Barking up the wrong myostatin tree?
Wyeth Pharmaceuticals of Madison, N.J., has announced it will not continue development of its experimental compound MYO-029 for muscular dystrophy.
MYO-029 is an antibody (immune-system protein) designed to stick to and interfere with the actions of myostatin, a protein that limits muscle growth. By blocking myostatin, it was hoped that muscles affected by muscular dystrophy would become larger and stronger. Development of MYO-029 was based in part on preclinical research funded by MDA.
“We're disappointed that MYO-029 will not be moving forward," said Sharon Hesterlee, MDA vice president of translational research. "But I doubt this is the end of the line for myostatin inhibition. MDA is looking at other ways to block myostatin and, of course, other strategies to improve muscle health.”
Beginning in 2005, with supplemental funding to trial sites from MDA, Wyeth began a phase 1-2 clinical trial to test the safety and tolerability of MYO-029 in 116 adults with Becker, facioscapulohumeral and limb-girdle types of muscular dystrophy.
The compound was found to be safe and well tolerated at three dosage levels, reports a paper published online todayin Annals of Neurology by Kathryn Wagner at Johns Hopkins University School of Medicine in Baltimore, and colleagues.
However, the investigators found no improvements in muscle strength or function, and no statistically significant muscle growth in trial participants. (The authors note that the study was not designed to measure efficacy.)
Investigators say the MYO-029 clinical trial supports the hypothesis that systemic administration of agents that block myostatin is safe enough for future studies and that further evaluation of more potent myostatin inhibitors than MYO-029 should be considered.
Wyeth Executive Vice President and Chief Medical Officer Gary Stiles says the company “remains committed to discovering and developing treatments for muscle diseases and continues to explore myostatin inhibition along with other strategies,” despite its discontinuation of the MYO-029 program.