[Ad hoc announcement pursuant to Art. 53 LR] Roche announces EMBARK
trial in Duchenne muscular dystrophy (DMD) did not reach primary
endpoint, but shows positive efficacy outcomes on all timed
functional key endpoints
- Elevidys-treated boys aged
4-7 years with Duchenne showed an increase on the North Star
Ambulatory Assessment (NSAA), a measure of motor function, compared
to placebo at 52 weeks but the primary endpoint was not
met
- For all key pre-specified
secondary functional endpoints, time to rise and 10-metre walk test
across age groups, clinically meaningful and statistically
significant treatment benefits were observed
- No new safety signals
observed, reinforcing the favourable and manageable safety profile
observed with Elevidys to date
- Further evaluation of data
is ongoing and Roche will discuss the path forward with health
authorities
Basel, 30 October 2023 - Roche (SIX: RO, ROG; OTCQX: RHHBY)
announced today topline results from the global, randomised,
double-blind Phase 3 EMBARK study of Elevidys™ (delandistrogene
moxeparvovec) in ambulatory boys (those who can walk) with Duchenne
muscular dystrophy aged 4-7 years. In the study, Elevidys-treated
patients improved 2.6 points on their NSAA total score 52 weeks
after treatment, compared to 1.9 points in placebo-treated patients
(0.65; n=125; P=0.24).
In all pre-specified, timed functional key secondary endpoints,
time to rise from floor and 10 metre walk test, clinically
meaningful and statistically significant improvements were
observed. Both endpoints are prognostic factors for disease
progression and loss of ability to walk. Additionally, a clinically
meaningful and statistically significant improvement was also
observed for the pre-specified secondary endpoint stride velocity
95th centile. This novel digital endpoint, qualified by the
European Medicines Agency (EMA), measures speed of walking via a
wearable device (Syde®). The time to ascend 4-steps secondary
endpoint also demonstrated consistent treatment benefit in favour
of Elevidys.
All data are being further analysed and will be discussed with
health authorities to determine the path forward. Detailed results
from the EMBARK study will be shared at an upcoming scientific
congress and a medical journal publication will be pursued.
“High unmet need remains in Duchenne and we are encouraged by
the consistent and meaningful results seen in all key secondary
functional endpoints for Elevidys, an innovative gene therapy,”
said Levi Garraway, M.D., Ph.D., Chief Medical Officer and Head of
Global Product Development, Roche. “We will work to further analyse
the EMBARK results and consult with health authorities as quickly
as possible. We sincerely thank all the boys, their families and
the wider Duchenne community involved in this important research
effort.”
All key pre-specified functional secondary endpoints
demonstrated robust evidence for a clinically meaningful treatment
benefit that was consistent across age groups in Elevidys-treated
patients compared to placebo at week 52. These include:
Functional endpoints:
Time to rise (TTR) |
Change vs Placebo LSM Difference (seconds) |
Overall (n=125) |
-0.64 (p=0.0025) |
Ages 4-5 (n=59) |
-0.50 |
Ages 6-7 (n=66) |
-0.78 |
10-metre walk test |
Change vs Placebo LSM Difference (seconds) |
Overall (n=125) |
-0.42 (p=0.0048) |
Ages 4-5 (n=59) |
-0.33 |
Ages 6-7 (n=66) |
-0.52 |
LSM = least squares mean
As part of a collaboration agreement Roche is working with
Sarepta Therapeutics to transform the future for the Duchenne
community, enabling those living with the disease to maintain and
protect their muscle function, keeping them stronger for longer.
Sarepta is responsible for managing regulatory approval and the
commercialisation of Elevidys in the US. Roche is responsible for
regulatory approvals and bringing Elevidys to patients across the
rest of the world. Sarepta is responsible for the manufacturing of
Elevidys and together, the companies are working on a comprehensive
joint clinical development plan to maximise the chances of broad
approval and access.
Elevidys clinical development programme
- Study 101 evaluating the safety of Elevidys in four ambulatory
participants aged between 4-<8 years old with Duchenne.
Four-year data show a durable response and consistent safety
profile.
- Study 102, a Phase 2 clinical trial evaluating the safety and
efficacy of Elevidys in patients with Duchenne aged 4-<8
years.
- Study 103 (ENDEAVOR), a two-part, open-label, Phase 1b study
assessing the Elevidys-dystrophin expression and safety of Elevidys
in five cohorts of boys with Duchenne representing different stages
of disease progression. This study is ongoing.
