Cytokinetics, Incorporated (Nasdaq: CYTK) is bullish today after announcing its positive topline results. The rsults from SEQUOIA-HCM (Safety, Efficacy, and Quantitative Understanding of Obstruction Impact of Aficamten in HCM), the pivotal Phase 3 clinical trial of aficamten in patients with symptomatic obstructive hypertrophic cardiomyopathy (HCM).
The results of SEQUOIA-HCM show that treatment with aficamten significantly improved exercise capacity compared to placebo, increasing peak oxygen uptake (pVO2) measured by cardiopulmonary exercise testing (CPET) by a least square mean difference (95% CI) of 1.74 (1.04 – 2.44) mL/kg/min (p=0.000002). The treatment effect with aficamten was consistent across all prespecified subgroups reflective of patient baseline characteristics and treatment strategies, including patients receiving or not receiving background beta-blocker therapy.
Aficamten was well-tolerated in SEQUOIA-HCM with an adverse event profile comparable to placebo. Treatment emergent serious adverse events occurred in 8 (5.6%) and 13 (9.3%) patients on aficamten and placebo, respectively. Core echocardiographic left ventricular ejection fraction (LVEF) was observed to be <50% in 5 patients (3.5%) on aficamten compared to 1 patient (0.7%) on placebo. There were no instances of worsening heart failure or treatment interruptions due to low LVEF.
“The results from SEQUOIA-HCM meet our high expectations for both efficacy and safety, demonstrating that aficamten added to standard of care therapy had a positive impact on exercise capacity as well as rapid and sustained effects on symptoms and functional class in patients with obstructive HCM while maintaining the safety and tolerability that we have previously observed,” said Fady I. Malik, M.D., Ph.D., Cytokinetics’ Executive Vice President of Research & Development. “We believe these results are consistent with those observed in REDWOOD-HCM, the Phase 2 clinical trial of aficamten, and FOREST-HCM, the ongoing open label extension clinical trial, and may reflect a profile enabling of aficamten to become the cardiac myosin inhibitor of choice among physicians and patients. We thank the patients, investigators and site personnel who participated in SEQUOIA-HCM and continue to support our ongoing clinical research and look forward to sharing the full results from this trial at a medical meeting in 2024.”
“Cardiac myosin inhibition represents an exciting new therapy option for patients with symptomatic obstructive HCM and I am pleased to see these impressive results from SEQUOIA-HCM,” said Martin Maron, M.D., Director, Hypertrophic Cardiomyopathy Center, Lahey Hospital and Medical Center, Burlington, MA; Tufts University School of Medicine, and National Principal Investigator of SEQUOIA-HCM. “A therapy like aficamten that improves exercise capacity in a clinically meaningful manner, absent low LVEF events that interrupt treatment, should be a welcome addition for HCM patients as well as the clinicians who treat them.”