New Data from Ambry Genetics Showed Concurrent RNA and DNA Testing Identified More Patients with Hereditary Breast Cancer than DNA Testing Alone

On December 10, 2019 Ambry Genetics (Ambry), a leading clinical genetic testing lab, reported that it will announce new data showing that conducting RNA and DNA tests for hereditary cancer risk at the same time identifies more patients with mutations that increase cancer risk than DNA testing alone (Press release, Ambry Genetics, DEC 10, 2019, View Source [SID1234552216]). To be presented at the San Antonio Breast Cancer Symposium (SABCS) this week, the data come from a study of 746 patients with breast cancer that received +RNAinsight, paired RNA and DNA genetic testing for hereditary cancer risk.

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Standard DNA testing for hereditary cancer risk excludes large portions of DNA, thereby missing some mutations. In addition, DNA testing can produce inconclusive results and fail to determine that an error in our DNA increases cancer risk. These limitations impact patients and their families because doctors may not have the information needed to recommend appropriate preventive, early detection, or therapeutic steps. Additionally, relatives may not be referred for genetic testing and obtain the care they would otherwise have gotten if they had learned they had mutations.

Adding RNA to DNA testing overcomes these limitations for a substantial number of patients as it provides considerably more evidence than DNA testing alone about whether our DNA has mutations.

The data showed that adding RNA genetic testing to DNA testing increased the diagnostic yield – the number of people found to have a mutation that increases cancer risk – across 16 hereditary breast and/or ovarian cancer genes. As a result of +RNAinsight, five breast cancer patients were identified to have mutations in clinically-actionable genes that would have otherwise been missed completely or the patient would have received inconclusive results if they had received DNA testing only. These findings included three women with mutations in BRCA1/2, one woman with a mutation in ATM, and one woman with a mutation in PMS2. Additionally, paired RNA and DNA genetic testing decreased the number of inconclusive results, giving patients more definitive answers about whether their breast cancers were hereditary. Additional results will be presented on an expanded breast cancer cohort at the meeting on Saturday, December 14th.

"These data further prove that paired RNA and DNA genetic testing for hereditary cancer should be the industry standard," said Holly LaDuca, MS, CGC, senior manager of Ambry’s clinical affairs research. "Our research has consistently shown that +RNAinsight provides clinicians with more accurate results, better informing patient care."

Researchers from Ambry will also present at SABCS new data from a pre-and post-test clinician survey that assessed how genetic testing for hereditary cancer impacted medical management, such as screening recommendations. The survey found that positive genetic testing results frequently lead to changes in management recommendations in both high risk (e.g. BRCA1) and moderate risk (e.g. ATM) genes. Changes to mammogram, breast MRI, and/or preventive surgery options were reported in 77.3% of positive individuals. Moreover, medical management changes largely adhered to published guidelines, indicating that clinicians are applying recommendations appropriately based on test results.

"With this survey data, clinicians are showing us that they truly do use genetic testing results to implement personalized recommendations, which can be life-saving for a patient," said Carrie Horton, MS, CGC, senior researcher in Ambry’s clinical affairs team. "These data provide further evidence that genetic testing is essential to comprehensive cancer care. Continued study in this area will aid clinicians, laboratories, health plans, and ultimately patients."

Below are summaries of each of the four studies that Ambry will present at SABCS 2019.

Friday, December 13, 5:00- 7:00 PM CST

P5-07-06, Black M, et. al., Performance of Polygenic Risk Score Combined with Clinical Assessment for Breast Cancer Risk

Findings suggest that the 100-SNP polygenic risk score significantly improves estimation of breast cancer risk based on non-genetic models, and can be used to further identify women at increased lifetime risk who would otherwise not be identified by clinical assessment alone.
Saturday, December 14, 7:00 – 9:00 AM CST

P6-08-35, Horton C, et. al., Impact of Multigene Panel Testing on Medical Management: Preliminary Results of a Pre- and Post- Test Clinician Survey

The data from this ongoing study demonstrate that positive genetic test results frequently lead to changes in medical management and in some cases therapeutic recommendations.
P6-08-08, LaDuca H, et. al., Concurrent DNA and RNA Genetic Testing Identifies More Patients with Hereditary Breast Cancer than DNA Testing Alone

