Sobi will showcase extensive research and clinical outcomes at ISTH 2025
"Attending ISTH is an important part of our commitment to collaboration and knowledge sharing so we can further advance treatments for those living with haemophilia, immune thrombocytopenia, and other rare blood conditions", said Lydia Abad-Franch, MD, Head of R&D and Medical Affairs, and Chief Medical Officer at Sobi.
"We are proud to be showcasing abstracts as part of the ISTH programme, including Doptelet abstracts that present new data related to children living with ITP, Altuvoct outcomes for perioperative management, and joint health outcomes from a long-term extension study, as well as final outcomes from the B-MORE study. Many people living with rare conditions have unmet needs, and we are proud to present additional data advancing treatments for these rare conditions."
Data to be presented at ISTH 2025
ALTUVOCT® (efanesoctocog alfa)
OC 64.4: Major Surgical Outcomes with Efanesoctocog Alfa: 4 Years' Experience in the XTEND Clinical Program
Presenting author: Liane Khoo
Oral presentation
Session date: Tuesday 24 June
Session time: 14:45 - 16:00 EDT
Location: 151 A&B, Walter E. Washington CC Convention Center
OC 20.5: Treatment of Bleeding Episodes with Efanesoctocog Alfa in Children: XTEND-ed Second Interim Analysis
Presenting author: Lynn Malec
Oral presentation
Session date: Sunday 22 June
Session time: 14:45 - 16:00 EDT
Location: Ballroom A-C, Walter E. Washington CC Convention Center
PB0847: Minor Surgeries Outcomes with Efanesoctocog Alfa: 4 Years' Experience in the XTEND Clinical Program
Presenting author: Pratima Chowdary
Poster presentation
Session date: Monday 23 June
Session time: 13:45 – 14:45 EDT
Location: Exhibition Hall
PB1425: Joint Health Outcomes with Efanesoctocog Alfa in Adults/Adolescents from XTEND-1 Continuing XTEND-ed
Presenting author: Christoph Königs
Poster presentation
Session date: Tuesday 24 June
Session time: 13:45 – 14:45 EDT
Location: Exhibition Hall
General Haemophilia
PB0778: Addressing unmet medical needs and health inequities in haemophilia A: expertconsensus statements
Presenting author: Cédric Hermans
Poster presentation
Session date: Monday 23 June
Session time: 13:45 – 14:45 EDT
Location: Exhibition Hall
PB0816: Extravascular distribution of factor IX: evidence and relevance for haemophilia B replacement therapy
Presenting author: Cédric Hermans
Poster presentation
Session date: Monday 23 June
Session time: 13:45 – 14:45 EDT
Location: Exhibition Hall
PB1439: Monitoring Joint Health in Haemophilia Patients in Spain: Updated Analysis of the JOIN-US Project
Presenting author: María Teresa Álvarez Román
Poster presentation
Session date: Tuesday 24 June
Session time: 13:45 – 14:45 EDT
Location: Exhibition Hall
ALPROLIX® (rFIXFc)
PB0868: Real-World Effectiveness and Usage
of Recombinant Factor IX Fc: Final Data from the B-MORE Study
Presenting author: Heidi Glosli
Poster presentation
Session date: Monday 23 June
Session time: 13:45 – 14:45 EDT
Location: Exhibition Hall
Doptelet® (avatrombopag)
PB0348: Consistent Response to Avatrombopag for the Treatment of Children with ITP Across Various Baseline Characteristics
Presenting author: Rachael F. Grace
Poster presentation
Session date: Sunday 22 June
Session time: 13:45 - 14:45 EDT
Location: Exhibition hall
PB0364: Evaluation of Efficacy and Safety of Avatrombopag in Children with ITP Based on Disease Duration
Presenting author: Rachael F. Grace
Poster presentation
Session date: Sunday 22 June
Session time: 13:45 - 14:45 EDT
Location: Exhibition hall
OC 65.3: Real-World Outcomes of Avatrombopag Treatment in Primary ITP Stratified by Prior TPO-RA Exposure
Presenting author: Srikanth Nagalla
Oral presentation
Session date: Tuesday 24 June 2025
Session time: 14:45 – 16:00 EDT
(Presentation time currently in programme: 15:15 – 15:30 EDT)
All abstracts are accessible through the official ISTH website. However, any late-breaking abstracts will only be made available later.
