Cytokinetics, Incorporated · NASDAQ:CYTK
Cytokinetics — Investor Presentation (January 2022)
39 slides from Cytokinetics's investor presentation, January 2022
Slide 1: Empowering Muscle, Empowering Lives
Sarcomere Directed Therapies EMPOWERING MUSCLE EMPOWERING LIVES Nefertari, diagnosed with heart failure Jillian, diagnosed with HCM Chuck, diagnosed with ALS CYTOKINETICS® and the C-shaped logo are registered trademarks of Cytokinetics in the U.S. and certain other countries Copyright©2022 CYTOKINET
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: Vision
Achieve regulatory approvals for at least two drugs arising from our pipeline Build commercial capabilities to market and sell our medicines reflective of their innovation and value VISION Our vision is to be the leading muscle biology biopharma company that Generate sustainable and growing revenues
Slide 4: Sarcomere Directed Drug Development
Sarcomere Directed Drug Development The sarcomere is a molecular structure found in skeletal and cardiac muscle that enables cardiac myocytes to contract and generate force ACTIVATE INHIBIT INHIBIT ACTIVATE ACTIVATE ACTIVATE Troponin Omecamtiv Reldesemtiv CK-136 CK-601 Aficamten CK-271 Mecarbil (Car
Slide 5: Pipeline of Novel Muscle-Directed Drug Candidates
Pipeline of Novel Muscle-Directed Drug Candidates Research Pre-Clinical Phase 1 Phase 2 Phase 3 CARDIAC MUSCLE Omecamtiv Mecarbil (Heart Failure) Aficamten (oHCM, nHCM, HFpEF) CK-271 CK-136 (Heart Failure, other) SKELETAL MUSCLE Reldesemtiv (ALS)* CK-601 Additional Skeletal Muscle Activators OTHER M
Slide 6: Omecamtiv Mecarbil
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 has not been established. 6
Slide 7: Heart Failure Is a Public Health Epidemic
Heart Failure Is a Public Health Epidemic ~6.5M Americans ≥20 years of age have HF; 1M new HF cases occur annually 1 High cost burden driven by hospitalizations; mean cost for each hospital stay ~$17K2 46% 127% 50% ~900,000 24% 49% Increase in Cost increase of HF patients who will Annual HF Patients
Slide 8: Pivotal Phase 3 Trial Design
Pivotal Phase 3 Trial Design Second largest clinical trial ever conducted in heart failure Overview Enrolled 8,256 patients at ~1,000 sites in 35 countries Primary Endpoint Composite of time to cardiovascular (CV) death or first HF event*, whichever occurs first Secondary Endpoints • Time to CV deat
Slide 9: Positive Primary Composite Endpoint
Positive Primary Composite Endpoint Treatment effect increased in more advanced patients 8% Relative Risk Reduction in Primary Composite Endpoint Treatment Effect Increased Progressively As (Time to First Heart Failure Event or CV Death)1 Baseline LVEF Decreased2 1.2 Treatment Effect (Ratio) 1.0 0.8
Slide 10: Greater Treatment Effect in Worsening HF
Greater Treatment Effect in Worsening HF Primary Outcome in Severe HF: HR = 0.80 (0.71, 0.90) Primary Outcome in Patients with LVEF (Severe HF defined as NYHA III-IV, EF ≤ 30%, HF ≤28%: HR 0.84; 95% CI 0.77, 0.92 hospitalization <6 mos)1,2 1. Felker GM, Omecamtiv Mecarbil in Patients with Severe Hea
Slide 11: Large and Growing Heart Failure Patient Population
Large and Growing Heart Failure Patient Population Prevalence in Adults (18+, USA) 2020 Estimates 2032 Estimates 6.2M1 8.0M3 Heart Failure Including HFpEF 3.1M2 4.0M3 HFrEF Population 2.0M2 2.6M3 HFrEF & EF ≤30% Proposed Cardiac Function Recent Event GDMT Limitations Omecamtiv Mecarbil Target Patien
Slide 12: Small Subset of Cardiologists Manage Majority of Patients
Small Subset of Cardiologists Manage Majority of Patients HFrEF Patient Concentration in Cardiologists Distribution of High-Volume Cardiologists 100% % of Patients 50% 0% 0% 50% 100% % of Cardiologists Allows for more targeted field team approach, focusing on <10,000 HCPs Symphony APLD (1/1/2019 – 1
Slide 13: Engagement Approach Allows Customizing and Broadening
Engagement Approach Allows Customizing and Broadening Customizing engagement by different types of customers Digital allows broader reach ~~ illustrative ~~ ~~ illustrative ~~ Targeted Broadening Targeted Community Reach Beyond HF Specialist Cardiologist Initial Target Field & Inside Digital Reimbur