Concurrent DNA and RNA genetic testing for hereditary cancer risk through +RNAinsight identified more disease-causing mutations compared to DNA-only testing. In this study, five patients with breast cancer learned of their genetic mutations as a result of +RNAinsight and would have been missed with DNA testing alone.
P6-08-04, Yadav S, et. al., Germline Mutations in Cancer Predisposition Genes in Patients with Invasive Lobular Carcinoma of the Breast

Invasive lobular carcinoma (ILC) is the second-most common type of breast cancer, but the mutations that increase patients’ risk of developing ILC are largely unknown. The findings from this study improve our understanding of the mutations that increase patients’ risks for ILC and suggest that multigene genetic testing should be considered for all ILC patients.

CNS Pharmaceuticals Received Positive FDA Pre-IND Guidance for Berubicin

On December 10, 2019 CNS Pharmaceuticals, Inc. (NASDAQ: CNSP) ("CNS" or the "Company"), a biotechnology company specializing in the development of novel treatments for brain tumors, reported that it received positive feedback from the U.S. Food and Drug Administration (FDA) for its Pre-IND (Investigational New Drug) meeting proposal to use a lyophilized drug product, Berubicin, in Phase II clinical trials (Press release, CNS Pharmaceuticals, DEC 10, 2019, View Source [SID1234552217]).

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In its positive response to the Company’s previously submitted Pre-IND request, the FDA indicated that the proposal to use a lyophilized drug product in the Phase II clinical trial appears reasonable. The FDA recommended that the existing supply of Berubicin be reprocessed by batch recrystallization. Furthermore, the FDA noted that the requested dosage regimen, which will be based on the Reata Phase I trial, was reasonable. The Company plans to reprocess its existing supply of Berubicin ahead of its upcoming potential Phase II study.

"We were encouraged by the initial feedback provided by the FDA as we believe the availability of our existing supply of Berubicin for a Phase II trial represents significant costs savings and eliminates excess risk and time," stated CEO of CNS, John M. Climaco. "We are continuing the process of preparing a new IND in accordance with the guidance we received from the FDA. We look forward to our upcoming potential Phase II trial evaluating the efficacy of Berubicin in subjects who have glioblastoma that has recurred or progressed following radiation therapy and temozolomide as we believe in Berubicin’s potential to offer oncologists the only anthracycline effective against brain cancer."

About Berubicin
Berubicin is an anthracycline, a class of drugs among the most powerful chemotherapy drugs and effective against more types of cancer than any other class of chemotherapeutic agents. Anthracyclines are designed to damage the DNA of targeted cancer cells by interfering with the action of the topoisomerase II, a critical enzyme enabling cell proliferation. Berubicin was developed at the MD Anderson Cancer Center (MDACC), the world’s largest cancer research facility. Berubicin appeared to demonstrate one Durable Complete Response in a Phase I human clinical trial conducted by a prior developer.

INTERIM 1-YEAR CLINICAL IMMUNE MONITORING DATA FROM DC VACCINE TRIAL IN AML PRESENTED AT ASH 2019 ANNUAL MEETING

On December 10, 2019 Medigene AG (FSE: MDG1, Prime Standard), a clinical stage immuno-oncology company focusing on the development of T cell immunotherapies, reported that Medigene’s researchers presented clinical immune monitoring data obtained during the first year of treatment from the ongoing Phase I/II clinical trial with Medigene’s DC vaccine for the treatment of acute myeloid leukemia (AML) at the 61st ASH (Free ASH Whitepaper) Annual Meeting (Orlando, USA) (Press release, MediGene, DEC 10, 2019, View Source [SID1234552258]).

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This ongoing DC vaccine clinical trial for the treatment of AML will be completed at the end of 2019 and topline data will be reported in the beginning of 2020.

