Cytokinetics, Incorporated · NASDAQ:CYTK
Cytokinetics — Investor Presentation (July 2021)
16 slides from Cytokinetics's investor presentation, July 2021
Slide 1: Sarcomere Directed Therapies: Empowering Muscle, Empowering Lives
Sarcomere Directed Therapies EMPOWERING MUSCLE EMPOWERING LIVES John, diagnosed with heart failure Jillian, diagnosed with HCM Chuck, diagnosed with ALS
Slide 2: Sarcomere Directed Drug Development
Sarcomere Directed Drug Development Cardiac muscle The sarcomere is a molecular structure found in skeletal and cardiac muscle that enables cardiac myocytes to contract and generate force Troponin Activate Inhibit Inhibit Omecamtiv CK-274 CK-271 Activate Mecarbil (Cardiac) (Cardiac) CK-136 Myosin (C
Slide 3: Symptomatic HCM: Orphan Indication
Symptomatic HCM: Orphan Indication US HCM Prevalence 1,085 High end est. (CVrg 2020) 1000 425 800 Diagnosis gap Low end est. (560-805K patients) 600 (Maron 2013) 400 High end (SHA 280 660 diagnosed HCM 200 pts. 2016-2021) 180 Low end (Maron 2013) 100 0 Diagnosed Patients Patient Prevalence Source: #
Slide 4: CK-274: Next-In-Class Cardiac Myosin Inhibitor
CK-274: Next-In-Class Cardiac Myosin Inhibitor Potential treatment for patients with HCM • Selective allosteric inhibitor of cardiac myosin discovered by company scientists independent of collaborations • Potential in vivo pharmacodynamic advantages related to distinctive binding site • Optimized fo
Slide 5: 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 CK-3773274 + 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
Slide 6: Patient Enrollment and Dosing
Patient Enrollment and Dosing 41 Total Enrolled Patients Final Dose Achieved (N) Cohort 1 Cohort 2 Placebo 5mg 10mg 15mg 10mg 20mg 30mg 13 4 5 5 9 4 1 6
Slide 7: Baseline Echocardiographic Data
Baseline Echocardiographic Data Baseline (Day 1 Pre-dose) Characteristic, mean Placebo CK-274 C1 + C2 Combined (N = 13) Cohort 1 (N = 14) Cohort 2 (N = 14) LVEF (%) 74.5 73.2 75.4 LVOT-G, Rest (mmHg) 52.1 53.8 58.2 LVOT-G, Valsalva (mmHg) 84.6 74.4 82.3 7
Slide 8: High Response Rates on Treatment with CK-274
High Response Rates on Treatment with CK-274 1/13 11/14 13/14 Responder # / Total # Responder Definition: Resting LVOT-G <30 mmHg and post-Valsalva LVOT-G <50 mmHg at Week 10 8
Slide 9: Resting Left Ventricular Outflow Tract Gradient
Resting Left Ventricular Outflow Tract Gradient Resting LVOT-G 80 Combined Placebo CK-274: Cohort 1 60 CK-274: Cohort 2 mmHg 40 20 mean ± SEM 0 0 2 4 6 8 10 Weeks Mean ± SEM Resting LVOT-G (mmHg) Baseline Week 2 Week 4 Week 6 Week 10 Placebo (n=13) 52.1 45.0 47.1 49.0 44.0 Cohort 1 (n = 14) 53.8 24.
Slide 10: Post-Valsalva Left Ventricular Outflow Tract Gradient
Post-Valsalva Left Ventricular Outflow Tract Gradient Valsalva LVOT-G 100 Combined Placebo 80 CK-274: Cohort 1 CK-274: Cohort 2 60 mmHg 40 20 mean ± SEM 0 0 2 4 6 8 10 Weeks Mean ± SEM Valsalva LVOT-G (mmHg) Baseline Week 2 Week 4 Week 6 Week 10 Placebo (n=13) 84.6 71.3 71.3 73.4 76 Cohort 1 (n = 14
Slide 11: Safety Data
Safety Data • Incidence of adverse events on CK-274 similar to placebo and mild or moderate • There were no treatment related serious adverse events reported by investigators • No patients who received CK-274 in Cohort 1 had an LVEF <50% • In Cohort 2, one patient with LVEF at baseline of 58% was up
Slide 12: Open Label Extension Trial
Open Label Extension Trial 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 CK-274 on LVOT-G; assessments of steady-state pharmacokinetics. • Cardiac MRI sub-study to assess ch
Slide 13: Engaging Regulatory Authorities to Inform Phase 3
Engaging Regulatory Authorities to Inform Phase 3 • Type C meeting with FDA to review Phase 3 clinical trial design • End of Phase 2 meeting to review final dose selection rationale for Phase 3 13
Slide 14: CK-274: Clinical Development Plan for HCM
CK-274: Clinical Development Plan for HCM Phase 1 Phase 2 Phase 3 Safety, PK & PD Proof of Concept, Dose Finding Pivotal Studies SAD & MAD oHCM patients oHCM patients Healthy Placebo Controlled Exercise Endpoint (peak VO2) IND Filed Volunteers Echocardiography Endpoints NDA Well tolerated dose Impro
Slide 15: Novel Approach May Address Multiple Unmet Patient Needs
Novel Approach May Address Multiple Unmet Patient Needs No FDA-approved therapies Obstructive HCM oHCM CK-274 (Cardiac myosin Inhibitor) nHCM HFpEF Non-Obstructive HCM Heart Failure with Preserved Ejection Fraction 15
Slide 16: Thank You
Sarcomere Directed Therapies THANK YOU John, diagnosed with heart failure Jillian, diagnosed with HCM Chuck, diagnosed with ALS