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VARIPURE demonstrated strong safety outcomes with no incidence of stroke and 99.7% acute effectiveness of the VARIPULSE™ Platform in nearly 800 enrolled patientsi IRVINE, Calif., Sept. 2, 2025 /PRNewswire/ — Johnson & Johnson (NYSE: JNJ) — Johnson & Johnson MedTech, a global leader in…
Largest Noninterventional Natural History Study of People Under 18 with MYBPC3-associated HCM with More than 200 Participants 93% of MyClimb Participants had the Nonobstructive Form of HCM for Which There Are No Approved Therapeutics Genotypic Status Identified as a Significant Predictor of Risk and Left Ventricular Mass Index May Serve as a Surrogate Marker for Poor Long-Term Outcomes SOUTH SAN FRANCISCO, Calif., Aug. 31, 2025 (GLOBE NEWSWIRE) — Tenaya Therapeutics, Inc. (NASDAQ: TNYA), a clinical-stage biotechnology company with a mission to discover, develop and deliver potentially curative therapies that address the underlying causes of heart disease, today announced the presentation of interim data from its ongoing MyClimb™ (ClinicalTrials.gov ID: NCT05112237) natural history study of pediatric participants with MYBPC3-associated hypertrophic cardiomyopathy (HCM) at the European Society of Cardiology Congress (ESC). “The interim data from MyClimb presented at ESC highlight the distinct risks of complications, heightened severity and rapid rate of disease progression experienced by pediatric patients with MYBPC3-associated HCM,” said Georgia Brugada, M.D., Pediatric Cardiologist, Head of Arrhythmias Unit, and Medical Consultant at SJD Barcelona Children’s Hospital and an investigator for the MyClimb study. “The data emerging from MyClimb underscore the urgent need for disease-modifying approaches to the treatment of children with MYBPC3-associated HCM, as well as the importance of genetic diagnosis, genetic counseling and close monitoring in this population.” “Data obtained from MyClimb offer actionable information for predicting those severe MYBPC3-associated HCM pediatric patients who may be at higher risk of death or severe complications, such as life-threatening ventricular arrhythmias, cardiovascular-related hospitalizations, heart failure, sudden cardiac arrest, and/or heart transplant,” said Whit Tingley, M.D., Ph.D., Tenaya’s Chief Medical Officer. “Among the new observations, we believe certain genetic profiles or higher left ventricular mass index can identify those children who are at substantially greater risk of serious potential complications, relatively rapid progression and poor long-term outcomes. These new insights into predictive risk factors can inform clinicians’ thinking on risk stratification and interventions and offer important clues for endpoints and eligibility criteria as we consider advancing genetic medicines such as TN-201 gene therapy in clinical trials for pediatric patients where there is high unmet need.” MyClimb is believed to be the largest natural history study of those under 18 diagnosed with MYBPC3-associated HCM. As of the July 2025 cutoff for this interim readout, 213 MYBPC3-associated HCM individuals diagnosed before the age of 18 were analyzed, with retrospective data for 173 and prospective data for 42 participants available. Participants were enrolled from 27 centers across the U.S., Canada, Spain and the UK. Key findings from MyClimb shared at ESC 2025 include: 93% of participants were classified as having the nonobstructive HCM phenotype, for which there are currently no approved treatment options.Participants were stratified based on three genetic inheritance patterns, revealing distinct risk profiles. Among the Homozygous group (also referred to as biallelic; those born with two pathogenic or likely pathogenic truncating variants in MYBPC3), nearly all died or required heart transplant before age one.Compound Heterozygous participants (with one pathogenic or likely pathogenic truncating variant and one missense variant in MYBPC3) had a median age of diagnosis of 2.9 years of age and experienced severe cardiomyopathy with significant arrythmia burden and high prevalence of heart-failure related hospitalization (63%), transplant or death (27%).Heterozygous children (with one pathogenic or likely pathogenic variant in MYBPC3) had a median age of diagnosis of 6.5 years of age. Among these, 27% experienced heart failure-related hospitalizations and 13% had arrhythmia-related symptoms. Initial modeling suggests Left Ventricular Mass Index (LVMI) was found to be a significant predictor of risk in compound heterozygous and heterozygous groups, with every 10-unit (g/m2) increase associated with a 10% higher hazard of a serious event. These findings indicate LVMI may serve as an appropriate surrogate marker to evaluate the early effectiveness of gene therapy in potential future pivotal studies. The poster, titled “Interim results from MyClimb, a natural history study of pediatric MYBPC3-associated hypertrophic cardiomyopathy (HCM)” is being presented as a moderated e-poster by Dr. Brugada at ESC 2025 today during the Pediatric cardiomyopathy session and is available in the “Our Science” section of Tenaya’s website. About MyClimb and Pediatric MYBPC3-Associated HCMMyClimb is a retrospective and prospective natural history study of MYBPC3-associated HCM participants diagnosed before the age of 18. The study was initiated in 2021 to characterize the association between genotype, structural, and functional cardiac measures over time. To date, MyClimb has enrolled more than 200 individuals at 29 sites worldwide and is believed to be the largest study of pediatric individuals with MYBPC3-associated HCM ever conducted. Mutations in MYBPC3, the gene that encodes cardiac myosin-binding protein C, are among the most common genetic causes of HCM. MYBPC3-associated pediatric-onset HCM is estimated to comprise ~17% of all MYBPC3-driven HCM cases, with 2% of patients presenting in infancy. In the U.S. alone, there are an estimated 3,000 children with the condition and 13,000 individuals who were diagnosed before the age of 18 and are now adults. Current treatment options for children with MYBPC3-associated HCM are limited and while interventions such as implantable cardio defibrillators and heart transplant may be lifesaving, they are also highly invasive, frequently associated with considerable complication rates and do not address the underlying cause of disease. About TN-201 Gene TherapyTN-201 is an adeno-associated virus serotype 9 (AAV9)-based gene therapy designed to deliver a working MYBPC3 gene