The poster presentation focused on clinical immune monitoring data from the trial. Several assay systems were used to assess the impact of the DC vaccine on T cell responses (specific for WT-1 and PRAME antigens) and other immunological parameters. Furthermore, the recurrence of WT-1-positive and/or PRAME-positive AML blasts, as well as the occurrence of AML-specific mutations in bone marrow, were investigated. The results of the 1-year interim assessment show that, upon in vitro stimulation with WT-1 or PRAME, T cells from peripheral blood of 75% (6 of 8) of relapsing patients were capable of producing the pro-inflammatory cytokine IFNgamma, while such a response was detected in only in 25% (3 of 12) of the patients in remission.

Dr. Kai Pinkernell, CMO and CDO of Medigene AG, comments: "These interim clinical immune monitoring data from our DC trial after one year of treatment shed light on the potential functionality of our vaccine. We believe that the detection of reactive T cells in peripheral blood may be linked to the concomitant presence of AML blasts in the periphery of relapsing patients. In addition, in most patients in remission the stable or decreasing levels of WT-1 and/or PRAME mRNA in the bone marrow would be consistent with a local response against AML antigens, despite a negative IFN-gamma response in peripheral blood. Further monitoring of the patients in remission in the second year of vaccination may bring further insights into the role of DC vaccination in the prevention of AML recurrence."

The poster entitled "DC Vaccination Induces Antigen Specific Immune Responses in AML Patients: A 1-Year Interim Assessment" is available at Medigene’s company website at: View Source

About Medigene’s DC trial:
A total of 20 subjects (median age 59, range 24 to 73) with AML (risk groups good, intermediate, poor: 13, 5, 2), in morphologic complete remission (CR) or complete remission with incomplete hematologic recovery (CRi) after induction or consolidation therapy, not eligible for allogeneic hematopoietic stem cell transplantation, were enrolled into this safety and feasibility Phase I/II trial, vaccinated and followed up for 12 months at the interim analysis timepoint. Subjects in this trial had AML that was positive for Wilms Tumor-1 (WT-1) antigen with or without positivity for Preferentially Expressed Antigen in Melanoma (PRAME). Vaccination with dendritic cells, presenting the antigens WT-1 and PRAME, was carried out monthly, with a higher frequency within the first 6 weeks. AML diagnoses had been established with a median of 9.8 months before the first vaccination (range 4.5 to 17.5 months), and the last chemotherapy infusion had been performed at a median of 6.9 months (range 2 to 14.8 months).

Interim results:
The vaccinations were well tolerated with no serious adverse events (SAEs) related to the treatment. The most common adverse events (AEs) were injection site related, accounting for 35% of all AEs and mild in nature (Grade 1). After a 12-month treatment period, the overall survival was 89% (18 of 20 patients, 95% confidence interval: 61 to 97%) and the progression free survival was 60% (12 of 20 patients, 95% confidence interval: 36 to 78%). Most relapses, 5 out of 8, occurred within the first 80 days after initiation of vaccination, suggesting a possible molecular relapse upon entering the study.

About Medigene’s DC vaccines:
In addition to Medigene’s development focus on T cell-receptor modified T cells (TCR-Ts), the Company has developed a new generation of antigen-tailored dendritic cell (DC) vaccines.
Dendritic cells (DC) can take up antigens, process them and present peptides on their surface in a form that can induce antigen-specific T cells to mature and proliferate. In this way, T cells recognize and eliminate tumor cells which bear the same antigen peptide on their surface. Dendritic cells can also induce natural killer cells (NK cells) to attack tumor cells. The scientific team of Medigene has developed new, fast and efficient methods for generating autologous (patient-specific) mature dendritic cells which have the relevant characteristics to generate very strong T cell and NK cell immune responses. The dendritic cells can be loaded with various tumor antigens to treat different forms of cancer. Since an immune response builds up over the total time of administration of the DC vaccine, this form of therapy is particularly designed for patients who suffer from a tumor disease which has been reduced to such an extent by chemotherapy that the prevention of the recurrence of the tumor disease is the main goal.

