Dilated Cardiomyopathy (DCM) poses a significant healthcare burden, affecting millions worldwide. The progressive weakening of the heart muscle leads to impaired pumping function, resulting in heart failure and, in severe cases, necessitating heart transplantation. In this blog post, we'll explore the current landscape of pharmaceutical assets in development for DCM, shedding light on both promising industry assets and academic-run clinical trials.
Current Treatment Approaches
Managing DCM involves a combination of lifestyle modifications, medications, and, in advanced cases, device therapy or heart transplantation. Common drugs used in the treatment of DCM include angiotensin-converting enzyme (ACE) inhibitors, beta-blockers, and angiotensin II receptor blockers (ARBs). These medications aim to alleviate symptoms, slow disease progression, and improve the overall quality of life for patients.
Next Gen Therapies to Watch
Industry Assets
Clinical development is most advanced for two novel approaches, focusing on myosin, a protein involved in muscle contraction, and beta-1-adrenoceptors, a crucial GPCR for cardiac function. Assets to watch are:
Danicamtiv is a small molecule orignially developed by MyoKardia, which was acquired by Bristol Myers Squibb for $13.1bn in 2020. The asset is an allosteric activator of myosin to increase contractility in genetic DCM. Currently, BMS explores the drug's efficacy and safety in a Phase II trial.
BC 007 is a DNA aptamer developed by Berlin Cures. The drug binds to and eliminates pathogenic autoantibodies directed against the beta-1-adrenoceptor. The neutralisation of these autoantibodies helps the GPCR to interact with adrenalin, its natural ligand. Berlin Cures investigated the asset in a Phase II trial completed end of 2022, and currently runs a Phase II study in Long COVID.
Preclinical development mostly happens in the gene therapy space, repairing various defective genes causative for dilated cardiomyopathies. Some interesting assets and platform companies to watch are:
AVB 401 is a BAG3-targeting gene therapy developed by Solid Biosciences. The asset is investigated for BAG3-mediated DCM, previously developed by AavantiBio, which was acquired by Solid Biosciences in 2022.
DINA004/5/6 are gene therapy approaches developed by DiNAQOR AG. The company uses its proprietary Engineered Heart Tissue technology and Loco-Regional Perfusion system to deliver gene therapies directly into the heart tissue and other solid organs. This approach limits systemic exposure, off-target effects, and allows for repeated dosing.
DWORF is a AAV-based gene therapy investigated by Tenaya Therapeutics. The therapy is designed to deliver the DWORF gene to DCM patients, which is a muscle specific micropeptide first discovered by Tenaya's co-founder Eric Olson. The target is promising in various heart failure syndromes, acting on the SERCA2a pathway.
REN 001 is an AAV9-based gene therapy orignially developed by Renovacor but acquired by Rocket Pharmaceuticals in 2022. The asset is investigated for BAG3-associated familial DCM, replacing the defective BAG3 gene.
PHL 001 from PHLOX Therapeutics is a gene therapy targeting lamin gene defects resulting in DCM. Cardiac laminopathies are aggressive forms of cardiomyopathies with a high unmet need.
Academic Trials
Moreover, several academic centers are currently running trials in DCM settings, trying out distinct approaches to reduce the immense healthcare burden. Here, stem cell therapies, gene therapies delivering TERT, and rejuvenating approaches using the secretome of cardiovascular progenitor cells are highlighted trials to keep an eye on. A summary of these trials is shown here:
Landscape Summary
The landscape of pharmaceutical assets in development for DCM is dynamic and promising. While current treatments aim to manage symptoms and slow disease progression, the emergence of myosin modulators, gene therapies, RNA therapeutics, and cell therapies signals a shift toward more targeted and personalized interventions.
As these innovative therapies progress through clinical trials, there is hope for a future where DCM patients can benefit from treatments that not only alleviate symptoms but also address the root causes of the disease. Collaborations between researchers, pharmaceutical companies, and healthcare professionals are crucial in advancing the field and bringing these potential breakthroughs to patients in need. The journey toward more effective and personalized treatments for DCM is underway, marking a hopeful chapter in the ongoing fight against cardiovascular diseases.
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