
Spacecraft Sends Back Historic First-Ever Images Of Sun's South Pole
Launched in February 2020 to take the first-ever close-up images of the sun, the European Space Agency's Solar Orbiter spacecraft has sent back humanity's first clear images of the sun's south pole. 'Today we reveal humankind's first-ever views of the sun's pole,' said Professor Carole Mundell, ESA's Director of Science. 'These new unique views from our Solar Orbiter mission are the beginning of a new era of solar science.'
Caption: This collage shows Solar Orbiter's view of the Sun's south pole on 16–17 March 2025, from a ... More viewing angle of around 15° below the solar equator. This was the mission's first high-angle observation campaign, a few days before reaching its current maximum viewing angle of 17°.
The unique views of the sun were captured on March 16-17, when Solar Orbiter was orbiting the sun from 15 degrees below the solar equator, enabling it to peek at the sun's south pole for the first time.
The Solar Orbiter's three cameras provide images of the sun in visible light, ultraviolet light, and light emanating from different temperatures of charged gas above the sun's surface, revealing distinct layers of the sun's atmosphere.
One of the mission's first major discoveries is a surprisingly disordered magnetic field at the sun's south pole. Data from one of its cameras reveals that both magnetic polarities — north and south — are present in the region, an unusual state linked to the current solar maximum (the peak of the sun's 11-year solar cycle). That could help scientists figure out why the sun's magnetic field flips during solar maximum, and more accurately predict it.
The Spectral Imaging of the Coronal Environment (SPICE) instrument on the ESA-led Solar Orbiter ... More spacecraft got its first good look at the Sun's south pole in March 2025.
Solar Orbiter's images represent the first-ever views of the sun from a different angle. Until now, almost all images of the sun have been taken from the same point of view on Earth or in Earth's vicinity — the ecliptic plane. If you think about the solar system as a fried egg with the sun as the yolk, all planets orbit in the white. Solar Orbiter is the first to tilt out of this zone. By March 2025, the spacecraft had achieved a 17 degrees inclination to the ecliptic, enabling direct observations of the sun's south pole. 'We didn't know what exactly to expect from these first observations – the sun's poles are literally terra incognita,' said Sami Solanki at the Max Planck Institute for Solar System Research, Germany, who works on one of Solar Orbiter's instruments.
A camera on the Solar Orbiter, called EUI, images the sun in ultraviolet light, revealing the million-degree charged gas in the sun's outer atmosphere, the corona. In April, ESA published the widest high-resolution view of the sun so far, assembled from 200 images taken by Solar Orbiter on March 9, 2025. The ultraviolet light images showed the corona. Solar scientists must learn about the corona because it's the source of the solar wind — a stream of charged particles emanating from the sun and impacting Earth — and the space weather that causes geomagnetic storms and auroras.
In February 2025, Solar Orbiter became the first Sun-watching spacecraft ever to tilt its orbit out ... More of the ecliptic plane.
Over the next several years, the Solar Orbiter will observe the sun moving toward solar minimum — the trough of the solar cycle — studying how its magnetic structure is reorganized.
The full dataset from these initial observations from Solar Orbiter's newly inclined orbit isn't expected until October 2025, but in the four years following that, solar scientists anticipate unprecedented data. 'This is just the first step of Solar Orbiter's 'stairway to heaven.' In the coming years, the spacecraft will climb further out of the ecliptic plane for ever better views of the sun's polar regions,' said Daniel Müller, ESA's Solar Orbiter project scientist. 'These data will transform our understanding of the sun's magnetic field, the solar wind, and solar activity.'