About acute myeloid leukemia (AML):
Acute myeloid leukemia is a malignant disease of the hematopoietic system, affecting mainly adults above 60 years of age. In Germany, about 3,600 cases are registered annually.
AML is caused by uncontrolled growth of dysfunctional hematopoietic precursor cells in the bone marrow. These cells prevent the generation of normal blood cells, causing a decrease in erythrocytes and platelets, for example. Typical symptoms of AML include anemia, fever, increased risk of infection, and bleeding. AML progresses rapidly and may be fatal within a few weeks or months, if untreated.
AML treatment is often started with intensive chemotherapy, followed by consolidation with or without allogeneic hematopoietic stem cell transplantation. Unfortunately, a significant proportion of patients suffer a relapse of the original disease. Depending on the biologic risk profile of the disease, age and co-morbidity the long-term survival is highly variable.

Vedanta Biosciences Announces Initiation of First-in-Patient Study of Immuno-Oncology Candidate VE800 In Combination with Bristol-Myers Squibb’s Opdivo® (Nivolumab)

On December 10, 2019 Vedanta Biosciences, Inc. (Vedanta Biosciences, Vedanta or the Company), a clinical-stage biopharmaceutical company developing a new category of therapies for immune-mediated diseases based on defined bacterial consortia, reported the initiation of a first-in-patient clinical study of VE800 in combination with Bristol-Myers Squibb’s programmed death-1 (PD-1) immune checkpoint inhibitor Opdivo (nivolumab) in patients with select types of advanced or metastatic cancer (Press release, Vedanta Biosciences, DEC 10, 2019, View Source [SID1234552202]). Vedanta also announced the formation of its Immuno-Oncology Scientific Advisory Board (SAB), which is comprised of experts in immunology, immuno-oncology and the microbiome, to support the planned clinical development of VE800.

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The study, which is being conducted at clinical centers in the United States, will evaluate the safety and tolerability and clinical activity of VE800 in combination with Opdivo, as measured by the confirmed overall response rate, in addition to other parameters. The open-label, non-randomized study will target enrollment of over 100 patients diagnosed with advanced or metastatic melanoma, gastric/gastroesophageal junction adenocarcinoma, or microsatellite-stable colorectal cancer. Eligible patients will receive daily VE800 dosing in combination with Opdivo. Topline results are anticipated in 2021.

"Despite unprecedented global investment in checkpoint inhibitors, there is still a major need for differentiated approaches to further enhance and expand responses in cancer," said Bernat Olle, Ph.D., co-founder and chief executive officer of Vedanta Biosciences. "The role the gut microbiota plays in influencing responses to immunotherapies has been ignored by previous approaches, so we are excited about the potential of microbiome modulation to open up an entirely new approach to cancer therapy."

VE800 is made up of 11 commensal bacterial strains that act in concert to activate cytotoxic CD8+ T cells, which are the vanguard of the immune system’s response to tumors and thus a key driver of effective immunotherapies. In preclinical studies, VE800 has been shown to enhance the ability of these T cells to infiltrate tumors, thereby promoting suppression of tumor growth and potentially enhancing survival. Preclinical data also suggest that VE800 may enhance the effects of checkpoint inhibitors.

Foundational work demonstrating VE800’s novel anti-tumor activity and cooperatively potentiated responses to checkpoint inhibitor therapies and various immune challenges was published in Nature by Vedanta and its scientific co-founder Kenya Honda, M.D., Ph.D., of Keio University School of Medicine. The research also showed that mice colonized with VE800 demonstrated enhanced therapeutic efficacy in a range of tumor models when VE800 was administered in conjunction with PD-1 or CTLA4 immune checkpoint inhibitors.

"The ability of bacterial consortia to mediate immune activity, including potential anti-cancer activity, is an exciting area for investigation in indications with some of the highest unmet medical need," said Hassane M. Zarour, M.D., co-leader of the Cancer Immunology and Immunotherapy Program of the Hillman Cancer Center, University of Pittsburgh, and a member of Vedanta’s newly formed Immuno-Oncology SAB. "We see enormous potential for this class of drugs to improve cancer patients’ outcomes."

