Chugai to Present Japanese Phase III Results on Alecensa® at ASCO

On May 19, 2016 Chugai Pharmaceutical Co., Ltd. (TOKYO: 4519) reported that the results of the Japanese phase III study (J-ALEX) of Alecensa, in ALK fusion gene positive non-small cell lung cancer (NSCLC) patients, will be presented at the annual meeting of the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) which will be held June 3 -7 in Chicago, IL (USA) (Press release, Chugai, MAY 19, 2016, View Source [SID:1234512571]). Results from the J-ALEX study will be presented at the oral abstract sessions scheduled for June 6 (CDT).

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Abstract #9008
Alectinib (ALC) versus crizotinib (CRZ) in ALK-inhibitor naïve ALK-positive non-small cell lung cancer (ALK+NSCLC): primary results from the J-ALEX study

The J-ALEX study was an open-label, randomized phase III study that compares the efficacy and safety between Alecensa and crizotinib. The J-ALEX study enrolled 207 ALK-inhibitor naïve patients with ALK fusion gene positive advanced or recurrent NSCLC, who either had not undergone chemotherapy or had undergone one chemotherapy regimen. The subjects were allocated to either the Alecensa arm or the crizotinib arm of the study in a one to one ratio. The primary endpoint of the J-ALEX study was progression free survival (PFS) as assessed by a blinded independent review board. The secondary endpoints included overall survival, objective response rate and safety, and other endpoints.

The PFS hazard ratio of the Alecensa arm to the crizotinib arm was 0.34 and Alecensa demonstrated significantly prolonged PFS (99.6826% CI: 0.17-0.70, stratified log-rank p<0.0001). Median PFS was not reached (95% CI: 20.3-Not Estimated) in the Alecensa arm while it was 10.2 months (95%CI: 8.2-12.0) in the crizotinib arm. In the Alecensa arm, constipation (36%) was an adverse event (AE) with >30% frequency, while in the crizotinib arm nausea (74%), diarrhea (73%), vomiting (59%), visual disturbance (55%), dysgeusia (52%), constipation (46%), ALT elevation (32%), and AST elevation (31%) were each seen in >30% patients. Grade 3-4 AEs occurred in 27% of the Alecensa arm and in 51% of the crizotinib arm, there were no treatment-related deaths in either arm.

In February, 2016, Chugai carried out a prospectively defined interim analysis, and had an independent data monitoring committee examine the results. Since the results showed that Alecensa significantly prolonged the PFS to a higher extent than anticipated, the committee decided to recommend an early discontinuation of the J-ALEX study.

"It was found in Japan earlier than in any other country in the world that ALK fusion gene serves as a powerful carcinogenic factor for some types of lung cancer. Alecensa was created by Chugai as a drug selectively inhibiting the activity of ALK fusion gene, and it was first approved in Japan in 2014 based on the Japanese clinical study data. The J-ALEX study, comparing Alecensa therapy directly with standard therapy, demonstrated superiority of Alecensa over standard therapy for the first time in the world. This finding not only greatly encourages the patients suffering from ALK fusion gene positive NSCLC but also illustrates the high level of drug development progression from basic research to clinical studies in Japan," said Chugai’s Director and Executive Vice President, Dr. Yutaka Tanaka. "Chugai is extremely proud of having developed Alecensa which has been shown to provide benefit to patients."

As a top pharmaceutical company in the field of oncology in Japan, Chugai believes that early treatment using Alecensa in ALK fusion gene positive NSCLC is expected to prolong these patients’ PFS and enable them to face their disease with hope for the future.

About Alecensa
Alecensa is a highly selective ALK inhibitor discovered by Chugai. It has been reported that 2 to 5 percent of patients with NSCLC express a chromosomal rearrangement which leads to fusion of the ALK gene with another gene.1) ALK kinase signalling is constantly active in cells with such fusion genes, resulting in uncontrolled growth of tumor cells and transforming the cells into tumor cells.2, 3) Alecensa exerts its anti-tumor effect by selectively inhibiting ALK kinase activity to inhibit tumor cell proliferation and induce cell death.4) In addition, Alecensa is not recognized by the transporter proteins in the blood brain barrier that actively pump molecules out of the brain. Alecensa is active in the central nervous system and has proven activity against brain metastases.

In Japan, Alecensa is available to patients with "ALK fusion gene positive unresectable, recurrent/advanced NSCLC" and is marketed by Chugai. In the US, Alecensa was approved in December 2015 for the indication of "anaplastic lymphoma kinase (ALK) positive, metastatic non-small cell lung cancer (NSCLC) in patients who have progressed on or those who are intolerant to crizotinib." In September 2015, Roche filed the MAA in Europe to the European Medicines Agency for the approval of "ALK fusion gene positive unresectable, recurrent/advanced NSCLC."

Amgen Highlights Data To Be Presented At 21st Congress Of The European Hematology Association

On May 19, 2016 Amgen (NASDAQ: AMGN) reported it will present new data from its oncology portfolio at the 21st Congress of the European Hematology Association (EHA) (Free EHA Whitepaper), June 9-12, 2016, in Copenhagen (Press release, Amgen, MAY 19, 2016, View Source;p=RssLanding&cat=news&id=2170004 [SID:1234512596]). Key data to be presented include studies evaluating BLINCYTO (blinatumomab), Kyprolis (carfilzomib), Aranesp (darbepoetin alfa) and Nplate (romiplostim). Data from the BLINCYTO TOWER study will be presented during the Presidential Symposium on Friday, June 10, and is recognized as a top abstract submitted to the Congress. This, along with other presentations, reinforces Amgen’s commitment to serve patients with hematologic malignancies through the development of innovative and novel products.

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"We are excited that the data from the TOWER study, which is the first randomized study of an immunotherapy to demonstrate overall survival benefit in adult patients with Ph-negative relapsed or refractory B-cell precursor acute lymphoblastic leukemia, will be featured at the Presidential Symposium this year at EHA (Free EHA Whitepaper)," said Sean E. Harper, M.D., executive vice president of Research and Development at Amgen. "This recognition along with other key data being presented validates our ongoing commitment to developing innovative therapies that have the potential to tackle unmet needs in complex-to-treat patient populations."

Key data include findings from clinical trials in acute lymphoblastic leukemia (ALL), multiple myeloma (MM), myelodysplastic syndrome (MDS) and immune thrombocytopenia (ITP):

BLINCYTO data

BLINCYTO was granted conditional marketing authorization by the European Commission (EC) last November and is the first bispecific T cell engager (BiTE) antibody construct approved in the European Union (EU) for the treatment of adults with Philadelphia chromosome-negative (Ph-) relapsed or refractory B-cell precursor ALL. Data from the comprehensive ALL development program to be presented will include:

Blinatumomab Improved Overall Survival in Patients with Relapsed/Refractory Philadelphia Negative B-Cell Precursor Acute Lymphoblastic Leukemia in a Randomized, Open-Label Phase 3 Study (TOWER)
Abstract No. S149, Oral presentation, Presidential symposium, Friday, June 10, 2016, 4:45 – 5 p.m. (CEST), Hall A1
ALL data

Trends in the Use of Hematopoietic Stem Cell Transplantation for Adults with Acute Lymphoblastic Leukemia (ALL): A Report From the Acute Leukemia Working Party of the European Society for Blood and Marrow Transplantation (EBMT)
Abstract No. S524, Oral presentation, Stem cell transplantation – Clinical 1, Saturday, June 11, 2016, 4:45 – 5 p.m. (CEST), Room H5
Kyprolis data

Kyprolis was granted marketing authorization by the EC last November for use in combination treatment of patients with relapsed multiple myeloma. Data to be presented include:

Carfilzomib, Lenalidomide, and Dexamethasone vs. Lenalidomide and Dexamethasone in Patients with Relapsed Multiple Myeloma: Analysis of Response and Progression-Free Survival Hazard Ratio Over Time
Abstract No. P275, Poster presentation, Innovative therapies for MM 1, Friday, June 10, 2016 , 5:15 – 6:45 p.m. (CEST), Poster area (Hall H)
Outcomes for Asian Patients with Relapsed Multiple Myeloma Treated with Carfilzomib and Dexamethasone vs. Bortezomib and Dexamethasone: A Subgroup Analysis of the Phase 3 ENDEAVOR Study (NCT01568866)
Abstract No. E1328, Eposter presentation, Myeloma and other monoclonal gammopathies – Clinical
Carfilzomib and Dexamethasone vs. Subcutaneous Bortezomib and Dexamethasone in Patients with Relapsed or Refractory Multiple Myeloma: Secondary Analysis from the Phase 3 Study ENDEAVOR (NCT01568866)
Abstract No. P659, Poster presentation, Innovative therapies for MM 4 Saturday, June 11, 2016, 5:30 – 7 p.m. (CEST), Poster area (Hall H)
Efficacy and Safety by Cytogenetic Risk Status: Phase 3 Study (ASPIRE) of Carfilzomib, Lenalidomide and Dexamethasone Versus Lenalidomide and Dexamethasone in Patients with Relapsed Multiple Myeloma
Abstract No. P663, Poster presentation, Innovative therapies for MM 4, Saturday, June 11, 2016, 5:30 – 7 p.m. (CEST), Poster area (Hall H)
A Sub-Study of the Phase 3 ENDEAVOR Study: Serial Echocardiographic Assessment of Patients with Relapsed Multiple Myeloma (RMM) Receiving Carfilzomib Plus Dexamethasone or Bortezomib Plus Dexamethasone
Abstract No. P664, Poster presentation, Innovative therapies for MM 4, Saturday, June 11, 2016, 5:30 – 7 p.m. (CEST), Poster area (Hall H)
Carfilzomib and Dexamethasone vs. Bortezomib and Dexamethasone: Subgroup Analysis of Patients with Relapsed Multiple Myeloma by Baseline Cytogenetic Risk Status (Phase 3 ENDEAVOR Study)
Abstract No. E1267, Eposter presentation, Myeloma and other monoclonal gammopathies – Clinical
Carfilzomib and Dexamethasone vs. Bortezomib and Dexamethasone in Patients with Relapsed Multiple Myeloma: Analysis of the Phase 3 ENDEAVOR Study by Age Subgroup
Abstract No. E1274, Eposter presentation, Myeloma and other monoclonal gammopathies – Clinical
Carfilzomib and Dexamethasone vs. Bortezomib and Dexamethasone: Subgroup Analysis of the Phase 3 ENDEAVOR Study to Evaluate the Impact of Prior Treatment on Patients with Relapsed Multiple Myeloma
Abstract No. E1266, Eposter presentation, Myeloma and other monoclonal gammopathies – Clinical
Weekly Carfilzomib with Dexamethasone for Patients with Relapsed or Refractory Multiple Myeloma: Updated Results from the Phase 1/2 Study CHAMPION-1 (NCT01677858)
Abstract No. P661, Poster presentation, Innovative therapies for MM 4, Saturday, June 11, 2016, 5:30 – 7 p.m. (CEST), Poster area (Hall H)
The Effect of Level of Response to Treatment on Associated Costs and Healthcare Resource Utilization: A Retrospective Chart Review Study in Patients with Symptomatic Multiple Myeloma
Abstract No. E1310, Eposter presentation, Myeloma and other monoclonal gammopathies – Clinical
Survival and Treatment Patterns in Patients with Symptomatic Multiple Myeloma (MM) in A Real-World Setting
Abstract No. E1280, Eposter presentation, Myeloma and other monoclonal gammopathies – Clinical
Description of Patient Characteristics, Treatment Patterns and Resource Use for Patients with Multiple Myeloma Treated in Three Local Health Units (LHUS) in Italy
Abstract No. E1327, Eposter presentation, Myeloma and other monoclonal gammopathies – Clinical
Overall Survival in Patients with Symptomatic Multiple Myeloma in the Real-World Setting: A Retrospective Analysis of the Pharos Registry in the Netherlands
Abstract No. E1292, Eposter presentation, Myeloma and other monoclonal gammopathies – Clinical
Aranesp data

Aranesp received initial EU approval within oncology in August 2002 and is indicated for the treatment of symptomatic anemia in adult cancer patients with non-myeloid malignancies receiving chemotherapy. Data to be presented include:

ARCADE (20090160): A Phase 3 Randomized Placebo-Controlled Double-Blind Trial of Darbepoetin Alfa in the Treatment Of Anemia in Patients with Low or Intermediate-1 Risk Myelodysplastic Syndromes (MDS)
Abstract No.S128, Oral presentation, Myelodysplastic syndromes – Clinical, Friday, June 10, 2016, 11:30 – 11:45 a.m. (CEST), Hall C14
Nplate data

