Celadon presents CUPID trial data at AHA

Celladon, a US privately-held biotechnology company that develops molecular therapies for the treatment of heart failure, presented Phase I data from the CUPID trial of its Mydicar genetically-targeted enzyme replacement therapy for advanced heart failure at the American Heart Association scientific sessions, held in New Orleans.

CUPID is a Phase I/II trial. The Phase I portion is an open-label, sequential-dose escalation study, while the Phase II portion is a randomized, double-blind, placebo-controlled, parallel-group, dose-ranging trial that compares the use of Mydicar at two or three dose levels with placebo. CUPID is expected to enroll 46 patients with advanced heart failure at 15 US medical centers.

The data demonstrated that Mydicar had an acceptable safety profile in these first nine patients, as determined by study investigators and an independent safety committee. In addition, improvements from baseline to six months across a number of parameters important in assessing heart failure status were observed, including symptomatic (five patients), functional (four), biomarker (two) and left ventricular function/remodeling (six). Of the nine patients treated, two with low levels of pre-existing antibodies to the adeno-associated viral vector did not show improvement in these parameters, the firm noted.