Last update 21 Mar 2024

Cardiomyopathy, Hypertrophic, Familial

Last update 21 Mar 2024

Basic Info

Synonyms

Asymmetric Septal Hypertrophy, Familial, CARDIOMYOPATHY, FAMILIAL HYPERTROPHIC, CMH

+ [30]

Introduction

An autosomal dominant inherited form of HYPERTROPHIC CARDIOMYOPATHY. It results from any of more than 50 mutations involving genes encoding contractile proteins such as VENTRICULAR MYOSINS; cardiac TROPONIN T; ALPHA-TROPOMYOSIN.

Drugs associated with Cardiomyopathy, Hypertrophic, Familial

Perhexiline Maleate

Target

CPT1A x calcium channel

Mechanism

CPT1A inhibitors [+1]

Active Org.

Weill Cornell Medicine

Originator Org.-

Active Indication

Angina Pectoris [+1]

Inactive Indication

Cardiomyopathy, Hypertrophic [+2]

Drug Highest PhaseApproved

First Approval Ctry. / Loc.-

First Approval Date20 Jan 1800

JN-210

Target-

Mechanism-

Active Org.

Jaan Biotherapeutics LLC

Originator Org.

Jaan Biotherapeutics LLC

Active Indication

Cardiomyopathy, Hypertrophic, Familial

Inactive Indication-

Drug Highest PhaseEarly Phase 1

First Approval Ctry. / Loc.-

First Approval Date20 Jan 1800

MYK-581

Target

Cardiac myosin

Mechanism

Cardiac myosin inhibitors

Active Org.

MyoKardia, Inc.

Originator Org.

MyoKardia, Inc.

Active Indication

Cardiomyopathy, Hypertrophic, Familial

Inactive Indication-

Drug Highest PhasePreclinical

First Approval Ctry. / Loc.-

First Approval Date20 Jan 1800

Clinical Trials associated with Cardiomyopathy, Hypertrophic, Familial

NCT06269640 / Not yet recruitingNot ApplicableIIT

NHLBI SESAME (SEptal Scoring Along Midline Endocardium) Early Feasibility Study

Background:
Some people have a condition in which the wall (septum) that separates the two main pumping chambers of the heart is too thick. This thick septum causes a condition called "left ventricular outflow tract obstruction" (LVOTO), which reduces blood flow out of the heart. LVOTO can cause serious heart disease; symptoms may include shortness of breath, chest pain, heart failure, or death. Researchers want to find better ways to treat LVOTO.
Objective:
To test a new procedure where excess tissue is sliced away from the septum in people with LVOTO. This procedure is called "septal scoring along midline endocardium" (SESAME).
Eligibility:
Adults aged 21 years with LVOTO.
Design:
Participants will have baseline tests. They will have imaging scans and tests of their heart structure and function. They will take a walking test and answer questions about how their heart condition affects their life.
Participants will stay in the hospital 2 to 6 days for the SESAME procedure.
They will be completely or partially asleep for the procedure. A tube will be inserted into the mouth and down the throat to take pictures of the heart. Pictures may also be taken with a tube inserted inside the heart.
Next, tubes will be inserted into the groin and guided through the blood vessels up to the heart. Guidewires will be inserted into the heart. Doctors will watch the path the wires take with x-rays and ultrasound. When the wire is in the correct place, it will be electrified to slice excess tissue away from the septum.
Participants will have 3 follow-up visits within 1 year....

Start Date20 Mar 2024

Sponsor / Collaborator

Lung and Blood Institute

NCT05687474 / RecruitingNot Applicable

Universal Genomic Newborn Screening in the Wallonia-Brussels Federation: Baby Detect

Newborn screening (NBS) is a global initiative of systematic testing at birth to identify babies with pre-defined severe but treatable conditions. With a simple blood test, rare genetic conditions can be easily detected, and the early start of transformative treatment will help avoid severe disabilities and increase the quality of life.
Baby Detect Project is an innovative NBS program using a panel of target sequencing that aims to identify 126 treatable severe early onset genetic diseases at birth caused by 361 genes. The list of diseases has been established in close collaboration with the Paediatricians of the University Hospital in Liege. The investigators use dedicated dried blood spots collected between the first day and 28 days of life of babies, after a consent sign by parents.

