Juventas Therapeutics announced that it has successfully completed its Phase I clinical trial evaluating the safety and preliminary efficacy of JVS-100 for treatment of patients with heart failure. Complete results from the trial will be reported at the 2011 Annual Meeting for the American Society for Gene & Cell Therapy on 19 May in Seattle, USA.
JVS-100 encodes Stromal-cell Derived Factor 1 (SDF-1), which has been shown in several pre-clinical studies to promote cardiac repair following a myocardial infarction through activation of natural stem-cell repair pathways, promotion of new blood vessel formation and prevention of on-going cell death.
Preliminary results from the clinical trial were recently reported at the Annual Meeting for the American College of Cardiology in New Orleans, USA. The trial enrolled 17 NYHA Class III heart failure patients, with left ventricular ejection fractions less than 40% and an average time from heart attack of 7.3 years. Three escalating JVS-100 doses were evaluated: 5mg (cohort 1), 15mg (cohort 2) and 30mg (cohort 3). The primary safety endpoint was the number of major adverse cardiac events (MACE) at 30 days.
Efficacy was assessed as changes from baseline in: echocardiographic parameters, cardiac perfusion via SPECT and clinical parameters including NYHA class, 6 minute walk distance (6MWd) and quality of life score (QOL). Safety data has been collected for all 17-patients and 4-month efficacy data has been collected from cohort 1 and 2 patients. The primary safety endpoint for the trial has been met with no adverse events likely related to drug. At 4 months post-therapy, patients are demonstrating dose-dependent improvements in all clinical parameters with clinically relevant improvements compared to baseline in QOL and 6MWd for patients from cohort 2. At 1-month after dosing in cohort 3 patients are showing similar clinically relevant improvements in QOL and 6MWd with some patients improving a full NYHA class.
“The preliminary safety and efficacy results from this clinical trial are encouraging,” said Douglas Losordo, principal investigator of the trial and director of the Feinberg Cardiovascular Research Institute at Northwestern University, USA. “The clinical responses observed are exciting and warrant further investigation of JVS-100 for treatment of heart failure in a Phase II clinical trial.”
In addition to its heart failure platform, Juventas has also received FDA authorisation to initiate a Phase II clinical trial evaluating the safety and efficacy for use of JVS-100 to treat patients with critical limb ischaemia.
“We have a strong understanding for the mechanisms of action through which SDF-1 works,” said Marc Penn, chief medical officer for Juventas and director for the Skirball Cardiovascular Cell Therapy Laboratory. “This understanding allows us to strategically and rapidly target JVS-100 to additional disease indications in which we believe JVS-100 will provide the greatest therapeutic benefit.”