Biogen Continues to Advance Innovation in SMA with New Data in
Adults and Infants to Be Presented at MDA Clinical and Scientific
Conference
Biogen (Nasdaq: BIIB) today announced it will present new data
illustrating the rapidly progressive nature of spinal muscular
atrophy (SMA) in adults, adolescents and older children at the
Muscular Dystrophy Association (MDA) Clinical and Scientific
Conference in Orlando, Florida (April 13-17, 2019). Other Biogen
presentations will highlight the benefits of pre-symptomatic
treatment with data from the NURTURE study, part of the SPINRAZA®
(nusinersen) global clinical trial program, and findings on the
role of neurofilament as a potential biomarker for predicting motor
function in SMA.
“The data show that, if left untreated, people with all types of
SMA continue to experience increased health challenges, and that
slowing the progression of the disease, even for those with less
severe forms, is critical to their ongoing health,” said Wildon
Farwell, M.D., senior medical director, clinical development at
Biogen. “With more than 6,600 individuals treated with SPINRAZA
across the post-marketing setting, expanded access program and
clinical trials and over six years of clinical data, we continue to
expand our in-depth knowledge of the disease and to gain real-world
experience on long-term safety and efficacy. We plan to
continue advancing research to help shape care for individuals with
SMA today and in the future.”
Biogen’s presentations on SMA and SPINRAZA include:
- Mortality Among a Large, Age-Diverse Population of Patients
With Spinal Muscular Atrophy (SMA): A U.S. Epidemiologic Study –
Poster 98 – Tuesday, April 16, 6:00 pm-8:00 pm ET
- Characteristics of Spinal Muscular Atrophy (SMA) Patients in
the neuroMuscular ObserVational Research (MOVR) Data Hub – Poster
100 – Tuesday, April 16, 6:00 pm-8:00 pm ET
- Phosphorylated Neurofilament Heavy Chain (pNF-H) and Motor
Function Achievement in Nusinersen-Treated Individuals With Spinal
Muscular Atrophy (SMA) – Poster 172 – Tuesday, April 16, 6:00
pm-8:00 pm ET
- Nusinersen in Infants Who Initiate Treatment in a
Presymptomatic Stage of Spinal Muscular Atrophy: Interim Results
From the Phase 2 NURTURE Study – Poster 174 – Tuesday, April 16,
6:00 pm-8:00 pm ET {Encore presentation}
- Symptoms and Complications Among Later Childhood, Adolescent
and Adult Spinal Muscular Atrophy Patients: A Natural History Study
within U.S. Hospitals – Poster Number 206 – Tuesday, April 16, 6:00
pm-8:00 pm 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 December 31, 2018, over 6,600 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. 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 been shown to increase
the amount of full-length SMN protein in individuals with SMA.
SPINRAZA is administered via intrathecal injection, which delivers
therapies directly into the cerebrospinal fluid (CSF) around the
spinal cord, where motor neurons degenerate in individuals with SMA
due to insufficient levels of SMN protein.
In the clinical trial program, SPINRAZA demonstrated a favorable
benefit-risk profile. The most common adverse reactions that
occurred in the SPINRAZA group were 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.
Biogen licensed the global rights to develop, manufacture and
commercialize SPINRAZA from Ionis Pharmaceuticals, Inc. (Nasdaq:
IONS), a leader in antisense therapeutics. Biogen and Ionis
conducted an innovative clinical development program, the largest
of its kind in SMA, that moved SPINRAZA from its first dose in
humans in 2011 to its first regulatory approval in five years.
About the SPINRAZA Clinical Program NURTURE is
an ongoing, open-label study of infants up to six weeks of age at
time of first dose, who were genetically diagnosed with SMA and had
not experienced any symptoms by the time of first dose. Data
presented at the World Muscle Society in October 2018 demonstrated
unprecedented efficacy in treating patients pre-symptomatically. In
that analysis, all NURTURE study participants were alive and did
not require permanent ventilation, in contrast to natural history
of SMA. Study participants achieved motor milestones with 100
percent sitting independently and 88 percent able to walk. All
NURTURE study participants were 14 months or older at the time of
the analysis. Participants included infants with two copies of the
SMN2 gene (n=15) who are likely to develop a fatal, early-onset
form of SMA known as Type 1, and infants with three copies of the
SMN2 gene (n=10) who typically develop SMA Type 2 or 3. People
living with SMA Types 2 and 3 may never be able to walk or will
lose that ability over time. No new safety concerns were
identified.
