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Cadrenal Therapeutics Announces Positive Outcome from FDA Type D Meeting for Phase 3 Registration Study of CAD-1005 in Heparin-Induced Thrombocytopenia

Coronary/Structural Heart Regulatory by 0 comments

FDA Alignment on Primary Endpoint and Path Forward for Phase 3 Registration Study CAD-1005 Targets a Significant Unmet Need, with Approximately 50,000 Confirmed Acute HIT Diagnoses Annually in the U.S. and an Estimated $2 Billion in Peak Annual Revenue Opportunity PONTE VEDRA, Fla., Aug. 31, 2026 (GLOBE NEWSWIRE) — Cadrenal Therapeutics, Inc. (Nasdaq: CVKD), a late-stage biopharmaceutical company advancing specialized therapies for critical care cardiology and orphan cardiovascular conditions, today announced positive feedback from a Type D Meeting with the U.S. Food and Drug Administration (FDA) held on July 28, 2026. During the meeting, Cadrenal and the FDA aligned on key aspects of the protocol and Statistical Analysis Plan (SAP) for the Phase 3 registrational study of CAD-1005, the Company’s first-in-class 12-lipoxygenase (12-LOX) inhibitor in development to treat heparin-induced thrombocytopenia (HIT). HIT is a potentially life-threatening immune reaction to heparin, a widely used blood thinner, and can lead to dangerous blood clots. In the U.S., heparin-induced thrombocytopenia (HIT) is a high-stakes emergency that affects approximately 50,000 patients with acute HIT each year. Current therapeutic options rely on standard anticoagulants to reduce thrombotic risk; however, they do not target the underlying immune mechanisms that drive this destructive cardiovascular cascade. CAD-1005 is a novel 12-LOX inhibitor designed to halt the core immune signaling pathway that drives platelet activation and vascular thrombosis. Developed as an essential add-on to standard anticoagulation, CAD-1005 targets a critical population and is projected to generate $2 billion in peak annual revenue. During the Type D meeting, the FDA agreed on an optimized definition of worsening HIT for the primary endpoint, based on progression of thrombotic events through Day 14 of treatment or hospital discharge. To ensure high-quality, reliable endpoint evaluation across clinical sites, the worsening component of the primary endpoint will also include extension of an existing thrombus into a new vascular segment or bed, avoiding potential site-to-site variability from manual size measurements. The updated composite primary endpoint will measure the proportion of Serotonin Release Assay-positive (SRA+) participants with adjudicated new or worsening composite thromboembolic events (CTEs) through Day 14 or hospital discharge. Additionally, the FDA agreed to use placebo control in the Phase 3 trial, with standard anticoagulation therapeutics for both the CAD-1005 and placebo control arms. “We are very pleased with the collaborative, constructive feedback from the FDA during this Type D meeting,” said Quang X. Pham, Chief Executive Officer of Cadrenal Therapeutics. “Securing agreement on the primary endpoint definition and the blinding protocols for our saline control provides greater clarity on the regulatory path forward for CAD-1005. We have incorporated the Agency’s recommendations into our Phase 3 protocol and Statistical Analysis Plan, strengthening the design of a registration study intended to evaluate whether CAD-1005 can reduce dangerous thrombotic events that persist in patients with HIT despite current anticoagulant therapies.” The Phase 3 trial design will also assess bleeding as a major safety endpoint using standard International Society on Thrombosis and Haemostasis (ISTH) criteria. All safety analyses will be conducted in the safety population of patients who receive at least one dose of the study drug. About Cadrenal Therapeutics, Inc. Cadrenal Therapeutics, Inc. is a late-stage biopharmaceutical company advancing specialized therapies for critical care cardiology and orphan cardiovascular conditions. The Company’s pipeline includes CAD-1005, tecarfarin, and frunexian. CAD-1005 is a novel investigational therapeutic in development for the treatment of heparin-induced thrombocytopenia (HIT) and Cardiac Surgery-Associated Acute Kidney Injury (CSA-AKI). CAD-1005 is designed to selectively inhibit 12-lipoxygenase (12-LOX), an enzyme central to platelet immune activation and thrombo-inflammatory signaling in HIT. CAD-1005 is intended to be used alongside existing standards of care and is being developed to address the underlying biological mechanisms that drive disease progression. CAD-1005 has an Orphan Drug Designation (“ODD”) from the U.S. Food and Drug Administration (“FDA”) for prophylaxis of thrombosis in patients with HIT, FDA Fast Track designation for the treatment and prevention of HIT, and an orphan designation from the European Medicines Agency for the treatment of platelet-activating factor 4 disorders. Second-generation 12-LOX oral therapeutics (CAD-2000) are also in development for chronic indications. The Company’s broader pipeline includes tecarfarin, a late-stage oral vitamin K antagonist designed to prevent heart attacks, strokes, and deaths from blood clots in patients requiring chronic anticoagulation, including those with end-stage kidney disease and atrial fibrillation, those with left ventricular assist devices, and potentially those with Kawasaki disease (KD), an acute, self-limited, febrile illness that primarily affects children under 5 years old and is the leading cause of acquired heart disease in developed countries. The Company recently submitted a request to the FDA for Rare Pediatric Disease Designation (RPDD) for tecarfarin for “Prevention of the Formation of Life-Threatening Blood Clots Inside Coronary Artery Aneurysms in Children with Kawasaki Disease”. Tecarfarin has also received Orphan Drug and Fast Track designations from the FDA. For more information, visit https://www.cadrenal.com/ and connect with the Company on LinkedIn. Safe Harbor Any statements in this press release about future expectations, plans, and prospects, as well as any other statements regarding matters that are not historical facts, may constitute “forward-looking statements.” The words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potentially,” “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. These statements include, without limitation, statements regarding the planned Phase 3 registration study of CAD-1005, the development of CAD-1005 to treat HIT; CAD-1005 potentially halting the core immune signaling pathway that drives platelet activation and vascular thrombosis; CAD-1005 being an essential add-on to standard anticoagulation; CAD-1005 unlocking a projected $2 billion in peak annual revenue; the worsening component of the primary endpoint of optimized definition of worsening HIT assessing extension of an existing thrombus into a new vascular segment or bed, avoiding potential site-to-site variability from manual size measurements; the updated composite primary endpoint measuring the proportion of Serotonin Release Assay-positive (SRA+) participants with adjudicated new or worsening composite thromboembolic events (CTEs) through Day 14 or hospital discharge; the regulatory path forward for CAD-1005; the registration study evaluating whether CAD-1005 can reduce dangerous thrombotic events that continue to occur in patients with HIT despite current anticoagulant therapies; the Phase 3 trial design evaluating bleeding as a major safety endpoint using standard International Society on Thrombosis and Haemostasis (ISTH) criteria; all safety analyses in the Phase 3 trial being conducted in the true safety population of patients who receive at least one dose of the study drug; the Company advancing specialized therapies for critical care cardiology and orphan cardiovascular conditions; CAD-1005 being successfully developed to treat HIT and CSA-AKI; CAD-1005 selectively inhibiting 12-LOX, an enzyme central to platelet immune activation and thrombo-inflammatory signaling in HIT; CAD-1005 being intended to be used alongside existing standards of care and being developed to address the underlying biological mechanisms that drive disease progression; second-generation 12-LOX oral therapeutics (CAD-2000) being developed for chronic indications; tecarfarin, a late-stage oral vitamin K antagonist, potentially preventing heart attacks, strokes, and deaths from blood clots in patients requiring chronic anticoagulation, including those with end-stage kidney disease and atrial fibrillation, those with left ventricular assist devices, and potentially those with Kawasaki disease; and the FDA’s ultimate decision regarding the Company’s request for RPDD for tecarfarin for the prevention of life-threatening blood clots inside coronary artery aneurysms in children with Kawasaki Disease; Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including the Company’s ability to advance its programs to clinical trial readiness; the Company’s ability to enter into development, licensing, and commercialization transactions for CAD-1005, frunexian, and tecarfarin; the Company’s ability to secure nondilutive grants to advance its programs; and the other risk factors described in the Company’s Annual Report on Form 10-K for the year ended December 31, 2025, and the Company’s subsequent filings with the Securities and Exchange Commission, including subsequent periodic reports on Quarterly Reports on Form 10-Q and Current Reports on Form 8-K. Any forward-looking statements contained in this press release speak only as of the date hereof and, except as required by federal securities laws, the Company specifically disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events, or otherwise. For more information, please contact: Lytham Partners, LLC Robert Blum, Managing Partner 602-889-9700 CVKD@lythampartners.com

