About Cerus

Cerus Corporation operates as a biomedical products company. The company focuses on developing and commercializing the INTERCEPT Blood System to enhance blood safety. The INTERCEPT Blood System, which is based on the company’s proprietary technology for controlling biological replication, is designed to reduce blood-borne pathogens in donated blood components intended for transfusion. The company’s INTERCEPT Blood System is intended for use with blood components and certain of their derivatives: platelets, plasma, red blood cells and to produce INTERCEPT Fibrinogen Complex, or IFC, and pathogen reduced plasma, cryoprecipitate reduced. The INTERCEPT Blood System for platelets, or platelet system, and the INTERCEPT Blood System for plasma, or plasma system, have received a broad range of regulatory approvals and certification, including but not limited to FDA approval in the U.S., CE Certificates of Conformity delivered in accordance with the Medical Devices Directive 93/42/EEC, or MDD, permitting the company to affix the CE Mark to its products and place them on the market in the European Union in accordance with the transitional provisions of the Medical Devices Regulation 2017/745, or MDR, and other jurisdictions that recognize the CE Mark, and are being marketed and sold in a number of countries around the world, including the U.S., certain countries in Europe, the Commonwealth of Independent States, or CIS, the Middle East, and Latin America and selected countries in other regions of the world. Additionally, the company has received FDA approval for the INTERCEPT Blood System for Cryoprecipitation. The INTERCEPT Blood System for Cryoprecipitation uses the company’s plasma system to produce IFC for the treatment and control of bleeding, including massive hemorrhage, associated with fibrinogen deficiency. In addition, the INTERCEPT Blood System for Cryoprecipitation is used to produce pathogen reduced plasma, cryoprecipitate reduced. The company sells the platelet and plasma systems using its direct sales force and through distributors and sell IFC or disposable kits to manufacture IFC in the U.S. using its direct sales force. The INTERCEPT Blood System for red blood cells, or the red blood cell system, is in development. In the U.S., the company is conducting two Phase 3 clinical trials - the RedeS study, to assess the safety and efficacy of INTERCEPT-treated red blood cells when compared to conventional, un-treated, red blood cells and the ReCePI study to evaluate the efficacy and safety of INTERCEPT-treated red blood cells in patients requiring transfusion for acute blood loss during surgery. In the European Union, or EU, the company completed the resubmission of its application for a CE Certificate of Conformity under the new Medical Device Regulation 2017/745, or MDR, in June 2021. Product Development The INTERCEPT Blood System is designed to broadly target and inactivate blood-borne pathogens, such as viruses (for example, HIV, West Nile, SARS, hepatitis B and C), bacteria and parasites, as well as potentially harmful white blood cells, while preserving the therapeutic properties of platelet, plasma, red blood cell and IFC transfusion products. The INTERCEPT Blood System has been shown to inactivate a broad array of pathogens and has the potential to reduce the risk of transfusion related transmission of pathogens for which testing is not completely effective, is not available or is not performed. The INTERCEPT Blood System also has the potential to inactivate most new pathogens before they are identified and before tests are developed and adopted commercially to detect their presence in donated blood. Products, Product Candidates and Development Activities INTERCEPT Blood System for Platelets, Plasma and Cryoprecipitation The platelet system and plasma system are designed to inactivate blood-borne pathogens in platelets and plasma donated for transfusion. Both systems received a CE Certificate of Conformity permitting the company to affix the CE Mark in the European Economic Area, or EEA, and FDA approval in the U.S. and are marketed and sold in a number of countries around the world, including the U.S., countries in Europe, the CIS, the Middle East and selected countries in other regions of the world. Separate approvals for use of INTERCEPT-treated platelet and plasma products have been obtained in France and Switzerland. In Germany and Austria, where approvals must be obtained by individual blood centers for use of INTERCEPT-treated platelets and plasma, several centers have obtained such approvals for use of INTERCEPT-treated platelets and one center has obtained such approval for use of INTERCEPT-treated plasma. The company received CE Certificates of Conformity in accordance with the MDD for its INTERCEPT platelet and plasma systems, which will expire in May 2024. The company has submitted its application and technical documentation