Latest news with #ACC.25
Business Wire
02-05-2025
- Business
- Business Wire
Hesperos Wins ACC.25 Pitch Competition as FDA Unveils Plan to Move Beyond Animal Testing
ORLANDO, Fla.--(BUSINESS WIRE)--Hesperos, a leader in recreating human biology for drug development with its Human-on-a-Chip ® technology, took top honors at the American College of Cardiology annual meeting (ACC.25). The showcase featured companies funded by the National Heart, Lung, and Blood Institute (NHLBI). Four companies were selected from a pool of more than 20 NHLBI-funded startups to present their technologies and commercialization strategies to a panel of clinical, translational medicine, and industry experts. Hesperos' winning presentation highlighted how its advanced multi-organ Human-on-a-Chip® models have accelerated development programs across a range of disease areas, with the FDA accepting data in support of four Phase II programs. Share 'This impressive achievement underscores Hesperos' innovative Human-on-a-Chip organ system technology that provides drug and (other medical product) developers more informative results about their products, more quickly and at lower cost than conventional animal testing. This validation of our service offerings by recognized experts aligns well with FDA's renewed focus on phasing out animal testing (see 10 April 2025 press release on said Christopher Long, PhD, Hesperos' Director of Automation and Engineering, who presented the pitch at ACC.25. Hesperos' winning presentation highlighted how its advanced multi-organ Human-on-a-Chip models have accelerated development programs across a range of disease areas, with the FDA accepting data in support of four Phase II programs, two of which that already have progressed to Phase III. Furthermore, NIH Director Dr. Jay Bhattacharya emphasized support for this shift in a 29 April 2025 press release on stating, 'This human-based approach will accelerate innovation, improve healthcare outcomes, and deliver life-changing treatments.' By providing clinically relevant functional readouts that often better predict human outcomes, Hesperos offers a faster, more capital-efficient pathway for drug development. With a growing track record of regulatory acceptance, the company continues to drive innovation, accelerating an industry shift away from animal testing and toward human-based approaches in therapeutic development. About Hesperos, Inc. Hesperos is a global contract research organization (CRO) specializing in preclinical drug development services through its Human-on-a-Chip ® platform. By replicating key aspects of human biology (and thus avoiding expensive, time-consuming and often less informative animal testing), Human-on-a-Chip ® organ-system models provide product development teams with more meaningful insights that can accurately predict an agent's therapeutic profile while lowering costs and accelerating development timelines. For more information, visit
Yahoo
31-03-2025
- Health
- Yahoo
Cleerly Unveils Groundbreaking Late-Breaking Clinical Trial Results Demonstrating AI-QCT's Predictive Power for Women's Cardiovascular Risk at ACC.25
New Findings from the CONFIRM2 Registry Reveal Significant Gender Disparities in Coronary Plaque Features and Associated Risks for Major Adverse Cardiovascular Events DENVER, March 31, 2025--(BUSINESS WIRE)--Cleerly, the leader in cardiovascular AI imaging, has announced revolutionary findings from its late-breaking clinical trial presented at the American College of Cardiology's Annual Scientific Session + Expo (ACC.25) in Chicago, Illinois. The study, titled "Artificial Intelligence-based Quantitative Computed Tomography (AI-QCT) Coronary Plaque Features Predict Risk More Pronounced in Females: The International Multicentric Registry CONFIRM2," was led by Gudrun M. Feuchtner, MD, MBA, and presented during the Clinical and Investigative Horizons I session on March 31st, 2025. This research is simultaneously published today in Circulation: Cardiovascular Imaging, a peer-reviewed journal of the American Heart Association. The findings demonstrate that AI-QCT features of coronary artery disease (CAD), as detected by Cleerly's AI-QCT, can identify women at high risk for major adverse cardiovascular events (MACE) and improves risk stratification - a population