Aficamten

Multinational Real-world Data Reinforce Favorable Clinical Pro Long-Term Safety SOUTH SAN FRANCISCO, Calif., May 11, 2026 (GLOBE NEWSWIRE) -- Cytokinetics, Incorporated (Nasdaq: CYTK) today announced the presentation of new data reinforcing the clinical pro MYQORZO ® (aficamten) at the European Society of Cardiology (ESC) Heart Failure 2026 Congress. The presentations include new analyses from SEQUOIA-HCM, the pivotal Phase 3 clinical trial of aficamten in patients with oHCM; MAPLE-HCM, the Phase 3 clinical trial of aficamten compared to metoprolol in patients with symptomatic obstructive HCM (oHCM); and FOREST-HCM, the open-label extension trial of aficamten . Collectively, the new evidence expands understanding of the effectiveness of cardiac myosin inhibition with aficamten compared directly to metoprolol , a beta blocker, as well as specific safety and durability characteristics of MYQORZO across patient demographics, clinical, and economic subgroups. “The breadth of research being presented at ESC Heart Failure 2026 reflects our commitment to deepening the scientific understanding of oHCM and heart failure,” said Stephen Heitner, M.D., Senior Vice President, Clinical Research and Development, Cytokinetics. “These new insights underscore the strength and consistency of the clinical pro MYQORZO, adding to the growing body of real-world evidence informing physician treatment decisions.” New Analyses Show MYQORZO Outperforms Metoprolol Across Sex and Doses A dose-dependent analysis from MAPLE-HCM compared aficamten to the beta-blocker metoprolol in patients with symptomatic oHCM. Key findings showed significant improvements in exercise capacity, outflow gradients, and N-terminal pro-B-type natriuretic peptide (NT-proBNP) across all treatment doses. Conversely, metoprolol failed to show improvement in these outcomes regardless of the dose administered (Figure 1). Figure 1 A secondary analysis of sex differences in MAPLE-HCM showed consistent benefits of MYQORZO in women. Despite women entering the trial (42%) with more severe baseline characteristics, MYQORZO delivered nearly identical improvements in peak oxygen consumption (pVO 2 ) for both sexes (+2.2 mL/kg/min). Both groups also saw significant gains in Kansas City Cardiomyopathy Questionnaire Clinical Summary Score (KCCQ-CSS) and reductions in cardiac biomarkers and NT-proBNP. Long-term Data Reinforce Safety and Efficacy Pro MYQORZO A prospective analysis of 122 patients who had interpretable ambulatory electrocardiogram (ECG) data at screening in the FOREST-HCM showed that long-term treatment with MYQORZO—up to 96 weeks—did not increase the incidence of arrhythmias in patients, including those who underwent withdrawal of beta-blocker therapy. Among 122 patients, the incidence of non-sustained ventricular tachycardia (NSVT) on ambulatory ECG remained stable through 96 weeks compared with baseline, with no increase in atrial fibrillation (AF) episodes and no newly identified subclinical AF. There was also no increase in arrhythmia frequency among a subgroup of 16 patients who discontinued beta-blocker therapy during aficamten treatment. These results represent the first prospective analysis of ambulatory ECG monitoring in patients treated with a cardiac myosin inhibitor and are consistent with the low incidence of clinically detected arrhythmias previously reported for patients with oHCM treated with aficamten . Additionally, an open-label extension study of aficamten in Chinese patients with symptomatic oHCM showed that aficamten was well tolerated using the same dosing strategy of individualized titration as was used globally. Through 48 weeks of treatment with aficamten , patients experienced no serious or severe treatment-emergent adverse events, no occurrences of LVEF 5% of patients and more commonly on MYQORZO than on placebo in the pivotal trial. INDICATIONS AND USAGE MYQORZO is indicated for the treatment of adults with symptomatic obstructive hypertrophic cardiomyopathy (oHCM) to improve functional capacity and symptoms. Please see full prescribing information , including BOXED WARING, for MYQORZO. About Hypertrophic Cardiomyopathy Hypertrophic cardiomyopathy (HCM) is a disease in which the heart muscle becomes abnormally thick. HCM