Presymptomatic Infants Continued to Achieve
Motor Milestones Generally Consistent with Normal Development in
New Interim Data Analysis
Positive Data Across a Broad Range of
Individuals with SMA Presented at the American Academy of Neurology
Annual Meeting
Biogen (NASDAQ:BIIB) will present Phase 3 end of study SPINRAZA®
(nusinersen) data from CHERISH, which demonstrated a highly
statistically significant and clinically meaningful improvement in
motor function in children with later-onset (most likely to develop
Type 2 or Type 3) spinal muscular atrophy (SMA) compared to
untreated children. The overall findings continue to support the
robust efficacy and favorable safety profile of SPINRAZA across a
broad range of individuals with SMA. The SPINRAZA development
program represents the largest body of clinical data of its kind in
SMA. SPINRAZA data will be presented at the American Academy of
Neurology (AAN) annual meeting in Boston, Mass., April 22-28,
2017.
“The CHERISH study, conducted in collaboration with Ionis,
further demonstrates the meaningful impact SPINRAZA can have in
children with later-onset SMA, and reaffirms the benefit of
treatment across SMA populations,” said Alfred Sandrock, M.D.,
Ph.D., executive vice president and chief medical officer at
Biogen. “Our clinical development program demonstrates the impact
of early treatment, which is confirmed by NURTURE data showing
significant motor milestone improvements generally consistent with
normal development in presymptomatic infants treated with
SPINRAZA.”
CHERISH: Later-onset SMA (Most Likely to Develop Type 2 or
Type 3)
CHERISH is a Phase 3, multicenter, randomized, double-blind,
sham-procedure controlled study to assess the efficacy and safety
of SPINRAZA in children with later-onset SMA. The 15-month study
investigated SPINRAZA in 126 non-ambulatory children 2 to 12 years
old who experienced symptom onset at greater than 6 months of
age.
In the CHERISH end of study analysis, children on SPINRAZA
demonstrated a highly statistically significant and clinically
meaningful improvement in motor function, as observed by the
treatment difference of 4.9 points in the mean change from baseline
to Month 15 in the Hammersmith Functional Motor Scale Expanded
(HFMSE) score (p=0.0000001). The HFMSE is a validated tool
specifically designed to assess motor function in children with
SMA. When measuring changes from baseline, children who received
SPINRAZA (n=84) achieved a 3.9 point mean improvement at Month 15,
while children who were not on treatment (n=42) experienced a mean
decline of 1.0 point. Primary endpoint results of the end of study
analysis were consistent with results observed at the interim
analysis.
Data from the other endpoints analyzed, including attainment of
new motor milestones and upper limb motor function, were
consistently in favor of children who received treatment.
SPINRAZA demonstrated a favorable safety profile.
Treatment-emergent adverse events (AEs), severe AEs and serious AEs
(SAEs) were reported less frequently in children treated with
SPINRAZA than those not on treatment. The majority of the AEs were
considered to be either related to SMA disease, common events in
the general population, or events related to the lumbar puncture
procedure. No children discontinued the study due to AEs.
“In CHERISH, most children with later-onset SMA treated with
SPINRAZA saw improvements in motor function and stabilization or
slowing of disease progression,” said Dr. Richard Finkel, chief of
neurology, Nemours Children's Hospital, Orlando, Florida. “As a
physician who has spent 37 years treating children with SMA, it’s
incredibly encouraging to see some patients on SPINRAZA achieve
milestones such as crawling and standing with assistance within the
clinical trial. These kinds of clinically meaningful improvements
are unprecedented and give new hope to individuals with SMA and
their families.”
NURTURE: Presymptomatic Infants with SMA
Biogen will also present new interim data from the Phase 2,
multicenter, open-label, single-arm NURTURE study evaluating
SPINRAZA for the treatment of infants under six weeks old with
genetically diagnosed SMA who were presymptomatic at treatment
initiation. At the time of the interim analysis, infants (n=20)
were enrolled for a median of 317.5 days, and all infants were
alive and none required respiratory intervention (chronic
non-invasive ventilation, invasive ventilation or tracheostomy).
