MARKETVUE® REPORT: Arrhythmogenic Cardiomyopathy Gene Therapy Research Gains Momentum

News provided by

Nov 07, 2023, 10:19 ET

~70% of physicians surveyed by REACH Market Research report there is an extremely high unmet medical need for novel treatments for ACM.

NEWTON, Mass., Nov. 7, 2023 /PRNewswire/ -- 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 REACH Market Research's 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."

To access REACH's MarketVue^® Report on ACM, visit https://reachmr.com or contact us at [email protected].

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.

About MarketVue^®

MarketVue^® reports are a rare disease focused, fresh alternative to traditionally long and outdated market research reports. MarketVue^® reports cover rare disease epidemiology and key market dynamics based on research from key opinion leader interviews, physician surveys, and secondary data.

About REACH Market Research

REACH is an independent pharmaceutical market research company focused on rare and niche diseases. With decades of experience in pharmaceutical market research and life sciences consulting, REACH fills an important gap in the market – accessible market research solutions for rare and niche diseases.

SOURCE REACH Market Research