U.S. Food and Drug Administration Approves Expanded Indication for Bristol Myers Squibb’s CAMZYOS® (mavacamten) for the Treatment of Symptomatic Obstructive Hypertrophic Cardiomyopathy (oHCM) in Adults and Pediatric Patients*

The expanded indication covers pediatric patients weighing 30 kg or more, while treatment remains subject to heart-function monitoring.

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CAMZYOS is the only FDA-approved therapy for the treatment of oHCM in a pediatric population

Approval based on positive results of Phase 3 SCOUT-HCM trial, building on the extensive clinical evidence for CAMZYOS in adults with oHCM

PRINCETON, N.J.--(BUSINESS WIRE)-- Bristol Myers Squibb (NYSE: BMY) today announced that the U.S. Food and Drug Administration (FDA) approved CAMZYOS^® (mavacamten) for the treatment of symptomatic obstructive hypertrophic cardiomyopathy (oHCM) to improve functional capacity and symptoms in adults and pediatric patients weighing 30 kg (66 lbs) or more. CAMZYOS now has the broadest indication of any cardiac myosin inhibitor (CMI). This expanded indication offers a new treatment option for patients with significant unmet need.¹

“Today’s approval of CAMZYOS for pediatric patients with symptomatic obstructive hypertrophic cardiomyopathy marks an important milestone for young patients and families who are facing a serious cardiovascular disease with significant unmet medical need,” said Al Reba, senior vice president, Immunology & Cardiovascular Commercialization, Bristol Myers Squibb. “For young patients living with this disease, the burden on daily life can be substantial during a critical stage of development, and we are proud to help bring an innovative new treatment option to this community. This approval also strengthens the growing body of clinical evidence supporting CAMZYOS, which is the most extensively studied cardiac myosin inhibitor with up to five years of follow up in adults, and its role in the treatment of oHCM.”

This FDA approval is based on positive results from the Phase 3 SCOUT-HCM trial, which evaluated the efficacy and safety of CAMZYOS (n=23) versus placebo (n=21) in patients aged 12 to <18 years with symptomatic NYHA (New York Heart Association) Class II-III obstructive hypertrophic cardiomyopathy. Enrolled patients had LVEF ≥60%, Valsalva LVOT peak gradient ≥30 mmHg and a maximal gradient ≥50 mmHg at rest or with provocation, and background oHCM therapy with a beta blocker, calcium channel blocker, disopyramide, or combinations was permitted.^1,2

CAMZYOS met the trial’s primary endpoint, demonstrating a statistically significant reduction in Valsalva left ventricular outflow tract (LVOT) gradient at Week 28 compared with placebo.^1,2

In SCOUT-HCM, no patients experienced left ventricular ejection fraction (LVEF) below 50% and no adverse events led to treatment discontinuation. No new adverse reactions were identified beyond the safety profile observed in adults. Serious adverse events occurred in 2 patients each in the CAMZYOS (9%) and placebo (10%) groups.^1,2

* Weighing 30 kg (66 lbs) or more

SCOUT-HCM Key Data^1,2†

	CAMZYOS (n=23)   Mean Change (SD)	Placebo (n=21)   Mean Change (SD)	Least-Squares Mean Difference from Placebo (95% CI)
Primary endpoint
Change from baseline to week 28 in provoked Valsalva LVOT gradient — mm Hg	−49.4 (42.6)	−1.8 (37.8)	−48.0 (−67.7 to −28.3) P<0.0001
Select secondary endpoints‡
Change from baseline to week 28 in resting LVOT gradient — mm Hg	−41 (35)	5.3 (23)	−47.0 (−62.7 to −31.4)
Change from baseline to week 28 in postexercise LVOT gradient — mm Hg	−31.2, 95% CI (−46.3 to −16.1) (n=16, Least-Squares Mean Change)	10.5, 95% CI (−5.5 to 26.6) (n=16, Least-Squares Mean Change)	−41.7 (−59.7 to −23.7)
Change from baseline to week 28 in maximal LV wall thickness — mm	−2.5, 95% CI (−3.7 to −1.3) (n=23, Least-Squares Mean Change)	−0.7, 95% CI (−2.1 to 0.7) (n=20, Least-Squares Mean Change)	−1.8 (−3.4 to −0.2)
† Efficacy analyses were performed in the randomized analysis population, which included all the patients who underwent randomization. LV denotes left ventricular.
‡ Does not include all secondary endpoints measured. Secondary endpoints are summarized descriptively with estimates of between-group differences and 95% confidence intervals. The widths of the confidence intervals were not adjusted for multiplicity; therefore, confidence intervals should not be interpreted as formal hypothesis testing.