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Beyond Air® Receives FDA Breakthrough Device Designation for LungFit® GO to Treat Nontuberculous Mycobacterial Pulmonary Disease (NTM-PD)

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GARDEN CITY, N.Y., Aug. 31, 2026 (GLOBE NEWSWIRE) — Beyond Air, Inc. (NASDAQ: XAIR) (“Beyond Air” or the “Company”), a commercial-stage medical device and biopharmaceutical company focused on harnessing the power of nitric oxide (NO) to improve patients’ lives, today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Device Designation to LungFit GO for the treatment of nontuberculous mycobacterial pulmonary disease (NTM-PD). The Breakthrough Device Designation underscores the significant unmet need in NTM-PD and builds upon clinical data generated with LungFit GO within this patient population. Results from the Company’s pilot study, which were published in the peer-reviewed journal Annals of the American Thoracic Society, showed that that adult subjects with chronic refractory NTM lung disease were able to safely self-administer high-dose intermittent inhaled nitric oxide therapy at home, with high treatment compliance and a favorable safety profile. The study also showed signs of meaningful improvement in a subset of patients across quality-of-life domains, along with a trend toward reduced mycobacterial burden, including one instance of culture conversion. “LungFit GO has the potential to transform nitric oxide from a hospital-based therapy into a practical home-based treatment platform for chronic pulmonary disease and represents a valuable asset within Beyond Air’s broader nitric oxide platform,” said Robert Goodman, Chief Executive Officer of Beyond Air. “We are excited to receive Breakthrough Device Designation, which validates both the significant unmet need facing patients with NTM-PD and the encouraging clinical experience generated to date with LungFit GO. Our focus and lead resource allocation remain firmly centered on our commercial business, however, this designation enhances the value of LungFit GO and expands the options available to us as we determine the best path forward for the asset, including through enhanced interactions with the FDA.” “While inhaled nitric oxide has been used in medicine for many years, its use has largely been confined to the hospital setting,” stated Kenneth Olivier, MD, MPH, Professor of Medicine and Director, Bronchiectasis/NTM Care and Research Center at the University of North Carolina – Chapel Hill. “The ability to deliver high-dose intermittent inhaled nitric oxide through a portable system in the home has the potential to address an important unmet need for patients with NTM-PD, who often require prolonged treatment. Breakthrough Device Designation is an encouraging step toward further evaluating this innovative approach in larger clinical studies.” Beyond Air plans to leverage the enhanced interactions available through the FDA’s Breakthrough Devices Program as it finalizes the design of a future clinical study for LungFit GO in NTM-PD. The Company believes the designation has the potential to streamline the clinical development process and support more efficient advancement of the program toward potential regulatory approval. FDA grants Breakthrough Device Designation when a device provides for more effective treatment or diagnosis of life-threatening or irreversibly debilitating human disease or conditions. The Breakthrough Device Designation is designed to provide timely access to medical devices to speed up development, assessment, and review for FDA approval. NTM-PD is a rare and serious chronic bacterial lung infection associated with substantial morbidity and mortality. The disease is considered an emerging global public health concern, as many infections are difficult to treat and often require prolonged multidrug antibiotic regimens that may continue for up to two years. These complex treatment courses can be difficult to tolerate, are associated with significant adverse events, and can have limited success in difficult-to-treat infections, including Mycobacterium abscessus complex (MABSC) and refractory Mycobacterium avium complex (MAC). As a result, NTM-PD remains a disease with significant unmet medical need, underscoring the need for novel therapeutic approaches. About Beyond Air®, Inc. Beyond Air is a commercial-stage medical device and biopharmaceutical company dedicated to harnessing the power of endogenous and exogenous nitric oxide (NO) to improve the lives of patients suffering from respiratory illnesses, neurological disorders, and solid tumors. The Company has received FDA approval and CE Mark for its first system, LungFit PH, for the treatment of term and near-term neonates with hypoxic respiratory failure. For more information, visit www.beyondair.net. About LungFit® Beyond Air’s LungFit is a cylinder-free, phasic flow generator and delivery system designated as a medical device by the U.S. Food and Drug Administration (FDA). The ventilator-compatible version of the device can generate NO from ambient air on demand for delivery to the lungs at concentrations ranging from 1 ppm to 80 ppm. The LungFit system could potentially replace large, high-pressure NO cylinders, providing significant advantages in the hospital setting, including greatly reducing inventory and storage requirements, improving overall safety by eliminating NO2 purging steps, and offering other operational benefits. LungFit can also deliver NO at concentrations at or above 80 ppm for potentially treating severe acute lung infections in the hospital setting (e.g., COVID-19, bronchiolitis) and chronic, refractory lung infections in the home setting (e.g., NTM). With the elimination of cylinders, Beyond Air intends to offer NO treatment in the home setting. About LungFit GO LungFit GO is an investigational device that has not been cleared or approved for commercial distribution by the FDA or any other regulatory authority. LungFit Go will be a portable device that weighs only ~20 lbs. and operates with a standard electrical outlet (120-240 volts). Since NO is generated from ambient air that flows through a reaction chamber, there is an unlimited supply. Beyond Air’s proprietary NO2 filters are required for the system to generate and safely deliver NO. Toxic levels of NO2 can result from high concentrations of NO without proper filtration. The filters are equipped with an RFID chip which programs the system with respect to NO concentration, flow rate and duration of therapy. The Company believes this design provides maximum flexibility for NO administration. Filters are single use and there are no special requirements for disposal. Alarms monitor system performance. *Beyond Air’s LungFit PH is approved for commercial use in the United States, European Union, and many other countries around the world. Beyond Air’s other LungFit systems are not approved for commercial use. Beyond Air is not suggesting or promoting NO use over 80 ppm, or use in the home setting, outside of an authorized investigational or clinical study setting. About Nitric Oxide Nitric Oxide (NO) is a potent molecule, naturally synthesized in the human body, proven to play a critical role in a broad array of biological functions. In the airways, NO targets the vascular smooth muscle cells that surround the small resistance arteries in the lungs. Currently, exogenous inhaled NO is used in adult respiratory distress syndrome, post certain cardiac surgeries and persistent pulmonary hypertension of the newborn to treat hypoxemia. Additionally, NO is believed to play a key role in the innate immune system and in vitro studies suggest that NO possesses anti-microbial activity not only against common bacteria, including both gram-positive and gram-negative, but also against other diverse pathogens. Forward-Looking Statements This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, statements regarding the potential benefits of the Breakthrough Device Designation granted for LungFit GO, including its potential to accelerate the Company’s interactions with the FDA and to streamline clinical development and regulatory review; the design, timing, initiation, and completion of a future pivotal clinical study for LungFit GO in NTM-PD; and the Company’s ability to obtain regulatory approval for, and to ultimately commercialize, LungFit GO Forward-looking statements may be identified by words such as “anticipate,” “believe,” “expect,” “intend,” “plan,” “potential,” “will,” “would,” “could,” “may,” and similar expressions, or by the use of future tense. Because forward-looking statements relate to future events, they are subject to inherent risks and uncertainties, many of which are beyond the Company’s control, that could cause actual results to differ materially from those expressed or implied in such statements. These risks and uncertainties include, but are not limited to, risks related to the design, timing, initiation, and results of the Company’s planned future pivotal clinical study for LungFit GO in NTM-PD; whether Breakthrough Device Designation will result in a faster development, assessment, or review process, or in any expedited approval, for LungFit GO; the fact that Breakthrough Device Designation does not guarantee that LungFit GO will receive marketing authorization, or that any such authorization will be obtained on the timeline the Company anticipates, or at all; the Company’s ability to obtain the financial and other resources necessary to complete development of LungFit GO; and other risks described in the “Risk Factors” section of Beyond Air, Inc.’s most recent Annual Report on Form 10-K and subsequent filings with the Securities and Exchange Commission. CONTACTS: Investor Relations contactCorey Davis, Ph.D.LifeSci Advisors, LLCcdavis@lifesciadvisors.com (212) 915-2577

