Poxel Announces Positive Preclinical Data for PXL065 in Hypertrophic Cardiomyopathy To Be Presented at the ESC Congress 2025
PXL065 demonstrated significant benefits in a HCM mouse model preventing pathological myocardial remodeling, including hypertrophy and fibrosis in the heart
Presentation of detailed results from preclinical study on Sept 1st, 2025, 11:15 am CEST, at the European Society of Cardiology 2025
Findings support the clinical development of PXL065 as a potential disease- modifying treatment for symptomatic and asymptomatic HCM patients
LYON, France, May 26, 2025--(BUSINESS WIRE)--Regulatory News:
POXEL SA (Euronext : POXEL - FR0012432516), a clinical stage biopharmaceutical company developing innovative treatments for chronic serious diseases with metabolic pathophysiology, including metabolic dysfunction-associated steatohepatitis (MASH) and rare metabolic disorders, today announces that the abstract featuring previously announced preclinical data demonstrating positive results for PXL065, the deuterium-stabilized R-enantiomer of pioglitazone, in hypertrophic cardiomyopathy1, has been accepted for presentation at the 2025 edition of the European Society of Cardiology (ESC) Congress (link), to be held jointly with World Congress of Cardiology, on September 1st, 2025 at 11:15 am CEST, in Madrid, Spain.
Prof. Dr. Cordula Wolf, Director of the Center for Rare Congenital Heart Diseases at the TUM University Hospital German Heart Center, stated: "The compelling results obtained in this study illustrate the potential of PXL065 in HCM, the most common genetic cardiac disorder. Current treatments have important limitations in efficacy, safety, or patient applicability. There is a clear unmet need for safe and effective disease-modifying therapies."
Thomas Kuhn, CEO of Poxel, added: "We are pleased to have the data with PXL065 in HCM be presented at one of the world's leading forums for cardiovascular science and medicine, which underscores both the quality and relevance of these findings. We look forward to further supporting PXL065 development for the treatment of HCM based on these promising results."
Hypertrophic Cardiomyopathy (HCM) is a genetic disorder marked by myocardial hypertrophy, cardiac fibrosis, ventricular dysfunction, arrhythmias, and an increased risk of sudden cardiac death. It is caused by mutations in sarcomere protein genes, leading to altered cell metabolism, including oxidative stress and mitochondrial dysfunction. The estimated prevalence of HCM is 0.2%, or 1/500 adults, and its incidence is around 5 per 100,000 person-years.
In connection with the mechanism of action of PXL065 on the inhibition of the mitochondrial pyruvate carrier (MPC) and on the inhibition of Acyl CoA Synthetase 4 (ACSL4) thus acting on oxidative stress, inflammation and fibrosis, PXL065 was successfully assessed in an established mouse model of hypertrophic cardiomyopathy.
This preclinical study was funded by the German Center for Cardiovascular Research (DZHK) and conducted at the TUM University Hospital German Heart Center by leading HCM expert Prof. Dr. Cordula Wolf. Poxel and the TUM University Hospital German Heart Center collaborated on the pre-clinical study based on Poxel's existing data and patent portfolio on PXL065 and prior research conducted by Prof. Dr. Cordula Wolf and her group on the disease mechanisms and therapeutic use of TZD's in HCM.
About Poxel SA
Poxel is a clinical stage biopharmaceutical company developing innovative treatments for chronic serious diseases with metabolic pathophysiology, including metabolic dysfunction-associated steatohepatitis (MASH) and rare disorders. For the treatment of MASH, PXL065 (deuterium-stabilized R-pioglitazone) met its primary endpoint in a streamlined Phase 2 trial (DESTINY-1). In rare diseases, development of PXL770, a first-in-class direct adenosine monophosphate-activated protein kinase (AMPK) activator, is focused on the treatment of adrenoleukodystrophy (ALD) and autosomal dominant polycystic kidney disease (ADPKD). TWYMEEG® (Imeglimin), Poxel's first-in-class product that targets mitochondrial dysfunction, is now marketed for the treatment of type 2 diabetes in Japan by Sumitomo Pharma and Poxel expects to receive royalties and sales-based payments. Poxel has a strategic partnership with Sumitomo Pharma for Imeglimin in Japan. Listed on Euronext Paris, Poxel is headquartered in Lyon, France, and has subsidiaries in Boston, MA, and Tokyo, Japan.
