Latest news with #LeviGarraway
Scottish Sun
15 hours ago
- Health
- Scottish Sun
‘Game-changing' Alzheimer's drug could STOP disease in its tracks – as the ‘most powerful weapon' against condition yet
Click to share on X/Twitter (Opens in new window) Click to share on Facebook (Opens in new window) A NEW "game-changing" drug could stop the progression of Alzheimer's disease, early trials suggest. Scientists say the treatment - called trontinemab - could be the most powerful weapon yet against dementia, as they look into giving the drug to people with no symptoms in the hopes of preventing disease. Sign up for Scottish Sun newsletter Sign up 1 Trontinemab is formulated to bypass the blood-brain barrier to target plaques clogging up brain cells Credit: Getty Phase two trial results for trontinemab were presented at he Alzheimer's Association International Conference in Toronto. Researchers said the drug seemed to clear plaques - the abnormal build-up of proteins around brain cells that cause damage and disrupt function - quicker than other drugs licensed to treat Alzheimer's. Trontinemab cleared out plaques and eliminated visible signs of the the disease within 28 weeks for nine out of 10 patients. Drug-maker Roche is now conducting another trial to see if plaque clearance also improves patients' memory. The 18-month study includes 1,600 people, the Telegraph reported. Separate research will examine whether the drug could be given to people without any signs of dementia, just as statins are used to ward off heart disease. Experts hope the drug could stop patients from developing dementia symptoms at all, if it is given early enough. They described the findings so far as 'very promising', suggesting that the drug was much more powerful than existing ones while leading to fewer side effects - as it was able to clear plaques better within seven months that other drugs can in 18 months. Roche's chief medical officer Levi Garraway said: 'Alzheimer's disease represents one of the greatest challenges in healthcare today and tackling it requires early detection and effective therapeutics. 'Trontinemab is designed to target a key driver of Alzheimer's disease biology more effectively in the brain. Common painkiller used for back pain ups risk of dementia by 29%, scientists warn "Combining new treatment avenues with advanced diagnostics may enable earlier and potentially more effective intervention. "With plans for phase three trials in both early symptomatic and pre-clinical Alzheimer's disease, we are advancing science with the goal of delaying - and ultimately preventing - progression of this devastating condition.' Prof Sir John Hardy, the chairman of molecular biology of neurological disease at University College London's Institute of Neurology told the Telegraph trontinemab could be 'game-changing'. The scientist, who was the first to identify the role of amyloid plaques in Alzeheimer's, said: 'This is absolutely great news. "It sucks the plaque out of the brain really quickly, much faster than we have seen with lecanemab or donanemab.' Both are Alzheimer's drugs approved for used in the UK, which target amyloid proteins in the brain to slow disease progression. However, neither treatment is offered on the NHS, as the National Institute for Health and Care Excellence (Nice) deemed them too expensive - in large part because patients need frequent scans to monitor for brain swelling and bleeds. Prof Hardy added: 'There is no doubt [trontinemab] could be game-changing. "We hope that if we can use these drugs to people early, we can halt the progression of disease, even before people have symptoms. "Now we need to see the size of the clinical effect. 'These results show it is much faster and safer than previous drugs, which means less monitoring. "That brings down the cost significantly, it means fewer MRI scans, so that would surely mean it would get Nice approval.' Early signs of dementia It's not unusual for your memory to lapse a bit as you get older. But dementia is different from 'just getting old' as it will cause noticeable - rather than gradual - changes to mental abilities and make managing everyday tasks and activities increasingly difficult. The symptoms of dementia may be small to start with, but get worse over time. Below are some examples of possible signs. Memory loss Memory loss is a key sign of dementia. This can include: Forgetting something you were only recently told. You may ask for the same information repeatedly – for example, 'Are the doors locked?' Putting objects in unusual places – for example, putting your house keys in the refrigerator. Being unable to learn new tasks, like how to use a new washing machine. Planning and decision making issues People with dementia can have difficulty with planning and decision making. This can include: Getting very confused when planning or thinking things through. Struggling to stay focused on a single task. Not making informed, careful decisions when dealing with money or looking at risks. Finding it hard to manage regular payments, budgets or monthly bills. Problems with language and understanding In people with dementia, this can manifest as: Having frequent problems finding the right word or regularly referring to objects as 'that thing'. Finding it hard to take part in conversations. Regularly being unable to follow what someone is saying even without distractions. Losing sense of time and place Dementia can cause problems with orientation, including: Losing track of the date, season or the passage of time. Getting lost in a place that is familiar or that