“The FDA approval of CAMZYOS for pediatric patients with symptomatic obstructive hypertrophic cardiomyopathy represents a landmark moment for pediatric cardiology. For the first time, children with this serious condition have a therapy that is FDA-approved to reduce left ventricular outflow tract obstruction,” said Joseph Rossano, MD, Principal Investigator of SCOUT-HCM and Chief of the Division of Cardiology at Children’s Hospital of Philadelphia. “This historic milestone was made possible by the outstanding efforts of the international SCOUT-HCM investigators and participating families whose commitment has transformed clinical research into a therapy with potential to meaningfully improve the lives of pediatric patients with symptomatic obstructive hypertrophic cardiomyopathy.”

“I cannot help but think back to my 12-year-old self receiving my diagnosis and being told there were no treatment options approved specifically for my disease,” said Lisa Salberg, CEO and founder of the Hypertrophic Cardiomyopathy Association. “This is truly a milestone moment for patients and families. Having a targeted therapy for cardiac myosin is a significant breakthrough, and it's a welcome change to bring this approval to a younger population. Thank you to all of the researchers, scientists, and the entire HCM ecosystem that has helped bring this new therapy to more patients.”

The full U.S. Prescribing Information for CAMZYOS includes a Boxed WARNING for the risk of heart failure. CAMZYOS reduces left ventricular ejection fraction (LVEF) and can cause heart failure due to systolic dysfunction. Echocardiogram assessments of LVEF are required prior to and during treatment with CAMZYOS. Initiation of CAMZYOS in patients with LVEF <55% is not recommended. Interrupt CAMZYOS if LVEF is <50% at any visit or if the patient experiences heart failure symptoms or worsening clinical status. Concomitant use of CAMZYOS with certain cytochrome P450 inhibitors or discontinuation of certain cytochrome P450 inducers may increase the risk of heart failure due to systolic dysfunction; therefore, the use of CAMZYOS is contraindicated with strong CYP2C19 inhibitors and moderate to strong CYP2C19 inducers or moderate to strong CYP3A4 inducers. Because of the risk of heart failure due to systolic dysfunction, CAMZYOS is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called the CAMZYOS REMS Program. Please see additional Important Safety Information including Boxed WARNING below.

CAMZYOS was first approved by the FDA in 2022 for the treatment of adults with symptomatic NYHA class II-III oHCM to improve functional capacity and symptoms and has since been approved by multiple global regulatory authorities for that indication. CAMZYOS has been prescribed by more than 5,000 healthcare providers (HCPs) to over 25,000 patients in the U.S. alone.³ Beyond this U.S. approval, the pediatric data is being discussed with multiple global regulatory authorities for potential pediatric indications.

Today’s approval of CAMZYOS for adults and pediatric patients weighing 30 kg (66 lbs) or more reinforces Bristol Myers Squibb’s commitment to delivering innovative therapies that can transform outcomes for patients living with serious cardiovascular diseases.

About Obstructive Hypertrophic Cardiomyopathy (oHCM) in Pediatric Patients
Hypertrophic cardiomyopathy (HCM) is the most common inherited cardiac disorder that may result from known or suspected genetic defects in sarcomeric proteins of the cardiac myocyte or due to unknown reasons (idiopathic).^2,4 Pediatric patients with obstructive HCM suffer substantial morbidity such as risk for ventricular arrhythmias, heart failure and atrial fibrillation.⁵ Management of obstructive HCM in pediatric patients has historically included therapies such as beta blockers, calcium channel blockers, disopyramide and, in some cases, invasive septal reduction procedures. Given the burden of this disease, there has been a longstanding need for additional treatment options.^2,4

