FDA authorizes Juventas' JVS-100 Phase II clinical trial for critical limb ischemia

Juventas Therapeutics, a clinical-stage regenerative medicine company developing novel therapies for cardiovascular disease, announces that the U.S. Food and Drug Administration (FDA) authorized commencement of a Phase II clinical trial evaluating the efficacy and safety of its lead product, JVS-100, for the treatment of critical limb ischemia (CLI).  JVS-100 encodes Stromal-cell Derived Factor 1 (SDF-1), which has been shown in several pre-clinical studies to promote tissue repair through activation of natural stem-cell repair pathways, promotion of new blood vessel formation and prevention of on-going cell death.  The new trial builds on the JVS-100 cardiovascular program that includes a U.S. Phase I heart failure (HF) clinical trial for which the Company recently completed enrollment of 17 patients.

"Juventas is excited to extend its clinical pipeline to include treatment of patients with CLI," stated Rahul Aras, Ph.D., President & CEO for Juventas Therapeutics.  "With our heart failure and critical limb ischemia programs moving into mid-stage clinical trials, the Company is focused on leveraging its regenerative medicine platform to address a range of cardiovascular indications with the goal of providing novel therapies to patients with limited options."

The randomized, placebo-controlled study will evaluate the efficacy and safety of JVS-100 in 48 patients (36 JVS-100 treated; 12 placebo) with Rutherford Class IV or V CLI.  The clinical trial will measure several efficacy endpoints including time to amputation, transcutaneous oximetry (TcPO2), Ankle-Brachial Index (ABI) and ulcer closure.  

"Critical limb ischemia is a devastating disease for which there is currently no FDA-approved drug for treatment," said Douglas Losordo, M.D., Principal Investigator for the trial and Director of the Feinberg Cardiovascular Research Institute at the Northwestern University Feinberg School of Medicine.  "Novel regenerative therapies, such as JVS-100, have the potential to restore blood flow and improve outcomes for these patients."

There are approximately 3 million U.S. citizens with critical limb ischemia.  The hospital treatment costs for these patients exceed $10 billion with average in-patient charges surpassing those for stroke or heart attack.  Within a year of CLI diagnosis, up to 50% of patients will undergo an amputation and 25% will die.

The Juventas therapeutic platform is based on discoveries made nearly a decade ago by Marc Penn, M.D., Ph.D., Director of the Bakken Heart-Brain Institute at the Cleveland Clinic and Juventas' Chief Medical Officer.  Dr. Penn demonstrated that SDF-1 is a strong chemo-attractant for hematopoietic stem cells (HSCs), with the potential to promote cardiac repair when delivered to the heart hours to months following a heart attack.  These findings were published in 2003 in The Lancet and have now been validated by several independent laboratories.  The SDF-1 repair pathway is conserved in most end organ systems, including the heart, peripheral vasculature, kidney, skin, liver and eye.

"Our work on SDF-1 originates from the hypothesis that we all have a natural stem cell based repair process that attempts to repair organs following injury," states Dr. Penn.  "We believed that if we could identify the molecular factors that activate this pathway, they could be delivered at a time post-injury to promote tissue preservation.  SDF-1 has proven to be a key player in driving repair and we are excited to see our findings translating to the clinic with the potential to help patients".