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).