RANCHO CORDOVA, Calif., Jan. 2, 2015 (GLOBE NEWSWIRE) -- Cesca Therapeutics Inc. (Nasdaq:KOOL), an autologous cell-based regenerative medicine company, today announced that it has received a letter from the U.S. Food and Drug Administration responding to the Company's investigational device exemption ("IDE") application for its pivotal CLIRST III clinical trial. The IDE application, filed on November 20, 2014, sought approval to begin a pivotal, randomized, placebo-controlled study of 224 late stage CLI patients at up to 60 clinical sites in the United States and India. In its letter, the FDA noted several deficiencies in the Company's submission, requesting, among other things, additional information on characterization of the output of the SurgWerks™ - CLI and VXP system, a plan for testing device output for conformity to predefined intra-operative release criteria prior to administration, modifications to the management of potential adverse events and a strengthening of the language in the risk clauses in the Informed Consent Document.
"The clarity of the feedback from the FDA is very helpful and we are now in the process of addressing the various points raised in their letter," commented Robin Stracey, Chief Executive Officer of Cesca. "We anticipate completing that work and submitting our amended IDE application sometime in February 2015," he continued.
In its letter, the FDA noted no deficiencies relating to the Company's overall trial design, including the patient population size of 224, the 3:1 randomization and the use of a blinded independent committee for determination of amputation decisions. The Company believes that, upon FDA approval, its study will be one of the first CLI trials to use a blinded independent committee review to assess the primary endpoint. Cesca included this element in its trial design specifically to overcome the trial bias observed in previous pivotal CLI trials with major amputation free survival rates as the primary endpoint.
Ken Harris, Cesca's President and the leader of the Company's clinical programs, commented, "Taken as a whole, we believe the questions and comments received from the FDA confirm that we have designed a sound clinical trial. We look forward to addressing the Agency's remaining concerns and resubmitting our application. If the amended application is approved by the FDA, we hope to be able to begin enrolling patients as early as April 2015."
CLI is the most severe form of Peripheral Arterial Disease ("PAD") affecting over two million patients in the United States. It is often associated with chronic foot and leg ulcers, leading to approximately two hundred thousand amputations per year. A diagnosis of CLI statistically results in a 25% mortality rate and a 25% amputation rate within one year.
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 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 FDA approval, timing of the Company's submission of IDE 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.