CLEVELAND, Dec. 9, 2013 /PRNewswire-USNewswire/ -- Juventas Therapeutics, a privately-held, clinical-stage company developing novel regenerative therapies for treatment of cardiovascular disease, announced today that the company has initiated enrollment of RETRO-HF, a Phase I/II clinical trial that will assess the safety and efficacy of JVS-100 administered by retrograde infusion to patients with symptomatic heart failure. The 12-person open-label, dose-escalation Phase I component will evaluate safety of performing retrograde infusion of JVS-100 dosed at 30 mg or 45 mg. It will be immediately followed by a 60-patient randomized, double-blinded, placebo-controlled Phase II component in which patients will receive JVS-100 at 30 mg or 45 mg or a placebo infusion. Enrollment is underway and Dr. Amit Patel, Director of Cardiovascular Regenerative Medicine at University of Utah, successfully dosed the first patient.
JVS-100, the Company's lead product, is a non-viral gene therapy that encodes stromal cell-derived factor 1 (SDF-1). It has been shown to promote tissue repair through recruitment of a patient's own stem cells to the damaged organ. Juventas recently completed enrollment of two Phase II trials. The 93-patient STOP-HF trial will measure safety and efficacy of JVS-100 administered by endo-myocardial injection to patients with symptomatic heart failure and the 48-patient STOP-CLI trial will measure safety and efficacy for JVS-100 in patients with critical limb ischemia.
Retrograde infusion is a minimally invasive technique in which a catheter is inserted through the venous system into a patient's coronary sinus. A balloon is then inflated in order to temporarily block blood flow out of the heart so that the gene therapy can be infused directly into the heart. The technique employs commonly used balloons and catheters that are already approved for delivery of non-biologic agents to the heart.
"RETRO-HF represents the first clinical trial in the world in which retrograde infusion is being used for delivery of a gene therapy to the heart" states Dr. Patel. "The concept has been discussed and studied pre-clinically for years and it is a significant step forward for the field to have the first successful clinical case behind us. Retrograde infusion has the potential to be a widely-used, cost-effective procedure to safely deliver biologics to the heart."
The RETRO-HF trial will enroll patients with symptomatic ischemic heart failure. Heart failure affects nearly six million Americans, with an estimated cost to our healthcare system in excess of $37 million. The patients targeted for treatment with JVS-100 have typically progressed to a stage where their options are limited to a heart transplant or assist device.
"The STOP-HF and RETRO-HF trials are designed to study JVS-100 in patients whose heart failure symptoms have progressed to a point that their health is deteriorating despite receiving optimal medical management," states Marc Penn, M.D., Ph.D. Founder and Chief Medical Officer for Juventas and Director of Cardiovascular Research at Summa Cardiovascular Institute, Summa Health System. "There is a large population of patients in the United States and world-wide who even with currently available therapies have significant physical limitations in their daily lives and are at high risk for worsening of their heart failure. We are very excited to see our seminal findings of a decade ago reach patients in need and look forward to next year when we are targeting clinical data from more than 150 heart failure patients enrolled in our Phase II heart failure program."
About Juventas Therapeutics
Juventas Therapeutics, headquartered in Cleveland, Ohio, is a privately-held clinical-stage biotechnology company developing regenerative therapies for cardiovascular disease. Founded in 2007 with an exclusive license from the Cleveland Clinic, Juventas has transitioned its therapeutic platform from concept to mid-stage clinical trials for treatment of heart failure and critical limb ischemia. Investors include New Science Ventures, Triathlon Medical Venture Partners, Takeda Ventures, Early Stage Partners, Fletcher Spaght Ventures, Venture Investors, Reservoir Venture Partners, The Cleveland Clinic, Glengary, North Coast Angel Fund, X Gen Ltd., JumpStart Inc., and Blue Chip Venture Co. The company has received support through the Ohio Third Frontier funded Cleveland Clinic Ohio BioValidation Fund, Global Cardiovascular Innovation Center and Center for Stem Cell & Regenerative Medicine.
The company's lead product, JVS-100 encodes Stromal–cell Derived Factor 1 (SDF-1). JVS-100 is a non-viral DNA plasmid that has been shown to protect and repair tissue following organ-injury in a broad range of pre-clinical disease models. The first clinical targets will be treatment of patients with symptomatic heart failure and critical limb ischemia. The company has demonstrated in pre-clinical models that JVS-100 therapy has the potential to repair tissue-damage following ischemic injury by recruiting the body's own stem cells to the damaged region, the prevention of cardiac cell death and promotion of new blood vessel growth in the heart. In addition to heart failure, Juventas is also developing JVS-100 for additional indications including acute myocardial infarction, chronic angina, and muscle regeneration.