Start Date01 Sep 2022

Sponsor / Collaborator

CHR Citadelle

[+5]

NCT05100420 / Enrolling by invitationNot ApplicableIIT

Hearts in Rhythm Organization Hypertrophic Cardiomyopathy Registry, Biobank and Imaging Data Repository (HiRO-HCM)

The Hearts in Rhythm Organization (HiRO) is a national network of Canadian researchers/clinicians, working towards a better understanding of the rare genetic causes of sudden cardiac death (SCD). The HiRO Hypertrophic Cardiomyopathy registry, biobank and imaging data repository (HiRO-HCM) is a multicenter study that will prospectively enroll patients with HCM as well as those carrying sarcomeric gene variants predisposing to HCM.
The objectives of HiRO-HCM are:
1. to better understand the natural history of the disease and identify clinical markers and biomarkers for adverse outcomes;
2. to derive and validate risk prediction models for disease expression, complications and response to therapy;
3. to better define the genetic architecture of sarcomeric and non-sarcomeric HCM.

Start Date23 Feb 2021

Sponsor / Collaborator

Montréal Heart Institute

[+1]

100 Clinical Results associated with Cardiomyopathy, Hypertrophic, Familial

100 Translational Medicine associated with Cardiomyopathy, Hypertrophic, Familial

0 Patents (Medical) associated with Cardiomyopathy, Hypertrophic, Familial

2,445

Literatures (Medical) associated with Cardiomyopathy, Hypertrophic, Familial

23 Feb 2024·GeroScience

Vascular smooth muscle cell-specific Igf1r deficiency exacerbates the development of hypertension-induced cerebral microhemorrhages and gait defects.

Article

Author: Lauren R Miller ; Marisa A Bickel ; Michaela L Vance ; Hannah Vaden ; Domonkos Nagykaldi ; Adam Nyul-Toth ; Elizabeth C Bullen ; Tripti Gautam ; Stefano Tarantini ; Andriy Yabluchanskiy ; Tamas Kiss ; Zoltan Ungvari ; Shannon M Conley

Cerebrovascular fragility and cerebral microhemorrhages (CMH) contribute to age-related cognitive impairment, mobility defects, and vascular cognitive impairment and dementia, impairing healthspan and reducing quality of life in the elderly. Insulin-like growth factor 1 (IGF-1) is a key vasoprotective growth factor that is reduced during aging. Circulating IGF-1 deficiency leads to the development of CMH and other signs of cerebrovascular dysfunction. Here our goal was to understand the contribution of IGF-1 signaling on vascular smooth muscle cells (VSMCs) to the development of CMH and associated gait defects. We used an inducible VSMC-specific promoter and an IGF-1 receptor (Igf1r) floxed mouse line (Myh11-Cre^ERT2 Igf1r^f/f) to knockdown Igf1r. Angiotensin II in combination with L-NAME-induced hypertension was used to elicit CMH. We observed that VSMC-specific Igf1r knockdown mice had accelerated development of CMH, and subsequent associated gait irregularities. These phenotypes were accompanied by upregulation of a cluster of pro-inflammatory genes associated with VSMC maladaptation. Collectively our findings support an essential role for VSMCs as a target for the vasoprotective effects of IGF-1, and suggest that VSMC dysfunction in aging may contribute to the development of CMH.

15 Jan 2024·Heliyon

Extracellular vesicles in cardiomyopathies: A narrative review.

Review

Author: A S Rizzuto ; A Faggiano ; C Macchi ; S Carugo ; C Perrino ; M Ruscica

Extracellular vesicles (EVs) are membrane-bound particles released by all cells under physiological and pathological conditions. EVs constitute a potential tool to unravel cell-specific pathophysiological mechanisms at the root of disease states and retain the potential to act as biomarkers for cardiac diseases. By being able to carry bioactive cargo (such as proteins and miRNAs), EVs harness great potential as accessible "liquid biopsies", given their ability to reflect the state of their cell of origin. Cardiomyopathies encompass a variety of myocardial disorders associated with mechanical, functional and/or electric dysfunction. These diseases exhibit different phenotypes, including inappropriate ventricular hypertrophy, dilatation, scarring, fibro-fatty replacement, dysfunction, and may stem from multiple aetiologies, most often genetic. Thus, the aims of this narrative review are to summarize the current knowledge on EVs and cardiomyopathies (e.g., hypertrophic, dilated and arrhythmogenic), to elucidate the potential role of EVs in the paracrine cell-to-cell communication among cardiac tissue compartments, in aiding the diagnosis of the diverse subtypes of cardiomyopathies in a minimally invasive manner, and finally to address whether certain molecular and phenotypical characteristics of EVs may correlate with cardiomyopathy disease phenotype and severity.