for CE Certificates of Conformity under the MDR for both its INTERCEPT platelet and plasma systems which are under review by its Notified Body. The U.S. Food and Drug Administration (FDA) has approved the platelet system for ex vivo preparation of pathogen-reduced apheresis platelet components collected and stored in 100% plasma or InterSol in order to reduce the risk of transfusion-transmitted infection, or TTI, including sepsis, and as an alternative to gamma irradiation for prevention of transfusion-associated graft versus host disease. In 2021, the company completed one of the two post-approval studies that FDA required as part of its approval of the platelet system - a haemovigilance study evaluating the incidence of acute lung injury following transfusion of INTERCEPT-treated platelets. The second required post-approval study - a recovery study of platelets treated with the platelet system - is in progress. The FDA has also approved the plasma system for ex vivo preparation of plasma in order to reduce the risk of TTI when treating patients requiring therapeutic plasma transfusion. The company has also received FDA approval for the INTERCEPT Blood System for Cryoprecipitation, which uses its plasma system to produce IFC for the treatment and control of bleeding, including massive hemorrhage, associated with fibrinogen deficiency and to produce pathogen reduced plasma, cryoprecipitate reduced. The company has contracted with several blood centers to produce IFC for it which it plans to sell directly to hospitals. All of the blood centers that the company has contracted with to produce IFC for it has submitted for their interstate licenses, or BLAs. Until Biologics License Applications (BLAs) are more broadly obtained, the company expects that its direct sales of IFC will be limited. Further, the hospital customers of blood centers may need to complete changes to their administrative processes of generating internal tracking codes to integrate INTERCEPT-treated products into their inventories prior to receiving INTERCEPT-treated components. In addition, the company estimates that the majority of platelets used in the U.S. are collected by apheresis, which is part of its FDA-approved label for the platelet system, though a significant minority are prepared from pooled random donor platelets derived from whole blood collections. INTERCEPT Blood System for Red Blood Cells The company completed a series of in vitro and in vivo tests with the red blood cell system, including successfully completing recovery and survival studies measuring red cell recovery twenty-four hours after transfusion. Previously, the company terminated Phase 3 clinical trials for acute and chronic anemia using a prior generation of the red blood cell system due to the detection of antibody reactivity to INTERCEPT-treated red blood cells, or RBCs, in two patients in the trial for chronic anemia. After unblinding the data from the original Phase 3 clinical trials, the company found that it had met the primary endpoint in the clinical trial for acute anemia. The company evaluated the antibodies detected and developed process changes to diminish the likelihood of antibody reactivity in RBCs treated with its modified process. The company has since successfully completed Phase 3 clinical trials of the red blood cell system for subjects with acute and chronic anemia patients to support an application for a CE Certificate of Conformity. The company filed its application for a CE Certificate of Conformity for the red blood cell system in December 2018 under the Medical Device Directive, or MDD, and in June 2021, the company completed the application for a CE Certificate of Conformity under the new MDR. The company previously completed a European Phase 3 clinical trial of RBCs treated with the INTERCEPT Blood System for acute anemia in cardiovascular surgery subjects announced that the trial met its primary endpoint, with preliminary analysis demonstrating that the mean hemoglobin content (53.1g) of INTERCEPT-treated RBCs, on day 35 of storage met the protocol-defined criteria for equivalence based on the inferiority margin of 5g compared to conventional RBCs (55.8g). Additionally, the company previously announced that the European Phase 3 clinical trial of chronic anemia evaluating INTERCEPT-treated RBCs in thalassemia subjects met its primary efficacy and safety endpoints. Regardless of the potential sufficiency of clinical data required to receive a CE Certificate of Conformity, the company understands that it will need to generate additional safety data from commercial use in order to achieve broad market acceptance, if ever certified. In the U.S., the company successfully completed a Phase 2 recovery and lifespan study. Subsequently, the company initiated a double-blind Phase 3 clinical study, known as the RedeS study, to assess the safety and efficacy of INTERCEPT-treated RBCs when