should be easy to find your way around – for example, a supermarket. Regularly being unable to follow what someone is saying even without distractions. Problems with vision and perception This can mean having problems making sense of what you see. For example, having difficulty judging distances on stairs, or mistaking reflections or patterns for other objects. Mood and behavioural changes Finally, dementia can also make people act differently or shift their mood. This can mean: Becoming withdrawn and losing interest in work, friends or hobbies. Feeling unusually sad, anxious, frightened or low in confidence. Getting easily upset at home, at work, with friends or in places that usually feel comfortable or familiar. Source: Alzheimer's Society Trials for lecanemab and donanemab showed the drugs helped clear toxic clumps out of the brain and slow nerve damage caused by Alzheimer's by 27 per cent and 35 per cent respectively, over the course of 18 months. But the treatments did present a risk of brain swelling and brain bleeds, meaning patients needed intense monitoring. Trontinemab appears to be safer and cause fewer side effects, with less than five per cent of patients showing abnormal results on their brain scans. All of these cases were quickly resolved, researchers said. The drug - administered by infusion into the bloodstream - also doesn't need to be given a frequently. Upcoming trials will examine the impact of giving the drug to patients once a month for six months, then every three months. Trontinemab has been designed to efficiently bypass the blood-brain barrier, a protective layer of cells that stops harmful substances in the blood stream from attacking the brain and spinal cord. This barrier can make it hard for many drugs to reach the brain. Prof Jonathan Schott, the chief medical officer at Alzheimer's Research UK, told the Telegraph: 'We urgently need a range of treatments for Alzheimer's that are effective and safe for the people affected by this devastating disease. 'Evidence presented at the Alzheimer's Association conference in Toronto on trontinemab is very promising, showing that the drug can effectively and rapidly clear amyloid from the brain, seemingly with very few side effects. 'We now need to see whether these early stage results carry through to later stage clinical trials, which are planned to start later this year, including in the UK. "These trials will show whether the drug is not only safe, but impacts on memory, thinking and quality of life.' He said it was 'exciting' that the drug would now be tested in some people without symptoms under the phase three trials.
The Sun
15 hours ago
- Health
- The Sun
‘Game-changing' Alzheimer's drug could STOP disease in its tracks – as the ‘most powerful weapon' against condition yet
A NEW "game-changing" drug could stop the progression of Alzheimer's disease, early trials suggest. Scientists say the treatment - called trontinemab - could be the most powerful weapon yet against dementia, as they look into giving the drug to people with no symptoms in the hopes of preventing disease. 1 Phase two trial results for trontinemab were presented at he Alzheimer's Association International Conference in Toronto. Researchers said the drug seemed to clear plaques - the abnormal build-up of proteins around brain cells that cause damage and disrupt function - quicker than other drugs licensed to treat Alzheimer's. Trontinemab cleared out plaques and eliminated visible signs of the the disease within 28 weeks for nine out of 10 patients. Drug-maker Roche is now conducting another trial to see if plaque clearance also improves patients' memory. The 18-month study includes 1,600 people, the Telegraph reported. Separate research will examine whether the drug could be given to people without any signs of dementia, just as statins are used to ward off heart disease. Experts hope the drug could stop patients from developing dementia symptoms at all, if it is given early enough. They described the findings so far as 'very promising', suggesting that the drug was much more powerful than existing ones while leading to fewer side effects - as it was able to clear plaques better within seven months that other drugs can in 18 months. Roche's chief medical officer Levi Garraway said: 'Alzheimer's disease represents one of the greatest challenges in healthcare today and tackling it requires early detection and effective therapeutics. 'Trontinemab is designed to target a key driver of Alzheimer's disease biology more effectively in the brain. "Combining new treatment avenues with advanced diagnostics may enable earlier and potentially more effective intervention. "With plans for phase three trials in both early symptomatic and pre-clinical Alzheimer's disease, we are advancing science with the goal of delaying - and ultimately preventing - progression of this devastating condition.' Prof Sir John Hardy, the chairman of molecular biology of neurological disease at University College London's Institute of Neurology told the Telegraph trontinemab could be 'game-changing'. The scientist, who was the first to identify the role of amyloid plaques in Alzeheimer's, said: 'This is absolutely great news. "It sucks the plaque out of the brain really quickly, much faster than we have seen with lecanemab or donanemab.' Both are Alzheimer's drugs approved for used in the UK, which target amyloid proteins in the brain to slow disease progression. However, neither treatment is offered on the NHS, as the National Institute for Health and Care Excellence (Nice) deemed them too expensive - in large part because patients need frequent scans to monitor for brain swelling and bleeds. Prof Hardy added: 'There is no doubt [trontinemab] could be game-changing. "We hope that if we can use these drugs to people early, we can halt the progression of disease, even before people have symptoms. "Now we need to see the size of the clinical effect. 