to heart muscle cells via a single intravenous infusion, increasing MyBP-C protein levels to address the underlying cause of MYBPC3-associated HCM. The MyBP-C protein plays a crucial role in regulating the heart’s contractility. Encouraging initial data from the first three patients to receive TN-201 at the 3E13 vg/kg dose level (Cohort 1) were presented at the American College of Cardiology meeting Tenaya intends to report longer-term follow-up data from Cohort 1 and initial data from Cohort 2 in the fourth quarter of 2025. The U.S. Food and Drug Administration has granted TN-201 Fast Track, Orphan Drug and Rare Pediatric Drug Designations. TN-201 has also received orphan medicinal product designation from the European Commission. About Tenaya TherapeuticsTenaya Therapeutics is a clinical-stage biotechnology company committed to a bold mission: to discover, develop and deliver potentially curative therapies that address the underlying drivers of heart disease. Tenaya’s pipeline includes clinical-stage candidates TN-201, a gene therapy for MYBPC3-associated hypertrophic cardiomyopathy (HCM) and TN-401, a gene therapy for PKP2-associated arrhythmogenic right ventricular cardiomyopathy (ARVC). Tenaya has employed a suite of integrated internal capabilities, including modality agnostic target validation, capsid engineering and manufacturing, to generate a portfolio of novel medicines based on genetic insights, including TN-301, a clinical-stage small molecule HDAC6 inhibitor for the potential treatment of heart failure and related cardio/muscular disease, and multiple early-stage programs in preclinical development aimed at the treatment of both rare genetic disorders and more prevalent heart conditions. For more information, visit www.tenayatherapeutics.com. Forward Looking StatementsThis press release contains forward-looking statements as that term is defined in Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. Statements in this press release that are not purely historical are forward-looking statements. Words such as “believe,” “may,” and similar expressions are intended to identify forward-looking statements. Such forward-looking statements include, among other things, the clinical, therapeutic and commercial potential of, and expectations regarding TN-201; the potential for MyClimb data to help decipher endpoints and eligibility criteria for TN-201 in clinical trials for pediatric patients; the planned timing to report interim results from MyClimb; and statements made by Tenaya’s Chief Medical Officer. The forward-looking statements contained herein are based upon Tenaya’s current expectations and involve assumptions that may never materialize or may prove to be incorrect. These forward-looking statements are neither promises nor guarantees and are subject to a variety of risks and uncertainties, including but not limited to: the potential failure of TN-201 to demonstrate safety and/or efficacy in clinical testing; actions and decisions of regulatory agencies; availability of MyClimb results at the referenced time; risks associated with the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics and operating as an early stage company; Tenaya’s continuing compliance with applicable legal and regulatory requirements; Tenaya’s ability to raise any additional funding it will need to continue to pursue its product development plans; Tenaya’s reliance on third parties; Tenaya’s manufacturing, commercialization and marketing capabilities and strategy; the loss of key scientific or management personnel; competition in the industry in which Tenaya operates; Tenaya’s ability to obtain and maintain intellectual property protection for its product candidates; general economic and market conditions; and other risks. Information regarding the foregoing and additional risks may be found in the section entitled “Risk Factors” in documents that Tenaya files from time to time with the Securities and Exchange Commission. These forward-looking statements are made as of the date of this press release, and Tenaya assumes no obligation to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law. Tenaya Contact Michelle CorralVP, Corporate Communications and Investor RelationsTenaya TherapeuticsIR@TenayaThera.com InvestorsAnne-Marie FieldsPrecision AQ annmarie.fields@precisionaq.com MediaWendy RyanTen Bridge Communicationswendy@tenbridgecommunications.com
V-DIFFERENCE is first study to show that Leqvio prioritized after statins helps more patients achieve guideline low-density lipoprotein cholesterol (LDL-C) goals early with reduced muscle pain1 85% of patients on Leqvio plus individually optimized lipid-lowering therapy (LLT) achieved LDL-C targets within 90 days vs. 31% of patients on placebo plus LLT1Patients on Leqvio plus LLT were 43% less likely to experience muscle-related adverse events compared to those on placebo plus LLT1 Basel, August 30, 2025 – Novartis today announced positive results from V-DIFFERENCE, a Phase IV study evaluating Leqvio® (inclisiran) compared to placebo, both administered on top of individually optimized lipid-lowering therapy (LLT), in patients with high cholesterol (hypercholesterolemia) who have not achieved guideline-recommended low-density lipoprotein cholesterol (LDL-C) goals1. These data will be presented in one of the Hot Line sessions of the 2025 European Society of Cardiology (ESC) Congress, held in Madrid, Spain, from August 29 to September 1, 2025. After 90 days of treatment with Leqvio on top of LLT, 85% of patients achieved their guideline-recommended LDL-C target compared to 31% of those receiving placebo on top of LLT (p