can be obstructive, when thickened muscle blocks blood flow, or non-obstructive, when blood flow is not blocked but heart function is still affected. In obstructive HCM, the thickening of cardiac muscle leads to the inside of the left ventricle becoming smaller, stiffer and less able to relax and fill with blood. Ultimately, HCM limits the heart’s pumping function, leading to reduced exercise capacity and a variety of symptoms. HCM is the most common monogenic inherited cardiovascular disorder, with well over 300,000 patients diagnosed in the U.S. 2 However, there are an estimated 400,000-800,000 additional patients who remain undiagnosed. 3,4,5 Recent analysis of a large claims database indicates that approximately half of patients with HCM have obstructive HCM (oHCM) and half have non-obstructive HCM (nHCM). 2 People with HCM are at high risk of also developing cardiovascular complications including atrial fibrillation, stroke and mitral valve disease. 6 People with HCM are at risk for potentially fatal ventricular arrhythmias and it is one of the leading causes of sudden cardiac death in younger people or athletes. 7 A subset of patients with HCM are at high risk of progressive disease leading to dilated cardiomyopathy and heart failure necessitating cardiac transplantation. About Cytokinetics Cytokinetics is a specialty cardiovascular biopharmaceutical company, building on its over 25 years of pioneering scientific innovations in muscle biology, and advancing a pipeline of potential new medicines for patients suffering from diseases of cardiac muscle dysfunction. Cytokinetics’ MYQORZO ® ( aficamten ) is a cardiac myosin inhibitor approved in the U.S., Europe and China for the treatment of adults with symptomatic obstructive hypertrophic cardiomyopathy (oHCM). Following positive topline results in ACACIA-HCM, a Phase 3 clinical trial of aficamten in patients with non-obstructive HCM, the company is preparing to present the full results at an upcoming medical meeting and discuss them with the U.S. FDA and other regulatory authorities. Cytokinetics is also developing omecamtiv mecarbil , an investigational cardiac myosin activator for the potential treatment of patients with heart failure with severely reduced ejection fraction and ulacamten , an investigational cardiac myosin inhibitor for the potential treatment of heart failure with preserved ejection fraction, while continuing pre-clinical research and development in muscle biology. For additional information about Cytokinetics, visit and follow us on X , LinkedIn , Facebook and YouTube . Forward-Looking Statements This press release contains forward-looking statements for purposes of the Private Securities Litigation Reform Act of 1995 (the “Act”). Cytokinetics disclaims any intent or obligation to update these forward-looking statements and claims the protection of the Act's safe harbor for forward-looking statements. Examples of such statements include, but are not limited to, statements, express or implied, related to Cytokinetics’ research and development activities; clinical trial initiation, design, enrollment, conduct, progress, continuation, completion, timing and results; regulatory submissions, review processes, approval timing and outcomes, including with respect to supplemental applications and approvals in jurisdictions outside the United States; the scope, expansion, modification, durability or continuation of labeling and promotional claims; commercial readiness, launch timing, market access and reimbursement; anticipated patient, prescriber and payer adoption; expectations regarding market opportunity, growth and market share; pipeline development and expansion into additional indications or geographies; access to and use of capital; and Cytokinetics’ business strategy, objectives and future plans. Such statements are based on management's current expectations and assumptions; however, actual results may differ materially due to various risks and uncertainties, including, but not limited to, uncertainties inherent in drug development and commercialization; the timing, conduct and outcomes of clinical trials; regulatory review and approval processes in the United States and other jurisdictions; differences in regulatory requirements, labeling, market