A Multi-Center, Double-Blind, Randomized, Placebo-Controlled Trial to Assess Efficacy and Safety of Omecamtiv Mecarbil in Patients With Symptomatic Heart Failure With Severely Reduced Ejection Fraction (COMET-HF)

The purpose of this study is to find out if the investigational drug called omecamtiv mecarbil can reduce the risk of the effects of heart failure, like hospitalization, transplantation, or death in patients with heart failure and severely reduced ejection fraction.

Start Date19 Dec 2024

Sponsor / Collaborator

Cytokinetics, Inc.

NCT04464525

/ WithdrawnPhase 3

Omecamtiv Mecarbil Post-trial Access Protocol for Subjects Completing Study 20110203 GALACTIC-HF

The primary objective of this study is to provide access to omecamtiv mecarbil for participants who have completed GALACTIC-HF 20110203.

Start Date18 Dec 2020

Sponsor / Collaborator

Cytokinetics, Inc.

NCT04175808

/ CompletedPhase 1

Single-dose and Randomized, Single-center, Placebo- and Active-controlled, Crossover Study to Assess the Effect of Omecamtiv Mecarbil (OM) on QT/QTc Intervals in Healthy Subjects

The primary objective of this study is to assess the effect of a single therapeutic (50 mg) oral dose of omecamtiv mecarbil (OM) on the QT interval / QT interval corrected for heart rate (QTc), relative to placebo, in healthy adults.
The QT interval is the section on an electrocardiogram (ECG) that represents the time it takes for the electrical system to fire an impulse through the ventricles and then recharge, or the time it takes for the heart muscle to contract and then recover.

Start Date14 Nov 2019

Sponsor / Collaborator

Cytokinetics, Inc.

100 Clinical Results associated with Omecamtiv Mecarbil

100 Translational Medicine associated with Omecamtiv Mecarbil

100 Patents (Medical) associated with Omecamtiv Mecarbil

284

Literatures (Medical) associated with Omecamtiv Mecarbil

01 Oct 2025·JACC-Heart Failure

Efficacy and Safety of Omecamtiv Mecarbil in Heart Failure With Reduced Ejection Fraction According to Age

Article

Author: Heitner, Stephen B ; Metra, Marco ; McMurray, John J V ; Teerlink, John R ; Claggett, Brian L ; Lu, Henri ; Vaduganathan, Muthiah ; Diaz, Rafael ; Solomon, Scott D ; Inciardi, Riccardo M ; Malik, Fady ; Felker, G Michael

BACKGROUND:

Older age is associated with a high prevalence of comorbidities and worse cardiovascular (CV) outcomes in patients with heart failure (HF) with reduced ejection fraction. Omecamtiv mecarbil, a selective cardiac myosin activator, exerts minimal effects on blood pressure and heart rate and has limited extracardiac activity, and thus it may be well-tolerated in older individuals. The safety profile and clinical benefits of omecamtiv mecarbil across the age spectrum remain uncertain.

OBJECTIVES:

This study aims to assess CV outcomes, treatment response, and tolerability to omecamtiv mecarbil according to age in patients enrolled in the GALACTIC-HF (Global Approach to Lowering Adverse Cardiac Outcomes Through Improving Contractility in Heart Failure) trial.

METHODS:

GALACTIC-HF was a randomized, global, double-blind clinical trial testing omecamtiv mecarbil vs placebo in patients with symptomatic HF with reduced ejection fraction. Key eligibility criteria included an age between 18-85 years, elevated natriuretic peptides, and a left ventricular ejection fraction (LVEF) ≤35%. We examined the treatment effect of omecamtiv mecarbil vs placebo for the primary endpoint of CV death or first HF event (hospitalization or urgent visit for HF) in both the overall population and the severe HF subgroup (LVEF ≤30%, NYHA functional class III/IV, HF hospitalization within 6 months), as well as safety outcomes according to prespecified age groups (<65 or ≥65 years).

RESULTS:

A total of 8,232 patients (age 64.5 ± 11.4 years, 54.5% ≥65 years, 21% female) were included. The rate of the primary outcome was 21.4 per 100 patient years (py) in those <65 years of age and 28.8 per 100 py in those ≥65 years of age. The treatment effect of omecamtiv mecarbil for the primary outcome (HR: 0.92 [95% CI: 0.86-0.99]; P = 0.03) was consistent in age groups (P_interaction = 0.76) and irrespective of age as a continuous variable (P_interaction = 0.19, after adjusting for treatment interactions with known modifiers of the effects of omecamtiv mecarbil: LVEF and baseline atrial fibrillation status). In patients with severe HF, omecamtiv mecarbil significantly reduced the risk of the primary outcome in both patients <65 years of age (HR: 0.77 [95% CI: 0.64-0.92]) and those ≥65 years of age (HR: 0.83 [95% CI: 0.71-0.97]; P_interaction = 0.47). The safety profile of omecamtiv mecarbil was consistent irrespective of age (P_interaction > 0.05 for all).

CONCLUSIONS:

In GALACTIC-HF, older individuals were well-represented and faced higher risks of CV events. Treatment with omecamtiv mecarbil was safe irrespective of age and reduced the risk of CV death or first HF event across the age spectrum, especially in those with severe HF. (Global Approach to Lowering Adverse Cardiac Outcomes Through Improving Contractility in Heart Failure [GALACTIC-HF]; NCT02929329.).