Nplate, a thrombopoietin receptor agonist, was approved in the EU in February 2009 for the treatment of adult chronic-immune (idiopathic)-thrombocytopenic-purpura (ITP) patients who are refractory to other treatments (e.g. corticosteroids, immunoglobulins). Data to be presented include:

Romiplostim in Splenectomized (Splnx) and Nonsplenectomized (Nonsplnx) Patients with Immune Thrombocytopenia (ITP)
Abstract No. S520, Oral presentation, Platelet disorders 1, Saturday, June 11, 2016, 5 – 5:15 p.m. (CEST), Room H4
Characterization of Patients with Immune Thrombocytopenia (ITP) Entering Remission in a Romiplostim Bone Marrow Study
Abstract No. P405, Poster presentation, Platelet disorders, Friday, June 10, 2016, 5:15 – 6:45 p.m. (CEST), Poster area (Hall H)
Safety and Efficacy of Long-Term Open-Label Dosing of Subcutaneous (SC) Romiplostim in Children with Immune Thrombocytopenia (ITP)
Abstract No. E1416, Eposter presentation, Platelet disorders
Primary Immune Thrombocytopenia Treated with Romiplostim in Routine Clinical Practice: A Retrospective Study from the United Kingdom Immune Thrombocytopenia Registry
Abstract No. E1426, Eposter presentation, Platelets disorders
Romiplostim in Children with Immune Thrombocytopenia: A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study
Abstract No. P401, Poster presentation, Platelet disorders, Friday, June 10, 2016, 5:15 – 6:45 p.m. (CEST), Poster area (Hall H)
Safety and Efficacy/Effectiveness of Second-Line Treatments in Patients with Immune Thrombocytopenia: A Systematic Review of the Literature
Abstract No. E1417, Eposter presentation, Platelets disorders
Abstracts are available and can be viewed on the EHA (Free EHA Whitepaper) website at View Source!*menu=16*browseby=2*sortby=1*media=3*label=9759.

About BLINCYTO (blinatumomab)

BLINCYTO is a bispecific CD19-directed CD3 T cell engager (BiTE) antibody construct that binds specifically to CD19 expressed on the surface of cells of B-lineage origin and CD3 expressed on the surface of T cells.

BLINCYTO was granted breakthrough therapy and priority review designations by the U.S. Food and Drug Administration, and is now approved in the U.S. for the treatment of Ph- relapsed or refractory B-cell precursor ALL. This indication is approved under accelerated approval. Continued approval for this indication may be contingent upon verification of clinical benefit in subsequent trials.

In November 2015, BLINCYTO was granted conditional marketing authorization in the European Union for the treatment of adults with Ph- relapsed or refractory B-cell precursor ALL.

Important EU BLINCYTO (blinatumomab) Safety Information

This product is subject to additional monitoring in the EU. All suspected adverse reactions should be reported in accordance with the national reporting system.

The adverse reactions described in this section were identified in the pivotal clinical study (N=189).The most serious adverse reactions that may occur during blinatumomab treatment include: infections (31.7%), neurologic events (16.4%), neutropenia/febrile neutropenia (15.3%), cytokine release syndrome (0.5%), and tumor lysis syndrome (0.5%). The most common adverse reactions were: infusion-related reactions (67.2%), infections (63.0%), pyrexia (59.8%), headache (34.4%), febrile neutropenia (28%), peripheral edema (25.9%), nausea (24.3%), hypokalaemia (23.8%), constipation (20.6%), anaemia (20.1%), cough (18.5%), diarrhea (18.0%), tremor (17.5%), neutropenia (17.5%), abdominal pain (16.9%), insomnia (15.3%), fatigue (15.3%), and chills (15.3%).

Please refer to the Summary of Product Characteristics for full European prescribing information.

Important Safety Information Regarding BLINCYTO (blinatumomab) U.S. Indication

This safety information is specific to the current U.S. approved indication.

WARNING: CYTOKINE RELEASE SYNDROME and NEUROLOGICAL TOXICITIES

Cytokine Release Syndrome (CRS), which may be life-threatening or fatal, occurred in patients receiving BLINCYTO. Interrupt or discontinue BLINCYTO as recommended.
Neurological toxicities, which may be severe, life-threatening or fatal, occurred in patients receiving BLINCYTO. Interrupt or discontinue BLINCYTO as recommended.
Contraindications

BLINCYTO is contraindicated in patients with a known hypersensitivity to blinatumomab or to any component of the product formulation.

Warnings and Precautions

Cytokine Release Syndrome (CRS): Life-threatening or fatal CRS occurred in patients receiving BLINCYTO. Infusion reactions have occurred and may be clinically indistinguishable from manifestations of CRS. Closely monitor patients for signs and symptoms of serious events such as pyrexia, headache, nausea, asthenia, hypotension, increased alanine aminotransferase (ALT), increased aspartate aminotransferase (AST), increased total bilirubin (TBILI), disseminated intravascular coagulation (DIC), capillary leak syndrome (CLS), and hemophagocytic lymphohistiocytosis/macrophage activation syndrome (HLH/MAS). Interrupt or discontinue BLINCYTO as outlined in the Prescribing Information (PI).

Neurological Toxicities: Approximately 50% of patients receiving BLINCYTO in clinical trials experienced neurological toxicities. Severe, life-threatening, or fatal neurological toxicities occurred in approximately 15% of patients, including encephalopathy, convulsions, speech disorders, disturbances in consciousness, confusion and disorientation, and coordination and balance disorders. The median time to onset of any neurological toxicity was 7 days. Monitor patients for signs or symptoms and interrupt or discontinue BLINCYTO as outlined in the PI.

Infections: Approximately 25% of patients receiving BLINCYTO experienced serious infections, some of which were life-threatening or fatal. Administer prophylactic antibiotics and employ surveillance testing as appropriate during treatment. Monitor patients for signs or symptoms of infection and treat appropriately, including interruption or discontinuation of BLINCYTO as needed.

Tumor Lysis Syndrome (TLS): Life-threatening or fatal TLS has been observed. Preventive measures, including pretreatment nontoxic cytoreduction and on treatment hydration, should be used during BLINCYTO treatment. Monitor patients for signs and symptoms of TLS and interrupt or discontinue BLINCYTO as needed to manage these events.

Neutropenia and Febrile Neutropenia, including life-threatening cases, have been observed. Monitor appropriate laboratory parameters during BLINCYTO infusion and interrupt BLINCYTO if prolonged neutropenia occurs.