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Medera Presents Late-Breaking Clinical Trial Data at ESC Congress 2026 Demonstrating Durable Improvement in HFpEF Cardiac Filling Pressures Following SRD-002 Gene Therapy

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80% (8/10) met the prespecified threshold for normalization of cardiac filling pressures at 12 months, and mean peak exercise pulmonary capillary wedge pressure (PCWP) was reduced by approximately 30% at 12 months following a single intracoronary dose of SRD-002 Favorable safety profile maintained across all 10 treated patients, with no treatment-related serious adverse events BOSTON, Aug. 31, 2026 (GLOBE NEWSWIRE) — Medera Inc. (“Medera”), a clinical-stage biopharmaceutical company pioneering next-generation therapeutics for cardiovascular disease, today announced 12-month data from its ongoing MUSIC-HFpEF Phase 1/2a clinical trial were presented as a Late-Breaking Clinical Trial (LBCT) at the European Society of Cardiology (ESC) Congress 2026 taking place August 28-31 in Munich, Germany. The selection marks the fourth LBCT recognition awarded to the MUSIC-HFpEF program at major international cardiology conferences, spanning successive stages of the trial’s development. The presentation, titled “Outcomes of Intracoronary AAV1/SERCA2a Gene Therapy in Patients With Heart Failure With Preserved Ejection Fraction: 12-Month Results From the MUSIC-HFpEF Phase 1b Trial” was delivered by Marat Fudim, MD, MHS, Head of Heart Failure Unit at Duke University Medical Center, during the session “Transforming the Heart: Novel Therapies for Myocardial Disease and Heart Failure” on Monday, August 31, 2026, from 11:15 a.m. to 12:15 p.m. CEST. The open-label, dose-escalation MUSIC-HFpEF trial is evaluating a single intracoronary infusion of SRD-002, a next-generation adeno-associated virus serotype 1 (AAV1).SERCA2a — sarcoplasmic reticulum Ca²⁺-ATPase 2a, the cardiac calcium pump that is down-regulated in heart failure and whose restoration improves calcium reuptake and diastolic relaxation—gene therapy, in adults with symptomatic HFpEF. Ten patients received either low-dose SRD-002 (3×1013 vg; n=5) or high-dose SRD-002 (4.5×1013 vg; n=5). Across patients from both cohorts, 80% (8/10) met the prespecified threshold for normalization of cardiac filling pressures at 12 months, and reduction in mean peak exercise pulmonary capillary wedge pressure (PCWP) was approximately 30% at 12 months, representing one of the largest sustained reductions in exercise filling pressure reported to date in an interventional HFpEF trial. The improvements were durable and showed a dose-response, with reductions generally greater from 6 to 12 months and from low to high dose. Supporting symptomatic, quality-of-life, and functional findings were consistent with the hemodynamic results: 83% (5/6) of patients with baseline New York Heart Association (NYHA) class III symptoms improved to class II, mean improvement in Kansas City Cardiomyopathy Questionnaire (KCCQ) score was +17.8 points, and functional capacity stabilized or improved in all patients. “These 12-month results are among the first to show sustained normalization of invasive exercise hemodynamics following a gene therapy in HFpEF,” said Marat Fudim, MD, MHS, Head of Heart Failure Unit at Duke University Medical Center. “The magnitude and durability of the filling pressure reductions we observed, paired with meaningful improvements in symptoms and quality of life, support impaired calcium handling as a disease-defining mechanism in HFpEF and reinforce the case for advancing this approach into randomized evaluation.” SRD-002 continued to demonstrate a favorable safety profile across all 10 treated patients, with no treatment-related serious adverse events, dose-limiting toxicities, treatment discontinuations, or Grade 3 or higher liver enzyme elevations reported. Intracoronary delivery enabled approximately 100- to 300-fold lower vector exposure compared with systemic intravenous AAV approaches while achieving targeted myocardial delivery without routine immunosuppression. “These data reinforce what we’ve seen across our HFpEF program: a favorable safety profile paired with durable and clinically meaningful improvement in the physiological abnormalities that define the disease. Particularly encouraging is that the improvement in cardiac filling pressures continued to evolve following a single administration of SRD-002,” said Ronald Li, PhD, Co-Founder and CEO of Medera. “We believe these findings support the potential for SRD-002 to become a one-time, disease-modifying therapy for HFpEF, and we look forward to advancing the program into randomized development.” These Phase 1/2a results support advancement to a randomized Phase 2b trial to further evaluate the efficacy and safety of SRD-002 as a potential one-time therapy for patients with HFpEF. For additional information about the MUSIC-HFpEF trial, visit ClinicalTrials.gov using the study identifier NCT06061549. About Heart Failure with Preserved Ejection Fraction (HFpEF) Heart failure affects more than 64 million people worldwide, with HFpEF accounting for approximately half of all heart failure cases. Patients with HFpEF experience morbidity and mortality comparable to those with heart failure with reduced ejection fraction (HFrEF). Despite its growing prevalence, currently approved therapies primarily improve symptoms and reduce cardiovascular risk rather than directly targeting the underlying myocardial abnormalities responsible for the disease. Consequently, HFpEF remains one of the largest unmet needs in cardiovascular medicine and an area of active therapeutic development. About Medera Inc. Medera is a clinical-stage biopharmaceutical company focused on targeting difficult-to-treat and currently incurable diseases by developing next-generation therapeutics. Medera operates via two business units: Sardocor, its clinical development arm advancing a pipeline of cardiac gene therapy trials (HFpEF, HFrEF, DMD-CM), and Novoheart, its preclinical subsidiary pioneering the world’s first and award-winning “mini-Heart” technology for human-based disease modelling, drug discovery, and toxicity testing. Novoheart’s human cardiac disease models have supported the preclinical development of Medera programs that have subsequently received FDA IND clearance, Fast Track designation for HFpEF and Orphan Drug Designation for DMD-CM, and the Company is advancing a broader pipeline of gene therapy, cell therapy, and small molecule candidates. For more information, visit www.medera.bio. Contacts Ally StubinICR Healthcare – Media Relations646.667.1861Ally.stubin@icrhealthcare.com Stephanie CarringtonICR Healthcare – Investor Relations646.277.1282Stephanie.carrington@icrhealthcare.com