26 Aug 2025·PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Danicamtiv reduces myosin’s working stroke but activates the thin filament by accelerating actomyosin attachment

Article

Author: Greenberg, Lina ; Squarci, Caterina ; Scott, Brent ; Campbell, Kenneth S. ; Greenberg, Michael J.

Heart failure is a leading cause of death worldwide, and even with current treatments, the 5-y transplant-free survival rate is only ~50 to 70%. As such, there is a need to develop new treatments for patients that improve survival and quality of life. Recently, there have been efforts to develop small molecules for heart failure that directly target components of the sarcomere, including cardiac myosin. Danicamtiv is one of these molecules; however, its direct effects on myosin’s single molecule mechanics and kinetics are not well understood. Using optical trapping techniques, stopped flow transient kinetics, and in vitro reconstitution assays, we found that danicamtiv reduces the size of cardiac myosin’s working stroke without affecting actomyosin detachment kinetics at the level of individual crossbridges. We demonstrate that danicamtiv accelerates actomyosin association kinetics, leading to increased recruitment of myosin crossbridges and subsequent thin filament activation at physiologically relevant calcium concentrations. We demonstrate important mechanistic differences with another cardiac myosin binding myotrope, omecamtiv mecarbil. Finally, we computationally model how the observed changes in mechanics and kinetics at the level of single crossbridges can contribute to increased cardiac contraction. Taken together, our results have important implications for the design of sarcomeric-targeting compounds for heart failure.

07 Jul 2025·JOURNAL OF GENERAL PHYSIOLOGY

Comparative mechanistic analysis of danicamtiv and omecamtiv mecarbil’s in vivo cardiac effects

Article

Author: Stelzer, Julian E. ; Holmes, Joshua B.

Danicamtiv is a second-generation myotropic sarcomere activator currently in clinical trials for treating heart failure with reduced ejection fraction. Initial clinical and preclinical studies suggest that danicamtiv improves upon the major shortcoming of the first-generation myotropic sarcomere activator, omecamtiv mecarbil (OM), which overly impaired diastolic function. However, no study has directly compared the in vivo cardiac effects of danicamtiv and OM to verify these claims. These direct comparisons are essential to understand the potential benefits of one drug over the other. Therefore, this study employed carefully controlled experiments with left ventricular pressure–volume loop and echocardiographic strain analysis to compare how danicamtiv and OM alter each phase of the cardiac cycle. Our results show that for similar increases in left ventricular stroke volume, danicamtiv reduced diastolic performance and myocardial relaxation less than OM. However, danicamtiv still significantly decreased diastolic function at higher doses, like OM. Furthermore, danicamtiv and OM elicited a qualitatively similar triphasic dose–response from the left ventricle. These similarities between danicamtiv and OM in the whole heart were surprising given recent evidence showing significant differences in the drugs’ molecular effects on myosin mechanics. We therefore conclude that danicamtiv likely has a wider therapeutic window than OM, but may be limited by the same trade-off between systolic and diastolic performance, driven by similar underlying mechanisms.

225

News (Medical) associated with Omecamtiv Mecarbil

19 Dec 2025

·ir.cytokinetics.com

Cytokinetics Announces FDA Approval of MYQORZO™ (aficamten) for the Treatment of Adults with Symptomatic Obstructive Hypertrophic Cardiomyopathy to Improve Functional Capacity and Symptoms