'These results show it is much faster and safer than previous drugs, which means less monitoring. "That brings down the cost significantly, it means fewer MRI scans, so that would surely mean it would get Nice approval.' Early signs of dementia It's not unusual for your memory to lapse a bit as you get older. But dementia is different from 'just getting old' as it will cause noticeable - rather than gradual - changes to mental abilities and make managing everyday tasks and activities increasingly difficult. The symptoms of dementia may be small to start with, but get worse over time. Below are some examples of possible signs. Memory loss Memory loss is a key sign of dementia. This can include: Forgetting something you were only recently told. You may ask for the same information repeatedly – for example, 'Are the doors locked?' Putting objects in unusual places – for example, putting your house keys in the refrigerator. Being unable to learn new tasks, like how to use a new washing machine. Planning and decision making issues People with dementia can have difficulty with planning and decision making. This can include: Getting very confused when planning or thinking things through. Struggling to stay focused on a single task. Not making informed, careful decisions when dealing with money or looking at risks. Finding it hard to manage regular payments, budgets or monthly bills. Problems with language and understanding In people with dementia, this can manifest as: Having frequent problems finding the right word or regularly referring to objects as 'that thing'. Finding it hard to take part in conversations. Regularly being unable to follow what someone is saying even without distractions. Losing sense of time and place Dementia can cause problems with orientation, including: Losing track of the date, season or the passage of time. Getting lost in a place that is familiar or that should be easy to find your way around – for example, a supermarket. Regularly being unable to follow what someone is saying even without distractions. Problems with vision and perception This can mean having problems making sense of what you see. For example, having difficulty judging distances on stairs, or mistaking reflections or patterns for other objects. Mood and behavioural changes Finally, dementia can also make people act differently or shift their mood. This can mean: Becoming withdrawn and losing interest in work, friends or hobbies. Feeling unusually sad, anxious, frightened or low in confidence. Getting easily upset at home, at work, with friends or in places that usually feel comfortable or familiar. Trials for lecanemab and donanemab showed the drugs helped clear toxic clumps out of the brain and slow nerve damage caused by Alzheimer's by 27 per cent and 35 per cent respectively, over the course of 18 months. But the treatments did present a risk of brain swelling and brain bleeds, meaning patients needed intense monitoring. Trontinemab appears to be safer and cause fewer side effects, with less than five per cent of patients showing abnormal results on their brain scans. All of these cases were quickly resolved, researchers said. The drug - administered by infusion into the bloodstream - also doesn't need to be given a frequently. Upcoming trials will examine the impact of giving the drug to patients once a month for six months, then every three months. Trontinemab has been designed to efficiently bypass the blood-brain barrier, a protective layer of cells that stops harmful substances in the blood stream from attacking the brain and spinal cord. This barrier can make it hard for many drugs to reach the brain. Prof Jonathan Schott, the chief medical officer at Alzheimer's Research UK, told the Telegraph: 'We urgently need a range of treatments for Alzheimer's that are effective and safe for the people affected by this devastating disease. 'Evidence presented at the Alzheimer's Association conference in Toronto on trontinemab is very promising, showing that the drug can effectively and rapidly clear amyloid from the brain, seemingly with very few side effects. 'We now need to see whether these early stage results carry through to later stage clinical trials, which are planned to start later this year, including in the UK. "These trials will show whether the drug is not only safe, but impacts on memory, thinking and quality of life.' He said it was 'exciting' that the drug would now be tested in some people without symptoms under the phase three trials.
Business Upturn
2 days ago
- Health
- Business Upturn
Roche presents new insights in Alzheimer's disease research across its diagnostics and pharmaceutical portfolios at AAIC
