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Gene Therapy Improves Cardiac Structure, Function in Patients With Ischemic Heart Failure
In new 12-month data presented at the European Society of Cardiology –– Heart Failure 2015 meeting, a single administration of 30 mg of JVS-100 (Juventas Therapeutics), a nonviral DNA plasmid gene therapy, improved cardiac structure, function, serum biomarkers, and clinical status in patients with severe ischemic heart failure 1year after treatment. The final results from the phase II STOP-HF clinical trial are in press at the European Heart Journal.
The randomized, double-blind, placebo-controlled study involved 93 patients at 16 clinical centers in the U.S.
JVS-100 encodes stromal cell-derived factor 1 (SDF-1), a naturally occurring signaling protein. When delivered directly to a site of tissue injury, the treatment induces the expression of SDF-1 proteins into the local environment for a period of approximately 3 weeks. The secretion of SDF-1 has been shown to create a homing signal that recruits the body’s own stem cells to the site of injury to induce tissue repair and regeneration. JVS-100 is being developed for the treatment of patients with advanced chronic cardiovascular disease, including heart failure and late-stage peripheral artery disease.
In the full study population, patients receiving the 30-mg dose of JVS-100 demonstrated improvements in objective measures of cardiac function and structure compared with placebo-treated patients, as measured by the median change in the left ventricle ejection fraction (LVEF) (3.5% greater than placebo) and in the left ventricular end-systolic volume (LVESV) (8.5 mL more than placebo). A composite score comprising the change from baseline in the 6-Minute Walk Test distance and in the Minnesota Living With Heart Failure Questionnaire also showed a trend toward an improved clinical status for patients treated with 30 mg of JVS-100 (2.5 points); however, the increase in the composite score in patients who received placebo (2.0 points) led to the trial missing its primary endpoint at 4 months.
A prespecified subanalysis of patients with the lowest-tertile LVEF at the time of enrollment demonstrated an 11% increase (P < 0.01) in LVEF 1 year after the patients received 30 mg of JVS-100 compared with placebo. In this high-risk group, 30 mg of JVS-100 also led to clinically meaningful improvements in the LVESV of –34 mL and in stroke volume of 25 mL. The same patients treated with LVS-100 demonstrated a clinically meaningful improvement in the composite score of 2.5, compared with an improvement of 1.2 in the placebo group.
Source: Juventas Therapeutics; May 26, 2015.