U.S. drugmaker Pfizer Inc said on Wednesday it reached an agreement with Sangamo Therapeutics Inc for rights to its gene therapy programs to treat the rare blood-clotting disorder hemophilia A, under which Pfizer will pay $70 million upfront.
Shares of Sangamo jumped 41 percent to $6.15 in after-the-bell trading.
Sangamo will be eligible for potential milestone payments of up to $475 million and tiered double-digit royalties on net sales if the treatment is approved.
Michael Goettler, global president of Pfizer Rare Disease, said the deal bolsters Pfizer’s pipeline in both gene therapy and hemophilia, noting that the company already has a collaboration for a hemophilia B treatment.
“We have an intention to become a leader in gene therapy and this is another step,” he said in an interview.
Sangamo is expected to begin human trials of one of its lead product candidates, SB-525, this quarter. Pfizer will fund and conduct any late-stage clinical trials.
Hemophilia A is caused by a genetic mutation resulting in insufficient activity of Factor VIII, a blood-clotting protein the body uses to stop bleeding. There are approximately 16,000 patients in the United States and more than 150,000 worldwide with hemophilia A.
(Reporting by Michael Erman; Editing by Leslie Adler)