By GlobeNewswire Published on July 28, 2025, 10:00 IST Trontinemab's Phase Ib/IIa Brainshuttle™ AD study continues to show rapid and robust clearance of amyloid plaques, with 91% becoming amyloid PET negative and ARIA-E remaining <5% Design of the Phase III TRONTIER 1 and 2 studies of trontinemab in early symptomatic Alzheimer's disease featured, with initiation planned in 2025 Plans for new Phase III trial investigating trontinemab in preclinical Alzheimer's disease, in people at high risk of cognitive decline New real-world data support Elecsys pTau217 as a standalone blood test, comparable to a PET scan, for rule-in and rule-out identification of amyloid pathology Basel, 28 July 2025 – Roche (SIX: RO, ROG; OTCQX: RHHBY) announced today that new data from its Alzheimer's development portfolio is being presented at the Alzheimer's Association International Conference (AAIC) in Toronto, Canada (July 27-30). These data exemplify the comprehensive approach Roche is taking in addressing Alzheimer's across the entire patient journey. Featured oral presentations include the latest results from the ongoing Phase Ib/IIa Brainshuttle™ AD study, which continue to support rapid and robust reduction of amyloid plaques, and design of the Phase III TRONTIER 1 and 2 studies of investigational trontinemab for early symptomatic Alzheimer's disease, with initiation planned later this year. As part of its growing Alzheimer's development programme, Roche announced today its plans for an additional Phase III trial to investigate trontinemab in preclinical Alzheimer's disease. The trial will focus on individuals at risk of cognitive decline, with the goal of potentially delaying or preventing the progression of the disease to symptomatic stages. 'Alzheimer's disease represents one of the greatest challenges in healthcare today and tackling it requires early detection and effective therapeutics,' said Levi Garraway, M.D., Ph.D., Roche's Chief Medical Officer and Head of Global Product Development. 'Trontinemab is designed to target a key driver of Alzheimer's disease biology more effectively in the brain. Combining new treatment avenues with advanced diagnostics may enable earlier and potentially more effective intervention. With plans for Phase III trials in both early symptomatic and preclinical Alzheimer's disease, we are advancing science with the goal of delaying —and ultimately preventing—progression of this devastating condition.' Late-breaking oral and poster presentations highlight the potential of Roche's Elecsys® pTau217 as a reliable and accessible blood-based biomarker test, providing comparable results to PET scan and cerebrospinal fluid (CSF) diagnostics for rule-in and rule-out diagnosis of amyloid pathology, a hallmark of Alzheimer's disease, across care settings. The test, which received Breakthrough Device Designation from the U.S. Food and Drug Administration last year, will also be utilised in Roche's TRONTIER studies. 'Blood based testing for Alzheimer's disease has the potential to greatly improve patient access and decrease the time to definitive disease diagnosis,' said Matt Sause, CEO of Roche Diagnostics. 'Our data show that the Elecsys pTau217 test performs comparably to PET scans but can be performed with a simple blood draw and analyzed in a routine clinical laboratory. This has the potential to transform the diagnosis of Alzheimer's and provide clear answers to caregivers, patients, and their families.' Up to 75% of people living with symptoms of Alzheimer's disease globally have not been diagnosed, and those who have, waited an average of 2.8 years1, and even less have received any form of treatment. Diagnostics play a crucial role in addressing the global challenge of Alzheimer's, not only to detect and identify people with the disease early, even before the first symptoms, but also to rule out those who may or may not benefit from specific treatments. Pharmaceuticals In a 90-minute Featured Research session, designs were shared for the Phase III studies, TRONTIER 1 and 2, which will initiate later this year, investigating the efficacy and safety of investigational trontinemab in people with early Alzheimer's disease. The primary endpoint will measure the change in cognition and function based on the Clinical Dementia Rating – Sum of Boxes scale after 18 months of treatment. Secondary endpoints will include assessments of cognition, function, behavioural symptoms, and quality of life. A pre-screening study, TRAVELLER, based on a brief clinical assessment and a plasma biomarker, which will be identified using the Elecsys pTau217 test, has also been initiated, to enable broader community outreach and extend access to these trials to more diverse populations representative of Alzheimer's disease. New data on the latest results for trontinemab from the completed dose-expansion part of the 1.8 mg/kg and 3.6 mg/kg cohorts from the ongoing Phase Ib/IIa Brainshuttle AD study continued to show rapid and robust reduction of amyloid plaques in the brain as measured by amyloid positron emission tomography (PET). In the 3.6 mg/kg cohort, trontinemab reduced amyloid levels below the 24 centiloid positivity threshold in 91% of participants (n=49/54) after 28 weeks of treatment; 72% (n=39/54) achieved deep clearance below 11 centiloids. These data were reinforced by early and significant reductions in fluid biomarkers of Alzheimer's disease, including total tau, phosphorylated Tau (pTau)181, pTau217, and neurogranin measured in CSF and continues to show a favourable safety and tolerability profile. Amyloid-related imaging abnormalities-edema/effusion (ARIA-E) continued to be observed in <5% of participants (blinded data; N=4/149 across 1.8 and 3.6mg/kg dose cohorts). All cases were radiographically mild, one was associated with mild and transient symptoms. Diagnostics Roche will present data on a new study comparing the pTau217/Ab42 plasma ratio to the high-throughput, fully automated Elecsys pTau217 assay. The presentation will report on the accuracy of these tools in detecting amyloid pathology. Together with the high throughput and full automation of the assay, these data will assess