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XYRA Announces First Patient Dosed in Europe in the Phase 2/3 HARBOR-AF Study of Budiodarone in Paroxysmal and Persistent Atrial Fibrillation

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LOS ALTOS, California, Aug. 31, 2026 (GLOBE NEWSWIRE) — Open-label, dose-escalation study using cardiac rhythm data from implantable and wearable AF monitors allows the treating physician to titrate the dose of budiodarone monthly. First European patient dosed in CLN-209 (HARBOR-AF)Study is the first to titrate an antiarrhythmic drug against continuously monitored AF burden (AFB) and long episodes of AF (LEAF) reported in real time from a range of wearable and implanted devicesHARBOR-AF study is starting as a 100 subject Phase 2 feasibility study in EU and will extend in 2027 to US sites as one of three Phase 3 pivotal registration studies the FDA requires for approval XYRA LLC, a private biopharmaceutical company committed to developing innovative therapies for cardiac rhythm disorders with high unmet need, today announced that the first patient in Poland has been dosed with budiodarone in HARBOR-AF (CLN-209), the company’s Phase 2/3 open-label, dose-escalation study evaluating budiodarone in subjects with paroxysmal and persistent atrial fibrillation (AF). HARBOR-AF is a global, multicenter, open-label, dose-escalation Phase 2/3 study of budiodarone in approximately 600 subjects with highly symptomatic paroxysmal and persistent AF who have failed rhythm control with antiarrhythmic drugs or AF catheter ablation, or who have failed rate control. The objective of the study is to determine, in a real-world setting, the effectiveness of dose-adjusted budiodarone therapy, guided by a range of currently available AF monitoring tools, in eliminating harmful LEAF greater than 5 hours over 6 months and reducing AF burden and symptoms. It will also help define the doses to be used in a future potential randomized, placebo-controlled registration study. HARBOR-AF is starting as a Phase 2 feasibility study in the European Union and is planned to extend to the United States as one of three Phase 3 pivotal registration studies the FDA requires for approval. Following a 28-day run-in period to confirm eligibility based on AF burden, multiple long episodes of AF (LEAF), and AF symptoms, subjects will enter a 6-month treatment period starting with budiodarone 200 mg twice daily. Treating physicians may escalate the dose monthly in 200 mg twice-daily increments, up to 800 mg twice daily, according to predefined responder criteria. Responder status is defined by the absence of LEAF greater than 5 hours and by maintaining cumulative AF burden below 5 hours or 21% in any rolling 24-hour period. The study is designed to identify, for each subject, the lowest effective dose of budiodarone that controls AF symptoms and suppresses prolonged AF episodes associated with increased risk of stroke, heart failure, and progression to permanent AF. This study uniquely integrates AF monitoring into both subject eligibility assessment and treatment response, using the resulting rhythm data to guide budiodarone dose titration. XYRA is using a range of implantable and wearable AF monitoring technologies available to cardiologists. Where present, AF data are collected from pre-implanted cardiac implantable electronic devices (CIEDs), including pacemakers and implantable loop recorders; when CIED data are not available, subjects are monitored using wearable devices. Rhythm data from either source are relayed to the treating physician and used to quantify AF burden and the number and duration of LEAF episodes monthly or quarterly. At each monthly visit, physicians review AF burden, LEAF, and Responder Status data to make individualized dose-titration decisions. Further details of the study design, eligibility criteria, and monitoring methods are available in the HARBOR-AF study listing on clinicaltrials.gov (NCT number: NCT07792239). “Evidence from large epidemiological studies indicates that stroke risk is influenced not only by the overall burden of atrial fibrillation but also by its pattern, particularly, prolonged episodes or clusters of episodes which we refer to as long episodes of AF, or LEAF. These are either discrete episodes of at least five hours or periods of daily AF burden exceeding five hours both readily identified in an individual with AF using modern digital technologies and AI tools, and probably long enough time in AF to form a clot in the left atrium.” said Peter Milner, M.D., FACC., founder and managing member of XYRA. “In the HARBOR-AF study, we plan to identify patients who experience frequent LEAF using a range of reliable, FDA-approved AF-monitoring technologies, including existing pacemakers and low-cost, convenient wearable devices such as patches and wristbands. Once each patient’s AF burden, risk profile, and symptoms have been established at baseline, we plan to administer budiodarone using a dose-escalation strategy to identify the appropriate dose for that individual over six months. Treatment will be guided by the AF-monitoring systems, with the aim of eliminating LEAF, thereby establishing a safe harbor, while reducing monthly AF burden and associated symptoms. We believe this study could establish a precedent of the first use of cardiac rhythm data collected from smart watches, beyond simply using them for irregular rhythm notifications.” Professor Andrzej Przybylski, M.D., Head of Cardiology, University of Rzeszów, Poland, and principal investigator at the first activated site, added, “We are pleased to be leading the clinical trial of budiodarone in Poland, the first antiarrhythmic drug to be individually dose adjusted to maximize its efficacy and safety using the wide range of AF monitoring tools currently available to us in practice. We believe that the combination of an investigational antiarrhythmic drug and AF monitoring tools may be a relatively low-cost solution to improve AF rhythm control and improve patient outcomes.” “The use of sophisticated continuous or near-continuous heart rhythm monitoring to assess AF-related stroke risk and guide individualized dose adjustment represents an exciting and innovative approach to evaluating the efficacy of budiodarone, a drug already well known to shorten or suppress episodes of atrial fibrillation,” said Professor A. John Camm, M.D., Emeritus Professor of Clinical Cardiology at City St George’s, University of London, a globally recognized leader in atrial fibrillation and long-standing advisor to the budiodarone program. “Joining XYRA as Chief Operating Officer at such a pivotal stage in the company’s development is immensely exciting. Having spent many years leading R&D and operations in large pharma, I joined XYRA because I strongly believe in the potential of budiodarone to transform outcomes for patients with AF,” said Charlie Rampersaud, XYRA’s Chief Operating Officer. “My immediate focus is on building the operational framework required to support next year’s randomized, placebo-controlled CLN-210 study.” Budiodarone is being developed under a regulatory pathway agreed with the FDA that incorporates wearable AF monitoring into the clinical development program and proposed labeling strategy. About Budiodarone Budiodarone is currently in Phase 2/3 clinical development and is a potentially first-in-class mixed ion channel blocker with esterase metabolism, with a significantly shorter half-life than amiodarone and no evidence of tissue accumulation in human or animal studies. In clinical studies to date, budiodarone has demonstrated reductions in AF burden and prolonged AF episodes, improvements in AF symptoms, and maintenance of sinus rhythm. About Atrial Fibrillation Atrial fibrillation (AF) is the most common sustained arrhythmia in adults, affecting more than 50 million people worldwide. It is associated with an increased risk of thromboembolism, impaired cardiac function, and other morbidities. While many patients experience symptomatic AF, the condition may also first be identified following a stroke, the development of left ventricular dysfunction, or incidentally. The management of AF has evolved as evidence supporting improved outcomes with rhythm-control strategies has accumulated. In appropriately selected patients, treatment increasingly aims to restore and maintain sinus rhythm rather than relying solely on ventricular rate control. Rhythm-control options include antiarrhythmic drugs (AADs), catheter ablation, or a combination of the two. About XYRA LLC XYRA LLC is a private biopharmaceutical company committed to developing innovative therapies for cardiac rhythm disorders with high unmet need. XYRA’s mission is to make AF management comparable to that of other common cardiac conditions like hypertension and hypercholesterolemia, in which upward dose titration of effective drugs, guided by simple, widely available monitoring tools to confirm compliance and response to therapy, has consistently improved outcomes. For information: www.xyra.us Media and Investor Relations Contact: press@xyra.us LinkedIn: Xyra, LLC

