Genentech, a member of the Roche Group, will present new data on its approved and investigational medicines for neurological conditions during the 70th American Academy of Neurology (AAN) Annual Meeting from April 21-27 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 Genentech 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, M.D., 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 will be shared for the first time through the OBOE (Ocrelizumab Biomarker Outcome Evaluation) study.
Additional 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 Genentech’s neuroscience pipeline will also be presented:
- Alzheimer’s disease is an important area of focus for Genentech. Four presentations on investigational anti-amyloid beta antibodies crenezumab and gantenerumab show promise in Genentech’s Alzheimer’s disease pipeline, and the findings have informed the design for both the CREAD and GRADUATE pivotal Phase III trial programs, 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 (1200 mg) 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 program 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 120 mg. Data from this study were used to determine an optimal dose now used in the ongoing CREAD pivotal program 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.
- 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 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||S6.002 (platform), Sunday, April 22, 1:12 – 1:24 p.m. PDT|
|Annualized Relapse Rate and Confirmed Disability Progression in Patients Receiving Continuous 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||P1.366 (poster), Sunday, April 22, 11:30 a.m. – 5:30 p.m. PDT|
|Confirmed Disability Progression in Different Subgroups of Patients with Relapsing Multiple Sclerosis Who Received Ocrelizumab or Interferon Beta-1a in the Phase III OPERA I and OPERA II Studies||P1.371 (poster), Sunday, April 22, 11:30 a.m. – 5:30 p.m. PDT|
|Establishment of Optimal Bioanalytical Parameters for Measuring Neurofilament Light Chain (Nf-L) in Multiple Sclerosis (MS) Subjects from Clinical Trial Cohorts||P1.413 (poster), Sunday, April 22, 11:30 a.m. – 5:30 p.m. PDT|
|Impact of Ocrelizumab on Cognition in Patients at Increased Risk of Progressive Disease||P1.420 (poster), Sunday, April 22, 11:30 a.m. – 5:30 p.m. PDT|
|Interim Analysis of the OBOE (Ocrelizumab Biomarker Outcome Evaluation) Study in Multiple Sclerosis (MS)||S24.002 (platform), Tuesday, April 24, 3:42 – 3:54 p.m. PDT|
|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||P3.376 (poster), Tuesday, April 24, 11:30 a.m. – 7:00 p.m. PDT|
|Safety of Ocrelizumab in Multiple Sclerosis: Updated Analysis in Patients with Relapsing and Primary Progressive Multiple Sclerosis||S36.001 (platform), Wednesday, April 25, 3:30 – 3:42 p.m. PDT|
|Effect of Ocrelizumab on Vaccine Responses in Patients with Multiple Sclerosis||S36.002 (platform), Wednesday, April 25, 3:42 – 3:54 p.m. PDT|
|FLOODLIGHT: Remote Self-monitoring is Accepted by Patients and Provides Meaningful, Continuous Sensor-based Outcomes Consistent with and Augmenting Conventional In-clinic Measures||P4.382 (poster), Wednesday, April 25, 11:30 a.m. – 7:00 p.m. PDT|
|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||P4.367 (poster), Wednesday, April 25, 11:30 a.m. – 7:00 p.m. PDT|
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
|P4.372 (poster), Wednesday, April 25, 11:30 a.m. – 7:00 p.m. PDT|
|Time to Cognitive Worsening in Patients with Relapsing Multiple Sclerosis in Ocrelizumab Phase III Trials||S44.005 (platform), Thursday, April 26, 4:18 – 4:30 p.m. PDT|
|Routine Laboratory Measures in the Controlled-treatment Period of Phase III Ocrelizumab Trials in Relapsing and Progressive Multiple Sclerosis||P5.425 (poster), Thursday, April 26, 11:30 a.m. – 7:00 p.m. PDT|
|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||P6.370 (poster), Friday, April 27, 11:30 a.m. – 5:30 p.m. PDT|
Real-world Experience with Ocrelizumab
|P6.356 (poster), Friday, April 27, 11:30 a.m. – 5:30 p.m. PDT|
|Crenezumab||Characterization of the Selective In Vivo and In Vitro Binding Properties of Crenezumab: Insights into Crenezumab’s Unique Mechanism of Action||P6.174 (poster), Friday, April 27, 11:30 a.m. – 5:30 p.m. PDT|
|Safety, Tolerability and Pharmacokinetics of Crenezumab in Mild-to-Moderate AD Patients Treated with Escalating Doses for up to 32.3 Months||P6.182 (poster) Friday, April 27, 11:30 a.m. – 5:30 p.m. PDT|
|Gantenerumab||Higher Dose Gantenerumab Leads to Significant Reduction in Amyloid Plaque Burden – Results for the Marguerite and Scarlet Road Open Label Extension Studies||S2.005 (platform), Sunday, April 22, 1:48 – 2:00 p.m. PDT|
|Optimizing the Gantenerumab Phase 3 Dosing Regimen Through PK/PD Modeling and Clinical Trial Simulations||P6.179 (poster), Friday, April 27, 11:30 a.m. – 5:30 p.m. PDT|
|RG7916||RG7916 Significantly Increases SMN Protein in SMA Type 1 Babies||004 (Emerging Science platform), Tuesday, April 24, 5:54 p.m. PDT|
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
|P4.453 (poster), Wednesday, April 25, 11:30 a.m. – 7:00 p.m. PDT|
|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||S46.003 (platform), Thursday, April 26, 3:54 – 4:06 p.m. PDT|
|Relationship Between Central and Peripheral SMN Protein Increase Upon Treatment with RO7034067 (RG7916)||S46.007 (platform), Thursday, April 26, 4:42 – 4:54 p.m. PDT|
A Long-term, Open-label Follow-up Study of Olesoxime in Patients with Type 2 or Non-ambulatory Type 3 Spinal Muscular Atrophy Who Participated in a Placebo-controlled Phase 2 Trial
|S46.002 (platform), Thursday, April 26, 3:42– 3:54 p.m. PDT|
|Effects of IONIS-HTTRx in Patients with Early Huntington’s Disease, Results of the First HTT-lowering Drug Trial||CT.002 (plenary session), Tuesday, April 24, 9:15 – 9:27 a.m. PDT|
A Randomized, Placebo-controlled, Double-blind, Phase 1b/2 Study of the Novel Anti-myostatin Adnectin RG6206 (BMS-986089) in Ambulatory Boys with Duchenne Muscular Dystrophy
P5.431 (poster), Thursday, April 26, 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: https://www.aan.com/conferences-community/annual-meeting