the potential of Elecsys pTau217 as an accurate standalone rule-in and rule-out test that could be scaled up for broad implementation in routine clinical practice worldwide. Additionally, results from a cohort-based model of healthcare utilisation in the U.S. demonstrated that using the Elecsys® pTau181 blood-based rule-out test in primary care scenarios improved diagnostic accuracy and reduced resource use compared with the current standard-of-care clinical, cognitive and imaging tests. If made available in primary care settings, the Roche Elecsys® pTau181 blood test has the potential to reliably avoid the need for further confirmatory testing in nearly all people who receive a negative result. This will avoid the need for these people to undergo unnecessary testing using CSF or PET, which often come with long wait times and high cost, resulting in further delays to diagnosis and cost to healthcare systems. Medicine Abstract title Presentation number (type) Presentation date (session) Time Abstracts will be available on the AAIC website. Pharmaceuticals Next wave of innovation in Alzheimer's disease therapeutics: The value of novel active transport mechanisms Featured Research Session (FRS), Talk 1 Room 718 27 Jul 2025, 2pm – 3.30pm EDT Cath Mummery, Roberto Villaseñor, Jens Niewoehner, Scarlett Barker, Luka Kulic Latest results from the dose-expansion part (Part 2) of the Brainshuttle™ AD study of trontinemab in people with Alzheimer's disease Featured Research Session (FRS), Talk 2 Room 71827 Jul 2025, 2pm – 3.30pm EDT Luka Kulic, Fabien Alcaraz, Gregory Klein, Stephen Salloway, Carsten Hofmann, João A. Abrantes, Stella Yilmaz, Denise Sickert, Maddalena Marchesi, Jakub Wojtowicz, Andres Schneider, Ruth Croney, David Agnew, Silke Ahlers, Paul Delmar, Hanno Svoboda, Iris Wiesel Interim biomarker results for trontinemab, a novel Brainshuttle™ antibody in development for the treatment of Alzheimer's disease Featured Research Session (FRS), Talk 3 Room 718 27 Jul 2025, 2pm – 3.30pm EDT Gregory Klein, Gil Rabinovici, Henrik Zetterberg, Matteo Tonietto, Tobias Bittner, Daria Rukina, Fabien Alcaraz, Carsten Hofmann, Maddalena Marchesi, Jakub Wojtowicz, Ruth Croney, David Agnew, João A. Abrantes, Franziska Schaedeli Stark, Silke Ahlers, Paul Delmar, Hanno Svoboda, Iris Wiesel, Luka Kulic TRONTIER 1 and TRONTIER 2: Pivotal trials of trontinemab in early symptomatic Alzheimer's disease Featured Research Session (FRS), Talk 4 Room 71827 Jul 2025, 2pm – 3.30pm EDT Janice Smith, Catherine Mummery, Jeffrey L. Cummings, Gil Rabinovici, Stephen Salloway, Reisa Sperling, Henrik Zetterberg, Angeliki Thanasopolou, Christopher Lane, Paul Delmar, Gregory Klein, Ruth Croney, Jakub Wojtowicz, Carsten Hofmann, Luka Kulic, Hideki Garren Diagnostics Evaluating the Impact on Diagnostic Performance and Healthcare Resource Utilization of Introducing a plasma rule-out test in the Alzheimer's Disease Diagnostic Pathway Poster #102729 July 27, 7:30am- 4:15pm EDT Sophie Roth , Gustaf Ortsäter, Joana Amorim Freire Location tbc Evaluating the Clinical Performance of the Elecsys pTau217 Plasma Immunoassay to Detect Amyloid Pathology in a Routine Clinical Practice Cohort Poster #96679 July 28, 7:30 am – 4:15 pm EDT Sayuri Hortsch , Niels Borlinghaus, Alexander Jethwa, David Caley, Annunziata Di Domenico, Craig Ritchie Clinical performance and effect of pre-analytical variation of plasma pTau217 alone versus the plasma pTau217/Aβ42 ratio for the identification of amyloid pathology Oral Developing Topics #108585 3-23-DEV Developing Topics on Tau Biomarkers July 29, 2025: 2:00 PM – 3:30 PM Christopher M. Rank, Joana Amorim Freire, Alexander Jethwa, Annunziata Di Domenico, Christina Rabe, Marc Suárez-Calvet , Colin L. Masters, Tobias Bittner Accuracy of cerebrospinal fluid biomarker ratios to determine amyloid positron-emission tomography status: a diagnostic test accuracy meta-analysis Poster #100941 July 28, 7:30 am – 4:15 pm EDT Pablo Martinez-Lage, Eino Solje, Julian G. Martins, Sraboni Sarkar Equity in diagnosis through adequate clinical trial design in diagnostic performance studies Poster #102804 July 30, 7:30am-4:15pm EDT Imke Kirste , David Caley, Clara Quijano Rubio, Margherita Carboni Investigating Differences in Patients Enrolled in a Clinical Study Based on Referral Type Poster #108110 July 30, 7:30am-4:15pm EDT Sophie Roth , Laura Schlieker, Sayuri Hortsch, Joana Amorim Freire,David Caley About trontinemab Trontinemab is an investigational Brainshuttle bispecific 2+1 amyloid-beta targeting monoclonal antibody specifically engineered for enhanced access to the brain to enable rapid reduction of amyloid in people with Alzheimer's disease. Trontinemab is designed for the efficient transport across the blood-brain barrier to target aggregated forms of amyloid beta and remove amyloid plaques in the brain. The uniqueness of trontinemab is based on Roche's proprietary Brainshuttle technology combining an amyloid beta-binding antibody with a transferring receptor (TfR1) shuttle module. As a result, high central nervous system (CNS) exposure of trontinemab may be achieved at low doses, leading to a rapid and deep amyloid clearance. Due to its unique properties, trontinemab might unlock the full potential of disease-modifying monoclonal antibodies by effectively penetrating the brain and potentially leading to slowing of disease progression. About Roche in Alzheimer's Disease With more than two decades of scientific research in Alzheimer's disease, Roche is working towards a day when we can detect and treat the disease early, in order to slow down, stop or even prevent its progression to preserve what makes people who they are. Today, the company's Alzheimer's disease portfolio spans investigational medicines for different targets, types and stages of the disease, including trontinemab. On the diagnostics side, it also includes approved and investigational tools, including digital and blood-based tests and CSF assays, aiming to more effectively detect, diagnose and monitor the disease. Yet the global challenges of Alzheimer's disease go well beyond the capabilities of science, and making a meaningful impact requires collaboration both within the Alzheimer's community and outside of healthcare. Roche will continue to work together with numerous partners with the hope to transform millions of lives. 