RANCHO CORDOVA, Calif., Dec. 05, 2016 (GLOBE NEWSWIRE) -- Cesca Therapeutics Inc. (NASDAQ: KOOL ), a market leader in automated cellular processing and point-of-care autologous cell-based therapeutics, today announced that on November 21, 2016 the Company has submitted an Investigational Device Exemption (IDE) Supplement to the U.S. Food and Drug Administration (FDA) for its previously approved pivotal trial. The study is designed to demonstrate the safety and effectiveness of the Company’s point-of-care, SurgWerks™ system, in the treatment of late stage, “no-option” and “poor-option”, CLI patients. The Company expects the FDA to submit its response to the IDE supplement within 30 days from submission.
“This IDE supplement is the result of extensive review of current literature, as well as discussions with independent advisors, key opinion leaders and the FDA. After taking everything into consideration, from scientific to clinical to reimbursement, we believe that amputation free survival, a hard clinical end-point, gives us the best chance for a successful pivotal CLI trial outcome, pre-marketing approval (PMA), and path to commercialization,” said Dr. Xiaochun “Chris” Xu, Cesca’s Interim Chief Executive Officer. “We remain dedicated to the development of safe and effective point-of-care autologous cell therapies, with CLI being just one of Cesca’s many opportunities in the space. We have several other vascular and orthopedic cellular therapies in our pipeline and are committed to opening up multiple clinical avenues to unlock the immense value and drive the success of the Company.”
Designed to demonstrate the safety and efficacy of the Company’s point-of-care SurgWerks™ system, the revised Phase III trial design will focus on the difference in AFS measured over 12 months between a treatment arm and a sham control arm. The general design of the study will include 1:1 randomization with a 12 month follow-up program. The estimated sample size needed to successfully demonstrate safety and a statistically powered efficacy result is 362 patients (181 patients in the treatment arm and in the sham control arm, factoring in an estimated dropout rate of 10%).
The supplement also details changes to the protocol designed to improve patient enrollment. These include expanding the cohort to include both “no option” and “poor option” Rutherford Category 4 and 5 CLI patients, using a sham control arm vs placebo, restricting severe diabetic R5 subjects, and stream-lining follow-up procedures.
The trial also includes clinically significant secondary endpoints, such as wound healing and improvement in rest pain, designed to show quality-of-life improvements, which are relevant to receiving reimbursement approval from The Centers for Medicare and Medicaid Services (CMS).
About Cesca Therapeutics Inc. Cesca Therapeutics Inc. ( www.cescatherapeutics.com ) is engaged in the research, development, and commercialization of cellular therapies and delivery systems 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 therapeutics. These include:
- The SurgWerks™ System (in development) - a proprietary system comprised of the SurgWerks Processing Platform, including devices and analytics, and indication-specific SurgWerks Procedure Kits for use in regenerative stem cell therapy at the point-of-care for vascular and orthopedic diseases.
- The CellWerks™ System (in development) - a proprietary cell processing system with associated analytics for intra-laboratory preparation of adult stem cells from bone marrow or blood.
- The AutoXpress® System (AXP ®) - a proprietary automated device and companion sterile disposable for concentrating hematopoietic stem cells from cord blood.
- The MarrowXpress™ System (MXP™) - a derivative product of the AXP and its accompanying sterile disposable for the isolation and concentration of hematopoietic stem cells from bone marrow.
- The BioArchive® System - an automated cryogenic device used by cord blood banks for the cryopreservation and storage of cord blood stem cell concentrate for future use.
- Manual bag sets for use in the processing and cryogenic storage of cord blood.
Forward-Looking Statement The statements contained herein may include statements of future expectations and other forward-looking statements, including statements concerning Cesca Therapeutics’ ability to demonstrate safety and efficacy of its CLI therapy from its clinical study, the timing of the FDA’s response to the IDE supplement, the ability to successfully develop any particular therapy, and the receipt of any related reimbursement approval, all of which 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. A more complete description of 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.
Company Contact: Cesca Therapeutics Inc. ir@cescatherapeutics.com Investor Contact: The Ruth Group Lee Roth / Tram Bui 646-536-7012 / 7035 lroth@theruthgroup.com / tbui@theruthgroup.com