Effects on Ability to Drive and Use Machines: Due to the possibility of neurological events, including seizures, patients receiving BLINCYTO are at risk for loss of consciousness, and should be advised against driving and engaging in hazardous occupations or activities such as operating heavy or potentially dangerous machinery while BLINCYTO is being administered.

Elevated Liver Enzymes: Transient elevations in liver enzymes have been associated with BLINCYTO treatment. The majority of these events were observed in the setting of CRS. The median time to onset was 15 days. Grade 3 or greater elevations in liver enzymes occurred in 6% of patients outside the setting of CRS and resulted in treatment discontinuation in less than 1% of patients. Monitor ALT, AST, gamma-glutamyl transferase (GGT), and TBILI prior to the start of and during BLINCYTO treatment. BLINCYTO treatment should be interrupted if transaminases rise to > 5 times the upper limit of normal (ULN) or if TBILI rises to > 3 times ULN.

Leukoencephalopathy: Although the clinical significance is unknown, cranial magnetic resonance imaging (MRI) changes showing leukoencephalopathy have been observed in patients receiving BLINCYTO especially in patients previously treated with cranial irradiation and anti-leukemic chemotherapy. Preparation and administration errors have occurred with BLINCYTO treatment. Follow instructions for preparation (including admixing) and administration in the PI strictly to minimize medication errors (including underdose and overdose).

Adverse Reactions

The most commonly reported adverse reactions (≥ 20%) in clinical trials were pyrexia (62%), headache (36%), peripheral edema (25%), febrile neutropenia (25%), nausea (25%), hypokalemia (23%), rash (21%), tremor (20%), diarrhea (20%) and constipation (20%).

Serious adverse reactions were reported in 65% of patients. The most common serious adverse reactions (≥ 2%) included febrile neutropenia, pyrexia, pneumonia, sepsis, neutropenia, device-related infection, tremor, encephalopathy, infection, overdose, confusion, Staphylococcal bacteremia, and headache.

U.S. Dosage and Administration Guidelines

BLINCYTO is administered as a continuous intravenous infusion at a constant flow rate using an infusion pump which should be programmable, lockable, non-elastomeric, and have an alarm. It is very important that the instructions for preparation (including admixing) and administration provided in the full Prescribing Information are strictly followed to minimize medication errors (including underdose and overdose).

Please see full U.S. Prescribing Information and medication guide for BLINCYTO at www.BLINCYTO.com.

About Kyprolis (carfilzomib)

Proteasomes play an important role in cell function and growth by breaking down proteins that are damaged or no longer needed.1 Kyprolis has been shown to block proteasomes, leading to an excessive build-up of proteins within cells.2 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.2 The irreversibility of Kyprolis’ binding has also been shown to offer a more sustained inhibition of the targeted enzymes.3

Kyprolis is approved in the United States, Argentina, Israel, Kuwait, Mexico, Thailand, Colombia, Korea, Canada and the European Union. Additional regulatory applications for Kyprolis are underway and have been submitted to health authorities worldwide.

Kyprolis is a product of Onyx Pharmaceuticals, Inc. Onyx Pharmaceuticals is a subsidiary of Amgen and holds development and commercialization rights to Kyprolis globally, excluding Japan. For more information, please visit www.kyprolis.com.

Important EU Kyprolis (carfilzomib) Safety Information

This medicinal product is subject to additional monitoring. This will allow quick identification of new safety information. Healthcare professionals are asked to report any suspected adverse reactions.

Kyprolis treatment should be supervised by a physician experienced in the use of anti-cancer therapy. The most serious side effects that may occur during Kyprolis treatment include: Cardiac toxicity, pulmonary toxicities, pulmonary hypertension, dyspnea, hypertension including hypertensive crises, acute renal failure, tumor lysis syndrome, infusion reactions, thrombocytopenia, hepatic toxicity, posterior reversible encephalopathy syndrome (PRES) and thrombotic thrombocytopenic purpura/hemolytic uremic syndrome (TTP/HUS). The most common side effects are anemia, fatigue, diarrhea, thrombocytopenia, nausea, pyrexia, dyspnea, respiratory tract infection, cough and peripheral edema.

Please refer to the Summary of Product Characteristics for full European prescribing information.

Important U.S. Safety Information Regarding Kyprolis (carfilzomib) for Injection

INDICATION(S)

KYPROLIS (carfilzomib) is indicated 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.
KYPROLIS (carfilzomib) is indicated as a single agent for the treatment of patients with relapsed or refractory multiple myeloma who have received one or more lines of therapy.
IMPORTANT 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 KYPROLIS administration.
Monitor patients for clinical signs or symptoms of cardiac failure or cardiac 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 KYPROLIS 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 in patients with baseline cardiac failure or who are at risk for cardiac failure.
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 (including blood pressure and fluid management) prior to starting treatment with KYPROLIS and remain under close follow-up.
Acute Renal Failure

Cases of acute renal failure and renal insufficiency adverse events (including renal failure) have occurred in patients receiving KYPROLIS. 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 in patients receiving KYPROLIS. Patients with multiple myeloma and 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. Withhold KYPROLIS until TLS is 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 in patients receiving KYPROLIS. Some events have been fatal. In the event of drug‐induced pulmonary toxicity, discontinue KYPROLIS.
Pulmonary Hypertension

Pulmonary arterial hypertension (PAH) was reported in patients treated with KYPROLIS. 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 KYPROLIS 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 KYPROLIS based on a benefit/risk assessment.
Hypertension

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

Venous thromboembolic events (including deep venous thrombosis and pulmonary embolism) have been observed with KYPROLIS. 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 oral contraceptives or a hormonal method of contraception associated with a risk of thrombosis should consider an alternative method of effective contraception during treatment with KYPROLIS in combination with dexamethasone or lenalidomide plus dexamethasone.
Infusion Reactions

Infusion reactions, including life‐threatening reactions, have occurred in patients receiving KYPROLIS. Symptoms include fever, chills, arthralgia, myalgia, facial flushing, facial 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 of KYPROLIS. Premedicate with dexamethasone to reduce the incidence and severity of infusion reactions. Inform patients of the risk and of symptoms of an infusion reaction and to contact a physician immediately if they occur.
Thrombocytopenia

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

Cases of hepatic failure, including fatal cases, have been reported during treatment with KYPROLIS. 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 in patients receiving KYPROLIS. Monitor for signs and symptoms of TTP/HUS. Discontinue KYPROLIS if diagnosis is suspected. If the diagnosis of TTP/HUS is excluded, KYPROLIS may be restarted. The safety of reinitiating KYPROLIS therapy in patients previously experiencing TTP/HUS is not known.
Posterior Reversible Encephalopathy Syndrome (PRES)

