Pluristem Receives Clearance from Germany to Initiate its Multinational Phase III Trial in Critical Limb Ischemia Targeting Early Conditional Marketing Approval
GlobeNewswire•January 17, 2017
PLX-PAD cells could potentially obtain early conditional marketing approval in Europe via the Adaptive Pathways pilot project based on positive interim efficacy data from first 125 patients
This approval joins those from the U.S. FDA and U.K. for this 250-patient Phase III Trial
Germany is Western Europe’s largest market for Critical Limb Ischemia
Enrollment expected to commence in the first half of 2017
HAIFA, Israel, Jan. 17, 2017 (GLOBE NEWSWIRE) -- Pluristem Therapeutics Inc. (PSTI) (PSTI), a leading developer of placenta-based cell therapy products, today announced that Germany’s health regulatory agency, the Paul Ehrlich Institute (PEI), has cleared Pluristem to begin enrollment in Germany for its pivotal Phase III trial of PLX-PAD cells to treat Critical Limb Ischemia (CLI) in patients who are unsuitable for revascularization. The trial was recently cleared by the United States' Food and Drug Administration (FDA) and the United Kingdom's Medicines & Healthcare products Regulatory Agency (MHRA).
PLX-PAD cells have been selected by the European Medicines Agency (EMA) to be developed for CLI via the Adaptive Pathways pilot project. Under this regulatory pathway, the multinational Phase III trial may potentially lead to early marketing approval of PLX-PAD for CLI in Europe, based on interim efficacy data from the first 125 patients that have completed a follow-up of 12 months. Data from the entire 250 patients will be submitted to the U.S. FDA for a Biologics License Application (BLA) targeting commercialization, and to the EMA to apply for full marketing approval.
“The potential for early marketing approval is an important achievement for us, and we are glad that we may help bring hope to CLI patients with no good treatment options. It is gratifying to see that regulatory authorities around the world are working to accelerate patient access to innovative treatments for medical conditions, such as CLI, that are unmet medical needs,” stated Pluristem Chairman and CEO, Zami Aberman.
The global Phase III trial will evaluate PLX-PAD cells in the treatment of CLI in a double-blind, randomized, placebo-controlled trial. An estimated 250 patients with CLI Rutherford Category 5, who are unsuitable candidates for revascularization, will be enrolled. Patients will be treated with 300 million cells or placebo, injected twice intramuscularly (IM) two months apart. The primary endpoint will be time to amputation or death, allowing for a survival analysis which enables collection of more data during the trial thereby significantly reducing the number of patients needed, to allow statistically significant results from a trial of this size.
About Critical Limb Ischemia
In CLI, fatty deposits block arteries in the leg, leading to greatly reduced blood flow. This causes leg pain at rest, non-healing ulcers and gangrene. Patients with CLI are at high risk for limb amputation and death within a year of diagnosis. While some conservative treatments exist to relieve pain, and provide localized ulcer care, most patients will ultimately need a revascularization procedure. CLI patients who cannot undergo revascularization procedures are left with poor treatment options, and have a severe unmet medical need. With over 700,000 citizens suffering from critical limb ischemia, Germany represents the single largest Western European market, as per the Sage group (2010). This reflects the fact that Germany has the highest regional prevalence of diabetes, as well as the greatest number of elderly citizens, which are major risk factors for CLI.
About the Adaptive Pathways Pilot Project
The purpose of EMA’s Adaptive Pathways pilot project is to shorten the time it takes for innovative medicines to reach patients with serious conditions that lack adequate treatment options. The pathway is open to clinical programs in early stages of development only. After a therapy is selected for the program, the Adaptive Pathways group conducts high level discussions and provides guidance to the applicant regarding the formal regulatory processes that precede a trial, targeting early approval and further expansion of the indications.
