“Biogen’s research efforts are focused on helping to improve the lives of the approximately one billion people affected by neurological disorders,1” said Alfred Sandrock, Jr., M.D., Ph.D., executive vice president and chief medical officer at Biogen. “The data we are presenting at AAN reflect the work we are conducting on multiple fronts, including research to better understand challenging nervous system diseases and the clinical development of investigational treatments for what we believe are some of the most significant unmet medical needs.”
Platform and poster presentations will highlight the benefits SPINRAZA (nusinersen) provides for individuals with spinal muscular atrophy (SMA) across the age and disease spectrum; the company’s multiple sclerosis (MS) therapies and non-therapeutic research collaborations designed to elevate the care of MS; and the company’s investigational therapies for Alzheimer’s disease, Parkinson’s disease (PD) and progressive supranuclear palsy (PSP).
Building on Substantial Data, Long-Term Benefits for Broad SMA Populations
- Biogen will present data demonstrating that with SPINRAZA treatment, older patients were able to walk longer distances while experiencing stable or less fatigue at the same time, in contrast to SMA natural history data. The study participants had Type 2 or 3 SMA and were ages 2-15 years at study enrollment.
- Several other analyses illustrating SPINRAZA’s effectiveness will be presented, including part one of the Phase 2 EMBRACE study as well as an interim analysis of the SHINE open-label extension study. Featured in AAN’s Emerging Science program, the SHINE analysis examined the longer-term safety and efficacy of SPINRAZA in infantile-onset SMA patients.
Elevating the Care of MS
- Data from Biogen’s collaborative research initiative to identify a quantitative blood biomarker of MS disease severity will be presented. These data further support serum neurofilament light (NfL) as a promising biomarker for disease severity stratification and treatment monitoring in MS. A biomarker like NfL levels may enhance treatment decision-making and ultimately lead to better long-term outcomes for people with MS.
- Biogen will present updates from MS PATHS (Multiple Sclerosis Partners Advancing Technology and Health Solutions), a collaboration with 10 leading MS centers in the U.S. and Europe that leverages technology deployed in routine care to generate standardized, high-quality data. Researchers anticipate more than 15,000 people with MS will participate in the study. MS PATHS allows researchers to evaluate common and disruptive MS symptoms, such as cognitive changes, to help drive more evidence-based, personalized treatment decisions.
- Real-world data will be presented that demonstrate people with relapsing MS treated with TECFIDERA® (dimethyl fumarate) or TYSABRI® (natalizumab) early in the course of their disease may experience better long-term outcomes.
- MRI and relapse results from the Phase 3 EVOLVE-MS-1 study for BIIB098 (formerly known as ALKS 8700) in patients with relapsing remitting MS will be featured in AAN’s Emerging Science program. BIIB098 is an oral, monomethyl fumarate (MMF) prodrug in Phase 3 development for the treatment of relapsing forms of MS.
Advancing the Company’s Neurodegenerative Pipeline
- Data from the Phase 1b study of aducanumab, Biogen’s investigational treatment for the early stages of Alzheimer’s disease, will be presented. The aducanumab 36-month data from the Phase 1b PRIME study have been identified by AAN as a 2018 Abstract of Distinction, a program recognizing top scientific achievements in each abstract topic area; 24-month titration data will also be presented.
Both slide presentations will be available concurrently with the applicable session on the Investor section of the Biogen company website, www.Biogen.com.
Aducanumab is currently being evaluated in two global Phase 3 studies, ENGAGE and EMERGE, which are designed to evaluate its safety and efficacy in slowing cognitive and functional impairment in people with mild cognitive impairment (MCI) due to Alzheimer’s disease and mild Alzheimer’s disease dementia. Aducanumab is thought to target aggregated forms of beta amyloid, including soluble oligomers and insoluble fibrils, which can form into amyloid plaque in the brain of Alzheimer’s disease patients. As of October 22, 2017, Biogen and Eisai Co. Ltd. are collaborating on the development and commercialization of aducanumab globally.
- Data from movement disorder programs will be presented at the meeting, including Phase 1 study results for BIIB092 (formerly BMS-986168), the tau-targeting antibody and investigational compound for PSP, as well as details about the design of the ongoing Phase 2 PASSPORT study. PSP is a rare neurodegenerative disease, considered to be a primary tauopathy, characterized by rapidly progressing physical impairments, such as difficulty speaking, swallowing and walking, as well as cognitive/behavioral impairments, such as apathy and dementia. Data for Biogen’s investigation compound for PD, BIIB054, an alpha-synuclein targeting antibody, include Phase 1 study results demonstrating a favorable pharmacokinetics, as well as a safety and tolerability profile which support further clinical development.
