Cytokinetics presents baseline characteristics from SEQUOIA-HCM, the pivotal phase III clinical trial of aficamten at the HCM Society scientific sessions.

“The baseline characteristics of SEQUOIA-HCM show that the patients enrolled into this pivotal trial align with our objectives for aficamten, which include assessing our next-in-class cardiac myosin inhibitor in a population with substantial deficit in exercise capacity and significant symptom burden despite background treatment with guideline directed medical therapies,” said Fady I. Malik, M.D., Ph.D., Cytokinetics’ Executive Vice President of Research & Development. “We look forward to announcing topline results from SEQUOIA-HCM by the end of the year and our hopefully elaborating on clinical effects to the benefit of patients.”

SEQUOIA-HCM: Baseline Characteristics: SEQUOIA-HCM was designed to evaluate aficamten in patients with symptomatic obstructive HCM on background medical therapy over a 24-week period. Patients enrolled in SEQUOIA-HCM were required to have severe left ventricular outflow tract (LVOT) obstruction as evidenced by a resting LVOT-G greater than 30 mmHg, a post-Valsalva peak LVOT-G greater than 50 mmHg, NYHA functional class II or III, and a peak VO2 less than 90% predicted.

SEQUOIA-HCM enrolled a total of 282 patients, with one third from the United States, one half from Europe and Israel, and the remainder from China. Patients were on average 59.1 years of age, 40.4% female, and 21% were non-white. Background medical therapy consisted of beta-blockers (61%), calcium channel blockers (26.6%), and disopyramide (12.8%); combination background therapy was permitted. At baseline, 75.9% of patients were NYHA functional class II, 23.8% were functional class III, and 0.4% were functional class IV. One quarter of patients were guideline-eligible for septal reduction therapy at the time of enrollment. The pooled mean (SD) for baseline peak VO2 was 18.5 (4.5) mL/kg/min or 56.9% (11.8) of age- and sex-predicted peak VO2, and for the Kansas City Cardiomyopathy Questionnaire Clinical Summary Score (KCCQ-CSS) was 74.7 (18.0). The geometric mean (Q1, Q3) high-sensitivity cardiac troponin I was 12.1 (7.7, 27.3) ng/L. Key baseline characteristics that remain blinded include left ventricular ejection fraction (LVEF), resting and Valsalva LVOT-G, and NT-proBNP.