The MarketVue®: Arrhythmogenic Cardiomyopathy market landscape report combines primary (KOL interviews) and secondary market research to empower strategic decision-making and provide a complete view of the market.
Every MarketVue® includes a disease overview, epidemiology (US and EU5), current treatment, unmet needs, pipeline and access and reimbursement chapter.
Topics covered in this report:
- Disease overview: Review the disease pathophysiology and potential druggable targets
- Epidemiology: Understand prevalence, diagnosed and drug-treated prevalence of the population and key market segments
- Current treatment: Understand the treatment decision tree and strengths and weaknesses of current on-label and off-label treatment
- Unmet needs: Identify opportunities to address treatment or disease management gaps
- Pipeline analysis: Compare current and emerging therapy clinical development strategy; their performance on efficacy, safety, and delivery metrics; and their potential to address unmet needs
- Value and access: Review the evidence needed to assess and communicate value to key stakeholders (e.g., providers, payers, regulators) and learn what competitors have done or are doing
Methodology:
Research for the MarketVue®: Arrhythmogenic Cardiomyopathy report is supported by 13 qualitative interviews with key opinion leaders and secondary research.
Geographies covered:
United States plus epidemiology for EU5 (France, Germany, Italy, Spain, United Kingdom).
Key companies mentioned:
- Rocket Pharmaceuticals
- Lexeo Therapeutics
- Tenaya Therapeutics
- StrideBio / Ginko Bioworks
- Ginkgo Bioworks
- BioMarin Pharmaceuticals
Key drugs mentioned:
- Beta blockers
- Sotalol (Betapace, Sorine)
- ACE inhibitors
- RP-A601
- TN-401
- STRX-330
- LX2020
- BMN365
- Spironolactone
- Flecainide (Tambocor)
Key takeaways from the report:
Arrhythmogenic Cardiomyopathy (ACM) is a rare hereditary cardiac disorder characterized by progressive replacement of myocardial tissue with fibrofatty deposits, leading to life-threatening arrhythmias and cardiac dysfunction.
ACM patients typically receive treatment with beta blockers and anti-arrhythmic drugs to manage symptoms and arrhythmias. However, according to this MarketVue® assessment, a striking 50% of patients face treatment failure, necessitating invasive procedures like implantable cardioverter-defibrillators (ICDs), catheter ablation, or even heart transplants which coincide with their own set of challenges.
The urgency for innovative treatments targeting the underlying disease pathophysiology to slow down or halt the progression of the disease, ultimately reducing the risk of life-threatening cardiac events is clear, however, the disease exhibits incomplete penetrance, making risk prediction and patient stratification challenging.
Cardiologist, U.S.: '[There is] the inability to actually prevent the underlying progression since there are no medications that will prevent progression or reduce the cause of arrhythmias, so even if they've got an ICD, they may get shocked from it or have poor quality of life.'
The ACM pipeline remains sparse, with two gene therapies entering early-stage clinical development targeting the PKP2 gene, the most prevalent ACM mutation:
- Rocket Pharmaceuticals’ AAVrh74-based gene therapy is in Phase 1
- Lexeo Therapeutics is on track to initiate a Phase 1/2 trial for their AAVrh10-based gene therapy following IND clearance in August 2023
These developments offer new hope to ACM patients, however, given the disease can be caused by genetic mutations outside of PKP2, these treatments would target less than half of diagnosed prevalent patients.
Table of Contents
1. DISEASE OVERVIEW
- A life-threatening, progressive inherited genetic condition impacting cardiac function
- Table 1.1. Most common ACM mutations
- Figure 1.1. Pathogenesis of ACM
2. EPIDEMIOLOGY & PATIENT POPULATIONS
- Prevalence of ACM
- Table 2.1. ACM diagnosed prevalence in the United States, 2023
- Figure 2.1. Prevalence estimates of ACM in the medical literature from 1994 - 2022
- Disease definition
- Figure 2.2. Diagnosed prevalent cases of PKP2+ ACM by region
- Table 2.2. Diagnosed prevalent ACM cases in the US and EU5
- Table 2.3 Average prevalence of ACM mutation type
3. DIAGNOSIS AND CURRENT TREATMENT
- Diagnosis overview
- Figure 3.1. Diagnostic pathway for ARVC patients
- Figure 3.2. Physician-reported average estimate of ACM patients that receive a diagnosis
- ACM genetic testing dynamics
- Figure 3.3. KOL estimate of their ACM patients who are PKP2+ with genetic testing
- Figure 3.4. Physician-reported barriers to genetic testing
- ACM treatment involves pharmacological and device interventions
- Figure 3.5. Treatment algorithm for management of ACM
- Figure 3.6. ACM treatment goals
- ACM treatment dynamics
- Figure 3.7. Current treatment patient share
- Table 3.1. Current ACM treatment outcomes
- Table 3.2. Upsides and downsides of current interventions used for ACM
- Key treatment dynamics that shape disease management and novel therapy use in ACM
- Table 3.3. Must-know ACM market dynamics
- Key treatment dynamics that shape disease management and drug use in ACM
- Figure 3.8. Important dynamics of ACM market evolution1
4. UNMET NEED
- Overview
- Figure 4.1. Top unmet needs in ACM
- Figure 4.2. Physician-reported perspectives on ACM treatment
- ACM disease burden and unmet needs
- Figure 4.3. Physician-reported disease burden for ACM patients and families
- Table 4.1. Most important attributes for new ACM treatment
- Physician perspectives on unmet needs in ACM
5. PIPELINE ANALYSIS
- Overview
- Table 5.1 Summary of the RP-A601 gene therapy trial
- ACM therapies in early pipeline follow similar framework
- Table 5.2. Comparison of preclinical ACM gene therapies
- Investigator-sponsored drug-based approaches to ACM
- Figure 5.1. Estimated proportion of population with positive AAV titers
- Table 5.3. Investigator-sponsored ACM trials
- Gene therapy in ACM
- Table 5.4. Physician-reported perspectives on ACM treatment and gene therapies
- Table 5.5. Attributes that will drive uptake for emerging ACM therapies
- Future considerations for gene therapy
- Table 5.6. Opportunities and threats for emerging AAV-based ACM gene therapies
- Physician perspectives on ACM gene therapies
6. VALUE & ACCESS
- Overview
- Table 6.1. Comparison of gene therapy pricing, U.S.
- Figure 6.1. Key reimbursement and access considerations for emerging therapies in ACM
- Key insights from the ACM market
- Table 6.2. Innovative payer coverage gene therapy insights
7. METHODOLOGY
- Primary market research approach
- Epidemiology methodology
- Table 7.1. ACM prevalence and population size references
- PKP2 mutation rate methodology
- Table 7.2. Mutation rate references
- Table 7.3. Pharmaceutical company ACM and PKP2 prevalence estimates
Samples
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Companies Mentioned
- Rocket Pharmaceuticals
- Lexeo Therapeutics
- Tenaya Therapeutics
- StrideBio / Ginko Bioworks
- Ginkgo Bioworks
- BioMarin Pharmaceuticals