that has historically been underdiagnosed and undertreated for CAD. The CONFIRM2 study is notable for including approximately 50% female participants and a diverse cohort of 3,500 individuals from 11 countries, highlighting the broad applicability of these findings in addressing cardiovascular health disparities. Key Findings on Gender Disparities in Cardiovascular Risk Higher Risk for Women: Despite a higher total AI-QCT CAD burden in men, similar increases in AI-QCT-derived plaque features (total plaque volume, calcified plaque, non-calcified plaque, and percentage atheroma volume) confer a significantly higher risk for MACE in women. Traditional risk scores performed poorly, while AI-QCT added significant predictive value for both sexes. Enhanced Detection and Risk Stratification: Cleerly's AI-QCT improves the detection and characterization of coronary atherosclerosis, offering more precise prognostic risk stratification for MACE that surpasses traditional risk factors. Gender-Specific Insights: AI-QCT reveals that certain coronary plaque features present a higher risk of MACE in women compared to men, emphasizing the need for tailored preventive strategies for women. Prognostic Value Consistency: The analysis shows that AI-QCT has strong prognostic value in both women and men, with stenosis and non-calcified plaque identified as the strongest independent predictors in women. Implications for Women's Heart Health These findings underscore the urgent unmet need to integrate AI-QCT into clinical practice for unbiased risk estimation in both men and women. Enhanced Precision & Personalization: Using AI-QCT-based risk stratification instead of traditional risk scores strongly enhances precision and enables tailored preventive care. Addressing Underdiagnosis & Undertreatment: Transitioning to an AI-QCT-based approach may eliminate bias and tackle the longstanding issue of underdiagnosis and undertreatment of CAD in women, ultimately improving outcomes. Increased Awareness & Tailored Therapies: AI-QCT's ability to identify higher risk in women should drive greater awareness and prompt reinforced anti-atherosclerotic therapies and other preventive measures tailored specifically to women. Potential for Sex-Specific Guidelines: These insights may lead to the development of sex-specific treatment guidelines, ensuring women receive the most appropriate and effective care for their cardiovascular health. "This research is a significant step forward in addressing the historical disparities in cardiovascular care for women. AI-QCT provides physicians with a powerful, unbiased tool that not only enhances detection but also enables more precise risk prediction and personalized care. These findings underscore the urgency of incorporating AI-QCT into clinical practice to improve outcomes for all patients, and especially for women," said Gudrun M. Feuchtner, MD, MBA, lead researcher of the study, from Medical University Innsbruck, Austria, on behalf of the CONFIRM2 investigator group led by Principal Investigator Alexander van Rosendael, MD, PhD, from Leiden University Medical Center and Ibrahim Danad, MD, from Radboud Medical University Center, Netherlands. About Cleerly Cleerly is the company on a mission to eliminate heart attacks by creating a new standard of care for heart disease. Through its FDA-cleared solutions driven by artificial intelligence, Cleerly supports comprehensive phenotyping of coronary artery disease, as determined from advanced noninvasive CT imaging. Cleerly's approach is grounded in science, based on millions of images from over 40,000 patients. Led by a world-class clinical and technical team, Cleerly enhances health literacy for each and every stakeholder in the coronary care pathway. For more information, please visit: View source version on Contacts Cleerly Media Contact Christy Sievertpress@
Associated Press
30-03-2025
- Health
- Associated Press
CPC Announces Semaglutide Significantly Improves Walking Distance, Symptoms and Quality of Life for People with PAD and Diabetes
