Basking Ridge-based Caladrius Biosciences, Inc., a development-stage biopharmaceutical company with multiple technology platforms targeting autoimmune and select cardiovascular indications, announced that the first patient has been dosed in the company’s Phase 2 clinical trial in Japan with its proprietary CD34 cell therapy (CLBS12) for the treatment of no-option critical limb ischemia (“CLI”).
This trial is a 35-patient prospective, randomized, controlled, multicenter study. Patients randomized to treatment will be dosed with autologous G-CSF-mobilized peripheral blood-derived CD34 cells (CLBS12) through intramuscular injection, in addition to receiving standard of care pharmacotherapy. Patients randomized to the control arm will receive standard of care pharmacotherapy alone. The primary endpoint is time to continuous CLI-free status, defined as two consecutive monthly visits in which the patient is determined by an independent adjudication committee to be “CLI-free”.
Based on discussions with the Japanese regulatory authorities and in accordance with the Japanese Pharmaceuticals and Medical Devices Law passed in November 2014, the Company believes that, with favorable results, the study will qualify for consideration of conditional approval for CLBS12 in Japan for the treatment of no-option CLI. The noted legislation expedites the development and commercialization of regenerative medicine therapies and grants conditional approval for regenerative medicines that demonstrate evidence for safety and the likelihood for efficacy.
“We are delighted to have dosed the first patient in this pivotal trial of CLBS12 in no-option CLI in Japan. As we previously reported, the study protocol and Chemistry, Manufacturing and Controls (“CMC”) strategy for the trial both were constructed in consultation with the Japanese Pharmaceutical and Medical Devices Agency. Based on those discussions, it was agreed that, should this trial be successful, it will qualify CLBS12 for consideration of early conditional approval in Japan for this indication,” said David J. Mazzo, Ph.D., President and Chief Executive Officer of Caladrius. “The study has a clinically relevant yet practical endpoint, time-to-CLI-free status, that encompasses a broader spectrum of improvement than do time-to-amputation or amputation-free survival, which are the historical endpoints in CLI studies.”
Commenting on the study, Douglas W. Losordo, M.D., FACC, FAHA, Senior Vice President of Clinical Medical and Regulatory Affairs and Chief Medical Officer of Caladrius, noted, “Our enthusiasm for this program is based on previous studies of autologous CD34 cell therapy for no-option CLI patients in both Japan and the U.S. suggesting that CD34 cell therapy was safe, led to improvement in CLI-free status and improved amputation-free survival.”1,2,3
“With favorable data, we expect to pursue a commercial partnership for CLBS12 as a treatment for no-option CLI in Japan. We view CLI as the entry point to explore the broader applicability of CD34 therapy, which could potentially offer us significant opportunities across multiple underserved cardiovascular indications,” added Dr. Mazzo.
To access more business news, visit njbmagazine.comRelated Articles: