Hypertrophic Cardiomyopathy - Pipeline Insight, 2024

This “Hypertrophic Cardiomyopathy- Pipeline Insight, 2024” report provides comprehensive insights about 10+ companies and 10+ pipeline drugs in Hypertrophic Cardiomyopathy pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

Geography Covered

• Global coverage

Hypertrophic Cardiomyopathy: Understanding

Hypertrophic Cardiomyopathy: Overview

Hypertrophic Cardiomyopathy (HCM) is a genetic disorder caused by the caused by a change or mutation in one or more genes and is mostly passed on through families. It is a heart muscle disease in which the muscle is thickened (hypertrophic). This thickening typically occurs in the lower left chamber of the heart, called the left ventricle. Thickening of the heart muscle can occur at the septum (muscular wall that separates the left and right side of the heart), the posterior wall or free wall (outside wall of the left ventricle), the apex (the bottom of the heart) or throughout the entire left ventricle. Most people with HCM have no symptoms or feel stable throughout their life. However, others do have symptoms like breathlessness, chest pain, palpitations, light-headedness and/or fainting. Some people find that their symptoms worsen in later life. This may be because their heart muscle has slowly become stiffer, making it harder for the heart to pump. There are two types of HCM including Obstructive, which is the most common type, hypertrophic obstructive cardiomyopathy (HOCM) means the wall (septum) between the left ventricle and right ventricle thickens. This can block (obstruct) blood flow from the left ventricle to the aorta (the body's main artery). The other type include non-obstructive HCM, in which the heart muscle thickens but does not block blood flow. HCM can lead to other heart conditions, such as heart failure and heart arrhythmias and in very rare cases, mostly associated with vigorous physical activity; it can cause a cardiac arrest. The diagnosis of HCM is based on family history, medical history, physical exam,and cardiac testing. To further define the muscle abnormality and to determine if there is scar in the heart a cardiac magnetic resonance imaging (MRI) may be recommended, while other tests such as Blood tests, electrocardiogram (EKG), exercise stress echo test and cardiac catheterization may also be recommended. The treatment for individuals with HCM is different depending on the person and is based on a number of features including the presence of outflow tract obstruction (blood isn’t able to leave the left ventricle because of the thickened muscle), the function of the heart (how well it is squeezing), the presence of any symptoms, the age and activity level of the patient, the presence of abnormal heart rhythms and the family history. Medication prescribed to reduce the amount of work the heart has to do and to relieve the symptoms include beta-blockers, calcium channel blockers, and anti-arrhythmics.

Hypertrophic Cardiomyopathy- Pipeline Insight, 2024 report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Hypertrophic Cardiomyopathy pipeline landscape is provided which includes the disease overview and Hypertrophic Cardiomyopathy treatment guidelines. The assessment part of the report embraces, in depth Hypertrophic Cardiomyopathy commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Hypertrophic Cardiomyopathy collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

Report Highlights

• The companies and academics are working to assess challenges and seek opportunities that could influence Hypertrophic Cardiomyopathy R&D. The therapies under development are focused on novel approaches to treat/improve hypertrophic cardiomyopathy.

Hypertrophic Cardiomyopathy: Emerging Drugs Chapters

This segment of the Hypertrophic Cardiomyopathy report encloses its detailed analysis of various drugs in different stages of clinical development, including phase III, II, I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.

Hypertrophic Cardiomyopathy: Emerging Drugs

Aficamten: CytokineticsAficamten is an investigational, oral, small molecule cardiac myosin inhibitor. It is designed to reduce the hypercontractility associated with HCM by blocking myosin from pulling, resulting in less contraction, or fewer hands on the rope. The drug is in the study, SEQUOIA-HCM (Safety, Efficacy, and Quantitative Understanding of Obstruction Impact of Aficamten in HCM), a Phase III randomized, placebo-controlled, double blind, multi-center clinical trial designed to evaluate aficamten in patients with symptomatic obstructive HCM on background medical therapy for 24 weeks.

