Aficamten

PDUFA Target Action Date for Aficamten Set for September 26, 2025; Commercial Launch Preparations Underway for First Potential Approval Five-Year Aspirations Outline Corporate Strategies to Becoming Leading Muscle Biology Specialty Biopharma Company SOUTH SAN FRANCISCO, Calif., Jan. 13, 2025 (GLOBE NEWSWIRE) -- Cytokinetics, Inc. (Nasdaq: CYTK) today provided guidance for corporate milestones expected to occur in 2025 and outlined its aspirational Vision 2030, five-year strategic objectives designed to propel Cytokinetics to becoming the leading muscle-focused specialty biopharmaceutical company intent on meaningfully improving the lives of patients through global access to innovative medicines. “In 2025 we are poised for a defining year with principal focus to the potential approval and commercial launch of aficamten for obstructive HCM in the United States. At the same time, we are executing on a robust clinical trials development program for aficamten inclusive of MAPLE-HCM, with results expected in the first half of this year as may potentially support the use of aficamten as monotherapy,” said Robert I. Blum, Cytokinetics’ President and Chief Executive Officer. “In addition, we are advancing our later-stage development programs for omecamtiv mecarbil and CK-586 in adjacent specialty cardiology indications with intention to further augment our pipeline over time. Cytokinetics is well positioned to prudently and meaningfully deliver increased shareholder value as we continue to execute well on key milestones and position the company to achieve longer-term aspirations defining of our Vision 2030.” Expected 2025 Milestones Cardiac Muscle Programs Aficamten (cardiac myosin inhibitor) Advance go-to-market strategies and prepare to commercially launch aficamten in the U.S. in 2H 2025, subject to approval by the U.S. Food & Drug Administration (FDA). Continue go-to-market plans in Germany and expand commercial readiness activities in Europe in 2025, in preparation for potential approval by the European Medicines Agency (EMA) in 1H 2026. Coordinate with Sanofi to support the potential approval of aficamten in China in 2H 2025, pending approval by the National Medical Products Administration (NMPA). Report topline results from MAPLE-HCM (Metoprolol vs Aficamten in Patients with LVOT Obstruction on Exercise Capacity in HCM), the Phase 3 clinical trial comparing aficamten as monotherapy to metoprolol as monotherapy in patients with symptomatic obstructive HCM, in 1H 2025. Complete patient enrollment of ACACIA-HCM (Assessment Comparing Aficamten to Placebo on Cardiac Endpoints In Adults with Non-Obstructive HCM), the pivotal Phase 3 clinical trial of aficamten in patients with non-obstructive HCM, in 2H 2025. Complete enrollment of the adolescent cohort in CEDAR-HCM (Clinical Evaluation of Dosing with Aficamten to Reduce Obstruction in a Pediatric Population in HCM), a clinical trial of aficamten in a pediatric population with symptomatic obstructive HCM, in 2H 2025. Omecamtiv mecarbil (cardiac myosin activator) Continue patient enrollment in COMET-HF (Confirmation of Omecamtiv Mecarbil Efficacy Trial in Heart Failure), a confirmatory Phase 3 clinical of omecamtiv mecarbil in patients with symptomatic heart failure with severely reduced ejection fraction (HFrEF) through 2025 to enable completion of enrollment in 2026. CK-586 (cardiac myosin inhibitor) Complete enrollment of the first two patient cohorts in AMBER-HFpEF, (Assessment of CK-586 in a Multi-Center, Blinded Evaluation of Safety and Tolerability Results in HFpEF), a Phase 2 clinical trial of CK-586 in patients with symptomatic heart failure with preserved ejection fraction (HFpEF) in 2H 2025. Skeletal Muscle Program CK-089 (fast skeletal muscle troponin activator) Complete the Phase 1 study of CK-089 in healthy human participants in 2025. Ongoing Research Continue ongoing pre-clinical development and research activities directed to additional muscle biology focused programs. Vision 2030 The Company also outlined its Vision 2030: “Empowering Muscle, Empowering Lives” with the following objectives: Innovation: Advance two approved products across three indications and ten novel molecular entities (NMEs) in our pipeline. Ignition: Achieve broad access and rapid use of our medicines in >15 countries throughout North America and Europe. Impact: Reach >100,000 patients globally with our medicines. Inspiration: Foster a patient-centric culture with emphasis on equitable access. Ingenuity: Extend our leadership in muscle biology deploying multiple therapeutic modalities. “Our Vision 2030 provides the aspirational roadmap, aligned with our corporate five-year strategic plan, that will propel us forward as a fully integrated and leading specialty biopharma company intent on delivering innovative medicines to patients around the world,” said Mr. Blum. “Vision 2030 articulates ambitious company goals to deliver product approvals, achieve broad access to our medicines, promote equitable access and advance our pioneering research to benefit patients, shareholders and employees.” About Cytokinetics Cytokinetics is a late-stage, muscle biology specialty biopharmaceutical company focused on discovering, developing and commercializing muscle biology-directed drug candidates as potential treatments for debilitating diseases in which muscle performance is compromised. As a leader in muscle biology and the mechanics of muscle performance, Cytokinetics is intent on meaningfully improving the lives of patients through global access to innovative medicines. Cytokinetics is readying for potential regulatory approvals and commercialization of aficamten, 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). Aficamten is also being evaluated in additional clinical trials enrolling patients with obstructive and non-obstructive HCM. Cytokinetics is also developing omecamtiv 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 from aficamten, 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 about Cytokinetics, visit www.cytokinetics.com and follow us on X, LinkedIn, Facebook and YouTube. Forward-Looking Statements This press release contains forward-looking statements for purposes of the Private Securities Litigation Reform Act of 1995 (the “Act”). Cytokinetics disclaims 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 of aficamten or any of our other drug candidates, our ability to obtain regulatory approval for aficamten for the treatment of obstructive hypertrophic cardiomyopathy or any other indication from FDA or any other regulatory body in the United States or abroad on a timely basis, or at all, the labeling or post-marketing conditions that FDA or another regulatory body may require in connection with the approval of aficamten, our ability to timely enroll and complete the ACACIA-HCM, AMBER-HFpEF, CEDAR-HCM, COMET-HF or CK-089 clinical trials, our ability to timely report the results of the MAPLE-HCM trial, and our ability achieve any element of our Vision 2030. 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 the Securities 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 that Cytokinetics makes in this press release speak only as of the date of this press release. Cytokinetics assumes 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 the CYTOKINETICS and C-shaped logo are registered trademarks of Cytokinetics in the U.S. and certain other countries. Contact: Cytokinetics Diane Weiser Senior Vice President, Corporate Affairs (415) 290-7757 Source: Cytokinetics, Incorporated

SOUTH SAN FRANCISCO, Calif., Dec. 23, 2024 (GLOBE NEWSWIRE) -- Cytokinetics, Incorporated (Nasdaq: CYTK) today announced that the European Medicines Agency (EMA) has validated the Marketing Authorization Application (MAA) for aficamten, 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 for aficamten already under review in both the U.S. and China, the validation of the MAA marks an important milestone in bringing this potential medicine to even more patients with HCM worldwide,” said Robert 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, and Quantitative Understanding of Obstruction Impact of Aficamten in HCM), the pivotal Phase 3 clinical trial of aficamten in patients with symptomatic obstructive HCM, which were published in the New England Journal of Medicine.1 The MAA validation follows the acceptance by the U.S. Food and Drug Administration (FDA) of the New Drug Application (NDA) for aficamten for the treatment of obstructive HCM. The FDA assigned the NDA a standard review with a Prescription Drug User Fee Act (PDUFA) target action date of September 26, 2025. About SEQUOIA-HCM SEQUOIA-HCM (Safety, Efficacy, and Quantitative Understanding of Obstruction Impact of Aficamten in HCM) was the pivotal Phase 3 clinical trial of aficamten in patients with symptomatic obstructive hypertrophic cardiomyopathy (HCM). The results from SEQUOIA-HCM showed that treatment with aficamten for 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 with aficamten versus 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 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. Additional analyses from SEQUOIA-HCM have demonstrated that treatment with aficamten is associated with improvements in cardiac structure, function, and biomarkers without negatively impacting systolic function. About Aficamten Aficamten is 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. Aficamten was 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, aficamten reduced 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 for aficamten is 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. Aficamten was evaluated in SEQUOIA-HCM, a positive pivotal Phase 3 clinical trial in patients with symptomatic obstructive hypertrophic cardiomyopathy (HCM). Aficamten received Breakthrough Therapy Designation for the treatment of symptomatic obstructive HCM from the U.S. Food & Drug Administration (FDA) as well as the National Medical Products Administration (NMPA) in China where it is currently also under review for potential approval. Aficamten is also currently being evaluated in MAPLE-HCM, a Phase 3 clinical trial of aficamten as monotherapy compared to metoprolol as monotherapy in patients with obstructive HCM; ACACIA-HCM, a Phase 3 clinical trial of aficamten in patients with non-obstructive HCM; CEDAR-HCM, a clinical trial of aficamten in a pediatric population with obstructive HCM; and FOREST-HCM, an open-label extension clinical study of aficamten in 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 the U.S.2,3,4 Two-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.5 People 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.6 A subset of patients with HCM are at high risk of progressive disease leading to dilated cardiomyopathy and heart failure necessitating cardiac transplantation. About Cytokinetics Cytokinetics is 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. Cytokinetics is readying for the potential commercialization of aficamten, 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). Aficamten is also being evaluated in additional clinical trials enrolling patients with obstructive and non-obstructive HCM. Cytokinetics is also developing omecamtiv 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 from aficamten, 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 about Cytokinetics, visit www.cytokinetics.com and follow us on X, LinkedIn, Facebook and YouTube. Forward-Looking Statements This press release contains forward-looking statements for purposes of the Private Securities Litigation Reform Act of 1995 (the “Act”). Cytokinetics disclaims 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 of aficamten or any of our other drug candidates, our ability to obtain regulatory approval for aficamten for the treatment of obstructive hypertrophic cardiomyopathy or any other indication from FDA or any other regulatory body in the United States or abroad, and the labeling or post-marketing conditions that FDA or another regulatory body may require in connection with the approval of aficamten. 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 the Securities 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 that Cytokinetics makes in this press release speak only as of the date of this press release. Cytokinetics assumes 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 the CYTOKINETICS and C-shaped logo are registered trademarks of Cytokinetics in the U.S. and certain other countries. Contact: Cytokinetics Diane Weiser Senior Vice President, Corporate Affairs (415) 290-7757 References: Source: Cytokinetics, Incorporated