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Acoramidis Demonstrates Reversal of Cardiac Structural Disease Progression and Functional Decline and Significantly Increases Days Alive and Free from Hospitalization in ATTR-CM

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– Acoramidis is the first therapy shown to potentially reverse cardiac structural disease progression and functional decline through 42 months based on CMR imaging, with up to half of patients showing clinically meaningful improvement in cardiac function in the completer analysis – Patients treated with acoramidis were observed to have an unprecedented 65 additional days alive and out of the hospital by Month 36 versus baseline placebo patients – Acoramidis demonstrated long-term efficacy and safety through 54 months across variant ATTR-CM subgroups, including p.Val142Ile and non-p.Val142Ile. These findings were simultaneously published in the European Journal of Heart Failure PALO ALTO, Calif., Aug. 30, 2026 (GLOBE NEWSWIRE) — BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the “Company”), a commercial-stage, multi-product biopharmaceutical company focused on developing medicines for genetic conditions, presented new analyses from the Phase 3 ATTRibute-CM study of Attruby® (acoramidis) in transthyretin amyloid cardiomyopathy (ATTR-CM), including the cardiac magnetic resonance imaging (CMR) substudy and the open-label extension (OLE) at the European Society of Cardiology (ESC) Congress 2026. Acoramidis is the only selective small molecule, orally administered, near-complete (≥90%) transthyretin (TTR) stabilizer. “The clinical community is excited about the potential to restore heart health found in these data. For a long time, patients living with ATTR-CM could only hope for a stop to the otherwise relentless progression of disease. These new CMR data from ATTRibute-CM shows evidence of reversal in a meaningful proportion of individuals treated with acoramidis, with roughly half showing improved left ventricular systolic function in the completer analysis, more than 2x the proportion observed in the natural history from a NAC cohort or in ATTRibute-CM participants treated with placebo. These findings support acoramidis as a therapy capable of altering the trajectory of this otherwise progressive disease,” said Marianna Fontana, M.D. of University College London, UK. “For patients and clinicians navigating ATTR-CM, this is an exciting signal that the treatment paradigm is shifting toward a therapy that could actively restore heart health rather than only manage decline.” The CMR substudy of ATTRibute-CM and its open-label extension provide the first evidence from serial CMR that a therapy can potentially reverse disease progression through Month 42. The findings presented by Awais Sheikh, MBChB of the National Amyloidosis Centre, London, UK were evaluated using two complementary analytical approaches, which found: In a completer analysis, clinically meaningful improvement from baseline in left ventricular (LV) systolic function was observed in 54% of acoramidis-treated patients versus 20% of placebo-treated patients at Month 30, and in 53% of continuous-acoramidis patients at Month 42For context, only 26% of completers in an independent natural history cohort demonstrated improved LV systolic function by Month 24 – approximately half the rate observed with acoramidis, suggesting that this magnitude of improvement falls outside the expected natural course of diseaseIn a conservative analysis, long-term acoramidis treatment was associated with clinically meaningful improvement from baseline in LV systolic function in approximately one-third of patients over 30-42 months. Improvement was observed in 34% of acoramidis-treated patients versus 9% of placebo-treated patients at Month 30 and in 30% of continuous-acoramidis patients at Month 42In addition, 46% of patients receiving continuous acoramidis demonstrated improvement from baseline in LV mass index at Month 42, providing evidence of favorable structural remodelingThese results provided sufficient evidence for BridgeBio to recently dose its first participant in ASCEND-ATTR, a Phase 3b/4 study designed to determine if acoramidis is associated with sustained improvement in myocardial structural disease progression, function and amyloid burden In a post-hoc analysis of ATTRibute-CM presented by Richard Wright, M.D. of the Pacific Heart Institute, U.S., acoramidis preserved significantly more time alive outside the hospital for patients with ATTR-CM. The analysis evaluated days lost to death and/or cardiovascular-related hospitalization (DLDCVH), a patient-centered measure that integrates all-cause mortality, cardiovascular-related hospitalizations, and length of stay into a single assessment of disease burden. Key findings included: In participants with ATTR-CM, acoramidis reduced the estimated mean percentage of DLDCVH to 7.5% versus 11.7% with placebo through Month 30Acoramidis preserved more than one month of additional time alive and out of the hospital (38 days) over 30 months with the benefit nearly doubling to 65 days (observed) over three years, and nearly tripling to up to 94 days (modelled estimates) over three years, reflecting progressive divergence in outcomes over time The p.Val142Ile genetic variant is the most common ATTR-CM genetic variant globally, disproportionately affecting individuals of Western African ancestry, with a carrier frequency of 3-4% in the U.S. Black population. Findings in the ATTRibute-CM OLE presented by Kevin Alexander, M.D. of Stanford University School of Medicine, U.S. showed continued benefit of acoramidis in 56 variant ATTR-CM (ATTRv-CM) patients, including 35 p.Val142Ile and 21 non-p.Val142Ile patients through Month 54, demonstrating: All-cause mortality (ACM) and cardiovascular mortality (CVM) were markedly lower in the continuous acoramidis arm versus placebo-to-acoramidis across both p.Val142Ile and non-p.Val142Ile variant subgroupsThrough Month 54, ACM was 30.4% with continuous acoramidis versus 66.7% with placebo-to-acoramidis in the p.Val142Ile subgroup, and 24.3% with continuous acoramidis versus 57.9% with placebo-to-acoramidis across the overall ATTRv-CM population, a consistent, more than two-fold difference in mortality favoring continuous treatmentThe ACM and CVM rates at Month 54 were notably high (~65%) in the p.Val142Ile group who were randomized to placebo in ATTRibute-CM, underscoring the substantial unmet medical need in this high-risk subgroupContinuous acoramidis achieved sustained increases in serum TTR (sTTR) and persistent attenuation of N-terminal pro-B-type natriuretic peptide (NT-proBNP) rise through Month 54 in both participants with p.Val142Ile or non-p.Val142Ile variantsThese Month 54 findings extend the survival benefit and favorable biomarker trends previously reported at Month 30, demonstrating the long-term durability of efficacy and safety of acoramidis in ATTRv-CM, including in the p.Val142Ile subgroupAcoramidis remained well tolerated through Month 54, with no new safety signals observed in the OLE In addition to the one oral presentation and two moderated posters highlighted, two additional moderated posters on acoramidis were shared at the ESC Congress 2026, including: Acoramidis Improves Health-Related Quality of Life in Wild-Type and Variant Transthyretin Amyloid Cardiomyopathy: An EQ-5D-5L Subgroup Analysis from ATTRibute-CM, presented by Emer Joyce, M.D., Ph.D. of The Mater Misericordiae University Hospital, IE Treatment with acoramidis resulted