access or promotional restrictions across jurisdictions; the ability to obtain, expand, maintain or continue desired labeling, promotional claims or commercial positioning for approved products; potential legal, intellectual property or regulatory constraints affecting commercialization and marketing claims; patient and prescriber acceptance of MYQORZO as compared to alternative therapies; the availability and terms of reimbursement from commercial and government payers; manufacturing, supply and distribution risks; competition; and the availability of sufficient capital to execute Cytokinetics’ business plans. These forward-looking statements speak only as of the date they are made, and Cytokinetics undertakes no obligation to subsequently update any such statement, except as required by law. For further information regarding these and other risks related to Cytokinetics’ business, investors should consult Cytokinetics’ filings with the Securities and Exchange Commission (the “SEC”). CYTOKINETICS ® and the CYTOKINETICS C-shaped logo are registered trademarks of Cytokinetics in the U.S. and certain other countries. MYQORZO ® is a registered trademark of Cytokinetics in the U.S. and the European Union. Contact: Cytokinetics Diane Weiser Senior Vice President, Corporate Affairs (415) 290-7757 References Hartman JJ, Hwee DT, Roebrt-Paganin J, et al. Aficamten is a small-molecule cardiac myosin inhibitor designed to treat hypertrophic cardiomyopathy. Nat Cardiovasc Res. 2024;3(8) :1003-1016. doi:10.1038/s44161-024-00505-0 Butzner M, et al. Epidemiology of Hypertrophic Cardiomyopathy in the United States From 2016 to 2023. JACC Adv. 2026. 2026;5(2):102552. doi:10.1016/j.jacadv.2025.102552 CVrg: Heart Failure 2020-2029, p 44; Maron et al. 2013 doi:10.1016/S0140-6736(12)60397-3; Maron et al 2018 10.1056/NEJMra1710575 Symphony Health 2016-2021 Patient Claims Data DoF; Maron MS, Hellawell JL, Lucove JC, Farzaneh-Far R, Olivotto I. Occurrence of Clinically Diagnosed Hypertrophic Cardiomyopathy in the United States. Am J Cardiol. 2016; 15;117(10):1651-1654. Gersh, B.J., Maron, B.J., Bonow, R.O., Dearani, J.A., Fifer, M.A., Link, M.S., et al. 2011 ACCF/AHA guidelines for the diagnosis and treatment of hypertrophic cardiomyopathy. A report of the American College of Cardiology Foundation/American Heart Association Task Force on practice guidelines. Journal of the American College of Cardiology and Circulation, 58, e212-260. A photo accompanying this announcement is available at

Welcome back to another edition of Endpoints Weekly! Earnings season is in full swing, but we had plenty of original reporting and exclusives you won’t find anywhere else this week. Chief among them: Andrew Dunn’s excellent story Friday on why biopharma is struggling to recruit young AI talent . Spoiler — it’s not just the pay gap with companies like OpenAI, though that’s definitely part of it. We also hosted a Post-Hoc Live recapping what we’ve seen in earnings so far, where I joined Drew Armstrong, Elizabeth Cairns and Leerink senior research analyst David Risinger to dig into the biggest stories in pharma right now. Paid subscribers can click here to watch a recording of the conversation. And, on Friday afternoon, the Wall Street Journal reported that President Donald Trump is planning to fire FDA Commissioner Marty Makary — make sure to read about that here . Have a great weekend! — Max Gelman 🇨🇳 A new trend is shaking up how biotechs are built, Kyle LaHucik observes in this feature story . Instead of launching with eye-popping early-stage science or a new platform, there’s a surge of startups with small teams, large checks and one or two clinical-stage assets in-licensed from China. The so-called NewCo model has been around for years, but its use with assets connected to China appears to be increasing. Endpoints News identified at least 18 private biotechs that have followed this route since the beginning of 2025. Previously, most biotechs started with a piece of fundamental science, then worked for years to build a drug around it that could eventually be tested. But with the explosion of trial-ready assets from China, many are skipping those early steps. Some companies are focusing on a single drug, hoping to expand into multiple indications with the same asset. Others have sourced a clinical-stage asset or two from China to kickstart their pipelines and build a foundation for additional discovery work. The model represents a fundamental shift in how biotechs are formed and grow, Kyle writes . The dynamics can be traced back to a handful of factors, including a yearslong funding downturn, an explosion in target and modality crowding, a desire to get quicker returns, a flocking to diseases with large patient populations, and a massive growth spurt in competition from R&D hubs like Shanghai. You can read more here. 