Positive new data for Roche’s Evrysdi in largest trial ever
undertaken in patients with previously-treated spinal muscular
atrophy (SMA)
- New two-year Evrysdi
data show improvement or maintenance of motor
function in people with SMA, a progressive neuromuscular disease
that can be fatal
- The JEWELFISH study
enrolled the broadest and most diverse patient population ever
studied in an SMA trial
- Longer-term safety
data consistent with that previously seen in
earlier trials and low study drop-out rate
- Evrysdi has proven efficacy
in babies, children and adults, with more than 7,000 patients
treated to date worldwide
Basel, 12 October 2022- Roche (SIX: RO, ROG; OTCQX: RHHBY) today
announced new two-year data from the JEWELFISH study evaluating
Evrysdi® (risdiplam) in people with Type 1, 2 or 3 SMA aged 6
months to 60 years at time of enrolment. Patients had been
previously treated with other approved or investigational
SMA-targeting therapies, including nusinersen (Spinraza(R)) or
onasemnogene abeparvovec (Zolgensma(R)). Data showed Evrysdi
improved or maintained motor function and led to rapid increases in
SMN protein levels which were sustained after 2-years of treatment.
These data will be presented at the 27th World Muscle Society (WMS)
congress, 11-15 October 2022.
“The consistent safety profile and exploratory efficacy we have
seen in the JEWELFISH study, the largest ever conducted in
previously treated patients, reinforces Evrysdi as a meaningful
treatment option across SMA populations,” said Dr. Claudia
Chiriboga, Professor of Neurology and Pediatrics, Department of
Neurology, Columbia University Medical Center, New York, USA. “The
findings add to our confidence when making treatment decisions for
previously-treated patients in need.”
The JEWELFISH study enrolled the broadest and most diverse
patient population ever studied in an SMA trial. Of the 174 people
enrolled, 36% (n=63) were adults, 63% (n=105) had a Hammersmith
Functional Motor Scale Expanded (HFMSE) score of less than 10 at
baseline, meaning their disease was very severe, and 83% (n=139)
had scoliosis. Forty-four percent (n=76) of those enrolled had
previously been treated with nusinersen (Spinraza), 41% (n=71) with
olesoxime*, 8% (n=14) with onasemnogene abeparvovec (Zolgensma) and
7% (n=13) with RG7800*.
People with SMA are unable to produce enough survival motor
neuron (SMN) protein, leading to debilitating and potentially fatal
muscle weakness. The study showed Evrysdi led to a two-fold
increase in median SMN protein levels versus baseline after 4 weeks
of treatment in all patient groups, irrespective of previous
treatment. The SMN protein levels achieved after 4 weeks of
treatment were maintained for over two years.
Observed through exploratory efficacy endpoints, the study also
suggests maintenance of motor function was sustained at two-years
of treatment as measured by change from baseline in Motor Function
Measure 32 (MFM-32), Revised Upper Limb Module (RULM) and HFMSE
total scores compared to the natural history of SMA in untreated
patients. A recent survey conducted by patient advocacy group SMA
Europe, more than 96% of people with SMA viewed disease
stabilization as progress in terms of their expectations of
treatment.
“These important data demonstrate the safety and efficacy of
Evrysdi in a broad, real-world population of people previously
treated with an SMA-targeting therapy, ” said Levi Garraway, M.D,
Ph.D, Roche’s Chief Medical Officer and Head of Global Products.
“Those enrolled in JEWELFISH had very severe disease, with over 80%
having scoliosis, so maintaining motor function–especially for a
progressive disease–can be potentially life-changing.”
The overall adverse event (AE) and serious adverse event (SAE)
profiles observed with Evrysdi treatment in JEWELFISH were
reflective of underlying disease. The rate of AEs decreased by more
than 50% between the first and second 6-month period, and then
remained stable thereafter. The rate of SAEs, including pneumonia,
decreased throughout the 24-month period, with a total reduction of
more than 50% by the second year. The most common AEs (reported in
≥ 12% of all patients:n=173) were pyrexia (24%), upper respiratory
tract infection (21%), headache (18%), nasopharyngitis (16%),
diarrhoea (14%), nausea (13%) and cough (12%). The most common SAEs
(reported in less than >2% of all patients) were pneumonia (3%)
respiratory failure (2%), respiratory distress (2%), lower
respiratory tract infection (2%) and upper respiratory tract
infection (2%). The most common AEs/SAEs were consistent with those
observed in treatment-naïve patients in our other three trials. Low
rates of discontinuation from the study were observed, with a 5%
rate per year over the 24-month period.
Roche leads the clinical development of Evrysdi as part of a
collaboration with the SMA Foundation and PTC Therapeutics.
About Evrysdi® (risdiplam)Evrysdi is a survival
motor neuron 2 (SMN2) splicing modifier designed to treat SMA
caused by mutations in chromosome 5q that lead to SMN protein
deficiency. Evrysdi is administered daily at home in liquid form by
mouth or by feeding tube.
Evrysdi is designed to treat SMA by increasing and sustaining
the production of the SMN protein in the central nervous system
(CNS) and peripheral tissues. SMN protein is found throughout the
body and is critical for maintaining healthy motor neurons and
movement.
