CONCORD, Calif.--(EON: Enhanced Online News)--Cerus Corporation (NASDAQ:CERS) today provided an update on the timeline for the INTERCEPT Blood System for Red Blood Cells (RBCs) in Europe. The target timing for CE Mark submission has been extended, and the company’s European red cell study in chronic anemia patients has completed enrollment.
“Our ability to provide safety and efficacy data generated by the chronic anemia study at the time of our anticipated product launch will be an important supplement to the clinical data already included for submission.”
Due to the need for additional time to complete quality control tests on the Chemistry, Manufacturing, and Control (CMC) registration lots required for regulatory submission, the company now plans to provide an update on new submission timing on its 4Q16 earnings call in early March. The CE Mark process for the INTERCEPT Blood System for Red Blood Cells includes review and approval by both TŰV SŰD as the Notified Body and Irish Health Products Regulatory Authority (HPRA) as the Competent Authority. Accordingly, a new submission filing date will need to be scheduled with TŰV SŰD Product Service GmbH.
Cerus’ SPARC trial (A Randomized Controlled Study to Evaluate Efficacy and Safety of INTERCEPT Treated Red Blood Cells in Subjects with Thalassemia Major Requiring Chronic RBC Transfusion) has now reached its enrollment target of at least 70 evaluable patients, and the company expects that study data will be available in time to support its anticipated European product launch. The SPARC study is being conducted in transfusion-dependent thalassemia major patients, an inherited blood disorder of abnormal hemoglobin resulting in excessive destruction of red blood cells.
“Chronic anemia patients such as those with thalassemia major and sickle cell disease require more blood transfusions than almost any other patient group, putting them at elevated lifetime risk for exposure to existing and emerging pathogens,” said Richard Benjamin, Cerus' chief medical officer. “Our ability to provide safety and efficacy data generated by the chronic anemia study at the time of our anticipated product launch will be an important supplement to the clinical data already included for submission.”
The SPARC study is a randomized, crossover trial in which patients receive two periods of transfusion support, consisting of one period each of conventional and INTERCEPT-treated RBCs over approximately 6 - 9 months in total. The primary efficacy endpoint is hemoglobin usage and the primary safety endpoint is immunogenicity. Hemoglobin usage was selected as the primary efficacy endpoint because it is correlated with the ability of transfused red blood cells to oxygenate tissues, persist in circulation, and suppress endogenous erythropoiesis. This endpoint is clinically relevant because hemoglobin consumption is correlated with iron burden, an important complication for patients despite availability of chelation therapies.
Red blood cells are disc-shaped cells containing hemoglobin which deliver oxygen from the lungs to all body tissues. Red blood cells are the most frequently transfused blood component, with over 80 million units transfused globally each year. Patients typically receive red blood cell transfusions to offset blood loss after trauma and during surgery, or to increase hemoglobin levels in patients with hereditary anemia.
ABOUT THE INTERCEPT BLOOD SYSTEM FOR RED BLOOD CELLS
Cerus reported positive results of the INTERCEPT Blood System for Red Blood Cells from a European Phase III acute clinical trial in 2015. If approved, the INTERCEPT Blood System for RBCs will be the only system commercially available in Europe to enhance the safety of red blood cell transfusions through pathogen inactivation. In addition to plans to submit for CE Mark registration in Europe, Cerus is also currently in dialogue with the U.S. Food & Drug Administration regarding a protocol design for a Phase III pivotal red blood cell study to be conducted in the continental United States.
Cerus Corporation is a biomedical products company focused in the field of blood transfusion safety. The INTERCEPT Blood System is designed to reduce the risk of transfusion-transmitted infections 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 of the INTERCEPT treatment is designed to inactivate established transfusion threats, such as Hepatitis B and C, HIV, West Nile Virus and bacteria, as well as emerging pathogens such as chikungunya, malaria and dengue. Cerus currently markets and sells the INTERCEPT Blood System for both platelets and plasma in the United States, 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 www.cerus.com for information about Cerus.
INTERCEPT and INTERCEPT Blood System are trademarks of Cerus Corporation.
Except for the historical statements contained herein, this press release contains forward-looking statements concerning Cerus’ products, prospects and expected results, including statements concerning Cerus’ plans to pursue CE Mark approval in Europe for the red blood cell system and the timing thereof; the potential for CE Mark approval for and commercial launch of Cerus’ red blood cell product in Europe; and Cerus’ plans to present data regarding the study results from its study in transfusion-dependent thalassemia major patients. Actual results could differ materially from these forward-looking statements as a result of certain factors, including, without limitation: risks associated with the uncertain and time-consuming regulatory process, including completion of all of the CMC activities necessary to support a regulatory submission; that Cerus may be unable to file for CE Mark approval of the red blood cell system in Europe in the anticipated timeframe or at all, and even if filed, that Cerus may be unable to obtain CE Mark approval, or any other regulatory approvals, of the red blood cell system in a timely manner or at all; Cerus’ belief that the results from its clinical trial will support its CE Mark submission, Cerus’ development of the INTERCEPT Blood System for red blood cells and its therapeutic potential, the risks associated with the uncertainty of clinical trial results, as well as other risks detailed in Cerus’ filings with the Securities and Exchange Commission, including in Cerus‘ Quarterly Report on Form 10-Q for the quarter ended September 30, 2016, filed with the SEC on November 4, 2016. Cerus disclaims any obligation or undertaking to update or revise any forward-looking statements contained in this press release.