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CUPID: AAV1/SERCA2a found safe, beneficial in patients with advanced HF
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Heart Failure Society of America 14th Annual Scientific Meeting
SAN DIEGO — New data from a phase 2 trial presented here showed favorable results, including group and individual improvements, for patients with HF who were treated with AAV1/SERCA2a, a genetically targeted enzyme replacement therapy for advanced HF.
Researchers for the Calcium Upregulation by Percutaneous Administration of Gene Therapy in Cardiac Disease (CUPID) trial set out to evaluate the safety and feasibility, as well as explore the activity and efficacy, of AAV1/SERCA2a (Mydicar, Celladon). They included patients who were aged 18 to 75 years and had an NYHA Class III or IV, ischemic or non-ischemic cardiomyopathy and left ventricular ejection fraction of 35% or less. Patients were excluded if they had anti-AAV1 neutralizing antibody titer of at least 1.2, clinically significant MI within 6 months, were likely to need HF-related surgery within the following 6 months and had an expected survival of less than 1 year.
Of the total study population (n=39; 34 men with a mean age of 60.5 years), 19 (48.7%) had ischemic cardiomyopathy, 14 (35.9%) had idiopathic cardiomyopathy and four (10.3%) had hypertensive cardiomyopathy. According to the study results, in patients treated with AAV1/SERCA2a vs. placebo, there was an improvement in group level success in the 6-minute walk test (P=.14) and end-systolic volume (P=.057); an improvement in the mean individual efficacy score (P=.052); and an improvement in duration of CV hospitalization (P=.08).
Regarding safety, Barry H. Greenberg, MD,with the University of California, San Diego, and study researcher, said in his presentation that although there were many adverse events in this very sick patient population, there was no evidence that any dose of AAV1/SERCA2a increased adverse events when compared with placebo.
“These encouraging results support further studies to determine the value of genetically targeted enzyme replacement of SERCA2a in advanced HF,” Greenberg said.
Michael R. Bristow, MD,of the University of Colorado Cardiovascular Institute, Denver, who provided commentary after the presentation, said the CUPID trial shows that AAV1/SERCA2a gene therapy can be given in a multicenter design with what appears an excellent safety profile.
In addition, Bristow said, “There is definite evidence of an efficacy signal, and I believe there is enough data here that this treatment should progress to the next stage, either phase 2b or 3. CUPID should serve as a catalyst for the development of other gene therapies for HF.” – by Brian Ellis
For more information:
- Greenberg B. LBCT I. Presented at: Heart Failure Society of America 14th Annual Scientific Meeting; Sept. 13-16, 2010; San Diego.
