Cytokinetics, Incorporated – Cytokinetics Announces Positive Topline Results from ACACIA-HCM, the Pivotal Phase 3 Clinical Trial of Aficamten in Patients with Non-Obstructive Hypertrophic Cardiomyopathy

Trial Met Dual Primary Endpoints of KCCQ and Maximal Exercise Performance 
With Consistent Positive Findings Across Key Secondary Endpoints

Company to Host Conference Call and Webcast Tuesday May 5 at 8:00 AM Eastern Time

SOUTH SAN FRANCISCO, Calif., May 05, 2026 (GLOBE NEWSWIRE) — Cytokinetics, Incorporated (Nasdaq: CYTK) at present introduced constructive topline outcomes from ACACIA-HCM (Assessment Comparing Aficamten to Placebo on Cardiac Endpoints In Adults with Non-Obstructive HCM), the pivotal Phase 3 medical trial of aficamten in sufferers with symptomatic non-obstructive hypertrophic cardiomyopathy (HCM).

ACACIA-HCM met each twin main endpoints, demonstrating statistically important enhancements from baseline to Week 36 in comparison with placebo in each Kansas City Cardiomyopathy Questionnaire Clinical Summary Score (KCCQ-CSS) and maximal train efficiency (pVO₂) (Table 1).

“Patients with non-obstructive HCM have no therapies approved to treat the underlying hypercontractility associated with the disease. We hope that will change with ACACIA-HCM which is the first clinical trial to demonstrate statistically significant improvements in exercise capacity and symptom burden in patients with non-obstructive HCM,” mentioned Fady I. Malik, M.D., Ph.D., Cytokinetics’ Executive Vice President of Research & Development. “We believe that the totality and consistency of evidence favoring aficamten across multiple patient-reported and physician-assessed endpoints of symptom improvement and physical function are clinically meaningful for patients with non-obstructive HCM.”

Table 1: Primary Endpoint Results

The enchancment in KCCQ was sturdy and constant all through the remedy interval in individuals on aficamten. Following washout, KCCQ decreased for individuals on aficamten to match the placebo group. At Week 36, pVO₂ elevated for individuals on aficamten, whereas it remained unchanged for individuals on placebo, constant with prior trials of aficamten in obstructive HCM (oHCM) (Figure 1).

Figure 1: Assessments of KCCQ and pVO₂

Statistically important (p<0.001) enhancements in comparison with placebo have been noticed in key secondary endpoints together with the proportion of individuals with enhancements in New York Heart Association (NYHA) Functional Class, the composite z-rating of ventilatory effectivity and pVO₂, and NT-proBNP.

There have been no new security alerts recognized. The share of individuals finishing deliberate dosing was comparable in these receiving aficamten or placebo (88.4% vs. 90.3%, respectively). Left ventricular ejection fraction (LVEF) <50% occurred in 27 (10%) individuals taking aficamten and in two (1%) individuals taking placebo. Two individuals on aficamten skilled a critical opposed occasion of coronary heart failure related with LVEF <50%. Treatment interruptions as a result of LVEF <40% occurred in 3% of individuals taking aficamten.

“We are grateful to the clinical trial investigators and staff, as well as the patients who participated in this trial,” Dr. Malik added. “We look forward to presenting the results from ACACIA-HCM at an upcoming medical meeting, as well as discussing them with the U.S. FDA and other regulatory authorities.”

Investor Webcast Information

Cytokinetics will host an investor convention name on May 5, 2026, at 8:00 AM Eastern Time to debate the topline outcomes from ACACIA-HCM. Interested events can register on-line at ACACIA-HCM Topline Results. The stay webcast will probably be obtainable on the Investors & Media part of the Cytokinetics web site at https://ir.cytokinetics.com/. A replay of the webcast will probably be archived on the Cytokinetics web site for six months.

About ACACIA-HCM

ACACIA-HCM was a Phase 3, multi-heart, randomized, double-blind, placebo-managed medical trial designed to judge the impact of aficamten in comparison with placebo in sufferers with symptomatic non-obstructive hypertrophic cardiomyopathy (nHCM). The twin main endpoint was the change in Kansas City Cardiomyopathy Questionnaire (KCCQ) Clinical Summary Score and alter in maximal train efficiency (pVO₂) from baseline to Week 36.