Cases of PRES have occurred in patients receiving KYPROLIS. PRES was formerly known as Reversible Posterior Leukoencephalopathy Syndrome. Consider a neuro‐radiological imaging (MRI) for onset of visual or neurological symptoms. Discontinue KYPROLIS if PRES is suspected and evaluate. The safety of reinitiating KYPROLIS therapy in patients previously experiencing PRES is not known.
Embryo-fetal Toxicity

KYPROLIS can cause fetal harm when administered to a pregnant woman based on its mechanism of action and findings in animals.
Females of reproductive potential should be advised to avoid becoming pregnant while being treated with KYPROLIS. Males of reproductive potential should be advised to avoid fathering a child while being treated with KYPROLIS. 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 events occurring in at least 20% of patients treated with KYPROLIS 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 events occurring in at least 20% of patients treated with KYPROLIS in monotherapy trials: anemia, fatigue, thrombocytopenia, nausea, pyrexia, dyspnea, diarrhea, headache, cough, edema peripheral.
Please see full Prescribing Information at www.kyprolis.com.

About Aranesp (darbepoetin alfa) in the U.S.

Aranesp is indicated for the treatment of anemia due to chronic kidney disease (CKD), including patients on dialysis and patients not on dialysis.

Aranesp is indicated for the treatment of anemia in patients with non-myeloid malignancies where anemia is due to the effect of concomitant myelosuppressive chemotherapy, and upon initiation, there is a minimum of two additional months of planned chemotherapy.

Limitations of Use:

Aranesp has not been shown to improve quality of life, fatigue, or patient well-being.

Aranesp is not indicated for use:

In patients with cancer receiving hormonal agents, biologic products, or radiotherapy, unless also receiving concomitant myelosuppressive chemotherapy.
In patients with cancer receiving myelosuppressive chemotherapy when the anticipated outcome is cure.
As a substitute for red blood cell transfusions in patients who require immediate correction of anemia.
Important EU Aranesp Safety Information

Aranesp is indicated for treatment of symptomatic anemia in adult cancer patients with non-myeloid malignancies receiving chemotherapy.

Aranesp is contraindicated in patients with poorly controlled hypertension.

As with all growth factors, there is a concern that epoetins could stimulate the growth of tumours. In several controlled studies, epoetins have not been shown to improve overall survival or decrease the risk of tumour progression in patients with anemia associated with cancer.

In controlled clinical studies, use of Aranesp and other ESAs have shown:

Shortened time to tumour progression in patients with advanced head and neck cancer receiving radiation therapy when administered to target Hb > 14 g/dL; ESAs are not indicated for use in this patient population
Shortened overall survival and increased deaths attributed to disease progression at 4 months in patients with metastatic breast cancer receiving chemotherapy when administered to target Hb 12-14 g/dL(7.5-8.7 mmol/l).
Increased risk of death when administered to target Hb of 12 g/dl (7.5 mmol/l) in patients with active malignant disease receiving neither chemotherapy nor radiation therapy; ESAs are not indicated for use in this patient population.
In view of the above, in some clinical situations blood transfusion should be the preferred treatment for the management of anemia in patients with cancer. The decision to administer recombinant erythropoietins should be based on a benefit-risk assessment with the participation of the individual patient, which should take into account the specific clinical context. Factors that should be considered in this assessment should include the type of tumour and its stage; the degree of anemia; life-expectancy; the environment in which the patient is being treated; and patient preference.

In patients with solid tumours or lymphoproliferative malignancies, if Hb >12 g/dL (7.5 mmol/l), the dose should be reduced according to the instructions provided in the Summary of Product Characteristics to minimise the potential risk of thromboembolic events. Platelet counts and haemoglobin level should also be monitored at regular intervals.

Discontinue use after the end of chemotherapy.

Please refer to the Summary of Product Characteristics for full European prescribing information.

Important U.S. Safety Information for Aranesp(darbepoetin alfa)

WARNING: ESAs INCREASE THE RISK OF DEATH, MYOCARDIAL INFARCTION, STROKE, VENOUS THROMBOEMBOLISM, THROMBOSIS OF VASCULAR ACCESS AND TUMOR PROGRESSION OR RECURRENCE

Chronic Kidney Disease:

In controlled trials, patients experienced greater risks for death, serious adverse cardiovascular reactions, and stroke when administered erythropoiesis-stimulating agents (ESAs) to target a hemoglobin level of greater than 11 g/dL.
No trial has identified a hemoglobin target level, Aranesp dose, or dosing strategy that does not increase these risks.
Use the lowest Aranesp dose sufficient to reduce the need for red blood cell (RBC) transfusions.
Cancer:

ESAs shortened overall survival and/or increased the risk of tumor progression or recurrence in clinical studies of patients with breast, non-small cell lung, head and neck, lymphoid, and cervical cancers.
Because of these risks, prescribers and hospitals must enroll in and comply with the ESA APPRISE Oncology Program to prescribe and/or dispense Aranesp to patients with cancer. To enroll in the ESA APPRISE Oncology Program, visit www.esa-apprise.com or call 1-866-284-8089 for further assistance.
To decrease these risks, as well as the risk of serious cardiovascular and thromboembolic reactions, use the lowest dose needed to avoid RBC transfusions.
Use ESAs only for anemia from myelosuppressive chemotherapy.
ESAs are not indicated for patients receiving myelosuppressive chemotherapy when the anticipated outcome is cure.
Discontinue following the completion of a chemotherapy course.
Aranesp is contraindicated in patients with uncontrolled hypertension, pure red cell aplasia (PRCA) that begins after treatment with Aranesp or other erythropoietin protein drugs, or serious allergic reactions to Aranesp.

Use caution in patients with CKD and coexistent cardiovascular disease and stroke. Patients with CKD and an insufficient hemoglobin response to ESA therapy may be at even greater risk for cardiovascular reactions and mortality than other patients. A rate of hemoglobin rise of > 1 g/dL over 2 weeks may contribute to these risks. In controlled clinical trials of patients with cancer, Aranesp and other ESAs increased the risks for death and serious adverse cardiovascular reactions. These adverse reactions included myocardial infarction and stroke. In controlled clinical trials, ESAs increased the risk of death in patients undergoing coronary artery bypass graft surgery (CABG) and the risk of deep venous thrombosis (DVT) in patients undergoing orthopedic procedures. Control hypertension prior to initiating and during treatment with Aranesp.