Further, most infants achieved motor milestone and growth parameter
gains generally consistent with normal development, such as head
control, independent sitting, standing and walking independently,
as measured by validated scales.
Three infants experienced AEs considered possibly related to
SPINRAZA by the investigator, all of which were resolved. No
infants have discontinued or withdrawn from the study due to AEs,
and no new safety concerns have been identified.
“The results from NURTURE are significant, as they continue to
demonstrate the importance of beginning SPINRAZA treatment as soon
as possible after an SMA diagnosis and the major impact that early
treatment may have across a broad range of SMA populations,” said
Sandrock.
For more information about SPINRAZA and U.S. prescribing
information, visit www.SPINRAZA.com.
The CHERISH and NURTURE slide presentations will be available
concurrently with the AAN sessions on the Investor section of the
Biogen company website, www.Biogen.com.
SPINRAZA Program Status
Biogen licensed the global rights to develop, manufacture and
commercialize SPINRAZA from Ionis Pharmaceuticals (NASDAQ:IONS), a
leader in antisense therapeutics. Biogen and Ionis conducted an
innovative clinical development program that moved SPINRAZA from
its first dose in humans in 2011 to its first regulatory approval
by the United States Food and Drug Administration (FDA) in
2016.2
SPINRAZA was first approved by the FDA on December 23, 2016
within three months of regulatory filing for the treatment of SMA
in pediatric and adult patients. In April 2017, the Committee for
Medicinal Products for Human Use (CHMP) of the European Medicines
Agency (EMA) adopted a positive opinion recommending the granting
of a marketing authorization for SPINRAZA for the treatment of 5q
SMA, following review under an Accelerated Assessment program. A
decision from the European Commission (EC) is expected in the next
few months. Biogen has also submitted regulatory filings in Japan,
Canada, Australia and Switzerland and plans to initiate additional
filings in other countries in 2017.
About SMA 2-6
Spinal muscular atrophy (SMA) is characterized by loss of motor
neurons in the spinal cord and lower brain stem, resulting in
severe and progressive muscular atrophy and weakness. Ultimately,
individuals with the most severe type of SMA can become paralyzed
and have difficulty performing the basic functions of life, like
breathing and swallowing.
Due to a loss of, or defect 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. 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 live beyond two years without respiratory
support. People with Type 2 or Type 3 SMA produce greater amounts
of SMN protein and have less severe, but still life-altering forms
of SMA.
To support awareness and education about SMA, Biogen has
launched Together in SMA in the United States and Japan. Together
in SMA is a program created to provide informational materials and
resources to the SMA community. Learn more
at www.TogetherinSMA.com (U.S.-only)
and www.TogetherinSMA.jp/ (Japan-only).
About SPINRAZA™ (nusinersen)
SPINRAZA is being developed globally for the treatment of
SMA.
SPINRAZA is an antisense oligonucleotide (ASO), using Ionis
Pharmaceuticals’ 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.7 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 patients with SMA.
SPINRAZA is administered via intrathecal injection, which
delivers therapies directly to the cerebrospinal fluid (CSF) around
the spinal cord,8 where motor neurons degenerate in patients with
SMA due to insufficient levels of SMN protein.9
The most common adverse reactions reported for SPINRAZA were
lower respiratory infection, upper 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 antisense
oligonucleotides. Individuals may be at increased risk of bleeding
complications. Renal toxicity has been observed after
administration of some antisense oligonucleotides. SPINRAZA is
present in and excreted by the kidney.
For complete SPINRAZA U.S. prescribing information please
visit www.SPINRAZA.com.
About Patient Support in the U.S.
As part of Biogen’s commitment to patients and families living
with SMA, the company has launched SMA360°™, which provides certain
services that address nonmedical barriers to access in the U.S.
These include logistical assistance, product education, insurance
benefits investigations and financial assistance. A list of the
SMA360° offerings is available at www.SPINRAZA.com.
