{"id":998742,"date":"2026-08-28T05:48:09","date_gmt":"2026-08-28T09:48:09","guid":{"rendered":"https:\/\/www.marketnewsdesk.com\/index.php\/cytokinetics-announces-positive-results-from-acacia-hcm-presented-in-hot-line-session-at-the-european-society-of-cardiology-esc-congress-2026-and-published-in-the-new-england-journal-of-medicine\/"},"modified":"2026-08-28T05:48:09","modified_gmt":"2026-08-28T09:48:09","slug":"cytokinetics-announces-positive-results-from-acacia-hcm-presented-in-hot-line-session-at-the-european-society-of-cardiology-esc-congress-2026-and-published-in-the-new-england-journal-of-medicine","status":"publish","type":"post","link":"https:\/\/www.marketnewsdesk.com\/index.php\/cytokinetics-announces-positive-results-from-acacia-hcm-presented-in-hot-line-session-at-the-european-society-of-cardiology-esc-congress-2026-and-published-in-the-new-england-journal-of-medicine\/","title":{"rendered":"Cytokinetics Announces Positive Results from ACACIA-HCM Presented in Hot Line Session at the European Society of Cardiology (ESC) Congress 2026 and Published in The New England Journal of Medicine"},"content":{"rendered":"
\n

\n ACACIA-HCM is the First Phase 3 Clinical Trial to Successfully Demonstrate Statistically Significant Improvements Across Both Patient-Reported and Physician-Assessed Endpoints in Non-Obstructive HCM<\/em>\n <\/p>\n

\n Supplemental New Drug Application to be Submitted to FDA in Fourth Quarter<\/em>\n <\/p>\n

\n Company to Host Investor Event and Webcast Today at 2:00 PM Central European Summer Time (8:00 AM Eastern Time)<\/em>\n <\/p>\n

SOUTH SAN FRANCISCO, Calif., Aug. 28, 2026 (GLOBE NEWSWIRE) — Cytokinetics, Incorporated (Nasdaq: CYTK) today announced the primary results from ACACIA-HCM (A<\/strong>ssessment C<\/strong>omparing A<\/em><\/strong>ficamten<\/em> to Placebo on C<\/strong>ardiac Endpoints I<\/strong>n A<\/strong>dults with Non-Obstructive HCM<\/strong>), the pivotal Phase 3 clinical trial of aficamten <\/em>in patients with symptomatic non-obstructive hypertrophic cardiomyopathy (nHCM) were presented in a Hot Line Session at the European Society of Cardiology (ESC) Congress 2026 in Munich, Germany, and simultaneously published in\u00a0The New England Journal of Medicine<\/em><\/a>.1<\/sup><\/p>\n

ACACIA-HCM met both dual primary endpoints, demonstrating statistically significant improvements from baseline to Week 36 compared to placebo in both Kansas City Cardiomyopathy Questionnaire Clinical Summary Score (KCCQ-CSS) and maximal exercise performance (pVO2<\/sub>). Results for the dual primary endpoint analysis consistently favored aficamten<\/em> across prespecified subgroups studied.<\/p>\n

Based on the positive results from ACACIA-HCM, Cytokinetics plans to submit a supplemental New Drug Application to the U.S. Food and Drug Administration (FDA) for aficamten<\/em> for the treatment of adult patients with symptomatic nHCM in the fourth quarter of 2026.<\/p>\n

\u201cACACIA-HCM is the first-ever positive clinical trial in non-obstructive HCM,\u201d said Stephen Heitner, M.D., Cytokinetics\u2019 Chief Medical Officer. \u201cAs shown in presentation and publication, the results from ACACIA-HCM are both statistically robust, consistent across prespecified subgroups and other secondary endpoints, and clinically impactful for these patients with non-obstructive HCM. As such, these data may potentially translate into a new treatment option for patients with HCM with no available medical treatments that directly address their underlying disease. If approved, we look forward to seeing aficamten <\/em>become the first cardiac myosin inhibitor to treat the full spectrum of symptomatic HCM.\u201d<\/p>\n

\u201cWe are grateful to the patient community and the clinical trial team who contributed to the successful completion of ACACIA-HCM, a fitting tribute to the patients and families who have struggled with this disease for many years and across generations. We are also pleased that most of the eligible patients from ACACIA-HCM are continuing treatment with aficamten<\/em> in FOREST-HCM,\u201d said Ahmad Masri, M.D., M.S., Director, Hypertrophic Cardiomyopathy Center and Cardiac Amyloidosis Program, Oregon Health & Science University and Principal Investigator of ACACIA-HCM. \u201cWe look forward to applying this study\u2019s groundbreaking scientific knowledge into clinical practice upon potential approval, addressing this area of HCM where patients do not have adequate treatment options.\u201d<\/p>\n