Study underscores the direct vascular effects of this class of drugs, researchers say AURORA, CO / ACCESS Newswire / March 29, 2025 / A novel study of a semaglutide, a glucagon-like peptide-1 (GLP-1) agonist used to treat type 2 diabetes, obesity and cardiovascular disease, semaglutide was found to significantly improve maximal walking distance in people with symptomatic peripheral artery disease (PAD) and type 2 diabetes, meeting the study's primary endpoint. The STRIDE Trial, presented at the American College of Cardiology's Annual Scientific Session (ACC.25), is the first to evaluate any GLP-1 agonist in PAD for this outcome. In addition to improvements in walking ability and function, people taking semaglutide also saw significant improvements in both symptoms and quality of life compared with those taking a placebo. People taking semaglutide also saw significant improvements in both symptoms and quality of life compared with those taking a placebo. Dr. Marc Bonaca, Executive Director of CPC presented the results and concluded, 'We have the first new treatment for PAD symptoms in 25 years.' PAD affects an estimated 12 million U.S. adults and over 200 million people worldwide. PAD happens when there is a build-up of fat and cholesterol, most commonly in the arteries of the legs. It's often associated with difficulty walking and poor circulation that can lead to non-healing wounds and a high rate of limb loss. People with PAD are at very high risk for serious complications, including acute limb ischemia - similar to a heart attack or stroke of the leg - that can lead to limb amputation or death if not treated quickly. 'Even at very early stages of PAD, people can't walk well, but they often don't know it's PAD. They may say, 'I've just slowed down,' 'I'm getting older,' or 'I have arthritis,' but they're actually severely functionally impaired and they often will self-limit what they are doing,' said Marc P. Bonaca, MD, MPH, professor of medicine and director of vascular research at the University of Colorado School of Medicine in Aurora, Colorado, and the study's lead author, adding that the last drug approved by the FDA for improving functional outcomes in PAD was cilostazol in 2000, so there is a huge unmet need. 'The only drug we have available is contraindicated in people with heart failure, has no benefits beyond improvement in symptoms, and causes a lot of side effects and, so overall it's used in less than 10% of people and so we really have limited options to improve function in PAD,' he said. 'The [issue] is that as PAD progresses, patients go on to get revascularization procedures to open arteries, and become at high risk for adverse cardiovascular and limb events.' For this randomized, placebo-controlled, double-blind trial, called STRIDE, 792 people with type 2 diabetes and early-stage symptomatic PAD were enrolled at 112 medical centers in 20 countries. Patients (67 years old on average, about 25% women, 67% white) were randomized to receive semaglutide (1 mg) or placebo for one year (52 weeks). Researchers assessed maximal walking distance - the maximum distance that patients were able to walk on a treadmill at 2 miles per hour at a 12% grade (similar to walking up a moderate hill). Function was assessed at baseline (median maximal walk distance was 186 meters), week 26, week 52 (primary endpoint), and week 57 (5 weeks after stopping treatment). The Colorado Prevention Center (CPC), affiliated with the University of Colorado School of Medicine, supported the treadmill endpoint through its core lab services. 'Despite the fact that people were recruited on the basis of reporting early-stage PAD observed that they were actually severely impaired and could only walk about one-tenth of a mile with symptom onset significantly earlier,' Bonaca said. 'We saw that the drug clearly worked. There was a clear early benefit at six months that continued to increase at one year.' Overall, patients in the semaglutide arm had median and mean improvements in walking distance compared to placebo of 26 meters and 39.9 meters respectively, giving a statistically significant improvement in the ratio of change from baseline to week 52 of 1.13 (p=0.0004). 