Natural endocrine therapy is designed to help restore key protein signaling in diseased muscle to ‘more youthful, healthy levels.’
Longevity biotech Juvena Therapeutics has initiated its first human clinical trial for its lead biologic therapy designed to regenerate muscle tissue. Described as “insulin for muscle”, JUV-161 is a muscle regenerating endocrine therapy that has demonstrated efficacy in preclinical models by promoting muscle fiber formation, reversing muscle atrophy, enhancing strength and endurance and improving metabolic health.
JUV-161 is initially being explored for Myotonic Dystrophy Type 1 (DM1), a debilitating inherited condition with no currently approved treatments that leads to progressive muscle degeneration, myotonia, cardiac complications, cataracts and insulin resistance. Last year, the therapy received Orphan Drug designation from the FDA for treatment of DM1.
The first-in-human trial, now enrolling participants in Australia, is a single-ascending dose study assessing safety, tolerability, pharmacokinetics and pharmacodynamics in healthy adult volunteers. The trial is intended to support Juvena’s broader “pipeline in a product” strategy, which aims to develop JUV-161 across multiple muscle disease indications. The company says it plans to file an Investigational New Drug application next year to advance into Phase II studies in patients with congenital DM1 and sarcopenia.
“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 – this includes for individuals living with myotonic dystrophy as well as a broad range of sarcopenic conditions associated with aging, obesity, or even diabetes,” Juvena CEO Dr Hanadie Yousef told us. “Because of its natural mechanism of action, we’ve shown and validated that it has efficacy across multiple preclinical myopathy models, ranging from natural age-related muscle-wasting in a sarcopenia model through to glucocorticoid induced atrophy.”
Based on an engineered form of the human Insulin-like Growth Factor 2 (IGF2), JUV-161’s approach centers on restoring AKT signaling, a key pathway that regulates muscle growth, survival and metabolism but is frequently impaired in both disease and aging.
“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,” said Yousef. “JUV-161 is a unique tissue restorative therapy that is different from other anti-sense, RNA-targeting, or gene therapy knockdown approaches currently being taken for DM1.”
Juvena’s drug discovery platform, JuvNET, integrates AI with stem-cell secretome biology to identify and engineer therapeutic proteins. It uses a proprietary protein library and combines quantitative proteomics, multi-omics data and high-content imaging to uncover regenerative protein candidates.
The company’s approach has produced a growing pipeline of biologics aimed at treating chronic muscle and metabolic disorders, including JUV-112, an obesity-targeting candidate that induces weight loss through increased energy expenditure rather than appetite suppression. To date, Juvena says it has identified over 50 biologically active protein hits with potential therapeutic benefit across organ systems affected by conditions such as pulmonary fibrosis, hepatic fibrosis and osteoarthritis.
“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,” said Juvena’s Chief Scientific Officer Dr Jeremy O’Connell.
In 2022, Juvena raised $41 million in a Series A funding round co-led by Mubadala Capital and Horizons Ventures, bringing its total funding to $50 million.
