Celladon to report results of its gene therapy for heart failure: CEO

By Deena Beasley (Reuters) - Celladon Corp expects to report this month results from a key trial of Mydicar, its experimental gene therapy for advanced heart failure, a condition in which the heart muscle cannot pump enough blood to meet the body's needs. The upcoming results are seen as a big test for the more than 20-year-old field, which has re-emerged after a series of disappointments and safety concerns. On Monday, Bristol-Myers Squibb Co acquired a nearly 5 percent stake in uniQure NV, sending shares of the Dutch gene therapy producer up 55 percent. "If our data are positive, I think there will be a lot of competition because it is an enormous market," Celladon Chief Executive Officer Krisztina Zsebo said in a recent interview with Reuters. More than 5 million Americans are living with heart failure, costing the nation around $32 billion each year, according to the Centers for Disease Control and Prevention. Gene therapies aim to insert corrective genes into malfunctioning cells. Celladon's Mydicar is a virus designed to deliver a gene that reconstitutes an enzyme responsible for heart muscle contraction. An early trial in patients with advanced heart failure found that Mydicar reduced hospitalizations by 88 percent after 12 months, and 80 percent at the three-year mark. A confirmatory, Phase 2b, trial of 250 patients will be unblinded at the end of this month. Mydicar has been granted both "breakthrough" and "fast-track" status by the Food and Drug Administration. Celladon is planning a larger, pivotal trial, but it is possible that if the upcoming data are strong enough, Mydicar could be approved in certain geographies, Zsebo said. She said studies have shown no evidence of toxicity, but there are questions about how long the effect of the gene therapy will persist. If Mydicar is approved, patients would probably be treated just once due to the likelihood that their bodies would develop antibodies against the introduced proteins. Shares of San Diego-based Celladon, which went public last year, closed at $17.49 on Wednesday, a drop of about 30 percent in the past four weeks. If Mydicar is approved, Zsebo said the gene therapy would be priced like any other biologic drug. She said Celladon would not seek any "crazy annuity-based pricing," nor would it set a price in the hundreds of thousands of dollars, which is what companies currently charge for enzyme replacement therapies used to treat rare diseases. (Reporting by Deena Beasley in Los Angeles; Editing by Lisa Shumaker)