- New data underscore benefits of
SPINRAZA® (nusinersen) across broad spinal muscular atrophy (SMA)
populations and include longer-term efficacy and safety assessments
from the SHINE study
- Data from Biogen’s multiple sclerosis
(MS) research programs include results from clinical investigation
of a potential biomarker for MS and new insights from MS PATHS, its
Learning Health System, which may inform future care
approaches
- Data from movement disorder programs
support further clinical development of Biogen’s investigational
compounds for Parkinson’s disease and progressive supranuclear
palsy
Biogen (Nasdaq: BIIB) announced it will present data from its
portfolio of marketed treatments and clinical development programs
for some of the most complex and difficult-to-treat
neurodegenerative diseases at the 70th annual meeting of the
American Academy of Neurology (AAN) in Los Angeles (April
21–27).
“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 ATROPHYPlatform Presentation
- 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
Posters
- 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
MULTIPLE SCLEROSISPlatform Presentations
- 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
Posters
- 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
PIPELINEPlatform Presentations
- 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
Poster
- 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
About SPINRAZA®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.
About TECFIDERA®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.
The efficacy and safety of TECFIDERA have been studied in a
large, global clinical program, which includes an ongoing long-term
extension study.
For additional important safety information, and the United
States full prescribing information, please
visit www.tecfidera.com or your respective country’s
website.
About TYSABRI®TYSABRI is a disease modifying
therapy (DMT) approved in more than 80 countries including the
United States, the European Union, Canada, Australia and
Switzerland. In the United States, TYSABRI is indicated as
monotherapy for the treatment of patients with relapsing forms of
MS. In the European Union, it is indicated as single disease
modifying therapy in adults with highly active relapsing-remitting
MS (RRMS) for patients with highly active disease activity despite
a full and adequate course of treatment with at least one DMT or
patients with rapidly evolving severe RRMS. TYSABRI is proven
effective, with over 10 years of experience in treating RRMS, and
more than 180,000 people treated worldwide and over 635,000
patient-years of experience.7
TYSABRI is a monoclonal antibody that selectively binds to
α4-integrin and is thought to interrupt the activity of
inflammatory cells in MS patients by blocking the interaction
between α4β1-integrin and vascular cell adhesion molecule-1.
Disruption of these molecular interactions prevents transmigration
of leukocytes across the endothelium into inflamed parenchymal
tissue. The specific mechanism(s) by which TYSABRI exerts its
effects in MS have not been fully defined.
TYSABRI has advanced the treatment of MS patients with its
proven ability to slow the progression of disability, reduce
relapse rates, and impact the number of MRI brain lesions with a
well-characterized safety profile. Data from the Phase 3 AFFIRM
trial, which was published in the New England Journal of
Medicine, showed that at two years, TYSABRI treatment led to a 68
percent relative reduction (p<0.001) in the annualized relapse
rate when compared with placebo and reduced the relative risk of
disability progression by 42 to 54 percent (12-24-week sustained
respectively, both p<0.001). TYSABRI increases the risk of PML,
a rare opportunistic viral infection of the brain which has been
associated with death or severe disability. Risk factors that
increase the risk of PML are the presence of anti-JCV antibodies,
prior immunosuppressant use and longer TYSABRI treatment duration.
Patients who have all three risk factors have the highest risk of
developing PML. TYSABRI increases the risk of developing
encephalitis and meningitis caused by herpes simplex and varicella
zoster viruses and clinically significant liver injury has also
been reported in the post-marketing setting. Serious,
life-threatening, and sometimes fatal cases have been reported in
the postmarketing setting in MS patients receiving TYSABRI. Other
serious adverse events that have occurred in TYSABRI-treated
patients include hypersensitivity reactions (e.g., anaphylaxis) and
infections, including opportunistic and other atypical infections.
Clinically significant liver injury has also been reported in the
post-marketing setting.
The overall benefit-risk profile of TYSABRI remains positive.
For additional important safety information and the full United
States prescribing information which includes a full list of
adverse events, please visit www.tysabri.com or your
respective country’s website.
