US-based Acceleron Pharma has started the Phase I clinical trial of its new drug, ACE-083, designed to selectively increase muscle mass and strength.
The drug works by blocking proteins in the transforming growth factor-beta (TGF-ß) protein superfamily that modulates muscle growth.
ACE-083 is being developed for diseases in which improved muscle strength in a specific set of muscles may provide clinical benefits, such as inclusion of body myositis and certain forms of muscular dystrophy.
Acceleron chief executive officer John Knopf said: "Acceleron has built a highly productive drug discovery platform based on our deep understanding of the biology of the TGF-ß protein superfamily, and we are proud to bring another first-in-class protein therapeutic into human clinical trials.
"There is an enormous unmet medical need to treat patients suffering from muscular and neuromuscular diseases, such as some forms of muscular dystrophy, and we believe that ACE-083 has the potential to treat a wide array of diseases in which patients have lost muscle mass and strength in specific muscles or muscle groups."
The company is focused on the discovery, development and commercialisation of new protein therapeutics for cancer and rare diseases.
Acceleron understands the biology of the TGF-ß protein superfamily, a large and diverse group of molecules that are key regulators in the growth and repair of tissues throughout the human body, and in targeting these pathways to develop important new medicines.
The company has built a highly productive research and development platform that has generated new clinical and pre-clinical protein therapeutic candidates with novel mechanisms of action.