About the Phase 3 SCOUT-HCM Trial^1,2,5
SCOUT-HCM (NCT06253221) is a Phase 3 randomized, double-blind, placebo-controlled, international trial that enrolled 44 adolescent patients (12 years to <18 years old) with symptomatic oHCM. The trial includes three treatment periods totaling up to 200 weeks: a 28-week placebo-controlled period, followed by a 28-week active-treatment period (when patients randomized to placebo cross over to CAMZYOS), and an open-label long-term extension period for up to 144 weeks. At baseline, 83% and 86% of patients were receiving beta blockers, 13% and 10% were receiving calcium channel blockers, and 17% and 24% were receiving disopyramide, in the CAMZYOS and placebo groups, respectively. Mean LVEF was 69.0% in the CAMZYOS group and 67.4% in the placebo group; mean Valsalva LVOT gradient was 78 mmHg in the CAMZYOS group and 81 mmHg in the placebo group. Patients were randomized in a 1:1 ratio and administered either CAMZYOS, starting dose of 2.5 mg CAMZYOS for body weight 35 to <45 kg, 5 mg CAMZYOS for body weight ≥45 kg, or placebo, once daily for 28 weeks. Dose adjustment was based on clinical echocardiogram parameters.

The primary endpoint is change from baseline to Week 28 in Valsalva LVOT gradient (mmHg). Secondary endpoints include change from baseline to Week 28 in efficacy parameters of resting and postexercise LVOT gradients, peak oxygen consumption (proportion of patients achieving an increase from baseline in pVO₂), symptoms (proportion of patients with at least 1 NYHA Class improvement) and HCM Symptom Questionnaire Shortness of Breath domain (HCMSQ SoB), plus safety and pharmacokinetic parameters.

Joseph Rossano, MD, is a paid consultant to Bristol Myers Squibb and served as the site principal investigator for the SCOUT-HCM study at Children’s Hospital of Philadelphia.

About CAMZYOS^® (mavacamten)
CAMZYOS^® (mavacamten) is the most extensively studied cardiac myosin inhibitor (CMI), approved by regulatory bodies in more than 60 countries and regions across five continents worldwide.^1,6 CAMZYOS is supported by the largest body of worldwide evidence in the CMI treatment class, with approvals resulting from several phase III clinical trials (EXPLORER-HCM, VALOR-HCM, SCOUT-HCM, as well as HORIZON-HCM in Japan and EXPLORER-CN in China), and up to five years of follow up in multiple pivotal trial long-term extensions (EXPLORER-LTE, VALOR-HCM LTE), providing additional data on the consistent and sustained results for CAMZYOS to improve symptoms and impact cardiac structure and function.^7,8,9

In the U.S., CAMZYOS is indicated for the treatment of symptomatic oHCM to improve functional capacity and symptoms in adults and pediatric patients weighing 30 kg (66 lbs) or more. In the European Union, CAMZYOS is indicated for the treatment of symptomatic (NYHA, class II-III) oHCM in adult patients.

A selective, reversible, allosteric inhibitor of cardiac myosin, CAMZYOS targets hypercontractility, the source of oHCM. Reduction in cardiac contractility with CAMZYOS treatment leads to reduced LVOT obstruction, improved energy consumption, and lower cardiac filling pressures in oHCM patients. These effects have translated to demonstrated symptom improvements in clinical studies for adults with symptomatic oHCM, enabling them to be more active in their daily lives. CAMZYOS can be used with or without background therapies, including for newly diagnosed patients.^1,9,10

CAMZYOS has been prescribed by more than 5,000 healthcare providers (HCPs) to over 25,000 patients in the U.S. alone.³

IMPORTANT SAFETY INFORMATION

WARNING: RISK OF HEART FAILURE

CAMZYOS reduces left ventricular ejection fraction (LVEF) and can cause heart failure due to systolic dysfunction.

Echocardiogram assessments of LVEF are required prior to and during treatment with CAMZYOS. Initiation of CAMZYOS in patients with LVEF <55% is not recommended. Interrupt CAMZYOS if LVEF is <50% at any visit or if the patient experiences heart failure symptoms or worsening clinical status.

Concomitant use of CAMZYOS with certain cytochrome P450 inhibitors or discontinuation of certain cytochrome P450 inducers may increase the risk of heart failure due to systolic dysfunction; therefore, the use of CAMZYOS is contraindicated with the following:

• Strong CYP2C19 inhibitors
• Moderate to strong CYP2C19 inducers or moderate to strong CYP3A4 inducers

Because of the risk of heart failure due to systolic dysfunction, CAMZYOS is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called the CAMZYOS REMS Program.

