Cerus Corporation Announces Positive Topline Results for the Phase 3 Clinical Trial of the INTERCEPT Blood System for Red Blood Cells in Cardiovascular Surgery Patients
Cerus (Nasdaq: CERS) announces positive results for ReCePI Phase 3 trial of INTERCEPT Red Blood Cells showing non-inferiority in acute kidney injury incidence compared to conventional RBCs. Safety data also favorable.
The positive results from Cerus Corporation's ReCePI Phase 3 clinical trial represent a pivotal moment in the field of transfusion medicine. The non-inferiority of INTERCEPT Red Blood Cells (RBCs) in preventing acute kidney injury (AKI) post-transfusion is an important finding. AKI is a important indicator of RBC efficacy as it directly relates to the tissue oxygen delivery capability of the transfused RBCs. The results indicate that the INTERCEPT RBCs are just as effective as conventional RBCs in this regard, which could have significant implications for patient outcomes in complex cardiac surgeries where transfusions are common.
When considering the safety profile, the trial reported a small difference in treatment-emergent adverse events (TEAEs) between INTERCEPT RBCs and conventional RBCs, which was not statistically significant. The absence of clinical significance related to treatment-emergent RBC antibodies in the INTERCEPT group is another reassuring safety signal. These findings, subject to further validation in ongoing studies like the RedeS Phase 3 clinical trial, could support a favorable safety assessment in the integrated analysis planned for the modular premarket approval (PMA) submission.
For stakeholders, particularly investors in Cerus Corporation, these results could signal the potential for market expansion of INTERCEPT RBCs if approved by regulatory bodies. The data's inclusion in a PMA submission could be a catalyst for increased investor confidence, reflecting the company's commitment to advancing the safety and efficacy of blood transfusions. However, it is essential to remain cautious as further analysis and regulatory review are necessary before any potential market impact can be fully realized.
In the healthcare industry, innovations that improve patient outcomes and demonstrate safety and efficacy can lead to shifts in standard treatment protocols. The INTERCEPT blood system's potential to reduce the risk of transfusion-transmitted infections has already been recognized in certain markets and this new data on the non-inferiority of INTERCEPT RBCs in preventing AKI could further solidify its position in the market.
One must consider the broader context of the transfusion medicine market, which is highly regulated and sensitive to innovation that can improve patient safety. The trial's results could encourage adoption in hospitals and transfusion centers, pending regulatory approval. This could eventually lead to increased demand for INTERCEPT RBCs, impacting Cerus Corporation's revenue and market share. However, the adoption rate will depend on factors such as cost-effectiveness, reimbursement policies and the educational efforts required to shift practitioners towards new technologies.
Long-term, if INTERCEPT RBCs are integrated into clinical practice, there could be downstream effects on the supply chain and blood bank operations, as well as potential impacts on competitors who may need to innovate to keep pace. It's important for investors to monitor how Cerus navigates the regulatory environment and positions INTERCEPT RBCs in an increasingly competitive landscape.
From a market perspective, the successful trial outcomes for INTERCEPT RBCs could indicate a strategic advantage for Cerus Corporation. The ability to prevent AKI with a non-inferior performance to conventional RBCs addresses a significant concern in cardiac surgery patients. This could lead to increased market penetration in the cardiac surgery segment, which is substantial given the high volume of procedures conducted annually.
Furthermore, the potential for INTERCEPT RBCs to be used more broadly in other surgical or medical scenarios where blood transfusions are required could open up new market segments for Cerus. It's important to note that market expansion is contingent upon regulatory approvals, which are based on a comprehensive assessment of the data, including safety and efficacy.
Investor sentiment may be buoyed by this positive news, but it's important to analyze the competitive landscape, the cost of INTERCEPT RBCs compared to conventional RBCs and the potential for insurance coverage or reimbursement. These factors will ultimately determine the commercial success of INTERCEPT RBCs and Cerus Corporation's financial performance.
03/19/2024 - 08:00 AM
Trial met the primary efficacy endpoint demonstrating non-inferiority of the incidence of acute kidney injury for INTERCEPT Red Blood Cells compared to conventional red blood cells
CONCORD, Calif. --(BUSINESS WIRE)--
Cerus Corporation (Nasdaq: CERS) today announced positive topline results for ReCePI, a pivotal Phase 3 clinical trial of pathogen reduced INTERCEPT Red Blood Cells (INTERCEPT RBCs) transfused to complex cardiac surgery patients. The trial met its primary efficacy endpoint, demonstrating non-inferiority for INTERCEPT RBCs compared to conventional RBCs as measured by the incidence of acute kidney injury (AKI) following transfusion of study RBCs. AKI is a sensitive transfusion efficacy indicator of RBC tissue oxygen delivery. In transfused subjects, by modified intent to treat (mITT pre-specified primary endpoint population) the incidence of AKI was 29.3% (46/157) for INTERCEPT RBC recipients compared to 28.0% (45/161) for conventional RBC recipients, demonstrating non-inferiority of INTERCEPT RBCs compared to conventional RBCs with an upper limit of the 95% confidence interval of 10.4% compared with a non-inferiority margin of 14.0% (p = 0.001).
The safety endpoint of the proportion of patients with any related treatment-emergent adverse events (TEAEs) within 28 days of last transfusion was not significantly different for INTERCEPT RBCs (2.5% ) compared to conventional RBCs (0.6% ) (p = 0.130). There was no clinical significance related to treatment-emergent RBC antibodies observed in five patients receiving INTERCEPT RBCs. The trial’s independent Data Safety and Monitoring Board (DSMB) evaluated these patients and was supportive of continued transfusion, having found no adverse health effects from trial transfusions. This safety endpoint is also being explored in the Company’s ongoing RedeS Phase 3 clinical trial of INTERCEPT RBCs. Cerus plans to include data from both the ReCePI and RedeS clinical trials in an integrated safety analysis as part of its planned modular premarket approval (PMA) submission.
“We are extremely pleased by the positive topline results of the ReCePI clinical trial,” stated Richard Benjamin, M.D., Ph.D., Cerus’ chief medical officer and principal investigator for the ReCePI clinical trial. “The ReCePI clinical trial is a pioneering trial in the field of transfusion medicine, providing critical information about pathogen reduced INTERCEPT RBCs compared to conventional RBCs. We continue to look forward to completing the RedeS clinical trial, Cerus’ ongoing pivotal Phase 3 clinical trial for INTERCEPT RBCs enrolling a broader patient population requiring RBC transfusion for acute and chronic anemia.”
“Today’s news from the ReCePI clinical trial is an important milestone for the INTERCEPT RBC program,” stated William “Obi” Greenman, Cerus’ president and chief executive officer. “We are now one meaningful step closer to potentially making pathogen reduction available for RBCs, the most commonly transfused blood component in the U.S. , with approximately 11 million transfused units annually. If approved, pathogen reduced RBCs would complement our existing commercial portfolio of INTERCEPT Systems for Platelets, Plasma, and Fibrinogen Complex, providing clinicians and patients access to the benefits of pathogen inactivation across all transfused components. We are grateful to all the patients, blood centers, and physicians who participated in the ReCePI clinical trial.”
Cerus anticipates initiating a modular PMA application to the U.S. Food and Drug Administration (FDA) in the second half of 2025, with the final PMA module submission planned for the second half of 2026, upon the anticipated completion of the RedeS clinical trial. Data from preclinical studies, manufacturing information, previously reported Phase 1 and Phase 2 clinical trials in the U.S. , and the successfully completed Phase 3 clinical trials in the EU (STARS and SPARC) will be included in the modular PMA submission.