11 Jan 2024·International journal of cardiology

Role of genetics in inflammatory cardiomyopathy.

Article

Author: Paul J Scheel ; Iside Cartella ; Brittney Murray ; Nisha A Gilotra ; Enrico Ammirati

Traditional cardiomyopathy paradigms segregate inflammatory etiologies from those caused by genetic variants. An identified or presumed trigger is implicated in acute myocarditis or chronic inflammatory cardiomyopathy but growing evidence suggests a significant proportion of patients have an underlying cardiomyopathy-associated genetic variant often even when a clear inflammatory trigger is identified. Recognizing a possible genetic contribution to inflammatory cardiomyopathy may have major downstream implications for both the patient and family. The presenting features of myocarditis (i.e. chest pain, arrhythmia, and/or heart failure) may provide insight into diagnostic considerations. One example is isolated cardiac sarcoidosis, a distinct inflammatory cardiomyopathy that carries diagnostic challenges and clinical overlap; genetic testing has increasingly reclassified cases of isolated cardiac sarcoidosis as genetic cardiomyopathy, notably altering management. On the other side, inflammatory presentations of genetic cardiomyopathies are likewise underappreciated and a growing area of investigation. Inflammation plays an important role in the pathogenesis of several familial cardiomyopathies, especially arrhythmogenic phenotypes. Given these clinical scenarios, and the implications on clinical decision making such as initiation of immunosuppression, sudden cardiac death prevention, and family screening, it is important to recognize when genetics may be playing a role.