'To put that into context, we usually think an increase in walking distance of 10 to 20 meters is clinically important in PAD, so this exceeded those expectations,' he said. The results were further supported by confirmatory secondary endpoints showing significant improvements in quality of life (as measured by the Vascular Quality of Life Questionnaire-6 score), symptoms represented by pain-free walking distance, and sustained improvement in maximal walking distance 5 weeks after stopping therapy. Safety was shown to be similar to what was seen in earlier trials with semaglutide, with non-serious gastrointestinal side effects the most commonly reported side effects. Patients' ankle brachial index, a measure of blood flow in the legs, was also significantly improved among those taking semaglutide compared with placebo. A post-hoc analysis looking at time to rescue treatment (the need for revascularization due to worsening symptoms) or death was also lower with semaglutide. Within one-year of treatment, patients taking semaglutide had a 54% reduction in their risk of dying or needing a medication or procedure to open blocked arteries in their legs due to worsening symptoms compared with those receiving a placebo (14 vs 30 patients). 'Taken together, the data support semaglutide for people with PAD and type 2 diabetes mellitus as a therapy that has cardiometabolic, cardiovascular and kidney benefits and improves function, symptoms, and quality of life.,' said Bonaca, 'There is more work to be done to understand the mechanism of benefit as the population had a median BMI of 28.6 and the relationship between the outcome and weight loss was very weak. This coupled with the increase in ABI really suggests a direct vascular effect. This also raises the question of whether patients with PAD and without type 2 diabetes mellitus could benefit and that should be investigated in future studies.' The study was limited in that it was only in patients who also had diabetes. In addition, there were fewer U.S. patients because revascularization is so common and that patients who had these procedures were excluded. As a result, there was also less diversity and very few Black patients. This study was funded by Novo Nordisk. It was simultaneously published online in The Lancet at the time of presentation. Dr. Marc P. Bonaca can be reached by media at [email protected] or 303-860-9900. Dr. Marc Bonaca will present the study, 'The Effect Of Once-weekly Subcutaneous Semaglutide On Functional Capacity In People With Type 2 Diabetes And Peripheral Artery Disease: Primary Results From The Phase 3b, Randomized, Placebo-controlled, Double Blind Stride Trial,' on March 29, 2025, at 9:30 a.m. CT in Chicago at the American College of Cardiology Meeting. CPC Clinical Research, an ARO affiliated with the University of Colorado, is conducting the study in collaboration with Novo Nordisk. 2115 N. Scranton St., Suite 2040
Yahoo
29-03-2025
- Health
- Yahoo
Anthos Therapeutics Shares New Data from the Landmark AZALEA-TIMI 71 Study Demonstrating the Factor XI Inhibitor Abelacimab Significantly Reduced Bleeding in Patients Regardless of Age or Bleeding Risk
CAMBRIDGE, Mass., March 29, 2025 (GLOBE NEWSWIRE) -- Anthos Therapeutics, Inc., a transformative, clinical-stage biopharmaceutical company developing innovative therapies for the treatment of cardiovascular metabolic diseases, is presenting two new analyses from its landmark AZALEA-TIMI 71 study at the American College of Cardiology Annual Scientific Session (ACC.25) demonstrating the novel Factor XI inhibitor abelacimab significantly reduced bleeding in patients regardless of age or bleeding risk. These data will be presented today during the moderated poster sessions from 2:30-4:30pm CT. In the first analysis, the safety of abelacimab, a novel Factor XI inhibitor, was compared to rivaroxaban, a direct oral anticoagulant (DOAC), by patient age. Inhibition of Factor XI with abelacimab significantly reduced the relative risk of major or clinically relevant non-major (CRNM) bleeding compared with rivaroxaban regardless of age, with potential for greater absolute risk reduction (ARR) with older patients. The ARR in patients 75 years of age and older was 6.2% compared to 4.2% in patients less than 75 years. In addition, abelacimab consistently reduced bleeding risk in patients 75 years of age and older regardless of renal function, body mass index (BMI), and the use of concomitant antiplatelet therapy. "Patients with atrial fibrillation, particularly older patients, are frequently at a high risk of bleeding. There is a need for safer anticoagulants that can prevent thrombotic events while minimizing excess bleeding,' said Christian T. Ruff, MD, MPH, senior investigator of TIMI Group, director General Cardiology, Brigham and Women's Hospital, and associate professor, Harvard Medical School. 