Cytokinetics Launches ASPEN-HCM: A Real-World Study Evaluating MYQORZO® (aficamten) in Symptomatic Obstructive Hypertrophic Cardiomyopathy

ASPEN-HCM aims to enroll approximately 400 patients to evaluate MYQORZO in routine clinical practice.

Rhea-AI Summary

Cytokinetics (CYTK) has started ASPEN-HCM, an observational study evaluating MYQORZO in adults with symptomatic obstructive hypertrophic cardiomyopathy, a heart-muscle thickening disorder.

Led by the University of California, San Francisco, the prospective, multicenter study will evaluate treatment safety and effectiveness in routine clinical practice across 13 cardiology practices and HCM centers. It aims to enroll approximately 400 patients over an anticipated 12-to-36-month period. Outcomes include changes in heart-pumping function, treatment discontinuations, blood-flow obstruction, functional classification and patient-reported outcomes.

The study will also examine healthcare resource use, hospitalization and procedure rates, cardiac structural changes and treatment patterns. It will investigate potential predictors of treatment response and whether MYQORZO avoids or delays invasive septal reduction therapy.

Positive

• Minor pointASPEN-HCM started to evaluate MYQORZO’s real-world safety and effectiveness in adults with symptomatic obstructive HCM.
• Minor point. Forward-looking: it has not happened yet and may not happen.Approximately 400 patients targeted across 13 practices and centers over an anticipated 12-to-36-month period.
• Minor point. Forward-looking: it has not happened yet and may not happen.Economic analyses planned to assess healthcare resource use, hospitalization and procedure rates before and after treatment.
• Minor point. Forward-looking: it has not happened yet and may not happen.Additional study objectives include treatment-response predictors and MYQORZO’s impact on avoiding or delaying invasive septal reduction therapy.

Negative

• None.

Key Figures

Study sites: 13 cardiology practices and HCM centers of excellence Planned enrollment: approximately 400 patients Study period: 12-to-36 months

Study sites

13 cardiology practices and HCM centers of excellence

ASPEN-HCM study design

Planned enrollment

approximately 400 patients

ASPEN-HCM study

Study period

12-to-36 months

Anticipated study period

Key Terms

lvef, rems, cyp2c9, nyha classification

4 terms

lvef medical

"reductions in left ventricular ejection fraction (LVEF)"

Left ventricular ejection fraction (LVEF) is a percentage that measures how much blood the heart’s main pumping chamber pushes out with each beat, like the share of water a pump empties from a bucket each cycle. Investors watch LVEF because it’s a key medical yardstick used to diagnose and track heart function, shaping demand for drugs, devices, clinical trials, insurance costs and the financial outlook of healthcare-related businesses.

rems regulatory

"under a Risk Evaluation and Mitigation Strategy (REMS)"

Risk Evaluation and Mitigation Strategies (REMS) are safety programs required by regulators for certain medications to make sure their benefits outweigh known dangers; they can include extra training, special certifications for prescribers, restricted distribution channels, patient monitoring, or paperwork. For investors, REMS act like a safety checkpoint that can slow or limit a drug’s sales, raise costs, complicate manufacturing and distribution, and affect revenue forecasts and legal risk, so they matter for a product’s market potential and timeline.

cyp2c9 medical

"metabolized primarily by CYP2C9"

CYP2C9 is a liver protein that acts like a chemical cleanup crew for many prescription drugs, breaking them down so the body can use or eliminate them. Variations in CYP2C9 activity can change how fast drugs are cleared, affecting safety, effective dose, and the risk of interactions—factors that influence clinical trial outcomes, regulatory labels, and market adoption of medications. For investors, CYP2C9-related issues can alter a drug’s commercial prospects or create demand for genetic tests and safer alternatives.

nyha classification medical

"New York Heart Association (NYHA) classification"

A four-level system used by clinicians to describe how much a person's heart disease limits their physical activity and daily life, based on symptoms like shortness of breath and fatigue. Class I means no limitation of ordinary activity; Class II means slight limitation with ordinary activity; Class III means marked limitation with less-than-ordinary activity; Class IV means symptoms at rest or with any physical activity. It is a symptom-based, clinical functional scale rather than a diagnostic test, so classification depends on patient-reported symptoms and clinician judgment and can change over time.

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Multi-center Prospective Study to Track Real-World Safety, Clinical Effectiveness, and Healthcare Resource Impact of MYQORZO in Routine Clinical Practice

SOUTH SAN FRANCISCO, Calif., Sept. 30, 2026 (GLOBE NEWSWIRE) -- Cytokinetics, Incorporated (Nasdaq: CYTK) today announced the start of ASPEN-HCM (Aficamten Study of Patient Experience and Novel Outcomes in Symptomatic Obstructive HCM), a prospective, multicenter, observational real-world study evaluating MYQORZO® (aficamten) in adult patients with symptomatic obstructive hypertrophic cardiomyopathy (oHCM). MYQORZO is a once-daily, oral 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.

“We are pleased to announce the start of ASPEN-HCM, marking an important next step in our ongoing commitment to hypertrophic cardiomyopathy research,” said Sanatan Shreay, Ph.D., Cytokinetics’ Executive Director, Head of Health Economics and Outcomes Research. “The clinical trials we conducted of MYQORZO provided strong evidence supporting its approval. Now, this study will build on that foundation by generating valuable real-world insights that can help inform clinical decision-making and support clinicians in their everyday practices.”