compared to conventional RBCs in regions impacted by the Zika virus epidemic. The RedeS study was expanded to other areas at risk for transfusion-transmitted infections. The company also received investigational device exemption, or IDE, approval from the FDA to initiate a Phase 3 clinical trial, known as the ReCePI study, that is designed to evaluate the efficacy and safety of INTERCEPT-treated RBCs in patients requiring transfusion for acute blood loss during surgery. In addition to successfully conducting and completing the RedeS and ReCePI studies, the company also understands that one or more additional in vitro studies will be required to be successfully completed and submitted to the FDA before the FDA will consider its red blood cell product for approval. INTERCEPT Blood System Technology Both the company’s platelet system and plasma system employ the same technology. Platelet or plasma components collected from blood donors are transferred into plastic INTERCEPT disposable kits and are mixed with its proprietary compound, amotosalen, a small molecule compound that has an affinity for nucleic acid. The disposable kits are then placed in an illumination device, or illuminator, where the mixture is exposed to ultra-violet A, or UVA, light. If pathogens, such as viruses, bacteria or parasites, as well as leukocytes, or white cells, are present in the platelet or plasma components, the energy from the UVA light causes the amotosalen to bond with the nucleic acid. Since platelets and plasma do not rely on nucleic acid for therapeutic efficacy, the INTERCEPT Blood System is designed to preserve the therapeutic function of the platelet and plasma components and IFC when used in human transfusions. The company has performed extensive toxicology testing on the residual amotosalen and its by-products and good safety margins have been demonstrated. The INTERCEPT Blood System, with its combination of the amotosalen and UVA light, is designed to inactivate leukocytes in the same manner it inactivates pathogens. Manufacturing and Supply The company has used, and intends to continue to use, third parties to manufacture and supply the illuminators, components, disposable kits and inactivation compounds that make up the INTERCEPT Blood System for use in clinical trials and for commercialization. With the exception of certain components, the company relies solely on Fresenius Kabi AG, or Fresenius, for the manufacture of disposable kits for the platelet and plasma systems. In May 2022, the company entered into the Second Amended and Restated Supply and Manufacturing Agreement, or the 2022 Agreement, with Fresenius Kabi AG, Fenwal France SAS, Fenwal International, Inc. and Fresenius Kabi Deutschland GmbH, or collectively, Fresenius, for the manufacture and production of disposable sets for the INTERCEPT Blood System until December 31, 2031. Under the terms of the 2022 Agreement, Fresenius is obligated to manufacture, and the company is obligated to purchase, finished disposable kits for the platelet and plasma systems. Components of the compound adsorption devices used in the company’s platelet and plasma disposable kits are manufactured by Porex Corporation, or Porex. In April 2017, the company entered into an amended and restated manufacturing and supply agreement with Porex for the continued supply of the compound adsorption devices. Porex is the company’s sole supplier for certain components of and manufacturing of the compound adsorption devices. Under the amended and restated Porex agreement, the company is no longer subject to a minimum annual purchase requirement; however, Porex has the right to terminate the agreement, upon twelve months’ prior written notice, if annual production falls below a mutually agreed threshold. The amended and restated Porex agreement was renewed in December 2021 and will continue until December 31, 2024. The company also has an amended and restated supply agreement with Purolite LLC (Purolite) for the supply of raw materials used to make the compound adsorption devices. The amended supply agreement expires in April 2024, and will automatically renew for an additional year unless either party has provided notice not to renew at least two years prior to the expiration. The company operates with an amended manufacturing and supply agreement with Piramal for the synthesis of amotosalen, the inactivation compound used in its platelet and plasma systems. Under this amended agreement, the company is subject to minimum annual purchase requirements. The term of the amended manufacturing and supply agreement with Piramal will expire on December 31, 2023 and will continue to automatically renew for successive two-year periods, unless terminated by either party upon providing at least one year prior written notice, in the company’s case, or at least two years prior written notice, in the case of Piramal. Marketing, Sales and Distribution The