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Investigational combination of first-in-class bispecific antibodies TALVEY®▼ (talquetamab) and TECVAYLI®▼ (teclistamab) shows deep and durable responses in heavily pretreated multiple myeloma patients with extramedullary disease
Results from the Phase 2 RedirecTT-1 study demonstrate deep responses with 78.9 percent overall response rate through dual targeting of GPRC5D and BCMA1 Data signal potential of novel, off-the-shelf approach in patients with extramedullary disease who face significant unmet needs1 BEERSE, BELGIUM, June 15, 2025 (GLOBE NEWSWIRE) -- Janssen-Cilag International NV, a Johnson & Johnson company, announced today new results from the Phase 2 RedirecTT-1 study evaluating the investigational combination of TALVEY®▼(talquetamab), the first European Commission (EC) approved GPRC5D-directed bispecific antibody, and TECVAYLI®▼(teclistamab), the first EC approved BCMA-directed bispecific antibody. The results show a high overall response rate (ORR) with durability in patients with triple-class exposed (TCE) relapsed/refractory multiple myeloma (RRMM) who have true extramedullary disease (EMD).1 EMD is defined as soft tissue/organ-associated plasmacytomas with no contact to bony structures as per International Myeloma Working Group (IMWG) criteria.2 RedirecTT-1 is the largest study dedicated to patients with EMD to date.1 These data were featured in a late-breaking oral presentation (Abstract #LB4001) at the 2025 European Hematology Association (EHA) Congress.1 EMD represents an aggressive form of multiple myeloma and occurs when myeloma cells spread and form tumours (plasmacytomas) elsewhere in the body, such as in soft tissues and organs.3 These patients often face limited treatment options and worse outcomes due to the complexity of the disease, including tumour heterogeneity, resulting in low ORRs and rapid relapses with current standard therapies.2,3 On average, TCE RRMM patients with EMD have an ORR of less than 40 percent and a median progression-free survival (PFS) of less than six months.4 'The investigational combination of talquetamab and teclistamab has demonstrated deep, durable responses in patients with relapsed or refractory multiple myeloma, and now shows great promise in those with extramedullary myeloma, where standard therapies often fall short,' said Yael Cohen, M.D., Head of Myeloma Unit, Tel-Aviv Sourasky Medical Center, Tel-Aviv, Israel.* 'Dual targeting of GPRC5D and BCMA may lead to a higher ORR and greater depth of response by mitigating target antigen-related escape. The RedirecTT-1 trial shows the power of this novel dual-targeting combination approach as a potential treatment option for patients with this disease.' The Phase 2 RedirecTT-1 study enrolled 90 patients with TCE RRMM with true EMD.1 Of these patients, 84.4 percent were triple-class refractory, 35.6 percent were penta-drug refractory, 20.0 percent had previously received BCMA CAR-T therapy, and 8.9 percent had previously received a bispecific antibody.1 The investigational combination of talquetamab and teclistamab led to a high ORR of 78.9 percent (95 percent confidence interval [CI]; 69.0–86.8), with more than half of patients (54.4 percent) achieving complete response or better.1 High responses were observed even in patients exposed to prior BCMA CAR-T or anti-FcRH5 bispecific antibodies (83.3 percent ORR; 58.6-96.4 and 75.0 percent ORR; 34.9-96.8, respectively).1 Among responders, 66.2 percent remained in response at the data cutoff, with a median follow-up of 13.4 months, signalling deep and durable responses.1 Treatment with the combination resulted in 61.0 percent of patients progression-free and alive at one year.1 Additionally, the combination led to durable responses, with 64.1 percent of patients maintaining response (median duration of response: 13.8 months) and 74.5 percent of patients alive at one year, while median overall survival was not yet reached.1 'Multiple myeloma remains a complex and heterogeneous disease, with extramedullary disease presenting a particularly aggressive and challenging to treat form,' said Ester in't Groen, EMEA Therapeutic Area Head Haematology at Johnson & Johnson Innovative Medicine. 'The RedirecTT-1 study reflects our strategy to harness novel mechanisms of action, such as the combination of these dual bispecific antibodies, to help redefine potential outcomes for subsets of patients who are currently faced with a poor prognosis and limited options.' The safety profile of the combination was consistent with previous reports of talquetamab and teclistamab as monotherapies, with no new safety signals identified.1 Patients were given the option to switch to once a month dosing potentially contributing to improved tolerability.1 Rates of discontinuation were low with the treatment combination of talquetamab and teclistamab due to adverse events (AEs).1 Four participants discontinued talquetamab only.1 Reports of cytokine release syndrome (CRS) and immune effector cell-associated neurotoxicity syndrome (ICANS) were mostly low grade.1 Of the ten patients who had Grade 5 AEs (11.1 percent), five were due to infections.1 There were five patient deaths due to infection and the rates of severe infection were similar to those observed with some BCMA bispecific antibody monotherapies.1 'Patients with extramedullary myeloma, especially those who have exhausted prior therapies, need more effective treatment options,' said Jordan Schecter, M.D., Vice President, Disease Area Leader, Multiple Myeloma, Johnson & Johnson Innovative Medicine. 