Cleveland, Ohio-based Juventas Therapeutics, developing novel regenerative therapies for treating cardiovascular disease, has released the 12-month results from its phase I heart failure study of JVS-100.

JVS-100 encodes Stromal cell-Derived Factor 1 (SDF-1) and has been shown to repair damaged tissue through recruitment of circulating stem cells to the site of injury, prevention of ongoing cell death and restoration of blood flow.

The 17-person, open-label, dose-escalation study targeted New York Heart Association (NYHA) class III heart failure patients, who represent approximately a quarter of the six million heart failure patients in the U.S. and account for half of all heart failure hospital admissions. The clinical trial met its primary safety endpoint with no serious adverse events deemed drug related. 

Fifteen of the 17 patients in the trial survived to a year. Patients receiving target therapeutic doses demonstrated clinically significant improvements at 12 months in six-minute walk distance and the Minnesota Living with Heart Failure Questionnaire.  Nearly half of the patients improved a full NYHA class, with multiple patients improving two full classes.

"The patient population we treated in this trial has a true unmet clinical need and tends to have rapidly deteriorating quality of life," said Marc Penn, M.D., Ph.D, founder and chief medical officer for Juventas and director of cardiovascular research at the Summa Cardiovascular Institute at Summa Health System.  "To see clinical symptomatic benefits of this magnitude maintained from four to 12 months after JVS-100 treatment suggests we are inducing fundamental changes in the heart of treated patients.  We believe this is consistent with our understanding of the mechanisms associated with JVS-100 and warrants further investigation."

Based on these results, the company is preparing to enroll a placebo-controlled, randomized, double-blinded phase II heart-failure clinical trial in the U.S. to further define the efficacy of JVS-100.  Also, Juventas has received FDA clearance to enroll a phase IIa trial evaluating safety and efficacy of JVS-100 in patients with critical limb ischemia.  The CLI trial is enrolling patients in the U.S. and India.  In addition to safety, the trial will assess time to amputation and other efficacy endpoints and will begin enrollment in the first quarter of 2012.