Cerus expects the data from the ReCePI clinical trial to be presented at upcoming medical conferences as well as to be submitted for peer reviewed publication.
Webcast
The Company will host a webcast at 9:00 A.M. EDT this morning, during which management will discuss the results of the ReCePI clinical trial. To listen to the live webcast, please visit the Investor Relations page of the Cerus website at http://www.cerus.com/ir.
A replay will be available on Cerus’ website approximately three hours after the call through April 2, 2024.
ABOUT ReCePI
The ReCePI Phase 3 clinical trial was a prospective, multicenter, randomized, double-blinded, controlled, non-inferiority design trial (ClinicalTrials.gov: NCT03459287) to evaluate the efficacy and safety of RBCs prepared with the INTERCEPT RBC System in patients requiring transfusion for acute blood loss during complex cardiac surgery. Adult subjects at high risk of need for RBC transfusion received either conventional (control) or INTERCEPT RBCs during and for 7 days after complex cardiovascular surgery (CVS), including thoracic aorta surgery. The primary efficacy endpoint was the proportion of transfused patients with acute kidney injury (AKI) defined by increased serum creatinine (≥0.3 mg/dL or 26.5 µmol/L) from pre-transfusion baseline, within 48 hours of surgery. Safety endpoints were defined as related TEAEs through 28 days after enrollment; or antibodies with confirmed specificity to INTERCEPT RBCs through 75 days after enrollment. The ReCePI trial enrolled and randomized a total of 581 patients across 18 clinical study sites. The mITT population included 321 patients requiring RBC transfusions in the trial. Not all enrolled patients required RBC transfusion. INTERCEPT RBCs used in the ReCePI trial were produced by several blood centers in the U.S.
The development program of the INTERCEPT Red Blood Cell System has been funded in part with federal funds from the Department of Health and Human Services; Administration for Strategic Preparedness and Response; BARDA, under contract number HHSO100201600009C.
ABOUT THE INTERCEPT BLOOD SYSTEM FOR RED BLOOD CELLS
The INTERCEPT Blood System for Red Blood Cells is designed to inactivate blood-borne pathogens and donor leukocytes in RBCs intended for transfusion. The RBC system is designed to prevent pathogen replication by using a small molecule compound added to RBC concentrates to form bonds within nucleic acids in pathogens and leukocytes that may be present in donated RBC collections. The nucleic acid targeted mechanism of action is designed to preserve the therapeutic qualities of the RBCs. RBCs, platelets, and plasma do not require nucleic acid replication for therapeutic efficacy. The RBC system is under development and is not yet approved anywhere in the world. Cerus has filed for a CE Mark for the RBC system under the Medical Device Regulation and anticipates initiating a modular PMA application submission to the FDA in the second half of 2025.
ABOUT CERUS
Cerus Corporation is dedicated solely to safeguarding the world’s blood supply and aims to become the preeminent global blood products company. Headquartered in Concord, California , the company develops and supplies vital technologies and pathogen-protected blood components to blood centers, hospitals, and ultimately patients who rely on safe blood. The INTERCEPT Blood System for platelets and plasma is available globally and remains the only pathogen reduction system with both CE mark and FDA approval for these two blood components. The INTERCEPT red blood cell system is under regulatory review in Europe , and in late-stage clinical development in the US. Also in the US, the INTERCEPT Blood System for Cryoprecipitation is approved for the production of Pathogen Reduced Cryoprecipitated Fibrinogen Complex (commonly referred to as INTERCEPT Fibrinogen Complex), a therapeutic product for the treatment and control of bleeding, including massive hemorrhage, associated with fibrinogen deficiency. For more information about Cerus, visit www.cerus.com and follow us on LinkedIn.
INTERCEPT and the INTERCEPT Blood System are trademarks of Cerus Corporation.