CONTRAINDICATIONS

CAMZYOS is contraindicated with concomitant use of:

• Strong CYP2C19 inhibitors
• Moderate to strong CYP2C19 inducers or moderate to strong CYP3A4 inducers

WARNINGS AND PRECAUTIONS

Heart Failure
CAMZYOS reduces systolic contraction and can cause heart failure or significantly reduce ventricular function. Patients who experience a serious intercurrent illness (eg, serious infection) or arrhythmia (eg, atrial fibrillation or other uncontrolled tachyarrhythmia) are at greater risk of developing systolic dysfunction and heart failure.

Assess the patient’s clinical status and LVEF prior to and regularly during treatment and adjust the CAMZYOS dose accordingly. New or worsening arrhythmia, dyspnea, chest pain, fatigue, palpitations, leg edema, or elevations in N-terminal pro-B-type natriuretic peptide (NT-proBNP) may be signs and symptoms of heart failure and should also prompt an evaluation of cardiac function.

Asymptomatic LVEF reduction, intercurrent illnesses, and arrhythmias require additional dosing considerations.

Initiation of CAMZYOS in patients with LVEF <55% is not recommended. Avoid concomitant use of CAMZYOS in patients on disopyramide, ranolazine, verapamil with a beta blocker, or diltiazem with a beta blocker as these medications and combinations increase the risk of left ventricular systolic dysfunction and heart failure symptoms and clinical experience is limited.

CYP450 Drug Interactions Leading to Heart Failure or Loss of Effectiveness
CAMZYOS is primarily metabolized by CYP2C19 and CYP3A4 enzymes. Concomitant use of CAMZYOS and drugs that interact with these enzymes may lead to life-threatening drug interactions such as heart failure or loss of effectiveness.

Advise patients of the potential for drug interactions, including with over-the-counter medications (such as omeprazole, esomeprazole, or cimetidine). Advise patients to inform their healthcare provider of all concomitant products prior to and during CAMZYOS treatment.

CAMZYOS Risk Evaluation and Mitigation Strategy (REMS) Program
CAMZYOS is only available through a restricted program called the CAMZYOS REMS Program because of the risk of heart failure due to systolic dysfunction. Notable requirements of the CAMZYOS REMS Program include the following:

• Prescribers must be certified by enrolling in the REMS Program
• Patients must enroll in the REMS Program and comply with ongoing monitoring requirements
• Pharmacies must be certified by enrolling in the REMS Program and must only dispense to patients who are authorized to receive CAMZYOS
• Wholesalers and distributors must only distribute to certified pharmacies

Further information is available at www.CAMZYOSREMS.com or by telephone at 1-833-628-7367.

Embryo-Fetal Toxicity
CAMZYOS may cause fetal toxicity when administered to a pregnant female, based on animal studies. Confirm absence of pregnancy in females of reproductive potential prior to treatment and advise patients to use effective contraception during treatment with CAMZYOS and for 4 months after the last dose. Combined hormonal contraceptives (CHCs) containing a combination of ethinyl estradiol and norethindrone may be used with CAMZYOS. However, CAMZYOS may reduce the effectiveness of certain other CHCs. If these CHCs are used, advise patients to add nonhormonal contraception (such as condoms) during concomitant use and for 4 months after the last dose of CAMZYOS.

ADVERSE REACTIONS

In the EXPLORER-HCM trial, adverse reactions occurring in >5% of patients and more commonly in the CAMZYOS group than in the placebo group were dizziness (27% vs 18%) and syncope (6% vs 2%). There were no new adverse reactions identified in VALOR-HCM. The safety of CAMZYOS in pediatric patients (≥35 kg) was evaluated in SCOUT-HCM. There were no new adverse reactions compared to those identified in adult studies.

Effects on Systolic Function
In the EXPLORER-HCM trial, mean (SD) resting LVEF was 74% (6) at baseline in both treatment groups. Mean (SD) absolute change from baseline in LVEF was -4% (8) in the CAMZYOS group and 0% (7) in the placebo group over the 30-week treatment period. At Week 38, following an 8-week interruption of trial drug, mean LVEF was similar to baseline for both treatment groups. In the EXPLORER-HCM trial, 7 (6%) patients in the CAMZYOS group and 2 (2%) patients in the placebo group experienced reversible reductions in LVEF <50% (median 48%: range 35-49%) while on treatment. In all 7 patients treated with CAMZYOS, LVEF recovered following interruption of CAMZYOS.