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. References [1] Roche Global Media Relations Phone: +41 61 688 8888 / e-mail: [email protected] 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 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 Investor Relations North America Loren KalmPhone: +1 650 225 3217 e-mail: [email protected] Attachment Media Investor Release AAIC 2025 English Disclaimer: The above press release comes to you under an arrangement with GlobeNewswire. Business Upturn takes no editorial responsibility for the same. Ahmedabad Plane Crash GlobeNewswire provides press release distribution services globally, with substantial operations in North America and Europe.
Business Wire
2 days ago
- Health
- Business Wire
Genentech and Roche Present New Insights in Alzheimer's Disease Research Across Its Diagnostics and Pharmaceutical Portfolios at AAIC
SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY) announced today that new data from its Alzheimer's development portfolio is being presented at the Alzheimer's Association International Conference (AAIC) in Toronto, Canada (July 27-30). These data exemplify the comprehensive approach Roche is taking in addressing Alzheimer's across the entire patient journey. Featured oral presentations include the latest results from the ongoing Phase Ib/IIa Brainshuttle™ AD study, which continue to support rapid and robust reduction of amyloid plaques, and design of the Phase III TRONTIER 1 and 2 studies of investigational trontinemab for early symptomatic Alzheimer's disease, with initiation planned later this year. As part of its growing Alzheimer's development program, Roche announced today its plans for an additional Phase III trial to investigate trontinemab in preclinical Alzheimer's disease. The trial will focus on individuals at risk of cognitive decline, with the goal of potentially delaying or preventing the progression of the disease to symptomatic stages. 'Alzheimer's disease represents one of the greatest challenges in healthcare today and tackling it requires early detection and effective therapeutics,' said Levi Garraway, M.D., Ph.D., chief medical officer and head of Global Product Development. 'Trontinemab is designed to target a key driver of Alzheimer's disease biology more effectively in the brain. Combining new treatment avenues with advanced diagnostics may enable earlier and potentially more effective intervention. With plans for Phase III trials in both early symptomatic and preclinical Alzheimer's disease, we are advancing science with the goal of delaying—and ultimately preventing—progression of this devastating condition.' Late-breaking oral and poster presentations highlight the potential of Roche's Elecsys ® pTau217 as a reliable and accessible blood-based biomarker test, providing comparable results to PET scan and cerebrospinal fluid (CSF) diagnostics for rule-in and rule-out diagnosis of amyloid pathology, a hallmark of Alzheimer's disease, across care settings. The test, which received Breakthrough Device Designation from the U.S. Food and Drug Administration last year, will also be utilized in Roche's TRONTIER studies. 'Blood based testing for Alzheimer's disease has the potential to greatly improve patient access and decrease the time to definitive disease diagnosis,' said Matt Sause, CEO of Roche Diagnostics. 'Our data show that the Elecsys pTau217 test performs comparably to PET scans but can be performed with a simple blood draw and analyzed in a routine clinical laboratory. This has the potential to transform the diagnosis of Alzheimer's and provide clear answers to caregivers, patients, and their families.' Up to 75% of people living with symptoms of Alzheimer's disease globally have not been diagnosed, and those who have, waited an average of 2.8 years, and even less have received any form of treatment. Diagnostics play a crucial role in addressing the global challenge of Alzheimer's, not only to detect and identify people with the disease early, even before the first symptoms, but also to rule out those who may or may not benefit from specific treatments. Pharmaceuticals In a 90-minute Featured Research session, designs were shared for the Phase III studies, TRONTIER 1 and 2, which will initiate later this year, investigating the efficacy and safety of investigational trontinemab in people with early Alzheimer's disease. The primary endpoint will measure the change in cognition and function based on the Clinical Dementia Rating – Sum of Boxes scale after 18 months of treatment. Secondary endpoints will include assessments of cognition, function, behavioral symptoms, and quality of life. A pre-screening study, TRAVELLER, based on a brief clinical assessment and a plasma biomarker, which will be identified using the Elecsys pTau217 test, has also been initiated, to