Circulation journal publishes Celladon's MYDICAR Phase 2 trial on advanced heart failure

Celladon Corp., a biopharmaceutical company focused on the discovery and development of innovative treatments for cardiovascular diseases, announces that the data from its completed Phase 2 CUPID clinical study of MYDICAR® for the treatment of advanced heart failure is being published today online in Circulation, an American Heart Association journal.

"I am very encouraged that our approach of restoring a critical enzyme that is depleted in advanced heart failure may lead to new treatment options for those patients that continue to decline despite the best standard drug and device therapies," said Mariell Jessup, M.D., Professor of Medicine at the University of Pennsylvania's School of Medicine, who was the principal investigator and lead author of the study.  "The clinical deterioration seen in the placebo patients receiving ongoing optimal standard therapy emphasizes the tremendous unmet medical need in people with advanced heart failure. Based on these results we are encouraged that MYDICAR may one day fill this need."  

The previously announced top line results of 39 patients met its primary safety and efficacy endpoints at 6 months for high dose MYDICAR versus placebo.  Additionally, 12 months after receiving a single infusion of MYDICAR, patients treated with the highest dose versus placebo had an 88 percent risk reduction (Hazard Ratio = 0.12).

• Death
• Need for left ventricular assist device (LVAD) or cardiac transplant
• Episodes of worsening of heart failure
• Number of heart failure-related hospitalizations

The mean duration of hospitalization in the MYDICAR high dose group during the 12-month period was 0.4 days per patient compared with 4.5 days per patient in the placebo group. This finding is especially noteworthy because heart failure is the leading cause of hospitalization in Americans 65 years of age and older.

Additionally, the 12-month CUPID data show that heart failure, which is a progressive disease, became stabilized in high dose MYDICAR-treated patients: heart failure symptoms, exercise tolerance, serum biomarkers and cardiac function essentially improved or remained the same while these parameters deteriorated substantially in patients treated with placebo and concurrent optimal drug and device therapy.

The safety profile from this study was very favorable, with no significant side-effects from MYDICAR therapy.

Roger Hajjar, M.D., Director, Cardiovascular Research Center, Mount Sinai School of Medicine, NY, and co-founder of Celladon said: "While conventional treatment modalities have reduced heart failure mortality in the last 20 years, there remains a critical need for new therapeutic approaches in patients with advanced heart failure. The successful and safe completion of the phase 2 CUPID trial may give patients with heart failure a new therapeutic option in the future."