News (Medical) associated with Cardiomyopathy, Hypertrophic, Familial

25 Oct 2023

·www.biospace.com

Tenaya Therapeutics Receives FDA Fast Track Designation for TN-201

TN-201 Being Developed for the Potential Treatment of MYBPC3-associated HCM Dosing in Phase 1 Clinical Trial Expected to Commence in Q3 2023 SOUTH SAN FRANCISCO, Calif., May 02, 2023 (GLOBE NEWSWIRE) -- Tenaya Therapeutics, Inc. (NASDAQ: TNYA), a clinical-stage biotechnology company with a mission to discover, develop and deliver potentially curative therapies that address the underlying causes of heart disease, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for its gene therapy product candidate, TN-201, being developed for the treatment of Myosin Binding Protein C3 (MYBPC3)-associated hypertrophic cardiomyopathy (HCM). TN-201 is Tenaya’s potential first-in-class adeno-associated virus (AAV)-based investigational gene therapy for the treatment of HCM caused by mutations in the MYBPC3 gene, the most common genetic cause of HCM. TN-201 is designed to deliver a fully functional MYBPC3 gene to restore normal levels of myosin-binding protein, which regulates the contraction and relaxation of the heart muscle. In preclinical studies of MYBPC3 knock-out models, TN-201 has been shown to halt disease progression and demonstrated significant and durable disease reversal and survival benefit after a single dose. “Receipt of Fast Track designation for TN-201 reflects the pressing unmet need among HCM patients whose disease is caused by MYBPC3 genetic mutations,” said Whit Tingley, M.D., Ph.D., Chief Medical Officer of Tenaya. “As we prepare to begin dosing patients later this year, we look forward to continued close collaboration with the FDA under this designation in support of TN-201’s development.” The FDA Fast Track program is designed to facilitate the development and expedite the review of drug candidates intended to treat serious conditions and for which nonclinical data demonstrates the potential to address unmet medical need. Companies with therapies that receive the Fast Track designation from the FDA are eligible for increased communication with the agency and may qualify for accelerated approval and priority review if relevant criteria are met. The goal of the program is to deliver approved treatments to patients with a serious or life-threatening condition as quickly as possible. This designation is not an assurance that regulatory approval will be received. TN-201 also has received Orphan Drug Designation from the FDA and Orphan Medicinal Product designation from the European Commission for the treatment of HCM due to mutations in the MYBPC3 gene. In January 2023, Tenaya announced that the FDA cleared its Investigational New Drug application (IND) for TN-201. Tenaya is initiating the MyPeak-1 Phase 1b clinical trial to assess the safety, tolerability and clinical efficacy of a one-time intravenous infusion of TN-201. The multi-center, open-label study will enroll adults diagnosed with MYBPC3-associated nonobstructive HCM. Tenaya anticipates the first patient will be dosed in this clinical trial during the third quarter of 2023. Tenaya is also conducting two non-interventional studies to support the development of TN-201: a study evaluating seroprevalence to AAV9 antibodies among adults with MYBPC3-associated HCM, and MyClimb, a natural history study of pediatric patients with MYBPC3-associated HCM. About MYBPC3-Associated Hypertrophic Cardiomyopathy Hypertrophic cardiomyopathy (HCM) is the most common inherited cardiac disorder and variants in the Myosin Binding Protein C3 (MYBPC3) gene are the most common genetic cause of HCM. MYBPC3-associated HCM is estimated to account for approximately 20 percent of the overall HCM population and to affect approximately 115,000 patients in the United States alone. MYBPC3-associated HCM is a chronic, progressive condition characterized by left ventricular thickening, hypercontractility, fibrosis, abnormal heart rhythms, cardiac dysfunction and impaired diastolic relaxation. This in turn leads to serious complications including debilitating symptoms such as shortness of breath, fainting and palpitations; heart failure; significant impairment in overall quality of life; and sudden cardiac death in some adults and children. There are currently no approved therapeutics addressing the underlying genetic cause of HCM. TN-201 is Tenaya’s first-in-class adeno-associated virus (AAV)-based gene therapy candidate being developed to treat hypertrophic cardiomyopathy (HCM) due to disease-causing variants in the MYBPC3 gene. About Tenaya Therapeutics Tenaya Therapeutics is a clinical-stage biotechnology company committed to a bold mission: to discover, develop and deliver potentially curative therapies that address the underlying drivers of heart disease. Leveraging its integrated and interrelated Gene Therapy, Cellular Regeneration and Precision Medicine platforms and proprietary core capabilities, the company is advancing a pipeline of novel therapies with diverse treatment modalities for rare genetic cardiovascular disorders and more prevalent heart conditions. Tenaya’s most advanced candidates include TN-201, a gene therapy for MYBPC3-associated hypertrophic cardiomyopathy (HCM), TN-401, a gene therapy for PKP2-associated arrhythmogenic right ventricular cardiomyopathy (ARVC), and TN-301, a small molecule HDAC6 inhibitor being initially developed for heart failure with preserved ejection fraction (HFpEF). Tenaya also has multiple early-stage programs progressing through preclinical development. For more information, visit . Forward Looking Statements This press release contains forward-looking statements as that term is defined in Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. Statements in this press release that are not purely historical are forward-looking statements. Words such as “potential,” “look forward,” “eligible,” “may,” “anticipates,” and similar expressions are intended to identify forward-looking statements. Such forward-looking statements include, among other things, statements regarding the therapeutic potential of TN-201 for MYBPC3-associated HCM patients; the timing for dosing of patients in the Phase 1b clinical trial evaluating TN-201; eligibility for increased communication with the FDA and potential qualification for accelerated approval and priority review for TN-201; and statements by Tenaya’s Chief Medical Officer. The forward-looking statements contained herein are based upon Tenaya’s current expectations and involve assumptions that may never materialize or may prove to be incorrect. These forward-looking statements are neither promises nor guarantees and are subject to a variety of risks and uncertainties, including but not limited to: the timing, scope and likelihood of regulatory filings and approvals; Tenaya’s ability to initiate and complete clinical trials for its product candidates; risks associated with the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics and operating as an early stage company; the potential for any clinical trial results to differ from preclinical, interim, preliminary, topline or expected results; the level of costs associated with Tenaya’s research, development, manufacturing and other activities and the company’s ability to raise any additional funding it will need to continue to pursue its business and product development plans; Tenaya’s ability to successfully operate a manufacturing facility for clinical or commercial supply; Tenaya’s commercialization and marketing capabilities and strategy; the loss of key scientific or management personnel; competition in the industry in which Tenaya operates; Tenaya’s reliance on third parties; Tenaya’s ability to obtain and maintain intellectual property protection for its product candidates; general economic and market conditions; and other risks. Information regarding the foregoing and additional risks may be found in the section entitled “Risk Factors” in documents that Tenaya files from time to time with the Securities and Exchange Commission. These forward-looking statements are made as of the date of this press release, and Tenaya assumes no obligation to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law. Contacts Investors Michelle Corral Vice President, Investor Relations and Corporate Communications Tenaya Therapeutics IR@tenayathera.com Media Wendy Ryan Ten Bridge Communications wendy@tenbridgecommunications.com