\n
\n ACACIA-HCM: Efficacy<\/u>
\n <\/strong>\n <\/p>\n

The baseline characteristics of patients enrolled in ACACIA-HCM were well-matched between treatment groups and consistent with a symptomatic nHCM phenotype with substantial disease burden despite normal left ventricular ejection fraction (LVEF).<\/p>\n

There were two pre-specified primary endpoints, KCCQ-CSS and pVO\u2082, evaluated in parallel. For each endpoint, a p-value of 0.025 or less was considered statistically significant. Compared to placebo, treatment with aficamten<\/em> demonstrated statistically significant improvements in both dual primary endpoints at 36 weeks (Table 1).<\/p>\n

\n Table 1: Dual Primary Endpoint Results<\/strong>\n <\/p>\n\n\n\n\n\n\n
\n Primary
Endpoints<\/strong>\n <\/td>\n		\n Change from Baseline to Week 36
<\/strong>
\n LSM [95% CI]<\/strong>\n <\/td>\n		\n
\n Aficamten
<\/em>
\n <\/strong>
\n vs Placebo<\/strong>
\n
\n LSM (95% CI)<\/strong>\n <\/td>\n		\n P-value<\/strong>\n <\/td>\n<\/tr>\n
\u00a0<\/td>\n\n Aficamten<\/em>\n <\/td>\nPlacebo<\/td>\n\u00a0<\/td>\n\u00a0<\/td>\n<\/tr>\n
KCCQ-CSS<\/td>\n11.4 [9.6 \u2013 13.2]<\/td>\n8.4 [6.6 \u2013 10.2]<\/td>\n3.0 (0.5 – 5.5)<\/td>\n0.021<\/td>\n<\/tr>\n
pVO2<\/sub> (ml\/kg\/min)<\/td>\n0.64 [0.32 \u2013 0.95]<\/td>\n-0.03 [-0.35 \u2013 0.28]<\/td>\n0.67 (0.22 – 1.1)<\/td>\n0.003<\/td>\n<\/tr>\n
LSM = least square mean; CI = confidence interval<\/td>\n<\/tr>\n<\/table>\n

\n
Improvements in KCCQ-CSS relative to placebo were noted beyond titration and throughout the treatment period in participants treated with aficamten<\/em>, such that patients remaining on treatment at 72 weeks demonstrated a least square mean (LSM) change from baseline in KCCQ-CSS of 7.0 points. Of note, following a washout of aficamten<\/em> for four weeks, the KCCQ-CSS declined from the end of treatment for participants on aficamten<\/em> to match that in the placebo group (Figure 1).<\/p>\n

\n Figure 1: Assessment of KCCQ-CSS <\/strong>\n <\/p>\n

\n \n <\/p>\n

\n The figure is based on the observed data, except that the inset box of Week 36 LSM difference and p-value are from the primary analysis imputed data.<\/em>\n <\/p>\n

At Week 36, pVO2<\/sub> increased for participants on aficamten<\/em> while it remained unchanged for participants on placebo (Figure 2).<\/p>\n

\n Figure 2: Assessment of pVO<\/strong>
\n
\n 2<\/strong>
\n <\/sub>\n <\/p>\n

\n \n <\/p>\n

The treatment effect of aficamten <\/em>on both dual primary endpoints was consistent across all prespecified subgroups, including baseline LVEF, atrial fibrillation status, genotype, age, sex, whether patients were receiving background beta-blocker therapy and whether patients had intracavitary obstruction at baseline (Figure 3).<\/p>\n

\n Figure 3: Pre-Specified Subgroups for KCCQ-CSS and pVO<\/strong>
\n
\n 2<\/strong>
\n <\/sub>\n <\/p>\n

\n \n <\/p>\n

Statistically significant improvements compared to placebo were observed in key ranked secondary endpoints, including New York Heart Association (NYHA) Functional Class, the composite z-score of two cardiopulmonary exercise testing (CPET) parameters (VE\/VCO2<\/sub> and pVO2<\/sub>), and NT-proBNP, a biomarker of cardiac wall stress. Statistical significance was not met on the endpoints of change from baseline in left atrial volume index or the time to first cardiovascular event for aficamten<\/em> compared to placebo (Table 2).<\/p>\n

