A Phase 3, Open Label, Single Arm Study to Evaluate Efficacy, Safety and Tolerability of Aficamten in Adult Japanese Patients With Symptomatic Obstructive Hypertrophic Cardiomyopathy

Researchers are looking for a better way to treat Japanese people who have symptomatic obstructive hypertrophic cardiomyopathy (symptomatic oHCM).
Obstructive hypertrophic cardiomyopathy (oHCM) is a type of heart disease where the heart muscles become thicker than normal due to over contraction. This thickening makes it harder than normal due to over contraction. This thickening makes it harder for the heart to pump blood out to the rest of the body. In symptomatic oHCM people with the condition experience symptoms like shortness of breath, chest pain, fainting, high blood pressure and irregular heartbeats.
The study treatment aficamten, also called BAY3723113, is under development to treat symptomatic oHCM. It aims to reduce the activity of cardiac myosin, a protein that helps heart muscles to contract, and thereby preventing over contraction and muscle thickening.
Although treatment options are available for symptomatic oHCM, there is still need for other treatment options that help target the root cause of the condition. In this study, researchers want to understand about the effects and long-term safety of aficamten in Japanese people with symptomatic oHCM.
The main purpose of the study is to learn how well aficamten works in Japanese with symptomatic oCHM.
For this, the researchers will check how participant's heart blood flow changes after 6 months of treatment. They do this by measuring the pressure needed for blood to leave the heart using a test called the left ventricular outflow tract (LVOT) gradient and a special breathing technique called Valsava maneuver.
Researchers will also look for:
* the number of participants who will have at least 1 level improvement on a scale doctors use to assess the effect of heart problems on daily activities after 3 and 6 months of treatment
* the change in the impact of heart problems on participant's daily lives based on their feedback on a questionnaire called Kansas City Cardiomyopathy Questionnaire - Clinical Summary Score (KCCQ-CSS) after 3 and 6 months of treatment.
This study will have 2 treatment periods: main treatment period and long-term treatment period. During the main treatment period, participants will take aficamten tablets once daily by mouth for up to 6 months. After completing this period, the participants who can join the long-term treatment period will continue taking aficamten until the drug becomes commercially available in Japan or the study ends.
Each participant will be in the study as long as they benefit from the treatment.
Participants will visit the study site:
* once before the treatment starts
* 9 times with a gap of 2 to 4 weeks between the visits during treatment under the main treatment period, and in the long-term treatment period, participants will visit almost every 3 months until the treatment ends.
* then 2 more times with a gap of 1 month between the visits after the treatment ends.
During the study, the study doctors and their team will:
* check participant's health by performing tests such as blood and urine tests, and checking heart health using an electrocardiogram (ECG) and echocardiogram (ECHO)
* ask the participants questions about how they are feeling and what adverse events are they having
An adverse event is any medical problem that a participant has during a study. Study doctors keep track of all adverse events, irrespective if they think it is related or not to the study treatment. In addition, the participants will be asked to complete a questionnaire on quality of life at certain time points during the study.
If the participant benefits from the treatment, treatment with aficamten after the end of the study might be possible.

Start Date30 Jun 2025

Sponsor / Collaborator

Bayer AG

NCT06412666

/ RecruitingPhase 2/3

A Phase 2/3 Multicenter, Randomized, Double-Blind, Placebo-Controlled and Open-Label Extension Trial to Evaluate the Efficacy and Safety of Aficamten in a Pediatric Population With Symptomatic Obstructive Hypertrophic Cardiomyopathy

The purpose of this study is to evaluate the efficacy, safety and PK of aficamten in a pediatric population with symptomatic obstructive hypertrophic cardiomyopathy (oHCM).

Start Date29 May 2024

Sponsor / Collaborator

Cytokinetics, Inc.

NCT06116968

/ RecruitingPhase 3

An Open-Label Study of Aficamten for Chinese Patients With Symptomatic Obstructive Hypertrophic Cardiomyopathy

This is an open-label extension study of China cohort in the phase 3 study (CY 6031) of aficamten for the treatment of obstructive HCM (oHCM) to collect long-term safety and tolerability data, including assessments of cardiac function and steady-state Pharmacokinetics (PK) during chronic dosing with aficamten.

Start Date14 Nov 2023

Sponsor / Collaborator-

100 Clinical Results associated with Aficamten

100 Translational Medicine associated with Aficamten

100 Patents (Medical) associated with Aficamten

Literatures (Medical) associated with Aficamten

01 Dec 2025·Journal of cardiovascular imaging

Cardiac myosin inhibitors in hypertrophic cardiomyopathy

Review

Author: Lim, Jaehyun ; Kim, Hyung-Kwan

Abstract:

Mavacamten, the first selective and reversible cardiac myosin inhibitor (CMI), has been introduced to the clinical arena for the treatment of obstructive hypertrophic cardiomyopathy (HCM). By reducing excessive actin-myosin cross-bridging, this agent decreases myocardial contractility and alleviates the dynamic left ventricular outflow tract (LVOT) obstruction in obstructive HCM. In the EXPLORER-HCM trial, mavacamten significantly improved exercise capacity, symptoms, and LVOT pressure gradients, while the VALOR-HCM trial proved it can obviate the need for septal reduction therapy in patients who were deemed to be candidates for septal reduction therapy. Notably, long-term data (MAVA-LTE study) has demonstrated sustained benefits up to 180 weeks, with < 10% experiencing transient reductions in left ventricular ejection fraction < 50% and only 1.3% of permanent discontinuation rate. Aficamten, a next-generation CMI with a shorter half-life, has also demonstrated comparable efficacy. Reverse remodeling following treatment was noted in both agents. In nonobstructive HCM, preliminary studies (MAVERICK-HCM trial and cohort 4 of REDWOOD-HCM trial) have reported improvements in cardiac serum biomarkers and symptoms. However, the preliminary results from phase 3 trials (ODYSSEY-HCM trial) revealed that primary endpoints were not met in nonobstructive HCM. Regarding safety, both were generally well tolerated. Although an LVEF reduction occurred in some patients, it was reversible with a dose reduction or a short-term drug cessation. These results emphasize careful dosing strategy with regular echocardiographic monitoring. Real-world data have also demonstrated consistent efficacy and safety across varying ethnic groups without new safety signals. CMI is a major advance in HCM management. However, future studies must provide data on hard clinical outcomes, such as heart failure hospitalization or death. Ongoing trials comparing CMI to traditional first-line therapies, such as β-blockers, will clarify their potential role as an initial therapeutic option.

22 Oct 2025·EUROPEAN HEART JOURNAL

Efficacy of aficamten in patients with obstructive hypertrophic cardiomyopathy and mild symptoms: results from the SEQUOIA-HCM trial

Article

Author: Coats, Caroline J ; Nassif, Michael E ; Claggett, Brian L ; Veselka, Josef ; Wohltman, Amy ; Malik, Fady I ; Maron, Martin S ; Barriales-Villa, Roberto ; Gimeno, Juan Ramon ; Heitner, Stephen B ; Januzzi, James L ; Kupfer, Stuart ; Jacoby, Daniel L ; Spertus, John A ; Masri, Ahmad ; Hegde, Sheila M ; Kulac, Ian J ; Olivotto, Iacopo

Abstract:

Background and Aims:

Patients with obstructive hypertrophic cardiomyopathy (oHCM) treated with aficamten in SEQUOIA-HCM (NCT05186818) demonstrated marked improvement in symptoms and functional capacity. This analysis explores whether oHCM and mild symptoms patients experience similar clinical benefits with aficamten as patients with more advanced limitations.

Methods:

Patients in SEQUOIA-HCM (N = 282) were grouped at baseline according to symptom severity. Mild symptoms (n = 118) were defined as New York Heart Association (NYHA) class II and Kansas City Cardiomyopathy Questionnaire Clinical Summary Score (KCCQ-CSS) ≥ 80, and moderate to severe symptoms (n = 150) as NYHA class II/III/IV and KCCQ-CSS <80. Primary endpoint was change in peak oxygen uptake (pVO2) from baseline to Week 24; secondary endpoints included change in NYHA class, KCCQ-CSS, outflow tract gradients, and N-terminal pro-B-type natriuretic peptide (NT-proBNP).

Results:

In aficamten-treated patients, change at Week 24 was not different between moderate to severe (1.8 mL/kg/min; n = 71) and mild (1.6 mL/kg/min; n = 62) symptom groups (P = .8). Likewise, the change in secondary endpoints (NYHA class, resting or Valsalva gradients, and NT-proBNP) did not differ significantly between the two symptom groups. Both groups experienced statistically significant improvements in KCCQ-CSS, but the extent of improvement was greater in the advanced symptom group (P = .02 for interaction). Treatment-emergent serious adverse events were infrequent in both groups.