Fast TrackPhase 1Orphan DrugGene Therapy

03 May 2023

·www.pharmaceutical-technology.com

Tenaya’s cardiomyopathy gene therapy gets fast track boost

Akosua Mireku @AkosuaMireku1 Close-up photo of a stressed man who is suffering from a chest pain and touching his heart area. Hypertrophic cardiomyopathy is a condition affecting the heart. Credit: damircudic via Getty Images The US Food and Drug Administration (FDA) has granted Tenaya Therapeutics’ gene therapy TN-201 a fast track designation for the treatment of myosin binding protein C3 (MYBPC3)-associated hypertrophic cardiomyopathy (HCM). If successful, TN-201 would be a first-in-class disease-modifying gene therapy for MYBPC3-associated HCM. Free Report How is the Biopharmaceutical industry evolving? 2021 was a year of continued innovation and change in the Biopharmaceutical industry. As the COVID-19 pandemic continues to take its toll on businesses worldwide, it’s time to look for new ways to create value, prepare for the future, and remain competitive in the ever-changing landscape. GlobalData’s expansive report examines the business environment and trends that shape the Biopharmaceutical industry. We highlight the most impactful emerging technologies, as well as the industry, regulatory, and macroeconomic factors that influence growth prospects. Access the report to: Benchmark the impact of major themes on the Biopharmaceutical industry. Gain a deeper "on the ground" perspective through exclusive opinions and analysis from industry respondents. Evaluate the effects of COVID-19 on the sector. Download the full report to understand what to expect and how to align your strategies for success. 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Our services are intended for corporate subscribers and you warrant that the email address submitted is your corporate email address. Thank you. Please check your email to download the Report. In January 2023, Tenaya announced FDA clearance for its Investigational New Drug (IND) application for TN-201. With this recent news, San Francisco-based Tenaya is initiating a multi-centre, open-label Phase Ib clinical trial for the drug. The company predicts that the first patient will be dosed in the MyPeak-1 trial in the third quarter of 2023. Tenaya is also running two supplementary non-interventional studies for the drug. One study is looking at the seroprevalence of AAV9 antibodies among adults with MYBPC3-associated HCM, and the other, MyClimb, is a natural history study of pediatric patients with MYBPC3-associated HCM. TN-201 is an intravenously delivered adeno-associated virus (AAV)- based investigational gene therapy that works by correcting mutations in the MYBPC3 gene, the most common cause of HCM. Mutations in this gene cause a decrease in the maximal force of cardiac contractions. Tenaya’s treatment delivers a functional MYPC3 gene, restoring myosin-binding protein levels to normal. TN-201 is one of four AAV-based gene therapies in Tenaya’s pipeline. In April 2022, Bristol Myers Squibb’s Camzyos (mavacamten) became the first FDA-approved cardiac myosin inhibitor to treat obstructive HCM. Results from a Phase III trial where Cytokinetics is studying its investigational cardiac myosin inhibitor aficamten are expected in December. HCM is the most common inherited cardiac disorder. In the US, MYBPC3-associated HCM accounts for approximately 20% of all HCM cases, affecting approximately 115,000 patients. According to the Mayo Clinic, HCM refers to “a disease in which the heart muscle becomes thickened (hypertrophied)”. Many people with HCM are asymptomatic. HCM can lead to other complications such as atrial fibrillation, blood clots, and strokes. In a May 2 press release , Tenaya’s CMO Dr. Whit Tingley said, “Receipt of fast track designation for TN-201 reflects the pressing unmet need among HCM patients whose disease is caused by MYBPC3 genetic mutations”. Free Report How is the Biopharmaceutical industry evolving? 2021 was a year of continued innovation and change in the Biopharmaceutical industry. As the COVID-19 pandemic continues to take its toll on businesses worldwide, it’s time to look for new ways to create value, prepare for the future, and remain competitive in the ever-changing landscape. GlobalData’s expansive report examines the business environment and trends that shape the Biopharmaceutical industry. 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Phase 3Fast TrackGene TherapyDrug ApprovalPhase 2

