Poxel Announces Positive Preclinical Data for PXL065 in Hypertrophic Cardiomyopathy To Be Presented at the ESC Congress 2025
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
Growth in Milk Banks Strengthens Neonatal Nutrition
In 2023, Spain recorded 320,656 births, of which 21,322 were premature, according to the National Statistics Institute. That figure represents 6.6% of all births, meaning that 1 in every 15 newborns is premature and requires specialized care, with nutrition playing a critical role. Human milk banks (HMBs) have become the cornerstone of neonatal care in Spain. The first facility opened in 2001 in Palma de Mallorca, followed by a second in 2007 at the 12 de Octubre University Hospital in Madrid, Spain. The network has since expanded to 22 centers across Spain, operating within blood and tissue banks, neonatology departments, and personalized nutrition units. A 2023 report from the Spanish Association of Human Milk Banks reported record growth in Spain, with several key measures, including the highest number of donors to date. A total of 2657 donors were accepted, the highest number since the establishment of the HMB network. Catalonia registered 737 donors, followed by Madrid with 335 donors and the Valencian Community in Spain with 224 donors. In total, 13,745 liters of milk were donated, with 11,781 liters pasteurized and 9949 liters distributed to hospitals, providing nutritional support for 4290 infants in 83 centers in Spain. The activities of HMBs in Spain have shown continuous and sustained growth since 2009. The number of accepted donors increased from 175 in 2009 to 2657 in 2023, reflecting a progressive increase in public awareness and participation in this type of altruistic donation. The volume of donated milk has also increased from 1144 liters in 2009 to 13,745 liters in 2023. The volume of milk distributed grew more than 10-fold over this period, rising from 774 liters in 2009 to 9949 liters, reflecting the expansion of HMBs' processing and distribution capacity. Consequently, the number of infant recipients grew from 333 in 2009 to 4290 in 2023. This increase underscores the vital and expanding clinical role of donor human milk, particularly in premature infants and those with specific medical needs. Clinical Benefits For premature infants or those with medical conditions, the World Health Organization, United Nations Children's Fund, Spanish Society of Neonatology, and Spanish Association of Pediatrics recommend donor human milk when the mother's milk is unavailable. Donor human milk provides protection against serious or potentially fatal illnesses and supports healthy growth and development. 'Human milk is now regarded as a true medicine,' said Carmen Pallás-Alonso, MD, PhD, head of Neonatology at Hospital 12 de Octubre University Hospital, and the director of the milk bank. Scientific evidence has shown that it reduces the risk of necrotizing enterocolitis, infection, and digestive disorders. Its benefits extend over the long term, supporting neurodevelopment and lowering cardiovascular risk in adulthood, including its positive effects on blood pressure and lipid profiles. Donor Selection Donors are healthy lactating women who breastfeed their own children and altruistically choose to donate excess milk to help sick or premature infants. Selection involves a personal interview to assess health and lifestyle, as well as serologic testing for HIV, syphilis, human T-lymphotropic virus, hepatitis B and C, and Chagas disease. Although donation can begin at any stage of lactation, it is recommended to wait 3-8 weeks postpartum to ensure that breastfeeding is well-established. Milk Processing At HMBs, milk donated by selected mothers is received, processed, analyzed, stored, and distributed to hospitals to feed newborns who cannot receive their mothers' milk. The collected milk undergoes rigorous safety and quality control. First, microbiological analysis detects any contamination, followed by macronutrient testing, including energy, fat, protein, and lactose, allowing healthcare professionals to match the nutritional content of milk to the requirements of each infant. Milk is then pasteurized using the Holder method (62.5 °C for 30 minutes) to eliminate pathogens, before being cooled, frozen, and stored until distribution. Online Milk Market Although uncommon in Spain, commercial online markets for human milk exist in countries such as the US and Canada. The Spanish Association of Pediatrics warns that this practice carries serious health risks because donor screening and processing cannot be verified. A study published in Pediatrics found that approximately 11% of human milk samples purchased online were contaminated with cow's milk. Laboratory analysis indicated that, in most cases, the level of contamination was sufficiently high to suggest deliberate adulteration rather than accidental exposure. Research and Global Context Associations such as the Spanish Association of Human Milk Banks, established in 2008 in Palma de Mallorca, Spain, and the European Milk Bank Association (EMBA), founded in Milan, Italy, in 2010, have been key to promoting safe and effective HMB operations through standardized protocols. These organizations foster international collaboration and help establish standardized protocols to ensure the quality, traceability, and safety of donated milk. The online market for human donor milk has expanded. This practice presents documented health risks because it operates without standard controls for donor screening, handling milk, or storage. These risks highlight the importance of stringent regulatory standards for milk distribution. The EMBA's Consensus Statement outlines over 100 evidence-based recommendations for milk banking, from donor selection to distribution, highlighting the importance of rigorous oversight of donor selection, heat treatment, storage, and distribution. These protocols not only safeguard vulnerable newborns but also preserve the beneficial properties of breast milk under controlled conditions, reinforcing its role as a therapeutic tool in hospitals. Breast milk is essential for newborns; however, recent studies have suggested that it may also help detect breast cancer in mothers months before routine diagnosis. In 2024, the Maimonides Institute of Biomedical Research of Córdoba (IMIBIC) in Córdoba, Spain, launched the Hera Project, led by Juan de la Haba-Rodríguez, MD, a medical oncologist and researcher at IMIBIC and Reina Sofía University Hospital, and Silvia Guil-Luna, PhD, a postdoctoral researcher affiliated with IMIBIC, Reina Sofía Hospital, and the University of Córdoba in Córdoba, Spain. The study identified postpartum breast cancer cases using biomarkers in breast milk. The results were presented at the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting in Chicago. Researchers at the Vall d'Hebron Institute of Oncology, part of the Vall d'Hebron Campus in Barcelona, Spain, have found that breast milk from patients diagnosed with breast cancer during pregnancy or postpartum contains circulating tumor DNA (ctDNA). This ctDNA can be detected through liquid biopsy of breast milk and could become a new tool for the early diagnosis of breast cancer in the postpartum period. The results of this study were published in Cancer Discovery , which reported the first detection of cell-free tumor DNA in breast milk from patients with breast cancer. Using droplet digital polymerase chain reaction, researchers detected tumor variants in 87% of breast milk samples, whereas 92% of matched blood plasma samples showed no variants. This indicated that breast milk may be a more effective medium for tumor DNA analysis. Next-generation sequencing confirmed these results, showing 71.4% sensitivity and 100% specificity. In two cases, tumor DNA was detected in breast milk collected 18 and 6 months before routine clinical diagnosis, highlighting its potential for earlier breast cancer detection.
Yahoo
5 hours ago
- Yahoo
Nicox Announces Positive Results from the NCX 470 Phase 3 Denali Trial in Glaucoma Patients
Press Release Nicox Announces Positive Results from the NCX 470 Phase 3 Denali Trial in Glaucoma Patients Achieving the primary endpoint in both Denali and the first NCX 470 Phase 3 trial, Mont Blanc, meets efficacy requirements for approval in the U.S. and China NCX 470 was also superior to latanoprost in the reduction of intraocular pressure (IOP) from baseline (p<0.05) at 3 out of 6 timepoints and numerically greater at 5 out of 6 timepoints NCX 470 0.1% was safe and well tolerated New Drug Application (NDA) submission in the U.S. currently expected in H1 2026 August 21, 2025 – release at 7:30 am CETSophia Antipolis, FranceNicox SA (Euronext Growth Paris: FR0013018124, ALCOX), an international ophthalmology company, today announced that once daily dosing of NCX 470 0.1%, a novel nitric oxide (NO)-donating bimatoprost eye drop, met the primary objective of non-inferiority in lowering intraocular pressure (IOP) from baseline compared to the standard of care, latanoprost 0.005%, in the Denali Phase 3 clinical trial involving 696 patients with open-angle glaucoma or ocular hypertension. The IOP-lowering effect from baseline for NCX 470 was 7.9 to 10.0 mmHg vs. 7.1 to 9.8 mmHg for latanoprost (reduction in time-matched IOP at 8 AM and 4 PM across the week 2, week 6 and month 3 visits). In a pre-specified secondary efficacy analysis of time-matched change from baseline IOP, the IOP reductions for NCX 470 0.1% were numerically greater than those for latanoprost at 5 of the 6 timepoints, and statistically significant (p<0.05) at 3 of the 6 timepoints, however overall statistical superiority was not achieved. These results are consistent with those of Mont Blanc, the first Phase 3 trial, successfully completed in 2022, and confirm the efficacy profile required for regulatory submissions in the U.S. and China. 