About ALTUVOCT® (efanesoctocog alfa)
ALTUVOCT® (efanesoctocog alfa) is indicated for the treatment and prophylaxis of bleeding in patients with haemophilia A. ALTUVOCT can be used for all age groups and any disease severity.
About Elocta®/Eloctate® (efmoroctocog alfa)
Elocta®/Eloctate® (efmoroctocog alfa) is a treatment and prophylaxis of bleeding in patients with haemophilia A. Elocta/Eloctate can be used for all age groups.
About Alprolix® (eftrenonacog alfa)
Alprolix® (eftrenonacog alfa) is a treatment and prophylaxis of bleeding in patients with haemophilia B. Alprolix can be used for all age groups.
About Doptelet® (avatrombopag)
Doptelet® (avatrombopag) is indicated for the treatment of primary chronic immune thrombocytopenia (ITP) in adult patients who are refractory to other treatments, and a treatment of severe thrombocytopenia in adult patients with chronic liver disease (CLD) who are scheduled to undergo an invasive procedure.
About Sobi
Sobi is a global biopharma company unlocking the potential of breakthrough innovations, transforming everyday life for people living with rare diseases. Sobi has approximately 1,900 employees across Europe, North America, the Middle East, Asia and Australia. In 2024, revenue amounted to SEK 26 billion. Sobi's share (STO:SOBI) is listed on Nasdaq Stockholm. More about Sobi at sobi.com and LinkedIn.
About Sanofi
Sanofi is an R&D driven, AI-powered biopharma company committed to improving people's lives and delivering compelling growth. Sanofi apply deep understanding of the immune system to invent medicines and vaccines that treat and protect millions of people around the world, with an innovative pipeline that could benefit millions more. Sanofi's team is guided by one purpose: to chase the miracles of science to improve people's lives; this inspires Sanofi to drive progress and deliver positive impact for Sanofi's people and the communities Sanofi serve, by addressing the most urgent healthcare, environmental, and societal challenges of our time.
Sanofi is listed on EURONEXT: SAN and NASDAQ: SNY
About the Sanofi and Sobi collaboration
Sobi and Sanofi collaborate on the development and commercialisation of Alprolix and Elocta/Eloctate. The companies also collaborate on the development and commercialisation of efanesoctocog alfa, or ALTUVIIIO® in the US, Japan, and Taiwan, and Altuvoct in Europe. Sobi has final development and commercialisation rights in the Sobi territory (essentially Europe, North Africa, Russia, and most Middle Eastern markets). Sanofi has final development and commercialisation rights in North America and all other regions in the world excluding the Sobi territory.
Contacts
For details on how to contact the Sobi Investor Relations Team, please click here. For Sobi Media contacts, click here.
This information was brought to you by Cision http://news.cision.com
https://news.cision.com/swedish-orphan-biovitrum-ab/r/sobi-will-showcase-extensive-research-and-clinical-outcomes-at-isth-2025,c4166601
The following files are available for download:
https://mb.cision.com/Main/14266/4166601/3514826.pdf
Sobi will showcase extensive research and clinical outcomes at ISTH 2025
View original content:https://www.prnewswire.co.uk/news-releases/sobi-will-showcase-extensive-research-and-clinical-outcomes-at-isth-2025-302486857.html
Hashtags
Try Our AI Features
Explore what Daily8 AI can do for you:
Comments
No comments yet...