in significant and clinically meaningful benefits in health-related quality of life (HRQoL) in both wild-type ATTR-CM (ATTRwt-CM) and ATTRv-CM. Greater impact on HRQoL versus placebo was observed in participants with ATTRv-CM Improvement of Health Status with Acoramidis in Patients with Wild-Type and Variant Transthyretin Amyloid Cardiomyopathy: KCCQ Domains Analysis from the ATTRibute-CM Study, presented by Nitasha Sarswat, M.D. of University of Chicago Medical Center, U.S. In ATTRibute-CM, acoramidis attenuated the decline in heart failure-related health status versus placebo in participants with ATTRwt-CM and ATTRv-CM, with consistent benefits observed across Kansas City Cardiomyopathy Questionnaire Overall Summary (KCCQ-OS) and individual domain scores. A numerical improvement was observed across almost all KCCQ domains in acoramidis-treated participants with ATTRwt-CM and ATTRv-CM relative to placebo As part of BridgeBio’s partnership with Yale’s Cardiovascular Data Science (CarDS) Lab to advance AI networks for earlier detection of ATTR-CM, three posters were presented at the ESC Congress 2026. Findings from the partnership included: A Novel AI-Derived Digital Biomarker for Monitoring Disease Progression in ATTR-CM: First-In-Trial Use of a Computer Vision AI-ECG Algorithm within a Phase 3 Pivotal Randomized Controlled Trial, presented by Rohan Khera, M.D. of Yale School of Medicine, U.S. This showed the first deployment of a computer vision AI-ECG algorithm, operating directly on ECG data, as a digital biomarker in a RCT (ATTRibute-CM). An image-based AI-ECG algorithm demonstrated discrimination across clinical subgroups at baseline and detected differential longitudinal changes between acoramidis and placebo over 30 months. These findings support the potential role of AI-ECG derived prediction scores as a scalable digital biomarker in clinical trials and potential routine cardiovascular care A Fully Decentralized, Patient-Led Digital Registry for ATTR-CM Integrating Multisystem EHR and Wearable Data: The DISCOVER-ATTR Study, presented by Aline Pedroso, Ph.D. of Yale School of Medicine A fully decentralized, patient-led digital registry can successfully aggregate longitudinal multisystem electronic health records (EHR) data and wearable physiologic signals in ATTR-CM. Early results show substantial data yield and feasibility of longitudinal mapping of care trajectories and multimodal risk prediction, providing a blueprint for next-generation registries in rare cardiovascular diseases Nationwide U.S. Federated Deployment of Artificial Intelligence for Multimodal Screening of ATTR Cardiomyopathy: First Multicenter Analysis from the TRACE-AI Network, presented by Bruno Batinica, MBChB of Yale School of Medicine In this largest-ever deployment of AI-electrocardiogram and AI-Echo models for opportunistic retrospective screening of individuals at risk of ATTR-CM, we demonstrate a large burden of probable undiagnosed ATTR-CM with prognostic implications. Leveraging this framework for screening holds promise for enabling broad, timely identification of patients to maximize the overall benefit of new therapies Acoramidis is approved as Attruby® by the U.S. FDA and is approved as BEYONTTRA® by the European Medicines Agency (EMA), Japanese Pharmaceuticals and Medical Devices Agency, Swissmedic, the Swiss Agency for Therapeutic Products, the UK Medicines and Healthcare Products Regulatory Agency, and the Brazilian Health Regulatory Agency (ANVISA) with all labels specifying near-complete stabilization of TTR. Additional data on the benefit of Attruby for individuals with ATTR-CM is planned for future medical meetings, including Heart Failure Society of America (HFSA) Annual Scientific Meeting 2026, taking place in Phoenix, Arizona on October 9-12, 2026. About Attruby® (acoramidis)INDICATIONAttruby 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 INFORMATIONAdverse ReactionsDiarrhea (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). 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,” “estimates,” “expects,” “hopes,” “intends,” “may,” “plans,” “projects,” “remains,” “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 include statements regarding the potential clinical significance and therapeutic implications of the data presented regarding acoramidis, including the potential clinical and therapeutic implications of observed changes in cardiac structure and function and the potential for acoramidis to alter the trajectory of ATTR-CM and restore heart health; the potential utility of AI-based tools and digital biomarkers for the detection, monitoring and screening of ATTR-CM in clinical trials and clinical practice; and BridgeBio’s plans to present additional data regarding Attruby at future medical meetings. Although the Company believes that its plans, intentions, expectations and strategies as reflected in or suggested by those forward-looking statements are reasonable, the Company can give no assurance that the 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, initial and ongoing data from the Company’s clinical trials not being indicative of final data, the design and success of ongoing and planned clinical trials, the risk that results from post hoc analyses, subgroup analyses or other analyses may not be predictive of future clinical outcomes or treatment effects, that observed improvements in cardiac structure, function or other measures may not be replicated in additional analyses or studies or translate into improved long-term clinical outcomes, that the potential utility of AI-based tools and digital biomarkers may not be demonstrated in further studies or translate into routine clinical use, that plans to present additional data may change, the impacts of current macroeconomic and geopolitical events, including changing conditions from hostilities in Ukraine and in Israel and the Middle East, increasing rates of inflation and changing interest rates, on business operations and expectations, as well as those risks set forth in the Risk Factors section of the Company’s most recent Quarterly Report on Form 10-Q and Annual Report on Form 10-K and the Company’s other filings with the U.S. Securities and Exchange Commission. Moreover, the Company 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 the Company’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 the forward-looking statements. Except as required by applicable law, BridgeBio assumes no obligation to update publicly any forward-looking statements, whether as a result of new information, future events or otherwise. About BridgeBioBridgeBio exists to develop transformative medicines for genetic conditions. Millions of people worldwide living with genetic conditions lack treatment options, often because drug development for small patient populations can be commercially challenging. We aim to bridge the gap between advancements in genetic science and meaningful medicines for underserved patient populations. Our decentralized, hub-and-spoke model is designed for speed, precision, and scalability. Autonomous and empowered teams focus on individual conditions, while a central hub provides the clinical, regulatory, and commercial capabilities needed to bring innovation to market. For more information, visit bridgebio.com and follow us on LinkedIn, X, Facebook, Instagram, YouTube, and TikTok. BridgeBio Media Contact:Kaitlyn Reilly, Director, Communicationscontact@bridgebio.com(650) 789-8220 BridgeBio Investor Contact:Kristen Kelleher, Director, Investor Relationsir@bridgebio.com