Phase III trial informed by comprehensive KARDIA data set generated through three Phase II studies: KARDIA-1, KARDIA-2 and KARDIA-3In the Phase II KARDIA-3 study, presented today as a late breaker at the European Society of Cardiology Congress 2025, zilebesiran demonstrated clinically meaningful reductions in office systolic blood pressure at month three with continuous control through month sixZilebesiran, a potential best-in-disease RNAi anti-hypertensive with twice-yearly subcutaneous dosing, demonstrated encouraging safety when combined with two or more antihypertensivesPhase III cardiovascular outcomes trial expected to be initiated by the end of the year Basel, 30 August 2025 – Roche (SIX: RO, ROG; OTCQX: RHHBY) and Alnylam (Nasdaq: ALNY) today announced the decision to initiate a Phase III cardiovascular outcomes trial (CVOT) to evaluate the ability of zilebesiran, a RNAi therapeutic, to reduce the risk of major adverse cardiovascular events in patients with uncontrolled hypertension. This decision was informed by the comprehensive KARDIA Phase II programme, including KARDIA 1, KARDIA 2 and the most recent KARDIA-3 study evaluating the efficacy and safety of zilebesiran in patients with uncontrolled hypertension and high cardiovascular (CV) risk, on two to four standard of care antihypertensives. In particular, KARDIA-3 aimed to define the patient population to be investigated in the Phase III CV outcomes trial. Results of KARDIA-3 showed that a single dose of zilebesiran (300 mg every six months, subcutaneous injection) resulted in clinically meaningful placebo-adjusted reductions of office systolic blood pressure (SBP) in all comers at the month three primary endpoint (-5.0 mmHg; p=0.0431) with sustained benefits out to month six (-3.9 mmHg; 95% CI: [-8.5, 0.7]). There were no additional benefits of the 600 mg dose at month three (-3.3 mmHg; p=0.1830) or month six (-3.6 mmHg; 95% CI: [-8.2, 1.0]). The overall KARDIA-3 study did not meet the pre-specified definition for statistical significance, because of a multiplicity statistical testing approach. However, the study met the aim of identifying the patient population that could potentially benefit the most from zilebesiran and also showed encouraging safety and clinically meaningful placebo adjusted reductions in blood pressure. As observed in the KARDIA-2 Phase II study, the KARDIA-3 results support a robust benefit of combining zilebesiran with a diuretic, a commonly used antihypertensive. In an analysis of patients that were on diuretics and had a baseline BP >140 mm Hg, the placebo-adjusted reduction was -9.2mmHg; (-17.3, -1.2) at month three and -8.3mmHg (-16.4, -0.2) at month six. A precedent for enhanced blood pressure reduction conferred by this type of combination is established in both literature and clinical practice. “Zilebesiran has the potential to become a best-in-disease treatment for many patients with uncontrolled hypertension. Its blood pressure-lowering effects and twice-yearly dosing could reduce the risk of serious health complications and death,” said Levi Garraway, MD, PhD, Roche’s chief medical officer and head of Global Product Development. “Detailed analysis of our comprehensive Phase II clinical trials have informed our decision to move zilbesiran into Phase III. Despite current treatment options, up to 80% of people with hypertension do not achieve adequate blood pressure control putting them at higher risk of cardiovascular events. Therefore, additional treatment options are needed.” Zilebesiran also demonstrated encouraging safety in patients with comorbidities on multiple background therapies – more than 90% of whom were receiving treatment with an ACE inhibitor or an Angiotensin Receptor Blocker (ARB). These findings reinforce confidence in zilebesiran’s ability to be combined with standard of care antihypertensives. As a result, the ZENITH (ZilebEsiraN CardIovascular OuTcome Study in Hypertension) Phase III trial has been submitted to global regulators and is expected to be initiated by the end of 2025. ZENITH will be a CVOT enrolling approximately 11,000 patients and evaluating zilebesiran (300 mg) every six months compared to placebo in patients with uncontrolled hypertension with either established CV disease or at high risk for CV disease on two or more antihypertensives, one being a diuretic. Hypertension is the primary cause of and number one modifiable risk factor for cardiovascular disease. An estimated one in three adults, over 1,2 billion people worldwide, have hypertension and despite the wide availability of antihypertensives, up to 80% of them do not achieve adequate blood pressure control. Poor adherence to daily oral therapies is an important contributor to poor blood pressure control and CV outcomes. An effective long-acting therapy that provides continuous control of blood pressure may help to reduce the burden of uncontrolled hypertension. With its growing cardiometabolic portfolio and strong diagnostic expertise, Roche is advancing transformative standards of care to improve the lives of people living with cardiometabolic diseases as well as reducing the significant burden on healthcare systems and society. About the KARDIA-3 study KARDIA-3 (NCT06272487), the third phase II study in the KARDIA programme, included two Cohorts (A and B). Cohort A assessed zilebesiran in patients with eGFR ≥ 45 mL/min/1.73m2, while Cohort B included patients with advanced kidney dysfunction (i.e., eGFR between 30 and
— 2025 ESC/EAS Dyslipidemia Guideline Focused Update Reaffirms High Dose Icosapent Ethyl as Class IIA Recommended Therapy in High-Risk or Very High-Risk Patients Based on REDUCE-IT — DUBLIN and BRIDGEWATER, N.J., Aug. 30, 2025 (GLOBE NEWSWIRE) — Amarin Corporation plc (NASDAQ:AMRN) today highlighted three sub-analyses from the REDUCE-IT® trial describing the impact of VASCEPA®/VAZKEPA® (icosapent ethyl – IPE) administration on cardiovascular disease (CVD) risk associated with Cardiovascular-Kidney-Metabolic (CKM) syndrome, major adverse cardiovascular events (MACE) stratified by baseline apolipoprotein B (ApoB) and fasting triglyceride rich lipoprotein cholesterol (TRL-C) levels, and on hospitalizations. All three post hoc analyses showed significant reductions in cardiovascular risk and outcomes in the populations studied. The data was presented at the European Society of Cardiology (ESC) Congress 2025 in Madrid, Spain. “The REDUCE-IT data continue to yield important insights into the clinical utility of VASCEPA/VAZKEPA and how icosapent ethyl can reduce cardiovascular risk across diverse patient populations,” said Deepak L. Bhatt, MD, MPH, MBA, Director of Mount Sinai Fuster Heart Hospital. “These new analyses reinforce the robustness of the original findings and highlight the potential of icosapent ethyl to address complex conditions like cardiovascular-kidney-metabolic syndrome and lipid-driven risk. The consistency of benefit observed across multiple high-risk subgroups supports its integration into contemporary treatment strategies as a complementary therapy aimed at improving patient outcomes.” Key findings from these post hoc analyses are outlined below: Icosapent Ethyl Reduces CVD Risk in Cardiovascular-Kidney-Metabolic Syndrome: REDUCE-IT CKM Cardiovascular-kidney-metabolic syndrome is a recently defined disorder linking metabolic syndrome risk factors to chronic kidney disease and cardiovascular disease. This analysis examined the incremental CVD risk of CKM in