'Our first-in-class bispecific antibodies talquetamab and teclistamab have transformed treatment for relapsed or refractory multiple myeloma. The RedirecTT-1 study underscores our commitment to advancing innovative therapies that attack the disease in different ways by building combinable and complementary regimens.' About Talquetamab Talquetamab received conditional marketing authorisation (CMA) from the European Commission (EC) in August 2023, as monotherapy for the treatment of adult patients with relapsed and refractory multiple myeloma (RRMM) who have received at least three prior therapies, including an immunomodulatory agent, a proteasome inhibitor, and an anti-CD38 antibody, and have demonstrated disease progression on the last therapy.5 The U.S. Food and Drug Administration (FDA) also granted talquetamab approval in August 2023, for the treatment of adult patients with RRMM who have received at least four prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent and an anti-CD38 antibody.6 Talquetamab is a bispecific T-cell engaging antibody that binds to CD3 on the surface of T-cells, and GPRC5D, a novel target which is highly expressed on the surface of multiple myeloma cells, with minimal to no expression detected on B-cells or B-cell precursors.5 For a full list of adverse events and information on dosage and administration, contraindications and other precautions when using talquetamab, please refer to the Summary of Product Characteristics. In line with European Medicine Agency (EMA) regulations for new medicines and those given conditional approval, talquetamab is subject to additional monitoring. About TeclistamabTeclistamab received EC approval in August 2022 for the treatment of patients with RRMM who have received at least three prior therapies, including an immunomodulatory agent, a proteasome inhibitor, and an anti-CD38 antibody, and have demonstrated disease progression on the last therapy.7 In August 2023, the EC approved a Type II variation application for teclistamab, providing the option for a reduced dosing frequency of 1.5mg/kg every two weeks in patients who have achieved a complete response (CR) or better for a minimum of six months.8 Teclistamab received approval from the U.S. FDA in October 2022 for the treatment of adult patients with RRMM who have received at least four prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent and an anti-CD38 antibody.9 Teclistamab is an off-the-shelf (or ready-to-use) bispecific antibody.9,10 Teclistamab, a subcutaneous injection, redirects T-cells through two cellular targets (BCMA and CD3) to activate the body's immune system to fight the cancer. Teclistamab is currently being evaluated in several combination studies.10,11,12,13,14 For a full list of adverse events and information on dosage and administration, contraindications and other precautions when using teclistamab, please refer to the Summary of Product Characteristics. In line with European Medicine Agency (EMA) regulations for new medicines and those given conditional approval, teclistamab is subject to additional monitoring. About Multiple MyelomaMultiple myeloma is currently an incurable blood cancer that affects a type of white blood cell called plasma cells, which are found in the bone marrow.15,16 In multiple myeloma, these malignant plasma cells continue to proliferate, accumulating in the body and crowding out normal blood cells, as well as often causing bone destruction and other serious complications.15,16 In the European Union, it is estimated that more than 35,000 people were diagnosed with multiple myeloma in 2022, and more than 22,700 patients died.17 Patients living with multiple myeloma experience relapses which become more frequent with each line of therapy18,19 while remissions become progressively shorter.18,19,20 Whilst some patients with multiple myeloma initially have no symptoms, others can have common signs and symptoms of the disease, which can include bone fracture or pain, low red blood cell counts, fatigue, high calcium levels, infections, or kidney damage.21 About Johnson & Johnson At Johnson & Johnson, we believe health is everything. Our strength in healthcare innovation empowers us to build a world where complex diseases are prevented, treated, and cured, where treatments are smarter and less invasive, and solutions are personal. Through our expertise in Innovative Medicine and MedTech, we are uniquely positioned to innovate across the full spectrum of healthcare solutions today to deliver the breakthroughs of tomorrow and profoundly impact health for humanity. Learn more at Follow us at Janssen-Cilag International NV, Janssen Pharmaceutica NV, Janssen-Cilag Limited, Janssen Biotech, Inc., and Janssen Research & Development, LLC are Johnson & Johnson companies. This press release contains 'forward-looking statements' as