For more information, please visit: www.poxelpharma.com
All statements other than statements of historical fact included in this press release about future events are subject to (i) change without notice and (ii) factors beyond the Company's control. These statements may include, without limitation, any statements preceded by, followed by or including words such as "target," "believe," "expect," "aim," "intend," "may," "anticipate," "estimate," "plan," "project," "will," "can have," "likely," "should," "would," "could" and other words and terms of similar meaning or the negative thereof. Forward-looking statements are subject to inherent risks and uncertainties beyond the Company's control that could cause the Company's actual results or performance to be materially different from the expected results or performance expressed or implied by such forward-looking statements. The Company does not endorse or is not otherwise responsible for the content of external hyperlinks referred to in this press release.
1 Press release as of March 20, 2025
View source version on businesswire.com: https://www.businesswire.com/news/home/20250526341087/en/
Contacts
Investor relations / Media NewCapAurélie Manavarere, Théo Martin / Arthur Rouilléinvestors@poxelpharma.com +33 1 44 71 94 94
Hashtags
Try Our AI Features
Explore what Daily8 AI can do for you:
Comments
No comments yet...
Related Articles
Medscape
3 hours ago
- Medscape
Artificial Blood: Sci-Fi Breakthrough or False Hope?
Japanese researchers have developed artificial blood, marking a significant step in this long-standing scientific pursuit. However, large-scale production remains a challenge. In France, the national blood service Établissement français du sang regularly raises alarms over critically low blood supplies, with daily donations falling short of the 10,000 units needed to meet transfusion demands. This challenge is further complicated by ABO/Rh compatibility issues, prompting targeted appeals for rare blood-type donors. This shortage is not unique to France. It is a global concern, often even more severe elsewhere, driving continued efforts to create viable artificial alternatives. Vesicle Trials Researchers at Nara University in Nara, Japan have created 'hemoglobin vesicles' (HbVs): Haemoglobin encapsulated within a lipid shell that mimics red blood cells. In a 2022 trial, investigators injected 100 mL of HbVs into 12 healthy volunteers, with no serious adverse events reported. The team plans to administer up to 400 mL to a larger cohort. If safety is confirmed, future studies will assess haemodynamic and respiratory effectiveness. Investigators hope to achieve industrial production and clinical use by 2030. Storage Benefits Artificial blood offers two key advantages over donor blood. First, it can be stored at room temperature for up to 2 years or up to 5 years if frozen, whereas donated blood expires after 6 weeks. Second, HbVs lack ABO and Rh antigens, making them theoretically compatible with all recipients. Notably, the solution turned purple. Failed Attempts The quest for artificial blood holds great promise, as tens of thousands die annually from blood shortages, particularly in low-income countries. However, previous efforts have been repeatedly stalled. In 2011, Luc Douay, MD, PhD, senior study author and Professor of Haematology at Université Pierre et Marie Curie in Paris, France, performed France's first transfusion using stem cell-derived red cells. In Japan, military physicians transfused experimental blood into rabbits in 2019 and reported encouraging results from stem cell-derived erythrocyte injections in the United Kingdom in 2022. 'All attempts have failed so far,' Douay said in 2017. While the latest Japanese trials offer renewed hope, experts emphasise the need to expand natural blood donation programs.