MYQORZO, a Cardiac Myosin Inhibitor, Directly Addresses Underlying Hypercontractility Associated with Obstructive HCM FDA Approval Based on Results of SEQUOIA-HCM MYQORZO is Company’s First FDA-Approved Medicine Company to Host Investor Conference Call Today at 4:30 PM Eastern Time SOUTH SAN FRANCISCO, Calif., Dec. 19, 2025 (GLOBE NEWSWIRE) -- Cytokinetics, Incorporated (Nasdaq: CYTK) today announced that the U.S. Food and Drug Administration (FDA) has approved MYQORZO™ (aficamten), 5 mg, 10 mg, 15 mg, 20 mg tablets for the treatment of adults with symptomatic obstructive hypertrophic cardiomyopathy (oHCM) to improve functional capacity and symptoms. MYQORZO is an allosteric and reversible inhibitor of cardiac myosin motor activity. In patients with oHCM, myosin inhibition with MYQORZO reduces cardiac contractility and left ventricular outflow tract (LVOT) obstruction. “This is a historic moment for our company and for the patients we serve, as we fulfill our promise to translate our science into medicines that may make a meaningful difference in patients’ lives,” said Robert I. Blum, Cytokinetics’ President and Chief Executive Officer. “Our first FDA approval stands as a testament to the strength of our science and the bold, trailblazing research that has defined Cytokinetics’ leadership in muscle biology. I’m pleased that the approved label and REMS reflect the distinct characteristics of MYQORZO including a straightforward, flexible dosing regimen, no requirement for drug-drug interaction monitoring and a predictable safety profile. I am profoundly grateful for the many years of passion and persistence shown by patients with obstructive HCM, as well as healthcare professionals, advocates, partners and employees who have contributed so importantly to reaching this key milestone.” The full U.S. Prescribing Information for MYQORZO includes a Boxed WARNING for the risk of heart failure. MYQORZO reduces left ventricular ejection fraction (LVEF) and can cause heart failure due to systolic dysfunction. Echocardiogram assessments are required prior to and during treatment with MYQORZO to monitor for systolic dysfunction. Initiation of MYQORZO in patients LVEF <55% is not recommended. Decrease the dose of MYQORZO if LVEF <50% and ≥40%. Interrupt the dose of MYQORZO if LVEF <40% or if the patient experiences heart failure symptoms or worsening clinical status due to systolic dysfunction. Because of the risk of heart failure due to systolic dysfunction, MYQORZO is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called the MYQORZO REMS Program. Please see additional Important Safety Information including Boxed WARNING below. MYQORZO is expected to be available in the U.S. in the second half of January 2026. Cytokinetics will support patients with MYQORZO & You™, a personalized program for patients prescribed MYQORZO in the U.S. to help navigate the treatment journey, provide disease and product education, and offer support with insurance benefits investigations or financial assistance for those eligible. For more information, call 833-MYQORZO (833-697-6796). “HCM is a heart muscle disease associated with a significant symptom burden. This approval of a new drug, MYQORZO, represents a meaningful addition to the treatment options available for symptomatic obstructive HCM patients,” said Martin Maron, M.D., Director, Hypertrophic Cardiomyopathy Center, Lahey Hospital and Medical Center, and Principal Investigator of SEQUOIA-HCM. “In SEQUOIA-HCM, MYQORZO improved exercise capacity and reduced symptoms while also being well-tolerated. For these reasons, MYQORZO represents an important step forward in how we care for people living with obstructive HCM.” “Living with symptomatic obstructive HCM means managing physical limitations and burdensome symptoms every day of your life,” said Lisa Salberg, Founder and CEO of the Hypertrophic Cardiomyopathy Association (HCMA). “For far too long, we’ve had few options to address our needs, and the approval of MYQORZO is a long-awaited and major addition to bring new hope to patients living with oHCM. We are so grateful to the team at Cytokinetics for listening to the patient community and working in true partnership to bring this therapy to so many in need.” About SEQUOIA-HCM The approval is based on the positive results from the pivotal Phase 3 clinical trial, SEQUOIA-HCM, published in the New England Journal of Medicine, which demonstrated robust efficacy, safety, and clinically meaningful benefits across symptoms, exercise capacity, hemodynamics, and biomarker endpoints. The results from SEQUOIA-HCM showed that treatment with MYQORZO for 24 weeks significantly improved exercise capacity compared to placebo, increasing peak oxygen uptake (pVO2) measured by cardiopulmonary exercise testing (CPET) by 1.8 mL/kg/min compared to baseline in patients treated with MYQORZO versus 0.0 mL/kg/min in patients treated with placebo (least square mean (LSM) difference [95% CI] of 1.74 mL/kg/min [1.04 - 2.44]; p=0.000002).1 The treatment effect of MYQORZO was consistent across all prespecified subgroups, including age, sex, patient baseline characteristics, and in patients receiving or not receiving background beta-blocker therapy. MYQORZO was well-tolerated, with no instances of worsening heart failure or treatment interruptions due to low LVEF. Treatment emergent serious adverse events occurred in 5.6% of patients on MYQORZO and 9.3% of patients on placebo. Core laboratory echocardiographic LVEF was observed to be <50% in 5 patients (3.5%) on MYQORZO compared to 1 patient (0.7%) on placebo. Hypertension (8% vs 2%) was the only adverse reaction occurring in >5% of patients and more commonly on MYQORZO than on placebo. MYQORZO-associated increases in blood pressure are consistent with relief of LVOT obstruction and improved cardiac output. Investor Webcast Information Cytokinetics will host an investor conference call on December 19, 2025, at 4:30 PM Eastern Time to discuss the FDA approval of MYQORZO. Interested parties can register online at https://myqorzo-fda-approval-call.open-exchange.net/. The live webcast will be available on the Investors & Media section of the Cytokinetics website at www.cytokinetics.com. A replay of the webcast will be archived on the Cytokinetics website for six months. INDICATION MYQORZO is indicated for the treatment of adults with symptomatic obstructive hypertrophic cardiomyopathy (oHCM) to improve functional capacity and symptoms. IMPORTANT SAFETY INFORMATION CONTRAINDICATIONS MYQORZO is contraindicated with concomitant use of rifampin. WARNING AND PRECAUTIONS Heart Failure MYQORZO reduces cardiac contractility, which can reduce LVEF and cause heart failure. Patients who experience a serious intercurrent illness (eg, serious infection) or arrhythmia (eg, new or uncontrolled atrial fibrillation) may be at greater risk of developing systolic dysfunction and heart failure. Assess patients’ clinical status and LVEF prior to and during treatment and adjust the MYQORZO dose accordingly. New or worsening arrhythmia, dyspnea, chest pain, fatigue, leg edema, or elevations in N-terminal pro-B-type natriuretic peptide may be signs and symptoms of heart failure. Initiation of MYQORZO in patients with LVEF <55% is not recommended. MYQORZO REMS Program MYQORZO is available only through a restricted program called the MYQORZO REMS Program, because of the risk of heart failure due to systolic dysfunction. Notable requirements of the MYQORZO REMS Program include: Prescribers must be certified by enrolling in the MYQORZO REMS Program Patients must enroll in the MYQORZO REMS Program and comply with ongoing monitoring requirements Pharmacies must be certified by enrolling in the MYQORZO REMS Program and must only dispense to patients who are authorized to receive MYQORZO Wholesalers and distributors must only distribute to certified pharmacies Further information is available at www.MYQORZOREMS.com, or at 1-844-285-7367. Cytochrome P450 Interactions Leading to Heart Failure or Loss of Effectiveness MYQORZO is metabolized primarily by CYP2C9, and to a lesser extent by CYP3A, CYP2D6, and CYP2C19 enzymes. Initiation of medications that inhibit multiple P450 pathways of MYQORZO elimination (eg, fluconazole, voriconazole, or fluvoxamine) or strong CYP2C9 inhibitors, and discontinuation of moderate-to-strong CYP3A inducers may lead to increased blood concentrations of aficamten and increase the risk of heart failure due to systolic dysfunction. Conversely, initiation of medications that induce P450 pathways of MYQORZO (eg, rifampin, moderate-to-strong CYP3A inducers) may lead to decreased blood concentrations of aficamten and potential loss of effectiveness. Assess LVEF 2 to 8 weeks after initiation of such inhibitors or after discontinuation of such inducers and adjust the dose of MYQORZO accordingly. Advise patients of the potential for drug interactions. Advise patients to inform their healthcare provider of all concomitant medications prior to and during MYQORZO treatment. ADVERSE REACTIONS Hypertension (8% vs 2%) was the only adverse reaction occurring in >5% of patients and more commonly on MYQORZO than on placebo in the pivotal trial.Please see full Prescribing Information, including Boxed WARNING and Medication Guide. About MYQORZO™ (aficamten) MYQORZO™ (aficamten) is a cardiac myosin inhibitor approved by the U.S. FDA for the treatment of symptomatic obstructive hypertrophic cardiomyopathy (oHCM) to improve functional capacity and symptoms. MYQORZO is an allosteric and reversible inhibitor of cardiac myosin motor activity. In patients with HCM, myosin inhibition with MYQORZO reduces cardiac contractility and left ventricular outflow tract (LVOT) obstruction. MYQORZO was engineered to achieve a predictable exposure response, rapid onset of action and reversibility.2 On December 17, 2025, the China National Medical Products Administration approved MYQORZO® (aficamten) for the treatment of oHCM. On December 12, 2025, the Committee for Medicinal Products for Human Use of the European Medicines Agency adopted a positive opinion recommending marketing authorization in the European Union for MYQORZO® (aficamten) with a decision expected from the European Commission in the first quarter of 2026. Aficamten is also under clinical investigation in ACACIA-HCM, a Phase 3 trial in patients with non-obstructive HCM (nHCM) and CEDAR-HCM, in a pediatric population with oHCM. Aficamten has not been deemed safe or effective for use in either of these patient populations. In addition, aficamten is being studied in FOREST-HCM, an open-label extension clinical study. 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. However, there are an estimated 400,000-800,000 additional patients who remain undiagnosed.3,4,5 Approximately half of patients with HCM have obstructive HCM (oHCM) and half have non-obstructive HCM (nHCM). 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 for the treatment of adults with symptomatic obstructive hypertrophic cardiomyopathy (HCM) to improve functional capacity and symptoms by the U.S. Food and Drug Administration and the China National Medical Products Administration. The Committee for Medicinal Products for Human Use of the European Medicines Agency adopted a positive opinion recommending marketing authorization in the European Union for MYQORZO® (aficamten) with a decision expected from the European Commission in first quarter in 2026. Aficamten is also being studied for the potential treatment of non-obstructive HCM. 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 www.cytokinetics.com 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 relating to any of our clinical trials, statements relating to our ability to obtain regulatory approval for aficamten in any jurisdiction by any particular date, if ever. Such statements are based on management’s current expectations, but actual results may differ materially due to various risks and uncertainties, including, but not limited to, potential difficulties or delays in the development, testing, regulatory approvals for trial commencement, progression or product sale or manufacturing, or production of Cytokinetics’ drug candidates that could slow or prevent clinical development or product approval; Cytokinetics’ drug candidates may have adverse side effects or inadequate therapeutic efficacy; the FDA or foreign regulatory agencies may delay or limit Cytokinetics’ ability to conduct clinical trials; Cytokinetics may be unable to obtain or maintain patent or trade secret protection for its intellectual property; standards of care may change, rendering Cytokinetics’ drug candidates obsolete; and competitive products or alternative therapies may be developed by others for the treatment of indications Cytokinetics’ drug candidates and potential drug candidates may target. For further information regarding these and other risks related to Cytokinetics’ business, investors should consult Cytokinetics’ filings with the Securities and Exchange Commission. CYTOKINETICS® and the CYTOKINETICS C-shaped logo are registered trademarks of Cytokinetics in the U.S. and certain other countries. MYQORZO™ is a trademark of Cytokinetics in the U.S., and a registered trademark in the European Union. References Contact:CytokineticsDiane WeiserSenior Vice President, Corporate Affairs(415) 290-7757 A photo accompanying this announcement is available at https://www.globenewswire.com/NewsRoom/AttachmentNg/b9f8fb42-7a54-4595-ac4a-01d585228003