\n Table 2: Secondary Endpoints<\/strong>\n <\/p>\n\n\n\n\n\n\n\n\n\n
\n Secondary
Endpoints<\/strong>\n <\/td>\n		\n Change from Baseline to Week 36<\/strong>
\n
\n LSM (95% CI)<\/strong>\n <\/td>\n		\n
\n Aficamten<\/em>
\n <\/strong>
\n vs
Placebo<\/strong>
\n
\n Treatment
Difference <\/strong>
\n
\n (95% CI)<\/strong>\n <\/td>\n		\n P-value<\/strong>\n <\/td>\n<\/tr>\n
\u00a0<\/td>\n\n Aficamten<\/em>\n <\/td>\nPlacebo<\/td>\n\u00a0<\/td>\n\u00a0<\/td>\n<\/tr>\n
Improvement of \u22651 NYHA class at week 36 \u2013 n (%)<\/td>\n108 (41.9)<\/td>\n72 (27.8)<\/td>\n14.1 (6.0 to 22.2)<\/td>\n<0.001<\/td>\n<\/tr>\n
Change in composite z-score of two CPET parameters (pVO2<\/sub> and VE\/VCO2<\/sub>) at week 36<\/td>\n0.05 (-0.01 to 0.12)<\/td>\n-0.09 (-0.14 to -0.05)<\/td>\n0.14 (0.07 to 0.22)<\/td>\n<0.001<\/td>\n<\/tr>\n
Proportional change in NT-proBNP level at week 36 \u2013 pg\/ml<\/td>\n0.43 (0.39 to 0.46)<\/td>\n1.00 (0.93 to 1.08)<\/td>\n0.43 (0.38 to 0.47)<\/td>\n<0.001<\/td>\n<\/tr>\n
Change in left atrial volume index at week 36<\/td>\n-0.17 (-1.12 to 0.79)<\/td>\n1.13 (0.19 to 2.08)<\/td>\n-1.30 (-2.64 to 0.04)<\/td>\n\n
0.058<\/td>\n<\/tr>\n
Time to first cardiovascular event \u2013 weeks<\/td>\n68.2 (66.2 to 70.2)<\/td>\n68.8 (66.9 to 70.7)<\/td>\n-0.6 (-3.3 to 2.2)<\/td>\n\n
0.678<\/td>\n<\/tr>\n
\n Treatment difference is from statistical modeling<\/em>\n <\/td>\n<\/tr>\n<\/table>\n

\n
\n
\n
ACACIA-HCM: Safety<\/u>
\n <\/strong>\n <\/p>\n

\n Aficamten<\/em>\u00a0was generally well-tolerated in the study. Non-fatal adverse events that led to early study drug discontinuation occurred in 18 (7.0%) and 5 (1.9%) patients on aficamten<\/em> and placebo, respectively. Serious adverse events occurred in 52 (20.2%) patients on aficamten<\/em> compared to 38 (14.7%) patients on placebo. In the time to the first cardiovascular event endpoint, an event (cardiovascular death, heart transplantation or left ventricular assist device, aborted sudden cardiac death, non-fatal stroke, heart failure hospitalization, or cardiac arrhythmia [atrial fibrillation or ventricular tachyarrhythmia] requiring treatment or hospitalization) occurred in 22 (8.5%) patients in the aficamten<\/em> arm and 20 (7.7%) in the placebo arm.<\/p>\n

LVEF <50% occurred in 27 (10.5%) patients taking aficamten<\/em> and in two (0.8%) patients taking placebo. At baseline, patients who experienced LVEF <50% tended to have lower LVEF, lower exercise capacity, and a higher frequency of atrial fibrillation history. Of the 27 patients with LVEF <50% on aficamten<\/em>, 21\u00a0completed treatment while receiving the same\u00a0or lower doses.<\/p>\n

As previously reported, two participants on aficamten<\/em> experienced a serious adverse event of heart failure associated with LVEF <50%. Ten additional patients on aficamten<\/em> had serious adverse events of heart failure not associated with LVEF <50%, compared to three patients on placebo. All heart failure events in patients treated with aficamten<\/em> occurred during dose titration or prior to Week 12 and were generally responsive to a short course of diuretic therapy.<\/p>\n

\n
\n ACACIA-HCM: Global Efficacy Analysis<\/u>
\n <\/strong>\n <\/p>\n

An additional analysis from ACACIA-HCM was also presented during the Hot Line presentation which evaluated the global efficacy of aficamten<\/em> in patients with symptomatic nHCM. The analysis assessed clinical improvements across five key domains: exercise capacity, cardiac structure, cardiac biomarkers, diastolic function and symptom burden.<\/p>\n

At 36 weeks, treatment with aficamten<\/em> demonstrated statistically significant improvements across all five efficacy metrics compared to placebo (p<0.001), with 53% of patients on aficamten<\/em> demonstrating a clinical response in three or more outcome measures compared to only 13% of patients on placebo. These results help place the primary results into clinical context and underscore the opportunity to impact several aspects of disease burden by addressing the underlying pathobiology in patients living with symptomatic nHCM. The results from this sub-analysis were also published in Circulation<\/em>.<\/a>2<\/sup><\/p>\n

