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adequacy, fairness, accuracy or completeness of, any information obtained from third-party sources. 2
Our Mission To bring forward new medicines to improve the healthspan of
people with devastating cardiovascular and neuromuscular diseases of impaired muscle function. 3
Now Available FDA-approved for the treatment of adults with symptomatic
oHCM to improve functional capacity and symptoms FDA: U.S. Food & Drug Administration; oHCM: obstructive hypertrophic cardiomyopathy Please see full Prescribing Information, including Boxed WARNING and Medication Guide 4 4
A Commitment to Muscle-Directed Medicines Drug Therapeutic Area Approved
& (Clinical Trial) Research Pre-Clinical Phase 1 Phase 2 Phase 3 Commercialized oHCM* Approved MYQORZO SEQUOIA-HCM in US, China & EU** oHCM (vs. metoprolol) Aficamten MAPLE-HCM Pediatric oHCM Aficamten CEDAR-HCM nHCM Aficamten
ACACIA-HCM HFpEF Ulacamten AMBER-HFpEF Omecamtiv HFrEF Mecarbil COMET-HF Muscular Dystrophy, CK-089 other Research Muscle Biology Directed *MYQORZO is approved for the treatment of adults with symptomatic oHCM to improve functional capacity and
symptoms. Please see full Prescribing Information, including Boxed WARNING and Medication Guide **MYQORZO is only approved in the U.S., China and the EU for the treatment of adults with symptomatic oHCM. Ulacamten, omecamtiv mecarbil and CK-089 are
investigational agents and have not been approved for use by any regulatory agency. Their safety and efficacy has not been established. oHCM: obstructive hypertrophic cardiomyopathy; nHCM: non-obstructive hypertrophic cardiomyopathy; HFpEF: heart
failure with preserved ejection fraction; HFrEF: heart failure with reserved ejection fraction 5
Positioned for Launch & Sustainable Growth COMMERCIAL RESEARCH &
DEVELOPMENT STRONG FINANCIAL POSITION Specialty Muscle-directed platform Cardiology ~$1.1B with multi-program pipeline Franchise cash, cash equivalents & investments (as of 3/31/26) sNDA for aficamten based on MAPLE-HCM under review with FDA,
PDUFA November 14, 2026* Access to further capital: Eligible to draw up to $175M from tranche Potential for aficamten in nHCM based on positive 7 loan provided by Royalty Pharma topline results from ACACIA-HCM Approved for the treatment of
Up to $150M funding of a pivotal trial of adults with symptomatic oHCM to Expansion & breadth: CEDAR-HCM (pediatric ulacamten by Royalty Pharma at its option improve functional capacity and oHCM) enrolling, FOREST-HCM (HCM OLE) ongoing
symptoms in U.S., China & Europe Building specialty cardiology pipeline: Omecamtiv mecarbil: Ph3 confirmatory trial enrolling (COMET-HF) Ulacamten: Ph2 trial enrolling (AMBER-HFpEF) oHCM: obstructive hypertrophic cardiomyopathy; sNDA:
Supplemental New Drug Application; FDA: Food and Drug Administration; PDUFA: Prescription Drug User Fee Act; nHCM: non-obstructive hypertrophic cardiomyopathy; OLE: open label extension *The results of MAPLE-HCM showed that the mean change in pVO
from baseline to Week 24 for aficamten was +1.1 mL/kg/min and -1.2 mL/kg/min for metoprolol (least-squares mean (LSM) difference between groups of 2.3 mL/kg/min (p<0.0001) 2 MYQORZO is only approved in the U.S., China and the EU for the treatment
of adults with symptomatic oHCM. Ulacamten, omecamtiv mecarbil and CK-089 are investigational agents and have not been approved for use by any regulatory agency. Their safety and efficacy has not been established. 6
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 culture with
emphasis on equitable access INGENUITY Extend leadership in muscle biology deploying multiple therapeutic modalities The company's Vision 2030 statements are a statement of goals and there can be no assurance that the goal indicated will be
achieved or, if achieved, will be achieved on this timeline. 7
Potential for Multiple Specialty Cardiology Launches Potential Drug 2024
2025 2026 2027 2028 2029 2030+ Indication Candidate oHCM MYQORZO oHCM Mono (aficamten) (MAPLE-HCM) nHCM (ACACIA-HCM) Omecamtiv HFrEF Mecarbil Ulacamten HFpEF Estimated date of potential future launch of each candidate for the respective indication.