- Study 301 (EMBARK), a Phase 3 global, randomised,
double-blinded and placebo-controlled study of Elevidys in
ambulatory Duchenne patients aged 4-<8 years old.
- The ENVOL trial (Study 302) a Phase 2 study in children with
Duchenne. The study aims to enrol 21 participants who are under 4
years of age, including newborns. Not yet started.
- The ENVISION trial (Study 303), a Phase 3 study in older
ambulatory/non-ambulatory patients which is now recruiting.
- The EXPEDITION long-term (5 year) follow up study (Study 305)
of participants who have received Elevidys in a previous clinical
study, which is not yet recruiting.
About EMBARKEMBARK is a multinational, Phase 3,
randomised, double-blind, two-part crossover, placebo-controlled
study assessing the safety and efficacy of Elevidys in ambulatory
boys with a confirmed mutation in the DMD gene, aged between 4 and
7 years. Eligible participants received a single dose of Elevidys
during either Part 1 or Part 2 of the study. The study is
ongoing.
Participants (n=125) received 1.33x1014 vg/kg of delandistrogene
moxeparvovec or placebo. In Part 1, participants were randomised
according to age (4-5 or 6-7) or NSAA total score at screening (≤22
or >22) to receive either Elevidys or placebo, with a follow-up
period for 52 weeks. In Part 2, participants crossed over -
meaning, those who were previously treated with placebo in Part 1
received Elevidys and participants who were previously treated with
placebo received Elevidys, with a follow-up period for 52
weeks.
The primary endpoint of the trial was change from baseline in
NSAA total score at week 52. Secondary endpoints include:
- The quantity of delandistrogene moxeparvovec micro-dystrophin
expression at Week 12 as measured by western blot of biopsied
muscle tissue (Part 1)
- Change from baseline to Week 52 in Time to Rise from Floor
- Change from baseline to Week 52 in 10-metre Walk/Run
(10MWR)
- Change from baseline to Week 52 in stride velocity 95th centile
(as measured by Syde®, a wearable device)
- Change from baseline to Week 52 in 100-metre Walk/Run
- Change from baseline to Week 52 in time to ascend 4 steps
Data not yet available for the following endpoints:
- Change from baseline to Week 52 in Patient-Reported Outcomes
Measurement Information System® (PROMIS®) mobility score
- Change from baseline to Week 52 in PROMIS® upper extremity
score
- Number of skills gained or improved at Week 52 as measured by
the NSAA
About ELEVIDYS™Elevidys™ (delandistrogene
moxeparvovec, also known as SRP-9001) is the first approved
disease-modifying therapy for Duchenne and is designed to address
the underlying cause of Duchenne through targeted skeletal,
respiratory and cardiac muscle expression of shortened dystrophin
produced by Elevidys. Elevidys is a one-time treatment administered
through a single (one-time) intravenous dose. Elevidys is
contraindicated in patients with any deletion in exons 8 and/or 9
in the DMD gene.
Elevidys received accelerated approval in the US in June 2023,
in the United Arab Emirates in August 2023 and in Qatar in
September 2023 for the treatment of ambulant children aged 4
through 5 years with Duchenne, who have a confirmed mutation in the
DMD gene.
About Duchenne muscular dystrophyDuchenne is a
rare, genetic, muscle-wasting disease that progresses rapidly from
early childhood. Approximately 1 in 3,500 - 5,000 boys worldwide
are born with Duchenne, while Duchenne in girls is very rare.
Everyone who has Duchenne will lose the ability to walk, upper
limb, lung and cardiac function and mean life expectancy is 28
years. A diagnosis of DMD will require full-time caregiving which
is most often provided by parents, the majority of whom will find
it difficult to carry out usual work or household activities and
suffer from depression and physical pain.
Duchenne is caused by mutations of the DMD gene, which affects
the production of the muscle protein, dystrophin. Dystrophin is a
critical component of a protein complex that strengthens muscle
fibers and protects them from injury during muscle contraction. Due
to a genetic mutation in the DMD gene, people with Duchenne do not
make functional dystrophin; their muscle cells are more sensitive
to injury and muscle tissue is progressively replaced with scar
tissue and fat.
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 neuromuscular diseases: Duchenne
muscular dystrophy, facioscapulohumeral muscular dystrophy,
myasthenia gravis and spinal muscular atrophy; neuro immune
diseases: multiple sclerosis and neuromyelitis optica spectrum
disorder; and neurodegenerative diseases: Alzheimer’s disease,
Huntington’s disease and Parkinson’s disease. 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 recognising our endeavour 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.
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