\n Investor Webcast Information<\/strong>\n <\/p>\n

Cytokinetics will host an in-person investor event in Munich, Germany on August 28, 2026, at 2:00 PM Central European Summer Time (8:00 AM Eastern Time). The event will also be simultaneously webcast live online. Interested parties must register to attend in-person or online at https:\/\/acacia-hcm-investor-event-esc-2026.open-exchange.net\/welcome<\/a>.<\/p>\n

Registered attendees may access the webcast by visiting the Investor & Media section of the Cytokinetics website at www.cytokinetics.com<\/a>. The webcast replay will be archived on the Cytokinetics website for six months.<\/p>\n

\n About ACACIA-HCM<\/strong>\n <\/p>\n

ACACIA-HCM was a Phase 3, multi-center, randomized, double-blind, placebo-controlled clinical trial designed to evaluate the effect of aficamten <\/em>compared to placebo in patients with symptomatic non-obstructive hypertrophic cardiomyopathy (nHCM). The dual primary endpoint was the change in Kansas City Cardiomyopathy Questionnaire (KCCQ) Clinical Summary Score and change in maximal exercise performance (pVO2<\/sub>) from baseline to Week 36.<\/p>\n

Secondary endpoints included the proportion of participants with \u22651 class improvement in NYHA functional class, and changes in the composite z-score of two cardiopulmonary exercise testing (CPET) parameters of sub-maximal exercise performance (VE\/VCO2<\/sub> and pVO2<\/sub>), NT-proBNP, and left atrial volume index (LAVI) from baseline to Week 36. After 36 weeks of treatment, participants continued treatment with aficamten<\/em> or placebo for up to 72 weeks to evaluate additional secondary and exploration analyses including the time to first cardiovascular event. The trial (outside Japan) concluded when at least 200 patients completed 52 weeks of treatment.<\/p>\n

ACACIA-HCM randomized and treated 517 participants (outside Japan) on a 1:1 basis with aficamten<\/em> or placebo. Randomization was stratified by persistent atrial fibrillation and presence of intracavitary obstruction. At screening, participants enrolled in ACACIA-HCM were 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) \u226560%, respiratory exchange ratio (RER) \u22651.00 and pVO2<\/sub> \u226490% predicted, NT-proBNP \u2265300 pg\/mL or \u2265900 pg\/mL if atrial fibrillation or atrial flutter were present at screening, NYHA functional class II or III and KCCQ Clinical Summary Score \u226485.<\/p>\n

Each patient received up to four escalating doses of aficamten <\/em>or placebo based on echocardiographic guidance. Participants who received aficamten<\/em> began with 5 mg dosed once daily. At weeks 2, 4 and 6 participants received an echocardiogram to determine if they would be up-titrated to escalating doses of 10, 15 or 20 mg. Dose escalation occurred only if a participant had an LVEF \u226560%. Participants who did not meet escalation criteria continued the same dose or were down-titrated if their LVEF was <50%.<\/p>\n

\n About MYQORZO\u00ae\u00a0(<\/strong>
\n
\n aficamten<\/em>
\n <\/strong>
\n )<\/strong>\n <\/p>\n

MYQORZO\u00ae<\/sup> (aficamten<\/em>) 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<\/sup><\/p>\n

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

\n INDICATION <\/strong>\n <\/p>\n

MYQORZO is indicated for the treatment of adults with symptomatic obstructive hypertrophic cardiomyopathy (oHCM) to improve functional capacity and symptoms.<\/p>\n

\n IMPORTANT SAFETY INFORMATION<\/strong>\n <\/p>\n\n\n\n
\n WARNING: RISK OF HEART FAILURE
<\/strong>\n <\/td>\n<\/tr>\n
\n
\n
\n <\/strong>
\n MYQORZO reduces left ventricular ejection fraction (LVEF) and can cause heart failure due to systolic dysfunction.<\/strong><\/p>\n

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 \u226540%. Interrupt the dose of MYQORZO if LVEF <40% or if the patient experiences heart failure symptoms or worsening clinical status due to systolic dysfunction<\/strong>
\n
\n .<\/em>
\n <\/strong><\/p>\n

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.<\/strong>\n <\/td>\n<\/tr>\n<\/table>\n

\n
\n
CONTRAINDICATIONS <\/strong>\n <\/p>\n

MYQORZO is contraindicated with concomitant use of rifampin.<\/p>\n

\n WARNINGS AND PRECAUTIONS<\/strong>\n <\/p>\n

\n Heart Failure<\/strong>\n <\/p>\n

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.<\/p>\n

Assess patients\u2019 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.<\/p>\n

Initiation of MYQORZO in patients with LVEF <55% is not recommended.<\/p>\n

\n MYQORZO REMS Program <\/strong>\n <\/p>\n

MYQORZO is available only through a restricted program called the MYQORZO REMS Program, because of the risk of heart failure due to systolic dysfunction.<\/p>\n

Notable requirements of the MYQORZO REMS Program include:<\/p>\n