'These data show that abelacimab significantly reduced the risk of major or CRNM bleeding, particularly among people over 75 who are vulnerable to such risks, and, if approved, could be a safer alternative for patients needing anticoagulation therapy.' In the second analysis, the safety of abelacimab was compared to rivaroxaban across a spectrum of bleeding risk, which was determined by using the direct oral anticoagulant (DOAC) score, a clinical tool leveraged to assess the risk of bleeding in patients who are prescribed DOACs. In patients with atrial fibrillation, abelacimab reduced rates of bleeding relative to rivaroxaban regardless of bleeding risk, with greater absolute safety benefit in those at higher bleeding risk. In the rivaroxaban arm, the rates of major or CRNM bleeding per 100 patient years increased stepwise across risk categories from 5.6% in the low risk category to 21.2% in the very high risk category. In contrast, the rates of bleeding in patients in the pooled abelacimab arm were 2.5% in the low-risk category to 7.1% in the very high-risk category. The ARR increased from 3.1% in the low risk category to 14.1% in the very high risk category (p-trend for ARR <0.001). These data suggest that, if approved, abelacimab may be especially attractive in patients with atrial fibrillation who are at high bleeding risk. "Research shows that 40% of patients with atrial fibrillation are not receiving optimal treatment when it comes to anticoagulation, in large part due to a fear of bleeding. This includes people living with atrial fibrillation who have a higher risk of bleeding, such as the elderly, patients taking anti-platelet agents, and those with other co-morbidities,' said Dan Bloomfield, MD, chief medical officer of Anthos Therapeutics. 'These results suggest that abelacimab may be safe across the spectrum of bleeding risk and may make it possible to anticoagulate some of the most vulnerable populations who otherwise would not be treated and therefore left unprotected from the risk of stroke.' Full results of the AZALEA-TIMI 71 study were published in January of this year in the New England Journal of Medicine (NEJM) and demonstrated a 62% reduction in major or CRNM bleeding with abelacimab 150 mg compared to rivaroxaban 20 mg in patients with atrial fibrillation. Abelacimab is currently in Phase 3 development with the lead indication for the prevention of stroke and systemic embolism in patients with atrial fibrillation (LILAC-TIMI 76), in addition to two studies in patients with cancer-associated thrombosis (ASTER and MAGNOLIA). Data from these trials are expected in the second half of 2026. AZALEA-TIMI 71 Study of Bleeding Risk by Age Relevant Study Details: Both abelacimab doses (90 mg and 150 mg) were pooled for this analysis. Cox proportional hazards were used to examine the primary outcome of major/CRNM bleeding with an interaction term for treatment*age (≥75 vs <75 yrs). Of 1,287 patients, 625 (49%) were ≥75 yrs at baseline. Patients ≥75 had lower BMI (28 vs. 32 kg/m2) and were less likely to be on antiplatelet therapy (17% vs 31%), but more likely to have Creatinine clearance (CrCl) ≤50 mL/min (33% vs. 8%) compared with younger patients (p<0.001 for each). AZALEA-TIMI 71 Study of Patient-Specific Bleeding Risk Relevant Study Details: Both abelacimab doses (90 mg and 150 mg) were pooled for this analysis. Patient-specific bleeding risk was categorized using the previously validated DOAC score. Overall, 8%, 33%, 37%, 16% and 5% of patients were categorized as very low (0-3), low (4-5), moderate (6-7), high (8-9) and very high (10) bleeding risk, respectively. Media Contact: media@ About Anthos TherapeuticsFounded by Blackstone Life Sciences (BXLS) in 2019, Anthos Therapeutics is a transformative, clinical-stage biopharmaceutical company with the exclusive global rights from Novartis Pharma AG to develop, manufacture and commercialize abelacimab. For more information, visit the Company's website and follow on Twitter and LinkedIn. About the AZALEA-TIMI 71 StudyLaunched in February 2021, the AZALEA-TIMI 