The estimated launch date requires positive clinical data and regulatory approval within projected timelines. MYQORZO (aficamten) is only approved in the U.S. and China for oHCM. Ulacamten and omecamtiv mecarbil are investigational drug candidates
and are not approved as safe or effective for any indication. 8
MYQORZO (aficamten) Clinical Evidence in oHCM Prescribing Information
MYQORZO Inhibits Cardiac Myosin Motor Activity oHCM causes cardiac
MYQORZO binds to and hypercontractility, inhibits cardiac myosin, impaired cardiac relaxation & reducing cardiac contractility increased energy consumption & LVOT obstruction MYQORZO Actin Active myosin head Deactivated myosin head oHCM:
obstructive hypertrophic cardiomyopathy; LVOT: left ventricular outflow tract Please see full Prescribing Information, including Boxed WARNING and Medication Guide 1 0
SEQUOIA-HCM: Pivotal Phase 3 Trial in oHCM Significant improvement in
exercise capacity and symptoms compared to placebo Treatment with MYQORZO for 24 weeks also significantly improved: Gradients 1 -50 mmHg placebo-corrected change in post-Valsalva LVOT-G (p<0.0001) Absolute Change from Baseline to Week 24 Symptoms
MYQORZO 2 Placebo +7.9 points in KCCQ-OSS (p<0.0001) 21 2 +7.8 points in SAQ7-SS (p<0.0001) 1 34% of patients had 1 class improvement in NYHA Class (p<0.0001) 20 Disease Status 1 78 fewer days eligible for septal reduction therapy
(p<0.0001) 19 Biomarkers 1 80% reduction in NT-proBNP (p<0.001) p pV VO O = = 0 0 .0 .0 m m l/l k /g k /g m /m inin 2 2 3 43% reduction in hs-cTnI (p<0.001) 18 Structure, Function & Remodeling Improvements in maximal wall
thickness, septal wall thickness, inferolateral Baseline Week 24 wall thickness, LV mass index, LV end systolic volume index, left atrial pVO peak oxygen uptake; LS: least squares; SE: standard error; LVOT-G: left ventricular outflow tract gradient;
KCCQ-OSS: Kansas City Cardiomyopathy 2: 4 Questionnaire Overall Summary Score; SAQ7-SS: Seattle Angina Questionnaire Summary Score, NYHA: New York Heart Association; NT-pro-BNP: N-terminal volume index, lateral e' velocity, lateral E/e'
(all p<0.01) prohormone of brain natriuretic peptide: hs-cTnI: high-sensitivity cardiac troponin I; LV: left ventricular Sources: 1. Maron M. Aficamten for Symptomatic Obstructive Hypertrophic Cardiomyopathy. N Engl J Med 2024 May
30;390(20):1849-1861 2. Sherrod C, et al. Effect of Aficamten on Health Status Outcomes in Obstructive Hypertrophic Cardiomyopathy: Results from SEQUOIA-HCM. JACC. 2024. 3. Coats CJ, et al. Cardiac Biomarkers and Effects of Aficamten in Obstructive
Hypertrophic Cardiomyopathy: The SEQUOIA-HCM Trial. Eur Heart J. 2024 4. Hegde S, et al. Impact of Aficamten on Echocardiographic Cardiac Structure and Function in Symptomatic Obstructive Hypertrophic Cardiomyopathy. JACC. 2024. Please see full
Prescribing Information, including Boxed WARNING and Medication Guide 1 1 pVO (mL/kg/min) 2
SEQUOIA-HCM: Safety Data MYQORZO Placebo Event, n (%) (n=142) (n=140)
Overall AEs 105 (73.9) 99 (70.7) Headache 11 (7.7) 10 (7.1) Hypertension 11 (7.7) 3 (2.1) Palpitations 10 (7.0) 4 (2.9) AEs with 5% incidence Upper respiratory infection 9 (6.3) 12 (8.6) COVID-19 8 (5.6) 9 (6.4) Dyspnea 8 (5.6) 8 (5.7) There