Aranesp increases the risk of seizures in patients with CKD. Monitor patients closely for new-onset seizures, premonitory symptoms, or change in seizure frequency.

For lack or loss of hemoglobin response to Aranesp, initiate a search for causative factors. If typical causes of lack or loss of hemoglobin response are excluded, evaluate for PRCA. Cases of PRCA and of severe anemia, with or without other cytopenias that arise following the development of neutralizing antibodies to erythropoietin have been reported in patients treated with Aranesp. This has been reported predominantly in patients with CKD receiving ESAs by subcutaneous administration. PRCA has also been reported in patients receiving ESAs for anemia related to hepatitis C treatment (an indication for which Aranesp is not approved). If severe anemia and low reticulocyte count develop during treatment with Aranesp, withhold Aranesp and evaluate patients for neutralizing antibodies to erythropoietin. Permanently discontinue Aranesp in patients who develop PRCA following treatment with Aranesp or other erythropoietin protein drugs. Do not switch patients to other ESAs.

Serious allergic reactions, including anaphylactic reactions, angioedema, bronchospasm, skin rash, and urticaria may occur with Aranesp. Immediately and permanently discontinue Aranesp if a serious allergic reaction occurs.

Adverse reactions (≥ 10%) in Aranesp clinical studies in patients with CKD were hypertension, dyspnea, peripheral edema, cough, and procedural hypotension. Adverse reactions (≥ 1%) in Aranesp clinical studies in cancer patients receiving chemotherapy were abdominal pain, edema, and thrombovascular events.

To see the Aranesp Prescribing Information, including Boxed Warnings, and Medication Guide visit www.aranesp.com.

About Nplate (romiplostim)

Nplate is approved in over 50 countries worldwide, including the U.S., European Union (EU), Canada, Australia, Russia, Mexico, Switzerland, Lichtenstein, Japan, Argentina, Israel, South Korea, Hong Kong, and Chile. Nplate also has received orphan designation for chronic ITP in the U.S. (2003), the EU (2005) and other parts of the world.

Nplate is the first FDA-approved treatment specifically for adult chronic ITP

In the U.S., Nplate is indicated for the treatment of thrombocytopenia in patients with chronic ITP who have had an insufficient response to corticosteroids, immunoglobulins or splenectomy. Nplate is not indicated for the treatment of thrombocytopenia due to myelodysplastic syndrome (MDS) or any cause of thrombocytopenia other than chronic ITP. Nplate should be used only in patients with ITP whose degree of thrombocytopenia and clinical condition increase the risk for bleeding. Nplate should not be used in an attempt to normalize platelet counts.

In the EU, Nplate is indicated for adult chronic-immune (idiopathic)-thrombocytopenic-purpura (ITP) patients who are refractory to other treatments (e.g. corticosteroids, immunoglobulins).

Nplate was named as a recipient of the U.S. Prix Galien 2009 "Best Biotechnology Product" award and also received the 2009 Scrip Awards for "Best New Drug." Nplate has also been honored with numerous awards throughout the EU, including a 2010 Prix Galien in France in the category of "Drugs for Rare Diseases," and the 2011 Prix Galien in Germany in the category of "Specialist Care." In September 2010, Nplate was awarded the 2010 International Prix Galien Award, an award granted every two years which recognizes the "best of the best" selected from previous national Prix Galien award recipients.

For more information about Nplate, please visit www.Nplate.com.

Important EU Nplate Safety Information

The EU Summary of Product Characteristics for Nplate lists the following Special Warnings and Precautions: Reoccurrence of thrombocytopenia and bleeding after cessation of treatment, increased bone marrow reticulin, thrombotic/thromboembolic complications, progression of existing MDS (in patients with MDS), medication errors, loss of response to Nplate, and effects on red and white blood cells.

The most common adverse reactions observed include hypersensitivity reactions (including cases of rash, urticarial and angioedema) and headache. As with all therapeutic proteins, there is a potential for immunogenicity.

Please refer to the Summary of Product Characteristics for full European prescribing information.

Important U.S. Nplate Safety Information

Risk of Progression of Myelodysplastic Syndromes to Acute Myelogenous Leukemia

In Nplate clinical trials of patients with myelodysplastic syndromes (MDS) and severe thrombocytopenia, progression from MDS to acute myelogenous leukemia (AML) has been observed.
Nplate is not indicated for the treatment of thrombocytopenia due to MDS or any cause of thrombocytopenia other than chronic ITP.
Thrombotic/Thromboembolic Complications

Thrombotic/thromboembolic complications may result from increases in platelet counts with Nplate use. Portal vein thrombosis has been reported in patients with chronic liver disease receiving Nplate.
To minimize the risk for thrombotic/thromboembolic complications, do not use Nplate in an attempt to normalize platelet counts. Follow the dose adjustment guidelines to achieve and maintain a platelet count of ≥ 50 x 109/L.
Loss of Response to Nplate

Hyporesponsiveness or failure to maintain a platelet response with Nplate should prompt a search for causative factors, including neutralizing antibodies to Nplate.
To detect antibody formation, submit blood samples to Amgen (1-800-772-6436). Amgen will assay these samples for antibodies to Nplate and thrombopoietin (TPO).
Discontinue Nplate if the platelet count does not increase to a level sufficient to avoid clinically important bleeding after 4 weeks at the highest weekly dose of 10 mcg/kg.
Laboratory Monitoring

Obtain CBCs, including platelet counts, weekly during the dose adjustment phase of
Nplate therapy and then monthly following establishment of a stable Nplate dose.
Obtain CBCs, including platelet counts, weekly for at least two weeks following discontinuation of Nplate.
Adverse Reactions

In the placebo-controlled trials, headache was the most commonly reported adverse drug reaction, occurring in 35% of patients receiving Nplate and 32% of patients receiving placebo. Headaches were usually of mild or moderate severity.
Most common adverse reactions (≥ 5% higher patient incidence in Nplate versus placebo) were Arthralgia (26%, 20%), Dizziness (17%, 0%), Insomnia (16%, 7%), Myalgia (14%, 2%), Pain in Extremity (13%, 5%) , Abdominal Pain (11%, 0%), Shoulder Pain (8%, 0%), Dyspepsia (7%, 0%), and Paresthesia (6%, 0%).
Nplate administration may increase the risk for development or progression of reticulin fiber formation within the bone marrow. This formation may improve upon discontinuation of Nplate. In a clinical trial, one patient with ITP and hemolytic anemia developed marrow fibrosis with collagen during Nplate therapy.
Please see full U.S. Prescribing Information and Medication Guide at www.Nplate.com