defined in the Private Securities Litigation Reform Act of 1995 regarding product development and the potential benefits and treatment impact of teclistamab and talquetamab. The reader is cautioned not to rely on these forward-looking statements. These statements are based on current expectations of future events. If underlying assumptions prove inaccurate or known or unknown risks or uncertainties materialise, actual results could vary materially from the expectations and projections of Johnson & Johnson. Risks and uncertainties include, but are not limited to: challenges and uncertainties inherent in product research and development, including the uncertainty of clinical success and of obtaining regulatory approvals; uncertainty of commercial success; manufacturing difficulties and delays; competition, including technological advances, new products and patents attained by competitors; challenges to patents; product efficacy or safety concerns resulting in product recalls or regulatory action; changes in behaviour and spending patterns of purchasers of health care products and services; changes to applicable laws and regulations, including global health care reforms; and trends toward health care cost containment. A further list and descriptions of these risks, uncertainties and other factors can be found in Johnson & Johnson's most recent Annual Report on Form 10-K, including in the sections captioned 'Cautionary Note Regarding Forward-Looking Statements' and 'Item 1A. Risk Factors,' and in Johnson & Johnson's subsequent Quarterly Reports on Form 10-Q and other filings with the Securities and Exchange Commission. Copies of these filings are available online at or on request from Johnson & Johnson. Johnson & Johnson does not undertake to update any forward-looking statement as a result of new information or future events or developments. * Yael Cohen, M.D., Head of Myeloma Unit, Tel-Aviv Sourasky Medical Center, Tel-Aviv, Israel., has provided consulting, advisory and speaking services to Janssen-Cilag International NV; she has not been paid for any media work. ### 1 Kumar S, et al. Phase 2 study of Talquetamab + Teclistamab in Patients with Relapsed/Refractory Multiple Myeloma with Extramedullary Disease: presentation at 2025 European Hematology Association (EHA) Congress; June 12-15, 2025. 2 Ho M, et Multiple Myeloma: Challenges and Opportunities. Curr. Oncol, 2025; 32: 182. 3 Blade J, et al. Extramedullary Disease in Multiple Myeloma: a Systematic Literature Review. Blood Cancer J, 2022; 12(3):45.4 Moreau P, et al. Outcomes of Patients With Extramedullary Disease in Triple-Class Exposed Relapsed/Refractory Multiple Myeloma From the Pooled LocoMMotion and MoMMent Studies. Clinical Lymphoma, Myeloma and Leukemia, 2025; 25: S2152-2650.5 European Medicines Agency. TALVEY Summary of Product Characteristics. Available at: Last accessed: June 2025.6 FDA. FDA Grants Accelerated Approval to Talquetamab-tgvs for Relapsed or Refractory Multiple Myeloma. Available at: Last accessed: June 2025.7 Janssen Marks First Approval Worldwide. Available at: Last accessed: June 2025.8 European Commission Approves Reduced Dosing Frequency for Janssen's Bispecific Antibody TECVAYLI®▼ (teclistamab). Available at: Last accessed: June 2025.9 U.S. FDA Approves TECVAYLI™ (teclistamab-cqyv), the First Bispecific T-cell Engager Antibody for the Treatment of Patients with Relapsed or Refractory Multiple Myeloma. Available at: Last accessed: June 2025. 10 European Medicines Agency. TECVAYLI Summary of Product Characteristics. Available at: Last accessed: June 2025.11 A Study of Teclistamab With Other Anticancer Therapies in Participants With Multiple Myeloma (MajesTEC-2). Available at: Last accessed: June 2025.12 A Study of the Combination of Talquetamab and Teclistamab in Participants With Relapsed or Refractory Multiple Myeloma. Available at: Last accessed: June 2025.13 A Study of Subcutaneous Daratumumab Regimens in Combination With Bispecific T Cell Redirection Antibodies for the Treatment of Participants With Multiple Myeloma. Available at: Last accessed: June 2025.14 A Study of Teclistamab in Combination With Daratumumab Subcutaneously (SC) (TecDara) Versus Daratumumab SC, Pomalidomide, and Dexamethasone (DPd) or Daratumumab SC, Bortezomib, and Dexamethasone (DVd) in Participants With Relapsed or Refractory Multiple Myeloma (MajesTEC-3). Available at: Last accessed: June 2025.15 Abdi J, et al. Drug Resistance in Multiple Myeloma: Latest Findings on Molecular Mechanisms. Oncotarget 2013;4(12):2186-2207.16 American Society of Clinical Oncology. Multiple Myeloma: Introduction. Available at: Last accessed: June 2025.17 ECIS - European Cancer Information System. Estimates of Cancer Incidence and Mortality in 2022, by Country. Multiple Myeloma. Available at: Last accessed: June 2025.18 Bhatt P, Kloock C, Comenzo R. Relapsed/Refractory Multiple Myeloma: A Review of Available Therapies and Clinical Scenarios Encountered in Myeloma Relapse. Curr Oncol. 2023;30(2):2322-2347.19 Hernández-Rivas JÁ, et al. The Changing Landscape of Relapsed and/or Refractory Multiple Myeloma (MM): Fundamentals and Controversies. Biomark Res. 2022;10(1):1-23.20 Gavriatopoulou M, et al. Metabolic Disorders in Multiple Myeloma. Int J Mol Sci. 2021;22(21):11430.21 American Cancer Society. Multiple Myeloma: Early Detection, Diagnosis and Staging. Available at: Last accessed: June 2025. CP-526056 June 2025 CONTACT: Media contact: Jenni Mildon jmildon@ +44 7920 418 552 Investor contact: Lauren Johnson investor-relations@