Vedanta’s newly announced Immuno-Oncology SAB will work closely with the Company’s scientific co-founders and leadership to further support the clinical development of VE800. The SAB includes:

Antoni Ribas, M.D., Ph.D.is a leading translational and clinical researcher in immuno-oncology with a focus on malignant melanoma. He is a professor of medicine, surgery and molecular and medical pharmacology at the University of California Los Angeles (UCLA), director of the tumor immunology program at the Jonsson Comprehensive Cancer Center, director of the Parker Institute for Cancer Immunotherapy Center at UCLA, chair of the Melanoma Committee at SWOG and president-elect 2019-2020 of the American Association for Cancer Research (AACR) (Free AACR Whitepaper).
Josep Tabernero, M.D., Ph.D. is a researcher focused on gastrointestinal cancers and cancer genetics. At Vall D’Hebron Institute of Oncology (VHIO), he is director of clinical research, co-director of the research unit for molecular therapy of cancer, head of the gastrointestinal and endocrine tumors group, and head of the medical oncology department of Vall d’Hebron University Hospital. He is also president of the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper).
Hassane Zarour, M.D. is a researcher with expertise in melanoma and skin lesions, immunotherapy and cancer vaccines. At the University of Pittsburgh Medical Center, he is a professor of medicine, immunology and dermatology, co-leader of the melanoma program and the James W. and Frances G. McGlothlin chair in melanoma immunotherapy research.
Diwakar Davar, MBBS, M.Sc. is a leader in microbiome science and using the microbiome to treat melanoma. He is an assistant professor of medicine at the University of Pittsburgh Medical Center.
Bertrand Routy, M.D., Ph.D. is recognized as one of the first researchers to demonstrate the negative impact of antibiotics and the microbiome on the efficacy of checkpoint inhibitor therapy. He is an assistant professor of hematology-oncology and director of the laboratory of immunotherapy / oncomicrobiome at the Centre hospitalier de l’Université de Montréal (CHUM).
Dan Littman, M.D., Ph.D. is a scientific co-founder of Vedanta and a leader in T cell biology, including differentiation and lineage specification. He is the Helen L. and Martin S. Kimmel professor of molecular immunology and pathology and professor in the department of microbiology at the Skirball Institute of Biomolecular Medicine at New York University Langone School of Medicine.
Sasha Rudensky, Ph.D. is a scientific co-founder of Vedanta and a leader in molecular mechanisms of CD4 T cell differentiation, particularly regulatory T cells. He is chair of the immunology program at Sloan Kettering Institute (SKI) and director of the Ludwig Center at Memorial Sloan Kettering (MSK).
About VE800

VE800 is Vedanta Biosciences’ proprietary, orally administered immuno-oncology product candidate. It is produced from pure, non-pathogenic clonal bacterial cell banks, which yield a standardized drug product in powdered form. VE800 consists of a rationally-defined bacterial consortium of 11 commensal strains that, acting in concert, activate cytotoxic CD8+ T cells, a type of white blood cell that is the predominant effector in cancer immunotherapy. In preclinical studies, VE800 has been shown to enhance the ability of these T cells to infiltrate tumors, thereby promoting suppression of tumor growth and enhancing survival. Preclinical data also suggest that VE800 may enhance the effects of checkpoint inhibitors. Vedanta is evaluating VE800 as a potential treatment for patients with advanced or metastatic cancers.

Amgen Data From Phase 3 CANDOR Study Combining KYPROLIS® (carfilzomib) And DARZALEX® (daratumumab) To Be Presented During Late-Breaking Session At American Society Of Hematology Annual Meeting

On December 10, 2019 Amgen (NASDAQ:AMGN) reported additional results from the primary analysis of the Phase 3 CANDOR study evaluating KYPROLIS (carfilzomib) in combination with dexamethasone and DARZALEX (daratumumab) (KdD) compared to KYPROLIS and dexamethasone alone (Kd) in patients with relapsed or refractory multiple myeloma (Press release, Amgen, DEC 10, 2019, View Source [SID1234552218]). The data will be presented in a late-breaking abstract session at the 61st American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting & Exposition.

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At a median follow up of 17 months, the study met its primary endpoint of progression-free survival (PFS), resulting in a 37% reduction in the risk of disease progression or death in patients receiving KdD (HR=0.63; 95% CI: 0.464, 0.854; p=0.0014). Median PFS was not reached for the KdD arm versus 15.8 months for the Kd arm.