secondary prevention patients with metabolic syndrome but without diabetes at baseline (n=2860) and the effect of IPE therapy. Subjects were divided into four baseline estimated glomerular filtration rate (eGFR) groups: ≥ 90, < 90, ≥ 60, and < 60 mL/min/1.73 m2. IPE treatment vs placebo showed a statistically significant reduction in the primary composite endpoint (CV death, nonfatal MI, nonfatal stroke, coronary revascularization, or unstable angina) in eGFR groups < 60, ≥ 60, and < 90. IPE treatment in the eGFR < 60 group showed a 44% relative risk reduction (RRR) for time to the first primary composite endpoint, hazard ration (HR) 0.56 (95% CI 0.39, 0.79), P = 0.001, absolute risk reduction (ARR) = 11.2%, number needed to treat (NNT) = 9. The analysis showed that IPE treatment reduced CVD risk in patients with CKM syndrome, thereby supporting a role for this therapy in these high CVD risk patients. Icosapent Ethyl Reduces Cardiovascular Risk Across Apolipoprotein B and Fasting Triglyceride Rich Lipoprotein Levels Increased ApoB levels due to triglyceride rich lipoproteins (TRL) are associated with increased cardiovascular risk, even when low-density lipoprotein cholesterol (LDL-C) levels are well controlled. This analysis assessed the impact of IPE on MACE (CV death, nonfatal MI, nonfatal stroke, coronary revascularization, or unstable angina) stratified by ApoB and fasting TRL-C. Relationships between quartiles of baseline ApoB concentration, baseline fasting TRL-C and risk for first MACE were analyzed. IPE treatment resulted in significant reductions in MACE, from the first through fourth baseline quartiles of ApoB: HR 0.72 (95% CI 0.58, 0.88), HR 0.73 (95% CI 0.59, 0.89), HR 0.76 (95% CI 0.63, 0.91), and HR 0.80 (95% CI 0.66, 0.97), respectively, all P ≤ 0.02. Additionally, there were significant reductions in MACE from the second through fourth baseline quartiles of TRL-C: HR 0.74 (95% CI 0.60, 0.90), HR 0.79 (95% CI 0.65, 0.96), and HR 0.68 (95% CI 0.56, 0.82), respectively, all P ≤ 0.02. This analysis showed that IPE significantly reduced MACE across all quartiles of baseline ApoB and TRL-C concentrations above the 25th percentile, supporting the use of IPE in patients with mild or moderate hypertriglyceridemia regardless of ApoB and TRL-C levels. Effects of Icosapent Ethyl on Risk and Duration of Hospitalizations and Death in REDUCE-IT This analysis of REDUCE-IT estimated the effects of IPE treatment on total hospitalizations and days lost to hospitalization and death. The analysis showed that among participants in REDUCE-IT, IPE reduced total hospitalizations and had favorable impacts on measures of days lost due to hospitalization and death. IPE significantly reduced total hospitalizations, HR 0.91 (95% CI 0.84, 0.98), P=0.017; increased the likelihood of surviving until end of study without hospitalization, odds ratio (OR) 1.12 (95% CI 1.02, 1.22), P=0.016; and had a lower rate of days lost among those who were hospitalized and/or died during follow-up, rate ratio (RR) 0.93 (95% CI 0.93, 0.94), P1% more frequent than placebo): arthralgia (2% vs 1%) and oropharyngeal pain (1% vs 0.3%).Adverse events may be reported by calling 1-855-VASCEPA or the FDA at 1-800-FDA-1088.Patients receiving VASCEPA and concomitant anticoagulants and/or anti-platelet agents should be monitored for bleeding. FULL U.S. FDA-APPROVED VASCEPA PRESCRIBING INFORMATION CAN BE FOUND AT WWW.VASCEPA.COM. Europe For further information about the Summary of Product Characteristics (SmPC) for VAZKEPA® in Europe, please visit: https://www.ema.europa.eu/en/documents/product-information/vazkepa-epar-product-information_en.pdf Globally, prescribing information varies; refer to the individual country product label for complete information. Forward-Looking Statements This press release contains forward-looking statements, which are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995, including beliefs about Amarin’s key achievements in 2024 and the potential impact and outlook for achievements in 2025 and beyond; Amarin’s 2025 financial outlook and cash position; Amarin’s overall efforts to expand access and reimbursement to VAZKEPA across global markets; expectations regarding potential strategic collaboration and licensing agreements with third parties, including our ability to attract additional collaborators, as well as our plans and strategies for entering into potential strategic collaboration and licensing agreements and the overall potential and future success of VASCEPA/VAZKEPA and Amarin that are based on the beliefs and assumptions and information currently available to Amarin. All statements other than statements of historical fact contained in this press release are forward-looking statements. These forward-looking statements are not promises or guarantees and involve substantial risks and uncertainties. A further list and description of these risks, uncertainties and other risks associated with an investment in Amarin can be found in Amarin's filings with the U.S. Securities and Exchange Commission, including Amarin’s quarterly report on Form 10-Q for the period ending June 30, 2025 and annual report on Form 10-K for the fiscal year ended 2024. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date they are made. Amarin undertakes no obligation to update or revise the information contained in its forward-looking statements, whether as a result of new information, future events or circumstances or otherwise. Amarin’s forward-looking statements do not reflect the potential impact of significant transactions the company may enter into, such as mergers, acquisitions, dispositions, joint ventures or any material agreements that Amarin may enter into, amend or terminate. Investors and others should note that Amarin communicates with its investors and the public using the company website (www.amarincorp.com), the investor relations website (www.amarincorp.com/investor-relations) including but not limited to investor presentations and investor FAQs, U.S. Securities and Exchange Commission filings, press releases, public conference calls and webcasts Availability of Other Information About Amarin Amarin communicates with its investors and the public using the company website (www.amarincorp.com) and the investor relations website (investors.amarincorp.com), including but not limited to investor presentations and FAQs, Securities and Exchange Commission filings, press releases, public conference calls and webcasts. The information that Amarin posts on these channels and websites could