Conclusions:

Patients with oHCM and mild symptoms treated with aficamten achieved significant improvement across a range of clinically relevant outcomes and generally similar to patients with more advanced symptoms. Less severely symptomatic patients could be considered for aficamten treatment.

07 Oct 2025·CIRCULATION

Characterization and Application of Novel Exercise Recovery Patterns That Reflect Cardiac Performance: A Substudy of the SEQUOIA-HCM Trial

Article

Author: Kulac, Ian J. ; Malik, Fady I. ; Owens, Anjali T. ; Wohltman, Amy ; Claggett, Brian L. ; Campain, Joseph ; Griskowitz, Catharine ; Kupfer, Stuart ; Prasad, Cheshta ; Moreno, Fabely ; Coats, Caroline J. ; Heitner, Stephen B. ; Newlands, Chloe ; Lee, Matthew M.Y. ; Landsteiner, Isabela ; Malhotra, Rajeev ; Minasian, Alexandra ; Giverts, Ilya ; Maron, Martin S. ; Jacoby, Daniel L. ; Lewis, Gregory D. ; McGinnis, Shaina ; Rupert, Lillian ; Iskenderian, Nick

BACKGROUND::

Post-exercise oxygen uptake recovery (VO 2 Rec) is slow in advanced heart failure. We sought to establish easily derived VO 2 Rec measures and evaluate their cardiospecificity and prognostic relevance in patients with dyspnea on exertion. We further sought to determine VO 2 Rec modifiability proportional to changes in cardiac function with disease-specific treatment of obstructive hypertrophic cardiomyopathy.

METHODS::

VO 2 Rec patterns were evaluated in relation to cardiac performance and the primary outcome of heart failure hospitalization or death in a referral cohort with dyspnea on exertion undergoing cardiopulmonary exercise testing with hemodynamic monitoring (MGH-ExS [Massachusetts General Hospital Exercise Study]). We then investigated longitudinal measures of VO 2 Rec in the pivotal phase 3 randomized controlled trial SEQUOIA-HCM (Safety, Efficacy, and Quantitative Understanding of Obstruction Impact of Aficamten in Hypertrophic Cardiomyopathy) of aficamten versus placebo for 24 weeks in participants with symptomatic obstructive hypertrophic cardiomyopathy. For both cohorts, VO 2 Rec was uniformly measured as time for VO 2 to decline by >0%, 12.5% (VO 2 T 12.5% ), 25%, and 50% of peak VO 2 .

RESULTS::

Among 814 MGH-ExS patients (58±16 years of age, 58% women), those with a longer VO 2 T 12.5% (≥35 versus <35 seconds) demonstrated elevated exercise pulmonary capillary wedge pressure to cardiac output slope ( P <0.0001) with no difference in peripheral oxygen extraction ( P =0.11). For each 15-second increase in VO 2 T 12.5% , the hazard ratio for heart failure hospitalization and all-cause death was 1.54 (95% CI, 1.35–1.76; P <0.001). In SEQUOIA-HCM participants with cardiopulmonary exercise testing at baseline and week 24 (n=263, 59.1±2.9 years of age, 41% women), baseline VO 2 T 12.5% was 45±20 seconds and improved 8 seconds (95% CI, −12 to −5 seconds; P <0.001) with aficamten treatment compared with placebo at 24 weeks. Participants treated with aficamten versus placebo were more likely to improve VO 2 T 12.5% by ≥15 seconds (odds ratio [OR], 3.7 [95% CI, 1.9–6.9]; number needed to treat=4.8). Shortening of VO 2 T 12.5% correlated with reduced NT-proBNP (N-terminal pro-B-type natriuretic peptide), high-sensitivity cardiac troponin I, and left ventricular outflow tract gradient (all P <0.005).

CONCLUSIONS::

This study established VO 2 T 12.5% as a new measure that reflects cardiac performance during exercise and predicted heart failure event-free survival. Furthermore, VO 2 T 12.5% improved proportional to improvements in left ventricular outflow tract gradient and cardiac biomarkers in response to aficamten treatment, a cardiospecific therapy for obstructive hypertrophic cardiomyopathy. The simplicity and physiological relevance of VO 2 T 12.5% support its regular inclusion in cardiopulmonary exercise testing protocols evaluating cardiac function during exercise.

