Biogen To Present Data At AAN Highlighting Its Innovative Marketed Treatments And Investigational Pipeline Programs For Complex Neurodegenerative Diseases

— Data demonstrate that pre-symptomatic infants with SMA treated with SPINRAZA® (nusinersen) over three years achieved motor milestones that are more consistent with normal childhood development — Late-breaking data expand on safety and exploratory efficacy results of a study investigating tofersen (BIIB067) for the treatment of amyotrophic lateral sclerosis with a confirmed superdioxide dismutate 1 (SOD1) mutation — Data from industry-leading multiple sclerosis (MS) portfolio and pipeline highlight ongoing innovations to meet the diverse needs of people living with MS, including additional pivotal data for diroximel fumarate

CAMBRIDGE, Mass., May 01, 2019 (GLOBE NEWSWIRE) — Biogen Inc. (Nasdaq: BIIB) today announced it will present new data across its industry-leading neuroscience portfolio and advancing clinical development programs at the 71st annual meeting of the American Academy of Neurology (AAN) in Philadelphia, PA. (May 4–11). Of Biogen’s 33 total presentations, key data will focus on spinal muscular atrophy (SMA), multiple sclerosis (MS) and amyotrophic lateral sclerosis (ALS).

“Biogen stands at the forefront of neuroscience, taking on some of the most challenging diseases in history as part of our steadfast commitment to supporting patients with a range of complex and rare neurological disorders,” said Alfred Sandrock, Jr., M.D., Ph.D., executive vice president and chief medical officer at Biogen. “Our data at AAN illustrate our drive to advance critical research for people who have limited or no treatment options.”

Research across Biogen’s clinical and development programs demonstrates the company’s commitment to further understanding neurodegenerative diseases with unmet need. SMA and MS data focus on validating biomarkers that aid in monitoring and detecting disease progression, while pipeline data underscore the company’s emphasis on thoughtful study design and understanding the patient journey.

SMA Data Presentations Reinforce SPINRAZA as Standard of Care in Treating SMA, Underscoring Therapy’s Long-Term Benefit for Infants, Children and Adults

— Results from the NURTURE study demonstrate that pre-symptomatic infants with SMA treated with SPINRAZA over three years achieved motor milestones more consistent with normal childhood development. — After three years of follow-up, interim results from the ENDEAR/CHERISH/SHINE open-label extension study show that treatment with SPINRAZA, particularly when initiated earlier, leads to progressive motor milestone improvements and increased survival rates for individuals with infantile-onset SMA. — Additional data suggest that phosphorylated neurofilament heavy chain (pNF-H) may emerge as a promising biomarker to predict disease activity in SMA.

Advancing Biogen’s Pipeline of Investigational Treatments for Neurodegenerative Diseases

— In a late-breaking presentation, Biogen will share expanded findings from its phase 1/2 study of tofersen (BIIB067) for the investigational treatment of ALS with a confirmed SOD1 mutation. Positive interim results of the study, as well as Biogen’s decision to exercise its option to obtain a license from Ionis Pharmaceuticals Inc. to develop and commercialize the asset, were announced in December 2018. Biogen has advanced the program to the ongoing phase 3 clinical study (VALOR), which is a continuation of the Phase 1/2 study. Biogen is collaborating with regulators to further define the scope of the clinical data package required to support the registration of BIIB067. — Other pipeline data to be presented advance the scientific community’s understanding of rare neurodegenerative diseases, including progressive supranuclear palsy (PSP), and provide updates on Biogen’s investigational programs for stroke and neuropathic pain.

Advancing Biogen’s Portfolio of Leading MS Therapies

— Safety and exploratory efficacy results from the ongoing pivotal EVOLVE-MS-1 study with diroximel fumarate (BIIB098) will be shared, which support the potential of Biogen and Alkermes’ investigational oral treatment as a new option for patients with relapsing MS. If approved, diroximel fumarate will be marketed under the brand name VUMERITY™, which has been conditionally accepted by the FDA and will be confirmed upon approval. — Committed to improving patient care and further understanding the benefit-risk profile of TYSABRI® (natalizumab), Biogen will present updated safety analyses evaluating extended interval dosing of natalizumab (of approximately every 6 weeks) compared to every 4-week dosing. — Biogen continues to engage in collaborative research efforts to identify potential biomarkers in MS, and will present results further confirming serum neurofilament light (sNfL) as a clinically useful tool for disease prognosis and treatment management.

Highlights of Biogen’s Platform and Poster Presentations:


— Nusinersen in Infants Who Initiate Treatment in a Presymptomatic Stage of Spinal Muscular Atrophy (SMA): Interim Efficacy and Safety Results From the Phase 2 NURTURE Study – Platform S25.001 – Tuesday, May 7, 1:00 p.m. ET — Interim Report on the Safety and Efficacy of Longer-Term Treatment With Nusinersen in Infantile-Onset Spinal Muscular Atrophy (SMA): Updated Results From the SHINE Study – Platform S25.004 – Tuesday, May 7, 1:33 p.m. ET — Association of Phosphorylated Neurofilament Heavy Chain (pNF-H) Levels With Motor Function Achievement in Individuals With Spinal Muscular Atrophy (SMA) Treated With Nusinersen – Platform S27.009 –Tuesday, May 7, 2:28 p.m. ET

