Genentech at The AAN

Genentech, a member of the Roche Group, will present new data on its approved and investigational medicines for neurological conditions during the 70^th 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:

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