Plaque Burden - Results for the Marguerite and Scarlet Road Open Label Extension Studies Optimising the Gantenerumab Phase 3 Dosing Regimen Through PK/PD Modeling and Clinical Trial Simulations RG7916 Significantly Increases SMN Protein in SMA Type 1 Babies Updated pharmacodynamic and Safety Data from SUNFISH Part 1, a Study Evaluating the Oral SMN2 Splicing Modifier RG7916 in Patients with Type 2 or 3 Spinal Muscular Atrophy Preliminary Evidence for Pharmacodynamics Effects of RG7916 in JEWELFISH, a Study in Patients with Spinal Muscular Atrophy who Previously Participated in a Study with Another SMN2-Splicing Targeting Therapy P6.370 (poster), Friday, 27 April, P6.356 (poster), Friday, 27 April, P6.174 (poster), Friday, 27 April, P6.182 (poster) Friday, 27 April, S2.005 (platform), Sunday, 22 April, 1:48 2:00 p.m. PDT P6.179 (poster), Friday, 27 April, 004 (Emerging Science platform), Tuesday, 24 April, 5:54 p.m. PDT P4.453 (poster), Wednesday, 25 S (platform), Thursday, 26 April, 3:54 4:06 p.m. PDT 5/8

6 Olesoxime RG6042 (IONIS-HTTRx) RG6206 Relationship Between Central and Peripheral SMN Protein Increase Upon Treatment with RO (RG7916) A Long-Term, Open-Label Follow-up Study of Olesoxime in Patients with Type 2 or Nonambulatory Type 3 Spinal Muscular Atrophy who Participated in a Placebo-controlled Phase 2 Trial Effects of IONIS-HTTRx in Patients with Early Huntington s Disease, Results of the First HTTlowering Drug Trial A Randomized, Placebo-Controlled, Double-Blind, Phase 1b/2 Study of the Novel Anti-Myostatin Adnectin RG6206 (BMS ) in Ambulatory Boys with Duchenne Muscular Dystrophy S (platform), Thursday, 26 April, 4:42 4:54 p.m. PDT S (platform), Thursday, 26 April, 3:42 3:54 p.m. PDT CT.002 (plenary session), Tuesday, 24 April, 9:15 9:27 a.m. PDT P5.431 (poster), Thursday, 26 April, 11:30 a.m. to 7:00 p.m. PDT Full session details and data presentation listings for the 2018 AAN Annual Meeting can be found at the meeting website: OCREVUS is now approved in over 55 countries across North America, South America, the Middle East, Eastern Europe, as well as in Australia, Switzerland and the European Union. Marketing applications are currently under review in more than 20 countries across the world. Follow Roche on Twitter and keep up to date with AAN 2018 Annual Meeting news and updates by using the hashtag #AANAM. About Roche in neuroscience Neuroscience is a major focus of research and development at Roche. The company s goal is to develop treatment options based on the biology of the nervous system to help improve the lives of people with chronic and potentially devastating diseases. Roche has more than a dozen investigational medicines in clinical development for diseases that include multiple sclerosis, Alzheimer s disease, spinal muscular atrophy, Parkinson s disease and autism. 6/8

7 About Roche Roche is a global pioneer in pharmaceuticals and diagnostics focused on advancing science to improve people s lives. The combined strengths of pharmaceuticals and diagnostics under one roof have made Roche the leader in personalised healthcare a strategy that aims to fit the right treatment to each patient in the best way possible. Roche is the world s largest biotech company, with truly differentiated medicines in oncology, immunology, infectious diseases, ophthalmology and diseases of the central nervous system. Roche is also the world leader in in vitro diagnostics and tissue-based cancer diagnostics, and a frontrunner in diabetes management. Founded in 1896, Roche continues to search for better ways to prevent, diagnose and treat diseases and make a sustainable contribution to society. The company also aims to improve patient access to medical innovations by working with all relevant stakeholders. Thirty medicines developed by Roche are included in the World Health Organization Model Lists of Essential Medicines, among them life-saving antibiotics, antimalarials and cancer medicines. Roche has been recognised as the Group Leader in sustainability within the Pharmaceuticals, Biotechnology & Life Sciences Industry nine years in a row by the Dow Jones Sustainability Indices (DJSI). The Roche Group, headquartered in Basel, Switzerland, is active in over 100 countries and in 2017 employed about 94,000 people worldwide. In 2017, Roche invested CHF 10.4 billion in R&D and posted sales of CHF 53.3 billion. Genentech, in the United States, is a wholly owned member of the Roche Group. Roche is the majority shareholder in Chugai Pharmaceutical, Japan. For more information, please visit All trademarks used or mentioned in this release are protected by law. Roche Investor Relations Dr. Karl Mahler Dr. Sabine Borngräber Phone: Phone: karl.mahler@roche.com sabine.borngraeber@roche.com Dr. Bruno Eschli Dr. Susann Manchado Phone: Phone: bruno.eschli@roche.com susann.manchado@roche.com Dr. Birgit Masjost Phone: birgit.masjost@roche.com 7/8

8 Investor Relations North America Neera Dahiya Ravindran, MD Loren Kalm Phone: Phone: /8