Cesca Therapeutics Inc (NASDAQ: KOOL): FDA Approval of Revisions for Pivotal Study for Treatment of CLI

Equities Staff  |

Cesca Therapeutics, Inc (NASDAQ: KOOL) is engaged in the research, development, and commercialization of cellular therapies and delivery systems for use in regenerative medicine.

On January 5, 2017, KOOL’s Critical Limb Ischemia (CLI is a severe obstruction of the arteries which markedly reduces blood flow to the extremities) clinical trial revision received FDA approval. Here is an important quote from KOOL’s press release after the approval, "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. With the SergWerks platform, KOOL wants to revolutionize how autologous cellular therapies are conducted; the process by which the implantation, transplantation, infusion, or transfer of human cells or tissue back into the individual from whom the cells or tissue were recovered."

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The revisions involved with the trial 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. The Rutherford Category is the clinical staging system for describing peripheral arterial disease, or the lack of blood to the extremities. KOOL’s participation pool has been expanded from the most severe degree of CLI to a lesser degree of CLI. 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.

Logistically, the sample size was increased from 224 to 362 patients, which in turn allowed the superiority margin to be reduced from 20% to 13%. 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. Dr. Xiaochun “Chris” Xu, Cesca’s Interim CEO said, “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…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.”

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