DRUG INTERACTIONS

Potential for Other Drugs to Affect Plasma Concentrations of CAMZYOS
CAMZYOS is primarily metabolized by CYP2C19 and to a lesser extent by CYP3A4 and CYP2C9. Inducers and inhibitors of CYP2C19 and moderate to strong inhibitors or inducers of CYP3A4 may affect the exposures of CAMZYOS.

Impact of Other Drugs on CAMZYOS:

• Strong CYP2C19 Inhibitors: Concomitant use increases CAMZYOS exposure, which may increase the risk of heart failure due to systolic dysfunction. Concomitant use is contraindicated
• Moderate to Strong CYP2C19 Inducers or Moderate to Strong CYP3A4 Inducers: Concomitant use decreases CAMZYOS exposure, which may reduce CAMZYOS’ efficacy. The risk of heart failure due to systolic dysfunction may increase with discontinuation of these inducers as the levels of induced enzyme normalizes. Concomitant use is contraindicated
• Weak CYP2C19 Inhibitors or Moderate CYP3A4 Inhibitors: Concomitant use with a weak CYP2C19 inhibitor or a moderate CYP3A4 inhibitor increases CAMZYOS exposure, which may increase the risk of adverse drug reactions. Initiate CAMZYOS at the recommended starting dosage in patients who are on stable therapy with a weak CYP2C19 inhibitor or a moderate CYP3A4 inhibitor. Reduce dose of CAMZYOS by one level (ie, 15 mg to 10 mg, 10 mg to 5 mg, 5 mg to 2.5 mg, or 2.5 mg to 1 mg [pediatric dosing]) in patients who are on CAMZYOS treatment and intend to initiate a weak CYP2C19 inhibitor or a moderate CYP3A4 inhibitor. Avoid initiation of concomitant weak CYP2C19 and moderate CYP3A4 inhibitors in patients who are on the lowest recommended dose of CAMZYOS. For short-term use (eg, 1 week), interrupt CAMZYOS for the duration of treatment with a weak inhibitor of CYP2C19 or a moderate inhibitor of CYP3A4. CAMZYOS may be reinitiated at the previous dose immediately on discontinuation of concomitant therapy.
• Moderate CYP2C19 Inhibitors or Strong CYP3A4 Inhibitors: Concomitant use with a moderate CYP2C19 inhibitor or strong CYP3A4 inhibitor increases CAMZYOS exposure, which may increase the risk of adverse drug reactions. Discontinuing use of a moderate CYP2C19 inhibitor or strong CYP3A4 inhibitor after long-term concomitant use may decrease CAMZYOS exposure, which may reduce CAMZYOS’ efficacy. Initiate CAMZYOS at one dose level below the recommended starting dose in patients who are on stable therapy with a moderate CYP2C19 inhibitor or a strong CYP3A4 inhibitor. Reduce dose of CAMZYOS by one level (ie, 15 mg to 10 mg; 10 mg to 5 mg; 5 mg to 2.5 mg; or 2.5 mg to 1 mg [pediatric dosing]) in patients who are on CAMZYOS and intend to initiate a moderate CYP2C19 inhibitor or a strong CYP3A4 inhibitor. Avoid initiation of concomitant moderate CYP2C19 and strong CYP3A4 inhibitors in patients who are on the lowest recommended dose of CAMZYOS. An increase in dose of CAMZYOS may be needed if the moderate inhibitor of CYP2C19 or strong inhibitor of CYP3A4 is discontinued after long-term concomitant use. Monitor for new or worsening symptoms. For short-term use (eg, 1 week), interrupt CAMZYOS for the duration of treatment with a moderate inhibitor of CYP2C19 or a strong inhibitor of CYP3A4. CAMZYOS may be reinitiated at the previous dose immediately on discontinuation of concomitant therapy.

Potential for CAMZYOS to Affect Plasma Concentrations of Other Drugs
CAMZYOS is an inducer of CYP3A4, CYP2C9, and CYP2C19. Concomitant use with CYP3A4, CYP2C9, or CYP2C19 substrates may reduce plasma concentration of these drugs. Closely monitor when CAMZYOS is used with concomitant CYP3A4, CYP2C9, or CYP2C19 substrates unless otherwise recommended in the Prescribing Information.