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[Ad hoc announcement pursuant to Art. 53 LR] Roche provides safety update on Elevidys™ gene therapy for Duchenne muscular dystrophy in non-ambulatory patients
After a thorough clinical review, the benefit-risk for the use of Elevidys in non-ambulatory patients with Duchenne has been re-assessed, following two cases of fatal acute liver failure Effective immediately, dosing of non-ambulatory patients, irrespective of age, is paused in the clinical setting; dosing of non-ambulatory patients is discontinued in the commercial setting Roche is working closely with relevant health authorities, investigators and prescribing physicians to ensure they are informed and patient care is being appropriately modified The benefit-risk of Elevidys treatment in ambulatory Duchenne patients remains positive and treatment guidance is unchanged Basel, 15 June 2025 - Roche (SIX: RO, ROG; OTCQX: RHHBY) announced today new dosing restrictions, effective immediately, for ELEVIDYS™ (delandistrogene moxeparvovec), for non-ambulatory Duchenne muscular dystrophy (DMD) patients, irrespective of age, in both clinical and commercial settings. In the commercial setting, non-ambulatory patients should no longer receive Elevidys. In the clinical trial setting, enrolment and dosing of non-ambulatory patients will be immediately paused until additional risk mitigation measures (e.g. immune modulatory treatment) are implemented in the study protocol. Health authorities, investigators and physicians are being informed so that patient care can be quickly adjusted. This decision follows careful assessment of two cases in non-ambulatory patients of fatal acute liver failure (ALF), an identified risk of Elevidys and other AAV-mediated gene therapies, which led to a reassessment of the benefit-risk profile as unfavourable for patients with DMD who are non-ambulatory. The new dosing restrictions do not impact the treatment of ambulatory DMD patients of any age, and the benefit-risk ratio remains positive in the ambulatory patient population. "We are deeply saddened by the loss of these two young men and are urgently working to mitigate any risks related to the use of Elevidys,' said Levi Garraway, M.D., Ph.D., Chief Medical Officer and Head of Global Product Development, Roche. "Patient safety is always our highest priority. Therefore, we have recommended halting treatment with Elevidys in non-ambulatory patients with immediate effect.' DMD is a rare, genetic, muscle-wasting disease that progresses rapidly from early childhood. Duchenne primarily affects males, with 1 in 5,000 boys born worldwide having Duchenne. Everyone with Duchenne will eventually lose the ability to walk, along with upper limb, lung and cardiac function. The two fatal ALF cases occurred in non-ambulatory patients, out of a total of approximately 140 non-ambulatory patients treated with Elevidys globally to date. Following the first case of fatal ALF, European regulators requested that Roche and Sarepta put temporary clinical holds on Elevidys studies 104 (NCT06241950), 302 (ENVOL, NCT06128564) and 303 (ENVISION Study 303, NCT05310071). The temporary clinical holds are still in effect. Outside of Europe, dosing will be paused, effective immediately, for the ENVISION trial. The dosing restrictions will also go into effect for future dosing of commercial non-ambulatory patients. Elevidys has been approved by regulatory authorities in eight Roche territories for the treatment of DMD including Bahrain, Brazil, Israel, Japan, Kuwait, Oman, Qatar, and the UAE. In 2019, Roche entered into a global collaboration agreement with Sarepta Therapeutics, Inc. to commercialise Elevidys in territories outside the U.S. Roche and Sarepta jointly manage the clinical studies for Elevidys. Roche is the sponsor of the ENVOL study; Sarepta is the sponsor for all other studies. Overview of the Elevidys clinical development programme Studies in non-ambulatory people with DMD Ongoing ENVISION (Study 303, NCT05881408), a global Phase III study investigating the safety and efficacy of Elevidys in participants who are ambulatory (aged 8 to <18 years old) and non-ambulatory (no age limitation). This study is already on temporary clinical hold in Europe. Outside of Europe, recruitment will be paused. ENDEAVOR (Study 103, NCT04626674), a two-part, open-label, Phase Ib study assessing Elevidys micro-dystrophin protein expression and safety of Elevidys in seven cohorts of boys with Duchenne, across different ages, mutations and stages of disease progression. No longer recruiting; long term follow up ongoing. Studies in ambulatory people with DMD Study 101 (NCT03375164), a Phase I/II study evaluating the safety of Elevidys in four ambulatory participants aged 4 to <8 years old with Duchenne. The study is complete. Study 102 (NCT03769116), a Phase II clinical trial evaluating the safety and efficacy of