REGISTRATION::

URL: https://www.clinicaltrials.gov ; Unique identifier: NCT05186818.

267

News (Medical) associated with Aficamten

05 Jan 2026

·www.fiercepharma.com

2025 drug approvals: Despite FDA tumult and macro uncertainty, biopharma scored with 55 new products

Dozens of new drugs made the grade in 2025, and a clutch of newly commercial biopharma companies are now underway with their first launches. Photo by Sarah Silbiger/Getty Images How did staff layoffs, inspection backlogs and unprecedented leadership turnover affect the ability of the FDA to approve new drugs in 2025?While it would be presumptuous to base conclusions solely on the number of drugs the FDA approved in 2025, it is noteworthy that FDA nods of new treatments fell short of previous years, though a surge of green lights in December helped make the final numbers more respectable.There were 46 novel drug approvals in 2025, compared to 55 in 2023 and 50 in 2024. Meanwhile, the FDA’s Center for Biologics Evaluation and Research endorsed 18 new biological treatments in 2025, compared to 25 in 2023 and 18 in 2024.The surge in December included seven novel approvals, which was the most in any month of 2025. There also were many more novel approvals (30) in the second half of 2025 than in the first half (16), indicating that the U.S. regulator functioned more efficiently as it gained stability through the year.In March 2025, the Department of Health and Human Services announced a plan to cut 10,000 jobs, including 3,500 at the FDA. In the following months, scores of companies revealed delays for their product approvals that were initiated by the agency.A study from RBC Capital Markets revealed there were more marketing applications rejected in the third quarter of last year than in the previous six periods. Additionally, the FDA delayed 11% of the reviews of those applications in the third quarter compared to an average delay rate of 4% from the previous six quarters.Decline in potential blockbusters In addition to the decline in approvals in 2025, there was a drop in the number of new meds that are expected to generate blockbuster sales.In 2024, there were nearly 20 new products approved that were expected to deliver sales of at least $1 billion by 2030, according to Evaluate Pharma. On this year’s list, only eight newly approved medicines carry those expectations.In the last two weeks of December, Cytokinetics secured a nod for cardiomyopathy treatment Myqorzo and GSK scored with asthma medicine Exdensur. Myqorzo has been projected by Evaluate Pharma to generate sales of $2.8 billion by 2030, with Exdensur’s estimate at $1.2 billion.Of the new drugs that were approved in 2025, the one expected to generate the highest sales by 2030 is AstraZeneca and Daiichi Sankyo’s Datroway, which is the first TROP2-directed antibody-drug conjugate to treat breast cancer. Five months after it was approved, the companies earned an FDA clearance to treat patients with non-small cell lung cancer (NSCLC) who have EGFR mutations.The new drug projected to generate the second-most revenue in 2030 is Insmed’s Brinsupri, which is the first DPP1 inhibitor to reach the market. It also is the first treatment for non-cystic fibrosis bronchiectasis and is projected to reach sales of $3.1 billion in 2030.Other products approved in 2025 that are expected to be blockbusters by 2030 are Vertex’s non-opioid pain reliever Journavx, Novartis’ Rhapsido for chronic spontaneous urticaria, Johnson & Johnson’s Imaavy for generalized myasthenia gravis and GSK’s Penmenvy, a meningococcal vaccine.There were many more high-profile drugs approved in 2024, including Madrigal Pharmaceuticals' Rezdiffra for metabolic dysfunction-associated steatohepatitis; Merck’s Winrevair for pulmonary arterial hypertension; Bristol Myers Squibb’s Cobenfy for schizophrenia; J&J’s Lazcluze for NSCLC; BridgeBio’s Attruby for cardiomyopathy; Vertex’s Alyftrek for cystic fibrosis; and a pair from Eli Lilly—Alzheimer’s disease treatment Kisunla and atopic dermatitis therapy Ebglyss. Treatment types in 2025 The FDA signed off on 14 oncology treatments last year compared to 15 in 2024. Of the 14, five are for NSCLC, including two for non-squamous NSCLC. The splurge of NSCLC nods came after the FDA blessed three new treatments in the indication in 2024. In addition to AstraZeneca and Daiichi Sankyo’s high-profile approval for breast cancer drug Datroway, Eli Lilly also scored a nod for its breast cancer treatment Inluriyo.The FDA gave its thumbs-up to two blood cancer treatments: Kura Oncology and Kyowa Kirin’s Komzifti for acute myeloid leukemia and Regeneron’s Lynozyfic for multiple myeloma. It’s a continued slowdown from 2023, when the FDA signed off on seven new blood cancer therapies.Of the FDA’s 46 novel drug approvals in 2025, 26 (57%) were designated as rare disease (orphan) treatments. Drugs that are for diseases with fewer than 200,000 patients in the U.S. are slotted into the orphan category. It was a higher rate of rare disease treatments than in the previous two years, when just over half of the new approved drugs were for orphan diseases.Three of the orphan approvals were for the same disorder, hereditary angioedema (HAE), and came in a span of 10 weeks. The splurge began with a nod for CSL’s Andembry. The FDA also endorsed Ionis Pharmaceuticals’ Dawnzera, which is the first RNA-targeted treatment for HAE. The other approval came for KalVista Pharmaceuticals' plasma kallikrein inhibitor Ekterly, which differs from the other injected treatments in that it is taken orally and is used on demand to quell acute attacks of HAE.After a record number of new cell and gene therapy (CGT) approvals in 2024 with nine, there was a slowdown in CGT nods in 2025 with five. It was the fewest since 2022, when the FDA also issued five CGT approvals. With the exception of Novartis’ nod for spinal muscular atrophy treatment Itvisma, the approvals were scored by small companies Abeona, Precigen, Neurotech and Fondazione Telethon, an Italian firm that became the first nonprofit to get a CGT across the FDA finish line. European companies excel in 2025 European companies won many more approvals for new products than their counterparts in the U.S. In fact, no U.S. company earned more than one FDA nod in 2025. Meanwhile, five European companies scored multiple FDA approvals last year.GSK and Novartis achieved three approvals each. GSK earned its nods for meningococcal vaccine Penmenvy, urinary tract and gonorrhea antibiotic Blujepa and severe asthma treatment Exdensur. Novartis won approvals for nephropathy drug Vanrafia, urticaria medicine Rhapsido and spinal muscular atrophy gene therapy Itvisma.The companies with two FDA endorsements were Sanofi, which secured endorsements for thrombocytopenia therapy Wayrilz and hemophilia drug Qfitlia; Boehringer Ingelheim, which won over the regulator with idiopathic pulmonary fibrosis drug Jascayd and lung cancer treatment Hernexeos; and Bayer, which scored with menopause treatment Lynkuet and lung cancer therapy Hyrnuo.It was a lean year for U.S. Big Pharma as AbbVie, Eli Lilly, Johnson & Johnson, Merck and Regeneron were the only top 20 companies that gained approvals for new drugs.Note: The numbers for our annual roundup typically vary slightly from the FDA's official list, because our report includes approvals for biologic therapeutics and vaccines but excludes diagnostic imaging agents, which are represented on the FDA's list.