71 study enrolled 1,287 patients across 95 global study sites, including the U.S. and Canada, Europe and Asia. With a median follow-up of 2.1 years, spanning more than 2,000 patient-years, the AZALEA-TIMI 71 study is the largest and longest head-to-head study of a Factor XI inhibitor compared to a standard-of-care anticoagulant. About AbelacimabAbelacimab is a novel, investigational, highly selective, fully human monoclonal antibody that binds tightly to Factor XI to block its activation and prevent the generation of the activated form (Factor XIa). This mimics natural Factor XI deficiency, which is associated with protection from thromboembolic disease.1 As a monoclonal antibody, abelacimab is not metabolized via the cytochrome P450 system or as a substrate for P-glycoprotein, meaning the risk of drug-drug interactions is very low.2 There is no need to adjust the dose based on age or renal/hepatic status.3 Factor XI inhibition offers the promise of hemostasis-sparing anticoagulation for the prevention and treatment of arterial and venous thromboembolic events.4 Abelacimab is the only Factor XI inhibitor being studied for both conditions. If approved, abelacimab is planned to be dosed subcutaneously (SC) monthly by patients with atrial fibrillation using an autoinjector to maintain near-complete inhibition in a chronic setting. It is also planned to be administered via an initial intravenous (IV) infusion for acute indications requiring immediate onset of action and then followed by subsequent monthly SC administration. In the AZALEA-TIMI 71 study, abelacimab 150 mg dosed subcutaneously once-monthly, inhibited Factor XI by 99%.5 In a PK/PD study, abelacimab administered by IV provided profound suppression of Factor XI within one hour after the start of therapy and maintained near maximal inhibition for up to 30 days.6 The results of the AZALEA-TIMI 71 study, published in the January 23, 2025, issue of New England Journal of Medicine (NEJM) showed a 62% reduction in major or clinically relevant non-major bleeding with abelacimab 150 mg compared with rivaroxaban 20 mg in patients with atrial fibrillation who are at moderate-to-high risk of stroke (P<0.001, HR 0.38, 95% Cl 0.24–0.60). Data also showed a 62% reduction in major bleeding alone with abelacimab 150 mg vs. rivaroxaban 20 mg (P=0.001, HR 0.33, 95% CI 0.16-0.66) and an 89% reduction in gastrointestinal (GI) bleeding).7 Abelacimab received a Fast Track Designation from the FDA in July 2022 for the treatment of thrombosis associated with cancer. In September 2022, abelacimab was also granted a Fast Track Designation for the prevention of stroke and systemic embolism in patients with atrial fibrillation. Abelacimab is an investigational agent and is not approved for any indication in any country. About the DOAC Score The direct oral anticoagulant (DOAC) score is a clinical tool used to assess the risk of bleeding in patients who are prescribed DOACs, a class of drugs used to prevent and treat thromboembolic disorders such as stroke in atrial fibrillation. The DOAC score helps clinicians evaluate the safety and appropriateness of DOAC therapy for individual patients by considering factors such as age, renal function, history of bleeding, and concurrent use of other medications that may increase bleeding risk. Forward-Looking StatementsThis press release contains 'forward-looking statements' within the meaning of the Private Securities Litigation Reform Act of 1995 that involve substantial risks and uncertainties, including statements regarding the potential benefit of abelacimab and our goals to further develop and commercialize abelacimab. All statements, other than statements of historical facts, contained in this press release, including statements regarding the company's strategy, future operations, future financial position, prospects, plans and objectives of management, are forward-looking statements. The words 'anticipate,' 'become,' 'believe,' 'continue,' 'could,' 'estimate,' 'expect,' 'intend,' 'may,' 'plan,' 'potential,' 'predict,' 'project,' 'should,' 'target,' 'will,' 'would' and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. In addition, the forward-looking statements included in this press release represent the company's views as of the date hereof and should not