Secondary endpoints included the proportion of individuals with ≥1 class enchancment in New York Heart Association (NYHA) useful class, and modifications in the composite z-rating of two cardiopulmonary train testing (CPET) parameters of sub-maximal train efficiency (VE/VCO₂ and pVO₂), NT-proBNP, and left atrial quantity index (LAVI) from baseline to Week 36. After 36 weeks of remedy, individuals continued remedy with aficamten or placebo for as much as 72 weeks to judge further secondary and exploratory analyses together with the time to first cardiovascular occasion. The trial (exterior Japan) concluded when not less than 200 individuals accomplished 52 weeks of remedy.

ACACIA-HCM randomized and handled 516 individuals (exterior Japan) on a 1:1 foundation with aficamten or placebo. Randomization was stratified by persistent atrial fibrillation and presence of intracavitary obstruction. At screening, individuals enrolled in ACACIA-HCM have been required to have resting left ventricular outflow tract gradient (LVOT-G) <30 mmHg and post-Valsalva LVOT-G <50 mmHg in addition to left ventricular ejection fraction (LVEF) ≥60%, respiratory alternate ratio (RER) ≥1.00 and peak VO₂ ≤90% predicted, NT-proBNP ≥300 pg/mL or ≥900 pg/mL if atrial fibrillation or atrial flutter have been current at screening, NYHA useful class II or III and KCCQ Clinical Summary Score ≤85.

Each affected person obtained as much as 4 escalating doses of aficamten or placebo based mostly on echocardiographic steering. Participants who obtained aficamten started with 5 mg dosed as soon as day by day. At weeks 2, 4 and 6 individuals obtained an echocardiogram to find out if they might be up-titrated to escalating doses of 10, 15 or 20 mg. Dose escalation occurred provided that a participant had an LVEF ≥60%. Participants who didn’t meet escalation standards continued the identical dose or have been down-titrated if their LVEF was <50%.

About MYQORZO^® (aficamten)

MYQORZO^® (aficamten) is a cardiac myosin inhibitor permitted in the U.S., China and European Union for the remedy of symptomatic obstructive hypertrophic cardiomyopathy (oHCM). In sufferers with oHCM, myosin inhibition with MYQORZO reduces cardiac contractility and consequently, left ventricular outflow tract (LVOT) obstruction. MYQORZO was engineered to realize a predictable publicity response, fast onset of motion and reversibility.¹

Aficamten can be underneath medical investigation in CEDAR-HCM, in a pediatric inhabitants with oHCM. Aficamten has not been deemed protected or efficient to be used in this affected person inhabitants. In addition, aficamten is being studied in FOREST-HCM, an open-label extension medical research.

INDICATION

MYQORZO is indicated for the remedy of adults with symptomatic obstructive hypertrophic cardiomyopathy (oHCM) to enhance useful capability and signs.

IMPORTANT SAFETY INFORMATION

CONTRAINDICATIONS

MYQORZO is contraindicated with concomitant use of rifampin.

WARNINGS AND PRECAUTIONS

Heart Failure

MYQORZO reduces cardiac contractility, which may scale back LVEF and trigger coronary heart failure.
Patients who expertise a critical intercurrent sickness (eg, critical an infection) or arrhythmia (eg, new or uncontrolled atrial fibrillation) could also be at higher danger of creating systolic dysfunction and coronary heart failure.

Assess sufferers’ medical standing and LVEF previous to and through remedy and alter the MYQORZO dose accordingly. New or worsening arrhythmia, dyspnea, chest ache, fatigue, leg edema, or elevations in N-terminal professional-B-kind natriuretic peptide could also be indicators and signs of coronary heart failure.

Initiation of MYQORZO in sufferers with LVEF <55% isn’t advisable.