enable broader community outreach and extend access to these trials to more diverse populations representative of Alzheimer's disease. New data on the latest results for trontinemab from the completed dose-expansion part of the 1.8 mg/kg and 3.6 mg/kg cohorts from the ongoing Phase Ib/IIa Brainshuttle AD study continued to show rapid and robust reduction of amyloid plaques in the brain as measured by amyloid positron emission tomography (PET). In the 3.6 mg/kg cohort, trontinemab reduced amyloid levels below the 24 centiloid positivity threshold in 91% of participants (n=49/54) after 28 weeks of treatment; 72% (n=39/54) achieved deep clearance below 11 centiloids. These data were reinforced by early and significant reductions in fluid biomarkers of Alzheimer's disease, including total tau, phosphorylated Tau (pTau)181, pTau217, and neurogranin measured in CSF and continues to show a favourable safety and tolerability profile. Amyloid-related imaging abnormalities-edema/effusion (ARIA-E) continued to be observed in <5% of participants (blinded data; N=4/149 across 1.8 and 3.6 mg/kg dose cohorts). All cases were radiographically mild, one was associated with mild and transient symptoms. Diagnostics Roche will present data on a new study comparing the pTau217/Ab42 plasma ratio to the high-throughput, fully automated Elecsys pTau217 assay. The presentation will report on the accuracy of these tools in detecting amyloid pathology. Together with the high throughput and full automation of the assay, these data will assess the potential of Elecsys pTau217 as an accurate standalone rule-in and rule-out test that could be scaled up for broad implementation in routine clinical practice worldwide. Additionally, results from a cohort-based model of healthcare utilization in the U.S. demonstrated that using the Elecsys ® pTau181 blood-based rule-out test in primary care scenarios improved diagnostic accuracy and reduced resource use compared with the current standard-of-care clinical, cognitive and imaging tests. If made available in primary care settings, the Roche Elecsys ® pTau181 blood test has the potential to reliably avoid the need for further confirmatory testing in nearly all people who receive a negative result. This will avoid the need for these people to undergo unnecessary testing using CSF or PET, which often come with long wait times and high cost, resulting in further delays to diagnosis and cost to healthcare systems. About trontinemab Trontinemab is an investigational Brainshuttle bispecific 2+1 amyloid-beta targeting monoclonal antibody specifically engineered for enhanced access to the brain to enable rapid reduction of amyloid in people with Alzheimer's disease. Trontinemab is designed for the efficient transport across the blood-brain barrier to target aggregated forms of amyloid beta and remove amyloid plaques in the brain. The uniqueness of trontinemab is based on Roche's proprietary Brainshuttle technology combining an amyloid beta-binding antibody with a transferring receptor (TfR1) shuttle module. As a result, high central nervous system (CNS) exposure of trontinemab may be achieved at low doses, leading to a rapid and deep amyloid clearance. Due to its unique properties, trontinemab might unlock the full potential of disease-modifying monoclonal antibodies by effectively penetrating the brain and potentially leading to slowing of disease progression. About Roche in Alzheimer's Disease With more than two decades of scientific research in Alzheimer's disease, Roche is working towards a day when we can detect and treat the disease early, in order to slow down, stop or even prevent its progression to preserve what makes people who they are. Today, the company's Alzheimer's disease portfolio spans investigational medicines for different targets, types and stages of the disease, including trontinemab. On the diagnostics side, it also includes approved and investigational tools, including digital and blood-based tests and CSF assays, aiming to more effectively detect, diagnose and monitor the disease. Yet the global challenges of Alzheimer's disease go well beyond the capabilities of science, and making a meaningful impact requires collaboration both within the Alzheimer's community and outside of healthcare. Roche will continue to work together with numerous partners with the hope to transform millions of lives. About Genentech in Neuroscience Neuroscience is a major focus of research and development at Genentech. Our goal is to pursue groundbreaking science to develop new treatments that help improve the lives of people with chronic and potentially devastating diseases. Genentech and Roche are 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 Genentech Founded more than 40 years ago, Genentech is a leading biotechnology company that discovers, develops, manufactures and commercializes medicines to treat patients with serious and life-threatening medical conditions. The company, a member of the Roche Group, has headquarters in South San Francisco, California. For additional information about the company, please visit
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Roche provides regulatory update on Elevidys™ gene therapy for Duchenne muscular dystrophy in the EU