Led by the University of California, San Francisco (UCSF), ASPEN-HCM will evaluate the safety and effectiveness of MYQORZO in routine clinical practice to generate prospective, real-world evidence on the clinical and economic impact of treatment.

“With our deep commitment to driving innovation in cardiology, this important study is designed to provide critical insights about the impact of the latest non-invasive treatment in HCM, the most common inherited heart disease,” said Theodore Abraham, M.D., co-director, UCSF HCM Center of Excellence.

ASPEN-HCM: Study Design

This prospective, multicenter, observational real-world study is designed to evaluate the clinical, economic, and patient impact of MYQORZO in adult patients with oHCM across 13 cardiology practices and HCM centers of excellence. Over an anticipated 12-to-36-month period, the study aims to enroll approximately 400 patients to characterize the real-world safety profile of MYQORZO.

The study will evaluate real-world safety and effectiveness outcomes, including reductions in left ventricular ejection fraction (LVEF), treatment discontinuations, changes in in left ventricular outflow tract gradient (LVOT–G), New York Heart Association (NYHA) classification, and patient-reported outcomes.

Secondary study objectives focus on analyzing healthcare resource utilization, conducting pre-and-post economic analyses on hospitalization and procedure rates, and tracking cardiac structural remodeling using parameters like left ventricular mass index and septal thickness.

Additionally, the study will investigate longitudinal treatment patterns, dose titration pathways, the prevalence and incidence of atrial fibrillation, potential predictors of treatment response, and the clinical impact of MYQORZO on avoiding or delaying invasive septal reduction therapy.

To learn more about ASPEN-HCM, visit www.clinicaltrials.gov.

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.

About MYQORZO® (aficamten)

MYQORZO^® (aficamten) is a cardiac myosin inhibitor approved in the U.S., China, European Union and United Kingdom for the treatment of symptomatic obstructive hypertrophic cardiomyopathy (oHCM). In patients with oHCM, myosin inhibition with MYQORZO reduces cardiac contractility and consequently, left ventricular outflow tract (LVOT) obstruction. MYQORZO was engineered to achieve a predictable exposure response, rapid onset of action and reversibility.³

Aficamten is also under clinical investigation in CEDAR-HCM in a pediatric population with oHCM. Safety and efficacy of aficamten have not been established in a pediatric patient population. In addition, aficamten is being studied in FOREST-HCM, an open-label extension clinical study.

INDICATION

MYQORZO is indicated for the treatment of adults with symptomatic obstructive hypertrophic cardiomyopathy (oHCM) to improve functional capacity and symptoms.

IMPORTANT SAFETY INFORMATION

WARNING: 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 with LVEF <55% is not recommended. Decrease the dose of MYQORZO if LVEF is <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.

CONTRAINDICATIONS

MYQORZO is contraindicated with concomitant use of rifampin.

WARNINGS AND PRECAUTIONS

Heart Failure

MYQORZO reduces cardiac contractility, which can reduce LVEF and cause heart failure.
Patients who experience a serious intercurrent illness (e.g., serious infection) or arrhythmia (e.g., 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 (e.g., 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 (e.g., 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.

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 approved in the U.S., including Boxed WARNING.

Please see full Summary of Product Characteristics approved in the European Union.

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, affecting approximately one out of 350 individuals worldwide.¹

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.³ 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.⁴ 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 approved in the U.S., China, European Union and United Kingdom for the treatment of adults with symptomatic obstructive hypertrophic cardiomyopathy (oHCM). Following positive results in ACACIA-HCM, a Phase 3 clinical trial of aficamten in patients with non-obstructive HCM (nHCM), the company plans to submit a Supplemental New Drug Application in Q4 2026. 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 our ability to obtain regulatory approval for aficamten in nonobstructive hypertrophic cardiomyopathy in any jurisdiction by any particular date, if ever, the number of patients comprising the eligible treatment population for aficamten, or market acceptance of aficamten for the treatment of nonobstructive hypertrophic cardiomyopathy. 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 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 registered trademark of Cytokinetics in the U.S., the European Union and the United Kingdom.

References

1. Tsenov et al. Healthcare access, symptom burden, and psychological impact in hypertrophic cardiomyopathy: a multinational patient-driven survey. Int J Cardiol Cardiovasc Risk Prev2025 Aug 4;27:200485. doi:10.1016/j.ijcrp.2025.200485.
2. 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.
3. 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.
4. Hong Y, Su WW, Li X. Risk factors of sudden cardiac death in hypertrophic.
   
   

Contact:
Cytokinetics
Diane Weiser
Senior Vice President, Corporate Affairs
(415) 290-7757

FAQ

AI-generated questions and answers. How Rhea-AI works. Not financial advice.

What is Cytokinetics’ ASPEN-HCM study evaluating?

ASPEN-HCM evaluates MYQORZO’s safety and effectiveness in routine clinical practice in adults with symptomatic obstructive hypertrophic cardiomyopathy. Led by the University of California, San Francisco, it is a prospective, multicenter observational study that also examines clinical, economic and patient outcomes.

What additional treatment patterns will Cytokinetics’ ASPEN-HCM study track?

ASPEN-HCM will track longitudinal treatment patterns, dose titration pathways, and the prevalence and incidence of atrial fibrillation. Cardiac structural assessments include left ventricular mass index and septal thickness.