be deemed to be material information. As a result, Amarin encourages investors, the media and others interested in Amarin to review the information that is posted on these channels, including the investor relations website, on a regular basis. This list of channels may be updated from time to time on Amarin’s investor relations website and may include social media channels. The contents of Amarin’s website or these channels, or any other website that may be accessed from its website or these channels, shall not be deemed incorporated by reference in any filing under the Securities Act of 1933. Amarin Contact Information Media Inquiries:Tegan BerryAmarin Corporation plcPR@amarincorp.com Investor Inquiries:Bob BurrowsWestern Avenue Advisers LLCInvestor.relations@amarincorp.com 1 François Mach, Konstantinos C Koskinas, Jeanine E Roeters van Lennep, Lale Tokgözoğlu, Lina Badimon, Colin Baigent, Marianne Benn, Christoph J Binder, Alberico L Catapano, Guy G De Backer, Victoria Delgado, Natalia Fabin, Brian A Ference, Ian M Graham, Ulf Landmesser, Ulrich Laufs, Borislava Mihaylova, Børge Grønne Nordestgaard, Dimitrios J Richter, Marc S Sabatine, ESC/EAS Scientific Document Group , 2025 Focused Update of the 2019 ESC/EAS Guidelines for the management of dyslipidaemias: Developed by the task force for the management of dyslipidaemias of the European Society of Cardiology (ESC) and the European Atherosclerosis Society (EAS), European Heart Journal, 2025;, ehaf190, https://doi.org/10.1093/eurheartj/ehaf190
– Acoramidis demonstrated a significant reduction in risk of CVM through 42 months post-randomization, with a 44% hazard reduction, setting a new standard for CVM outcomes for patients with ATTR-CM – Acoramidis also demonstrated a significant 46% hazard reduction in the risk of the composite outcome of CVM or first CVH through 42 months – Acoramidis demonstrated higher rates of disease stabilization or improvement versus disease progression as compared to placebo as reflected in change from baseline in NT-proBNP and NAC Stage – In the ATTRibute-CM study, acoramidis demonstrated the most rapid benefit seen in any Phase 3 study of ATTR-CM to date in both ATTRv-CM and ATTRwt-CM patients – In as few as 3 months, the time to first event (ACM or CVH) durably separated relative to placebo- A 42% reduction in composite ACM and recurrent CVH events relative to placebo at Month 30- A 50% reduction in the cumulative frequency of CVH events relative to placebo at Month 30- Acoramidis is approved as Attruby® by the U.S. FDA and is approved as BEYONTTRA® by the European Commission, Japanese Pharmaceuticals and Medical Devices Agency and UK Medicines and Healthcare Products Regulatory Agency PALO ALTO, Calif., Aug. 30, 2025 (GLOBE NEWSWIRE) — BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the “Company”), a new type of biopharmaceutical company focused on genetic diseases, presented data from the ATTRibute-CM open label extension (OLE) through Month 42, which demonstrated a statistically significant reduction in CVM in the overall transthyretin amyloid cardiomyopathy (ATTR-CM) population. These data were presented in an oral presentation at the European Society of Cardiology (ESC) Congress 2025, taking place in Madrid, Spain from August 29 – September 1, 2025. Acoramidis is a selective, small molecule, orally administered, near-complete (≥90%) transthyretin (TTR) stabilizer. “These results from the ATTRibute-CM open-label extension study add to the growing body of evidence supporting acoramidis as a potentially transformative therapy for patients with ATTR-CM. At 42 months post-randomization, acoramidis achieved an impressive 44% reduction in the hazard of cardiovascular mortality. These findings highlight acoramidis’s meaningful impact on patient outcomes and address a critical unmet need for those living with ATTR-CM,” said Kevin Alexander, M.D. of Stanford University School of Medicine, USA. Details from the oral presentation, Acoramidis Reduces Cardiovascular Mortality (CVM): Results at Month 42 from the ATTRibute-CM Open-Label Extension (OLE) Study, presented by Dr. Alexander, included: Acoramidis treatment administered for 42 months led to a 44% hazard reduction in CVM compared with the placebo to acoramidis treatment groupThese findings demonstrate the long-term clinical benefits of acoramidis for reducing CVM in ATTR-CM, and the importance of early and sustained treatment In addition to the oral presentation, two posters were shared with additional analyses at 30 month results from ATTRibute-CM. These findings included: Acoramidis-mediated Improvement in NT-proBNP at Month 30 Compared with Placebo in Patients with ATTR-CM: Results from the ATTRibute-CM Study, presented by Nitasha Sarswat, M.D. of UChicago Medicine, USA Acoramidis treatment resulted in improved or stable N-terminal pro-B-type natriuretic peptide (NT-proBNP), a biomarker used to assess heart failure and monitor its progression, at Month 30 in about 50% of study participants compared with fewer than 20% with placebo, indicating a clinically meaningful improvement in NT-proBNP and better stabilization of their disease Acoramidis Has a Beneficial Effect Compared with Placebo on Change from Baseline in NAC ATTR Stage at Month 30 in Patients with ATTR-CM: Results from the ATTRibute-CM Study, presented by Julian Gillmore, M.D., Ph.D., University College London’s Centre for Amyloidosis, UK Acoramidis treatment resulted in a greater proportion of participants whose NAC Stage, a staging system developed by the National Amyloidosis Centre (NAC) to classify patients based on disease severity, protect against heart damage and improve cardiovascular function improved or remained stable at Month 30 compared with placebo, indicating better stabilization of their disease Bayer, BridgeBio’s European licensing partner, will also have a poster shared by Francesco Cappelli, M.D. of Careggi University Hospital, Florence, IT, showing acoramidis achieved clinically meaningful improvements from baseline in NT-proBNP and/or six-minute walk distance test across 30 months in patients with ATTR-CM. In March 2024, BridgeBio entered into an exclusive licensing agreement with Bayer Consumer Care AG to commercialize BEYONTTRA in Europe for the treatment for ATTR‑CM. Acoramidis is approved as Attruby by the U.S. FDA and is approved as BEYONTTRA by the European Commission, Japanese Pharmaceuticals and Medical Devices Agency, and the UK Medicines and Healthcare Products Regulatory Agency with all labels specifying near-complete stabilization