Evrysdi was granted PRIME designation by the European Medicines
Agency (EMA) in 2018 and Orphan Drug Designation by the U.S. Food
and Drug Administration in 2017. In 2021 Evrysdi was awarded Drug
Discovery of the Year by the British Pharmacological Society as
well as the Society for Medicines Research award for Drug
Discovery. Evrysdi is currently approved in 91 countries and the
dossier is under review in a further 18 countries.
Evrysdi is currently being evaluated in five multicentre trials
in people with SMA:
- FIREFISH (NCT02913482) – an
open-label, two-part pivotal clinical trial in infants with Type 1
SMA. Part 1 was a dose-escalation study in 21 infants with the
primary objective of assessing the safety profile of risdiplam in
infants and determining the dose for Part 2. Part 2 is a pivotal,
single-arm study of risdiplam in 41 infants with Type 1 SMA treated
for 2 years, followed by an open-label extension. Enrolment for
Part 2 was completed in November 2018. The primary objective of
Part 2 was to assess efficacy as measured by the proportion of
infants sitting without support after 12 months of treatment, as
assessed by the Gross Motor Scale of the Bayley Scales of Infant
and Toddler Development – Third Edition (BSID-III) (defined as
sitting without support for 5 seconds). The study met its primary
endpoint.
- SUNFISH (NCT02908685) – SUNFISH is
a two-part, double-blind, placebo controlled pivotal study in
people aged 2-25 years with Types 2 or 3 SMA. Part 1 (n=51)
determined the dose for the confirmatory Part 2. Part 2 (n=180)
evaluated motor function using the total score of Motor Function
Measure 32 (MFM-32) at 12 months. MFM-32 is a validated scale used
to evaluate fine and gross motor function in people with
neurological disorders, including SMA. The study met its primary
endpoint.
- JEWELFISH (NCT03032172) – an
open-label exploratory trial designed to assess the safety,
tolerability, pharmacokinetics and pharmacodynamics in people with
SMA aged 6 months to 60 years who received other investigational or
approved SMA therapies for at least 90 days prior to receiving
Evrysdi. The study has completed recruitment (n=174).
- RAINBOWFISH (NCT03779334) – an
open-label, single-arm, multicentre study, investigating the
efficacy, safety, pharmacokinetics, and pharmacodynamics of
risdiplam in babies (~n=25), from birth to six weeks of age (at
first dose) with genetically diagnosed SMA who are not yet
presenting with symptoms. The study is ongoing.
- MANATEE (NCT05115110) – a global
phase 2/3 clinical study to evaluate the safety and efficacy of
GYM329 (RO7204239), an anti-myostatin molecule targeting muscle
growth, in combination with Evrysdi for the treatment of SMA in
patients 2-10 years of age. The FDA Office of Orphan Products
Development granted GYM329 Orphan Drug Designation for the
treatment of patients with SMA in December 2021. The study is
currently recruiting.
About SMASMA is a severe, progressive
neuromuscular disease that can be fatal. It affects approximately
one in 10,000 babies and is the leading genetic cause of infant
mortality. SMA is caused by a mutation of the survival motor neuron
1 (SMN1) gene, which leads to a deficiency of SMN protein. This
protein is found throughout the body and is essential to the
function of nerves that control muscles and movement. Without it,
nerve cells cannot function correctly, leading to muscle weakness
over time. Depending on the type of SMA, an individual’s physical
strength and their ability to walk, eat or breathe can be
significantly diminished or lost.
About Roche in NeuroscienceNeuroscience is a
major focus of research and development at Roche. Our goal is to
pursue groundbreaking science to develop new treatments that help
improve the lives of people with chronic and potentially
devastating diseases.
Roche is investigating more than a dozen medicines for
neurological disorders, including multiple sclerosis, spinal
muscular atrophy, neuromyelitis optica spectrum disorder,
Alzheimer’s disease, Huntington’s disease, Parkinson’s disease and
Duchenne muscular dystrophy. Together with our partners, we are
committed to pushing the boundaries of scientific understanding to
solve some of the most difficult challenges in neuroscience
today.
About Roche Founded in 1896 in Basel,
Switzerland, as one of the first industrial manufacturers of
branded medicines, Roche has grown into the world’s largest
biotechnology company and the global leader in in-vitro
diagnostics. The company pursues scientific excellence to discover
and develop medicines and diagnostics for improving and saving the
lives of people around the world. We are a pioneer in personalised
healthcare and want to further transform how healthcare is
delivered to have an even greater impact. To provide the best care
for each person we partner with many stakeholders and combine our
strengths in Diagnostics and Pharma with data insights from the
clinical practice.
In recognizing our endeavor to pursue a long-term perspective in
all we do, Roche has been named one of the most sustainable
companies in the pharmaceuticals industry by the Dow Jones
Sustainability Indices for the thirteenth consecutive year. This
distinction also reflects our efforts to improve access to
healthcare together with local partners in every country we
work.
Genentech, in the United States, is a wholly owned member of the
Roche Group. Roche is the majority shareholder in Chugai
Pharmaceutical, Japan.
For more information, please visit www.roche.com.
All trademarks used or mentioned in this release are protected
by law.Spinraza is a registered trademark of Biogen MA Inc.
Zolgensma is a registered trademark of Novartis AG.
*RG7800 and olesoxime are no longer in development as
investigational treatments for patients with SMA.
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