[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:
|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.
EMBARK 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
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 dystrophy
Duchenne 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 Neuroscience
Neuroscience 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.
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.
All trademarks used or mentioned in this release are protected by law.
Roche Global Media Relations
Phone: +41 61 688 8888 / e-mail: email@example.com
|Hans Trees, PhD|
Phone: +41 79 407 72 58
Phone: +41 79 771 05 25
Phone: +44 797 32 72 915
Phone: +41 79 461 86 83
Phone: +41 79 327 54 74
Phone: +49 172 6367262
Phone: +41 79 205 27 03
Phone: +41 79 935 81 48
Roche Investor Relations
|Dr. Bruno Eschli|
Phone: +41 61 68-75284
|Dr. Sabine Borngräber|
Phone: +41 61 68-88027
|Dr. Birgit Masjost|
Phone: +41 61 68-84814
|Dr. Gerard Tobin|
Phone: +41 61 68-72942
Investor Relations North America
Phone: +1 650 225 3217
AttachmentTo view this piece of content from www.globenewswire.com, please give your consent at the top of this page.
To view this piece of content from ml-eu.globenewswire.com, please give your consent at the top of this page.
About GlobeNewswire by notified
One Liberty Plaza - 165 Broadway
NY 10006 New York
GlobeNewswire by notified is one of the world's largest newswire distribution networks, specializing in the delivery of corporate press releases financial disclosures and multimedia content to the media, investment community, individual investors and the general public.
Subscribe to releases from GlobeNewswire by notified
Subscribe to all the latest releases from GlobeNewswire by notified by registering your e-mail address below. You can unsubscribe at any time.
Latest releases from GlobeNewswire by notified
Managers’ transactions9.12.2023 18:12:40 CET | Press release
December 9, 2023 Announcement no. 21 Managers’ transactions COPENHAGEN, DENMARK and BOSTON, MA, December 9, 2023, (GLOBE NEWSWIRE) – BioPorto A/S (BioPorto or the Company) (CPH:BIOPOR), has received notice pursuant to article 19 of Regulation (EU) no. 596/2014 of the below transaction related to shares in BioPorto made by persons discharging managerial responsibilities in BioPorto and/or persons closely related with them. 1. Details of the person discharging managerial responsibilities/person closely associated a) Name Singer Asefzadeh Family Holding Trust 2. Reason for the notification a) Position/status Closely associated person to Michael S. Singer, member of the Board of Directors of BioPorto A/S b) Initial notification/amendment Initial notification 3. Details of the issuer a) Name BioPorto A/S b) LEI 5299004SWFL5JAN4W830 4. Details of the transaction(s) a) Description of the financial instrument type of instrument and Identification code Shares, ISIN code DK0011048619 b) Nature o
New Pivotal Data for Bispecific Antibody Epcoritamab (DuoBody® CD3xCD20) Demonstrates High Overall and Complete Responses in Patients with Hard-To-Treat Relapsed/Refractory Follicular Lymphoma (FL)9.12.2023 18:00:00 CET | Press release
Media Release COPENHAGEN, Denmark; December 9, 2023 Data from the pivotal phase 1/2 EPCORE™ NHL-1 study showed 82 percent overall response rate (ORR), 63 percent complete response (CR) and 67 percent minimal residual disease (MRD) negativity in patients with relapsed/refractory (R/R) follicular lymphoma (FL) treated with subcutaneous epcoritamab Results presented at the 65th American Society of Hematology (ASH) Annual Meeting and Exposition include data from an optimized step-up dosing schedule for FL patients showing meaningful reduction in risk and severity of cytokine release syndrome (CRS) Follicular lymphoma is the second most common form of non-Hodgkin’s lymphoma, is considered incurable and can be difficult to treat in the R/R setting Genmab A/S (Nasdaq: GMAB) and AbbVie (NYSE: ABBV) todayannounced new data from the ongoing phase 1/2 EPCORE™ NHL-1 clinical trial investigating epcoritamab (DuoBody® CD3xCD20), a T-cell engaging bispecific antibody administered subcutaneously, demo
Galapagos presents new encouraging data at ASH 2023 from ongoing CD19 CAR-T studies with GLPG5201 and GLPG51019.12.2023 18:00:00 CET | Press release
Additional safety and efficacy data further support potential of innovative, decentralized approach to CAR-T manufacturing and transformational impact on patients with severe hematologic cancersTwo poster presentations include recent data updates and additional data not included in the ASH abstracts Galapagos to host a Key Opinion Leader (KOL) event with live webcast on Sunday, 10 December 2023 at 11:00 am PT/20:00 CET Mechelen, Belgium; 9 December 2023, 18:00 CET; Galapagos NV (Euronext & NASDAQ: GLPG) topresent additional encouraging clinical data from the ongoing Phase 1/2 CD19 CAR-T studies, EUPLAGIA-1 with GLPG5201 and ATALANTA-1 with GLPG5101, in patients with relapsed/refractory chronic lymphocytic leukemia (rrCLL), with or without Richter transformation, and non-Hodgkin lymphoma (rrNHL), during two poster sessions at the 65th American Society of Hematology (ASH) Annual Meeting taking place in San Diego, from 9-12 December. “We are very pleased to share promising new data from o
New data reinforce the benefit of early preventative treatment with Roche’s Hemlibra for babies with severe haemophilia A9.12.2023 17:30:00 CET | Press release
Phase III HAVEN 7 primary data presented at ASH 2023 provide additional confidence in the favourable efficacy and safety profile of subcutaneous Hemlibra given soon after birth 1At nearly two years median follow-up in the descriptive, single-arm study, no babies experienced spontaneous bleeds requiring treatment, and all treated bleeds were as a result of trauma 1Safety results were consistent with previous studies of Hemlibra, with no new safety signals observed 1The HAVEN 7 study was developed in collaboration with the haemophilia A community, to generate additional evidence for the prophylactic treatment of infants with haemophilia A Basel, 09 December 2023 - Roche (SIX: RO, ROG; OTCQX: RHHBY) announced today that the primary analysis of the Phase III HAVEN 7 study reinforced the efficacy and safety of Hemlibra® (emicizumab) in previously untreated or minimally treated infants with severe haemophilia A without factor VIII inhibitors. Results showed that Hemlibra achieved meaningful
Bulletin from Annual General Meeting in SkiStar AB9.12.2023 16:00:00 CET | Press release
At SkiStar AB (publ)’s annual general meeting, held in Sälen on 9 December 2023, the following decisions were made. A dividend of SEK 2.60 per share was adopted. Record day 12 December 2023.Lena Apler, Fredrik Paulsson, Gunilla Rudebjer, Anders Sundström, Anders Svensson and Vegard Søraunet were re-elected to the board and Carina Åkerström was elected as new board member.Anders Sundström was re-elected chairman of the board.Board fees, including committee fees, were raised to a total of SEK 2,890,000 (2022: SEK 2,810,000). The fees shall be distributed as follows: SEK 670,000 (650,000) to the chairman of the board and SEK 310,000 (300,000) each to the other non-executive directors. Audit committee members will receive total fees of SEK 240,000 (unchanged), distributed as follows: SEK 120,000 to the committee chairman and SEK 60,000 to each of the other two members. Remuneration committee members will receive total fees of SEK 120,000 (unchanged), distributed as follows: SEK 60,000 to t