May 31 2010
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.
The Company and Celladon first entered into collaboration and manufacturing agreements in 2004. In 2009, Targeted Genetics licensed its adeno-associated virus (AAV) vector serotype and manufacturing technology to Celladon and manufactured clinical supplies of MYDICAR. Under the 2009 license agreement between Targeted Genetics and Celladon, if MYDICAR is developed and commercialized by Celladon, then Targeted Genetics could receive multiple milestone payments totaling up to $20 million, starting with a $5 million milestone payment if a MYDICAR Phase III human clinical trial commences. In addition, the Company could receive a 10% royalty on commercial sales of MYDICAR, subject to certain reductions in some cases. Alternatively, if Celladon enters into a partnering transaction for MYDICAR or a sale transaction of Celladon or its MYDICAR assets, the Company could receive a $5 million milestone at the start of Phase III plus 10% of future partnering revenue or sale proceeds received by Celladon and, subject to certain reductions in some cases, a royalty of 10% on sales of MYDICAR.
"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," said Susan Robinson, President and CEO of Targeted Genetics. Ms. Robinson continued, "Should the product candidate MYDICAR proceed successfully through development and onto commercialization, it could provide a much needed advancement for the treatment of patients with heart failure, a significant clinical validation for the field of gene therapy, and a revenue stream for Targeted Genetics."
Targeted Genetics Corporation