Juvena Therapeutics Announces Initiation of First-in-Human Clinical Trial of JUV-161

• JUV-161 is a first-in-class muscle regenerating biologic that will address myopathies starting with Myotonic Dystrophy Type 1
• JUV-161 was discovered using Juvena’s proprietary JuvNET platform, an AI and stem cell secretome-based drug discovery and development platform

/EIN News/ -- REDWOOD CITY, Calif., May 19, 2025 (GLOBE NEWSWIRE) -- Juvena Therapeutics, Inc. (“Juvena”), a clinical-stage biotechnology company pioneering tissue restorative biologics for chronic muscle and metabolic diseases, announced today that it is enrolling participants into its clinical trial evaluating JUV-161, a first-in-class fusion protein that enhances muscle regeneration through its pro-myogenic, insulin-resistance modulating, and anti-inflammatory properties. JUV-161 is a muscle regenerating endocrine therapy being investigated for the treatment of Myotonic Dystrophy Type 1 (DM1) and Sarcopenia. JUV-161 received FDA Orphan Disease Drug designation for DM1 in 2024.

“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 CEO and co-founder of Juvena Therapeutics, Dr. Hanadie 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.”

“As the world’s largest myotonic dystrophy patient advocacy organization, founded by families impacted by DM and supporting research toward treatments and cures, the Myotonic Dystrophy Foundation is super excited about Juvena Therapeutics’ lead candidate, JUV-161, entering the clinical testing phase with a truly novel approach,” said Myotonic Dystrophy Foundation, Chief Science Officer, Dr. Andy Rohrwasser. “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.”

“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. “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.”

About the Study
Juvena has received approval from Australia’s Human Research Ethics Committees (HREC) to commence the study and is now enrolling participants. The first-in-human, single-ascending dose study is evaluating the safety, tolerability and pharmacokinetics and pharmacodynamics of JUV-161 in healthy adult volunteers.

The trial is part of the company’s global clinical “pipeline in a product” strategy. Testing in healthy volunteers will enable Juvena to expand to multiple potential muscle disease indications in the near future. Next year, Juvena expects to submit an Investigational New Drug (IND) application for JUV-161 and to initiate two Phase II studies in people living with congenital DM1 and sarcopenia. To learn more about the study visit www.clinicaltrials.gov.

About DM1
Today, there are no FDA-approved therapeutics for DM1, a progressive, multi-systemic, inherited autosomal dominant genetic disease that causes muscle degeneration, with weakness and shrinkage of the muscle tissue, myotonia, heart abnormalities, cataracts and insulin resistance, amongst other symptoms. An estimated one in 2,100 people, or over 3.6 million individuals globally, have DM1, representing a major unmet medical need.¹²³

About JUV-161
Often referred to as a potential “insulin for muscle,” JUV-161 modulates muscle regeneration and metabolism, enhancing overall muscle health. The fusion protein has demonstrated a unique ability to restore muscle fiber formation, counter muscle atrophy, enhance muscle strength and endurance, and improve muscle metabolism in multiple preclinical disease models ranging from models of dystrophies to models of sarcopenia resulting from aging, obesity, diabetes, and inflammation.

The long-lasting investigational biologic acts by activating and restoring AKT signaling, a pathway that regulates cell survival, growth, and metabolism, and is often dysregulated in muscle-wasting diseases and as people naturally age. Identified by Juvena’s AI-driven drug discovery and development platform, JuvNET, JUV-161’s primary mechanism of action is derived from an engineered human Insulin-like Growth Factor 2 (IGF2) protein developed for subcutaneous injection.

About JuvNET: A Unique AI-driven Discovery Platform Mapping the Therapeutic Potential of Secreted Proteins
JUV-161 is the lead therapeutic candidate discovered by JuvNET, which screens Juvena’s proprietary protein library of human stem-cell secreted proteins with therapeutic potential using unbiased quantitative proteomics, multi-omics and high-content imaging data, combined with a variety of artificial intelligence technologies. The company’s pipeline of biologics for myopathies and metabolic diseases, all produced by JuvNET, include JUV-112, an obesity targeting biologic which induces fat and weight loss via a non-appetite suppressive, muscle sparing, energy expenditure inducing mechanism. Additionally, Juvena’s platform has identified over 50 secreted protein hits to date across multiple metabolic diseases and organ systems.

About Juvena Therapeutics
Juvena Therapeutics is a platform-driven, clinical-stage company unlocking the therapeutic potential of stem-cell secreted proteins and translating them into a growing pipeline of engineered biologics for chronic muscle and metabolic diseases. Juvena is achieving this through its fully integrated, end-to-end Artificial Intelligence (AI)-enabled drug discovery and development platform, JuvNET, that combines a compounding database mapping secreted proteins to specific disease phenotypes, in silico and in vitro human cell screening, pharmacology, and protein engineering capabilities. In addition to a pipeline of biologic drug candidates spanning chronic muscle and obesity-related diseases, the company has generated dozens of hits with tissue restorative benefits spanning pulmonary and hepatic fibrosis and osteoarthritis. To learn more visit https://www.juvenatherapeutics.com/ or follow the company on LinkedIn @Juvena-Therapeutics.

Press Inquiries
Shira Derasmo
Cuttlefish Communications
+1 917-280-2497
shira@cuttlefishpr.com

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¹ https://www.mda.org/disease/myotonic-dystrophy
² https://www.myotonic.org/myotonic-dystrophy-glance
³ https://www.mda.org/disease/myotonic-dystrophy