Cytokinetics (CYTK) investor relations material

Company strategy and pipeline

• Focused on developing muscle-directed medicines, especially targeting cardiac diseases through myosin modulation.

• Lead candidate Aficamten is a cardiac myosin inhibitor pending FDA review for obstructive hypertrophic cardiomyopathy (oHCM), with a PDUFA date set for December 26 and potential approvals in China and Europe.

• Additional studies include MAPLE (monotherapy in oHCM), ACACIA (non-obstructive HCM, nHCM), and pediatric studies, with ACACIA results expected in the first half of next year.

• Other pipeline assets include Omecamtiv for heart failure with reduced ejection fraction and another myosin inhibitor for preserved ejection fraction, supporting a specialty cardiology franchise.

• Business strategy targets high unmet need, specialty cardiology indications with a concentrated customer segment and aims for high ROI and payer positioning.

Clinical trial results and regulatory outlook

• SEQUOIA phase III study of Aficamten in oHCM delivered promising results, forming the basis for NDA submission.

• MAPLE-HCM phase III study showed significant improvement in peak VO2 compared to standard of care, with positive secondary analyses presented at major cardiology meetings.

• ACACIA phase III trial in nHCM has completed enrollment, with results expected in the first half of 2026.

• Clinical data from REDWOOD Cohort 4 and FOREST studies support the safety, tolerability, and efficacy of Aficamten, including high-dose tolerability and significant improvements in functional class and NT-proBNP.

• Regulatory discussions indicate a differentiated risk mitigation profile for Aficamten, with flexible dosing and monitoring, minimal drug-drug interactions, and potential for less burdensome REMS requirements.

Commercial and financial positioning

• Anticipates a commercial launch of Aficamten shortly after regulatory approval, with expectations to expand the cardiac myosin inhibitor class and achieve preferential market share within the first year.

• Current market for cardiac myosin inhibitors is highly concentrated, with 600-700 physicians accounting for over 80% of prescriptions.

• Financially strong, ending June with ~$1 billion in cash and equivalents, plus access to $425 million from Royalty Pharma and a $650 million refinancing.

• Recent refinancing and capital structure adjustments reinforce readiness for go-to-market activities.

• Commercial differentiation expected through less burdensome dosing and monitoring, unlocking a larger prescriber base and more patients.