- Interim results from RESPOND study show improved motor
function in most participants treated with SPINRAZA after
Zolgensma® (onasemnogene abeparvovec)
CARLSBAD, Calif., June 30,
2023 /PRNewswire/ -- Ionis Pharmaceuticals (Nasdaq:
IONS) today announced that its partner Biogen presented new data
highlighting the potential benefit of SPINRAZA®
(nusinersen) in infants and toddlers living with spinal muscular
atrophy (SMA). Interim results from the RESPOND study showed
improved motor function in most participants treated with SPINRAZA
following treatment with Zolgensma® (onasemnogene
abeparvovec). The data were presented today at the SMA Research
& Clinical Care Meeting hosted by Cure SMA.
RESPOND is an ongoing two-year, Phase 4 open-label study to
evaluate clinical outcomes and safety following treatment with
SPINRAZA in infants and toddlers with SMA who have unmet medical
needs after treatment with Zolgensma. Interim efficacy results at
six months from 29* study participants treated with SPINRAZA
show:
- Improvements in motor function in most participants as measured
by increased mean total Hammersmith Infant Neurological Examination
Section 2 (HINE-2) score from baseline.
-
- Participants with two SMN2 copies (n=24) improved by a mean of
over 5 points on HINE-2.
- All participants with three SMN2 copies (n=3) improved; a mean
change from baseline was not calculated due to the small number of
participants.
- Most participants (25/27) with investigator-reported suboptimal
motor function at baseline improved.
After a median of 230.5 days in the study, serious adverse
events (AEs) were reported in 13/38 (34%) participants. No serious
AEs were considered related to SPINRAZA or led to study withdrawal.
No new emerging safety concerns have been identified in enrolled
participants who received SPINRAZA after Zolgensma. Additional
interim clinical outcomes from the RESPOND study are being
presented at the conference.
"SPINRAZA is a foundation of care for people living with spinal
muscular atrophy. The early results from RESPOND show that SPINRAZA
may further improve muscle performance in patients treated with
gene therapy whose outcomes have not met clinical expectations,"
said C. Frank Bennett, Ph.D.,
executive vice president and chief scientific officer of Ionis.
New Analysis Evaluating Real-World Impact of SPINRAZA
A systematic literature review and meta-analysis evaluating
real-world impact of SPINRAZA for infantile-onset SMA was presented
and highlights the importance of generating real-world evidence to
achieve a comprehensive understanding of the treatment benefits of
SPINRAZA. Improvements in motor function and motor milestones
observed in real-world studies were greater than or comparable to
those observed in clinical trials, and patients continued to
improve with longer duration of SPINRAZA treatment.
About SPINRAZA® (nusinersen)
SPINRAZA is approved in more than 60 countries to treat infants,
children and adults with spinal muscular atrophy (SMA). As a
foundation of care in SMA, more than 14,000 individuals have been
treated with SPINRAZA worldwide.1
SPINRAZA is an antisense oligonucleotide (ASO) that targets the
root cause of SMA by continuously increasing the amount of
full-length survival motor neuron (SMN) protein produced in the
body.2 It is administered directly into the central
nervous system, where motor neurons reside, to deliver treatment
where the disease starts.3
SPINRAZA has demonstrated sustained efficacy across ages and SMA
types with a well-established safety profile based on data in
patients treated up to 8 years,4 combined with
unsurpassed real-world experience. The nusinersen clinical
development program encompasses more than 10 clinical studies,
which have included more than 460 individuals across a broad
spectrum of patient populations, including two randomized
controlled studies (ENDEAR and CHERISH). The SHINE and NURTURE
open-label extension studies are evaluating the long-term impact of
SPINRAZA. The most common adverse events observed in clinical
studies were respiratory infection, fever, constipation, headache,
vomiting and back pain. Laboratory tests can monitor for renal
toxicity and coagulation abnormalities, including acute severe low
platelet counts, which have been observed after administration of
some ASOs.
Biogen licensed the global rights to develop, manufacture and
commercialize SPINRAZA from Ionis. Please click here for Important
Safety Information and full Prescribing Information for SPINRAZA in
the U.S., or visit your respective country's product
website.
About Ionis Pharmaceuticals, Inc.
For more than 30 years, Ionis has been a leader in RNA-targeted
therapy, pioneering new markets and changing standards of care.
Ionis currently has four marketed medicines and a promising
late-stage pipeline highlighted by cardiovascular and neurological
franchises. Our scientific innovation began and continues with the
knowledge that sick people depend on us, which fuels our vision to
become the leader in genetic medicine, utilizing a multi-platform
approach to discover, develop and deliver life-transforming
therapies.
To learn more about Ionis visit www.ionispharma.com and follow
us on Twitter @ionispharma.
Ionis' Forward-looking Statements
This press release includes forward-looking statements regarding
Ionis' business and the therapeutic and commercial potential of
SPINRAZA®, Ionis' technologies and other products in
development. Any statement describing Ionis' goals, expectations,
financial or other projections, intentions or beliefs is a
forward-looking statement and should be considered an at-risk
statement. Such statements are subject to certain risks and
uncertainties including those related to our commercial products
and the medicines in our pipeline, and particularly those inherent
in the process of discovering, developing and commercializing
medicines that are safe and effective for use as human
therapeutics, and in the endeavor of building a business around
such medicines. Ionis' forward-looking statements also involve
assumptions that, if they never materialize or prove correct, could
cause its results to differ materially from those expressed or
implied by such forward-looking statements. Although Ionis'
forward-looking statements reflect the good faith judgment of its
management, these statements are based only on facts and factors
currently known by Ionis. As a result, you are cautioned not to
rely on these forward-looking statements. These and other risks
concerning Ionis' programs are described in additional detail in
Ionis' annual report on Form 10-K for the year ended Dec. 31, 2022, and the most recent Form 10-Q
quarterly filing, which are on file with the Securities and
Exchange Commission. Copies of these and other documents are
available from the Company.
In this press release, unless the context requires otherwise,
"Ionis," "Company," "we," "our," and "us" all refer to Ionis
Pharmaceuticals and its subsidiaries.
Ionis Pharmaceuticals® is a registered trademark of Ionis
Pharmaceuticals, Inc. SPINRAZA® is a registered
trademark of Biogen.
*Two participants in the RESPOND study were not assessed at Day
183 and, therefore, not included in the mean calculation.
1 Based on commercial patients, early access
patients, and clinical trial participants through December 31, 2022.
2 SPINRAZA U.S. Prescribing Information. Available at:
https://www.spinraza.com/content/dam/commercial/specialty/spinraza/caregiver/en_us/pdf/spinraza-prescribing-information.pdf.
Accessed: June 2023.
3 SPINRAZA U.S. Prescribing Information. Available at:
https://www.spinraza.com/content/dam/commercial/specialty/spinraza/caregiver/en_us/pdf/spinraza-prescribing-information.pdf.
Accessed: June 2023.
4 Core Data sheet, Version 13, October 2021. SPINRAZA. Biogen Inc, Cambridge, MA.
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SOURCE Ionis Pharmaceuticals, Inc.