Drug ApprovalOrphan Drug

05 Jan 2026

·www.drugs.com

FDA Approves Myqorzo for Symptomatic Obstructive Hypertrophic Cardiomyopathy

MONDAY, Jan. 5, 2026 -- The U.S. Food and Drug Administration has approved Myqorzo (aficamten) for the treatment of adults with symptomatic obstructive hypertrophic cardiomyopathy (oHCM). Myqorzo is a cardiac myosin inhibitor that directly addresses underlying hypercontractility associated with oHCM and aids left ventricular outflow tract obstruction. Myqorzo is available in 5-, 10-, 15-, and 20-mg tablets and the U.S. Prescribing Information for Myqorzo includes a boxed warning for the risk of heart failure. Myqorzo reduces left ventricular ejection fraction (LVEF) and can cause heart failure due to systolic dysfunction, making echocardiogram assessments required before and during treatment to monitor for systolic dysfunction. The approval is based on the phase 3 SEQUOIA-HCM clinical trial that showed treatment with Myqorzo for 24 weeks significantly improved exercise capacity and increased peak oxygen uptake by 1.8 mL/kg/minute compared with baseline, versus 0.0 mL/kg/minute in patients treated with placebo (least square mean difference, 1.74 mL/kg/minute). The treatment effect of Myqorzo was persistent across all prespecified subgroups (e.g., age, sex, patient baseline characteristics) and regardless of background beta-blocker therapy. Myqorzo was well tolerated, with no instances noted of worsening heart failure or treatment interruptions due to low LVEF. Treatment-emergent serious adverse events occurred in 5.6 percent of patients treated with Myqorzo versus 9.3 percent taking placebo. Hypertension was the only adverse reaction occurring in >5 percent of patients, more commonly seen in patients on Myqorzo versus placebo (8 versus 2 percent). "I'm pleased that the approved label and REMS [Risk Evaluation and Mitigation Strategy] reflect the distinct characteristics of Myqorzo, including a straightforward, flexible dosing regimen, no requirement for drug-drug interaction monitoring, and a predictable safety profile," Robert I. Blum, the president and chief executive officer of Cytokinetics, said in a statement. Approval of Myqorzo was granted to Cytokinetics. More Information