Elevidys in patients with Duchenne aged 4 to <8 years. The study is complete. Study 104 (NCT06241950), a Phase I open-label, systemic gene delivery study to evaluate the safety, tolerability and expression of Elevidys in association with imlifidase in individuals aged 4 to 9 years with pre-existing antibodies to recombinant adeno-associated virus serotype, rAAVrh74. The study is on temporary clinical hold in Europe. HORIZON (Study 105, NCT06597656), a Phase I open-label, systemic gene delivery study to evaluate the safety, tolerability and expression of Elevidys following plasmapheresis in individuals aged 4 to 8 years with pre-existing antibodies to adeno-associated virus serotype, AAVrh74. The study is recruiting ambulatory patients. EMBARK (Study 301, NCT05096221), a multinational, Phase III, randomised, double-blind, placebo-controlled study assessing the safety and efficacy of Elevidys in ambulatory boys aged 4 to 7 years. The study duration is two years. The study is complete. ENVOL (Study 302, NCT06128564), a Phase II study evaluating the safety of Elevidys and expression of Elevidys micro-dystrophin protein in young children, including babies and newborns. The study is on temporary clinical hold in Europe and the UK. EXPEDITION (Study 305, NCT05967351), a Phase III long-term five-year follow-up study evaluating the safety and efficacy of Elevidys in those who have received Elevidys in a previous clinical study. EXPEDITION is enrolling by invitation. About Elevidys™ (delandistrogene moxeparvovec) Elevidys is a one-time treatment administered through a single intravenous dose and the first and only approved gene therapy for Duchenne. It is designed to target the underlying cause of Duchenne by delivering new instructions to cells to produce Elevidys-dystrophin in skeletal, respiratory and cardiac muscles. Elevidys aims to slow the progression of Duchenne by delaying the need for a wheelchair, protecting the heart from damage and a person's ability to breathe without a respirator for as long as possible. Elevidys uses adeno-associated virus (AAV) vector technology and consists of three parts: a transgene, promoter and vector. Its unique construct optimises delivery to all muscle types, including those of interest for Duchenne treatment. A robust clinical trial programme to understand its potential in a broad range of people with Duchenne, of all ages, ambulatory status and a wide range of DMD gene mutations is ongoing. To date, more than 900 individuals with Duchenne have been treated with Elevidys through Roche's clinical development program and in real-world settings. Elevidys has already been approved for the treatment of DMD by 10 regulatory authorities around the world, including the US and Japan. Elevidys is being developed by Roche in collaboration with Sarepta Therapeutics. About Duchenne muscular dystrophy Duchenne muscular dystrophy (DMD) is a rare, genetic, muscle-wasting disease that progresses rapidly from early childhood. Duchenne primarily affects males, with 1 in 5,000 boys born worldwide having Duchenne. Everyone with Duchenne will eventually lose the ability to walk, along with upper limb, lung and cardiac function. Average life expectancy is only 28 years. The physical, emotional and financial impact of Duchenne on those affected, their families and caregivers, is profound. Duchenne is an X-linked, rare neuromuscular disease caused by pathogenic variants (mutations) in the DMD gene that disrupt the production of functional dystrophin protein, leading to progressive and irreversible muscle weakness, diminished quality of life and premature death. Dystrophin strengthens and protects muscles and without it, normal activity causes excessive damage to muscle cells as they are more sensitive to injury. Over time, muscle tissue is replaced with scar tissue and fat, causing muscles to weaken. Although Duchenne progresses differently in each individual, its devastating trajectory is well established. Those with Duchenne will eventually lose the ability to use and move their limbs, to breathe on their own and are susceptible to respiratory infections. Muscle damage to the heart causes cardiomyopathy, including rhythm abnormalities and heart failure. Early diagnosis is important for timely intervention to prolong muscle function and preserve quality of life. There is a critical need for disease-modifying treatments that address the underlying cause of DMD before irreversible muscle loss occurs. 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 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. For over 125 years, sustainability has been an integral part of Roche's business. As a science-driven company, our greatest contribution to society is developing innovative medicines and diagnostics that help people live healthier lives. Roche is committed to the Science Based Targets initiative and the Sustainable Markets Initiative to achieve net zero by 2045. 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 All trademarks used or mentioned in this release are protected by law. Roche Global Media Relations Phone: +41 61 688 8888 / e-mail: Hans Trees, PhD Phone: +41 79 407 72 58 Sileia Urech Phone: +41 79 935 81 48 Nathalie Altermatt Phone: +41 79 771 05 25 Lorena Corfas Phone: +41 79 568 24 95 Simon Goldsborough Phone: +44 797 32 72 915 Karsten Kleine Phone: +41 79 461 86 83 Nina Mählitz Phone: +41 79 327 54 74 Kirti Pandey Phone: +49 172 6367262 Yvette Petillon Phone: +41 79 961 92 50 Dr Rebekka Schnell Phone: +41 79 205 27 03 Roche Investor Relations Dr Bruno Eschli Phone: +41 61 68-75284 e-mail: Dr Sabine Borngräber Phone: +41 61 68-88027 e-mail: Dr Birgit Masjost Phone: +41 61 68-84814 e-mail: Investor Relations North America Loren Kalm Phone: +1 650 225 3217 e-mail: Attachment Ad Hoc Media Investor Release Update on Elevidys EnglishError in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