"This primary analysis of the CANDOR study adds to the body of evidence supporting the combination of KYPROLIS and DARZALEX, two powerful targeted agents for multiple myeloma," said David M. Reese, M.D., executive vice president of Research and Development at Amgen. "KYPROLIS has demonstrated deep and sustained responses in treating patients with multiple myeloma that have relapsed. The CANDOR study now offers additional insight into the effectiveness of this combination as a potential new treatment option for relapsed myeloma patients."

In addition to meeting the primary endpoint, the KdD combination demonstrated efficacy in key secondary endpoints, including overall response rate (ORR), minimal residual disease (MRD) negative-complete response at 12 months and overall survival (OS). The ORR was 84.3% versus 74.7% (p=0.0040), and the rate of complete response or better was 28.5% versus 10.4% for the KdD and Kd arms, respectively. The analysis found the MRD-negative complete response rate at 12 months was 12.5% for KdD versus 1.3% for Kd (p<0.0001), a nearly 10-times higher response rate versus Kd-treated patients. The median OS was not reached in either group (HR=0.75; 95% CI: 0.49, 1.13; p=0.08).

"With the increasing use of frontline lenalidomide based therapies, there is an emerging need for lenalidomide-sparing regimens at relapse," said Saad Usmani, M.D., chief of the Plasma Cell Disorders Division and the director of Clinical Research in Hematologic Malignancies, Atrium Health’s Levine Cancer Institute (LCI). "The CANDOR trial demonstrates the potential efficacy of a lenalidomide-sparing regimen that combines two effective targeted agents and provides deep and durable responses upon relapse."

The safety of KdD was consistent with the known safety profiles of the individual agents. The most frequently reported (≥ 20% of subjects in either treatment arm [KdD, Kd]) treatment emergent adverse events included thrombocytopenia, anemia, diarrhea, hypertension upper respiratory tract infection, fatigue, and dyspnea. The incidence of treatment emergent grade 3 or higher, serious, and fatal adverse events was higher in the KdD arm compared to the Kd arm. The rate of treatment discontinuation due to AEs was similar in both arms.

Additional efficacy endpoints and key subgroup analyses will be presented at future meetings.

About CANDOR
CANDOR, a randomized, open-label Phase 3 study of KYPROLIS, dexamethasone and DARZALEX (KdD) compared to KYPROLIS and dexamethasone (Kd), evaluated 466 relapsed or refractory multiple myeloma patients who have received one to three prior therapies. Patients were treated until disease progression. The primary endpoint was PFS, and the key secondary endpoints were overall response rate, minimal residual disease and overall survival. PFS was defined as time from randomization until disease progression or death from any cause.

In the first arm, patients received KYPROLIS twice weekly at 56 mg/m2 and dexamethasone in combination with DARZALEX. In the second arm (control), patients received KYPROLIS twice weekly at 56 mg/m2 and dexamethasone.

CANDOR was initiated as part of a collaboration with Janssen, and under the terms of the agreement, Janssen co-funded the study. For more information about this trial, please visit www.clinicaltrials.gov under trial identification number NCT03158688.

About Multiple Myeloma
Multiple myeloma is an incurable blood cancer, characterized by a recurring pattern of remission and relapse.1 It is a rare and life-threatening disease that accounts for approximately one percent of all cancers.2,3 Worldwide, approximately 160,000 people are diagnosed with multiple myeloma each year, and 106,000 patient deaths are reported on an annual basis.2

About KYPROLIS (carfilzomib)
Proteasomes play an important role in cell function and growth by breaking down proteins that are damaged or no longer needed.4 KYPROLIS has been shown to block proteasomes, leading to an excessive build-up of proteins within cells.5 In some cells, KYPROLIS can cause cell death, especially in myeloma cells because they are more likely to contain a higher amount of abnormal proteins.4,5

Since its first approval in 2012, approximately 130,000 patients worldwide have received KYPROLIS. KYPROLIS is approved in the U.S. for the following:

In combination with dexamethasone or with lenalidomide plus dexamethasone for the treatment of patients with relapsed or refractory multiple myeloma who have received one to three lines of therapy
As a single agent for the treatment of patients with relapsed or refractory multiple myeloma who have received one or more lines of therapy
KYPROLIS is also approved in Algeria, Argentina, Australia, Bahrain, Belarus, Brazil, Canada, Chile, Colombia, Ecuador, Egypt, European Union, Hong Kong, India, Israel, Japan, Jordan, Kuwait, Lebanon, Macao, Malaysia, Mexico, Morocco, New Zealand, Oman, Peru, Philippines, Qatar, Russia, Saudi Arabia, Singapore, S. Korea, Switzerland, Taiwan, Thailand, Turkey and United Arab Emirates.