Drug ApprovalPhase 3Clinical Result

22 Dec 2025

·www.einpresswire.com

FDA approves drug to improve functional capacity and symptoms in adults with rare inherited heart condition

Action The U.S. Food and Drug Administration (FDA) approved Myqorzo (aficamten) tablets to treat adults with symptomatic obstructive hypertrophic cardiomyopathy (oHCM) to improve functional capacity and symptoms. Disease or Condition Symptomatic oHCM is an inherited condition where patients have thickened heart muscle and reduced blood flow from the left side of the heart to the rest of the body, causing symptoms such as shortness of breath, fatigue, and potentially life-threatening cardiac events. Myqorzo Effectiveness The effectiveness and safety of Myqorzo were studied in 282 adults with symptomatic oHCM randomly assigned to receive Myqorzo or placebo for 24 weeks. At the end of the study, participants receiving Myqorzo had an increase in exercise capacity measured by peak oxygen uptake compared to no change in exercise capacity among those receiving placebo. Also, 59 percent of participants receiving Myqorzo experienced an improvement in physical activity limitations (measured using the New York Heart Association Classification system) compared to 24 percent of individuals receiving placebo. Safety Information Myqorzo reduces contraction of the heart muscle which can cause heart failure. Because of the risk for heart failure, patients treated with Myqorzo must be monitored with echocardiograms, an imaging test that shows how well the heart is working. Because of the heart failure risk and need for monitoring, Myqorzo is only available through a restricted program called the Myqorzo Risk Evaluation and Mitigation Strategy (REMS). Patients must also avoid certain prescription medicines that interfere with the metabolism (breakdown) of Myqorzo. See the prescribing information for additional information on risks associated with Myqorzo. Designations Myqorzo received Orphan Drug Designation and Breakthrough Therapy Designation for this indication. Content current as of: 12/22/2025 Legal Disclaimer: EIN Presswire provides this news content "as is" without warranty of any kind. We do not accept any responsibility or liability for the accuracy, content, images, videos, licenses, completeness, legality, or reliability of the information contained in this article. If you have any complaints or copyright issues related to this article, kindly contact the author above.

Drug ApprovalOrphan DrugAHABreakthrough TherapyClinical Result

100 Deals associated with Aficamten

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Approved

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Indication	Country/Location	Organization	Date
Hypertrophic obstructive cardiomyopathy	China	Cytokinetics, Inc.	16 Dec 2025

Developing

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Indication	Highest Phase	Country/Location	Organization	Date
Hypertrophic Cardiomyopathy without Obstruction	Phase 3	United States	Cytokinetics, Inc.	30 Aug 2023
Hypertrophic Cardiomyopathy without Obstruction	Phase 3	China	Cytokinetics, Inc.	30 Aug 2023
Hypertrophic Cardiomyopathy without Obstruction	Phase 3	Japan	Cytokinetics, Inc.	30 Aug 2023
Hypertrophic Cardiomyopathy without Obstruction	Phase 3	Argentina	Cytokinetics, Inc.	30 Aug 2023
Hypertrophic Cardiomyopathy without Obstruction	Phase 3	Australia	Cytokinetics, Inc.	30 Aug 2023
Hypertrophic Cardiomyopathy without Obstruction	Phase 3	Brazil	Cytokinetics, Inc.	30 Aug 2023
Hypertrophic Cardiomyopathy without Obstruction	Phase 3	Canada	Cytokinetics, Inc.	30 Aug 2023
Hypertrophic Cardiomyopathy without Obstruction	Phase 3	Denmark	Cytokinetics, Inc.	30 Aug 2023
Hypertrophic Cardiomyopathy without Obstruction	Phase 3	France	Cytokinetics, Inc.	30 Aug 2023
Hypertrophic Cardiomyopathy without Obstruction	Phase 3	Germany	Cytokinetics, Inc.	30 Aug 2023

