Cesca Therapeutics Lobbies for IDE for Critical Limb Ischemia Trial

Rancho Cordova, Calif.-based Cesca Therapeutics Inc., an autologous cell-based regenerative medicine company, announced today that it recently participated in a pre-submission IDE (investigational device exemption) teleconference with the U.S. Food and Drug Administration’s (FDA) Center for Biologics Evaluation and Research. The call was to discuss the company’s proposed pivotal clinical trial design for the treatment of critical limb ischemia (CLI) using its CLIRST (critical limb ischemia rapid stem cell therapy) treatment protocol delivered via the company’s proprietary Surgwerks-CLI kit.

“We are quite pleased with the feedback we received from the FDA regarding our proposed trial design and our conclusion that our Surgwerks-CLI kit as outlined in our pre-submission is properly designated a class III product that follows an IDE-PMA (pre-market approval) regulatory pathway,” said CEO Matthew Plavan. “Good trial design and critical analysis of pivotal trial endpoints leads to better clinical outcomes. With that in mind, we believe our call with the FDA bodes well on our upcoming IDE submission.”

CLIRST is Cesca’s proprietary method and bedside platform technology using a patient’s own bone marrow stem cells and is in the clinical development stage. Cesca’s intended goal in its planned pivotal trial is to demonstrate prevention or delay in major limb amputation in Rutherford 5 patients, as compared to the standard of care. This therapy is targeted as an alternative to major limb amputation having a current cost of care in the United States of approximately $15 billion.

The Surgwerks-CLI therapy kit contains optimized stem cell harvesting, selection, and delivery disposables and can be completed on a patient in less than 60 minutes in the operating room with mild sedation, according to Cesca. Surgwerks-CLI patient cells never leave the operating room, meet the FDA definition for “minimally manipulated” cells, and are delivered to the patient in a single procedure.

The company sponsored a Phase 1B clinical trial for CLIRST which demonstrated safety and early efficacy results treating no-option patients suffering from CLI. The Safety and Efficacy of Autologous Bone Marrow Mononuclear Cells in Patients With Severe Critical Limb Ischemia trial was a single center, unblinded, single arm study in 17 patients with late-stage CLI. The original study results were announced in January 2014. The trial achieved both its primary safety endpoint and secondary efficacy endpoints at an average of 26 months having no serious adverse events determined to be related to the therapy, and achieving >82 percent in the protocol amputation free survival rates, and statistically significant improvement in pain reduction, 6-minute walking distance, open wound healing and, vasculogenesis over the 12 month follow-up period in the treated leg.

Critical limb ischemia afflicts an estimated 2 million people combined in the United States, European Union and Indian sub-continent, and results in approximately 500,000 amputations each year. The overall prevalence and incidence in the United States increases with age and diabetes status and five year mortality rates post limb amputation reaches nearly 50 percent.

The company expects to submit an IDE application for its U.S.-based CLI pivotal clinical trial with the FDA in October of 2014 and to fund its trial with a portion of the proceeds received from its recently completed equity financing of $11.3 million.