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Bayer Announces Phase III REVEAL Study with Investigational PET Radiotracer Iodine 124 Evuzamitide Presented as Late-Breaker at ESC 2026

Radiology by 0 comments

WHIPPANY, N.J.–(BUSINESS WIRE)–Bayer today announced the presentation of first detailed results from the Phase III REVEAL study, an investigator-initiated study by Brigham and Women’s Hospital, that evaluated the investigational positron emission tomography (PET) radiotracer iodine 124 evuzamitide (I 124 evuzamitide) for the diagnosis of cardiac amyloidosis.1 The pivotal study met its primary endpoints in the assessment of sensitivity and specificity for the diagnosis of cardiac amyloidosis b

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CRISPR Therapeutics Presents Phase 1a Data for CTX310® Demonstrating Deep and Durable ANGPTL3 Editing, Triglyceride and LDL Lowering at ESC Congress 2026

Coronary/Structural Heart by 0 comments

-Data presented in a late-breaking presentation at the European Society of Cardiology (ESC) Congress 2026- -Phase 1a clinical data for CTX310® continued to demonstrate robust, dose-dependent reductions in circulating ANGPTL3 with a mean reduction from baseline of 79% (maximum 89%), a mean reduction in triglycerides (TG) of 48% (maximum 78%), and a mean reduction of low-density lipoprotein (LDL) of 53% (maximum 84%) at the highest dose- -CTX310 was well tolerated with no treatment-related serious adverse events, no ≥Grade 3 changes in liver transaminases, and no additional treatment-related adverse events since the previous update- -Findings simultaneously published in The New England Journal of Medicine entitled “Durability of CRISPR-Cas9 Gene Editing Targeting ANGPTL3 with CTX310”- ZUG, Switzerland and BOSTON, Aug. 28, 2026 (GLOBE NEWSWIRE) — CRISPR Therapeutics (Nasdaq: CRSP) today presented durability data from its Phase 1a clinical trial evaluating CTX310®, an investigational, in vivo CRISPR/Cas9 gene editing therapy targeting ANGPTL3. A single-course treatment with CTX310 produced deep and durable reductions in circulating ANGPTL3, triglycerides (TG), and low-density lipoprotein (LDL) that were sustained through one year of follow-up. At the highest dose, mean reductions from baseline were 79% (maximum 89%) for ANGPTL3, 48% (maximum 78%) for TG, and 53% (maximum 84%) for LDL. These extended follow-up data demonstrate the durability of CTX310’s effect and support its potential to deliver long-lasting lipid lowering after a single intravenous (IV) infusion. These data were presented today during a late breaking session at the European Society of Cardiology (ESC) Congress 2026 and extend the Phase 1 results previously presented. The data were also published today in The New England Journal of Medicine (NEJM) in a peer reviewed article entitled “Durability of CRISPR-Cas9 Gene Editing Targeting ANGPTL3 with CTX310.” CRISPR Therapeutics is advancing CTX310 in a Phase 1b clinical trial, with U.S. and ex-U.S. trials ongoing, and expects to provide an additional update on the CTX310 program in the second half of 2026. “These durability data mark an important next step for the CTX310 program and for the field of in vivo gene editing,” said Naimish Patel, M.D., Chief Medical Officer, CRISPR Therapeutics. “Evidence that a single dose can produce lasting lipid lowering is central to our goal of developing one-time treatments for cardiometabolic diseases. These results provide strong support for continued advancement of CTX310 and our broader cardiovascular gene editing portfolio, and we look forward to sharing new data from the severe hypertriglyceridemia cohort of the CTX310 Phase 1b trial in the second half of this year.” “For patients at high cardiovascular risk, the biggest challenge is often not starting therapy but staying on it, since daily medications require lifelong adherence that many patients are unable to maintain,” said Luke Laffin, M.D., principal investigator and Medical Director of the Cleveland Clinic Coordinating Center for Clinical Research. “A single infusion producing durable reductions at one-year is an encouraging signal that a one-time approach could help close that adherence gap.” “What is compelling about this update is that the reductions in ANGPTL3, triglycerides, and LDL from a single infusion have persisted out to one-year, suggesting a sustained biological effect,” said Steven E. Nissen, M.D., senior author of the study and Chief Academic Officer at the Cleveland Clinic Heart, Vascular and Thoracic Institute. “A one-time treatment with this degree of durability could represent a meaningful advance in how we manage lifelong lipid disorders.” Phase 1a Clinical Trial Design The Phase 1a portion of the study was an open label, dose-escalation trial evaluating single-course IV doses of CTX310 ranging from 0.1 to 0.8 mg/kg (lean body weight) targeting ANGPTL3 in four patient groups: homozygous familial hypercholesterolemia (HoFH), severe hypertriglyceridemia (sHTG), heterozygous familial hypercholesterolemia (HeFH), or mixed dyslipidemias (elevated TG and LDL). Eligible participants had uncontrolled TG levels >150 mg/dL and/or LDL cholesterol >100 mg/dL (or >70 mg/dL for those with established ASCVD) despite background standard of care per local guidelines. The majority of participants were receiving statins and/or ezetimibe, while 40% were taking PCSK9 inhibitors. The trial was designed to evaluate safety and tolerability as primary endpoints, with changes in circulating ANGPTL3 protein, TG, and LDL as secondary endpoints.   