Important U.S. KYPROLIS (carfilzomib) Safety Information

Cardiac Toxicities

New onset or worsening of pre-existing cardiac failure (e.g., congestive heart failure, pulmonary edema, decreased ejection fraction), restrictive cardiomyopathy, myocardial ischemia, and myocardial infarction including fatalities have occurred following administration of KYPROLIS. Some events occurred in patients with normal baseline ventricular function. Death due to cardiac arrest has occurred within one day of administration.
Monitor patients for signs or symptoms of cardiac failure or ischemia. Evaluate promptly if cardiac toxicity is suspected. Withhold KYPROLIS for Grade 3 or 4 cardiac adverse events until recovery, and consider whether to restart at 1 dose level reduction based on a benefit/risk assessment.
While adequate hydration is required prior to each dose in Cycle 1, monitor all patients for evidence of volume overload, especially patients at risk for cardiac failure. Adjust total fluid intake as clinically appropriate.
For patients ≥ 75 years, the risk of cardiac failure is increased. Patients with New York Heart Association Class III and IV heart failure, recent myocardial infarction, conduction abnormalities, angina, or arrhythmias may be at greater risk for cardiac complications and should have a comprehensive medical assessment prior to starting treatment with KYPROLIS and remain under close follow-up with fluid management.
Acute Renal Failure

Cases of acute renal failure, including some fatal renal failure events, and renal insufficiency adverse events (including renal failure) have occurred. Acute renal failure was reported more frequently in patients with advanced relapsed and refractory multiple myeloma who received KYPROLIS monotherapy. Monitor renal function with regular measurement of the serum creatinine and/or estimated creatinine clearance. Reduce or withhold dose as appropriate.
Tumor Lysis Syndrome

Cases of Tumor Lysis Syndrome (TLS), including fatal outcomes, have occurred. Patients with a high tumor burden should be considered at greater risk for TLS. Adequate hydration is required prior to each dose in Cycle 1, and in subsequent cycles as needed. Consider uric acid lowering drugs in patients at risk for TLS. Monitor for evidence of TLS during treatment and manage promptly, and withhold until resolved.
Pulmonary Toxicity

Acute Respiratory Distress Syndrome (ARDS), acute respiratory failure, and acute diffuse infiltrative pulmonary disease such as pneumonitis and interstitial lung disease have occurred. Some events have been fatal. In the event of drug‐induced pulmonary toxicity, discontinue KYPROLIS.
Pulmonary Hypertension

Pulmonary arterial hypertension (PAH) was reported. Evaluate with cardiac imaging and/or other tests as indicated. Withhold KYPROLIS for PAH until resolved or returned to baseline and consider whether to restart based on a benefit/risk assessment.
Dyspnea

Dyspnea was reported in patients treated with KYPROLIS. Evaluate dyspnea to exclude cardiopulmonary conditions including cardiac failure and pulmonary syndromes. Stop KYPROLIS for Grade 3 or 4 dyspnea until resolved or returned to baseline. Consider whether to restart based on a benefit/risk assessment.
Hypertension

Hypertension, including hypertensive crisis and hypertensive emergency, has been observed, some fatal. Control hypertension prior to starting KYPROLIS. Monitor blood pressure regularly in all patients. If hypertension cannot be adequately controlled, withhold KYPROLIS and evaluate. Consider whether to restart based on a benefit/risk assessment.
Venous Thrombosis