Certain Combined Hormonal Contraceptives (CHCs): Progestin and ethinyl estradiol are CYP3A4 substrates. Concomitant use of CAMZYOS may decrease exposures of certain progestins, which may lead to contraceptive failure. CHCs containing a combination of ethinyl estradiol and norethindrone may be used with CAMZYOS, but if other CHCs are used, advise patients to add nonhormonal contraception (such as condoms) or use an alternative contraceptive method that is not affected by CYP450 enzyme induction (eg, intrauterine system) during concomitant use and for 4 months after the last dose of CAMZYOS.

Drugs That Reduce Cardiac Contractility
Expect additive negative inotropic effects of CAMZYOS and other drugs that reduce cardiac contractility. Avoid concomitant use of CAMZYOS in patients on disopyramide, ranolazine, verapamil with a beta blocker, or diltiazem with a beta blocker as these medications and combinations increase the risk of left ventricular systolic dysfunction and heart failure symptoms and clinical experience is limited.

If concomitant therapy with a negative inotrope is initiated, or if the dose of a negative inotrope is increased, monitor LVEF closely until stable doses and clinical response have been achieved.

SPECIFIC POPULATIONS

Pregnancy
CAMZYOS may cause fetal harm when administered to a pregnant female. Advise pregnant females about the potential risk to the fetus with maternal exposure to CAMZYOS during pregnancy. There is a pregnancy safety study for CAMZYOS. If CAMZYOS is administered during pregnancy, or if a patient becomes pregnant while receiving CAMZYOS or within 4 months after the last dose of CAMZYOS, healthcare providers should report CAMZYOS exposure by contacting Bristol Myers Squibb at 1-800-721-5072 or www.bms.com.

Lactation
The presence of CAMZYOS in human or animal milk, the drug’s effects on the breastfed infant, or the effects on milk production are unknown. The developmental and health benefits of breastfeeding should be considered along with the mother’s clinical need for CAMZYOS and any potential adverse effects on the breastfed child from CAMZYOS or from the underlying maternal condition.

Females and Males of Reproductive Potential
Confirm absence of pregnancy in females of reproductive potential prior to initiation of CAMZYOS. Advise females of reproductive potential to use effective contraception during treatment with CAMZYOS and for 4 months after the last dose. CHCs containing a combination of ethinyl estradiol and norethindrone may be used with CAMZYOS. However, CAMZYOS may reduce the effectiveness of certain other CHCs. If these CHCs are used, advise patients to add nonhormonal contraception (such as condoms) or use an alternative contraceptive method during concomitant use and for 4 months after the last dose of CAMZYOS.

Please see U.S. Full Prescribing Information, including Boxed WARNING.

Bristol Myers Squibb: Changing the Course of Cardiovascular Disease
Bristol Myers Squibb is inspired by a single vision—transforming patients’ lives through science. Cardiovascular disease is the leading cause of death worldwide, and despite major advances in how we prevent and treat it, the human and societal burden continues to worsen over time.¹¹ Whether a cardiovascular disease that affects millions of people around the world, or a rarer condition, the need is the same: new and better treatment options that allow people to continue to live their fullest lives.

Bristol Myers Squibb is committed to developing new treatments to address the global burden of cardiovascular disease. Building on our 70-year legacy of discovering and delivering paradigm-changing cardiovascular medicines, we are leveraging our experience and expertise, taking cardiovascular research to the next level to deliver meaningful, life‑changing outcomes for patients.

About Bristol Myers Squibb: Transforming Patients’ Lives Through Science
At Bristol Myers Squibb, our mission is to discover, develop and deliver innovative medicines that help patients prevail over serious diseases. We are pursuing bold science to define what’s possible for the future of medicine and the patients we serve. For more information, visit us at BMS.com or follow us on LinkedIn, X, YouTube, Facebook and Instagram.