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[Ad hoc announcement pursuant to Art. 53 LR] Roche provides safety update on Elevidys™ gene therapy for Duchenne muscular dystrophy in non-ambulatory patients
After a thorough clinical review, the benefit-risk for the use of Elevidys in non-ambulatory patients with Duchenne has been re-assessed, following two cases of fatal acute liver failure Effective immediately, dosing of non-ambulatory patients, irrespective of age, is paused in the clinical setting; dosing of non-ambulatory patients is discontinued in the commercial setting Roche is working closely with relevant health authorities, investigators and prescribing physicians to ensure they are informed and patient care is being appropriately modified The benefit-risk of Elevidys treatment in ambulatory Duchenne patients remains positive and treatment guidance is unchanged Basel, 15 June 2025 - Roche (SIX: RO, ROG; OTCQX: RHHBY) announced today new dosing restrictions, effective immediately, for ELEVIDYS™ (delandistrogene moxeparvovec), for non-ambulatory Duchenne muscular dystrophy (DMD) patients, irrespective of age, in both clinical and commercial settings. In the commercial setting, non-ambulatory patients should no longer receive Elevidys. In the clinical trial setting, enrolment and dosing of non-ambulatory patients will be immediately paused until additional risk mitigation measures (e.g. immune modulatory treatment) are implemented in the study protocol. Health authorities, investigators and physicians are being informed so that patient care can be quickly adjusted. This decision follows careful assessment of two cases in non-ambulatory patients of fatal acute liver failure (ALF), an identified risk of Elevidys and other AAV-mediated gene therapies, which led to a reassessment of the benefit-risk profile as unfavourable for patients with DMD who are non-ambulatory. The new dosing restrictions do not impact the treatment of ambulatory DMD patients of any age, and the benefit-risk ratio remains positive in the ambulatory patient population. "We are deeply saddened by the loss of these two young men and are urgently working to mitigate any risks related to the use of Elevidys,' said Levi Garraway, M.D., Ph.D., Chief Medical Officer and Head of Global Product Development, Roche. "Patient safety is always our highest priority. Therefore, we have recommended halting treatment with Elevidys in non-ambulatory patients with immediate effect.' DMD is a rare, genetic, muscle-wasting disease that progresses rapidly from early childhood. Duchenne primarily affects males, with 1 in 5,000 boys born worldwide having Duchenne. Everyone with Duchenne will eventually lose the ability to walk, along with upper limb, lung and cardiac function. The two fatal ALF cases occurred in non-ambulatory patients, out of a total of approximately 140 non-ambulatory patients treated with Elevidys globally to date. Following the first case of fatal ALF, European regulators requested that Roche and Sarepta put temporary clinical holds on Elevidys studies 104 (NCT06241950), 302 (ENVOL, NCT06128564) and 303 (ENVISION Study 303, NCT05310071). The temporary clinical holds are still in effect. Outside of Europe, dosing will be paused, effective immediately, for the ENVISION trial. The dosing restrictions will also go into effect for future dosing of commercial non-ambulatory patients. Elevidys has been approved by regulatory authorities in eight Roche territories for the treatment of DMD including Bahrain, Brazil, Israel, Japan, Kuwait, Oman, Qatar, and the UAE. In 2019, Roche entered into a global collaboration agreement with Sarepta Therapeutics, Inc. to commercialise Elevidys in territories outside the U.S. Roche and Sarepta jointly manage the clinical studies for Elevidys. Roche is the sponsor of the ENVOL study; Sarepta is the sponsor for all other studies. Overview of the Elevidys clinical development programme Studies in non-ambulatory people with DMD Ongoing ENVISION (Study 303, NCT05881408), a global Phase III study investigating the safety and efficacy of Elevidys in participants who are ambulatory (aged 8 to <18 years old) and non-ambulatory (no age limitation). This study is already on temporary clinical hold in Europe. Outside of Europe, recruitment will be paused. ENDEAVOR (Study 103, NCT04626674), a two-part, open-label, Phase Ib study assessing Elevidys micro-dystrophin protein expression and safety of Elevidys in seven cohorts of boys with Duchenne, across different ages, mutations and stages of disease