EMA's CHMP issued an opinion not to recommend Elevidys™ (delandistrogene moxeparvovec) for the treatment of ambulatory individuals with Duchenne muscular dystrophy (DMD) Roche will continue its dialogue with the EMA to explore a potential path forward to make Elevidys available to individuals living with DMD in the EU Roche believes the benefit-risk remains positive in the ambulatory Duchenne population Elevidys is the first and only disease-modifying gene therapy for DMD Basel, 25 July 2025 – Roche (SIX: RO, ROG; OTCQX: RHHBY) announced today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) issued a negative opinion on the conditional marketing authorisation (CMA) for Elevidys™ (delandistrogene moxeparvovec) for ambulatory individuals aged three to seven years with Duchenne muscular dystrophy (DMD). Given the high unmet need in DMD, Roche plans to continue to work with the EMA to explore a potential path forward. 'We are disappointed by the CHMP's negative opinion, given the urgent need for disease-modifying therapies for children in the EU living with Duchenne,' said Levi Garraway, M.D., Ph.D., Chief Medical Officer and Head of Global Product Development, Roche. 'With an average life expectancy of only 28 years, achieving disease stabilisation is a major advance for individuals living with Duchenne, their families and caregivers. We are confident in the value Elevidys can bring to ambulatory patients.' The CHMP opinion is based on data from the largest and broadest gene therapy clinical programme in DMD to date, including results from the pivotal Phase III EMBARK study that showed treatment with Elevidys provided sustained stabilisation or slowing of disease progression, and a consistent and manageable safety profile in ambulatory patients. To date, more than 900 individuals with DMD, 760 of whom are ambulatory, have been treated with Elevidys in clinical and real-world settings. While the primary endpoint was not met in EMBARK after one year, Elevidys showed clinically meaningful and statistically significant improvements across important secondary endpoints of functional outcome measures when compared to placebo. Longer term efficacy data were also submitted to EMA, including two-year results from the EMBARK study and three-year pooled efficacy analysis from three other Elevidys studies that showed clinically meaningful improvements across key measures of motor function. One-year data from part one of the EMBARK study were published in Nature Medicine in October 2024 and results from year two were shared at this year's Muscular Dystrophy Association clinical & scientific conference in Dallas, TX. DMD is a rare, genetic, muscle-wasting disease that progresses rapidly from early childhood. 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. Roche recognises there is a significant unmet medical need for those living with Duchenne and the urgency for treating children before DMD progresses to provide the best possible chance for improved outcomes. We are actively working with health authorities across the globe to bring Elevidys to patients and their families as soon as possible. Elevidys is the first and only approved gene therapy targeting the underlying cause of disease that consistently demonstrates stabilisation or slowing of DMD disease progression, with durable effects on functional and biological outcomes and muscle health. About Duchenne muscular dystrophy DMD 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 Elevidys™ (delandistrogene moxeparvovec) Elevidys™ (delandistrogene moxeparvovec, also known as SRP-9001) is the first approved disease-modifying gene 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 intravenous dose. Elevidys is contraindicated in individuals with any deletion in exons 8 and/or 9 in the DMD gene. Elevidys has been studied in the largest and broadest gene therapy clinical development program in DMD with the longest follow-up (up to six years) in patients of all ages, and with various DMD mutations. To date, more than 900 individuals with DMD (more than 760 of whom are ambulatory) have been treated with Elevidys across clinical and real-world settings and Elevidys is now approved in nine countries, including Japan. Important Elevidys Updates: On 15 June, Roche announced new dosing restrictions for Elevidys for non-ambulatory DMD patients, irrespective of age, in both clinical and commercial settings. These measures followed two reported fatalities in the non-ambulatory DMD population. On 22 July, in response to the U.S. Food and Drug Administration (FDA)'s request to Sarepta, Roche took additional measures towards initiating a voluntary and temporary pause of any new orders of Elevidys to countries outside the U.S. that reference the FDA as the basis for their local approval, and in Named Patient Supply (NPS) countries. Discussions with other relevant health authorities are ongoing. Roche will immediately respect requests to halt new orders and shipments from health authorities. Patient safety is Roche's highest priority. Based on the totality of available data, Roche believes that the benefit-risk profile is positive in the ambulatory patient population. To date, approximately 760 ambulatory DMD patients have been treated with Elevidys in clinical and real-world settings and there have been no treatment-related fatalities. Elevidys is being developed by Roche in collaboration with Sarepta Therapeutics 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: [email protected] 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 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 Investor Relations North America Loren KalmPhone: +1 650 225 3217 e-mail: [email protected] Attachment Media Investor Release Elevidys CHMP Opinion English Disclaimer: The above press release comes to you under an arrangement with GlobeNewswire. Business Upturn takes no editorial responsibility for the same. Ahmedabad Plane Crash