Clinical ResultDrug ApprovalPhase 3

17 Dec 2025

·www.globenewswire.com

Cytokinetics Announces NMPA Approval of MYQORZO® (aficamten) in China for Patients with Obstructive Hypertrophic Cardiomyopathy

Approval Triggers Milestone Payment of $7.5 Million from Sanofi; Cytokinetics Eligible to Receive Additional Milestone Payments and Royalties on Net Sales in Greater China SOUTH SAN FRANCISCO, Calif., Dec. 17, 2025 (GLOBE NEWSWIRE) -- Cytokinetics, Incorporated (Nasdaq: CYTK) today announced that MYQORZO® (aficamten) has been approved by the China National Medical Products Administration (NMPA) for the treatment of adults with New York Heart Association (NYHA) class II-III obstructive hypertrophic cardiomyopathy (oHCM), to improve exercise capacity and symptoms. Under the terms of its license and collaboration agreement with Cytokinetics, Sanofi has exclusive rights to develop and commercialize MYQORZO for the treatment of patients with obstructive and non-obstructive hypertrophic cardiomyopathy (HCM) in Greater China. The approval of MYQORZO in oHCM in China triggers a $7.5 million milestone payment from Sanofi to Cytokinetics. Cytokinetics remains eligible to receive up to $142.5 million in development and commercial milestone payments from Sanofi as well as royalties in the low-to-high teens on future sales of MYQORZO in Greater China. MYQORZO is only approved for use in China. Aficamten is currently under regulatory review in the U.S, where the 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. On December 12, 2025 the Committee for Medicinal Products for Human Use (CHMP) of the EMA adopted a positive opinion recommending marketing authorization in the European Union (EU) for aficamten, and a final decision is anticipated from the European Commission in the first quarter of 2026. About MYQORZO® (aficamten) MYQORZO® (aficamten) is an investigational selective, small molecule cardiac myosin inhibitor discovered following an extensive chemical optimization program that was conducted with careful attention to therapeutic index and pharmacokinetic properties.1 MYQORZO was designed to reduce the number of active actin-myosin cross bridges during each cardiac cycle and consequently suppress the myocardial hypercontractility that is associated with HCM. In preclinical models, MYQORZO reduced myocardial contractility by binding directly to cardiac myosin at a distinct and selective allosteric binding site, thereby preventing myosin from entering a force producing state. The development program for MYQORZO is assessing its potential as a treatment that improves exercise capacity as measured by peak oxygen uptake (pVO2) and relieves symptoms in patients with HCM. MYQORZO was evaluated in SEQUOIA-HCM, a positive pivotal Phase 3 clinical trial in patients symptomatic obstructive hypertrophic cardiomyopathy (HCM). MYQORZO received Breakthrough Therapy Designation for the treatment of symptomatic HCM from the U.S. Food & Drug Administration (FDA) and for the treatment of symptomatic obstructive HCM from the National Medical Products Administration (NMPA) in China. Aficamten is also currently under clinical investigation in ACACIA-HCM, a Phase 3 trial in patients with non-obstructive HCM and CEDAR-HCM, in a pediatric population with oHCM. Aficamten has not been deemed safe or effective for use in either of these patient populations. In addition, aficamten is being studied in FOREST-HCM, an open-label extension clinical study. About Hypertrophic Cardiomyopathy Hypertrophic cardiomyopathy (HCM) is a disease in which the heart muscle (myocardium) becomes abnormally thick (hypertrophied). The thickening of cardiac muscle leads to the inside of the left ventricle becoming smaller and stiffer, and thus the ventricle becomes less able to relax and fill with blood. This ultimately limits the heart’s pumping function, resulting in reduced exercise capacity and symptoms including chest pain, dizziness, shortness of breath, or fainting during physical activity. HCM is the most common monogenic inherited cardiovascular disorder, with approximately 280,000 patients diagnosed, however, there are an estimated 400,000-800,000 additional patients who remain undiagnosed in the U.S.2,3,4 Two-thirds of patients with HCM have obstructive HCM (oHCM), where the thickening of the cardiac muscle leads to left ventricular outflow tract (LVOT) obstruction, while one-third have non-obstructive HCM (nHCM), where blood flow isn’t impacted, but the heart muscle is still thickened. People with HCM are at high risk of also developing cardiovascular complications including atrial fibrillation, stroke and mitral valve disease.5 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.6 