Syros Pharmaceuticals to Present on its Two Lead Programs, SY-1425 and SY-1365, at 21st Congress of the European Hematology Association

On May 19, 2016 Syros Pharmaceuticals reported that preclinical data on its lead program, SY-1425, in genomically defined subsets of patients with acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS) will be highlighted in an oral presentation at the 21st Congress of the European Hematology Association (EHA) (Free EHA Whitepaper) taking place June 9-12 in Copenhagen, Denmark (Press release, Syros Pharmaceuticals, MAY 19, 2016, View Source [SID:1234512614]). The Company will also present new preclinical data on its first-in-class selective cyclin-dependent kinase 7 (CDK7) inhibitor, SY-1365, in acute leukemia.

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SY-1425 in Novel Genomically Defined Subset of AML and MDS Patients Using its gene control platform, Syros identified a subset of AML and MDS patients whose tumors have a highly specialized regulatory region of non-coding DNA, known as a superenhancer, associated with the RARA gene. The super-enhancer associated with RARA is believed to lead to over-production of the RARα transcription factor, locking cells in an immature, undifferentiated and proliferative state. Treatment with SY-1425, an oral, potent and selective agonist of RARα, appears to promote differentiation of cancer cells with the RARA-associated super-enhancer, inhibiting the cancer’s growth. The oral presentation at EHA (Free EHA Whitepaper) will detail SY-1425’s mechanism of action as well as in vitro and in vivo data showing that a biomarker for the RARA super-enhancer discovered by Syros is predictive of response to treatment with SY-1425 in models of AML, including a survival benefit observed in the mice with the RARA biomarker when treated with SY-1425. Syros is on track to advance SY-1425 into a Phase 2 trial in mid-2016 in subsets of AML and MDS patients whose tumors are positive for the RARA biomarker.

Date & Time: Sunday, June 12, from 9-9:15 a.m. CEST Presentation Title: Super-Enhancer Analysis Defines Novel AML and MDS Sub-Types Sensitive to SY-1425, a Potent and Selective RARα Agonist Session Title: AML Biology – Novel Targeted Therapies Presenter: Michael R. McKeown, Ph.D., Senior Scientist, Syros Pharmaceuticals Abstract Number: S807 Location: Bella Center, Auditorium 2

CDK7 Inhibition as a Novel Treatment Strategy for Acute Leukemia
Certain cancers, including AML and acute lymphoblastic leukemia (ALL), are dependent on high and constant expression of transcription factors for their growth and survival and have been shown to be particularly responsive to selective inhibition of the transcriptional kinase CDK7. The poster presentation at EHA (Free EHA Whitepaper) details preclinical data demonstrating that SY-1365, the Company’s first-in-class selective and potent CDK7 inhibitor, preferentially kills cancer cells by inducing robust and dose-dependent apoptosis in acute leukemia cell lines while not inducing apoptosis in non-cancerous cells. The data also show that SY-1365 produces a significant survival benefit in patient-derived xenograft models of AML. Syros expects to advance SY-1365 into a Phase 1/2 trial in the first half of 2017 in patients with acute leukemia, including AML and ALL.

Date & Time: Saturday, June 11, from 5:30-7 p.m. CEST Presentation Title: First-in-Class CDK7 Inhibitor Induces Robust Apoptosis in Acute Myeloid Leukemia and Demonstrates Durable In Vivo Efficacy Session Title: Acute Myeloid Leukemia – Biology 3 Presenter: Yoon J. Choi, Ph.D., Senior Scientist, Syros Pharmaceuticals Abstract Number: P558 Location: Bella Center, Hall H, Poster Area

OncoCyte to Release Bladder Cancer Data at the 2016 American Society of Clinical Oncology Annual Meeting

On May 19, 2016 OncoCyte Corporation (NYSE MKT: OCX), a developer of novel, non-invasive tests for the early detection of cancer, reported the abstract of data from a bladder cancer study that will be presented as a poster and also highlighted during a live panel discussion on June 6, 2016, during the 2016 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting to be held in Chicago, Illinois (Press release, BioTime, MAY 19, 2016, View Source [SID:1234512597]).

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"Our bladder test results are highly encouraging and reflect the depth of quality diagnostics products in our robust pipeline," commented OncoCyte Chief Executive Officer William Annett. "There is a high unmet need for non-invasive tests for bladder cancer and our gene expression classifiers have been shown to provide superior sensitivity in a procedure that is less invasive to patients than the current standard of care. Our results indicate the feasibility of using a urine-based diagnostic test to detect urothelial carcinoma both in screening (hematuria) and recurrence settings."

The abstract entitled Derivation of Gene Expression Classifiers for the Non-invasive Detection of Bladder Cancer in the Hematuria and Recurrence Surveillance Populations, describes OncoCyte’s recent results in the development of a urine-based test for bladder cancer. The detection of bladder cancer is typically accomplished with a combination of cystoscopy and urine cytology, each with inherent limitations. Urine cytology lacks the desired level of sensitivity, whereas cystoscopy is relatively invasive for routine screening and recurrence surveillance. OncoCyte’s study describes the development of four gene expression classifiers (GECs) optimized for the non-invasive detection of both high-grade and low-grade urothelial carcinoma in patients presenting with hematuria or for bladder cancer recurrence surveillance. The results for high-grade screening, high-grade recurrence, low-grade screening and low-grade recurrence were all obtained from single urine samples utilizing four different algorithms. A multi-center study involving 241 patient urine samples was used in the development of this assay.

OncoCyte’s approach of sequential GECs optimized for the detection of high-grade and low-grade malignancies provides the necessary data to distinguish between these different types of lesions and benign conditions in a non-invasive manner. Low-grade urothelial carcinoma is usually a non-aggressive cancer, whereas high-grade urothelial carcinoma is more aggressive, invasive and causes significantly more cancer-related mortality than low-grade urothelial carcinoma.

The GEC optimized for the detection of high-grade urothelial carcinoma in patients presenting with hematuria performed, with a cross-validated ROC AUC of 0.93, while the low-grade performed with an AUC of 0.81. In the recurrence surveillance cohort, the detection of high-grade performed with an AUC of 0.81 and low-grade with an AUC of 0.64. The following table summarizes these results.

