FDA's BPAC renders positive opinion on Phase III clinical trial of INTERCEPT Blood System

Nov 17 2009

Cerus Corporation (NASDAQ:CERS) announced today that the FDA's Blood Products Advisory Committee (BPAC) rendered a positive opinion on the proposed hemostatic efficacy and safety endpoints for a potential U.S. Phase III clinical trial of the INTERCEPT Blood System for platelets. The Committee disagreed with the safety margins for the trial proposed by Cerus, and recommended that the trial design include more stringent safety margins for comparing INTERCEPT-treated platelets and conventional platelets. In addition, the Committee rendered a positive opinion on a proposed pathway forward in which successful completion of the proposed Phase III trial would be followed by a post-marketing randomized control study and concurrent staged roll-out of the product.

"The Committee's guidance supports further development of INTERCEPT in the United States," said Claes Glassell, president and chief executive officer of Cerus. "We now need to conduct further discussions with the FDA to agree upon a final protocol. The more stringent safety margins recommended by the Committee may require a clinical trial with a larger number of patients than had been proposed. We expect that it will take at least 12 months to complete the clinical trial preparations and partnering arrangements necessary for commencement of the potential trial.”

The proposed Phase III clinical trial is designed as a randomized, double-blinded, non-inferiority trial to assess the hemostatic efficacy and safety of routine use of INTERCEPT-treated platelets compared to platelets prepared with conventional processes. The proposed primary efficacy endpoint is number of days of Grade 2 bleeding (World Health Organization scale), and the proposed primary safety endpoint is the incidence of acute lung injury (ALI).

Previously, Cerus completed a U.S. Phase III trial for INTERCEPT-treated platelets, the SPRINT trial, which enrolled 645 patients. The SPRINT trial met its primary endpoint of the proportion of patients with Grade 2 bleeding, indicating non-inferiority of INTERCEPT platelets compared to conventional platelets. While acknowledging this outcome, the FDA expressed concerns regarding observed differences between the test and control arms of the study and had indicated that it needed more data to support a pre-market approval application. The new Phase III trial will be designed to resolve the FDA's questions regarding hemostatic efficacy and safety.

The FDA seeks the advice of an advisory committee such as the BPAC when evaluating a potential new product. A final decision is made by the FDA and while the FDA is not required to follow the advice of its advisory committee, it often does.