Safety and Tolerability Single-course ascending doses of CTX310 were administered to 15 participants across sequential cohorts, and all participants completed at least one-year of follow-up as of the data cutoff. CTX310 was generally well tolerated, and no dose-limiting toxicities or serious adverse events related to treatment. Adverse events were generally mild to moderate. As previously reported in Laffin et al. (2025), one participant experienced an allergic reaction that resolved the following day with supportive care. Infusion-related reactions occurred in three participants (two at 0.6 mg/kg and one at 0.8 mg/kg dose), all Grade 2. All events resolved, and all participants completed their infusions. Beyond the transient aminotransferases elevation previously reported in one participant shortly after treatment, there were no liver function test elevations throughout the remainder of the trial. The safety events described above were previously reported in Laffin et al. (New England Journal of Medicine, 2025) and reflect the initial treatment period of the Phase 1a clinical trial. No new treatment-related safety events were observed during extended follow-up. Efficacy Highlights These new results build upon previously disclosed clinical data from 15 participants across four sequential cohorts, corresponding to lean body weight-based doses of DL1 [0.1 mg/kg], DL2 [0.3 mg/kg], DL3 [0.6 mg/kg], DL3.5 [0.7 mg/kg] and DL4 [0.8 mg/kg]. All participants had at least one-year of follow-up. Dose dependent reductions in circulating ANGPTL3 protein were sustained through one-year following CTX310 infusion.Among participants treated at 0.8 mg/kg, ANGPTL3 reductions of up to 89% were observed, with a mean reduction of 79% at one-year following CTX310 infusion. Next Steps The Phase 1b portion of the trial examines a fixed flat dose regimen of CTX310 equivalent to the most efficacious dose in Phase 1a (0.8mg/kg). CRISPR Therapeutics continues to anticipate sharing an update from the CTX310 Phase 1b clinical trial in the second half of 2026, focused on severe hypertriglyceridemia patients. About In Vivo Liver Editing Programs CRISPR Therapeutics has established a proprietary lipid nanoparticle (LNP) delivery platform to enable gene editing in the liver using both CRISPR/Cas9 and its novel, proprietary SyNTase™ editing technology. The Company’s in vivo portfolio includes three cardiovascular programs: CTX310, targeting angiopoietin-related protein 3 (ANGPTL3), in development for heterozygous and homozygous familial hypercholesterolemia, mixed dyslipidemias, and severe hypertriglyceridemia; CTX340™, targeting angiotensinogen (AGT), in development for refractory hypertension; and CTX321™, targeting LPA, in development for patients with elevated lipoprotein(a) [Lp(a)]. In addition, the Company’s disclosed development candidates also include CTX460™, targeting SERPINA1 using SyNTase editing, for the treatment of alpha-1 antitrypsin deficiency (AATD). About CRISPR Therapeutics CRISPR Therapeutics is a leading biopharmaceutical company focused on developing transformative gene-based medicines for serious human diseases. Founded over a decade ago as an early pioneer in CRISPR/Cas9 gene editing, the Company has evolved from a pioneering research-stage organization into an industry leader, marking a historic milestone with the approval of CASGEVY® (exagamglogene autotemcel [exa-cel]), the world’s first CRISPR-based therapy, for eligible patients with sickle cell disease and transfusion-dependent beta thalassemia. Today, CRISPR Therapeutics is advancing a broad, diversified pipeline spanning hemoglobinopathies, cardiovascular disease, autoimmune disease, oncology, regenerative medicine and rare diseases. The Company is also expanding its gene editing toolkit through SyNTase™ editing, its novel, proprietary platform designed to enable precise, efficient, and scalable gene correction. To accelerate its impact, CRISPR Therapeutics has established strategic collaborations with leading biopharmaceutical partners, including Vertex Pharmaceuticals. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Boston, Massachusetts and San Francisco, California. To learn more, visit www.crisprtx.com. CRISPR THERAPEUTICS® standard character mark and design logo, CTX310®, CTX321™, CTX340™, CTX460™ and SyNTase™ are trademarks and registered trademarks of CRISPR Therapeutics AG. All other trademarks and registered trademarks are the property of their respective owners. Reference Laffin L, et al. Phase 1 Trial of CRISPR-Cas9 Gene Editing Targeting ANGPTL3. The New England Journal of Medicine. 2025. CRISPR Special Note Regarding Forward-Looking Statements Statements contained in this press release regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Such statements include, but are not limited to, statements regarding any or all of the following: (i) CRISPR Therapeutics preclinical studies, clinical trials and pipeline products and programs, including, without limitation, manufacturing capabilities, status of such studies and trials, potential expansion into new indications and expectations regarding data, safety and efficacy generally; (ii) data included in the above-described oral presentation and any associated abstracts or posters, data included in the above-described article in The New England Journal of Medicine, as well as the ability to use data from ongoing and planned clinical trials for the design and initiation of further clinical trials; and (iii) the therapeutic value, development, and commercial potential of gene editing technologies and therapies, including CRISPR/Cas9 and SyNTase, as well as other technologies. Risks that contribute to the uncertain nature of the forward-looking statements include, without limitation, the risks and uncertainties discussed under the heading “Risk Factors” in CRISPR Therapeutics most recent annual report on Form 10-K and in any other subsequent filings made by CRISPR Therapeutics with the U.S. Securities and Exchange Commission. 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. We disclaim any obligation or undertaking to update or revise any forward-looking statements contained in this press release, other than to the extent required by law. Investor Contact: +1-617-307-7503 ir@crisprtx.com Media Contact: +1-617-315-4493 media@crisprtx.com

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