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 innovation in muscle biology, and advancing a pipeline of potential new medicines for patients suffering from diseases of cardiac muscle dysfunction with intention to create a muscle biology franchise business focused on medicines designed to optimize muscle performance for patients with cardiac and other diseases of muscle dysfunction. MYQORZO® (aficamten), the company’s cardiac myosin inhibitor, is approved for the treatment of patients with obstructive hypertrophic cardiomyopathy (HCM) in China, and under regulatory review in the U.S. The Committee for Medicinal Products for Human Use of the European Medicines Agency adopted a positive opinion recommending marketing authorization in the European Union for aficamten, and a final decision is anticipated from the European Commission in the first quarter of 2026. Cytokinetics is also developing omecamtiv mecarbil, a cardiac myosin activator, in patients with heart failure with severely reduced ejection fraction, ulacamten, a cardiac myosin inhibitor with a mechanism of action distinct from aficamten, for the potential treatment of heart failure with preserved ejection fraction and CK-089, a fast skeletal muscle troponin activator with potential therapeutic application to a specific type of muscular dystrophy and other conditions of impaired skeletal muscle function. For additional information about Cytokinetics, visit www.cytokinetics.com and follow us on X, LinkedIn, Facebook and YouTube. About the Sanofi and Cytokinetics Collaboration In 2024, Sanofi acquired exclusive rights to develop and commercialize MYQORZO® (aficamten) from Corxel Pharmaceuticals (CORXEL) for the treatment of patients with obstructive and non-obstructive hypertrophic cardiomyopathy (HCM) in Greater China. Previously, CORXEL acquired the rights to develop and commercialize MYQORZO in Greater China from Cytokinetics in accordance with Cytokinetics’ global registration programs. Forward-Looking Statements This press release contains forward-looking statements for purposes of the Private Securities Litigation Reform Act of 1995 (the “Act”). Cytokinetics claims the protection of the Act’s Safe Harbor for forward-looking statements. Examples of such statements include, but not limited to, statements, express or implied, relating to our or our partners’ research and development and commercial readiness activities, our receipt of regulatory approval by FDA or any other regulatory authority to enable our commercialization of aficamten in the United States or any other jurisdiction by any date, if ever, or our earning or receiving any milestone payments or specific quantum of royalties from commercialization of MYQORZO in Greater China. Such statements are based on management's current expectations, but actual results may differ materially due to various risks and uncertainties, including, but not limited to, the commercialization activities of Sanofi, the actions of regulatory activities, and the results of our on-going clinical trials. For further information regarding these and other risks related to Cytokinetics’ business, investors should consult Cytokinetics’ filings with the Securities and Exchange Commission, particularly under the caption “Risk Factors” in Cytokinetics’ Quarterly Report on Form 10-Q for the quarter ended September 30, 2025. Forward-looking statements are not guarantees of future performance, and Cytokinetics’ actual results of operations, financial condition and liquidity, and the development of the industry in which it operates, may differ materially from the forward-looking statements contained in this press release. Any forward-looking statements that Cytokinetics makes in this press release speak only as of the date of this press release. Cytokinetics assumes no obligation to update its forward-looking statements whether as a result of new information, future events or otherwise, after the date of this press release. 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 China. References: Chuang C, Collibee S, Ashcraft L, et al. Discovery of Aficamten (CK-274), a Next-Generation Cardiac Myosin Inhibitor for the Treatment of Hypertrophic Cardiomyopathy. J Med Chem. 2021;64(19):14142–14152. https://doi-org.sutd.idm.oclc.org/10.1021/acs.jmedchem.1c01290CVrg: Heart Failure 2020-2029, p 44; Maron et al. 2013 DOI: 10.1016/S0140-6736(12)60397-3; Maron et al 2018 10.1056/NEJMra1710575Symphony 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.Hong Y, Su WW, Li X. Risk factors of sudden cardiac death in hypertrophic cardiomyopathy. Current Opinion in Cardiology. 2022 Jan 1;37(1):15-21 Contact:Cytokinetics Diane WeiserSenior Vice President, Corporate Affairs(415) 290-7757

