EXPLORER-HCM Study Design^1,2

The efficacy and safety of CAMZYOS was evaluated in EXPLORER-HCM, a Phase 3, randomized, double-blind, placebo-controlled study that enrolled 251 adult patients with symptomatic NYHA Class II–III obstructive HCM, LVEF ≥55%, and LVOT peak gradient ≥50 mmHg at rest or with provocation. Patients were randomized in a 1:1 ratio to receive a starting dose of 5 mg of CAMZYOS (n=123) or placebo (n=128) once daily for 30 weeks. The dose was periodically adjusted to optimize patient response (following LVEF, Valsalva LVOT Gradient, and pharmacokinetics). 92% of patients were on background therapy with a beta blocker or calcium channel blocker in the pooled CAMZYOS (n=119/123) and placebo (n=112/128) groups.

Patients could achieve the primary composite functional endpoint with either ≥1 NYHA class improvement and ≥1.5 mL/kg/min improvement in pVO₂, or no worsening in NYHA class and ≥3.0 mL/kg/min improvement in pVO₂.

Secondary endpoints included the effect of CAMZYOS and placebo on postexercise LVOT peak gradient and the proportion of patients with improvement in NYHA class. Exploratory endpoints included the effect on Valsalva LVOT gradient and the proportion of patients with postexercise LVOT gradient <30 mmHg; all endpoints were assessed from baseline to Week 30.

EXPLORER-LTE Study Design³

The EXPLORER-LTE cohort is a part of MAVA-LTE, a single-arm, open-label, ongoing extension of the Phase 3 EXPLORER-HCM study to evaluate the long-term safety and efficacy of CAMZYOS, starting at 5 mg. 231 of 251 eligible patients from the EXPLORER-HCM trial enrolled in the long-term extension, after an 8-week washout period; the dose was periodically adjusted to optimize patient response, based on LVEF and Valsalva LVOT gradient. The majority of patients (92%) were on background therapy with a BB or CCB at baseline. The primary endpoint of the study was safety and tolerability.

EXPLORER-LTE Study Limitations

• These LTE data are not included in the CAMZYOS US Full Prescribing Information and caution should be used in interpreting the data; there are limitations with the data including decreased sample size and different continuation rates based on the continued involvement of responders and attrition of nonresponders
• MAVA-LTE, including the EXPLORER-LTE cohort (n=231), is a single-arm study without an active comparator, and data were not statistically tested for significance but are only descriptive in nature
• Patients enrolled in EXPLORER-LTE were part of either the CAMZYOS or placebo arm in the EXPLORER-HCM study; therefore, baseline characteristics for EXPLORER-LTE changed due to time and the consolidation of both arms to one cohort

CAMZYOS delivered significant improvements in both symptoms and obstruction in adults with symptomatic NYHA Class II–III obstructive HCM in EXPLORER-HCM. At Week 30, 2x as many patients on CAMZYOS achieved the primary composite endpoint of increased exercise capacity (pVO₂) and saw improvement or no worsening in NYHA class vs placebo [CAMZYOS (37%; n=45/123), placebo (17%; n=22/128); difference (95% CI): 19% (9, 30); P=0.0005].¹

• Exploratory Endpoint: In EXPLORER-HCM, patients achieved reductions in mean provoked Valsalva LVOT gradient by the first clinical visit (Week 4)* that were sustained through Week 30^1,2  
  • Mean (SD) changes were -49 (34) mmHg for the CAMZYOS group and -12 (31) mmHg for the placebo group¹
• Secondary Endpoint: At Week 30, patients on CAMZYOS had a mean change in postexercise LVOT peak gradient of -47 mmHg vs
-10 mmHg with placebo (P<0.0001)¹
• Exploratory Endpoint: 57% of patients on CAMZYOS no longer had obstruction (postexercise LVOT gradient <30 mmHg) vs 7% of patients on placebo at Week 30²
• These prespecified exploratory endpoints were not powered for significance, and statistical comparisons have not been made
• Secondary Endpoint: 65% of patients improved ≥1 NYHA class vs 31% on placebo (P<0.0001)¹
• Click here to explore the full data

*Week 4 marked the first scheduled assessment of LVOT gradient following the initiation of CAMZYOS.¹

CAMZYOS results observed in EXPLORER-HCM were sustained at ^~3.5 years in EXPLORER-LTE.^1-5

• CAMZYOS reduced Valsalva LVOT gradient at Week 30 in EXPLORER-HCM, with results sustained at ^~3.5 years in EXPLORER-LTE. The mean (SD) change in Valsalva LVOT gradient from baseline to Week 180 in EXPLORER-LTE was -55 (34) mmHg. Overall, 83% (n=191) achieved a reduced Valsalva LVOT gradient of ≤30 mmHg, or below obstructive levels at the time of analysis (data cutoff)^1-4
• In EXPLORER-LTE, 78% of patients improved their NYHA classification and 66% (n=63/95) of patients were asymptomatic 
(NYHA Class I) at ^~3.5 years³
  • 29 patients (31%) were NYHA Class II and 3 patients (3%) were NYHA Class III³
• These prespecified exploratory endpoints were not powered for significance, and statistical comparisons have not been made

Click here to dive into the EXPLORER-HCM and EXPLORER-LTE data.

