Rare Daily Staff

Pfizer said that it is terminating two ongoing clinical studies evaluating its experimental drug domagrozumab for the treatment of Duchenne muscular dystrophy a rare, progressive, and fatal genetic disorder.

DMD is caused by a genetic mutation that results in a deficiency of dystrophin, a protein that is essential to healthy muscles. DMD results in muscle weakness from early childhood. Patients with the condition can lose the ability to walk as early as age 10, followed by loss of the use of their arms. The disease leads to premature death in the mid-twenties due to heart and respiratory failure.

Pfizer said that domagrozumab failed to meet its primary endpoint in a mid-stage safety and efficacy study and an open-label extension study. Further evaluation of the trial data including secondary endpoints did not support a significant treatment effect, the company said.

“We are disappointed by these results and while we are not progressing with the studies, the data will contribute to a greater understanding of this disease and we will evaluate the total data set to see if there is a place for this medicine in muscular diseases,” said Seng Cheng, senior vice president and chief scientific officer, Pfizer Rare Disease Research Unit. “We are extremely grateful to all those involved with this trial, especially the boys who participated, and their families.”

Pfizer said it will continue research in DMD and rare neuromuscular diseases. Pfizer has an ongoing clinical trial in DMD with a gene therapy that carries a shortened version of the human dystrophin gene.

August 30, 2018
Photo: Seng Cheng, senior vice president and chief scientific officer, Pfizer Rare Disease Research Unit