Drug ApprovalPhase 3License out/inBreakthrough TherapyClinical Result

17 Dec 2025

·ir.cytokinetics.com

Cytokinetics Announces NMPA Approval of MYQORZO® (aficamten) in China for Patients with Obstructive Hypertrophic Cardiomyopathy

Approval Triggers Milestone Payment of $7.5 Million from Sanofi; Cytokinetics Eligible to Receive Additional Milestone Payments and Royalties on Net Sales in Greater China SOUTH SAN FRANCISCO, Calif., Dec. 17, 2025 (GLOBE NEWSWIRE) -- Cytokinetics, Incorporated (Nasdaq: CYTK) today announced that MYQORZO® (aficamten) has been approved by the China National Medical Products Administration (NMPA) for the treatment of adults with New York Heart Association (NYHA) class II-III obstructive hypertrophic cardiomyopathy (oHCM), to improve exercise capacity and symptoms. Under the terms of its license and collaboration agreement with Cytokinetics, Sanofi has exclusive rights to develop and commercialize MYQORZO for the treatment of patients with obstructive and non-obstructive hypertrophic cardiomyopathy (HCM) in Greater China. The approval of MYQORZO in oHCM in China triggers a $7.5 million milestone payment from Sanofi to Cytokinetics. Cytokinetics remains eligible to receive up to $142.5 million in development and commercial milestone payments from Sanofi as well as royalties in the low-to-high teens on future sales of MYQORZO in Greater China. MYQORZO is only approved for use in China. Aficamten is currently under regulatory review in the U.S, where the 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. On December 12, 2025 the Committee for Medicinal Products for Human Use (CHMP) of the EMA adopted a positive opinion recommending marketing authorization in the European Union (EU) for aficamten, and a final decision is anticipated from the European Commission in the first quarter of 2026. About MYQORZO® (aficamten) MYQORZO® (aficamten) is an investigational selective, small molecule cardiac myosin inhibitor discovered following an extensive chemical optimization program that was conducted with careful attention to therapeutic index and pharmacokinetic properties.1 MYQORZO was designed to reduce the number of active actin-myosin cross bridges during each cardiac cycle and consequently suppress the myocardial hypercontractility that is associated with HCM. In preclinical models, MYQORZO reduced myocardial contractility by binding directly to cardiac myosin at a distinct and selective allosteric binding site, thereby preventing myosin from entering a force producing state. The development program for MYQORZO is assessing its potential as a treatment that improves exercise capacity as measured by peak oxygen uptake (pVO2) and relieves symptoms in patients with HCM. MYQORZO was evaluated in SEQUOIA-HCM, a positive pivotal Phase 3 clinical trial in patients symptomatic obstructive hypertrophic cardiomyopathy (HCM). MYQORZO received Breakthrough Therapy Designation for the treatment of symptomatic HCM from the U.S. Food & Drug Administration (FDA) and for the treatment of symptomatic obstructive HCM from the National Medical Products Administration (NMPA) in China. Aficamten is also currently under clinical investigation in ACACIA-HCM, a Phase 3 trial in patients with non-obstructive HCM and CEDAR-HCM, in a pediatric population with oHCM. Aficamten has not been deemed safe or effective for use in either of these patient populations. In addition, aficamten is being studied in FOREST-HCM, an open-label extension clinical study. About Hypertrophic Cardiomyopathy Hypertrophic cardiomyopathy (HCM) is a disease in which the heart muscle (myocardium) becomes abnormally thick (hypertrophied). The thickening of cardiac muscle leads to the inside of the left ventricle becoming smaller and stiffer, and thus the ventricle becomes less able to relax and fill with blood. This ultimately limits the heart’s pumping function, resulting in reduced exercise capacity and symptoms including chest pain, dizziness, shortness of breath, or fainting during physical activity. HCM is the most common monogenic inherited cardiovascular disorder, with approximately 280,000 patients diagnosed, however, there are an estimated 400,000-800,000 additional patients who remain undiagnosed in the U.S.2,3,4 Two-thirds of patients with HCM have obstructive HCM (oHCM), where the thickening of the cardiac muscle leads to left ventricular outflow tract (LVOT) obstruction, while one-third have non-obstructive HCM (nHCM), where blood flow isn’t impacted, but the heart muscle is still thickened. People with HCM are at high risk of also developing cardiovascular complications including atrial fibrillation, stroke and mitral valve disease.5 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.6 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 innovation in muscle biology, and advancing a pipeline of potential new medicines for patients suffering from diseases of cardiac muscle dysfunction with intention to create a muscle biology franchise business focused on medicines designed to optimize muscle performance for patients with cardiac and other diseases of muscle dysfunction. MYQORZO® (aficamten), the company’s cardiac myosin inhibitor, is approved for the treatment of patients with obstructive hypertrophic cardiomyopathy (HCM) in China, and under regulatory review in the U.S. The Committee for Medicinal Products for Human Use of the European Medicines Agency adopted a positive opinion recommending marketing authorization in the European Union for aficamten, and a final decision is anticipated from the European Commission in the first quarter of 2026. Cytokinetics is also developing omecamtiv mecarbil, a cardiac myosin activator, in patients with heart failure with severely reduced ejection fraction, ulacamten, a cardiac myosin inhibitor with a mechanism of action distinct from aficamten, for the potential treatment of heart failure with preserved ejection fraction and CK-089, a fast skeletal muscle troponin activator with potential therapeutic application to a specific type of muscular dystrophy and other conditions of impaired skeletal muscle function. For additional information about Cytokinetics, visit www.cytokinetics.com and follow us on X, LinkedIn, Facebook and YouTube. About the Sanofi and Cytokinetics Collaboration In 2024, Sanofi acquired exclusive rights to develop and commercialize MYQORZO® (aficamten) from Corxel Pharmaceuticals (CORXEL) for the treatment of patients with obstructive and non-obstructive hypertrophic cardiomyopathy (HCM) in Greater China. Previously, CORXEL acquired the rights to develop and commercialize MYQORZO in Greater China from Cytokinetics in accordance with Cytokinetics’ global registration programs. Forward-Looking Statements This press release contains forward-looking statements for purposes of the Private Securities Litigation Reform Act of 1995 (the “Act”). Cytokinetics claims the protection of the Act’s Safe Harbor for forward-looking statements. Examples of such statements include, but not limited to, statements, express or implied, relating to our or our partners’ research and development and commercial readiness activities, our receipt of regulatory approval by FDA or any other regulatory authority to enable our commercialization of aficamten in the United States or any other jurisdiction by any date, if ever, or our earning or receiving any milestone payments or specific quantum of royalties from commercialization of MYQORZO in Greater China. Such statements are based on management's current expectations, but actual results may differ materially due to various risks and uncertainties, including, but not limited to, the commercialization activities of Sanofi, the actions of regulatory activities, and the results of our on-going clinical trials. For further information regarding these and other risks related to Cytokinetics’ business, investors should consult Cytokinetics’ filings with the Securities and Exchange Commission, particularly under the caption “Risk Factors” in Cytokinetics’ Quarterly Report on Form 10-Q for the quarter ended September 30, 2025. Forward-looking statements are not guarantees of future performance, and Cytokinetics’ actual results of operations, financial condition and liquidity, and the development of the industry in which it operates, may differ materially from the forward-looking statements contained in this press release. Any forward-looking statements that Cytokinetics makes in this press release speak only as of the date of this press release. Cytokinetics assumes no obligation to update its forward-looking statements whether as a result of new information, future events or otherwise, after the date of this press release. 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 China. References: Contact:Cytokinetics Diane WeiserSenior Vice President, Corporate Affairs(415) 290-7757

Drug ApprovalPhase 3License out/inBreakthrough TherapyClinical Result

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KEGG	Wiki	ATC	Drug Bank
D09648	Omecamtiv Mecarbil	-	DB11816