Ninerafaxstat: ImbriaNinerafaxstat is a novel, investigational cardiac mitotrope in development for the treatment of cardiovascular disease. As a partial fatty acid oxidation (pFOX) inhibitor, ninerafaxstat is designed to shift myocardial substrate utilization in favor of glucose oxidation to generate more ATP per unit of oxygen consumed thereby increasing myocardial metabolic efficiency. The drug is currently being investigated in three Phase II proof-of-concept clinical trials in patients with non-obstructive HCM. IMPROVE-HCM Phase II clinical trial is a randomized, double-blind, placebo-controlled clinical trial investigating the safety and efficacy of ninerafaxstat in patients with non-obstructive HCM.

TN-201: Tenaya TherapeuticsTN-201, an adeno-associated virus (AAV)-based gene therapy designed to treat adults and children with HCM due to MYBPC3 gene mutations, the most prevalent form of genetic HCM. TN-201 is intended to address the underlying cause of disease by delivering a fully functional MYBPC3 gene to restore normal levels of MYBPC3 protein with the hope of potentially halting disease progression and reversing the course of genetic HCM after a single treatment. Recently in 2023, the company has received clearance of the Investigational New Drug (IND) application from the US Food and Drug Administration (FDA) to begin clinical testing of TN-201. The company initiated Phase Ib clinical trial enrolling symptomatic adults who have been diagnosed with MYBPC3-associated non-obstructive HCM and expect to begin patient dosing in the third quarter of 2023.

Hypertrophic Cardiomyopathy: Therapeutic Assessment

This segment of the report provides insights about the different Hypertrophic Cardiomyopathy drugs segregated based on following parameters that define the scope of the report, such as:

Major Players in Hypertrophic Cardiomyopathy

There are approx. 10+ key companies which are developing the therapies for Hypertrophic Cardiomyopathy. The companies which have their Hypertrophic Cardiomyopathy drug candidates in the most advanced stage, i.e. phase III include, Cytokinetics.

Phases

This report covers around 10+ products under different phases of clinical development like

• Late stage products (Phase III)
• Mid-stage products (Phase II)
• Early-stage product (Phase I) along with the details of
• Pre-clinical and Discovery stage candidates
• Discontinued & Inactive candidates

Route of Administration

Hypertrophic Cardiomyopathy pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as

• Intra-articular
• Intraocular
• Intrathecal
• Intravenous
• Ophthalmic
• Oral
• Parenteral
• Subcutaneous
• Topical
• Transdermal

Molecule Type

Products have been categorized under various Molecule types such as

• Oligonucleotide
• Peptide
• Small molecule

Product Type

Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.

Hypertrophic Cardiomyopathy: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase III, II, I, preclinical and discovery stage. It also analyses Hypertrophic Cardiomyopathy therapeutic drugs key players involved in developing key drugs.

Pipeline Development Activities

The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Hypertrophic Cardiomyopathy drugs.

Hypertrophic Cardiomyopathy: Report Insights

• Hypertrophic Cardiomyopathy Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

Hypertrophic Cardiomyopathy: Report Assessment

• Pipeline Product Profiles
• Therapeutic Assessment
• Pipeline Assessment
• Inactive drugs assessment
• Unmet Needs

Key Questions

Current Treatment Scenario and Emerging Therapies:

• How many companies are developing Hypertrophic Cardiomyopathy drugs?
• How many Hypertrophic Cardiomyopathy drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Hypertrophic Cardiomyopathy?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Hypertrophic Cardiomyopathy therapeutics?
• What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies?
• What are the clinical studies going on for Hypertrophic Cardiomyopathy and their status?
• What are the key designations that have been granted to the emerging drugs?

Key Players

• Cytokinetics
• Imbria
• Bristol-Myers Squibb
• Tenaya Therapeutics
• Lexeo Therapeutics, Inc.
• BioMarin

Key Products

• Aficamten
• Ninerafaxstat
• MYK-224
• TN-201
• LX2022
• BMN 293

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