ROC AUCs

Low Grade High Grade
Screening (Hematuria) 0.81 0.93

Recurrence 0.64 0.81
"These data establishes the feasibility of our approach, and we look forward to continuing our larger, multicenter study to further validate these findings," added Karen B. Chapman, Ph.D., OncoCyte’s Vice President of Research, who led the study.

About Bladder Cancer

Bladder cancer has been projected to have the highest lifetime treatment costs per patient of all cancers. The high recurrence rate and ongoing invasive monitoring requirements drive the financial burden of this disease. The detection of bladder cancer in Hematuria and Recurrence patients is routinely accomplished with a combination of urine cytology and cystoscopy which is invasive, and lacks the desired level of sensitivity.

AstraZeneca highlights continued progress of oncology pipeline at ASCO 2016

On May 19, 2016 AstraZeneca and its global biologics research and development arm, MedImmune, reported that they will provide an update on their extensive oncology pipeline at the annual meeting of the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) in Chicago, USA, on 3-7 June 2016 (Press release, AstraZeneca, MAY 19, 2016, View Source [SID:1234512562]).

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Highlights will include new data demonstrating the strength and versatility of AstraZeneca’s industry-leading line of DNA damage response (DDR) medicines in multiple types of cancer. New data will highlight the continued momentum behind AstraZeneca’s numerous immuno-oncology (IO) programmes, and showcase small-molecule developments including Tagrisso (osimertinib) in leptomeningeal (brain) disease and the highly-selective Bruton’s tyrosine kinase (BTK) inhibitor, acalabrutinib, in chronic lymphocytic leukaemia (CLL).

Sean Bohen, Executive Vice President, Global Medicines Development and Chief Medical Officer at AstraZeneca, said: "Oncology is a strategic priority for AstraZeneca because of the potential of our broad pipeline to offer transformational therapies in cancer care. At ASCO (Free ASCO Whitepaper), we will update on our next-generation portfolio focusing on DNA damage response as a breakthrough paradigm in cancer treatment, including new long-term overall survival data for Lynparza. Our increased commitment to DDR therapies complements developments in our exciting immuno-oncology pipeline, from which we are expecting clinical results over the coming year."

DDR: A promising scientific platform, a leading position for AstraZeneca

DDR is a term describing the network of cellular pathways that minimise the daily impact of DNA damage. Currently, many cancers are known to have defects in DDR pathways, which makes them dependent on and therefore, highly sensitive to inhibition of the remaining DDR pathways. Targeting DDR deficiencies to preferentially kill cancer cells, while minimising the impact on normal cells, has potential for more selective, better tolerated therapies to improve survival in multiple cancers.

AstraZeneca is developing a comprehensive pipeline of compounds that target molecular pathways across the DDR system. These include the PARP inhibitor Lynparza (olaparib); Wee1 inhibitor AZD1775, ATM inhibitor AZD0156; ATR inhibitor AZD6738, and Aurora B Kinase inhibitor AZD2811. These compounds act on different cell-cycle points to prevent tumour cells from reproducing.

At the ASCO (Free ASCO Whitepaper) congress, DDR presentations will highlight:

The potential for maintenance of DDR therapies as shown by Lynparza overall survival data from Study 19 in ovarian cancer (Abstract # 5501). This abstract has been selected as a "Best of ASCO (Free ASCO Whitepaper)" abstract.
Opportunities for combination approaches with DDR and immuno-oncology therapies as shown in a Phase I study of the PD-L1 inhibitor, durvalumab, in combination with Lynparza or a VEGFR inhibitor, cediranib, in women’s cancers (Abstract # 3015)
The importance of selecting patients with a DDR pathway defect using the right diagnostic tool (abstract #4041)
The potential of DDR therapies against multiple biological DDR targets in different tumour types, with studies of the highly-selective WEE1 inhibitor, AZD1775, in advanced high-grade serous ovarian cancer (Abstract # TPS5610), squamous cell carcinoma of the head and neck (SCCHN) (Abstract # TPS6106), advanced solid tumours (Abstract # TPS2608) and glioblastoma (GBM) (Abstract # 2008)
Immuno-Oncology: Robust development momentum on track for read-outs in H1 2017

AstraZeneca is leading in a number of first-line studies with its IO strategy, where combined PD-L1 and CTLA-4 blockade – through the combination of durvalumab and tremelimumab – may address a significant unmet medical need for cancer patients who may not benefit from PD-1 pathway drugs in monotherapy.

Key updates include presentations covering pre-clinical data, late-stage trials and biomarker research:

Early study results of durvalumab monotherapy in urothelial bladder cancer from Phase Ib Study 1108 (Abstract # 4502)
Final results from a Phase III study of tremelimumab in mesothelioma (Abstract # 8502)
New study results on safety and clinical activity of durvalumab as first-line treatment in non-small cell lung cancer (NSCLC) (Abstract # 9029)
Ongoing investigation of the potential synergistic effects of durvalumab and the CTLA-4 inhibitor, tremelimumab, in bladder cancer (DANUBE trial – Abstract # TPS4574) and SCCHN (KESTREL trial – Abstract # TPS6101)
Enhanced understanding of PD-L1 biomarker expression in relation to primary versus metastatic tumours and sample age (Abstract # 3025)
Tagrisso in brain metastasis; acalabrutinib in CLL

AstraZeneca’s strong heritage in developing innovative targeted small molecules was underscored by the recent approval of Tagrisso as the first indicated treatment for EGFR T790M mutation-positive metastatic NSCLC in the US, EU and Japan. At ASCO (Free ASCO Whitepaper), new data will highlight the importance of Tagrisso activity in leptomeningeal disease through its ability to penetrate the blood-brain barrier. Further presentations will show the growing role of circulating tumour DNA (ctDNA) testing for diagnosis and treatment monitoring.

Key updates will also include presentation on the potential of our potent, highly-selective BTK inhibitor, acalabrutinib, in chronic lymphocytic leukaemia (CLL):

Data from the BLOOM study of Tagrisso in patients with leptomeningeal disease as a complication of EGFRm-metastatic NSCLC (Abstract # 9002)
Intensive plasma ctDNA profiling in experimental trials to identify markers of acquired drug resistance (Abstract # 11530)
Acalabrutinib – preliminary results from a first-line study as first-line therapy in CLL (Abstract # 7521) and in a Phase II study in combination with pembrolizumab in metastatic pancreatic cancer (Abstract # 4130)