Highlights of Biogen’s platform and poster presentations:
SPINAL MUSCULAR ATROPHY
- Longer-term Assessment of the Safety and Efficacy of Nusinersen for the Treatment of Infantile-onset Spinal Muscular Atrophy (SMA): An Interim Analysis of the SHINE Study – Platform 003 – Tuesday, April 24, 5:51-5:54 p.m. PT
- Evaluating Ambulatory Function and Fatigability in Children Treated with Nusinersen – Poster P2.322 – Monday, April 23, 11:30 a.m.-7:00 p.m. PT
- Safety and Efficacy of Nusinersen in Infants/Children with Spinal Muscular Atrophy (SMA): Part 1 of the Phase 2 EMBRACE Study – Poster P2.324 – Monday, April 23, 11:30 a.m.-7:00 p.m. PT
- Characterization of Later Childhood/Adult Spinal Muscle Atrophy Patients and Their Transitions of Care Within U.S. Hospitals – Poster P4.454 – Wednesday, April 25, 11:30 a.m.-7:00 p.m. PT
- Serum Neurofilament Light (NfL): Towards a Blood Test for Prognosis and Disease/Treatment Monitoring in Multiple Sclerosis Patients – Platform S24.003 – Tuesday, April 24, 3:54-4:06 p.m. PT
- MRI and Relapse Results for ALKS 8700 in Patients with Relapsing Remitting Multiple Sclerosis: 1-year Interim Results from the Phase 3 EVOLVE-MS-1 Study – Platform 006 – Tuesday, April 24, 5:45-7:00 p.m. PT
- Benchmarks of Cognitive Performance in a Large, Representative Patient Population – Platform S44.007 – Thursday, April 26, 4:42-4:54 p.m. PT
- Comparative Effectiveness of Dimethyl Fumarate Versus Fingolimod and Teriflunomide on the Risk of Relapse in MS Patients Switching from First-generation Platform Therapies in the US – Poster P1.374 – Sunday, April 22, 11:30 a.m.-5:30 p.m. PT
- A Comparative Effectiveness Analysis Applying a 3-Way Propensity Score Matching to Real-world Data from the MSBase Registry in Preparation for a Cost-effectiveness Model: Patients Switching Within First-line Agents or to Either Natalizumab or Fingolimod in Active Relapsing-remitting Multiple Sclerosis (RRMS) – Poster P1.369 – Sunday, April 22, 11:30 a.m.-5:30 p.m. PT
- A Cost-effectiveness Analysis Using Real-world Data from the MSBase Registry: Comparing Natalizumab to Fingolimod in Patients with Inadequate Response to Disease-modifying Therapies in Relapsing-remitting Multiple Sclerosis (RRMS) in Scotland – Poster P1.364 – Sunday, April 22, 11:30 a.m.-5:30 p.m. PT
- Comparison of Techniques for Measurement of Brain Volume in Multiple Sclerosis Patients – Poster P3.354 – Tuesday, April 24, 11:30 a.m.-7:00 p.m. PT
- The Functional Brain Network Remains Stable in Natalizumab-treated Multiple Sclerosis Patients: A One Year Study – Poster P3.369 – Tuesday, April 24, 11:30 a.m.-7:00 p.m. PT
- The Multiple Sclerosis Partners Advancing Technology and Health Solutions (MS PATHS) Patient Cohort – Poster P4.381 – Wednesday, April 25, 11:30 a.m.-7:00 p.m. PT
- Natalizumab Extended Interval Dosing (EID) is Associated with a Significant Reduction in Progressive Multifocal Leukoencephalopathy (PML) Risk Compared with Standard Interval Dosing (SID) in the TOUCH® Prescribing Program – Poster P4.475 – Wednesday, April 25, 11:30 a.m.-7:00 p.m. PT
- Clinical Trial and Post-Marketing Reports Indicate No Increased Risk of Herpes Zoster in Patients Treated with Delayed-release Dimethyl Fumarate – Poster P5.362 – Thursday, April 26, 11:30 a.m.-7:00 p.m. PT
- Real-world Strategies for Management of Gastrointestinal Events in Patients Treated with Delayed-release Dimethyl Fumarate: EFFECT Gastrointestinal Sub-study Results – Poster P5.345 – Thursday, April 26, 11:30 a.m.-7:00 p.m. PT
- Longer Duration of Natalizumab Exposure May Lessen Return of Disease Activity Following a Switch from Natalizumab to an Oral Therapy: Modelling Real-world Data from Relapsing-remitting Multiple Sclerosis (RRMS) Patients in the TYSABRI®Observational Program (TOP) – Poster P6.379 – Friday, April 27, 11:30 a.m.-5:30 p.m. PT
- Real-world Improvement Across Functional Systems (FS) in Relapsing-remitting Multiple Sclerosis (RRMS) Patients Treated with Natalizumab in the TYSABRI Observational Program (TOP) – Poster P6.383 – Friday, April 27, 11:30 a.m.-5:30 p.m. PT
