Juvena gets FDA Orphan Drug nod for muscle regeneration therapy

Longevity biotech goes after rare muscle disease with plans to expand into diseases of aging, including sarcopenia.
Longevity biotech startup Juvena Therapeutics has been granted the FDA’s Orphan Drug designation for using its flagship drug candidate, JUV-161, to address Myotonic Dystrophy Type 1 (DM1), an inherited disease that is the most common form of adult muscular dystrophy.

The Redwood, CA based company said that JUV-161 has demonstrated the ability to restore muscle fiber formation, counteract muscle atrophy, enhance muscle strength and endurance, and improve metabolism in various animal models. Human trials of JUV-161 are anticipated to commence later this year.

“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.

While DM1 is a rare condition (Orphan Drug designation is reserved for treatments for diseases affecting fewer than 200,000 people) it is also relevant in a longevity context.

“People living with the condition display accelerated hallmarks of aging,” Juvena’s CEP Dr Hanadie Yousef told us. “In fact, the FDA considers Myotonic Dystrophy Type 1 to be a progeroid disease.”

Yousef says that JUV-161’s ability to enhance muscle regeneration, metabolism, and function, is essentially counteracting the effects of aging and degenerative disease.

“It works in multiple muscle disease models, including models of sarcopenia, aged muscle injury and glucocorticoid induced muscle atrophy,” she said. “We are developing JUV-161 first for Myotonic Dystrophy Type 1, with an accelerated path to approval, well-defined inclusion and exclusion criteria and patient advocacy foundations and KOLs. Once we succeed in translating JUV-161 and showing its safety and its potential for efficacy, we will have an easier path to expand to larger diseases of aging, such as sarcopenia.”

Juvena’s AI discovery platform mines the secretomes of human pluripotent stem cells to identify secreted proteins that target the biology of aging to restore tissue homeostasis and develop them as disease-modifying biologics. The company says it has identified multiple secreted protein hits that show disease-modifying effects across various 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, Juvena’s Chief Scientific Officer.

Juvena gets FDA Orphan Drug nod for muscle regeneration therapy