Highlights

  • Juvena Therapeutics has received the FDA’s orphan drug designation — providing tax incentives for conducting clinical trials — for its drug JUV-161.
  • JUV-161 human trials will begin toward the end of the year with hopes of restoring muscle and improving strength and endurance in patients with a condition of progressive muscle weakening — myotonic dystrophy type I.
  • In addition to potentially treating myotonic dystrophy type I, JUV-161 could alleviate age-related muscle dysfunction.

The California-based biotech company Juvena Therapeutics has been granted the FDA’s orphan drug designation for JUV-161 — a drug discovered through an AI platform that treats myotonic dystrophy type I (DM1). DM1 is an inherited disease and the most common form of adult muscular dystrophy — where muscles progressively weaken and shrink.

The FDA’s orphan drug status qualifies Juvena Therapeutics for incentives like tax credits for clinical trials. The orphan drug designation also grants Juvena Therapeutics with a potential seven years of market exclusivity, where only Juvena Therapeutics can sell the drug, following the FDA’s drug approval process.

Juvena Therapeutics’ New Drug Discovery May Also Alleviate Age-Associated Muscle Deterioration

As for the new drug discovered with AI technology, JUV-161, researchers have shown that it restores muscle fiber formation, counteracts muscle weakening, improves muscle strength and endurance, and improves metabolism in various DM1 animal models like mice. In that regard, human JUV-161 trials are expected to commence later this year.

While DM1 is a rare disease, it is also relevant in the context of longevity. In fact, people with DM1 exhibit accelerated hallmarks of aging. Along those lines, the FDA considers DM1 to be an accelerated aging disease (a progeroid disease), according to Juvena’s Dr. Hanadie Yousef. As such, Yousef says that JUV-161’s muscle regeneration, metabolism, and muscle function-enhancing capabilities are essentially counteracting some of the effects of muscle aging.

Moreover, JUV-161 works in multiple age-related disease models. Examples include age-related muscle loss (sarcopenia) and aged muscle injury. Along those lines, Yousef says that JUV-161 is being developed for DM1 first and foremost but that once it is shown to be safe and effective, there will be an easier path for uses in diseases of aging like sarcopenia. Such a path may entail off-label prescriptions, aside from being used to treat DM1, for age-related conditions pertaining to deteriorated muscle function. Such a scenario would be similar to how Ozempic, a type II diabetes medicine, is being prescribed off-label for weight loss.

Juvena Therapeutics Utilizes AI-Identified Stem Cell Secreted Proteins to Target Aging

The AI-based drug discovery platform used to propose JUV-161 is examining stem cell-secreted proteins (part of the secretome) to identify proteins that target the biology of aging. In doing so, Juvena Therapeutics hopes to restore the function of aging tissues and alleviate various age-related muscle and metabolic diseases, as JUV-161 does.

The company says it has already identified multiple stem cell-secreted proteins that show capabilities in improving age-related diseases across multiple organs. Although not explicitly stated, it is possible that the benefits of these proteins have already been demonstrated in animal models like mice.

Some examples of secreted compounds already FDA-approved for medicinal purposes include insulin (secreted by pancreas cells) for diabetes and human growth hormone (secreted by pituitary gland cells) to boost lean body mass, according to Jeremy O’Connell, Juvena’s Chief Scientific Officer. He adds that these secreted proteins can have life-changing impacts. However, the massive analyses and search power needed to map this class of proteins has been a gargantuan challenge for the biotech industry. With the advent of AI-based drug discovery, such as the platform Juvena used to identify JUV-161, the discovery of new proteins from this class to reverse age-related diseases in humans may have gotten easier.