Juvena Therapeutics Begins First Human Trial of JUV-161

Juvena Therapeutics, a biotechnology company based in Redwood City, California, has initiated a clinical trial for its pioneering drug, JUV-161. This innovative therapy is designed to enhance muscle regeneration and holds promise for treating conditions like Myotonic Dystrophy Type 1 (DM1) and Sarcopenia. DM1 is a progressive genetic disorder that leads to muscle degeneration and other systemic issues, affecting millions worldwide and representing a significant unmet medical need. JUV-161, classified as a first-in-class muscle regenerating endocrine therapy, is developed using Juvena's proprietary AI and stem cell-based drug discovery platform, JuvNET.

JUV-161 stands out due to its unique mechanism as a fusion protein with pro-myogenic, insulin-resistance modulating, and anti-inflammatory properties. The therapy aims to restore critical protein signaling in diseased muscle tissues to youthful, healthy levels. By doing so, it seeks to improve muscle health and the overall quality of life for individuals affected by conditions that involve muscle loss. In 2024, JUV-161 received the FDA Orphan Disease Drug designation for its potential in treating DM1.

The Myotonic Dystrophy Foundation has expressed optimism about this development, highlighting JUV-161's potential to address muscle regeneration and metabolism, thus improving muscle function and health. This positive reception from the foundation underscores the potential impact of Juvena’s approach to developing a novel therapy for DM1 patients.

Juvena’s clinical trial, which has received approval from Australia's Human Research Ethics Committees, involves a first-in-human, single-ascending dose study. This trial focuses on evaluating the safety, tolerability, pharmacokinetics, and pharmacodynamics of JUV-161 in healthy adult volunteers. By starting with healthy participants, Juvena aims to gather essential data that will support the expansion of this therapy to other muscle-related diseases. Juvena plans to submit an Investigational New Drug application and start Phase II studies for both DM1 and Sarcopenia in 2026.

JUV-161's development leverages the JuvNET platform, which integrates quantitative proteomics and AI to analyze human stem cell secretome biology. This approach allows Juvena to discover life-changing regenerative and metabolic therapies. JUV-161, sometimes referred to as a potential "insulin for muscle," functions by modulating muscle regeneration and metabolism. It has shown efficacy in enhancing muscle fiber formation, countering muscle atrophy, and improving muscle strength and metabolism in various preclinical disease models. The therapy works by activating and restoring the AKT signaling pathway, which is crucial for cell survival, growth, and metabolism but often disrupted in muscle-wasting diseases.

Juvena Therapeutics continues to utilize JuvNET to advance its pipeline of biologics for myopathies and metabolic diseases. This platform has not only identified JUV-161 but also other promising candidates such as JUV-112, targeting obesity without suppressing appetite. Indeed, Juvena’s approach to biologic drug discovery holds promise for addressing numerous metabolic diseases and restoring tissue health across various organ systems.