Cautionary Statement Regarding Forward-Looking Statements
This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995 regarding, among other things, the research, development and commercialization of pharmaceutical products. All statements that are not statements of historical facts are, or may be deemed to be, forward-looking statements. Such forward-looking statements are based on current expectations and projections about our future financial results, goals, plans and objectives and involve inherent risks, assumptions and uncertainties, including internal or external factors that could delay, divert or change any of them in the next several years, that are difficult to predict, may be beyond our control and could cause our future financial results, goals, plans and objectives to differ materially from those expressed in, or implied by, the statements. These risks, assumptions, uncertainties and other factors include, among others, whether CAMZYOS^® (mavacamten) for the treatment of adults and pediatric patients weighing 30 kg (66 lbs) or more with symptomatic obstructive hypertrophic cardiomyopathy (oHCM) will be commercially successful, any marketing approvals, if granted, will have significant limitations on their use, and that continued approval of CAMZYOS^® (mavacamten) for the additional indication described in this release may be contingent upon verification and description of clinical benefit in confirmatory trials. No forward-looking statement can be guaranteed. Forward-looking statements in this press release should be evaluated together with the many risks and uncertainties that affect Bristol Myers Squibb’s business and market, particularly those identified in the cautionary statement and risk factors discussion in Bristol Myers Squibb’s Annual Report on Form 10-K for the year ended December 31, 2025, as updated by our subsequent Quarterly Reports on Form 10-Q, Current Reports on Form 8-K, and other filings with the Securities and Exchange Commission. The forward-looking statements included in this document are made only as of the date of this document and except as otherwise required by applicable law, Bristol Myers Squibb undertakes no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events, changed circumstances or otherwise.

References

1. CAMZYOS [package insert]. Princeton, NJ: Bristol-Myers Squibb Company; 2026.
2. Rossano JW, Canter C, Wolf CM, et al. Mavacamten in Adolescents with Obstructive Hypertrophic Cardiomyopathy. N Engl J Med. Published online March 29, 2026. doi:10.1056/NEJMoa2601103
3. Data on file. Total unique patients started on CAMZYOS. Valid as of July 2026. Princeton, NJ: Bristol-Myers Squibb Company; 2026.
4. Ommen SR, Ho CY, Asif IM, et al. 2024 AHA/ACC/AMSSM/HRS/PACES/SCMR Guideline for the Management of Hypertrophic Cardiomyopathy: A Report of the American Heart Association/American College of Cardiology Joint Committee on Clinical Practice Guidelines. Circulation. 2024;149(23):e1239-e1311. doi:10.1161/CIR.0000000000001250
5. Rossano J, Canter C, Wolf C, et al. Mavacamten in symptomatic adolescent patients with obstructive hypertrophic cardiomyopathy: design of the phase 3 SCOUT-HCM trial. Am Heart J. 2026;292:107283. doi:10.1016/j.ahj.2025.107283
6. Bristol Myers Squibb. U.S. Food and Drug Administration accepts for priority review Bristol Myers Squibb’s supplemental new drug application for Camzyos (mavacamten) to treat adolescents with symptomatic obstructive hypertrophic cardiomyopathy (oHCM). News release. June 1, 2026. Accessed July 22, 2026.
7. Desai MY, Wolski K, Owens A, et al; VALOR-HCM Investigators. Mavacamten in patients with hypertrophic cardiomyopathy referred for septal reduction: week 128 results from VALOR-HCM. Circulation. 2025;151(19):1378-1390. doi:10.1161/CIRCULATIONAHA.124.072445
8. Owens AT et al. Treatment With Mavacamten for Up to 5 Years in Patients With Obstructive Hypertrophic Cardiomyopathy: Final Results From MAVA-LTE. Oral presentation at ESC 2026; August 28-31, 2026; Munich, Germany
9. Olivotto I, Oręziak A, Barriales-Villa R, et al. Mavacamten for treatment of symptomatic obstructive hypertrophic cardiomyopathy (EXPLORER-HCM): a randomised, double-blind, placebo-controlled, phase 3 trial. Lancet. 2020;396(10253):759-769.
10. Anderson RL, Trivedi DV, Sarkar SS, et al. Deciphering the super relaxed state of human β-cardiac myosin and the mode of action of mavacamten from myosin molecules to muscle fibers. Proc Natl Acad Sci U S A. 2018;115(35): E8143-E8152.
11. Global, Regional, and National Burden of Cardiovascular Diseases and Risk Factors in 204 Countries and Territories, 1990-2023. JACC. 2025 Dec, 86 (22) 2167–2243. https://doi.org/10.1016/j.jacc.2025.08.015

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