were no serious adverse cardiovascular SAEs 8 (5.6) 13 (9.3) events associated with Cardiac AEs 24 (16.9) 21 (15.0) MYQORZO treatment in Discontinuations 5 (3.5) 4 (2.9) SEQUOIA-HCM New-onset atrial fibrillation (AF) 1 (0.7) 1 (0.7) Appropriate ICD
shock 0 1 (0.7) a LVEF <50% by core laboratory 5 (3.5) 1 (0.7) Dose reduction based on 7 (4.9) 1 (0.7) site-read LVEF <50% a 1 placebo- and 1 MYQORZO-treated patient overlap with dose reduction based on site-read LVEF <50%. AE, adverse
event; SAE, serious adverse event; ICD: implantable cardioverter defibrillator; LVEF: left ventricular ejection fraction Coats CJ. Dosing and Safety Profile of Aficamten in Symptomatic Obstructive Hypertrophic Cardiomyopathy. J Am Heart Assoc 2024 1
MAPLE-HCM: Phase 3 Monotherapy Trial in oHCM Aficamten superior to
standard-of-care beta blocker metoprolol Relative to metoprolol, aficamten also improved: Gradients Mean Change from Baseline to Week 24 in pVO 2 1 LSM difference of -30 mmHg in resting LVOT-G (p<0.0001) 1 LSM difference of -35 mmHg in
post-Valsalva LVOT-G (p<0.0001) 22 Symptoms 21 1 LSM difference of +6.9 points in KCCQ-CSS (p<0.0001) 1 51% of patients improved 1 NYHA FC (vs. 26% on metoprolol) (p<0.001) 20 Biomarkers 19 2 -81% in NT-proBNP (-73% for aficamten vs.
+42% for metoprolol) (p<0.001) 2 -28% in hs-cTnI (-43% for aficamten vs. -17% for metoprolol) (p<0.001) 18 Aficamten Metoprolol Structure, Function & Remodeling 17 2 1 LSM difference of -7.0 mL/m in left atrial volume index (p<0.0001)
Baseline Week 24 LSM difference (SE) vs. metoprolol 2.3 (0.39) mL/kg/min, P<0.0001 CI: Confidence interval; pVO peak oxygen uptake; LSM: least squares mean; KCCQ-CSS: Kansas City Cardiomyopathy Questionnaire Clinical Summary Score; NYHA FC: New
York Heart Association Functional Class; NT-pro-BNP: N-terminal prohormone of brain natriuretic 2: peptide: hs-cTnI: high-sensitivity cardiac troponin I 1. Garcia-Pavia, P, et al. Aficamten or Metoprolol Monotherapy for Obstructive Hypertrophic
Cardiomyopathy. N Engl J Med. 2025 2. Lakdawala, NK et al. The Effect of Aficamten vs. Metoprolol on Cardiac Biomarkers in Obstructive Hypertrophic Cardiomyopathy. AHA 2025. MYQORZO (aficamten) is only approved in the U.S., China and the EU for the
treatment of adults with symptomatic oHCM. 1 3 Mean (95% CI) pVO (mL/kg/min) 2
MAPLE-HCM: Safety Aficamten (n=88) Metoprolol (n=87) Patients with any
SAE 7 (8.0) 6 (6.9) Patients with any AE that led to early treatment withdrawal of aficamten or 1 (1.1) 3 (3.4) a metoprolol Patients with AE that led to temporary interruption of aficamten or metoprolol 1 (1.1) 1 (1.1) b c Patients with dose
reduction due to adverse events 1 (1.1) 4 (4.6) d e Patients with 1 dose down-titration 4 (4.5) 26 (29.9) Mean (SD) change in LVEF at Week 24 vs baseline 5.3% (4.7) 0.50% (3.7) f LVEF <50% by core lab 1 (1.1) 0 Values are n
(%). a In the aficamten group, 1 patient had sudden death after a brief viral illness. In the metoprolol group, AEs leading to early treatment discontinuation are ischemic stroke, hypotension, and fractured humerus due to fall (n=1 each). b In the
aficamten group, 1 patient had a dose reduction due to an AE of dizziness. c In the metoprolol group, 4 patients had dose reduction due to AEs of lightheadedness (n=2), bradycardia (n=1), and fatigue (metoprolol, n=1). d In the aficamten group, 3