market for the INTERCEPT Blood System, including the U.S. market, is dominated by a relatively small number of blood collection organizations. In October 2021, all the U.S. blood centers had to be compliant with the FDA guidance document, ‘Bacterial Risk Control Strategies for Blood Collection Establishments and Transfusion Services to Enhance the Safety and Availability of Platelets for Transfusion’, or the Final Guidance Document. While the company’s obtaining a CE Certificate of Conformity permitting it to affix the CE Mark and sell the platelet and plasma systems to blood centers in Germany, blood centers in Germany must still obtain both local manufacturing approval and national marketing authorization from the Paul Ehrlich Institute, or PEI, a German governmental regulatory body overseeing the marketing authorization of certain medical products, before being allowed to sell platelet and plasma components treated with the INTERCEPT Blood System to transfusing hospitals and physicians. As of December 31, 2022, several blood centers in Germany have received such requisite approvals and authorizations for the platelet system. INTERCEPT-treated platelets received national reimbursement in Germany in 2018 at a premium to untreated platelets. In October 2021, the company entered into a new agreement with Etablissement Francais du Sang (EFS) to supply platelet disposable kits. The agreement for supply of platelet disposable kits provides for a base term of two years, with two options for EFS to extend for one year each. In January 2020, the company entered into a new agreement with EFS to supply plasma disposable kits and maintenance services for illuminators for a base term of two years, with two options for EFS to extend for one year each. EFS exercised the second option in September 2022. The company has entered into manufacturing agreements with certain blood centers to produce IFC for it. In addition, the company has entered into agreements with certain blood centers and blood center affiliate organizations to sell the INTERCEPT Blood System for Cryoprecipitation. In order to successfully commercialize IFC, the company will need to generate commercial use data in order to influence the market and sell directly to hospital users and blood center producers of cryoprecipitate. The company is adding to and reorganizing its existing commercial teams to commercialize IFC. In the U.S., the company obtained Healthcare Common Procedure Coding System (HCPCS) reimbursement codes for hospital outpatient billing and payment of INTERCEPT-treated platelets and plasma in 2015, and for IFC and the derivative, pathogen-reduced plasma, cryoprecipitate reduced in 2021. The company maintains a wholly owned subsidiary, Cerus Europe B.V., headquartered in the Netherlands, which focuses its efforts on marketing and selling the INTERCEPT Blood System in a number of countries in Europe, the Commonwealth of Independent States (CIS), the Middle East and selected countries in other regions around the world. The company has a small scientific affairs group in the U.S. and the Netherlands that supports its commercialization efforts, as well as hospital affairs professionals, to help educate hospitals and physicians on its products, clinical trial history and publications. The company has a small group of individuals to which it may add to in the future to market and sell IFC in the U.S. The company has a small number of employees focused on servicing the markets in Asia-Pacific and Latin American regions and rely primarily on distributors to market and sell its products in those regions. In February 2021, the company entered into an Equity Joint Venture Contract with Shandong Zhongbaokang Medical Implements Co., Ltd., or ZBK, to establish Cerus Zhongbaokang (Shandong) Biomedical Co., LTD., which it refers to as the JV, for the purpose of developing, obtaining regulatory approval for, and eventual manufacturing and commercialization of the INTERCEPT blood transfusion for platelets and red blood cells in the People’s Republic of China. The company own 51% of equity in the JV. The JV will need to obtain regulatory approval for the INTERCEPT Blood System for platelets and red blood cells before it can begin commercializing in China. The company’s business is dependent on the marketing and commercialization of the INTERCEPT Blood System to customers, such as blood banks, hospitals, distributors and other health care providers that have a need for a pathogen reduction system to treat blood products for transfusion. Government Contracts The company operates directly under three contracts with the U.S. Federal Agencies, one with Biomedical Advanced Research and Development Authority (BARDA), one with the FDA, and another with the Department of Defense (DoD). Inventory Requirements and Product Return Rights The company’s platelet and plasma disposable kits have received regulatory approval