Cytokinetics Announces European Medicines Agency Validation of Marketing Authorization Application for Aficamten for the Treatment of Obstructive Hypertrophic Cardiomyopathy

CYTK 12.23.2024

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SOUTH SAN FRANCISCO, Calif.,Dec. 23, 2024(GLOBE NEWSWIRE) --Cytokinetics, Incorporated(Nasdaq: CYTK) today announced that theEuropean Medicines Agency(EMA) has validated the Marketing Authorization Application (MAA) foraficamten, a next-in-class cardiac myosin inhibitor, for the treatment of obstructive hypertrophic cardiomyopathy (HCM). The MAA will now be reviewed by the Committee for Medicinal Products for Human Use (CHMP).

“With regulatory filings foraficamtenalready under review in both theU.S.andChina, the validation of the MAA marks an important milestone in bringing this potential medicine to even more patients with HCM worldwide,” saidRobert I. Blum, Cytokinetics’ President and Chief Executive Officer. “We look forward to working with EMA in connection with their review of our application.”

The MAA 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 MAA validation follows the acceptance by theU.S. Food and Drug Administration(FDA) of the New Drug Application (NDA) foraficamtenfor the treatment of obstructive HCM. The FDA assigned the NDA a standard review with a Prescription Drug User Fee Act (PDUFA) target action date ofSeptember 26, 2025.

About SEQUOIA-HCM

SEQUOIA-HCM (Safety,Efficacy, andQuantitativeUnderstanding ofObstructionImpact ofAficamteninHCM) was the pivotal Phase 3 clinical trial ofaficamtenin patients with symptomatic obstructive hypertrophic cardiomyopathy (HCM).

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 improvements in cardiac structure, function, and biomarkers without negatively impacting systolic function.

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.Cytokineticsis readying for the potential commercialization ofaficamten,a next-in-class cardiac myosin inhibitor following positive results from SEQUOIA-HCM, the pivotal Phase 3 clinical trial in patients with obstructive hypertrophic cardiomyopathy (HCM).Aficamtenis also being evaluated in additional clinical trials enrolling patients with obstructive and non-obstructive HCM.Cytokineticsis also developingomecamtiv mecarbil, a cardiac myosin activator, in patients with heart failure with severely reduced ejection fraction (HFrEF), CK-586, a cardiac myosin inhibitor with a mechanism of action distinct fromaficamten,for the potential treatment of heart failure with preserved ejection fraction (HFpEF) and CK-089, a fast skeletal muscle troponin activator with potential therapeutic application to a specific type of muscular dystrophy and other conditions of impaired skeletal muscle function.

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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Source: Cytokinetics, Incorporated

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