Related Articles
CBS News
2 hours ago
- CBS News
Patient numbers at NIH hospital have dropped under Trump, jeopardizing care
The number of people receiving treatment at the National Institutes of Health Clinical Center — the renowned research hospital that cares for patients with rare or life-threatening diseases — has tumbled under the second Trump administration, according to government documents and interviews with current and former NIH employees. NIH documents viewed by KFF Health News show a pronounced decline in patients at the 200-bed hospital from February through April, a time that coincides with the Department of Health and Human Services' mass firings of government employees, the gutting of scientific research, and the administration's broad crackdown on immigration. The average number of patients being treated daily during that time hovered between 60 and 80, with the April numbers falling to the lower end of that range. By contrast, in October, about 80 patients per day on average were at the hospital. The number of cancer clinical trial participants at the hospital as of July was down about 20% from last year, one NIH cancer scientist said. KFF Health News agreed not to identify the scientist and others who participated in this article who were not authorized to speak to the press and feared retaliation. The numbers "really don't look too good," Pius Aiyelawo, acting CEO of the clinical center, said during a May 23 meeting of the NIH Clinical Center Research Hospital Board. As of April 30, the average number of patients in the hospital per day had declined by 5.7% compared with the same period a year ago. Adults and children with cancer, people who need bone marrow transplants, and people with rare diseases or infections are among the patients who receive care at no charge at the NIH hospital, according to former officials. Clinicians there provide potentially lifesaving treatments as part of clinical trials, often to people who have run out of options. Research at the hospital has also led to breakthroughs about cancer, traumatic brain injury, and AIDS, among other ailments. James Gilman, a physician who was CEO of the clinical center from 2017 until retiring in January, said the center has driven important advances against disease "that couldn't have happened anywhere else." Former officials said the drop in patients this year is a consequence of the upheaval the Trump administration has caused at the NIH, the world's largest public funder of scientific research. Current and former employees say an exodus of clinicians, scientists, and other staffers has limited how many patients can be treated. Morale has tanked because of widespread firings and the administration's cancellation of grants that funded research into health disparities, vaccines, the health of LGBTQ+ people, and more. Contracts have been cut, and scientists have seen delays in getting essential supplies for clinical research. "Every day seems to be some type of breaking point," one NIH worker said. During the May board meeting, a video of which KFF Health News viewed, Aiyelawo attributed the decrease in patients coming to the hospital to the departure of NIH investigators — the researchers on studies — and less patient recruitment. He also noted 11 recent departures of clinical center staffers. They included Christine Grady, a nurse who led the center's bioethics department and the wife of Anthony Fauci, the former head of the NIH's infectious diseases institute who became a lightning rod for conservatives during the COVID-19 pandemic. HHS has fired more than 1,200 NIH employees this year as part of its purge of the federal workforce, but the true number of departures is almost certainly higher. Others have opted for early retirement or quit because they opposed the Trump administration's orders. Gilman said the NIH hospital relies on a "very complex ecosystem and network to find patients who are not too sick" to potentially be enrolled in a clinical trial. When researchers leave, "those patients are lost," he said. The clinical center's 2025 annual report said there were roughly 1,500 research studies underway in 2024, including studies focused on cancer, infectious disease, heart and lung conditions, and blood disorders. Clinical trials accounted for about half. The National Cancer Institute — which is the largest of the NIH's 27 institutes and has been crippled by cuts and chaos this year — typically has the most patients needing inpatient care, Gilman said. "What has happened here since January has been a pretty traumatic time for that ecosystem," he said, "and there are pieces of it that will take a long time to rebuild, if indeed they get a chance to rebuild." During the May board meeting, Aiyelawo said