be relied upon as representing the company's views as of any date subsequent to the date hereof. The company anticipates that subsequent events and developments will cause the company's views to change. However, while the company may elect to update these forward-looking statements at some point in the future, the company specifically disclaims any obligation to do so. 1 Goodman et al. Crit Pathways in Cardiol. June 20242 Hsu et al. J Am Coll Cardiol. Aug. 2021 3 Hsu et al. J Am Coll Cardiol. Aug. 2021 4 Hsu et al. J Am Coll Cardiol. Aug. 2021 5 TIMI Study Group website, AZALEA 716 Yi BA et al. J Thromb Haemost. Oct. 20217 Ruff C et al. N Engl J Med. Jan. 2025Sign in to access your portfolio
Yahoo
29-03-2025
- Health
- Yahoo
Imbria Presents Positive Clinical Data from Phase 2 IMPROVE-DiCE Trial of Ninerafaxstat in Patients with Cardiometabolic HFpEF at ACC.25
– Ninerafaxstat improves cardiac energetics, cardiac reserve capacity, 6-minute walk distance and heartfailure-related health status (KCCQ) in cardiometabolic HFpEF – – Findings support further development of ninerafaxstat in HFpEF and adjacent CV indicationssuch as non-obstructive hypertrophic cardiomyopathy (nHCM) – BOSTON, March 29, 2025 (GLOBE NEWSWIRE) -- Imbria Pharmaceuticals, Inc., a clinical stage, cardiometabolic company developing novel therapies designed to enhance cellular energetics, today announced positive clinical results from Part 2 of the IMPROVE-DiCE Phase 2 trial demonstrating that ninerafaxstat improved cardiac energetics, left ventricular reserve capacity, exercise capacity and patient-reported heart failure symptoms and physical limitations in patients with cardiometabolic heart failure with preserved ejection fraction (HFpEF). The results were presented at the American College of Cardiology's Annual Scientific Session & Expo (ACC.25) in Chicago, IL during the moderated poster session titled 'Innovations and Insights in Heart Failure With Preserved Ejection Fraction: Emerging Therapies, Biomarkers and Mechanistic Studies.' 'These findings validate the mechanistic rationale for targeting impaired myocardial energetics in HFpEF and demonstrate ninerafaxstat's potential to restore resting cardiac energetics and enhance functional cardiac reserve,' said Oliver Rider, MRCP(UK), DPhil (Oxon), Professor of Cardiovascular Medicine, University of Oxford. 'Importantly, these results suggest ninerafaxstat could improve daily symptoms and physical capacity for people living with HFpEF, a condition with few effective treatments available today.' Key findings from the trial include: Treatment of patients with cardiometabolic HFpEF with ninerafaxstat for 12 weeks resulted in a statistically significant (P=0.02) increase from baseline in cardiac phosphocreatine to adenosine triphosphate (PCr/ATP) ratio, consistent with an improvement in cardiac energy reserves and meeting the trial's primary objective. Of note, the largest energetic responses to ninerafaxstat were observed in those with the most severe energy deficit at baseline. Significant improvement in left ventricular (LV) systolic reserve capacity with exercise, reflecting an increase in the heart's ability to augment its stroke volume during exercise (P=0.03), a key abnormality in patients with HFpEF. Consistent with enhanced LV reserve capacity, an increase in 6-minute walk distance of ~14 m (P=0.02) was observed. Statistically significant and clinically meaningful improvements in heart failure-related health status, measured by the Kansas City Cardiomyopathy Questionnaire-Clinical Summary Score (KCCQ-CSS), were observed in patients most symptomatically limited at baseline, pre-specified as those with KCCQ-CSS ≤80 (~8 points; P=0.04). 'Approximately 50% of heart failure cases are related to HFpEF. In the U.S. alone, the prevalence is expected to rise to 8.5 million by 2030. Within the HFpEF population, at least 80% are living with type 2 diabetes mellitus (T2DM) and obesity. There are no current treatments aimed at targeting mitochondrial energy generation to improve patient symptoms, quality of life, and functional capacity. We believe these validating results pave the way to support further investigation of ninerafaxstat in cardiometabolic HFpEF,' said Jai Patel, MRCP(UK), Chief Medical Officer at Imbria. 