R&D Status

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Indication	Highest Phase	Country/Location	Organization	Date
Heart Failure	NDA/BLA	United States	Cytokinetics, Inc.	04 Feb 2022
Chronic heart failure	NDA/BLA	European Union	Cytokinetics, Inc.	-
Heart failure with reduced ejection fraction	Phase 3	United States	Cytokinetics, Inc.	09 Apr 2019
Heart failure with reduced ejection fraction	Phase 3	Canada	Cytokinetics, Inc.	09 Apr 2019
Heart failure with reduced ejection fraction	Phase 3	France	Cytokinetics, Inc.	09 Apr 2019
Heart failure with reduced ejection fraction	Phase 3	Germany	Cytokinetics, Inc.	09 Apr 2019
Heart failure with reduced ejection fraction	Phase 3	Hungary	Cytokinetics, Inc.	09 Apr 2019
Heart failure with reduced ejection fraction	Phase 3	Italy	Cytokinetics, Inc.	09 Apr 2019
Heart failure with reduced ejection fraction	Phase 3	Netherlands	Cytokinetics, Inc.	09 Apr 2019
Heart failure with reduced ejection fraction	Phase 3	Poland	Cytokinetics, Inc.	09 Apr 2019

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT02929329 (GALACTIC-HF, Pubmed \| JACC Heart Fail)	Phase 3	Heart failure with reduced ejection fraction	8,232	Omecamtiv Mecarbil	hdbijcoxgr(gfxwfkwlin) = ddfyyfosgm vresrvrcqb (axggkdcwoy ) View more	Positive	01 Oct 2025
				Placebo	hdbijcoxgr(gfxwfkwlin) = mgrhqsinzn vresrvrcqb (axggkdcwoy ) View more
NCT02929329 (GALACTIC-HF, Pubmed \| JACC Heart Fail)	Phase 3	Hydrops Fetalis \| Reduced ejection fraction co-occurrent and due to chronic heart failure \| Chronic heart failure natriuretic peptides	-	Omecamtiv mecarbil	vzjqhxxftd(kinxwuyzxj): adjusted HR = 0.8 (95% CI, 0.73 - 0.88) View more	Positive	01 Dec 2023
				Placebo
NCT03759392 (METEORIC-HF, CTgov)	Phase 3	Heart failure with reduced ejection fraction	276	Omecamtiv Mecarbil (Omecamtiv Mecarbil)	sbtheaifqf(ljhrxifllk) = qqmruukcqc bmztcmsvvm (ishxwtaqsd, 0.1718) View more	-	07 Mar 2023
				Placebo (Placebo)	sbtheaifqf(ljhrxifllk) = lghevitdih bmztcmsvvm (ishxwtaqsd, 0.2412) View more
NCT02929329 (GALACTIC-HF, Pubmed \| Eur J Heart Fail)	Not Applicable	Heart failure with reduced ejection fraction	8,232	Omecamtiv mecarbil	tupcdjbqbp(qwkcuxguzp): HR = 0.94 (95% CI, 0.8 - 1.09), P-Value = 0.095	-	04 Jan 2023
				Placebo
NCT03759392 (METEORIC-HF, Pubmed) Manual	Phase 3	Chronic heart failure	276	Omecamtiv Mecarbil	xlktutqbdk(pppabwcuyu) = xaffepwdyy qrewnddpip (insmqwpppc ) View more	Negative	19 Jul 2022
				Placebo	xlktutqbdk(pppabwcuyu) = xfowpbskrh qrewnddpip (insmqwpppc ) View more
NCT02929329 (GALACTIC-HF, Pubmed \| JAMA Cardiol)	Phase 3	Heart Failure	8,232	Omecamtiv mecarbil	ubbpwsbkoj(ktppgqpgmp): HR = 0.88 (95% CI, 0.75 - 1.03)	-	13 Oct 2021
				Placebo
NCT02929329 (GALACTIC-HF, CTgov)	Phase 3	Hydrops Fetalis \| Reduced ejection fraction co-occurrent and due to chronic heart failure \| Chronic heart failure	8,256	Placebo (Placebo)	qrmuicjctb = yshhyzodqz owjicocipd (xtmfogipkg, mcpqlnjofh - iakolteamn) View more	-	20 Jul 2021
				Standard of Care+Omecamtiv Mecarbil (Omecamtiv Mecarbil)	qrmuicjctb = xpvwmxgcdv owjicocipd (xtmfogipkg, fxdwkggttp - ayuwphynic) View more
NCT02929329 (GALACTIC-HF, Pubmed \| J Am Coll Cardiol)	Not Applicable	Hydrops Fetalis \| Reduced ejection fraction co-occurrent and due to chronic heart failure \| Chronic heart failure	-	Omecamtiv Mecarbil	zxdcfbxbcy(dbxipipcmy): Hazard Ratio = 0.83 (95% CI, 0.73 - 0.95)	Positive	28 Apr 2021
				Placebo
NCT02929329 (GALACTIC-HF, Pubmed \| Br Dent J \| N Engl J Med) Manual	Phase 3	Heart Failure, Systolic	8,232	Omecamtiv Mecarbil	qveowunobu(zhgcypmiic) = aqeufvjggi bypcrxhsfy (xrghniajgq ) View more	Positive	14 Jan 2021
				Placebo	qveowunobu(zhgcypmiic) = xzqpjyzhmf bypcrxhsfy (xrghniajgq ) View more
NCT02695420 (CTgov)	Phase 2	Heart failure with reduced ejection fraction	81	25 mg Omecamtiv Mecarbil	mztfcbokao(cckssuledd) = psuxcvsmcj uavozngjvo (yhkswjudav, 4.7) View more	-	31 Jan 2020

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