'The Denali trial results validate the robust therapeutic profile of NCX 470 observed in the Mont Blanc trial, replicating the efficacy and safety outcomes. Consistent results across the two large Phase 3 studies strengthen the case for regulatory submissions and support NCX 470's potential as an important treatment option for glaucoma patients. I look forward to seeing further data presented at future ophthalmology meetings.' said Don Budenz, Coordinating Investigator of the Denali 470 Glaucoma Denali Phase 3 Topline Results Summary Non-inferiority was met vs. latanoprost 0.005% in the primary efficacy analysis. IOP-lowering effect from baseline was 7.9 to 10.0 mmHg for NCX 470 0.1% vs. 7.1 to 9.8 mmHg for latanoprost 0.005% (reduction in time-matched IOP at 8 AM and 4 PM across the week 2, week 6 and month 3 visits). In a pre-specified secondary efficacy analysis of time-matched change from baseline IOP, NCX 470 0.1% was statistically superior (p<0.05) to latanoprost 0.005% in intraocular pressure reduction from baseline at 3 of the 6 timepoints, and numerically greater at 5 out of 6 timepoints but did not reach the overall statistical superiority pre-specified as a secondary efficacy endpoint. The difference in IOP reduction between NCX 470 0.1% and latanoprost 0.005% was up to 0.8 mmHg in favor of NCX 470. NCX 470 0.1% was well tolerated; the most common adverse event was conjunctival hyperaemia in 22.0% of the NCX 470 0.1% patients vs. 9.2% of latanoprost 0.005% patients. In the NCX 470 0.1% patients, there were no ocular serious adverse events and no treatment-related non-ocular serious adverse events. 10.1% of patients on NCX 470 0.1% discontinued compared to 6.6% on latanoprost 0.005%, which includes patients out to 12 months. The long-term safety profile is consistent with that seen in the shorter, 3-month, Mont Blanc trial. 'With a second Phase 3 trial demonstrating the necessary efficacy and safety profile for submission of New Drug Applications – NDAs – in both the United States and China, we are now moving forward towards potential recurrent NCX 470 royalty revenue from 2027 onwards. Our established partnerships with Kowa and Ocumension enable us to accelerate NDA preparations, whilst also allowing us to focus on potential future strategies for the company.' said Gavin Spencer, Chief Executive Officer of Company plans to publish further data at upcoming ophthalmology industry events and is also planning a pre-NDA meeting with the FDA, in collaboration with Kowa.A presentation of the results of the Denali trial is available on our website, Denali Trial DesignSimilar to Mont Blanc, Denali is a randomized, multi-regional, double-masked, parallel group trial that evaluated the safety and efficacy of NCX 470 ophthalmic solution, 0.1% compared to latanoprost ophthalmic solution, 0.005% in 696 patients in 90 sites in the U.S. and China. Latanoprost is the most widely prescribed first-line therapy for open-angle glaucoma or ocular hypertension. The primary efficacy evaluation was based on reduction from baseline in mean time-matched IOP at 6 timepoints: 8 AM and 4 PM at week 2, week 6 and month 3. The Denali trial also included a long term safety extension through to 12 months and was jointly conducted and equally financed with our Chinese partner, Ocumension Therapeutics. NCX 470 Licensing Deals Based on these results, Nicox will receive a milestone payment of €5 million from Kowa. Royalties on net sales in the U.S. will start at 8% with royalties also payable in other territories of the July 2025 agreement with Kowa. The total potential milestones payable under the agreement are valued at €127 million and full details can be found in the Press Release issued on 17 July 2025. Separate milestone payments and royalties are due under the Kowa NCX 470 agreement for has co-funded the Denali trial and Nicox will receive royalties of 6% to 12% on net sales of NCX 470 in the Chinese, Korean and Southeast Asian NCX 470NCX 470, Nicox's lead clinical product candidate, is a novel NO-donating bimatoprost eye drop being developed for the lowering of IOP in patients with open-angle glaucoma or ocular hypertension. NDA filings are in preparation in the U.S. and China and Phase 3 clinical development is underway in Japan. The first two Phase 3 trials completed, Mont Blanc and Denali, have been designed to fulfil the regulatory requirements for safety and efficacy Phase 3 trials to support NDA submissions in both the U.S. and in China, and both studies are required for the submissions. A separate Phase 3 clinical program is underway to support Japanese approval. Should NCX 470 be developed for other territories, there may be additional requirements. NCX 470 is exclusively licensed to Ocumension Therapeutics in China, Korea and Southeast Asia and to Kowa in the rest of the world. About Nicox Nicox SA is an international ophthalmology company developing innovative solutions to help maintain vision and improve ocular health. Nicox's lead program in clinical development is NCX 470 (bimatoprost grenod), a novel nitric oxide-donating bimatoprost eye drop, for lowering intraocular pressure in patients with open-angle glaucoma or ocular hypertension, licensed to Ocumension Therapeutics for the Chinese, Korean and Southeast Asian markets and to Kowa in the rest of the world. Nicox