'Ninerafaxstat's unique therapeutic profile has the potential to make a meaningful impact in cardiometabolic HFpEF and pathophysiologically adjacent disease states such as nHCM.' A copy of the poster will be available in the 'Media Center' section of the Imbria website at About ninerafaxstatNinerafaxstat is an innovative treatment for cardiac disorders characterized by an imbalance of energy supply and demand in the heart. To maintain normal contractile function, the heart requires substantial amounts of energy, which is produced primarily by the mitochondria in the form of adenosine triphosphate (ATP). The heart normally uses two main fuels for energy generation: fatty acids and glucose. Ninerafaxstat, a partial fatty acid oxidation (pFOX) inhibitor, acts to shift the heart's preference from fatty acids towards glucose. This shift in metabolism leads to more efficient mitochondrial energy generation with the potential for improved cardiac function both at rest and during exercise. Ninerafaxstat is a simple orally administered compound with no dose titration or monitoring required, no clinically significant drug-drug interactions and can be used on top of standard of care cardiovascular treatments. About IMPROVE-DiCEIMPROVE-DiCE is the first clinical trial utilizing multi-nuclear spectroscopy, including state-of-the-art hyperpolarized MR spectroscopy, and MRI to quantify the cardiac energetic, metabolic and functional responses to an investigational metabolic modulator in cardiovascular disease. It is a two-part, Phase 2 clinical trial evaluating the safety, tolerability and pharmacodynamic effects of ninerafaxstat. Part 1 enrolled pre-HFpEF patients with type 2 diabetes and obesity and demonstrated normalization of cardiac energetics, significant reduction in cardiac steatosis and improvements in left ventricular diastolic filling rate, an important component of heart failure. The results of Part 1 were presented at the European Society of Cardiology Congress in August 2022. Part 2 of IMPROVE-DiCE enrolled symptomatic patients with cardiometabolic HFpEF and assessed the impact of ninerafaxstat on cardiac energetics, cardiac reserve function, exercise capacity and heart failure symptoms. The trial was conducted at the Oxford Centre for Clinical Magnetic Resonance Research (OCMR), Radcliffe Department of Medicine at the University of Oxford, UK and was led by Professor Oliver Rider. For more information, please visit (Identifier: NCT04826159). About heart failure with preserved ejection fraction (HFpEF)Over half of all patients with heart failure have preserved ejection fraction, a clinical syndrome characterized by an inability of the heart to pump blood adequately to the body without pathological increases in filling pressures at rest or during exertion. As with other forms of heart failure, HFpEF is associated with cardiac energy deficiency resulting from impaired mitochondrial energy generation. This leads to impaired cardiac functional reserve on exertion and is associated with exercise-induced pulmonary congestion, resulting in hallmark heart failure symptoms of exertional breathlessness, fatigue, and markedly reduced exercise capacity leading to severe impairment in quality of life. Despite being associated with severe morbidity and mortality, HFpEF has few evidence-based therapies. Within the clinical syndrome of HFpEF, the cardiometabolic HFpEF phenotype, in which chronic cardiometabolic stress resulting from type 2 diabetes and obesity are key drivers of heart failure pathophysiology, is rapidly emerging as the most prevalent form. About ImbriaImbria is a privately held, clinical stage company developing novel therapies for patients with life-altering cardiometabolic disorders. Our clinical pipeline is focused on restoring or improving the cell's ability to produce energy in cardiometabolic disorders where energetic impairment is a fundamental contributor to disease pathogenesis, symptoms and functional deficits. The lead product candidate, ninerafaxstat, has completed multiple Phase 2 clinical trials in three indications: nHCM, stable angina, and HFpEF. In Phase 1 and 2 clinical trials, ninerafaxstat was shown to be well tolerated. For additional information, please visit ContactKomal JoshiImbria Pharmaceuticals, in to access your portfolio