The ENDEAR study was a thirteen-month, international, phase 3,
multicenter, double-blind, sham-controlled study of 121 patients
with infantile-onset SMA (most likely to develop Type 1). Results
from the pivotal study, which were published in the New England
Journal of Medicine, evaluated the efficacy and safety in patients
that onset of signs and symptoms of SMA before six months of age.
Patients treated with SPINRAZA in the ENDEAR study achieved
clinically meaningful improvement in achievement of motor
milestones compared to untreated study participants with 51 percent
vs. 0 percent demonstrating Hammersmith Infant Neurological
Examination section 2 (HINE-2) motor milestone response, an
assessment which evaluates eight motor-milestone categories, based
on the defined criteria.
CHERISH was a fifteen-month, phase 3, randomized, double-blind,
sham-controlled study investigating SPINRAZA in 126 non-ambulatory
patients with later-onset SMA (most likely to develop SMA Type 2 or
3). Patients included had onset of signs and symptoms at greater
than 6 months of age, and an age of 2 to 12 years at screening. The
final analysis of CHERISH data found that children receiving
SPINRAZA experienced a highly statistically significant and
clinically meaningful improvement in motor function compared to
those who did not receive treatment with a treatment difference of
4.9 points on the Hammersmith Functional Motor Scale Expanded.
About SMA5 SMA is a rare, genetic,
neuromuscular disease that is characterized by loss of motor
neurons in the spinal cord and lower brain stem, resulting in
severe and progressive muscle atrophy and weakness. About 1 in
10,000 live births have a diagnosis of SMA. Ultimately, individuals
with SMA can lose the ability to walk and have difficulty
performing the basic functions of life, such as breathing and
swallowing, which results in significant healthcare intervention
and caregiver assistance. Left untreated, the majority of infants
with the most severe form of the disease (Type 1) do not live
beyond their second birthday without respiratory intervention.
People with childhood or adult onset SMA (Type 2 or 3) produce
greater amounts of SMN protein resulting in less severe, but still
life-altering forms of the disease.2
Due to a deletion of, or mutation in, the SMN1 gene, people with
SMA do not produce enough survival motor neuron (SMN) protein,
which is critical for the maintenance of motor neurons. The
severity of SMA correlates with the amount of SMN protein an
individual has. People with Type 1 SMA, the form that requires the
most intensive and supportive care, produce very little SMN protein
and do not achieve the ability to sit without support or typically
live beyond two years without respiratory support. People with Type
2 and Type 3 SMA produce greater amounts of SMN protein and have
less severe, but still life-altering forms of SMA.2
About Biogen At 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, MS and
neuroimmunology, movement disorders, neuromuscular disorders, acute
neurology, neurocognitive disorders, pain and ophthalmology. 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
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Biogen Safe Harbor This news release
contains forward-looking statements, including statements made
pursuant to the safe harbor provisions of the Private Securities
Litigation Reform Act of 1995, about the potential benefits, safety
and efficacy of SPINRAZA; the results of certain real-world data;
our research and development program for the treatment of SMA; the
identification and treatment of SMA; and risks and uncertainties
associated with drug development and commercialization. These
statements may be identified by words such as “aim,” “anticipate,”
“believe,” “could,” “estimate,” “expect,” “forecast,” “goal,”
“intend,” “may,” “plan,” “possible,” “potential,” “will,” “would”
and other words and terms of similar meaning. 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. You should not place undue reliance on these statements
or the scientific data presented.
These statements involve risks and uncertainties that could
cause actual results to differ materially from those reflected in
such statements, including without limitation the occurrence of
adverse safety events and/or unexpected concerns that may arise
from additional data or analysis; risks of unexpected costs or
delays; failure to protect and enforce our data, intellectual
property and other proprietary rights and uncertainties relating to
intellectual property claims and challenges; product liability
claims; and third party collaboration risks. 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 we have 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 news 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.
Reference: 1. 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.
2. Finkel R, Chiriboga C, Vajsar J, et al. Treatment of
infantile-onset spinal muscular atrophy with nusinersen: a phase 2,
open-label, dose-escalation study. Lancet.
2016;388(10063):3017-3026.
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. Darras B, Markowitz J, Monani U, De Vivo D. Chapter 8 -
Spinal Muscular Atrophies. In: Vivo BTD, ed. Neuromuscular
Disorders of Infancy, Childhood, and Adolescence (Second Edition).
San Diego: Academic Press; 2015:117-145.
MEDIA CONTACT:
David Caouette +1 617 679 4945
public.affairs@biogen.com |
INVESTOR
CONTACT: Matt Calistri +1 781 464 2442
IR@biogen.com |
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