Slide 14: Medicare Is Major Payer with Select Key Players
Medicare Is Major Payer with Select Key Players Medicare is largest payer; enrollment highly concentrated with nearly 3 of 4 patients in only 5 plans Medicare Part D Enrollment (2019) Estimated Payer Mix Entresto Based Payer Mix on Entresto® (2018) 2018 Data Managed Medicaid 8% Top 5 plans have 75%
Slide 15: Commercial Supply Chain Operating Model
Commercial Supply Chain Operating Model Planning, Analytics & Master Data 2021-2022 Manufacturing, Finance Science & Quality Technology 2021-2022 External Commercial Manufacturing Supply 2021-2022 Chain Logistics 2021-2022 Commercial R&D Sales & Marketing Product Strategy 2023+ Not for Promotional U
Slide 16: Second Phase 3 Clinical Trial Underway
Second Phase 3 Clinical Trial Underway Investigating effect of omecamtiv mecarbil on exercise tolerance Results expected in early 2022 Primary endpoint: Exercise tolerance - Change in pVO2 by CPET from baseline to Week 20 Enrolling patients with LVEF ≤35 percent, NYHA class II or III, and reduced ex
Slide 17: Aficamten
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 has not been established. 17
Slide 18: In US, Large HCM Population With Many Undiagnosed
In US, Large HCM Population With Many Undiagnosed HCM Prevalence Potential Undiagnosed Patients Currently ~400-800K ~280K diagnosed, 700 – ~190K oHCM 1,100K symptomatic patients Estimated ~400-800K nHCM* 280K Diagnosed Patients un-diagnosed patients oHCM* ~280K Mava success 190K Now Future nHCM: non
Slide 19: Aficamten: Aspirational Target Profile
Aficamten: Aspirational Target Profile Potential next-in-class cardiac myosin inhibitor Functional Improvement: Improved exercise capacity Efficacy Symptom Improvement: One or two class improvement in NYHA class Quality of Life: KCCQ improvement Minimal drug-drug interactions Safety and Maintain LVE
Slide 20: Phase 2 Clinical Trial Design
Phase 2 Clinical Trial Design Two sequential dose-finding cohorts (with third cohort assessing patients on disopyramide) Randomization Patients with Aficamten + SoC End of Study Screening symptomatic oHCM, 2 and resting or provoked LVOT 1 gradient ≥ 50 mmHg Placebo + SoC Study Visits Screen W-1 D1 W
Slide 21: Response Rates on Treatment with Aficamten
Response Rates on Treatment with Aficamten 1/13 11/14 13/14 100 Responder # / Total # 7.7% 78.6% 92.9% Not Responder • Consistent, clinically Responder 80 meaningful reductions in LVOT Responder Definition: Resting LVOT-G <30 gradients within two weeks mmHg and post- 60 Valsalva LVOT-G <50 • No trea
Slide 22: REDWOOD-HCM: Efficacy
REDWOOD-HCM: Efficacy Reductions in LVOT gradients Resting LVOT-G Valsalva LVOT-G 80 100 80 Combined Placebo 60 Aficamten: Cohort 1 60 Aficamten: Cohort 2 mmHg mmHg 40 LVOT-G ≥50 mmHg 40 * ** ** ** * 20 ** ** *** *** * P < 0.05 *** ** 20 ** ** ** ** P < 0.001 mean ± SEM *** mean ± SEM *** P < 0.0001
Slide 23: Change from Baseline in NT-proBNP & NYHA Class
Change from Baseline in NT-proBNP & NYHA Class Change from Baseline NT-proBNPto Week 10 Improvement in Heart Failure Symptoms (NYHA Class) Week 10 Responder Definition: Improvement in NYHA Class ≥1 0 100 31% -200 80 (n=4) 43% (n=6) 64% (n=9) NTproBNP (pg/mL) -400 60 % Total -600 40 69% (n=9) 57% (n=
Slide 24: REDWOOD-HCM: Cohort 3
REDWOOD-HCM: Cohort 3 Enrolling patients on background therapy of disopyramide Results expected in Q1 2022 Patients with End of Study Screening symptomatic oHCM, and resting or Aficamten + SoC AND disopyramide provoked LVOT gradient ≥ 50 mmHg Study Visits Screen W-1* D1 W2 W4 W6** W8* W9* W10 W12 W1
Slide 25: REDWOOD-HCM: Open Label Extension
REDWOOD-HCM: Open Label Extension REDWOOD-HCM OLE open for eligible patients who completed REDWOOD-HCM • Primary endpoint: incidence of AEs & LVEF <50 • Secondary endpoints: measures of long-term effects of aficamten on LVOT-G; assessments of steady-state pharmacokinetics • Cardiac MRI sub-study to
