Juvena Therapeutics Receives FDA Orphan Drug Designation for JUV-161 for the Treatment of Myotonic Dystrophy Type 1
23 Jan 2024
Orphan Drug
Biologic treatment that restores muscle fiber formation leads Juvena’s pipeline of assets to treat myopathies, metabolic diseases and obesity
REDWOOD CITY, Calif.--(BUSINESS WIRE)-- Juvena Therapeutics, a biotechnology company unlocking the potential of stem cell-secreted proteins to treat muscle and metabolic diseases including muscular dystrophies and obesity, today announced the U.S. Food and Drug Administration (FDA) granted Orphan Drug designation to the company’s flagship candidate, JUV-161. JUV-161 is an investigational therapeutic for the treatment of Myotonic Dystrophy Type 1 (DM1), a rare, multi-systemic, autosomal dominant inherited disease and the most common form of adult muscular dystrophy.
“People living with DM1 deserve safe, effective, and rejuvenating treatments that can repair and restore tissue health to improve muscle function and metabolism,” said Dr. Hanadie Yousef, Co-Founder and CEO of Juvena Therapeutics. “As we advance Juvena’s first biologic initially focused on the treatment of DM1, we look forward to exploring potential treatments for an entire class of disorders that remain major unmet medical needs.”
JUV-161, which was identified by Juvena’s AI-driven drug discovery and development platform, has demonstrated an ability to restore muscle fiber formation, counter muscle atrophy, enhance muscle strength and endurance, and improve metabolism in multiple animal models. Human trials are anticipated to commence this year. JUV-161 includes an engineered human IGF-2 protein developed for subcutaneous injection.
“Until treatments are available, DM1 will continue to progressively limit individuals' strength and their ability to do everyday things like walking, talking, swallowing, and even breathing,” said Dr. Tanya Stevenson, CEO of the Myotonic Dystrophy Foundation. “We are excited about the unique approach Juvena Therapeutics is advancing because of its potential to safely promote muscle regeneration, improve metabolism, rejuvenate lives, and ultimately to more holistically address the disease.”
Juvena’s platform mines thousands of proteins secreted by human stem cells, screens their potential to restore tissue homeostasis, and engineers novel biologic medicines into life-saving treatments. The company has identified an expanded proprietary multi-dimensional library of regenerative proteins. JUV-161 leads Juvena’s preclinical pipeline of engineered biologics for multiple myopathies and metabolic diseases. Additionally, Juvena has identified multiple secreted protein hits that induce disease-modifying effects across several metabolic organs and therapeutic areas.
“FDA-approved biologics based on secreted proteins such as insulin, human growth hormone, and erythropoietin, can have life-changing impacts; however, the massive search power needed to systematically map this complex class of proteins has challenged the industry's ability to continue discovering proteins that reverse disease in humans,” said Dr. Jeremy O'Connell, Co-Founder and Chief Scientific Officer of Juvena Therapeutics. “We are thrilled with the recognition of our lead candidate, JUV-161, as a potential addition to this class of drugs, and the success of Juvena’s platform in revolutionizing the study of secreted proteins as therapeutics.”
The FDA's Office of Orphan Products Development grants orphan status to support the development of medicines for rare disorders that affect fewer than 200,000 people in the U.S. Orphan Drug designation provides certain benefits, including market exclusivity upon regulatory approval, exemption of FDA application fees, and tax credits for qualified clinical trials.
About DM1
DM1 affects an estimated 1 in 2,100 people and can present at any age. People living with DM1 experience a range of signs and symptoms which may include muscle wasting and weaknessweaknessweaknessweaknessweaknessweaknessweakness, myotonia (slow muscle relaxation), cataracts, heart conduction and rhythm abnormalities, abnormal blood sugar control, and breathing problems. Currently, there are no treatments for DM1.
About Juvena Therapeutics
Juvena Therapeutics is a computationally-driven biopharmaceutical company on a mission to develop disease-modifying biologics to treat chronic muscle and metabolic diseases. The company’s approach stems from unlocking the therapeutic potential of stem-cell secreted proteins and translating them into a growing pipeline of engineered biologics. In addition to JUV-161, Juvena is progressing multiple preclinical programs, including a novel obesity asset with a unique mechanism. Juvena is achieving this through a fully integrated, end-to-end Artificial Intelligence (AI)-enabled drug discovery and development platform 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. To learn more about Juvena Therapeutics, visit JuvenaTherapeutics.com or follow the company on LinkedIn @Juvena-Therapeutics.
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