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT06412666 \| NCT05186818 (CEDAR-HCM, Pubmed \| Circ Heart Fail)	Phase 3	Hypertrophic obstructive cardiomyopathy	65	Aficamten	klttmwjqaq(zsfpufjqil) = xxlrpjomwt lizqtksfhu (wczfzjarel )	Positive	05 Dec 2025
NCT05767346 (MAPLE-HCM, Pubmed \| J Am Coll Cardiol)	Phase 3	Hypertrophic obstructive cardiomyopathy	175	Aficamten 5 to 20 mg	qtjydyplku(jygscpwxeq) = eitkqtfniq bttcirlzyy (wtiddjwqkp ) View more	Positive	01 Dec 2025
				Metoprolol 50 to 200 mg	-
NCT05767346 (MAPLE-HCM, Pubmed \| J Am Coll Cardiol)	Phase 3	Hypertrophic obstructive cardiomyopathy	175	Aficamten	gezvzhbeez(hfhwpduhhy) = kgywvkexyl mqnsppsevg (qumojqaxdk ) View more	Positive	01 Nov 2025
				Metoprolol	gezvzhbeez(hfhwpduhhy) = lldbtowcfd mqnsppsevg (qumojqaxdk ) View more
NCT04848506 (FOREST-HCM, Company_Website) Manual	Phase 2/3	Hypertrophic Cardiomyopathy without Obstruction	34	Aficamten (post-REDWOOD-HCM Cohort 4)	ahgevsqoyd(tgnirhfzjk) = lippuqclng mloqyztkls (ldaglfysgf ) View more	Positive	29 Sep 2025
NCT05767346 (MAPLE-HCM, Company_Website) Manual	Phase 3	Hypertrophic obstructive cardiomyopathy	-	Aficamten	bvgeqjhaky(eqiveuhjvw) = sflngdihvn wincqkslhf (lrmltkrmis ) View more	Superior	29 Sep 2025
				Metoprolol	-
NCT05767346 (MAPLE-HCM, Pubmed \| N Engl J Med) Manual	Phase 3	Hypertrophic obstructive cardiomyopathy	175	Aficamten	zwchlaufax(iuglemxodw) = whpgbotgwk kgjdfyaysy (cwfejiplck, 0.5 - 1.7)	Superior	11 Sep 2025
				Metoprolol	zwchlaufax(iuglemxodw) = lcldstqbja kgjdfyaysy (cwfejiplck, -1.7 to -0.8)
NCT04848506 (FOREST-HCM, Pubmed \| JACC Heart Fail)	Phase 2/3	Hypertrophic obstructive cardiomyopathy	213	Aficamten 5 mg	nmijycuhbl(ycjxjdishf) = 2 (4.3%) asymptomatic and transient instances of left ventricular ejection fraction <50% (range: 47%-49%), neither resulting in drug discontinuation vnsnmgfslj (btjnmfonns ) View more	Positive	01 Aug 2025
				Aficamten ≤20 mg
NCT05767346 (MAPLE-HCM, GlobeNewswire \| NEWS) Manual	Phase 3	Hypertrophic obstructive cardiomyopathy	-	Aficamten	ftixnhrfuv(ctlylcggmb) = MAPLE-HCM met its primary endpoint, demonstrating a statistically significant improvement in peak oxygen uptake (pVO2) from baseline to Week 24 for aficamten compared to metoprolol. fdgifustyv (znvoiguwpv ) Met	Positive	13 May 2025
				Metoprolol
NCT05186818 (SEQUOIA-HCM, GlobeNewswire) Manual	Phase 3	Cardiomyopathy, Hypertrophic	-	Aficamten	aiecmeluqe(niooqjhuzz) = atiatdncja vkjphshqgp (qkrhvgygbk, 3.1) View more	Positive	16 Nov 2024
				Placebo	aiecmeluqe(niooqjhuzz) = rftoekobyc vkjphshqgp (qkrhvgygbk, 2.7) View more
NCT05186818 (SEQUOIA-HCM, Pubmed \| J Am Coll Cardiol)	Phase 3	Hypertrophic obstructive cardiomyopathy	282	Aficamten	dsgqlcymvi(ipyfjndajp) = eultcbtmod ywowghfslb (ydctjyfbzl, -25 to -6) View more	Positive	01 Nov 2024

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