Cerus Corporation (NASDAQ: CERS) announced today that it has submitted an IDE supplement to the U.S. Food and Drug Administration (FDA) for the company’s proposed Phase III trial of the INTERCEPT Blood System for red cells in patients receiving chronic red cell transfusion support for sickle cell disease (SCD) or thalassemia. “We look forward to working with FDA to finalize the Phase III study protocol to support U.S. licensure of INTERCEPT red cells for support of patients with chronic anemia,” commented Dr. Laurence Corash, Cerus’ chief medical officer. “Chronically transfused patients are at increased risk both from newly emerging pathogens, such as dengue virus, and from known infectious agents, such as Babesia, for which there are no currently licensed tests. Pathogen inactivation of red cells offers a potential new treatment option for this vulnerable patient population, who may face lifetime exposure to blood transfusion.” The proposed trial design includes approximately 400 patients, who would receive transfusions of INTERCEPT or control red cells for treatment of sickle cell disease or thalassemia. Proposed endpoints of the study are designed to assess both the efficacy and safety of INTERCEPT red cells. FDA has provided guidance that an approximately 60 red cell unit paired in vitro study and an additional Phase I recovery and survival study using the final clinical disposable sets and reagents in healthy subjects will be required prior to initiation of the proposed Phase III trial. These prerequisite studies may need to be finished and the data submitted prior to the FDA’s review of the Phase III protocol supplement filed in the IDE. Previous discussions with the FDA indicated that Cerus’ Phase III protocol could be reviewed under a Special Protocol Assessment (SPA), but upon receipt of the application the FDA amended its position and requested use of the IDE route. The proposed Phase III study size and endpoints in the IDE supplement are identical to those previously planned for SPA review.
An approved Investigational Device Exemption (IDE) is required to allow use of an investigational device in a clinical study to collect safety and efficacy data to support a Premarket Approval (PMA) application. More information about the IDE process is available from the FDA website: http://www.fda.gov/MedicalDevices/DeviceRegulationandGuidance/HowtoMarketYourDevice/InvestigationalDeviceExemptionIDE/default.htmABOUT CERUS Cerus Corporation is a biomedical products company focused on commercializing the INTERCEPT Blood System to enhance blood safety. The INTERCEPT system is designed to reduce the risk of transfusion-transmitted diseases by inactivating a broad range of pathogens such as viruses, bacteria and parasites that may be present in donated blood. The nucleic acid targeting mechanism of action enables INTERCEPT treatment to inactivate established transfusion threats, such as hepatitis B and C, HIV, West Nile virus and bacteria, and is designed to inactivate emerging pathogens such as influenza, malaria and dengue. Cerus currently markets and sells the INTERCEPT Blood System for both platelets and plasma in Europe, the Commonwealth of Independent States, the Middle East and selected countries in other regions around the world. The INTERCEPT red blood cell system is in clinical development. See http://www.cerus.com for more information. INTERCEPT and the INTERCEPT Blood System are trademarks of Cerus Corporation. This press release contains forward-looking statements. Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements, including, without limitation, statements relating to the proposed Phase III INTERCEPT red cell system trial, the therapeutic and commercial potential of the INTERCEPT red cell system, and future regulatory matters, including those related to the IDE process with the FDA, the number and scope of additional studies required prior to initiating the proposed Phase III INTERCEPT red cell system trial, and potential regulatory approval. These forward-looking statements are based upon Cerus’ current expectations. Actual results could differ materially from these forward-looking statements as a result of certain factors, including, without limitation, risks associated with the uncertain clinical development and regulatory approval process, including the risks that Cerus’ IDE application may not be reviewed or approved by the FDA in a timely manner or at all, that the FDA may require that we conduct additional unanticipated clinical studies to initiate Phase III development of the INTERCEPT red cell system and/or to support potential approval of INTERCEPT red cells, and that we may never obtain FDA approval of INTERCEPT red cells, risks associated with Cerus’ need for additional capital, including the risk that Cerus may be unable to raise the funds necessary to conduct the proposed Phase III INTERCEPT red cell system trial or any other trials of the INTERCEPT red cell system required by the FDA, and other risks detailed in the Cerus' filings with, the Securities and Exchange Commission (SEC), including in Cerus' annual report on Form 10-K for the year ended December 31, 2011, filed with the SEC on March 5, 2011. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date of this press release. Cerus does not undertake any obligation to update any forward-looking statements as a result of new information, future events, changed assumptions or otherwise.