CAMZYOS REMS Data⁶

The FDA approved CAMZYOS in April 2022 with a REMS program in collaboration with BMS to ensure monitoring for heart failure due to systolic dysfunction. As this is not a clinical study, there was no required institutional review board approval. The data are based on submitted forms completed by HCPs and pharmacies and were focused on compliance with 4 key program elements:

• HCP/pharmacy certifications and training
• Patient enrollment and monitoring with echocardiograms
• Screening for contraindicated concomitant medications and medications with drug interactions that require dosage adjustment
• Ensuring that CAMZYOS dispensing occurred only when the previous 3 elements were completed and consistent with the REMS program

These outcomes are from REMS Assessment Reports provided to the FDA over a period of 34 months. The following are a summary of data from reports covering April 28, 2022, to February 27, 2025. There were 11,982 patients that received at least 1 dispense of CAMZYOS, and 11,007 patients had PSFs submitted as of February 27, 2025.

Data Limitations

• These single-arm data are not included in the US Full Prescribing Information for CAMZYOS; caution should be used in interpreting the data, as they were not statistically tested for significance but are descriptive in nature
• REMS data collection is designed to demonstrate that the required safe use conditions have been met, and does not provide robust clinical data on safety, efficacy, or outcomes
• Current data also do not provide exact values for various echocardiographic variables (eg, LVEF, Valsalva LVOT gradient), nor does it provide any information about potential confounding etiologies
• The REMS program relies on individual providers making an accurate diagnosis of obstructive HCM prior to initiating CAMZYOS
• There is a potential for underreporting:
  • It is possible that not all PSFs were submitted, and it is possible these patients experienced an unrelated decrease in LVEF, experienced an adverse event, potentially had insurance coverage issues, etc
  • The REMS program is designed to ensure that patients are eligible to continue therapy, and there is little incentive to report events that do not permit continued treatment with CAMZYOS

In a 34-month CAMZYOS REMS data capture of over 11,000 patients, reductions in Valsalva LVOT gradient were sustained over time, and real-world LVEF findings were consistent with CAMZYOS EXPLORER-HCM clinical trial experience.^1,6

• REMS data collection is designed to assess that the required safe use conditions have been met and does not provide robust clinical data on safety, efficacy, or outcomes. Provider discretion is advised. Results are not powered for significance

Click here for more details on the CAMZYOS REMS Program Data. 

At Week 30, mean (SD) changes in Valsalva LVOT gradient were -49 (34) mmHg for the CAMZYOS group and -12 (31) mmHg for the 
placebo group.¹

In EXPLORER-HCM, reduction in mean Valsalva LVOT gradient was observed by the first clinical visit (Week 4)^* and sustained through Week 30.²

This prespecified exploratory endpoint was not powered for significance, and statistical comparisons have not been made.

• Patient response should be assessed as individualized (through LVEF and Valsalva LVOT gradient) and monitored via echo through scheduled follow-up¹

*Week 4 marked the first scheduled assessment of LVOT gradient following the initiation of CAMZYOS.¹

No head-to-head studies have been done, and cross-trial comparisons cannot be made. 

• CMI trials should be interpreted within the context of each individual study and cannot be directly compared in the absence of 
head-to-head data due to differences including enrollment criteria, study design, patient population, and endpoints
• For CAMZYOS, the evidence base includes the pivotal Phase III trial, EXPLORER-HCM, EXPLORER Long Term Extension Study, and REMS real-world data capture^1,3,6

The CAMZYOS safety profile is supported by EXPLORER-HCM and is reinforced by long-term follow-up through ^~3.5 years in EXPLORER-LTE.^1,3

• In EXPLORER-HCM, adverse reactions occurring in >5% of patients and more commonly with CAMZYOS than placebo were dizziness (27% vs 18%) and syncope (6% vs 2%); syncope (0.8%) was the only adverse reaction leading to discontinuation in patients receiving CAMZYOS¹
• In EXPLORER-LTE, 91% of patients remained on CAMZYOS at data cutoff (n=211/231)³
• Through Week 180, drug-related serious TEAEs occurred in 10 patients (4.3%), including decreased ejection fraction (n=5), cardiac failure (n=3), atrial fibrillation, and atrial flutter (both n=1)

Click here for full safety information.

