RANCHO CORDOVA, Calif., June 16, 2015 (GLOBE NEWSWIRE) -- Cesca Therapeutics Inc. (Nasdaq:KOOL), an autologous cell-based regenerative medicine company, today announced that the U.S. Food and Drug Administration (FDA) has granted full approval for an Investigational Device Exemption (IDE) for the Company's pivotal clinical trial, named the CLIRST III trial, to evaluate Cesca's SurgWerks™-CLI and VXP System for the treatment of patients with late-stage, no-option, critical limb ischemia (CLI). This debilitating and progressive disease results in approximately 70,000 major limb amputations, per year, in the United States alone (Sage Group, Aug, 2013).
The CLIRST III pivotal trial will involve 204 subjects at up to 60 hospital sites in the United States with additional enrollment to be conducted outside the U.S. up to the anticipated total subject enrollment of 224. The study will be a 3:1 randomized, double blinded, placebo-controlled trial, having an adaptive interim analysis for repowering (if necessary), and with a primary endpoint of major amputation-free survival. Additional trial characteristics include the use of an independent, centralized panel of physicians for adjudicating/determining both the inclusion of a prospective subject, as well as determining the final outcome of an enrolled subject at the 12 month follow-up period. The trial is understood to be the first such pivotal/Phase III trial for this disease to use a blinded review committee to reduce potential bias in both physicians and subjects. Reduction of bias is important for better assessment and objective determination of whether an amputation is necessary.
Cesca's cell therapy technology for this indication, known as the SurgWerks-CLI and VXP System and intended to facilitate limb salvage, is regulated as a Class III medical device and has been determined to be CMS Category B by the FDA. Data from the pivotal trial will be used in support of a future premarket approval (PMA) application. The CLIRST III trial follows a successful feasibility study conducted in India.
"This CLIRST III trial approval is a significant milestone for us as we become a fully integrated cell therapy company. It is also an important development for patients who are facing the devastating loss of a limb from advanced critical limb ischemia. We are pleased to have received the FDA's approval without condition, and look forward to moving ahead with enrollment. This approval also reinforces our view that Cesca has a unique advantage in being able to run highly cost effective pilot studies within its India operation that can in turn result in U.S. pivotal trial clearances. Cesca is proud to join the ranks of a very select handful of pivotal stage regenerative medicine companies," said Ken Harris, Cesca's President and head of clinical development.
Prior to enrollment of the first subject, the Company will, among other things, request Medicare coverage for routine subject treatment costs, as allowed under the Medicare Prescription Drug, Improvement and Modernization Act of 2003 (ACT). Effective January 1, 2015, ACT allows reimbursement for routine care and service items that are incurred as part of an IDE study involving a Category B device.
Robin C. Stracey, Chief Executive Officer of Cesca Therapeutics added, "I am very proud of what our clinical team has accomplished under Ken's leadership. In part as a result of extensive dialog with the FDA's clinical trials specialists, the group has developed clinical guidelines that will not only better serve medical professionals running the trials but, most importantly, benefit the patients who suffer the consequences of CLI. We look forward to getting the trial underway."
About Cesca Therapeutics Inc.
Cesca Therapeutics Inc. (www.cescatherapeutics.com) is engaged in the research, development and commercialization of autologous cell-based therapeutics for use in regenerative medicine. The Company is a leader in the development and manufacture of automated blood and bone marrow processing systems that enable the separation, processing and preservation of cell and tissue therapy products. These include:
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SurgWerks™; proprietary stem cell therapy point-of-care kits for the treatment of vascular and orthopedic indications that integrate the following indication specific elements:
- Cell harvesting
- Cell processing and selection
- Cell diagnostics
- Cell delivery
- CellWerksTM; a proprietary stem cell laboratory kit for the processing of target cells used in the treatment of oncological and hematological disorders.
- The AutoXpress® (AXP); a proprietary automated device, along with companion sterile blood processing disposables, for the harvesting of stem cells from cord blood.
- The MarrowXpress® (MXP); a derivative product of the AXP and its accompanying disposable bag set, for the isolation and concentration of stem cells from bone marrow. Self-powered and microprocessor-controlled, the MXP contains flow control optical sensors that volume-reduces blood from bone marrow to a user defined volume in 30 minutes while retaining over 90% of mononuclear cells (MNCs).
- The Res-Q™ 60 (Res-Q); a point-of-care system designed for the preparation of cell concentrates, including stem cells, from bone marrow aspirates for bone marrow concentrate (BMC) and whole blood for platelet rich plasma (PRP).
- The BioArchive® System; an automated cryogenic device, used by cord blood stem cell banks in more than 30 countries, for cryopreservation and archiving of cord blood stem cell units for transplant.
Forward Looking Statement:
The statements contained herein may include statements of future expectations and other forward looking statements that are based on management's current views and assumptions and involve known and unknown risks and uncertainties that could cause actual results, performance or events to differ materially from those expressed or implied in such statements, including our ability to initiate and prosecute the pivotal CLIRST III trial, our ability to obtain efficacy consistent with prior CLI studies, continued FDA approval, Medicare reimbursement approval, timing of the Company's future submission of IDE, PMA and/or Medicare reimbursement applications, or amendments to such applications, and outcomes from such submissions. Further, clinical trial outcomes are not predictable, and results may vary from the Company's expectations, including the start of any such clinical trials, patient follow up issues, and costs associated with such trials. Further description of other risks that could cause actual events to differ from the outcomes predicted by Cesca Therapeutics' forward-looking statements is set forth under the caption "Risk Factors" in Cesca Therapeutics annual report on Form 10-K and other reports it files with the Securities and Exchange Commission from time to time, and you should consider each of those factors when evaluating the forward-looking statements. Contact: Cesca Therapeutics Inc. http://www.cescatherapeutics.com. Cesca may, at its discretion, choose to publish on-going interim notifications, requests for further information as received from the FDA, Medicare or equivalent foreign agencies, but as a general policy only makes announcements regarding material or significant information, such as filing of applications, approvals, initiation of studies, and conclusions.