Forward Looking Statements
Except for the historical statements contained herein, this press release contains forward-looking statements concerning Cerus’ products, prospects and expected results, including statements relating to: the therapeutic and commercial potential of INTERCEPT RBCs, including Cerus potentially making pathogen reduction available for RBCs; Cerus’ planned modular premarket approval (PMA) submission and the anticipated timing thereof, including Cerus’ plan to include data from both the ReCePI and RedeS clinical trials in an integrated safety analysis as part of its planned modular PMA submission; the anticipated completion of the RedeS clinical trial and the anticipated timing thereof; and other statements that are not historical fact. 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 development and regulatory process, including the risks that (a) the topline results reported herein and related findings and conclusions are subject to change following a more comprehensive review of the data from the ReCePI clinical trial, (b) Cerus’ planned modular PMA submission for INTERCEPT RBCs may be delayed or may not occur for a variety reasons, including those outside of the control of Cerus, (c) Cerus may be unable to report data from its RedeS clinical trial in a timely manner or at all and that even if reported, such data may not be supportive of Cerus’ planned modular PMA submission for INTERCEPT RBCs, (d) even if submitted, Cerus’ planned modular PMA submission for INTERCEPT RBCs may not be accepted by the FDA for review in a timely manner or at all or if accepted for review, may not be approved by the FDA in a timely manner or at all, (e) Cerus’ expectation that, if Cerus’ planned modular PMA submission for INTERCEPT RBCs is approved, the FDA would require a post marketing study, (f) Cerus may be unable to obtain CE Mark approval of INTERCEPT RBCs in a timely manner or at all, and (g) Cerus may otherwise be unable to obtain the requisite regulatory approvals to advance its pipeline programs and bring them to market in a timely manner or at all; risks related to product safety, including the occurrence of antibody reactivity to INTERCEPT RBCs; Cerus’ ability to maintain an effective, secure manufacturing supply chain, including the risks that (a) Cerus’ supply chain could be negatively impacted as a result of the evolving impact of macroeconomic developments, including the ongoing military conflicts in Ukraine and Israel , rising interest rates, inflation and the evolving effects of the COVID-19 pandemic, (b) Cerus’ manufacturers could be unable to comply with extensive FDA and foreign regulatory agency requirements, and (c) Cerus may be unable to maintain its supply agreements with its third party suppliers; risks related to Cerus’ ability to demonstrate to the transfusion medicine community and other health care constituencies that pathogen reduction is safe, effective and economical; risks associated with Cerus’ need for additional funding; risks associated with macroeconomic developments, including ongoing military conflicts in Ukraine and Israel and the COVID-19 pandemic and resulting global economic and financial disruptions, and the current and potential future negative impacts to Cerus’ business operations; and other risks detailed in Cerus’ filings with the Securities and Exchange Commission, including under the heading “Risk Factors” in Cerus’ Annual Report on Form 10-K for the year ended December 31, 2023, filed with the SEC March 5, 2024. Cerus disclaims any obligation or undertaking to update or revise any forward-looking statements contained in this press release.
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Jessica Hanover – Vice President, Corporate Affairs
Source: Cerus Corporation
What are the topline results of Cerus 's ReCePI Phase 3 trial?
The trial showed non-inferiority of INTERCEPT Red Blood Cells in acute kidney injury incidence compared to conventional RBCs.
What is the ticker symbol for Cerus ?
The ticker symbol for Cerus is CERS.
What is the primary efficacy endpoint met in the ReCePI trial?
The trial demonstrated non-inferiority of INTERCEPT Red Blood Cells in the incidence of acute kidney injury compared to conventional RBCs.
What is the safety endpoint related to treatment-emergent adverse events in the trial?
The safety endpoint showed no significant difference between INTERCEPT RBCs and conventional RBCs in related treatment-emergent adverse events.
Who is the chief medical officer and principal investigator for the ReCePI clinical trial?
Richard Benjamin, M.D., Ph.D., serves as Cerus' chief medical officer and principal investigator for the ReCePI clinical trial.