About BiogenAt Biogen, our mission is clear: we are
pioneers in neuroscience. Biogen discovers, develops, and delivers
worldwide innovative therapies for people living with serious
neurological and neurodegenerative diseases. One of the world’s
first global biotechnology companies, Biogen was founded in 1978 by
Charles Weissmann, Heinz Schaller, Kenneth Murray and Nobel Prize
winners Walter Gilbert and Phillip Sharp, and today has the leading
portfolio of medicines to treat multiple sclerosis; has introduced
the first and only approved treatment for spinal muscular atrophy;
and is focused on advancing neuroscience research programs in
Alzheimer’s disease and dementia, multiple sclerosis and
neuroimmunology, movement disorders, neuromuscular disorders, pain,
ophthalmology, neuropsychiatry, and acute neurology. Biogen also
manufactures and commercializes biosimilars of advanced
biologics.
We routinely post information that may be important to investors
on our website at www.biogen.com. To learn more, please visit
www.biogen.com and follow us on social media – Twitter, LinkedIn,
Facebook, YouTube.
Biogen Safe HarborThis press release contains
forward-looking statements made pursuant to the safe harbor
provisions of the Private Securities Litigation Reform Act of 1995
about additional results from the Phase 2 EMBRACE study, Phase 2
SHINE study, and CS2/CS12 study of SPINRAZA, the Phase 3 EVOLVE
MS-1 study of BIIB098, the Phase 1b study of aducanumab, the Phase
1 study of BIIB092, and/or the Phase 1 study of BIIB054, the
potential clinical effects of SPINRAZA, TECFIDERA, TYSABRI,
BIIB098, aducanumab, BIIB092 and/or BIIB054, the identification and
treatment of MS and Alzheimer’s disease, the treatment of SMA, and
clinical studies on Parkinson’s disease and PSP. These statements
may be identified by words such as “aim,” “anticipate,” “believe,”
“could,” “estimate,” “expect,” “forecast,” “intend,” “may,” “plan,”
“possible,” “potential,” “will,” and other words and terms of
similar meaning, and are based on our current beliefs and
expectations. You should not place undue reliance on these
statements or the scientific data presented. Drug development and
commercialization involve a high degree of risk, and only a small
number of research and development programs result in
commercialization of a product. Results in early stage clinical
trials may not be indicative of full results or results from later
stage or larger scale clinical trials and do not ensure regulatory
approval.
These statements involve risks and uncertainties that could
cause actual results to differ materially from those reflected in
such statements, including without limitation, the risk that we may
not fully enroll our clinical trials or enrollment will take longer
than expected, unexpected concerns may arise from additional data,
analysis or results obtained during our clinical trials, regulatory
authorities may require additional information or further studies,
or may fail or refuse to approve or may delay approval of our drug
candidates, the occurrence of adverse safety events, or we may
encounter other unexpected hurdles. The foregoing sets forth many,
but not all, of the factors that could cause actual results to
differ from our expectations in any forward-looking statement.
Investors should consider this cautionary statement, as well as the
risk factors identified in our most recent annual or quarterly
report and in other reports filed with the Securities and Exchange
Commission. These statements are based on our current beliefs and
expectations and speak only as of the date of this press release.
We do not undertake any obligation to publicly update any
forward-looking statements, whether as a result of new information,
future developments, or otherwise.
1 World Health Organization. Neurological disorders: Public
health challenges. 2007. Available at:
http://www.who.int/mental_health/neurology/chapter_4_neuro_disorders_public_h_challenges.pdf?ua=1.2
Hua Y, Sahashi K, Hung G, Rigo F, Passini MA, Bennett CF, Krainer
AR. Antisense correction of SMN2 splicing in the CNS rescues
necrosis in a type III SMA mouse model. Genes Dev. 2010 Aug 1;
24(15):16344-44.3 Evers MM, Toonen LJ, van Roon-Mom WM. Antisense
oligonucleotides in therapy for neurodegenerative
disorders. Adv Drug Deliv Rev. 2015;87:90-103.4 Lunn MR,
Wang CH. Spinal muscular
atrophy. Lancet. 2008;371(9630):2120-2133.5 Combined
post-marketing and clinical trials exposure to TECFIDERA as of 31
January 2018.6 TECFIDERA is approved in the European Union for
relapsing-remitting multiple sclerosis.7 Global Natalizumab
(TYSABRI) Postmarketing PML Update, March 2018.
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BiogenMEDIA CONTACT:David Caouette, +1
617-679-4945public.affairs@biogen.comorINVESTOR CONTACT:Matt
Calistri, +1 781-464-2442IR@biogen.com
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