patients had 4 down-titration events based on site-read LVEF <50% (n=3) and due to an AE (n=1). e In the metoprolol group, 26 patients had 31 down-titration events based on SBP <90 mmHg (n=5), HR <50 bpm (n=17), and AE (n=4). f No
associated AE with this LVEF <50%. Garcia-Pavia, P. et al. Aficamten vs Metoprolol as Monotherapy for Symptomatic Obstructive Hypertrophic Cardiomyopathy. ESC 2025. MYQORZO (aficamten) is only approved in the U.S., China and the EU for the
treatment of adults with symptomatic oHCM. 1 4
Aficamten nHCM Prescribing Information Medication Guide 1 5
ACACIA-HCM: Pivotal Phase 3 Trial in nHCM Patients with: Trial
enrolled over 516 Aficamten Symptomatic symptomatic nHCM patients (NYHA Class II/III) nHCM, Dual primary endpoint: change KCCQ-CSS Placebo 30 and 85 in KCCQ Clinical Summary Score and peak VO2 from LVEF 60% baseline
to Week 36 NT-proBNP 300 ng/mL 5-20 mg doses; 6-week titration period Secondary endpoints: a b Part 1 Part 2 Change in Ve/VCO2 Study Visits d Titration EOT EOS Left atrial volume index (LAVI) Screen D1 W2 W4
W6 W8 W12 W24 W36 W48 W60 W72 W76 NT-proBNP c Echocardiogram Proportion of patients with 1 CPET class improvement in NYHA from KCCQ baseline to Week 36 NYHA Time to first cardiovascular event Dose Titration Biomarkers
a Part 1: All participants followed until week 36 MYQORZO is only approved in the U.S., China and the EU for the treatment of b Part 2: Participants completing Week 36 continue until either Week 72 (followed by EOS at Week 76) OR the last randomized
participant in Part 1 completes Week adults with symptomatic oHCM. 36. c Site-read focused echocardiogram for titration visit (sole criterion). Aficamten dose range 5-20 mg. d 4-week follow up after last dose 1 6 Screening Review by medical monitor
team Randomization (1:1) N = 420 Week 36 Primary Analysis KCCQ & pVO2 plus Secondary Week 72 End of Treatment (EOT) End of Study (EOS)
ACACIA-HCM: Phase 3 Trial of Aficamten in nHCM Full results to be
presented at upcoming medical meeting Change from Baseline to Week 36 Aficamten vs Placebo No new safety signals identified Primary Endpoints p-value LSM (95% CI) LSM (95% CI) % participants completing planned dosing: Aficamten Placebo Similar
between groups (88.4% on aficamten vs. 90.3% on placebo) KCCQ-CSS 11.4 (9.6 - 13.2) 8.4 (6.6 - 10.2) 3.0 (0.5 - 5.5) 0.021 LVEF <50%: 27 (10%) on aficamten vs. 2 (1%) on placebo; pVO (ml/kg/min) 0.64 (0.32 - 0.95) -0.03 (-0.35
- 0.28) 0.67 (0.22 - 1.1) 0.003 2 2 participants on aficamten had a serious adverse event of HF associated with LVEF <50% Treatment Interruptions Statistically significant (p<0.001) improvements observed in key secondary endpoints:
Treatment interruptions due to LVEF <40% 1. Proportion of participants with improvements in NYHA Functional Class occurred in 3% of patients on aficamten 2. Composite z-score of ventilatory efficiency and pVO 2 3. N-terminal pro-B-type
natriuretic peptide (NT-proBNP) nHCM: non-obstructive hypertrophic cardiomyopathy; pVO : peak oxygen uptake; LSM: least squares mean; CI: confidence interval; KCCQ-CSS: Kansas City Cardiomyopathy Questionnaire Clinical Summary Score; NYHA: New York