Trial Success for Gene Therapy, Delivered the Helix™ Way

Juventas JVS-100 Gene Therapy delivered to heart muscle using the Biocardia Helix system, has been delivering promising clinical data, as presented at the European Society of Cardiology Heart Failure Congress in Greece.

BioCardia®, Inc., a leader in cardiovascular regenerative medicine, today announced the presentation by Juventas Therapeutics of their positive four-month results for their STOP-HF Trial at the European Society of Cardiology Heart Failure Congress in Greece.

Background

Juventas Therapeutics’ JVS-100 is a non-viral gene therapy which results in cells expressing stromal cell-derived factor 1 (SDF-1). The therapy has been shown to improve cardiac function in patients with advanced heart failure more than a decade after having a heart attack. SDF-1 has been shown in previous studies to promote tissue repair through activating an endogenous stem cell repair pathway.

BioCardia’s Helix™  is a steerable, two-catheter system that enables delivery of biologic therapies such as JVS-100 to the heart muscle from within the chamber of the heart. BioCardia claims unique performance advantages for its system, including crossing the aortic valve over a wire to prevent damage; enhanced navigation using the company’s steerable Morph® guiding catheter; a helical needle which screws into the myocardium for stable delivery; and the use of contrast at the base of the needle to confirm tissue engagement.

STOP-HF is a double-blinded, placebo-controlled, multi-center trial of Juventas’ non-viral DNA plasmid therapy, “JVS-100” in patients with symptomatic heart failure. Interim top-line four-month efficacy data demonstrated dose-dependent improvements in echocardiographic parameters and biomarkers for patients having received the treatment. Specifically, a subpopulation of high risk patients who received the high dose of JVS-100 (30 mg) demonstrated improvements relative to placebo in left ventricular end systolic volume (LVESV) p<0.05, left ventricular end diastolic volume (LVEDV) p=0.11, left ventricular ejection fraction (LVEF) p=0.23 and NTproBNP levels (a simple blood test that predicts the risk of worsening heart failure in patients with known cardiovascular disease) p=0.12.

The big news for BioCardia is that the therapy is delivered by its Helix™ Transendocardial Delivery System (known as Helical Infusion System/HIS in the trial). A total of 93 patients were enrolled in this trial at 16 academic and community hospitals in the United States.

Patients enrolled in STOP-HF had a prior history of a heart attack and years later developed symptomatic heart failure as defined by an ejection fraction less than 40 percent and poor quality of life and exercise tolerance as measured by the Minnesota Living with Heart Failure Questionnaire (MLWHQ) and six minute walk distance (6MWD), respectively. Patients were randomized to placebo or treatment with two different doses of JVS-100. In addition to safety, data for several efficacy endpoints is being collected at four months and one-year post-treatment including heart failure related hospitalizations, major adverse cardiac events, and changes in 6MWD, MLWHQ scores, and ejection fraction. 

In the trial, the Helix system performed well, with no instances of treatment emergent mortality, myocardial infarction, aortic dissection, arterial spasm or stroke, all major adverse events believed to have occurred in other clinical trials using other transendocardial and intracoronary biotherapeutic delivery platforms.

Company comments

“BioCardia congratulates the Juventas team and the talented clinical investigators on the performance of a rigorous clinical program, and we are honored that Juventas selected the Helix system for use in its STOP-HF trial,” said BioCardia Chief Executive Officer Peter Altman. “The Helix system’s growing safety experience adds value to therapeutic development programs, enhancing the probability of both technical and commercial success for biotherapeutic cardiovascular regenerative medicine products.”

Source: BioCardia, Inc., Business Wire

published: May 20, 2014 in: Cardio, Clinical Studies/Trials

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