Value Proposition
· GCPII inhibitor has a low IC50 (<5 nM), suggesting greater potency and less systemic toxicity.
· Functional improvements after treatment include 95% preservation of muscle mass, 96% preservation of grip strength, 25% greater isometric force.
· Decreased decline in neuromuscular transmission compared to control.
Technology Description
Researchers at Johns Hopkins have found that muscle atrophy is associated with glutamate carboxypeptidase II (GCPII) activity in muscle macrophages. They developed a system to deliver a GCPII inhibitor to macrophages and showed in mice that it not only preserves muscle mass and function (by up to 25% by some measures of function), but also nerve signal conductivity.
Unmet Need
Sarcopenia, a loss of muscle mass and function that increases the risk of falls and fractures, affects 10-16% of the global population. Currently there are no drugs that are FDA-approved to treat sarcopenia. Many attempts have increased muscle mass without increasing muscle function. The current gold standard for sarcopenia treatment is exercise, which leads to a minor functional improvement. The need for an effective sarcopenia treatment is exacerbated by the recent popularity of weight loss drugs. These cause significant decreases in muscle mass, jeopardizing their viability as long-term treatments. Thus, there exists a need for more effective sarcopenia treatments.
Stage of Development
· Preclinical studies in aged mice have been completed and demonstrate preserved muscle mass, neuromuscular conduction, and functional improvements.
· Current efforts are focused on characterization of a lead drug conjugate.
Data Availability
· Data available upon request.
Publications
- WO 2025/193538
- Related Technology: C17127