💰 Candid’s effort to prove T cell engagers’ potential in autoimmune diseases is getting picked up by one of Europe’s oldest pharma companies, Kyle LaHucik writes . UCB is paying $2 billion in cash to acquire Candid, with up to $200 million in milestones on the line. Candid had been planning to go public this summer via a reverse merger with Rallybio. But unsolicited interest from multiple companies “led toward a broader evaluation,” CEO Ken Song said. “I was a bit surprised,” Song told Endpoints . But he noted that there has been “an increasing appreciation broadly across the industry on the benefit of T cell engagers for B cell depletion in a variety of autoimmune diseases.” Song has been committed to testing the modality for autoimmune diseases, previously claiming that Candid would “prove or disprove” the approach “for the entire industry.” The deal marks UCB’s second biotech acquisition in as many months, in what has so far been a busy year for M&A. Drugmakers have signed at least 30 acquisitions so far this year, according to an Endpoints tally. 💩 Scientists trying to understand why cancer immunotherapy works startlingly well for some patients — and not others — have found a potential answer in one cancer survivor’s poop . Kanvas Biosciences studied a woman’s stool and identified roughly 50 strains of bacteria that, collectively, created just the right conditions for immunotherapy to cure her advanced colorectal cancer. Researchers also say they’ve figured out how to grow that microbial community in the lab and package them in pills, senior science correspondent Ryan Cross reports . Kanvas raised $48 million in a Series A to run a clinical trial later this year testing those pills. The money will also fund two other microbe-based therapies: one to help reduce colon inflammation caused by immunotherapy, and another, backed by the Gates Foundation, aimed at healing the guts of patients who can’t properly absorb nutrients. Read more from Ryan here . 🤝 GSK inked a $1 billion biobucks deal with Suzhou-based SiranBio, expanding its reach in the cardiometabolic space. GSK will pay $55 million upfront for the rights to SA030 — an siRNA in Phase 1 for obesity — outside of China, Hong Kong, Macau and Taiwan. SiranBio has been building up the drug candidate as a next-generation obesity treatment, with the potential to “achieve selective fat reduction without compromising muscle mass.” It goes after activin receptor-like kinase 7, or ALK7, which is the same target as one of Arrowhead Pharmaceuticals’ obesity drugs. But GSK sees potential beyond obesity. A GSK spokesperson told Endpoints News that the company doesn’t plan to develop the program “for the sole purpose of weight loss.” Rather, GSK said it will test the candidate in cardiometabolic disease patients with “chronic conditions that affect the lung, liver and kidney.” The deal also extends GSK’s dealmaking streak in China. Over the last couple of years, it has done deals with Hengrui, Hansoh and Chimagen. Read more here from Kyle LaHucik. 🫀 This week, Cytokinetics said its heart drug Myqorzo succeeded in a Phase 3 study, showing improvements in symptoms and exercise capacity in patients with the non-obstructive form of hypertrophic cardiomyopathy (nHCM). The drug already won approval for the obstructive form of the condition (oHCM) late last year. Myqorzo competes with Bristol Myers Squibb’s Camzyos in oHCM. And Cytokinetics’ new data would potentially open up a whole new market, in an indication where Camzyos notably failed in its pivotal trial last year. Both drugs work by inhibiting the heart muscle protein cardiac myosin. Read more about the pivotal win here . DON’T MISS: