Cytokinetics, Incorporated · NASDAQ:CYTK
Cytokinetics — Q1 2025 Earnings Presentation
63 slides from Cytokinetics's earnings presentation, May 6, 2025
Slide 1: Empowering muscle, empowering lives
EMPOWERING muscle EMPOWERING lives
Slide 2: Forward-Looking Statements
Forward-Looking Statements This presentation 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 Ha
Slide 3: Our Mission
Our Mission To bring forward new medicines to improve the healthspan of people with devastating cardiovascular and neuromuscular diseases of impaired muscle function. 3
Slide 4: A Commitment to Muscle-Directed Cardiac Medicines
A Commitment to Muscle-Directed Cardiac Medicines Protein Target Therapeutic Area Drug Candidate Research Pre-Clinical Phase 1 Phase 2 Phase 3 Approval U.S. PDUFA date 12/26/25 oHCM Aficamten China NDA & EU MAA on file Myosin oHCM Aficamten (Monotherapy*) Pediatric oHCM Aficamten Myosin-Targeted The
Slide 5: Strong Financial Position
Strong Financial Position Well-capitalized to execute launch & advance R&D pipeline ~$1.1B in cash, cash equivalents and investments as of March 31, 2025 Further access to capital Proceeds of $75M from Tranche 4 loan received in April 2025 through term loans[1] with Eligible to draw up to $100M in 2
Slide 6: Myosin Platform Fuels Multiple Milestones and Increased Value
Myosin Platform Fuels Multiple Milestones and Increased Value Potential Regulatory Results Approval For illustrative purposes only Aficamten Aficamten SEQUOIA- oHCM HCM Results Potential Approval Enhancing Aficamten U.S. PDUFA date of December oHCM Mono shareholder Emerging 26, 2025, China NDA & EU
Slide 7: Innovation Ignition Impact
INNOVATION Advance 2 approved products across 3 indications and 10 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 cu
Slide 8: Building a Specialty Cardiology Franchise
Building a Specialty Cardiology Franchise 8
Slide 9: Addressing Difficult to Treat Populations Within Heart Failure
Addressing Difficult to Treat Populations Within Heart Failure Specialty cardiology franchise strategy applies to markets with similar characteristics HFrEF HFpEF HCM Severely Supranormal EF reduced EF Opportunity for aficamten as Addressing underserved segment of HF Expanding into adjacent populati
Slide 10: Building a Specialty Cardiology Franchise Anchored by Aficamten
Building a Specialty Cardiology Franchise Anchored by Aficamten Potential patient market for specialty cardiology franchise strategy Myosin Myosin-Targeted Therapies 800K4 HFpEF + EF ≥ 65% in 2030 ~840K3 HFrEF + EF <30% in 2029 CK-586 HFpEF With nHCM as a 250-400K1,2 proxy, potential to nHCM patient
Slide 11: Potential for Multiple Specialty Cardiology Launches
Potential for Multiple Specialty Cardiology Launches Drug Potential Candidate Indication 2024 2025 2026 2027 2028 2029 2030+ oHCM Aficamten oHCM Mono (MAPLE-HCM) nHCM (ACACIA-HCM) Omecamtiv HFrEF Mecarbil CK-586 HFpEF Aficamten, omecamtiv mecarbil and CK-586 are investigational drugs and are not app
Slide 12: Specialty Cardiology Business Has Potential for High ROI
Specialty Cardiology Business Has Potential for High ROI Concentrated Prescribers Higher Revenue Per Prescriber Opportunity To Grow ~80K cardiologists/PCPs treat CV diseases Market Through Diagnosis ~10K $$$$/ DIAGNOSIS 50 % cardiologists HCP treat ~80% HCM $/HCP Distribution Limited to Differentiat
Slide 13: Potential Benefits of a Specialty Cardiology Franchise
Potential Benefits of a Specialty Cardiology Franchise • Significant FTE cost savings • 1:1 customer – rep relationship • Reduced operational support • Single point of contact for teams, IT systems & analytics Customer HCP & office staff enables • Efficiency in sales training, Financial Experience i
Slide 14: HCP & Patient-Directed HCM Awareness Campaigns Launched
HCP & Patient-Directed HCM Awareness Campaigns Launched HCP Portal CRM Registration (Medscape) Paid Social Educational Materials (Facebook) HCP Market Patient Market Development Website Development Website HCMBeyondTheHeart.com/HCP HCMBeyondTheHeart.com Paid Search Programmatic/ Programmatic/Digital
Slide 15: Aficamten
G Cytokinetics’ Aficamten Aficamten is an investigational agent and has not been approved for use by the U.S. Food & Drug Administration (FDA) or any regulatory agency. The safety and effectiveness of this product have not been established. 15
Slide 16: Opportunity for CMIs in Diagnosed, Symptomatic HCM Patients
Opportunity for CMIs in Diagnosed, Symptomatic HCM Patients US Prevalence of HCM1 700K to 1.1M ~65% ~35% US oHCM1,3 nHCM1,3 USDiagnosed HCM oHCM Prevalence ~300K2 nHCM Prevalence 450-700K1,3 250-400K1,3 Diagnosed oHCM Diagnosed nHCM ~200K2,3 ~100K2,3 Sympt. Sympt. >130K4,5 >40K4,5 Projections and fo
Slide 17: $10B Potential Market of CMI-Eligible Patients
$10B Potential Market of CMI-Eligible Patients Diagnosis of HCM anticipated to grow 5x the rate of the general U.S. population US and EU HCM Patients in 2035 Illustrative 880K1,2 Expected global population of diagnosed patients with Projected Diagnosed HCM Patients oHCM & nHCM (5x the growth rate of
Slide 18: Market Research Shows Aficamten May Achieve High Share & Grow Category
Market Research Shows Aficamten May Achieve High Share & Grow Category • If approved with target profile, aficamten may expand total CMI market & create opportunity in newly treated CMI patients Key attributes that may drive preference include the potential for: LVOT gradient Change in NYHA Pharmaco
Slide 19: SEQUOIA-HCM: Pivotal Phase 3 Trial
SEQUOIA-HCM: Pivotal Phase 3 Trial Primary endpoint: Change in pVO2 by CPET from baseline to Week 24 Randomization Aficamten + SoC End of Study Patients with Screening oHCM treated Secondary objectives include measuring with SOC with post-Valsalva 1 change in KCCQ & improvement in peak LVOT-G ≥50 mm
Slide 20: SEQUOIA-HCM: Primary Endpoint
SEQUOIA-HCM: Primary Endpoint Significant improvement in exercise capacity compared to placebo Results presented at Heart Failure 2024 and published in NEJM Absolute Change from Baseline to Week 24 LS mean Change from Baseline to Week 24 Aficamten 2.5 P=0.000002 Placebo 21 2.0 ΔpVO2 (mL/kg/min) pVO2
Slide 21: SEQUOIA-HCM: Secondary & Exploratory Endpoints
SEQUOIA-HCM: Secondary & Exploratory Endpoints Significant improvement in gradients by ~60% with no significant adverse change in LVEF LS mean difference: Resting LVOT-G Valsalva LVOT-G - 50 mmHg P<0.001 P<0.001 100 100 90 90 Resting LVOT-G (mmHg) Valsalva LVOT-G (mmHg) 80 80 70 70 60 60 50 50 40 40
Slide 22: SEQUOIA-HCM: Secondary & Exploratory Endpoints
SEQUOIA-HCM: Secondary & Exploratory Endpoints Significant improvement in patient symptom burden and quality of life KCCQ-OSS2 ≥1 NYHA FC Improvement1 P<0.001 SAQ7-SS2 P<0.001 Proportion of patients with ≥1 100 NYHA FC improvement (%) 90 80 58% 70 60 50 40 30 20 10 0 2 4 6 8 12 16 20 24 Weeks Mean d
Slide 23: SEQUOIA-HCM: Safety Data
SEQUOIA-HCM: Safety Data Placebo Aficamten Event, n (%) (n=140) (n=142) Overall AEs 99 (70.7) 105 (73.9) Headache 10 (7.1) 11 (7.7) Hypertension 3 (2.1) 11 (7.7) Palpitations 4 (2.9) 10 (7.0) AEs with ≥5% incidence Upper respiratory infection 12 (8.6) 9 (6.3) COVID-19 9 (6.4) 8 (5.6) Dyspnea 8 (5.7)
Slide 24: SEQUOIA-HCM: CMR Sub-Study
SEQUOIA-HCM: CMR Sub-Study Aficamten associated with favorable cardiac remodeling Among 50 of the 284 eligible patients who opted to complete the CMR sub-study there was: Left Ventricular Mass Index (LVMI) (g/m2) -15.4 (-24.5,-6.3); P=0.001 • Significant improvement in LVMI • Favorable cardiac remod
Slide 25: Integrated Safety Analysis
Integrated Safety Analysis Analysis represents 206 patient-years* of exposure to aficamten a Cumulative Placebo-controlled aficamten-treated b pool pool Aficamten Aficamten Placebo • <4% of patients experienced LVEF <50% Number of participants 283 170 153 c LVEF <50% , n (%) 11 (3.9) 9 (5.3) 1 (0.7)
Slide 26: Implementation of Dosing in Real-World Setting (FOREST-HCM)
Implementation of Dosing in Real-World Setting (FOREST-HCM) Low incidence of LVEF <50% in patients with oHCM treated with aficamten 5% N = 282 5% N = 267 4% 4.2% 4% Incidence LVEF <50% Incidence LVEF <50% Placebo-corrected 3% 3% 3.0% 2.8% 2% 2% 1% 1% 1 patient in common 0% 0% Core Lab Site Site ✓ No
Slide 27: Cardiologists Located in Concentrated Geographic Clusters Across the US
Cardiologists Located in Concentrated Geographic Clusters Across the US ~75% of the HCM patient volume is treated by ~10,000 cardiologists HCM Patient Concentration by Cardiologist Geographic Distribution of HCM Patients 100% 90% 80% 70% 60% 50% 40% 30% 20% 10% 0% 7,000 10,000 … 30,000 Note: include
Slide 28: U.S. Sales Team Designed Based on Efficiency & Customer Feedback
U.S. Sales Team Designed Based on Efficiency & Customer Feedback Traditional Models Our Design Principles Several functions with very focused roles Simple model creating quality experience Overwhelmed customers, “It’s too much” Hire team with deep experience in specialty Hospital Rep Community Strat
Slide 29: Building a Patient-Centric Treatment Experience
Building a Patient-Centric Treatment Experience Landscape assessment to identify Detailed integrated CMI specific current trends coupled with primary treatment experience build underway and secondary insights to understand in collaboration with clinicians and current challenges and barriers to patie
Slide 30: Strategy in Place to Support Market Access at Launch
Strategy in Place to Support Market Access at Launch Payer value proposition PIE engagements with key Channel & dispensing Patient support strengthened with payer accounts strategy designed to services will provide clinical & HEOR enhance patient robust prior- evidence experience authorization & med
Slide 31: Advancing EU Launch Readiness Activities
Advancing EU Launch Readiness Activities Key Key Hires in Hires Zug andin Europe Munich KeyKey Activities Activities to Support to Support Launch Launch Highly experienced hires in Zug, Switzerland in Design the EU distribution model & select EU 3PL Regulatory, Medical Affairs, Commercial, Market Ac
Slide 32: Gated Build of Commercial Infrastructure
Gated Build of Commercial Infrastructure Sales representative hiring to occur in proximity to approval 2/3 of hiring to occur at-approval Activities initiated upon key de-risking events Underway before SEQUOIA-HCM readout Market access strategy Commercial leadership Pricing strategy Marketing Distri
Slide 33: Global Presence of Aficamten & Progress of Marketing Applications
Global Presence of Aficamten & Progress of Marketing Applications 2025 2025 Partnered with United States China Sanofi in China; U.S. PDUFA NDA on file with date 12/26/25 priority review 2026 Europe Japan MAA on file Partnered with in Europe Bayer in Japan Aficamten is an investigational drug and is
Slide 34: Strategies for Success: First-Time Biotech Launches
Strategies for Success: First-Time Biotech Launches Cytokinetics’ commercial strategy validated by industry findings First-time launchers are more likely to succeed if they: Develop a multi-asset portfolio Invest in SG&A at launch year & continue over subsequent years Leverage precision-based market
Slide 35: Positive Topline Results from MAPLE-HCM
Positive Topline Results from MAPLE-HCM • Positive topline results from MAPLE-HCM demonstrating superiority of aficamten to metoprolol in patients with obstructive HCM • Expect to share full results at an upcoming major medical meeting Aficamten is an investigational drug and is not approved by any
Slide 36: Ongoing Clinical Trials of Aficamten
Ongoing Clinical Trials of Aficamten Pivotal Phase 3 clinical Clinical trial in a pediatric Open-label extension trial in nHCM population with oHCM clinical study in HCM Enrollment complete; Expect to complete enrollment Ongoing data expected in 1H 2026 of adolescent cohort in 2H 2025 Aficamten is a
Slide 37: ACACIA-HCM: Pivotal Phase 3 Trial in nHCM
ACACIA-HCM: Pivotal Phase 3 Trial in nHCM Enrollment complete; results expected 1H 2026 Patients with: Randomization • Trial enrolled over 516 Aficamten End of Study Symptomatic symptomatic nHCM patients N = 420 Screening (1:1) (NYHA Class (EOS) II/III) nHCM, • Dual primary endpoint: change KCCQ-CSS
Slide 38: Non-Obstructive HCM: A Growing and Underserved Population
Non-Obstructive HCM: A Growing and Underserved Population Projected HCM Prevalence • Significant underserved segment of the HCM population Present • No effective medical or surgical HCM Prevalence nHCM treatment options (1/2) nHCM • Diagnosis trends indicate that (1/3) nHCM is growing at a faster ra
Slide 39: Omecamtiv Mecarbil
G Cytokinetics’ Omecamtiv Mecarbil Omecamtiv mecarbil is an investigational agent and has not been approved for use by the U.S. Food & Drug Administration (FDA) or any regulatory agency. The safety and effectiveness of this product have not been established. 39
Slide 40: Omecamtiv Mecarbil: Potential for High-Risk Severe HF Patients Despite GDMT
Omecamtiv Mecarbil: Potential for High-Risk Severe HF Patients Despite GDMT Efficient, pragmatic Phase 3 clinical trial High Unmet Need Ph 3 clinical trial results in 8,000 patients point to important treatment benefit The large and growing heart failure population faces frequent Planning hospitaliz
Slide 41: GALACTIC-HF: Clinical Trial Overview
GALACTIC-HF: Clinical Trial Overview Phase 3 clinical trial Event-driven clinical trial; 8,256 patients randomized in 35 countries at 944 clinical trial sites Chronic HFrEF Patients Randomization 1:1 Currently hospitalized for Omecamtiv mecarbil + Standard HF Therapy End of Study primary reason of H
Slide 42: Primary Composite Endpoint
Primary Composite Endpoint 50 Placebo HR = 0.92 45 (95% CI, 0.86–0.99) 40 Omecamtiv Cumulative incidence, % P = 0.025 mecarbil 35 30 CV Death 25 HR = 1.01 (95% CI, 0.92–1.11) P = 0.86 20 Omecamtiv 15 mecarbil 10 5 0 0 6 12 18 24 30 36 Patients Months (30 days) since randomization at risk, n Placebo
Slide 43: Benefit Observed to Increase as Baseline LVEF Decreased
Benefit Observed to Increase as Baseline LVEF Decreased Incidence of Primary Composite Endpoint Relative Treatment Effect on Primary Endpoint 60 50 Placebo 1.2 Incidence Rate (per 100 py) 40 Treatment Effect (Ratio) Treatment 1.0 Effect 30 Omecamtiv 0.8 mecarbil 20 0.6 4,456 patients 3,776 patients
Slide 44: Large Treatment Effect in Easily Defined HF Population
Large Treatment Effect in Easily Defined HF Population Nom N Hazard Ratio (95% CI) ARR p-value All Patients 8232 0.025 2.1 LVEF <30% 4704 <0.001 4.9 + Hosp <3 mos 2836 <0.001 6.2 + SBP <110 1881 0.004 7.2 + Class III/IV 2249 <0.001 8.9 + NT-proBNP ≥1000 pg/mL 2852 <0.001 8.8 0.6 0.7 0.8 0.9 1 1.1 1.
Slide 45: Residual Risk is High Despite Best Therapy
Residual Risk is High Despite Best Therapy DAPA-HF Trial: Patients on GDMT including SGLT2-i DAPA-HF Trial Residual Risk DAPA-HF trial (dapagliflozin group) • Primary endpoint: CV Death/HF hospitalization/urgent HF visit • 4744 patients • Renin-angiotensin system blocker 94% • Dapagliflozin 96% • Mi
Slide 46: Phase 3 Confirmatory Clinical Trial Design
Phase 3 Confirmatory Clinical Trial Design Currently enrolling COMET-HF: Confirmation of Omecamtiv Mecarbil Efficacy Trial in Heart Failure 2-week washout period LVEF < 30% 2-week run-in period • Primary endpoint: time to within 6 mo Omecamtiv Mecarbil + Standard of Care CV death, HF events, Direct
Slide 47: Large and Growing Target Patient Population in US
Large and Growing Target Patient Population in US Prevalence in Adults (18+, USA) 2020 Estimates 2029 Estimates 7.1M1 8.6M3 Heart Failure Including HFpEF 3.5M2 4.3M3 HFrEF Population 2.4M2 2.8M3 HFrEF Patients with EF<30% ~720K4 ~840K4 High risk HF patients with EF<30% Proposed Cardiac Function Mark
Slide 48: Higher Event Rate & Costs in Patients with Severely Reduced EF
Higher Event Rate & Costs in Patients with Severely Reduced EF Accounts for ~60% of HFrEF hospitalizations5 35% of patients with severely reduced EF re-hospitalized within 1 year6 $15,493 per HF re-hospitalization7 Direct costs from HF re-hospitalizations projected to increase from $3.9 billion in 2
Slide 49: The Business Case for Omecamtiv Mecarbil
The Business Case for Omecamtiv Mecarbil Significant clinical need, lack of treatments drives higher price potential in HF with severely reduced EF “Severely Reduced EF” US Price Potential Premium to market Disease Severely Reduced EF Market Insights Severity LVEF <30 Payer ~1M patients Positioning
Slide 50: CK-586
G Cytokinetics’ CK-586 is an investigational agent and has not been approved for use by the U.S. Food & Drug Administration (FDA) or any regulatory agency. The safety and effectiveness of this product have not been established. 50
Slide 51: Heart Failure with Preserved Ejection Fraction (HFpEF)
Heart Failure with Preserved Ejection Fraction (HFpEF) Despite broad use of standard treatments & advances in care, the prognosis for patients with HF is poor1 ~75% ~84% HFpEF patients will HFpEF patients will Subset of HFpEF patients Significant increase Lifetime healthcare costs die within five ye
Slide 52: CK-586: Distinct Mechanism of Action from Aficamten
CK-586: Distinct Mechanism of Action from Aficamten “Fewer hands pulling on the rope” Before CK-586 After CK-586 CK- Mechanochemical 586 domain ATP Post-Powerstroke State Actin sliding Actin sliding CK-586 is an investigational drug and is not approved by any regulatory agency. Its safety and effica
Slide 53: CK-586: Shallow In Vivo Concentration-Response
CK-586: Shallow In Vivo Concentration-Response CK-586 has a shorter half-life than aficamten Pharmacodynamic window Fractional shortening IC50 /IC10 ratio mavacamten 2.8x aficamten 9.9x CK-586 9.3x IC10: plasma concentration at 10% relative reduction in fractional shortening IC50: plasma concentrati