of TTR. More data on the benefit of Attruby for ATTR-CM patients are planned for future medical meetings. About Attruby™ (acoramidis) INDICATION Attruby is a transthyretin stabilizer indicated for the treatment of the cardiomyopathy of wild-type or variant transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular death and cardiovascular-related hospitalization. IMPORTANT SAFETY INFORMATION Adverse Reactions Diarrhea (11.6% vs 7.6%) and upper abdominal pain (5.5% vs 1.4%) were reported in patients treated with Attruby versus placebo, respectively. The majority of these adverse reactions were mild and resolved without drug discontinuation. Discontinuation rates due to adverse events were similar between patients treated with Attruby versus placebo (9.3% and 8.5%, respectively). About BridgeBio Pharma, Inc.BridgeBio Pharma, Inc. (BridgeBio) is a new type of biopharmaceutical company founded to discover, create, test, and deliver transformative medicines to treat patients who suffer from genetic diseases. BridgeBio’s pipeline of development programs ranges from early science to advanced clinical trials. BridgeBio was founded in 2015 and its team of experienced drug discoverers, developers and innovators are committed to applying advances in genetic medicine to help patients as quickly as possible. For more information visit bridgebio.com and follow us on LinkedIn, Twitter, Facebook, Instagram, and YouTube. BridgeBio Forward-Looking StatementsThis press release contains forward-looking statements. Statements in this press release may include statements that are not historical facts and are considered forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended (the “Securities Act”), and Section 21E of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), which are usually identified by the use of words such as “anticipates,” “believes,” “continues,” “could,” “estimates,” “expects,” “hopes,” “intends,” “may,” “plans,” “projects,” “potential,” “seeks,” “should,” “will,” and variations of such words or similar expressions. BridgeBio intends these forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in Section 27A of the Securities Act and Section 21E of the Exchange Act. These forward-looking statements, including statements regarding the potential of acoramidis as a transformative therapy for patients with ATTR-CM and its potential to result in better stabilization of disease, reflect BridgeBio’s current views about its plans, intentions, expectations, and strategies, which are based on the information currently available to BridgeBio and on assumptions it has made. Although BridgeBio believes that its plans, intentions, expectations, and strategies as reflected in or suggested by these forward-looking statements are reasonable, it can give no assurance that such plans, intentions, expectations, or strategies will be attained or achieved. Furthermore, actual results may differ materially from those described in the forward-looking statements and will be affected by a number of risks, uncertainties, and assumptions, including, but not limited to: the risks associated with BridgeBio’s dependence on third parties for development; regulatory authorities requiring additional studies or data to support the continued or expanded commercialization of acoramidis; whether data and results meet regulatory requirements or are sufficient for continued development, review, or approval; and whether other regulatory agencies agree with BridgeBio’s strategies or data interpretations. These risks also include impacts from global health emergencies, such as delays in regulatory reviews and other activities, manufacturing and supply chain interruptions, adverse effects on healthcare systems, and disruption of the global economy; and the impacts of macroeconomic and geopolitical events, including changing conditions from hostilities in Ukraine and in Israel and the Gaza Strip, increasing inflation rates, and fluctuating interest rates on BridgeBio’s operations and expectations. Additional risks are described in the Risk Factors section of BridgeBio’s most recent Annual Report on Form 10-K, Quarterly Report on Form 10-Q, and other filings with the U.S. Securities and Exchange Commission. Moreover, BridgeBio operates in a very competitive and rapidly changing environment in which new risks emerge from time to time. These forward-looking statements are based upon the current expectations and beliefs of BridgeBio’s management as of the date of this press release and are subject to certain risks and uncertainties that could cause actual results to differ materially from those described in these statements. Except as required by applicable law, BridgeBio assumes no obligation to publicly update any forward-looking statements, whether as a result of new information, future events, or otherwise. BridgeBio Media Contact: Bubba Murarka, Executive Vice President, Corporate Development contact@bridgebio.com(650)-789-8220 BridgeBio Investor Contact: Chinmay Shukla, Senior Vice President, Strategic Finance ir@bridgebio.com
Positive Trial Demonstrates Superiority of Aficamten to Standard-of-Care Beta-Blocker Metoprolol Primary Endpoint Result Consistent Across All Prespecified Subgroups Company to Host Investor Event and Webcast Tuesday September 2, 2025, at 8:00 AM Eastern Time SOUTH SAN FRANCISCO, Calif., Aug. 30, 2025 (GLOBE NEWSWIRE) — Cytokinetics, Incorporated (Nasdaq: CYTK) today announced that primary results from MAPLE-HCM (Metoprolol vs Aficamten in Patients with LVOT Obstruction on Exercise Capacity in HCM) were presented in a Hot Line Session at the European Society of Cardiology Congress 2025 in Madrid, Spain, and simultaneously published in The New England Journal of Medicine.1 MAPLE-HCM is a Phase 3 randomized, double-blind, active-comparator clinical trial of aficamten compared to metoprolol in patients with symptomatic obstructive hypertrophic cardiomyopathy (oHCM). MAPLE-HCM enrolled 175 patients, randomized on a 1:1 basis to receive aficamten or metoprolol as monotherapy. Compared to SEQUOIA-HCM, the pivotal Phase 3 clinical trial of aficamten, MAPLE-HCM was designed to include patients with less severe oHCM, enrolling patients without obstruction at rest and with higher predicted peak oxygen uptake (pVO2). “This important study has the potential to inform our approach to treating obstructive HCM, as MAPLE-HCM provides the field its first look at a cardiac myosin inhibitor compared directly to a beta-blocker,” said Pablo Garcia-Pavia, M.D., Ph.D., Head of the Inherited Cardiac