Cytokinetics Announces FDA Acceptance of New Drug Application for Aficamten for the Treatment of Obstructive Hypertrophic Cardiomyopathy

CYTK 12.02.2024

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Drug:unknown-unknown aficamten
Diseases:obstructive hypertrophic cardiomyopathy
Diseases:non-obstructive hypertrophic cardiomyopathy
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PDUFA Target Action Date Set forSeptember 26, 2025

SOUTH SAN FRANCISCO, Calif.,Dec. 02, 2024(GLOBE NEWSWIRE) --Cytokinetics, Incorporated(Nasdaq: CYTK) today announced that theU.S.Food & Drug Administration (FDA) has accepted the company’s New Drug Application (NDA) foraficamten, a next-in-class cardiac myosin inhibitor, for the treatment of obstructive hypertrophic cardiomyopathy (HCM).

The FDA assigned the NDA a standard review with a Prescription Drug User Fee Act (PDUFA) target action date ofSeptember 26, 2025. The FDA is not currently planning to hold an advisory committee meeting to discuss the application.

“The NDA acceptance foraficamtenby FDA is a significant milestone that moves our company another step closer to hopefully translating our pioneering science to the potential benefit of patients suffering from obstructive HCM. The results from SEQUOIA-HCM, the pivotal Phase 3 clinical trial, which form the foundation of the NDA, demonstrated thataficamtenhas a positive impact on exercise capacity, clinical outcomes, symptom burden and cardiac biomarkers in patients with HCM, with a consistent effect across all prespecified subgroups and a favorable safety and tolerability profile,” saidRobert I. Blum, Cytokinetics’ President and Chief Executive Officer. “If approved by FDA, we believeaficamtenmay expand utilization of cardiac myosin inhibitors and become the preferred choice amongst physicians and patients while also anchoring our emerging specialty cardiology franchise arising from Cytokinetics’ industry-leading muscle biology directed research.”

The NDA is supported by the results from SEQUOIA-HCM (Safety,Efficacy, andQuantitativeUnderstanding ofObstructionImpact ofAficamteninHCM), the pivotal Phase 3 clinical trial ofaficamtenin patients with symptomatic obstructive HCM, which were published in theNew England Journal of Medicine.1

The results from SEQUOIA-HCM showed that treatment withaficamtenfor 24 weeks significantly improved exercise capacity compared to placebo, increasing peak oxygen uptake (pVO2) measured by cardiopulmonary exercise testing (CPET) by 1.8 ml/kg/min compared to baseline in patients treated withaficamtenversus 0.0 ml/kg/min in patients treated with placebo (least square mean (LSM) difference [95% CI] of 1.74 mL/kg/min [1.04 - 2.44]; p=0.000002). Statistically significant improvements were observed in all 10 prespecified secondary endpoints, including Valsalva left ventricular outflow tract (LVOT) gradient,New York Heart Association(NYHA) Functional Class, Kansas City Cardiomyopathy Clinical Summary Score (KCCQ-CSS), and proportion with LVOT gradient <30 mmHg, each at 12 and 24 weeks, as well as duration of guideline eligibility for septal reduction therapy (SRT), and total workload during CPET at 24 weeks. Treatment emergent serious adverse events occurred in 5.6% and 9.3% of patients onaficamtenand placebo, respectively. Core echocardiographic left ventricular ejection fraction (LVEF) was observed to be <50% in 5 patients (3.5%) onaficamtencompared to 1 patient (0.7%) on placebo. There were no instances of worsening heart failure or treatment interruptions due to low LVEF.

Additional analyses from SEQUOIA-HCM have demonstrated that treatment withaficamtenis associated with favorable cardiac remodeling as well as improvements in cardiac structure, function, and biomarkers without negatively impacting systolic function.

The FDA previously grantedaficamtenOrphan Drug Designation for the treatment of symptomatic HCM inJanuary 2021and Breakthrough Therapy Designation for the treatment of obstructive HCM inDecember 2021.

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 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, 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 theU.S. Food & Drug Administration(FDA) as well as theNational Medical Products Administration(NMPA) inChinawhere it is currently also under review for potential approval.

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; 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 theU.S.2,3,4Two-thirds of patients with HCM have obstructive HCM (oHCM), where the thickening of the cardiac muscle leads to left ventricular outflow tract (LVOT) obstruction, while one-third have non-obstructive HCM (nHCM), where blood flow isn’t impacted, but the heart muscle is still thickened. People with HCM are at high risk of also developing cardiovascular complications including atrial fibrillation, stroke and mitral valve disease.5People with HCM are at risk for potentially fatal ventricular arrhythmias and it is one of the leading causes of sudden cardiac death in younger people or athletes.6A subset of patients with HCM are at high risk of progressive disease leading to dilated cardiomyopathy and heart failure necessitating cardiac transplantation.

AboutCytokinetics

Cytokineticsis a late-stage, specialty cardiovascular biopharmaceutical company focused on discovering, developing and commercializing muscle biology-directed drug candidates as potential treatments for debilitating diseases in which cardiac muscle performance is compromised. As a leader in muscle biology and the mechanics of muscle performance, the company is developing small molecule drug candidates specifically engineered to impact myocardial muscle function and contractility. Following positive results from SEQUOIA-HCM, the pivotal Phase 3 clinical trial evaluatingaficamten,a next-in-class cardiac myosin inhibitor, in obstructive hypertrophic cardiomyopathy (HCM),Cytokineticsis progressing regulatory submissions foraficamtenfor the treatment of obstructive HCM in the US,Europe, andChina.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, 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.Cytokineticsis also developingomecamtiv mecarbil, a cardiac muscle activator, in patients with heart failure with severely reduced ejection fraction (HFrEF), CK-586, a cardiac myosin inhibitor with a mechanism of action distinct fromaficamtenfor the potential treatment of heart failure with preserved ejection fraction (HFpEF), and CK-089, a fast skeletal muscle troponin activator (FSTA) for the potential treatment of a specific type of muscular dystrophy.

For additional information aboutCytokinetics, visitwww.cytokinetics.comand follow us onX,LinkedIn,FacebookandYouTube.

Forward-Looking Statements

This press release contains forward-looking statements for purposes of the Private Securities Litigation Reform Act of 1995 (the “Act”).Cytokineticsdisclaims 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 inthe United Statesor abroad, and the labeling or post-marketing conditions that FDA or another regulatory body may require in connection with the approval ofaficamten. Such statements are based on management’s current expectations, but actual results may differ materially due to various risks and uncertainties, including, but not limited to the risks related to Cytokinetics’ business outlines in Cytokinetics’ filings with theSecurities and Exchange Commission. Forward-looking statements are not guarantees of future performance, and Cytokinetics’ actual results of operations, financial condition and liquidity, and the development of the industry in which it operates, may differ materially from the forward-looking statements contained in this press release. Any forward-looking statements thatCytokineticsmakes in this press release speak only as of the date of this press release.Cytokineticsassumes no obligation to update its forward-looking statements whether as a result of new information, future events or otherwise, after the date of this press release.

CYTOKINETICS® and theCYTOKINETICSand C-shaped logo are registered trademarks ofCytokineticsin theU.S.and certain other countries.

Contact:CytokineticsDiane WeiserSenior Vice President, Corporate Affairs(415) 290-7757

References:

  1. Maron, MS, et al. Aficamten for Symptomatic Obstructive Hypertrophic Cardiomyopathy. N Engl J Med. DOI: 10.1056/NEJMoa2401424
  2. 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
  3. Symphony Health2016-2021 Patient Claims Data DoF;
  4. 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.
  5. 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 theAmerican College of Cardiology Foundation/American Heart Association Task Forceon practice guidelines.Journal of the American College of Cardiology and Circulation, 58, e212-260.
  6. Hong Y, Su WW,Li X. Riskfactors of sudden cardiac death in hypertrophic cardiomyopathy. Current Opinion in Cardiology. 2022Jan 1;37(1):15-21

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