Cytokinetics, Incorporated announced new data relating to aficamten and hypertrophic cardiomyopathy (HCM), were presented at the American Heart Association Scientific Sessions 2024 in Chicago, IL.

“These analyses add to the growing body of evidence supporting the safety and efficacy profile of aficamten and build upon primary findings related to peak VO₂ and improvement in health-related quality of life, while demonstrating a significant and durable reduction in the need for septal reduction therapy,” said Stephen Heitner, M.D., Vice President, Head of Clinical Research. “In addition, analyses presented of real-world data shed light on the disparities that exist in the cost of care for HCM, underscoring the need for improved equity in healthcare across gender and race.”

Treatment with Aficamten Improves VO₂ Recovery; Data from a pre-specified exploratory analysis from SEQUOIA-HCM (Safety, Efficacy, and Quantitative Understanding of Obstruction Impact of Aficamten in HCM) presented showed that treatment with aficamten from 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 with aficamten significantly shortened times for VO₂Rec to decline by 12.5% (VO₂Rec T_12.5%), 25% (VO₂Rec T_25%) and 50% (VO₂Rec T_50%) 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. Additionally, a decrease in VO₂Rec T_12.5% 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)..

Treatment with Aficamten Results 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 of aficamten on 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 receiving aficamten and placebo in any of the five domains of the EQ-5D-5L index. Treatment with aficamten improved 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 receiving aficamten subsequently decreased. These data demonstrate that treatment with aficamten yielded 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 that aficamten improves QoL as measured by Kansas City Cardiomyopathy Questionnaire (KCCQ).

Treatment with Aficamten Durably Reduces SRT-Eligibility After 12 Weeks in Open-Label Extension; Findings from an analysis from FOREST-HCM (Follow-Up, Open-Label, Research Evaluation of Sustained Treatment with Aficamten in HCM), the open-label extension clinical study of aficamten in patients with HCM, related to the efficacy and safety of aficamten in 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 with aficamten, 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, New York Heart Association (NYHA) Functional Class, NT-proBNP and resting and Valsalva left ventricular outflow tract (LVOT) gradient. Changes in left ventricular ejection fraction (LVEF) were modest and similar between SRT-eligible and SRT-ineligible patients. Instances of LVEF <50% and atrial fibrillation or flutter were rare, and similar between groups. These results demonstrate that treatment with aficamten may provide a safe, durable and effective alternative to SRT in many patients with obstructive HCM.

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  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 January 2013 to December 2021 using real-world data from Optum Market Clarity database. Among 5,129 patients identified with obstructive HCM, 52% were female, the mean age was 63.9 years, 77.6% were white and 40% were Medicare recipients. Compared to females, male patients had higher costs including overall total ($71,581 vs $63,710; p=0.014), medical ($70,395 vs $62,455; p=0.013), ambulatory ($16,024 vs $10,776; p<0.001), office visits ($1,906 vs $1,573; p<0.001) and outpatient visits ($14,118 vs $9,202; p<0.001). Compared to white patients, Black patients had significantly higher inpatient admissions costs ($54,572 vs $42,686; p=0.015), Hispanic patients had greater emergency room costs ($1,724 vs $791; p<0.001) and Asian patients had greater office costs ($2,094 vs $1,800; p<0.001). Patients aged 18-39 years had higher costs across all categories (p<0.001) compared to patients 40 or older, except inpatient admissions and prescriptions. Overall, these real-world analyses showed that, for patients with obstructive HCM, being a younger male was associated with increased healthcare costs, with additional differences in cost across race/ethnicity.