Diseases and Heart Failure Unit, Department of Cardiology, Hospital Universitario Puerta de Hierro and Full Professor, Centro Nacional de Investigaciones Cardiovasculares in Madrid, Spain. “In showing that aficamten is superior to metoprolol on all clinically relevant efficacy endpoints, these results call into question the reliance on beta-blockers as the initial treatment modality for obstructive HCM that has prevailed for over 60 years.” “These results demonstrate that aficamten meaningfully improves exercise capacity in patients with obstructive HCM while treatment with metoprolol resulted in a meaningful reduction in exercise capacity,” said Fady I. Malik, M.D., Ph.D., Cytokinetics’ Executive Vice President of Research & Development. “The clinical difference in the two treatments is reinforced by the effect of aficamten on the secondary endpoints. Compared to first-line standard-of-care metoprolol, treatment with aficamten had a larger effect on measures of symptoms, functional class, and LVOT gradients. Importantly, these effects were achieved in a broader patient population with oHCM than previously studied in SEQUOIA-HCM, inclusive of patients in MAPLE-HCM with less severe disease as measured by objective metrics of disease burden.” Aficamten is an investigational drug candidate currently under regulatory review in the U.S; the Food and Drug Administration (FDA) is reviewing a New Drug Application (NDA) for aficamten with a Prescription Drug User Fee Act (PDUFA) target action date of December 26, 2025. Results of MAPLE-HCM The primary endpoint in MAPLE-HCM was the mean change from baseline in pVO2 for aficamten compared to metoprolol after 24 weeks of treatment. For aficamten, the mean change in pVO2 from baseline to Week 24 was +1.1 mL/kg/min (95% CI 0.5 to 1.7) and for metoprolol was -1.2 mL/kg/min (95% CI -1.7 to -0.8). The primary endpoint was statistically significant with a least-squares mean (LSM) difference between groups of 2.3 mL/kg/min (95% CI 1.5 to 3.1; p
HONG KONG, Aug. 29, 2025 /PRNewswire/ — LifeTech Scientific Corporation (“LifeTech” or the “Company”, together with its subsidiaries, the “Group”, stock code: 1302.HK), a medical device company specializing in minimally invasive interventional solutions for cardio-cerebrovascular and…
CHICAGO–(BUSINESS WIRE)–GE HealthCare (Nasdaq: GEHC) today announced the launch of the Vivid™ Pioneer, its most advanced, ultra-premium and adaptive cardiovascular ultrasound system yet, completely redesigned to support clinicians with extraordinary imaging in 2D, 4D and color flow, streamlined workflow, and enhanced diagnostic confidence. The ultrasound system recently received CE Mark […]
US FDA 510(k) submission remains on track for calendar Q3 2025Successful validation testing of Novasight Hybrid™ system with key opinion leaders Recent academic publications underscore importance of intravascular imaging and potential of hybrid IVUS / OCT$20 million financing completed in April strengthens balance sheet and supports anticipated U.S. launch TORONTO, Aug. 29, 2025 (GLOBE NEWSWIRE) — Conavi Medical Corp. (TSXV: CNVI) (OTCQB: CNVIF) (“Conavi Medical” or the “Company”), a commercial-stage medical device company focused on designing, manufacturing, and marketing imaging technologies to guide minimally invasive cardiovascular procedures, today announced its financial results for the fiscal quarter ended June 30, 2025. “Fiscal Q3 was an important quarter as we completed validation testing with leading interventional cardiologists, giving us confidence that our next generation Novasight™ is designed to be a best-in-class product,” said Thomas Looby, CEO of Conavi Medical. “We remain on track to submit our 510(k) application to the U.S. FDA in calendar Q3 2025, a key milestone that positions us for an anticipated U.S. launch in the first half of calendar 2026. Importantly, the $20 million financing completed in April strengthens our position as we work towards these key milestones.” A recent publication in the Journal of the American College of Cardiology (JACC) underscores the rising importance of intravascular imaging in the treatment of patients with complex coronary artery disease, a market that continues to grow as physicians increasingly rely on imaging to guide better outcomes. Combined with hands-on validation by experts using our system, these developments highlight the strong clinical and commercial momentum behind our hybrid IVUS / OCT technology and its potential to transform patient care. Business Highlights: Successful Validation Testing with Key Opinion Leaders: In July 2025, Conavi conducted two pre-clinical animal experiments with several leading interventional cardiologists, confirming that next generation Novasight meets key customer specifications for usability, imaging performance, and workflow integration. These studies validated market readiness and established that the system is competitive with best-in-class stand-alone IVUS and OCT solutions. 510(k) Regulatory Submission Remains on Track: The Company remains on track to submit its 510(k) application to the U.S. FDA in Q3 of calendar 2025, a critical milestone toward its planned U.S. launch in H1 calendar 2026. Transfer to Production Underway: Conavi advanced preparations to transition next generation Novasight from development to manufacturing. Transfer to production is expected to be completed to coincide with the planned U.S. launch in H1 calendar 2026. Peer-Reviewed JSCAI Publication: In July 2025, Journal of the Society for Cardiovascular Angiography & Interventions (JSCAI) published a case study with images from the first-generation Novasight system highlighting hybrid IVUS / OCT imaging as a breakthrough modality for guiding complex coronary interventions, further validating Conavi’s unique clinical approach. $20 Million Financing Strengthens Balance Sheet: In April 2025, Conavi completed an upsized equity offering for gross proceeds of $20 million. Led by U.S. institutional investors, the financing provides resources to complete the regulatory submission, advance transfer to production, and prepare for anticipated U.S. commercial launch of Novasight. Upcoming Targeted Milestones (calendar dates): Q3 2025 Planned submission of next generation Novasight U.S. FDA 510(k) application. Q4 2025 Publish additional whitepapers and case studies to drive awareness. H1 2026 Expected FDA clearance and U.S. commercial launch of next generation Novasight. Fiscal Q3 2025 Financial Results All amounts are in Canadian dollars unless specified otherwise: