Cytokinetics Presents New Data Relating to Aficamten and Hypertrophic Cardiomyopathy at the American Heart Association Scientific Sessions 2024
CYTK 11.16.2024

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Two New Analyses from SEQUOIA-HCM Demonstrate Treatment with Aficamten Improves Post-Exercise Oxygen Uptake Recovery and Quality of Life
New Data from FOREST-HCM Demonstrate Aficamten Durably Reduces the Proportion of Patients Guideline Eligible for Septal Reduction Therapy
Analyses of Real-World Data
“These analyses add to the growing body of evidence supporting the safety and efficacy profile ofaficamtenand build upon primary findings related to peak VO2and improvement in health-related quality of life, while demonstrating a significant and durable reduction in the need for septal reduction therapy,” said
Treatment withAficamtenImproves VO2Recovery
Data from a pre-specified exploratory analysis from SEQUOIA-HCM (Safety,Efficacy, andQuantitativeUnderstanding ofObstructionImpact ofAficamteninHCM) presented today showed that treatment withaficamtenfrom baseline to Week 24 resulted in significantly shortened post-exercise oxygen-uptake (VO2) recovery (VO2Rec). Prolonged VO2Rec has previously been linked to adverse outcomes in patients with other forms of heart failure. The analysis demonstrated that treatment withaficamtensignificantly shortened times for VO2Rec to decline by 12.5% (VO2Rec T12.5%), 25% (VO2Rec T25%) and 50% (VO2Rec T50%) of peak VO2, corresponding to absolute reductions relative to placebo of 8 seconds (p<0.001), 7 seconds (p<0.001) and 8 seconds (p=0.01), respectively (Table 1). Additionally, a decrease in VO2Rec T12.5%corresponded to a decrease in NT-proBNP levels (p<0.001), high-sensitivity cardiac troponin I levels (hs-cTnI) (p<0.001), resting left ventricular outflow tract gradient (LVOT-G) (p=0.003) and Valsalva LVOT-G (p=0.003).
Table 1. Effect ofAficamtenon Post-Exercise Oxygen Uptake Recovery in Patients with Obstructive HCM | ||||||||||
AFICAMTEN | PLACEBO | |||||||||
Variable | n | Baseline | Week 24 | Absolutedifference (SD) | n | Baseline | Week 24 | Absolutedifference (SD) | TreatmentEffect(95% CI) | p-value |
PeakVO2(mL/kg/min) | 133 | 18.4 ± 4.5 | 20.2 ± 5.2 | 1.8 ± 3.1 | 130 | 18.6 ± 4.6 | 18.6 ± 4.7 | 0.0 ± 2.7 | 1.7 (1.0, 2.4) | p<0.001 |
VO2 | 134 | 19 ± 20 | 15 ± 18 | -4 ± 19 | 129 | 17 ± 19 | 18 ± 19 | 1 ± 19 | -4 (-8, -0) | p=0.047 |
VO2recovery 12.5% (s) | 126 | 45 ± 20 | 38 ± 18 | -7 ± 19 | 127 | 45 ± 22 | 46 ± 23 | 1 ± 16 | -8 (-12, -5) | p<0.001 |
VO2recovery 25% (s) | 123 | 66 ± 21 | 60 ± 19 | -6 ± 18 | 126 | 70 ± 27 | 70 ± 28 | -0 ± 17 | -7 (-11, -3) | p<0.001 |
VO2recovery 50% (s) | 117 | 115 ± 32 | 107 ± 32 | -8 ± 27 | 116 | 116 ± 38 | 116 ± 36 | 0 ± 26 | -8 (-15, -2) | p=0.01 |
Treatment withAficamtenResults in Sustained and Significant Improvements in Health-Related Quality of Life
Data were also presented from an additional pre-specified exploratory analysis of SEQUOIA-HCM that evaluated the effect ofaficamtenon patient-reported health status using two quality of life (QoL) measurements, EuroQol 5-Dimension 5-Level (EQ-5D-5L) and EuroQol Visual Analogue Scale (EQ-VAS). EQ-5D-5L (range from 0 to 1) and EQ-VAS (range from 0 to 100) were measured at baseline through Week 24, with higher scores indicating better QoL. At baseline, there were no differences between patients receivingaficamtenand placebo in any of the five domains of the EQ-5D-5L index. Treatment withaficamtenimproved the EQ-5D-5L index score by 0.04 (p=0.008) and the EQ-VAS score by 4.5 points (p=0.002) compared to placebo, with significant differences observed as early as eight weeks after treatment initiation (p=0.005). Following withdrawal of treatment at the end of the clinical trial, QoL benefits in patients who were receivingaficamtensubsequently decreased. These data demonstrate that treatment withaficamtenyielded early, sustained and significant improvement in overall health-related QoL among patients with obstructive HCM as measured by EQ-5D-5L, reinforcing previously reported data showing thataficamtenimproves QoL as measured by Kansas City Cardiomyopathy Questionnaire (KCCQ).