SMA360° services from Biogen are available only to those
eligible patients who have been prescribed SPINRAZA in the U.S. To
learn more about the program and receive additional information
about these services, please contact an SMA Support Coordinator at
1-844-4SPINRAZA (1-844-477-4672) Monday-Friday 8:30
a.m.-8:00 p.m. EST.
About Biogen
Through cutting-edge science and medicine, Biogen discovers,
develops and delivers innovative therapies worldwide for people
living with serious neurological and neurodegenerative diseases.
Founded in 1978, Biogen is a pioneer in biotechnology and today the
Company has the leading portfolio of medicines to treat multiple
sclerosis, has introduced the first and only approved treatment for
spinal muscular atrophy, and is at the forefront of neurology
research for conditions including Alzheimer’s disease, Parkinson’s
disease and amyotrophic lateral sclerosis. Biogen also manufactures
and commercializes biosimilars of advanced biologics. For more
information, please visit www.biogen.com. Follow us on social media
– Twitter, LinkedIn, Facebook, YouTube.
Biogen Safe Harbor
This press release contains forward-looking statements,
including statements relating to the potential benefits, safety and
efficacy of SPINRAZA, the status of current regulatory filings, and
plans for additional regulatory filings in other jurisdictions.
These statements may be identified by words such as “believe,”
“except,” “may,” “plan,” “potential,” “will” and similar
expressions, and are based on our current beliefs and expectations.
You should not place undue reliance on these statements. These
statements involve risks and uncertainties that could cause actual
results to differ materially from those reflected in such
statements, including uncertainty of success in commercialization
of SPINRAZA, which may be impacted by, among other things, the
level of preparedness of healthcare providers to treat patients,
difficulties in obtaining or changes in the availability of
reimbursement for SPINRAZA, the effectiveness of sales and
marketing efforts, problems with the manufacturing process for
SPINRAZA, the occurrence of adverse safety events, failure to
obtain regulatory approvals in other jurisdictions, failure to
protect intellectual property and other proprietary rights, product
liability claims, third party collaboration risks, and the other
risks and uncertainties that are described in the Risk Factors
section of Biogen’s most recent annual or quarterly report and in
other reports Biogen has filed with the U.S. Securities and
Exchange Commission (SEC). Any forward-looking statements speak
only as of the date of this press release and we assume no
obligation to update any forward-looking statement.
1. Biogen. SPINRAZA USPI. December 2016. 2. 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. 3. Lefebvre S, Burglen L, Reboullet S, et al.
Identification and characterization of a spinal muscular
atrophy-determining gene. Cell.1995;80(1):155-165. 4. Mailman MD,
Heinz JW, Papp AC, et al. Molecular analysis of spinal muscular
atrophy and modification of the phenotype by SMN2. Genet Med.
2002;4(1):20-26. 5. Monani UR, Lorson CL, Parsons DW, et al. A
single nucleotide difference that alters splicing patterns
distinguishes the SMA gene SMN1 from the copy gene SMN2. Hum Mol
Genet. 1999;8(7):1177-1183. 6. Peeters K, Chamova T, Jordanova A.
Clinical and genetic diversity of SMN1-negative proximal spinal
muscular atrophies. Brain.2014;137(Pt 11):2879-2896. 7. 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. 8. Evers MM, Toonen LJ, van Roon-Mom WM. Antisense
oligonucleotides in therapy for neurodegenerative disorders. Adv
Drug Deliv Rev. 2015;87:90-103. 9. Lunn MR, Wang CH. Spinal
muscular atrophy. Lancet. 2008;371(9630):2120-2133.
View source
version on businesswire.com: http://www.businesswire.com/news/home/20170424005505/en/
MEDIA:BiogenLigia Del Bianco, +1
781-464-3260public.affairs@biogen.comorINVESTORS:BiogenBen
Strain, +1 781-464-2442IR@biogen.com
Ionis Pharmaceuticals (NASDAQ:IONS)
Historical Stock Chart
From Mar 2024 to Apr 2024
Ionis Pharmaceuticals (NASDAQ:IONS)
Historical Stock Chart
From Apr 2023 to Apr 2024