also has a preclinical research program on NCX 1728, a nitric oxide-donating phosphodiesterase-5 inhibitor, with Glaukos. Nicox's first product, VYZULTA® in glaucoma, licensed exclusively worldwide to Bausch + Lomb, is available commercially in the U.S. and over 15 other territories. Nicox generates revenue from ZERVIATE® in allergic conjunctivitis, licensed in multiple geographies, including to Harrow, Inc. in the U.S., and Ocumension Therapeutics in the Chinese, and in the majority of Southeast Asian markets. Nicox, headquartered in Sophia Antipolis, France, is listed on Euronext Growth Paris (Ticker symbol: ALCOX) and is part of the CAC Healthcare index. For more information Analyst coverage H.C. Wainwright & Co Yi Chen New York, U.S. The views expressed by analysts in their coverage of Nicox are those of the author and do not reflect the views of Nicox. Additionally, the information contained in their reports may not be correct or current. Nicox disavows any obligation to correct or to update the information contained in analyst reports. Contacts NicoxGavin SpencerChief Executive OfficerT +33 (0)4 97 24 53 00communications@ Disclaimer The information contained in this document may be modified without prior notice. This information includes forward-looking statements. Such forward-looking statements are not guarantees of future performance. These statements are based on current expectations or beliefs of the management of Nicox S.A. and are subject to a number of factors and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements. Nicox S.A. and its affiliates, directors, officers, employees, advisers or agents, do not undertake, nor do they have any obligation, to provide updates or to revise any forward-looking factors which are likely to have a material effect on Nicox's business are presented in section 3 of the 'Rapport Annuel 2024' which is available on Nicox's website ( this press release may be drafted in the French and English languages. If both versions are interpreted differently, the French language version shall prevail. Nicox Sophia Antipolis, Bâtiment C, Emerald Square, Rue Evariste Galois, 06410 Biot, FranceT +33 (0)4 97 24 53 00 Attachment EN_NCX470DenaliToplineAugust2025_PR_FINAL
Gizmodo
17 hours ago
- Gizmodo
Stone Age People Brutalized Their Prisoners of War, New Evidence Suggests
When we think of Stone Age people, most imagine small communities living in caves, cutting into their most recent hunt with primitive tools, and imitating their environment with illustrative rock art. People during the Neolithic, however—the last stage of the Stone Age (around 9000 to 3300 BCE)—also waged wars and absolutely demolished their enemies. In a study published today in the journal Science Advances, researchers present horrific evidence suggesting that Neolithic people in northeastern France mutilated foreign invaders. Their findings might represent some of the earliest known indications of gruesome victory celebrations related to war. The international team of researchers analyzed skeletal remains and severed limbs from burial pits dating to between 4300 and 4150 BCE at two sites near Strasbourg, Achenheim and Bergheim. 'A total of 82 humans are analyzed,' they wrote in the study. Their analysis revealed 'differences between victims and nonvictims and suggest that the former were members of invading groups brutally killed, perhaps exposed and deposited in pits—together with trophies in the form of severed upper limbs—by local groups in what might be one of the earliest well-documented instances of martial victory celebrations in prehistoric Europe,' they explained. Previous research in the Upper Rhine Valley had already shown that this time period was marked by military invasions and cultural upheaval, but scientists didn't know whether the human remains at Achenheim and Bergheim were of locals or foreigners and/or prisoners of war. In addition to the severed upper limbs, the researchers also identified injuries such as skull fractures that had not healed. The team, including Valladolid University's Teresa Fernandez-Crespo, suggests these victims met violent ends during war. The individuals without these sorts of unhealed injuries, on the other hand, likely received a regular burial. To investigate the differences between those who were brutally killed and those who weren't, Fernandez-Crespo and her colleagues conducted isotopic analyses. By identifying ratios of isotopes—variants of the same element—in the remains, they discovered that, while the non-victims were locals, the victims of war violence came from other regions. According to the researchers, this indicates that the victims were likely invaders killed by locals. 'In view of their demise, it is probable that the identities of these victims can be attributed to socially remote, nonlocal enemies that became trophies or captives during battles or raids and that may have been considered by their captors as not properly 'human' and hence warranting such treatment,' the researchers explained. For once, the term 'overkill' applies literally.