Treatment withAficamtenDurably Reduces SRT-Eligibility After 12 Weeks in Open-Label Extension
Findings from an analysis from FOREST-HCM (Follow-Up,Open-Label,ResearchEvaluation ofSustainedTreatment withAficamteninHCM), the open-label extension clinical study ofaficamtenin patients with HCM, related to the efficacy and safety ofaficamtenin patients who at baseline were guideline-eligible for septal reduction therapy (SRT) were also presented. Of the 280 patients with obstructive HCM enrolled in FOREST-HCM with ≥12 weeks of follow-up at the time of this analysis, 97 (35%) met guideline eligibility criteria for SRT at baseline; after 12 weeks of treatment withaficamten, only 3 (3%) remained SRT guideline-eligible. When comparing those patients who were SRT guideline-eligible versus those who were not at baseline, there were similar, robust improvements in KCCQ,
Analyses of Real-World Data Reveals Differences in Costs Across Gender, Age and Race/Ethnicity in Patients with Obstructive HCM
A new health economics and outcomes research (HEOR) study presented today evaluated the impact of sociodemographic characteristics on cost of care in patients with obstructive HCM. These retrospective analyses included adults diagnosed with obstructive HCM from
AboutAficamten
Aficamtenis 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 and as may translate into next-in-class potential in clinical development.Aficamtenwas 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 hypertrophic cardiomyopathy (HCM). In preclinical models,aficamtenreduced 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 foraficamtenis assessing its potential as a treatment that improves exercise capacity and relieves symptoms in patients with HCM as well as its potential long-term effects on cardiac structure and function. Aficamtenwas evaluated in SEQUOIA-HCM (Safety,Efficacy, andQuantitativeUnderstanding ofObstructionImpact ofAficamteninHCM), a positive pivotal Phase 3 clinical trial in patients with symptomatic obstructive hypertrophic cardiomyopathy (HCM).Aficamtenreceived Breakthrough Therapy Designation for the treatment of symptomatic obstructive HCM from the
Aficamtenis also currently being evaluated in MAPLE-HCM, a Phase 3 clinical trial ofaficamtenas monotherapy compared to metoprolol as monotherapy in patients with obstructive HCM, ACACIA-HCM, a Phase 3 clinical trial ofaficamtenin patients with non-obstructive HCM, and CEDAR-HCM, a clinical trial ofaficamtenin a pediatric population with obstructive HCM, and FOREST-HCM, an open-label extension clinical study ofaficamtenin patients with HCM.
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
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This press release contains forward-looking statements for purposes of the Private Securities Litigation Reform Act of 1995 (the “Act”). Cytokinetics disclaims any intent or obligation to update these forward-looking statements and claims the protection of the Act’s Safe Harbor for forward-looking statements. Examples of such statements include, but are not limited to, statements express or implied relating to the properties or potential benefits ofaficamtenor any of our other drug candidates, our ability to obtain regulatory approval foraficamtenfor the treatment of obstructive hypertrophic cardiomyopathy or any other indication from FDA or any other regulatory body in
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References:
- CVrg: Heart Failure 2020-2029, p 44; Maron et al. 2013 DOI: 10.1016/S0140-6736(12)60397-3; Maron et al 2018 10.1056/NEJMra1710575
Symphony Health 2016-2021 Patient Claims Data DoF;- Maron MS, Hellawell JL, Lucove JC,
Farzaneh-Far R , Olivotto I. Occurrence of Clinically Diagnosed Hypertrophic Cardiomyopathy inthe 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. 2022Jan 1 ;37(1):15-21

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