- Aducanumab Titration Dosing Regimen: 24-Month Analysis from PRIME, a Randomized, Double-Blind, Placebo-Controlled Phase 1b Study in Patients with Prodromal or Mild Alzheimer’s Disease – Platform S2.003 – Sunday, April 22, 1:24-1:36 p.m. PT
- Aducanumab 36-Month Data from PRIME: A Randomized, Double-blind, Placebo-controlled Phase 1b Study in Patients with Prodromal or Mild Alzheimer’s Disease – Platform S2.004 – Sunday, April 22, 1:36-1:48 p.m. PT
- Randomized, Double-blind, Placebo-controlled, Single Ascending Dose Study of Anti-alpha-synuclein Antibody BIIB054 in Patients with Parkinson’s Disease –Platform S26.001 – Tuesday, April 24, 3:30-3:42 p.m. PT
- Multiple Ascending Dose Study of the Tau-directed Monoclonal Antibody BMS-986168 in Patients with Progressive Supranuclear Palsy – Platform S27.004 – Wednesday, April 25, 8:24-8:32 a.m. PT
- Efficacy and Safety of BIIB092 in Patients with Progressive Supranuclear Palsy: PASSPORT Phase 2 Study Design – Poster P6.073 – Friday, April 27, 11:30 a.m.-5:30 p.m. PT
SPINRAZA is being developed globally for the treatment of SMA.
SPINRAZA is an antisense oligonucleotide (ASO), using Ionis Pharmaceutical Inc.’s proprietary antisense technology, that is designed to treat SMA caused by mutations or deletions in the SMN1 gene located in chromosome 5q that leads to SMN protein deficiency. SPINRAZA alters the splicing of SMN2 pre-mRNA in order to increase production of full-length SMN protein.2 ASOs are short synthetic strings of nucleotides designed to selectively bind to target RNA and regulate gene expression. Through use of this technology, SPINRAZA has the potential to increase the amount of full-length SMN protein in individuals with SMA.
SPINRAZA must be administered via intrathecal injection, which delivers therapies directly to the cerebrospinal fluid (CSF) around the spinal cord,3 where motor neurons degenerate in individuals with SMA due to insufficient levels of SMN protein.4
SPINRAZA demonstrated a favorable benefit-risk profile. The most common adverse reactions reported for SPINRAZA were upper respiratory infection, lower respiratory infection, and constipation. Serious adverse reactions of atelectasis were more frequent in SPINRAZA-treated patients. Coagulation abnormalities and thrombocytopenia, including acute severe thrombocytopenia, have been observed after administration of some ASOs. Individuals may be at increased risk of bleeding complications. Renal toxicity has been observed after administration of some ASOs. SPINRAZA is present in and excreted by the kidney.
For additional important safety information, and the United States full prescribing information, please visit www.spinraza.com or your respective country’s website.
TECFIDERA is an oral therapy for relapsing forms of MS, including relapsing-remitting MS, the most common form of MS. More than 310,000 patients have been treated with TECFIDERA worldwide with over 540,000 patient-years of experience.5 TECFIDERA has been proven to reduce the rate of MS relapses, slow the progression of disability, and impact the number of MS brain lesions, while demonstrating a favorable benefit-risk profile in people with relapsing forms of MS, notably newly diagnosed and early switch populations.6 In clinical trials, the most common adverse events associated with TECFIDERA were flushing and gastrointestinal (GI) events. Other side effects include a decrease in mean lymphocyte counts during the first year of treatment, which then plateaued, and liver function abnormalities, which resolved upon treatment discontinuation. TECFIDERA is contraindicated in patients with a known hypersensitivity to dimethyl fumarate or any of the excipients of TECFIDERA. Rare cases of progressive multifocal leukoencephalopathy (PML), a rare opportunistic viral infection of the brain which has been associated with death or severe disability, have been seen with TECFIDERA patients in the setting of prolonged moderate to severe lymphopenia.