Venous thromboembolic events (including deep venous thrombosis and pulmonary embolism) have been observed. Thromboprophylaxis is recommended for patients being treated with the combination of KYPROLIS with dexamethasone or with lenalidomide plus dexamethasone. The thromboprophylaxis regimen should be based on an assessment of the patient’s underlying risks.
Patients using hormonal contraception associated with a risk of thrombosis should consider an alternative method of effective contraception during treatment.
Infusion Reactions

Infusion reactions, including life‐threatening reactions, have occurred. Signs and symptoms include fever, chills, arthralgia, myalgia, facial flushing, facial edema, laryngeal edema, vomiting, weakness, shortness of breath, hypotension, syncope, chest tightness, or angina. These reactions can occur immediately following or up to 24 hours after administration. Pre-medicate with dexamethasone to reduce the incidence and severity of infusion reactions. Inform patients of the risk and of symptoms and seek immediate medical attention if they occur.
Hemorrhage

Fatal or serious cases of hemorrhage have been reported. Hemorrhagic events have included gastrointestinal, pulmonary, and intracranial hemorrhage and epistaxis. Promptly evaluate signs and symptoms of blood loss. Reduce or withhold dose as appropriate.
Thrombocytopenia

KYPROLIS causes thrombocytopenia with recovery to baseline platelet count usually by the start of the next cycle. Monitor platelet counts frequently during treatment. Reduce or withhold dose as appropriate.
Hepatic Toxicity and Hepatic Failure

Cases of hepatic failure, including fatal cases, have occurred. KYPROLIS can cause increased serum transaminases. Monitor liver enzymes regularly regardless of baseline values. Reduce or withhold dose as appropriate.
Thrombotic Microangiopathy

Cases of thrombotic microangiopathy, including thrombotic thrombocytopenic purpura/hemolytic uremic syndrome (TTP/HUS), including fatal outcome have occurred. Monitor for signs and symptoms of TTP/HUS. Discontinue if diagnosis is suspected. If the diagnosis of TTP/HUS is excluded, KYPROLIS may be restarted. The safety of reinitiating KYPROLIS is not known.
Posterior Reversible Encephalopathy Syndrome (PRES)

Cases of PRES have occurred in patients receiving KYPROLIS. If PRES is suspected, discontinue and evaluate with appropriate imaging. The safety of reinitiating KYPROLIS is not known.
Increased Fatal and Serious Toxicities in Combination with Melphalan and Prednisone in Newly Diagnosed Transplant-ineligible Patients

In a clinical trial of transplant-ineligible patients with newly diagnosed multiple myeloma comparing KYPROLIS, melphalan, and prednisone (KMP) vs bortezomib, melphalan, and prednisone (VMP), a higher incidence of serious and fatal adverse events was observed in patients in the KMP arm. KMP is not indicated for transplant-ineligible patients with newly diagnosed multiple myeloma.
Embryo-fetal Toxicity

KYPROLIS can cause fetal harm when administered to a pregnant woman.
Females of reproductive potential should be advised to avoid becoming pregnant while being treated with KYPROLIS and for 6 months following the final dose. Males of reproductive potential should be advised to avoid fathering a child while being treated with KYPROLIS and for 3 months following the final dose. If this drug is used during pregnancy, or if pregnancy occurs while taking this drug, the patient should be apprised of the potential hazard to the fetus.
ADVERSE REACTIONS

The most common adverse reactions in the combination therapy trials: anemia, neutropenia, diarrhea, dyspnea, fatigue, thrombocytopenia, pyrexia, insomnia, muscle spasm, cough, upper respiratory tract infection, hypokalemia.
The most common adverse reactions in monotherapy trials: anemia, fatigue, thrombocytopenia, nausea, pyrexia, dyspnea, diarrhea, headache, cough, edema peripheral.
Please see full Prescribing Information at www.kyprolis.com.

About Amgen Oncology
Amgen Oncology is searching for and finding answers to incredibly complex questions that will advance care and improve lives for cancer patients and their families. Our research drives us to understand the disease in the context of the patient’s life – not just their cancer journey – so they can take control of their lives.

For the last four decades, we have been dedicated to discovering the firsts that matter in oncology and to finding ways to reduce the burden of cancer. Building on our heritage, Amgen continues to advance the largest pipeline in the Company’s history, moving with great speed to advance those innovations for the patients who need them.

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