Poster Presentations

— Symptoms and Complications Over Three Years Among Later Childhood, Adolescent and Adult Spinal Muscular Atrophy Patients: A Natural History Study Within U.S. Hospitals – Poster P1.6-051 – Sunday, May 5, 11:30 a.m. – 6:30 p.m. ET — Ambulation Status, Role Participation and Caregiver Assistance Among Individuals With Spinal Muscular Atrophy Type III: Results from the Cure SMA Membership Survey – Poster P1.6-061 –Sunday, May 5, 11:30 a.m. – 6:30 p.m. ET — Interim Report on the Safety and Efficacy of Longer-Term Treatment With Nusinersen in Later-onset Spinal Muscular Atrophy (SMA): Results from the SHINE Study – Poster P1.6-063 – Sunday, May 5, 11:30 a.m. – 6:30 p.m. ET

PIPELINEEmerging Science (Late Breaking) Presentation

— Safety, PK, PD, and Exploratory Efficacy in Single and Multiple Dose Study of a SOD1 Antisense Oligonucleotide (BIIB067) Administered to Participants With ALS – Poster 007–Tuesday, May 7, 11:45 a.m. – 12:45 p.m. ET

Poster Presentations

— CONVEY, A Phase 2 Placebo-Controlled, Double-Blind, Enriched Enrollment Randomized Withdrawal Study Design of Vixotrigine for the Treatment of Pain in Participants With Confirmed Small Fiber Neuropathy – Poster P2.6-068 – Monday, May 6, 11:30 a.m. – 6:30 p.m. ET — Design of a Phase III Study of Intravenous Glibenclamide (BIIB093) for Large Hemispheric Infarction: the CHARM Study – Poster P2.3-036 – Monday, May 6, 11:30 a.m. – 6:30 p.m. ET — The Diagnostic Journey of Patients With Progressive Supranuclear Palsy (PSP) in US Electronic Medical Record Data – Poster P3.8-007 – Tuesday, May 7, 11:30 a.m. – 6:30 p.m. ET — Incidence of Venous Thromboembolic Events Among Amyotrophic Lateral Sclerosis in a US Health Insurance Claims Database – Poster P4.6-003 – Wednesday, May 8, 11:30 a.m. – 6:30 p.m. ET

MULTIPLE SCLEROSISPlatform Presentations

— Natalizumab Reduces Serum Concentrations of Neurofilament Light Chain in Secondary Progressive Multiple Sclerosis Patients From the Phase 3 ASCEND Study – Platform S12.008 – Monday, May 6,1:00 – 3:00 p.m. ET — Reduced Risk of Progressive Multifocal Leukoencephalopathy (PML) Associated With Natalizumab Extended Interval Dosing (EID): Updated Analysis of the TOUCH® Prescribing Program Database – PlatformS26.006 – Tuesday, May 7, 1:00 – 3:00 p.m. ET — Serum Neurofilament Light (sNfL) for Disease Prognosis and Treatment Monitoring in Multiple Sclerosis Patients: Towards Implementation into Clinical Care – PlatformS26.001 – Tuesday, May 7,1:00 – 3:00 p.m. ET — Pregnancy and Infant Outcomes With Interferon Beta: Data From the European Interferon Beta Pregnancy Registry and MS Preg Study Conducted in Finland and Sweden –Platform S49.005 – Thursday, May 9, 1:00 – 3:00 p.m. ET

Poster Presentations

— Dimethyl Fumarate Exerts Selective Effects on Key B Cell Subsets and IgG Levels Which May Contribute to Its Therapeutic Benefit in MS While Maintaining Protective Humoral Immunity – Poster P.2.2-069 – Monday, May 6,11:30 a.m. – 6:30 p.m. ET — Diroximel Fumarate (DRF) in Patients With Relapsing-Remitting Multiple Sclerosis: Interim Efficacy and Safety Results From the Phase 3 EVOLVE-MS- Study – Poster P3.2-060 –Tuesday, May 7, 11:30 a.m. – 6:30 p.m. ET — Effects of Dimethyl Fumarate on Brain Volume Change in Relapsing-remitting Multiple Sclerosis: A Pooled Analysis of the Phase 3 DEFINE and CONFIRM Studies – Poster P3.2-064 –Tuesday, May 7, 11:30 a.m. – 6:30 p.m. ET — Evaluating the Efficacy and Safety of 6-Week Extended Interval Dosing of Natalizumab via a Prospective, Controlled, Randomized, Open-Label, Rater-Blinded Phase 3b Study – Poster P3.2-095 – Tuesday,May 7, 11:30 a.m. – 6:30 p.m. ET — Longitudinal Stability of Anti–JC Virus (JCV) Antibody Index Over 2 Years in Multiple Sclerosis (MS) Patients Treated With Natalizumab in the ASCEND Study –Poster P4.2-009 –Wednesday, May 8 11:30 a.m. – 6:30 p.m. ET — Delayed-release Dimethyl Fumarate Treatment Shifts the Immune Repertoire in Patients With Relapsing-Remitting Multiple Sclerosis: Results of PROCLAIM, an Open-label Phase 3 Study – PosterP4.2-053 –Wednesday, May 8 11:30 a.m. – 6:30 p.m. ET

About SPINRAZA® (nusinersen)1-4SPINRAZA is the first and only approved medicine for the treatment of spinal muscular atrophy (SMA) and is currently available in more than 40 countries. As of March 31, 2019 over 7,500 individuals with SMA are being treated with SPINRAZA worldwide, based on patients across the post-marketing setting, Expanded Access Program (EAP) and clinical trial participants.

SPINRAZA is an antisense oligonucleotide (ASO) developed using Ionis’ proprietary antisense technology that is designed to treat the root cause of SMA. SPINRAZA alters the splicing of SMN2 pre-mRNA in order to increase production of full-length SMN protein. […]

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