MYQORZO REMS Program

MYQORZO is offered solely by a restricted program known as the MYQORZO REMS Program, as a result of of the danger of coronary heart failure as a result of systolic dysfunction.

Notable necessities of the MYQORZO REMS Program embody:

• Prescribers have to be licensed by enrolling in the MYQORZO REMS Program
• Patients should enroll in the MYQORZO REMS Program and comply with ongoing monitoring necessities
• Pharmacies have to be licensed by enrolling in the MYQORZO REMS Program and should solely dispense to sufferers who’re approved to obtain MYQORZO
• Wholesalers and distributors should solely distribute to licensed pharmacies

Further info is offered 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 medicines that inhibit a number of P450 pathways of MYQORZO elimination (eg, fluconazole, voriconazole, or fluvoxamine) or sturdy CYP2C9 inhibitors, and discontinuation of reasonable-to-sturdy CYP3A inducers could result in elevated blood concentrations of aficamten and improve the danger of coronary heart failure as a result of systolic dysfunction. Conversely, initiation of medicines that induce P450 pathways of MYQORZO (eg, rifampin, reasonable-to-sturdy CYP3A inducers) could result in decreased blood concentrations of aficamten and potential loss of effectiveness. Assess LVEF 2 to eight weeks after initiation of such inhibitors or after discontinuation of such inducers and alter the dose of MYQORZO accordingly.

Advise sufferers of the potential for drug interactions. Advise sufferers to tell their healthcare supplier of all concomitant medicines previous to and through MYQORZO remedy.

ADVERSE REACTIONS

Hypertension (8% vs 2%) was the solely opposed response occurring in >5% of sufferers and extra generally on MYQORZO than on placebo in the pivotal trial.

INDICATIONS AND USAGE

MYQORZO is indicated for the remedy of adults with symptomatic obstructive hypertrophic cardiomyopathy (oHCM) to enhance useful capability and signs.

Please see full Prescribing Information, together with Boxed WARNING.

About Hypertrophic Cardiomyopathy

Hypertrophic cardiomyopathy (HCM) is a illness in which the coronary heart muscle turns into abnormally thick. HCM may be obstructive, when thickened muscle blocks blood stream, or non-obstructive, when blood stream isn’t blocked however coronary heart operate continues to be affected. In obstructive HCM, the thickening of cardiac muscle results in the inside of the left ventricle changing into smaller, stiffer and fewer capable of loosen up and fill with blood. Ultimately, HCM limits the coronary heart’s pumping operate, resulting in diminished train capability and a range of signs.

HCM is the commonest monogenic inherited cardiovascular dysfunction, with effectively over 300,000 sufferers recognized in the U.S. However, there are an estimated 400,000-800,000 further sufferers who stay undiagnosed.^2,3,4 Recent evaluation of a big claims database signifies that roughly half of sufferers with HCM have obstructive HCM (oHCM) and half have non-obstructive HCM (nHCM).⁵

People with HCM are at excessive danger of additionally creating cardiovascular problems together with atrial fibrillation, stroke and mitral valve illness.⁶ People with HCM are in danger for doubtlessly deadly ventricular arrhythmias and it’s one of the main causes of sudden cardiac dying in youthful individuals or athletes.⁷ A subset of sufferers with HCM are at excessive danger of progressive illness resulting in dilated cardiomyopathy and coronary heart failure necessitating cardiac transplantation. There aren’t any at the moment permitted therapies for nHCM.

About Cytokinetics

Cytokinetics is a specialty cardiovascular biopharmaceutical firm, constructing on its over 25 years of pioneering scientific improvements in muscle biology, and advancing a pipeline of potential new medicines for sufferers struggling from illnesses of cardiac muscle dysfunction. Cytokinetics’ MYQORZO^® (aficamten) is a cardiac myosin inhibitor permitted in the U.S., European Union and China for the remedy of adults with symptomatic obstructive hypertrophic cardiomyopathy (oHCM). Cytokinetics can be creating omecamtiv mecarbil, an investigational cardiac myosin activator for the potential remedy of sufferers with coronary heart failure with severely diminished ejection fraction and ulacamten, an investigational cardiac myosin inhibitor for the potential remedy of coronary heart failure with preserved ejection fraction, whereas persevering with pre-medical analysis and improvement in muscle biology.