CAMZYOS targets hypercontractility in obstructive HCM and reduces cardiac contractility, so changes in LVEF can occur, which is why LVEF monitoring is built into treatment.¹

• CAMZYOS reduces LVEF and can cause heart failure due to systolic dysfunction. Following initiation, patients may develop heart failure while taking a cardiac myosin inhibitor like CAMZYOS. Regular LVEF and Valsalva LVOT gradient assessment is required for careful dose titration to achieve an appropriate target Valsalva LVOT gradient while maintaining LVEF ≥50% and avoiding heart 
failure symptoms¹
• Echocardiogram assessments of LVEF and Valsalva LVOT gradient are required prior to and during treatment¹
• In EXPLORER-HCM, mean LVEF remained above 60% at all trial visits, with results sustained at ^~3.5 years^1-3
• LVEF reductions were reversible and affected a limited number of patients in EXPLORER-HCM; 7 (6%) patients in the CAMZYOS group and 2 (2%) patients in the placebo group experienced reversible reductions in LVEF <50% while on treatment, and all 7 CAMZYOS-treated patients recovered following interruption¹
• In EXPLORER-LTE through Week 180, 20 patients (8.7%) experienced reductions in LVEF <50% in a total of 22 events
• In the real-world dataset from the REMS Program, the rates of LVEF <50% were consistent with the data from the initial clinical trials of CAMZYOS^1,6
• Click here for more details on LVEF from the CAMZYOS REMS Program

As part of the CAMZYOS REMS program, every patient is assessed for DDIs and contraindications before starting and during treatment by prescribers and dispensing specialty pharmacists, providing opportunities for clinical interventions.⁶

• In a recent ~3-year REMS analysis of >147,000 drug interaction and counseling checklists, <1% of checklists identified drug interactions⁶

Click here for a review of DDIs and Contraindications with CAMZYOS.

Patients with a history of AFib were allowed to enroll in the EXPLORER-HCM trial.²

• Patients with paroxysmal or intermittent AFib on screening ECG, and patients with persistent or permanent AFib not on anticoagulation for at least 4 weeks or not adequately rate-controlled within 6 months of screening were excluded from the trial²
• At baseline, 10% of patients in the CAMZYOS group and 18% of patients in the placebo group had a history of AFib²
• At Week 30, there were no differences in AFib events between the treatment groups (serious cardiac adverse events of AFib occurred in 2% of patients on CAMZYOS and 3% of patients on placebo; any AFib occurred in 6 patients (5%) in both the CAMZYOS and placebo groups)²
• With prolonged CAMZYOS use at ^~3.5 years, ^~14% of patients in EXPLORER-LTE experienced AFib episodes; about half of those patients had reported an underlying history of AFib at the time of enrollment³

Considering an incidence of AFib of ^~2%–5% per year among patients with HCM, these rates are not higher than expected in the overall obstructive HCM patient population.^7,8

CAMZYOS dosing follows a predictable and manageable echo monitoring approach, guided by echocardiographic and clinical parameters to optimize efficacy while monitoring safety.¹

• Treatment is initiated at 5 mg once daily, with titration to the appropriate dose (2.5 mg, 5 mg, 10 mg, or 15 mg) based on LVEF, Valsalva LVOT gradient, and clinical response¹
• In EXPLORER-HCM, 82% of patients completed the trial on 5 mg or 10 mg at Week 30, indicating that most patients achieved target dose (based on LVEF, Valsalva LVOT gradient, and pharmacokinetics) within a relatively narrow and predictable dosing range¹
  • 7% were receiving the 2.5 mg dose and 11% were receiving 15 mg at Week 30¹

For additional echo monitoring guidance and dosing evaluation considerations, see the Interactive Dosing Guide or refer to the Dosing and Administration and Clinical Studies sections of the US Full Prescribing Information.

In the initiation phase, echocardiograms are scheduled during Weeks 4, 8, and 12 per the dosing algorithm in the US Full Prescribing Information (Section 2). LVEF must be assessed before initiation; initiation or up-titration is not recommended if LVEF is <55%.¹

In the maintenance phase (Week 12 or later), if the patient is stable with LVEF ≥55% and Valsalva LVOT gradient <30 mmHg, clinical status and echo are rechecked after 6 months and then every 6 months thereafter; check clinical status after 3 months during the first 6-month cycle. For additional Maintenance Phase scenarios, such as LVEF 50-<55% or LVEF ≥55% and Valsalva LVOT gradient
≥30 mmHg, consult the US Full Prescribing Information (Section 2, Figure 2). Additional echocardiograms may be needed after dosing changes, treatment interruption, or starting certain medications known to interact with CAMZYOS.¹

If LVEF <50%, interrupt treatment and recheck echo parameters every 4 weeks until LVEF returns to at least 50%.

