Domagrozumab

Domagrozumab is an experimental drug that was under development by Pfizer for the treatment of Duchenne muscular dystrophy. The drug is a humanized monoclonal antibody that targets myostatin, a protein that inhibits muscle growth. However, in August 2018, Pfizer announced that it was discontinuing the development of domagrozumab due to lack of efficacy.

History

Domagrozumab was initially developed by Pfizer as a potential treatment for Duchenne muscular dystrophy (DMD), a rare and severe form of muscular dystrophy. The drug was designed to target myostatin, a protein that inhibits muscle growth. By blocking the action of myostatin, it was hoped that domagrozumab could promote muscle growth and improve muscle function in people with DMD.

In 2012, Pfizer initiated a Phase 2 clinical trial to evaluate the safety and efficacy of domagrozumab in patients with DMD. However, in August 2018, Pfizer announced that it was discontinuing the development of the drug due to lack of efficacy. The decision was based on the results of an interim analysis of the Phase 2 trial, which showed that domagrozumab did not meet the primary efficacy endpoint.

Mechanism of Action

Domagrozumab is a humanized monoclonal antibody that targets myostatin, a protein that inhibits muscle growth. Myostatin is produced in skeletal muscle cells, circulates in the blood and acts on muscle tissue by binding to a cell-bound receptor called the activin type II receptor. Domagrozumab binds to myostatin and prevents it from binding to its receptor, thereby inhibiting its activity. This is expected to result in increased muscle mass and strength.

Clinical Trials

The Phase 2 clinical trial of domagrozumab was a randomized, double-blind, placebo-controlled study that enrolled patients with DMD. The primary endpoint of the trial was the change in total lean body mass (as measured by DEXA) from baseline to week 48. Secondary endpoints included changes in muscle strength, physical function, and quality of life.

Despite the initial promise, the trial did not meet its primary endpoint, leading to the discontinuation of the drug's development.

See Also

• Duchenne muscular dystrophy
• Pfizer
• Myostatin
• Monoclonal antibody