NIH Director Jay Bhattacharya "is very aware" that fewer people are getting treated at the hospital "and we're doing everything we can to be able to get those numbers up." The drop in patients this year isn't isolated to people needing inpatient care, NIH documents show. As of the end of April, outpatient visits were down 8.5% compared with the same period in the prior fiscal year. The number of new patients overall had declined by 6.7%, to about 3,370 people. In response to questions, HHS spokesperson Andrew Nixon wrote in an emailed statement that the clinical center "remains fully operational and continues to provide world-class clinical research and patient care. Every day, patients from across the country and around the globe come here to participate in cutting-edge studies that drive scientific discovery and improve health outcomes." "As the crown jewel of research and discovery, the Clinical Center is a top priority" under Bhattacharya's leadership, Nixon said. "We are committed to fully leveraging its capabilities as the nation's hub for clinical research innovation. Our focus remains on empowering the research community and advancing the critical mission of making medical breakthroughs possible right here on the NIH campus." Even before President Trump began his second term, the hospital had struggled with lagging patient numbers. Before the pandemic, it averaged more than 110 patients daily. Those numbers plummeted starting in 2020, government documents show. During the 2022 fiscal year, there were about 73 patients, on average, in the hospital per day. While yearly figures have increased since then, they have not gone back to pre-pandemic levels. NIH documents show that the hospital saw an average of roughly 81 patients per day during fiscal 2024, which ended in September. Still, one NIH worker said: "This is a manufactured crisis. COVID was not." The federal government has also moved to tighten rules surrounding visitors from abroad, which likely limits how many people living in the U.S. without legal status would come to the NIH for care. Before Mr. Trump, officials developed a new visitor policy for the NIH that required people who aren't U.S. citizens or legal permanent residents to register online before arriving. But its implementation was delayed, Gilman said. It did not launch until late January, after President Joe Biden was no longer in office and around the time the Trump administration began its deportation operation. The Department of Homeland Security has carried out widespread raids and arrests and allowed immigration authorities unprecedented access to various federal data sources — including tax information and Medicaid recipients' personal data — as part of its immigration enforcement efforts. The clinical center's most recent annual report said around 600 patients in 2024 were from abroad. Now "international patients are terrified to come," said one recently departed clinician. "They don't know what will happen to them." KFF Health News is a national newsroom that produces in-depth journalism about health issues and is one of the core operating programs at KFF — the independent source for health policy research, polling, and journalism.
Medscape
2 hours ago
- Medscape
Meta-Analysis Finds Biologic Switches Effective in Psoriasis
TOPLINE: Interclass biologic switching is effective and safe in patients with psoriasis, though switching from an anti-tumor necrosis factor (anti-TNF)-alpha to an anti-interleukin (IL)-17A treatment was associated with higher risk for adverse events (AEs), according to a meta-analysis. METHODOLOGY: To evaluate the safety and effectiveness of switching treatments after an initial biologic treatment fails, researchers conducted a meta-analysis of 24 randomized clinical trials published through January 25, 2025, which included 12,661 adults with psoriasis who switched from one biologic agent to another within the same class or in a different class. Eight switching categories were analyzed. The primary endpoint was the Psoriasis Area and Severity Index (PASI) 90 score, and secondary endpoints included safety. TAKEAWAY: PASI 90 improved significantly in patients after interclass biologic switching both at week 4 (11 studies; odds ratio [OR], 6.53; 95% CI, 2.58-16.51) and long term (OR, 28.61; 95% CI, 12.89-63.47). All switches were effective in the short term, whereas most switches achieved a PASI 90 response in the long term, except for switches from anti-IL-17A agents to anti-IL-17A/F agents. Long-term, marked improvements were observed when switching from anti-TNF-alpha agents to anti-IL-23p19 agents (OR, 23.72; 95% CI, 4.29-130.98) and from anti-IL-12/23p40 agents to anti-IL-23p19 agents (OR, 19.87; 95% CI, 10.40-37.94). No major safety