Click here for more information about dosing.

The CAMZYOS Risk Evaluation and Mitigation Strategy (REMS) program is designed to help support closer care and monitor patient safety through LVEF echo monitoring for detection of heart failure and assessing for drug interactions. 

Click here for more information on CAMZYOS REMS.

Certification can be completed online at CAMZYOSREMS.com or by printing and faxing the HCP Enrollment Form to 833-299-9539, calling 833-628-7367, or emailing REMSSupport@bms.com.

• To become certified, HCPs must review the CAMZYOS Prescribing Information, review the Program Overview, review the Education Program for Healthcare Providers and Pharmacies, successfully complete the Healthcare Provider Knowledge Assessment, and enroll by completing the Healthcare Provider Enrollment Form
• HCPs will be notified within 1 business day if they have been certified to prescribe CAMZYOS

Prior to initiating treatment, confirm your patients eligibility and assess baseline clinical status¹:

• Confirm absence of pregnancy and use of effective contraception in females of reproductive potential
• Assess LVEF by echo; do not initiate treatment in patients with LVEF <55%
• Consider and assess for drug interactions prior to and throughout treatment

Start & Maintain patients on CAMZYOS with 3 steps:

1. Enroll
2. Prescribe
3. Maintain and monitor

Your patients and practice must maintain the echocardiogram schedule to avoid unnecessary interruptions. If echocardiograms are not completed on time, the medication will not be dispensed.

Learn more about starting and maintaining patients on CAMZYOS here.

Once CAMZYOS has been prescribed, MyCAMZYOS can assist with Patient Access Support, Free Trial Program, Bridge Program, Co-Pay Assistance, and Echocardiogram Co-Pay Program if eligible. 

Click here for more support. 

You can enroll your patients in MyCAMZYOS at account.covermymeds.com or by calling 855-CAMZYOS (855-226-9967).

Once the patient is enrolled, the program will check the patient's insurance type and benefits. If the patient is deemed commercially insured, they may be eligible for the CAMZYOS Co-Pay Program and pay as little as $10 for a 30-day supply for their prescription. They may also be eligible for echocardiogram co-pay assistance and may pay as little as $0 in out-of-pocket costs per echocardiogram procedure, subject to an annual maximum benefit of $2500.

Patients are responsible for any costs that exceed the maximum benefit.

Commercially insured patients who experience a 5-day coverage determination delay may be eligible for the bridge program.


The patient will be provided CAMZYOS at no cost until coverage is received, a prior authorization is denied and not appealed, or for 18 dispenses, whichever is earlier.

Traditional oral medication can be used to manage symptoms. Traditional treatment includes beta blockers, calcium channel blockers, and disopyramide. These medications are used for symptom management of obstructive HCM, and are recommended treatment options by the AHA/ACC/MS Guideline.⁴ However, most are not approved by the FDA specifically for obstructive HCM, also known as oHCM.

When the obstruction remains and symptoms persist with 1L pharmacologic medication (BB or CCB), CAMZYOS, as a CMI, is an
AHA/ACC/MS Guideline-recommended treatment option. Unlike most medications commonly prescribed for obstructive HCM, CAMZYOS is a CMI specifically designed to target hypercontractility, the source of obstructive HCM.^1,4 

Additionally, invasive procedures are an option for treating the obstruction. Alcohol septal ablation and septal myectomy, a type of surgery, can be performed (also known as septal reduction therapy). In removing the obstruction, these procedures may also result in symptom relief.⁴

Excess myosin-actin cross-bridge formation and dysregulation of the super-relaxed state are mechanistic hallmarks of HCM.¹

CAMZYOS is an allosteric and reversible inhibitor selective for cardiac myosin. CAMZYOS modulates the number of myosin heads that can enter “on actin” (power-generating) states, thus reducing the probability of force-producing (systolic) and residual (diastolic) cross-bridge formation.¹

CAMZYOS shifts the overall myosin population towards an energy-sparing, recruitable, super-relaxed state. In HCM patients, myosin inhibition with CAMZYOS reduces dynamic LVOT obstruction and improves cardiac filling pressures.¹

Click here for more information.

If a dose is missed, it should be taken as soon as possible, and the next scheduled dose should be taken at the usual time the following day.¹ 

Exact timing of the dose during the day is not essential, but two doses should not be taken on the same day.¹ 

Swallow capsules whole. Do not break, open, or chew the capsules.¹ 

Click here for more information on CAMZYOS dosing.