and certification for shelf lives ranging from 6 to 24 months. The company’s INTERCEPT Blood System for Cryoprecipitation has received regulatory approval and certification for a shelf life of 12 months. Although the company has regulatory approval and certification for its products in most regions for up to a 24-month shelf life, the FDA has limited its platelet product to a six-month shelf life and has asked for additional stability and aging data on newly manufactured lots which will require the company to manage its U.S. supply chain closely until it generates the requested data and receive approval for longer shelf lives from the FDA. Illuminators and replacement parts do not have regulated expiration dates. The company sells the INTERCEPT Blood System directly to blood banks, hospitals, universities, and government agencies, as well as to distributors in certain regions. Generally, the company’s contracts with its customers do not provide for open return rights, except within a reasonable time after receipt of goods in the case of defective or non-conforming product. The company has also entered into agreements with certain blood centers and blood center affiliate organizations to sell the INTERCEPT Blood System for Cryoprecipitation for their production of IFC and sale to their hospital customers. Government Regulation The company and its products are comprehensively regulated in the U.S. by the FDA and by comparable governmental authorities in other jurisdictions. In March 2020, the company received an extension of the CE Certificate of Conformity to 2024, under the MDD. While the company’s extension of registration is based on the MDD for the platelet and plasma systems. A separate CE Certificate of Conformity in accordance with the MDR must be received for the red blood cell system to be sold in the European Union and in other countries recognizing the CE Mark. The company filed its application for a CE Certificate of Conformity of the red blood cell system in December 2018 under the MDD, and in June 2021, the company completed the process to resubmit its application under the new MDR. The company has also recently received FDA approval for the INTERCEPT Blood System for Cryoprecipitation, which uses its plasma system to produce IFC for the treatment and control of bleeding, including massive hemorrhage, associated with fibrinogen deficiency and to produce pathogen reduced plasma, cryoprecipitate reduced. With FDA approval of its platelet and plasma systems and the INTERCEPT Blood System for Cryoprecipitation, the company is required to continue to comply with applicable FDA and other regulatory requirements related to, among other things, labeling, packaging, storage, advertising, promotion, record-keeping and reporting of safety and other information. In addition, the company’s manufacturers and their facilities are required to comply with extensive FDA and foreign regulatory agency requirements, including, in the U.S., ensuring that quality control and manufacturing procedures conform to FDA-mandated current Good Manufacturing Practice, or cGMP, and Quality System Regulation, or QSR, requirements. As such, the company and its contract manufacturers are subject to continual review and periodic inspections. The company is also required to report certain adverse events and production problems, if any, to the FDA, competent authorities of the EU Member States and Notified Bodies, and foreign regulatory authorities, when applicable, and FDA, competent authorities of the EU Member States, or other foreign regulatory authorities may require it to recall products as a result of adverse events or production problems. For example, the company’s promotional materials and training methods must comply with FDA and other applicable laws and regulations, including the prohibition of the promotion of unapproved, or off-label, uses. The company supplies the INTERCEPT Blood System for Cryoprecipitation to select blood centers that manufacture IFC for it. The company plans to sell the finished IFC made by its manufacturing blood center partners directly to hospitals. Similar to the company’s platelet and plasma products, any blood center manufacturing IFC will need to complete their process validations and obtain site-specific licenses from the FDA Center for Biologics Evaluation and Research (CBER) before it or they can sell finished IFC to hospital customers outside of the states producing IFC. Competition In the U.S., INTERCEPT-treated plasma faces competition from Octapharma AG, which is commercializing treated fresh frozen plasma for certain indications in the U.S., as well as from diagnostic and testing companies approved for the detection of pathogens in donated blood products, including bacterial and viral pathogens. History Cerus Corporation was founded in 1991. The company was incorporated in California in 1991 and reincorporated in Delaware in 1996.