Yahoo
3 hours ago
- Yahoo
Workplace Injuries Are Reduced with Employee Appreciation
Research finds that employee recognition can decrease workplace injuries among French workers by up to 50% LOUGHTON, England, June 10, 2025--(BUSINESS WIRE)--Latest research reveals that frequently recognising employees for their efforts and achievements can decrease the likelihood of workplace injuries among French workers by up to 50%. This is a key finding from O.C. Tanner's 2025 Global Culture Report which gathered data and insights from 38,075 workers from 27 countries including 1,208 from France. The report highlights how feeling unappreciated worsens mental health and this then increases the likelihood of workplace accidents. Robert Ordever, European MD of O.C. Tanner explains: "There's a clear link between deteriorating mental health and greater instances of workplace injuries, with mental illness often leading to decreased concentration, reduced cognitive function, and impaired decision-making abilities. Workers struggling with their mental health have their condition worsened when they feel unappreciated by their managers and colleagues." The Report highlights how regularly recognising employees for their efforts and achievements, not only improves workers' wellbeing, but noticeably reduces the odds of them reporting a workplace accident. Employees suffering from burnout are 50% less likely to have a workplace accident when their organisation has a formal recognition programme in place. For those workers with probable depression, their odds of reporting a workplace accident are 11% when their organisation is WITHOUT a recognition programme. For workers with probable depression but WITH a recognition programme in place, their odds of reporting a workplace accident are reduced to just 8%. Ordever says, "Implementing a recognition programme that allows all employees to regularly express gratitude to colleagues, not only reduces the likelihood of burnout, anxiety, and depression, but also cuts the number of workplace injuries. But it's important to note that having a recognition programme is not enough in itself. The organisation must champion the giving of appreciation on a daily basis, and encourage recognition that's sincere, tailored to the individual and meaningful. When the importance of recognition is understood and prioiritised, organisations can enjoy truly impactful business outcomes." About 2025 Global Culture Report The O.C. Tanner Institute uses multiple research methods to support the Global Culture Report, including interviews, focus groups, cross-sectional surveys, and a longitudinal survey. Qualitative findings came from 27 focus groups among employees, leaders, and HR practitioners of larger organisations. About O.C. Tanner O.C. Tanner is the global leader in personalised employee recognition solutions that improve workplace cultures so people feel appreciated, do their best work, and stay. Its Culture Cloud is a suite of apps and solutions, including recognition, service awards, wellbeing, leadership, and celebrations that help people thrive at work. O.C. Tanner provides these and other services for thousands of the most respected companies in the world. For more information visit View source version on Contacts Liz Boote, Loudhailer PR E: T: +44(0)7795 362727 Sign in to access your portfolio
Washington Post
6 hours ago
- Washington Post
High Seas Treaty gains momentum as 18 new countries pledge support
NICE, France — Eighteen countries ratified the High Seas Treaty on Monday, bringing the total to 49 — just 11 short of the 60 needed for the ocean agreement to enter into force. The surge in support, occurring during the U.N. Ocean Conference in Nice, France, adds momentum to what could become a historic shift in how the world governs the open ocean. Here's what the treaty is, why it matters and what happens next. Formally known as the Agreement on Biodiversity Beyond National Jurisdiction, the High Seas Treaty is the first legally binding agreement focused on protecting marine biodiversity in international waters. These waters, which are beyond the jurisdiction of any single country, make up nearly two-thirds of the ocean and almost half the surface of the planet. Until now, there has been no comprehensive legal framework to create marine protected areas or enforce conservation on the high seas. Despite their remoteness, the high seas are under growing pressure from overfishing, climate change and the threat of deep-sea mining . Environmental advocates warn that without proper protections, marine ecosystems in international waters face irreversible harm. 'Until now, it has been the wild west on the high seas,' said Megan Randles, global political lead for oceans at Greenpeace. 'Now we have a chance to properly put protections in place.' The treaty is also essential to achieving the global '30x30' target — an international pledge to protect 30% of the planet's land and sea by 2030. The treaty creates a legal process for countries to establish marine protected areas in the high seas, including rules for destructive activities like deep-sea mining and geo-engineering. It also establishes a framework for technology-sharing, funding mechanisms and scientific collaboration among countries. Crucially, decisions under the treaty will be made multilaterally through conferences of parties (COPs) rather than by individual countries acting alone. Once 60 countries ratify the treaty, a 120-day countdown begins before it officially enters into force. That would unlock the ability to begin designating protected areas in the high seas and put oversight mechanisms into motion. As of Monday evening, 49 countries and the EU had ratified, meaning 11 more are needed to trigger that countdown. The first Conference of the Parties (COP1) must take place within one year of the treaty's entry into force. That meeting will lay the groundwork for implementation, including decisions on governance, financing and the creation of key bodies to evaluate marine protection proposals. Environmental groups are pushing to surpass the required 60 ratifications, and to do so quickly – the more countries that ratify, the stronger and more representative the treaty's implementation will be. There's also a deadline: only countries that ratify by COP1 will be eligible to vote on critical decisions that determine how the treaty will operate. 'To reach 60 ratifications would be an absolutely enormous achievement, but for the treaty to be as effective as possible, we need countries from all over the world to engage in its implementation,' said Rebecca Hubbard, director of the High Seas Alliance. 'So the next step will be to go from 60 to global.' The surge in support on Monday has raised hopes that 2025 could mark a turning point for high seas protection. 'We're on the brink of making high seas history,' Hubbard said. ___ Follow Annika Hammerschlag on Instagram @ahammergram ___ The Associated Press receives support from the Walton Family Foundation for coverage of water and environmental policy. The AP is solely responsible for all content. For all of AP's environmental coverage, visit
