RANCHO CORDOVA, Calif., Jan. 05, 2017 (GLOBE NEWSWIRE) — Cesca Therapeutics Inc. (NASDAQ:KOOL), a market leader in automated cellular processing and point-of-care autologous cell-based therapies, announced today that the Company received approval from the U.S. Food and Drug Administration (FDA) for significant revisions to the Company’s pivotal study for treatment of Critical Limb Ischemia (CLI). The CLI clinical trial is designed to demonstrate the safety and efficacy of the Company’s point-of-care SurgWerks™ system for the treatment of CLI patients with limited or no treatment options. The study was first approved by FDA in June 2015, but was not initiated at that time pending funding.
The changes approved by the FDA are intended to increase patient enrollment by expanding the patient pool from Rutherford Category 5 patients only, to also include Rutherford Category 4 patients, or patients with a less severe form of the disease. In addition, the study population has been expanded to include patients who are poor candidates for either surgery or endovascular therapies, as opposed to only those patients with no viable treatment options. Finally, the FDA accepted to change the control arm from a placebo, which was both invasive and carried measureable safety risk, to a much less risky sham procedure.
Although the primary endpoint remains Amputation Free Survival (AFS), a standard in all current CLI drug and biologic trials, the FDA recognized the need to increase the available patient population in order to properly power the clinical trial. Compared to the initial study design, the sample size was increased from 224 to 362 patients, which in turn allowed the superiority margin to be reduced from 20% to 13%. We believe that the reduced superiority margin is both achievable and clinically relevant. Due to the increased patient pool, it is expected that enrollment rates will be better than previous CLI clinical trials.
Other improvements to the study include a reduction in study-related testing, improved statistical methods, and streamlined study oversight, all of which are intended to reduce costs and improve patient recruitment and investigator participation.
“We are pleased the FDA approved our IDE supplement. The changes, especially expanding the patient population and the improved statistical methods, allow us to strike an important balance between patient enrollment rates, superiority margin, cost and time”, said Dr. Xiaochun “Chris” Xu, Cesca’s Interim CEO. “With this compelling trial design we look forward to finding strategic partners to help develop and commercialize the SurgWerks™ CLI system. We remain committed to advancing our innovative point-of-care platforms and devices and making Cesca the leading solution provider in the broader cell therapy and healthcare space.”
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.
The statements contained herein may include statements of future expectations and other forward-looking statements that are based on management’s current views, beliefs 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. These statements include those concerning the effect of the changes approved by the FDA as they relate to patient enrollment, safety risk, efficacy, cost and achievability and those concerning the ability of the Company to finding strategic partners for development and commercialization. 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.
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