Targeted Genetics Corporation (Pink Sheets:TGEN) ("Company") announced that its partner, Celladon Corporation, presented full data today that show the first Phase II trial with MYDICAR® for the treatment of advanced heart failure has met its primary safety and efficacy endpoints, as prospectively defined in the Phase II trial protocol.
“The presentation today provided the detail supporting Celladon's announcement in April that the MYDICAR trial had met its protocol objectives. The statistical significance achieved on clinically meaningful endpoints and the safety profile emerging with MYDICAR puts Celladon in a favorable position to plan future development of this candidate”
The study met its primary safety and efficacy endpoints for high dose MYDICAR® versus placebo. The primary efficacy endpoint is a composite endpoint that encompasses the simultaneous assessment of patients' clinical outcomes, exercise tolerance, heart failure symptoms, biomarkers, and cardiac function. Barry Greenberg, M.D., Professor of Medicine at the University of California, San Diego, presented the data in advanced heart failure patients as a late-breaking trial presentation at Heart Failure Congress 2010, the annual meeting of the Heart Failure Association of the European Society of Cardiology.
High dose MYDICAR® treated patients had a statistically significant reduction in cardiovascular events as defined by death, the need for left ventricular assist device (LVAD) or cardiac transplant, worsening of heart failure or heart failure related hospitalizations, which translated into a of 50 percent risk-reduction in favor of high dose MYDICAR® (hazard ratio 50 percent versus placebo.
Additionally, patients treated with high dose MYDICAR® improved significantly in their heart failure symptoms, their exercise tolerance, serum biomarkers and cardiac function. Specifically, the quality of life worsened by +3.4 points for placebo treated patients but improved by -10.3 points for MYDICAR® treated patients, as measured by the Minnesota Living with Heart Failure® Questionnaire; exercise tolerance, measured with the six-minute walk test, decreased (worsened) in the placebo group by 87 meters but increased by one meter in the MYDICAR® group. A serum biomarker of heart failure, NT-ProBNP, worsened by +5540 pg/mL in placebo treated patients, and improved by -13.5 pg/mL in MYDICAR® treated patients, cardiac function worsened (heart further enlarged) by 18.2 mL (left ventricular end-systolic volume) in the placebo group, but improved by -9.6 mL in the MYDICAR® group, indicating a reverse remodeling of the damaged heart in the MYDICAR® treated patients.