differences were observed overall, except for increased serious adverse events (AEs) when switching from an anti-TNF-alpha agent to an anti-IL-17A agent (OR, 2.45; 95% CI, 1.25-4.83). Switching from anti-TNF-alpha agents to anti-IL-23p19, anti-IL-17A, or anti-IL-12/23p40 agents was associated with infection rates of 0.62%, 0.54%, and 0.39%, respectively. The highest risk for Candida infection (0.16%) was observed when switching from anti-TNF-alpha agents to anti-IL-17A/F agents. Switching to a different biologic class showed comparable effectiveness and safety with continuing the same agent, with regards to AEs. IN PRACTICE: This systematic review and meta-analysis found that 'interclass biologic switching was effective, and there were no safety differences for most patients,' the study authors wrote. 'Switching to anti-IL-23p19, anti-IL-17A, or anti-IL-12/23p40 agents from anti-TNF-alpha agents posed the greatest risk of infection,' they added, recommending 'vigilance for infections while switching to different biologics.' SOURCE: The study was led by Miao Zhang, MD, Department of Dermatology, Yueyang Hospital of Integrated Traditional Chinese and Western Medicine, Shanghai University of Traditional Chinese Medicine, Shanghai, China, and was published online on August 6 in JAMA Dermatology. LIMITATIONS: Limitations included heterogeneity in study designs, potential differences between biologics and batch variability which could have affected effectiveness comparisons after switching, insufficient data for several comparisons. DISCLOSURES: The study was supported by the National Natural Science Foundation of China, the Key Discipline Construction Project of Shanghai's 3-Year Action Plan for Strengthening the Construction of Public Health System, Shanghai Oriental Talent Program for Top-notch Project, CACMS Innovation Fund, Shanghai Healthy Special Project, The Shanghai 2022 Science and Technology Innovation Action Plan Medical Innovation Research Special Project, the Clinical Research Plan of Shanghai Shenkang Hospital Development Center, the High-level Chinese Medicine Key Discipline Construction Project, Evidence-based dermatology base sponsored by State Administration of Traditional Chinese Medicine, and the Shanghai Hospital Development Center Foundation. The authors reported having no conflicts of interest. This article was created using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication.
CNN
7 hours ago
- CNN
What you should know about metabolism, according to a nutrition scientist
Food & healthFacebookTweetLink Follow EDITOR'S NOTE: The podcast Chasing Life With Dr. Sanjay Gupta explores the medical science behind some of life's mysteries big and small. You can listen to episodes here. (CNN)— Think speeding up your metabolism is key to losing weight? Metabolism has grown into one of the biggest buzzwords in the wellness industry — a tricky code that, if deciphered, is believed to unlock the secrets of weight loss and overall health. Despite the term's ubiquity, one scientist says many people don't understand much of how this basic bodily process works. 'They think it has something to do with how much food we can eat without gaining weight or something like that,' Dr. Kevin Hall told CNN Chief Medical Correspondent Dr. Sanjay Gupta recently on his podcast, Chasing Life. Hall wants to elevate metabolism for different reasons. 'Metabolism is just this incredible biochemical process that basically turns the food that we eat and the oxygen that we breathe into essentially … everything we are and everything we do,' he said. A leading nutrition and metabolism scientist, Hall is well-known for his research on contestants from the reality show 'The Biggest Loser' that helped explain what set participants who kept the weight off apart from those who gained it back. He spent more than two decades at the National Institutes of Health. His later work on ultraprocessed foods investigated its link to obesity, including one study that demonstrated it actively caused participants to overeat. Hall announced his early retirement and exit from NIH in April, citing censorship of how his research findings were communicated. He's now coauthor of the new book 'Food Intelligence: The Science of How Food Both Nourishes and Harms Us' alongside journalist Julia Belluz. Metabolism and weight loss enter conversations arm in arm, but this, according to Hall, can be reductive. 'I just find it incredibly frustrating sometimes that this idea of metabolism, this really beautiful physiological process that is so fundamental to life, is kind of talked about as, 'Oh well, if you take this supplement, you can boost your metabolism and lose weight,'' he explained to Gupta. You can listen to the full episode here. Is a faster or higher metabolism really the highway to weight loss? Hall is here to set the record straight, highlighting three misconceptions about metabolism that show there is more to it than meets the eye. One common belief is that a slower metabolism results in a higher body weight. But mostly, Hall said, the opposite is true. 