16 Feb 2022

·www.biospace.com

Sage and Biogen, BMS Boast Positive Data in Depression, HCM

With positive results from another Phase III study in major depressive disorder, Sage Therapeutics and Biogen are on their way to potential approval of a new therapeutic. This morning, the two companies announced positive topline results from the Phase III CORAL study of zuranolone. Data showed that zuranolone demonstrated a rapid and statistically significant reduction in depressive symptoms after three days over a two-week treatment period. The CORAL study builds on previously released positive zuranolone data, including the Phase III SHORELINE study, which was released late last year. SHORELINE is part of the company’s overall LANDSCAPE clinical program assessing zuranolone. With multiple positive data points, Sage and Biogen, which hooked up on MDD in 2020, believe that zuranolone has three potential real world uses for the treatment of MDD. Data supports zuranolone as a monotherapy, a maintenance treatment and additive therapy. In the CORAL study, 50 mg of zuranolone, an investigational oral neuroactive steroid GABAA receptor positive allosteric modulator, co-initiated with a standard of care antidepressant, was compared to standard-of-care and placebo. The trial demonstrated a mean change from baseline in the 17-item Hamilton Rating Scale for Depression (HAMD-17) after three days for patients who were treated with zuranolone. The CORAL Study supports the potential of zuranolone, when co-initiated with standard of care, to accelerate the benefit of depression treatment compared to treatment with antidepressants alone. Barry Greene, the chief executive officer of Sage Therapeutics, said the data from the CORAL study is clinically meaningful, and when combined with the overall LANDSCAPE data, zuranolone can play an essential role in the treatment of MDD. “The LANDSCAPE data support zuranolone as a monotherapy, and since many people in the previously completed studies were already on maintenance ADTs (antidepressants), we believe our data also support zuranolone as additive therapy. The CORAL Study further supports the use of zuranolone to accelerate the benefit of conventional ADTs in treating MDD with a well-tolerated safety profile,” Greene said in a statement. “Including the CORAL Study, zuranolone now has six positive clinical studies, and we remain on track to start the rolling submission for a New Drug Application in MDD early this year with completion targeted for the second half of 2022.” Sage Chief Innovation Officer Jeff Jonas added that studies across the LANDSCAPE program have generated data supporting the safety and efficacy of zuranolone. “With the data from the CORAL Study in hand, we now have positive data on the safety and efficacy of zuranolone when co-initiated with a standard of care ADT. Collectively, clinical trials with zuranolone suggest the potential for broad use in the treatment of MDD, as a monotherapy, adjunctive use or when co-initiated with ADT, suggesting that there is ample opportunity for zuranolone, if approved, to be considered as a treatment option for the full 6.8 million population who are initiating a new ADT,” Jonas said in an email sent to BioSpace. Priya Singhal, head of global safety and regulatory sciences and interim head of R&D at Biogen, noted that zuranolone co-initiated with standard of care may offer more rapid relief from depressive symptoms than current standard of care taken alone. “Based on the collective results observed across the LANDSCAPE clinical development program, we believe that zuranolone has the potential to offer a new clinically meaningful treatment option for people with major depressive disorder,” Singhal said in a statement. In the CORAL study, zuranolone continued to demonstrate a positive safety profile. The 50 mg dose alongside standard of care antidepressants was generally well-tolerated by patients. Sage and Biogen were not the only companies to announce positive Phase III results this morning. Bristol Myers Squibb reported positive data from its Phase III VALOR-HCM program assessing mavacamten in adults with symptomatic obstructive hypertrophic cardiomyopathy (obstructive HCM) who are eligible for septal reduction therapy. Full data from the Phase III study will be shared at the American College of Cardiology 71st Annual Scientific Session & Expo in April. With the positive data in hand, BMS intends to approach global regulatory agencies to support the potential approval of mavacamten, an allosteric modulator of cardiac myosin. The drug, which BMS gained in its 2020 $13.1 billion acquisition of Myokardia, could be approved for the treatment of symptomatic, obstructive hypertrophic cardiomyopathy (HCM). Obstructive HCM is a progressive disease that thickens the heart walls and makes it harder to expand normally and fill with blood. Mavacamten is a selective cardiac myosin inhibitor that targets the underlying pathophysiology of obstructive HCM. The experimental drug has been shown to reduce cardiac muscle contractility by inhibiting excessive myosin-actin cross-bridge formation, resulting in hypercontractility, left ventricular hypertrophy, and reduced compliance. The company has an April 28 PDUFA date for that indication. “We are encouraged by the findings from this important study, which add to the growing body of clinical evidence that supports the promise of mavacamten for patients living with obstructive HCM,” Roland Chen, senior vice president of cardiovascular development at BMS said in a statement.

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