Juvena Therapeutics Initiates First-in-Human Trial of JUV-161 for Muscle Regeneration in Myotonic Dystrophy
核心洞察
Juvena Therapeutics (搜索) has begun enrolling participants in the first human clinical trial of JUV-161, a novel fusion protein designed to enhance muscle regeneration for treating Myotonic Dystrophy Type 1 (搜索) and sarcopenia (搜索).
JUV-161, described as "insulin for muscle," works by restoring AKT (搜索) signaling pathways that regulate muscle growth and metabolism, offering a unique approach compared to existing RNA-targeting or gene therapy strategies.
The therapy was discovered using Juvena's proprietary JuvNET platform, which combines AI and stem cell secretome biology to identify therapeutic proteins with regenerative potential.
Juvena Therapeutics (搜索) has launched its first-in-human clinical trial for JUV-161, a first-in-class fusion protein designed to regenerate muscle tissue in patients with Myotonic Dystrophy Type 1 (搜索) (DM1 (搜索)) and other muscle-wasting conditions. The clinical-stage biotechnology company, which specializes in tissue restorative biologics, is now enrolling healthy adult volunteers in Australia after receiving approval from the country's Human Research Ethics Committees (HREC).
JUV-161, which received FDA Orphan Disease Drug designation for DM1 (搜索) in 2024, represents a novel approach to treating muscle degeneration by enhancing muscle regeneration through pro-myogenic, insulin-resistance modulating, and anti-inflammatory properties.
A Novel Approach to Muscle Regeneration
Unlike other therapeutic strategies for DM1 (搜索) that focus on RNA-targeting, antisense technologies, or gene therapy knockdown approaches, JUV-161 functions as a natural endocrine protein therapy. The long-lasting biologic acts by activating and restoring AKT (搜索) signaling, a critical pathway that regulates cell survival, growth, and metabolism, which is often dysregulated in muscle-wasting diseases and during the natural aging process.
"JUV-161 is a natural, endocrine protein therapy that will help restore critical protein signaling in diseased muscle to more youthful, healthy levels, so that we can restore muscle health and ultimately restore the quality of life of individuals living with many indications where muscle loss reduces the quality of life or lifespan," explained Dr. Hanadie Yousef, CEO and co-founder of Juvena Therapeutics (搜索).
The investigational therapy is based on an engineered form of human Insulin-like Growth Factor 2 (IGF2) protein developed for subcutaneous injection. Often referred to as a potential "insulin for muscle," JUV-161 has demonstrated impressive results in preclinical studies, including:
- Restoration of muscle fiber formation
- Reversal of muscle atrophy
- Enhancement of muscle strength and endurance
- Improvement of muscle metabolism
These effects have been observed across multiple preclinical disease models, ranging from dystrophies to sarcopenia (搜索) resulting from aging, obesity, diabetes, and inflammation.
Addressing a Significant Unmet Need
Myotonic Dystrophy Type 1 (搜索) is a progressive, multi-systemic, inherited autosomal dominant genetic disease that affects approximately one in 2,100 people globally, representing over 3.6 million individuals. The condition causes muscle degeneration, weakness, myotonia, heart abnormalities, cataracts, and insulin resistance, among other symptoms. Currently, there are no FDA-approved therapeutics for DM1 (搜索).
Dr. Andy Rohrwasser, Chief Science Officer at the Myotonic Dystrophy Foundation (搜索), expressed enthusiasm about the trial: "This milestone is great news for myotonic dystrophy (DM) families because JUV-161 targets muscle regeneration and metabolism to improve overall muscle function and health and has the potential to improve and restore muscle."
Clinical Trial Design and Future Plans
The current single-ascending dose study is evaluating the safety, tolerability, pharmacokinetics, and pharmacodynamics of JUV-161 in healthy adult volunteers. This trial is part of Juvena's global clinical "pipeline in a product" strategy, which aims to expand to multiple potential muscle disease indications.
Looking ahead, Juvena expects to submit an Investigational New Drug (IND) application for JUV-161 next year and initiate two Phase II studies in people living with congenital DM1 (搜索) and sarcopenia (搜索).
"With JUV-161's broad applicability, we're really excited about the potential to develop and commercialize it towards many indications in which it can restore muscle health and ultimately lead to restoring the quality of life for individuals living with myotonic dystrophy as well as a broad range of sarcopenic conditions associated with aging, obesity, or even diabetes," Dr. Yousef noted.
AI-Driven Drug Discovery Platform
JUV-161 is the lead therapeutic candidate discovered using Juvena's proprietary JuvNET platform, which combines artificial intelligence with stem cell secretome biology to identify and develop novel biologics. The platform screens Juvena's proprietary protein library of human stem-cell secreted proteins using unbiased quantitative proteomics, multi-omics, and high-content imaging data, integrated with various AI technologies.
"With JUV-161, we're thrilled to bring to the clinic the first asset emerging from our JuvNET platform which deconvolutes human stem cell secretome biology with a combination of quantitative proteomics and AI," said Dr. Jeremy O'Connell, Chief Scientific Officer and co-founder of Juvena Therapeutics (搜索). "As we continue to unlock the therapeutic potential of a proven yet understudied source of biologics, Juvena is poised to identify life-changing muscle regenerative and pro-metabolic therapies in high demand across disease categories."
The company's pipeline extends beyond JUV-161 to include other biologics for myopathies and metabolic diseases. Among these is JUV-112, an obesity-targeting biologic that induces fat and weight loss through a non-appetite suppressive mechanism that spares muscle and increases energy expenditure. To date, Juvena's platform has identified over 50 secreted protein hits across multiple metabolic diseases and organ systems, including candidates with potential benefits for pulmonary and hepatic fibrosis and osteoarthritis.
Juvena Therapeutics (搜索) raised $41 million in a Series A funding round in 2022, co-led by Mubadala Capital and Horizons Ventures, bringing its total funding to $50 million. This financial backing supports the company's mission to translate stem-cell secreted proteins into engineered biologics for chronic muscle and metabolic diseases.