progression. No longer recruiting; long term follow up ongoing. Studies in ambulatory people with DMD Study 101 (NCT03375164), a Phase I/II study evaluating the safety of Elevidys in four ambulatory participants aged 4 to <8 years old with Duchenne. The study is complete. Study 102 (NCT03769116), a Phase II clinical trial evaluating the safety and efficacy of Elevidys in patients with Duchenne aged 4 to <8 years. The study is complete. Study 104 (NCT06241950), a Phase I open-label, systemic gene delivery study to evaluate the safety, tolerability and expression of Elevidys in association with imlifidase in individuals aged 4 to 9 years with pre-existing antibodies to recombinant adeno-associated virus serotype, rAAVrh74. The study is on temporary clinical hold in Europe. HORIZON (Study 105, NCT06597656), a Phase I open-label, systemic gene delivery study to evaluate the safety, tolerability and expression of Elevidys following plasmapheresis in individuals aged 4 to 8 years with pre-existing antibodies to adeno-associated virus serotype, AAVrh74. The study is recruiting ambulatory patients. EMBARK (Study 301, NCT05096221), a multinational, Phase III, randomised, double-blind, placebo-controlled study assessing the safety and efficacy of Elevidys in ambulatory boys aged 4 to 7 years. The study duration is two years. The study is complete. ENVOL (Study 302, NCT06128564), a Phase II study evaluating the safety of Elevidys and expression of Elevidys micro-dystrophin protein in young children, including babies and newborns. The study is on temporary clinical hold in Europe and the UK. EXPEDITION (Study 305, NCT05967351), a Phase III long-term five-year follow-up study evaluating the safety and efficacy of Elevidys in those who have received Elevidys in a previous clinical study. EXPEDITION is enrolling by invitation. About Elevidys™ (delandistrogene moxeparvovec) Elevidys is a one-time treatment administered through a single intravenous dose and the first and only approved gene therapy for Duchenne. It is designed to target the underlying cause of Duchenne by delivering new instructions to cells to produce Elevidys-dystrophin in skeletal, respiratory and cardiac muscles. Elevidys aims to slow the progression of Duchenne by delaying the need for a wheelchair, protecting the heart from damage and a person's ability to breathe without a respirator for as long as possible. Elevidys uses adeno-associated virus (AAV) vector technology and consists of three parts: a transgene, promoter and vector. Its unique construct optimises delivery to all muscle types, including those of interest for Duchenne treatment. A robust clinical trial programme to understand its potential in a broad range of people with Duchenne, of all ages, ambulatory status and a wide range of DMD gene mutations is ongoing. To date, more than 900 individuals with Duchenne have been treated with Elevidys through Roche's clinical development program and in real-world settings. Elevidys has already been approved for the treatment of DMD by 10 regulatory authorities around the world, including the US and Japan. Elevidys is being developed by Roche in collaboration with Sarepta Therapeutics. About Duchenne muscular dystrophy Duchenne muscular dystrophy (DMD) is a rare, genetic, muscle-wasting disease that progresses rapidly from early childhood. Duchenne primarily affects males, with 1 in 5,000 boys born worldwide having Duchenne. Everyone with Duchenne will eventually lose the ability to walk, along with upper limb, lung and cardiac function. Average life expectancy is only 28 years. The physical, emotional and financial impact of Duchenne on those affected, their families and caregivers, is profound. Duchenne is an X-linked, rare neuromuscular disease caused by pathogenic variants (mutations) in the DMD gene that disrupt the production of functional dystrophin protein, leading to progressive and irreversible muscle weakness, diminished quality of life and premature death. Dystrophin strengthens and protects muscles and without it, normal activity causes excessive damage to muscle cells as they are more sensitive to injury. Over time, muscle tissue is replaced with scar tissue and fat, causing muscles to weaken. Although Duchenne progresses differently in each individual, its devastating trajectory is well established. Those with Duchenne will eventually lose the ability to use and move their limbs, to breathe on their own and are susceptible to respiratory infections. Muscle damage to the heart causes cardiomyopathy, including rhythm abnormalities and heart failure. Early diagnosis is important for timely intervention to prolong muscle function and preserve quality of life. There is a critical need for disease-modifying treatments that address the underlying cause of DMD before irreversible muscle loss occurs. 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 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. For over 125 years, sustainability has been an integral part of Roche's business. As a science-driven company, our greatest contribution to society is developing innovative medicines and diagnostics that help people live healthier lives. 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