As previously reported, in the two preceding fiscal years and in FY Q2 2025, the Company focused on development of the next-generation Novasight system and on incorporating clinical user feedback from its earlier Novasight system. This focus and shift in resources continued into FY Q3 2025. For the quarter ended June 30, 2025, the Company recorded revenue of $63,000 compared to $401,000 for the same period in the prior year. Operating expenses for the three months ended June 30, 2025, were $4.7 million, compared to $8.8 million for the same period in the prior year. The operating loss for Q3 FY 2025 was $4.6 million, compared to $8.9 million for Q3 FY 2024. This drop largely stems from a reduction in research & development expenses, which were $3.2 million in FY Q3 2025, compared to $6.3 million in the same period in FY 2024, reflecting a reduction in costs as the next-generation system nears FDA submission. The Q3 FY 2025 net loss was $3.6 million, or $0.05 per common share, compared to a net loss of $13.0 million, or $2.12 per common share, in the three-month period ended June 30, 2024. The net loss in Q3 2025 was principally due to operating costs, partially offset by a gain in the fair value of warrant liability; the loss in Q3 FY 2024 was principally due to operating costs. In both periods, the majority of operating costs were attributable to research & development activities. As of June 30, 2025, cash and cash equivalents were $11.3 million, up from $0.4 million as of September 30, 2024. For detailed financial results, please refer to Conavi Medical’s filings on SEDAR+ and the Company’s website www.conavi.com. Additional Disclosure Regarding $20M Public Offering Further to the Company’s press release dated April 23, 2025 regarding the closing of its public equity offering for aggregate gross proceeds of $20 million (the “Offering”), the Company confirms that in connection with the Offering, it paid a cash commission of $1,034,657.75 and issued 2,408,350 compensation options to Bloom Burton Securities Inc., the sole and exclusive agent for the Offering. In addition, in connection with the Offering, 77,700 and 35,000 compensation options were issued to Raymond James Ltd. and Research Capital Corporation, respectively. About Conavi MedicalConavi Medical is focused on designing, manufacturing, and marketing imaging technologies to guide common minimally invasive cardiovascular procedures. Its patented Novasight Hybrid™ System is the first to combine intravascular ultrasound (IVUS) and optical coherence tomography (OCT) into a single device, enabling simultaneous and co-registered imaging of coronary arteries. The Novasight Hybrid™ System has regulatory clearance in the U.S., Canada, China, and Japan. For more information, visit conavi.com. Cautionary Statement Regarding Forward-Looking Information This news release contains “forward-looking statements” within the meaning of applicable Canadian and U.S. securities laws, which reflect the current expectations of management of Conavi’s future growth, results of operations, performance and business prospects and opportunities. Forward-looking statements are frequently, but not always, identified by words such as “may”, “would”, “could”, “will”, “anticipate”, “believe”, “plan”, “expect”, “intend”, “estimate”, “potential for” and similar expressions, although these words may not be present in all forward-looking statements. Forward-looking statements that appear in this release may include, without limitation, references to Conavi’s plans for the commercialization of its Novasight Hybrid™ System and expected FDA clearance and the commercial launch of next generation Novasight in the U.S. These forward-looking statements reflect management’s current beliefs with respect to future events, and are based on information currently available to management that, while considered reasonable by management as of the date on which the statements are made, are inherently subject to significant business, economic and competitive uncertainties and contingencies which could result in actions, events, conditions, results, performance or achievements to be materially different from those projected in the forward-looking statements. Forward-looking statements involve significant risks, uncertainties and assumptions and many factors could cause Conavi’s actual results, performance or achievements to be materially different from any future results, performance or achievements that may be expressed or implied by such forward-looking statements. Such factors and assumptions include, but are not limited to, Conavi’s ability to retain key personnel; its ability to execute on its business plans and strategies; and other factors listed in the “Risk Factors” sections of the joint information circular of Conavi dated August 30, 2024 and in the final short form prospectus of Conavi dated April 15, 2025 (each of which may be viewed at www.sedarplus.com). Should one or more of these risks or uncertainties materialize, or should assumptions underlying the forward-looking statements prove incorrect, actual results, performance, or achievements may vary materially from those expressed or implied by the forward-looking statements contained in this news release. These factors should be considered carefully, and prospective investors should not place undue reliance on the forward-looking statements. Although the forward-looking statements contained in the news release are based upon what management currently believes to be reasonable assumptions and Conavi has attempted to identify important factors that could cause actual actions, events, conditions, results, performance or achievements to differ materially from those described in forward-looking statements, Conavi cannot assure prospective investors that actual results, performance or achievements will be consistent with these forward-looking statements. Except as required by law, Conavi expressly disclaims any intention or obligation to update or revise any forward-looking statements whether as a result of new information, future events or otherwise. Accordingly, investors should not place undue reliance on forward-looking statements. All the forward-looking statements are expressly qualified by the foregoing cautionary statements. Neither the TSX Venture Exchange nor its Regulation Services Provider (as that term is defined in the policies of the TSX Venture Exchange) accepts responsibility for the adequacy or accuracy of this press release. Contact:Stefano PiconeChief Financial Officer(416) 483-0100