'Generally, larger people have faster metabolisms than smaller people,' he told Gupta. Hall attributed the pervasiveness of this myth to the way researchers designed early metabolism studies. Scientists initially tried to match up participants' caloric intake to their weight, but they didn't account for the fact that these calorie counts were self-reported. It turned out that those with obesity often underreported the number of calories they ate to a larger degree than their leaner counterparts. This, Hall said, 'led people to a conclusion that, well, if they're eating less calories and they're maintaining their weight, then they must be burning less calories. And maybe the reason why they have obesity is because they have slow metabolisms.' Modern technology now allows researchers to rely on data, not participants' own reports. 'When we actually directly measure people's metabolisms, people with obesity have higher metabolic rates on average than people who are lean,' Hall noted. But the myth that we should be boosting metabolism to treat obesity has stubbornly persisted — and has at times led to deadly consequences. When Stanford University researchers discovered that the chemical compound 2,4-dinitrophenol, or DNP, increased metabolism levels in the 1930s, they enthusiastically promoted it as a safe and effective tool for treating obesity. While DNP did boost metabolism, it also led to side effects that included blindness and death, causing federal drug regulators to quickly withdraw it from the market. 'Maybe,' Hall suggested, 'it shouldn't be too much of a surprise later to realize that with something so fundamentally important to life as metabolism, you can't just turn it up and turn it down with a pharmaceutical drug and not expect some pretty severe side effects, including death.' Like weight, age might not affect metabolism as much as you may assume. 'It turns out that until you get to very advanced ages, we're talking 70s and 80s, it looks like our metabolic rate is roughly constant,' Hall said. There are, of course, changes in the aging body that can impact metabolic rates. People 'past the age of 30 or so,' Hall said, are 'tending to lose muscle mass or tending to lose lean mass, and they're tending to gain fat mass. And that alone is anticipated to result in a lower metabolism.' That's because lean muscle burns more calories than fat. But it's these age-related changes — that are not inevitable — and not age itself, that influence one's metabolism. 'Once you get rid of that effect … it doesn't seem like there's a fundamental aging effect to slow metabolism as, as we get older,' Hall concluded. A couple ways to combat the loss of lean muscle mass as you age is by engaging in twice-a-week strength training and getting enough protein in your diet (but no need to go overboard). Another pillar of metabolism mythology is the idea that a slow metabolism is the enemy of continued weight loss. Interventions such as dieting are believed to slow metabolism to the point that you can't lose any additional weight. But keeping your metabolism from slowing down isn't the key to sustained weight loss, Hall said. In fact, it's just the opposite. 'The people who are most successful at losing weight and keeping it off are the ones who sport the slowest metabolisms or the greatest reductions in metabolism,' he said. 'It's kind of like stretching a spring, right?' The more intensely someone diets or exercises, the more weight they'll lose and the more their metabolism will slow down, he explained in his book. Hall's research showed that a slower metabolism 'didn't seem to determine anyone's ability to lose weight or keep it off in the short or long run.' By uncoupling metabolism from the weight loss conversation, Hall said he hopes everyone can appreciate the phenomenon for what it really is. Misinformation about metabolism 'has really distracted people, I think, from the real beauty of what this is,' he told Gupta. 'It's harnessing the continuous flow of matter and energy in our food and in our breath and powering every cell in our body, as well as the bodies and cells of practically every organism that we have ever encountered,' Hall said. 'It's a fundamental component of life, and it's just incredibly fascinating.' We hope these three insights help you understand your metabolism better. Listen to the full episode here. And join us next week for a new episode of Chasing Life.