For further details about Cytokinetics, go to www.cytokinetics.com and comply with us on X, LinkedIn, Facebook and YouTube.

Forward-Looking Statements

This press launch incorporates ahead-wanting statements for functions of the Private Securities Litigation Reform Act of 1995 (the “Act”). Cytokinetics disclaims any intent or obligation to replace these ahead-wanting statements and claims the safety of the Act’s Safe Harbor for ahead-wanting statements. Examples of such statements embody, however aren’t restricted to, statements referring to the enrollment, anticipated outcomes or timing of completion of any of our medical trials, the medical meaningfulness, persuasiveness or interpretation of medical trial outcomes, together with for functions of regulatory approval, labeling, or market acceptance, the outcomes of lengthy-time period, secondary or exploratory analyses, together with analyses of time to first cardiovascular occasion, statements referring to our skill to acquire regulatory approval for aficamten in nonobstructive hypertrophic cardiomyopathy in any jurisdiction by any explicit date, if ever, the quantity of sufferers comprising the eligible remedy inhabitants for aficamten, or market acceptance of aficamten for the remedy of nonobstructive hypertrophic cardiomyopathy. Such statements are based mostly on administration’s present expectations, however precise outcomes could differ materially as a result of varied dangers and uncertainties, together with, however not restricted to, potential difficulties or delays in the improvement, testing, regulatory approvals for trial graduation, development or product sale or manufacturing of Cytokinetics’ drug candidates that might sluggish or stop medical improvement or product approval; Cytokinetics’ drug candidates could have opposed uncomfortable side effects or insufficient therapeutic efficacy; the FDA or international regulatory businesses could delay or restrict Cytokinetics’ skill to conduct medical trials; Cytokinetics could also be unable to acquire or preserve patent or commerce secret safety for its mental property; requirements of care could change, rendering Cytokinetics’ drug candidates out of date; and aggressive merchandise or various therapies could also be developed by others for the remedy of indications Cytokinetics’ drug candidates and potential drug candidates could goal. For additional info relating to these and different dangers associated to Cytokinetics’ enterprise, buyers ought to seek the advice of Cytokinetics’ filings with the Securities and Exchange Commission together with the danger components included in Cytokinetics’ most up-to-date Annual Report on Form 10-Ok and subsequent studies filed with the SEC.

CYTOKINETICS^® and the CYTOKINETICS C-formed brand are registered emblems of Cytokinetics in the U.S. and sure different nations.

MYQORZO^® is a registered trademark of Cytokinetics in the U.S. and the European Union.

References

1. Hartman JJ, Hwee DT, Robert-Paganin J, et al. Aficamten is a small-molecule cardiac myosin inhibitor designed to deal with hypertrophic cardiomyopathy. Nat Cardiovasc Res. 2024;3(8) :1003-1016. doi:10.1038/s44161-024-00505-0
2. 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
3. Symphony Health 2016-2021 Patient Claims Data DoF;
4. 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.
5. 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
6. Gersh, B.J., Maron, B.J., Bonow, R.O., Dearani, J.A., Fifer, M.A., Link, M.S., et al. 2011 ACCF/AHA pointers for the prognosis and remedy of hypertrophic cardiomyopathy. A report of the American College of Cardiology Foundation/American Heart Association Task Force on follow pointers. Journal of the American College of Cardiology and Circulation, 58, e212-260.
7. Hong Y, Su WW, Li X. Risk components of sudden cardiac dying in hypertrophic cardiomyopathy. Current Opinion in Cardiology. 2022 Jan 1;37(1):15-21

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

A photograph accompanying this announcement is offered at https://www.globenewswire.com/NewsRoom/AttachmentNg/adcf634e-a2a7-4cdf-98b3-ca658eab1b35

Figure 1: Assessments of KCCQ and pVO2

Figure 1: Assessments of KCCQ and pVO2

Source: Cytokinetics, Incorporated