FDA Approves Commencement of Phase III Pivotal Trial as Amended
Requires Additional Validation of TcPO2 as a Surrogate to Support Subsequent PMA Approval
RANCHO CORDOVA, Calif., Aug. 01, 2016 (GLOBE NEWSWIRE) -- Cesca Therapeutics Inc. (NASDAQ: KOOL ), an autologous cell-based regenerative medicine company, is today updating investors on the status of the U.S. Food and Drug Administration’s (FDA) review of its Investigational Device Exemption (IDE) Supplement for a phase III pivotal trial, designed to demonstrate the safety and effectiveness of the Company’s SurgWerks TM system for the treatment of late stage, no option, critical limb ischemia (CLI) patients.
In addition to a number of protocol changes intended to improve patient enrollment and study flow, the IDE Supplement, submitted at the end of May, proposed a change in the primary efficacy endpoint from Amputation Free Survival (AFS) to Change in Transcutaneous Oxygen Pressure (TcPO2). The Company believes that published literature in peer reviewed journals over many years has shown a strong correlation between improvements in TcPO2 over time and limb salvage, a correlation which has since been reinforced by Cesca’s own feasibility study data. The Company had previously concluded that Amputation-Free Survival (AFS) would not be practical as a primary endpoint for the study because it couples limb-related outcomes with all-cause mortality, a characteristic that unreasonably burdens the statistical analysis necessary to specifically demonstrate treatment effectiveness in preventing amputations among late stage CLI patients.
The FDA has approved the changes in Cesca’s pivotal trial design as proposed in the IDE Supplement, including the use of Change in TcPO2 as the primary measure of efficacy. There are now no regulatory impediments to the Company enrolling patients and beginning the trial. However, in its feedback, the FDA also indicated that regardless of the outcome of the study, it would require the Company to further validate TcPO2 as a surrogate for clinical outcome for the proposed indication prior to granting marketing (PMA) approval. In light of this, the Company is now engaged in a follow-on dialog with the FDA to explore various alternative pathways to securing a PMA. “We are obviously very pleased to have regulatory approval to commence our Phase III pivotal clinical trial incorporating the changes detailed in our IDE Supplement,” said Robin Stracey, Cesca’s CEO. “Before we do so, however, we want to also be sure we have a clear and unambiguous pathway to marketing approval for the SurgWerks TM system once the study has concluded. We are therefore actively engaged in a dialog with the Agency to understand and evaluate alternative pathways to a PMA, including possible adaptation of our SurgWerks program to encompass earlier stage CLI patients, a change that could substantially increase our overall market opportunity. There may be several rounds of discussion with the FDA as we further refine our plan,” he added.
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:
- T h e 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.
- T h e 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.
- T h e AutoXpress® System (AXP®) - a proprietary automated device and companion sterile disposable for concentrating hematopoietic stem cells from cord blood.
- T h e 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.
- T h e 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.
- M anual bag sets for use in the processing and cryogenic storage of cord blood.
Forward-Looking Statements and Safe Harbor Statement under the Private Securities Litigation Reform Act of 1995 This press release includes statements of future expectations and other forward-looking statements regarding, among other things, the Company’s SurgWerks TM system and the phase III pivotal clinical trial, that are within the meaning of the “safe harbor” provisions of the Private Securities Litigation Reform Act of 1995. These statements are based on management’s current views and assumptions, speak only as of the date hereof and are subject to change. Forward-looking statements can often be identified by words such as “anticipates,” “belief,” “intend,” “may,” “could,” “would,” “potential,” “continue,” and similar expressions and include, but are not limited to, statements regarding research, development and commercialization of the SurgWerks TM system, the clinical literature concerning TcPO2, the Company’s understanding of its feasibility study data, the initiation of Phase III pivotal clinical trial, if at all, the timing for the enrollment of patients, if any, the outcome of the trial, the overall market opportunity of the SurgWerks TM system, the status of any dialog with the FDA, and the progress, if any, towards PMA approval. These forward-looking statements are not guarantees of future results and are subject to known and unknown risks and uncertainties that could cause actual results, performance or events to differ materially and adversely 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 these forward-looking statements is set forth under the caption "Risk Factors" in our Annual Report on Form 10-K, in our Quarterly Reports on Form 10-Q, and in other reports filed with the Securities and Exchange Commission from time to time, and you should also consider each of those factors when evaluating the forward-looking statements. We undertake no obligation to revise or update publicly any forward-looking statements for any reason, except as required by law.
C o mpany Contact: Cesca Therapeutics Inc. ir@cescatherapeutics.com
I n v e stor Contact : The Ruth Group Lee Roth / Tram Bui 646-536-7012 / 7035 lroth@theruthgroup.com / tbui@theruthgroup.com