Slide 54: Phase 1 Data Support Advancement to Phase 2 Clinical Trial
Phase 1 Data Support Advancement to Phase 2 Clinical Trial Phase 1 study design: 7 SAD cohorts (10 mg to 600 mg) & 2 MAD cohorts (100 & Plasma Concentration Change in LVEF vs. 200 mg once daily), 10 participants each (mean [SD]) over time after single ascending doses of CK-586 CK-586 Plasma Concentr
Slide 55: Phase 2 Study Schema
Phase 2 Study Schema Currently enrolling AMBER-HFpEF: Assessment of CK-586 in a Multi-Center, Blinded Evaluation of Safety and Tolerability Results in HFpEF 6 WEEKS 6 WEEKS 2 weeks 1 Enrolling HFpEF SoC + Placebo Cohort 1 patients with: n=12 Washout SoC + 150 mg CK-586 SoC + 300 mg CK-586 • LVEF ≥ 6
Slide 56: CK-586: Focusing on Patients with HFpEF and EF ≥ 60%
CK-586: Focusing on Patients with HFpEF and EF ≥ 60 7.9M 4.0M (2024) 1.0M (EF>65) to 2.0M (EF > 60) Heart Failure Prevalence in 2024, US 50% of HF with HFpEF (EF ≥ 50%) ~20% of HF with EF ≥ 60% 2024 prevalence calculated using published, historic estimate and projected to 2024 using age adjusted US
Slide 57: CK-586 May Address Unmet Needs of HFpEF Patients
CK-586 May Address Unmet Needs of HFpEF Patients Proposed Mechanistic Benefits Target Product Profile • Statistically significant reduction in composite of mortality and • CK-586 may benefit cardiac relaxation hospitalization outcomes during diastole • Oral QD tablet • CK-586 may reduce symptoms and
Slide 58: Financials & Milestones
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Slide 59: Strong Financial Position
Strong Financial Position Well-capitalized to execute launch & advance R&D pipeline ~$1.1B in cash, cash equivalents and investments as of March 31, 2025 Further access to capital Proceeds of $75M from Tranche 4 loan received in April 2025 through term loans[1] with Eligible to draw up to $100M in 2
Slide 60: 2025 Financial Guidance
2025 Financial Guidance Guidance Issued on Feb. 27, 2025 GAAP Operating Expense[1] $670M to $710M Stock-based Compensation included in GAAP Operating Expense $120M to $110M The financial guidance does not include the effect of GAAP adjustments as may be caused by events that occur subsequent to publ
Slide 61: Robust Pipeline, Upcoming Commercial Launch & Solid Financial Position
Robust Pipeline, Upcoming Commercial Launch & Solid Financial Position U.S. PDUFA date of December 26, 2025 China NDA and Commercial for aficamten EU MAA on file U.S go-to-market strategies anchored in European commercial readiness optimized market access & patient experience activities underway Afi
Slide 62: Expected 2025 Milestones
Expected 2025 Milestones Aficamten Omecamtiv Mecarbil Continue advancing go-to-market strategies & prepare Continue patient enrollment in COMET-HF through to launch aficamten in the U.S. in 2H 2025 2025 with objective to complete enrollment in 2026 Report topline results from MAPLE-HCM in May 2025 C
Slide 63: Thank you
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