Slide 26: SEQUOIA-HCM: Phase 3 Trial
SEQUOIA-HCM: Phase 3 Trial Start-up activities underway Primary endpoint: Change in pVO2 by CPET from baseline to Week 24 Randomization Patients with Aficamten + SoC End of Study oHCM Screening treated with 1 Secondary objectives include measuring SOC with post-Valsalva change in KCCQ & improvement
Slide 27: Franchise Strategy
Franchise Strategy 27
Slide 28: Launch Guiding Principles Strengthen Franchise Build
Launch Guiding Principles Strengthen Franchise Build Patient and Creating broad value for cardiac patients and build long-term, deep relationships customer centric with cardiologists with multiple CV medicines Leverage Go-to-Market synergies between multiple CV medicines, enabling Cost-efficient eff
Slide 29: Limited Incremental Cost For Future U.S. CV Launches
Limited Incremental Cost For Future U.S. CV Launches Building Today … … To Lead Tomorrow To optimize value capture for potential To support future launches and establish launch of omecamtiv mecarbil Cytokinetics as a CV leader • Building deep, long-term relationships • Significant overlap between HF
Slide 30: Reldesemtiv
Reldesemtiv Reldesemtiv 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 has not been established. 30
Slide 31: Phase 2 Clinical Trial in ALS
Phase 2 Clinical Trial in ALS Results presented at American Academy of Neurology 2019 Annual Meeting Double-Blind, Placebo-Controlled: 12 weeks Placebo (n = 110) Parallel group, dose ranging study enrolled 458 patients with ALS in the US, Canada, Australia and 150 mg BID (n = 110) Europe evaluating
Slide 32: Primary Endpoint: SVC
Primary Endpoint: SVC Change from baseline in percent predicted SVC at week 12 -6.46 -4.97 -4.62 -4.58 0 Percent Predicted SVC Change From -1 Baseline at Week 12 (95% CI) -2 -3 Primary Analysis* -4 P = 0.11 for weighted -5 dose-response relationship -6 -7 -8 * -9 Placebo 150 mg BID 300 mg BID 450 mg
Slide 33: Change From Baseline: All Active vs Placebo
Change From Baseline: All Active vs Placebo* Results support progression to potential Phase 3 clinical trial SVC Change From Baseline ALSFRS-R Change From Baseline (All Active vs Placebo) (All Active vs Placebo) 0.0 0.0 LS Mean (±SE) of Change in Functional Rating *P = 0.10 *P = 0.01 Scale – Revised
Slide 34: Phase 3 Clinical Trial Design
Phase 3 Clinical Trial Design Plan to enroll 555 patients; interim analysis for futility expected in 2022 Enrolling patients with ALS in the US, Reldesemtiv Canada, Australia (N= 555) 300 mg BID End of Study and Europe Screening Randomization evaluating change 2:1 from baseline Stratification: ALSFR
Slide 35: Financial Outlook
Financial Outlook 35
Slide 36: Monetizing Our Pipeline to Bolster Balance Sheet
Monetizing Our Pipeline to Bolster Balance Sheet Symmetry of deals for omecamtiv mecarbil and aficamten affords symmetry for development and potential launches and supports franchise strategy Sold 4.5% royalty Sold 3.5-4.5% royalty Transactions on worldwide net sales of on worldwide net sales of wit
Slide 37: More Than 2 Years Cash Runway
More Than 2 Years Cash Runway* Recent deals support plans for commercial launch & expansion of late-stage pipeline $669M At Q3 2021 + $70M upfront & near-term + $330M in potential milestone capital from Ji Xing & RTW payments + royalties from Ji Xing + $150M at closing and near-term + $300M in poten
Slide 38: Expected Milestones in 2022
Expected Milestones in 2022 Financial guidance and elaboration on milestones during Q4 2021 earnings call in February Update on plans for expansion Results from METEORIC-HF Expect results from Cohort 3 of of development program for expected in early 2022 REDWOOD-HCM in Q1 2022 aficamten in Q1 2022 S
Slide 39: Thank You
Sarcomere Directed Therapies THANK YOU Nefertari, diagnosed with heart failure Jillian, diagnosed with HCM Chuck, diagnosed with ALS Copyright©2022 CYTOKINETICS, All Rights Reserved Actual patients who consented to use of their name, image, and condition.