8-K – Current report

On September 23, 2015 OncoGenex Pharmaceuticals, Inc. (NASDAQ: OGXI) reported initial results from the Phase 2 Rainier study evaluating apatorsen in combination with ABRAXANE (paclitaxel protein-bound particles for injectable suspension) (albumin-bound) and gemcitabine compared to ABRAXANE and gemcitabine alone in patients with untreated metastatic pancreatic cancer (Filing, 8-K, OncoGenex Pharmaceuticals, SEP 23, 2015, View Source [SID:1234507534]). The addition of apatorsen to ABRAXANE and gemcitabine did not demonstrate a survival benefit compared to ABRAXANE and gemcitabine alone. The study was sponsored and conducted by Sarah Cannon Research Institute (SCRI) and further results will be presented by SCRI at a future medical meeting.

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Pancreatic cancer accounts for approximately 338,000 new cases each year worldwide. In the U.S., it continues to be the fourth leading cause of cancer death. Most pancreatic cancer patients will die within the first year of diagnosis, and five-year survival rates are less than 10 percent.

"Over the last decade, very few treatments have been able to demonstrate a survival benefit in this very difficult-to-treat cancer," said Johanna Bendell, MD, Director of the GI Cancer Research Program, SCRI, and a primary investigator on the trial. "We understand the dire need for new treatment options and are thankful to the patients who participated in this trial."
The most common grade 3/4 treatment-related toxicities on the apatorsen arm were anemia, neutropenia and fatigue, also consistent with the chemotherapy regimen side effects. These and other adverse events (AEs) observed on the apatorsen arm were similar to those seen in previous trials, with the exception of an increase in grade 4 or greater AEs and serious AEs in this pancreatic cancer trial. While patients in the apatorsen arm had fewer treatment discontinuations due to progressive disease, more patients discontinued therapy due to AEs.

"While we are disappointed with the Rainier results, we also recognize the challenges associated with developing effective treatments for such a lethal and complex disease. We remain confident in our broader apatorsen program, which includes ongoing Phase 2 clinical trials in lung, prostate and bladder cancers," said Scott Cormack, President and CEO of OncoGenex.

Recent Apatorsen Data
Results from an exploratory analysis of the Phase 2 Borealis-1 trial showed that patients with metastatic bladder cancer with poor prognostic features experienced a reduction in risk of death with the addition of 600 mg apatorsen added to first-line chemotherapy, compared to chemotherapy alone.

About Apatorsen and ORCA
Apatorsen (OGX-427) is a once-weekly intravenous (IV) experimental drug that is designed to inhibit production of heat shock protein 27 (Hsp27) to disable cancer cells’ defenses and overcome treatment resistance. Hsp27 in an intracellular protein that protects cancer cells by helping them survive, leading to resistance and more aggressive cancer phenotypes. Both the potential single-agent activity and synergistic activity of apatorsen with cancer treatments may increase the overall benefit of existing therapies and augment the durability of treatment outcomes, which could lead to increased patient survival. The ORCA (Ongoing Studies Evaluating Treatment Resistance in CAncer) program encompasses clinical trials of apatorsen. For more information on apatorsen and ORCA, please visit www. OncoGenex.com or www.orcatrials.com.

Takeda to Highlight Ixazomib Clinical Development Programat Upcoming International Myeloma Workshop

On September 24, 2015 Takeda Pharmaceutical Company Limited (TSE: 4502) reported that it will highlight the ixazomib clinical development program during multiple poster sessions at the upcoming 15th International Myeloma Workshop (IMW 2015) to be held in Rome, Italy, from September 23 to 26, 2015 (Press release, Takeda, SEP 23, 2015, View Source [SID:1234507529]).

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"IMW presents an important opportunity for the multiple myeloma community to come together," said Dixie-Lee Esseltine, MD, FRCPC, Vice President, Oncology Therapeutic Area Unit, Takeda. "Ixazomib’s TOURMALINE program, which we anticipate will have over 3,000 patients when fully enrolled, is the embodiment of Takeda’s commitment to addressing the unmet needs in multiple myeloma, and now contains a pivotal trial in every major segment of this patient population. We look forward to presenting an overview of this program at IMW and engaging with our partners in the fight against multiple myeloma."

Ixazomib is currently under review with the European Medicines Agency (EMA) and the U.S. Food and Drug Administration (FDA) for the treatment of patients with relapsed and/or refractory multiple myeloma, and has been granted accelerated assessment and priority review, respectively. The regulatory submissions are based on the results of the first pre-specified interim analysis of the pivotal Phase 3 trial TOURMALINE-MM1, an international, randomized, double-blind, placebo controlled clinical trial of 722 patients designed to evaluate the superiority of once-a-week oral ixazomib plus lenalidomide and dexamethasone over placebo plus lenalidomide and dexamethasone in adult patients with relapsed and/or refractory multiple myeloma. Additional filings in other countries are planned to begin this year. Ixazomib is the first oral proteasome inhibitor in late stage clinical development for the treatment of patients with multiple myeloma.

Ixazomib presentations at IMW 2015 are as follows:

Format/Timing: Poster Discussion; Thursday, September 24, 2015 (6:40-7:40 p.m. CEST)
Abstract No: 0110
Abstract Title: Four Phase 3 Studies of the Oral Proteasome Inhibitor (PI) Ixazomib for Multiple Myeloma in the Newly-Diagnosed, Relapsed/Refractory, and Maintenance
Settings: Tourmaline-MM1, -MM2, -MM3, and -MM4
Authors: Jesus San Miguel, Philippe Moreau, Vincent Rajkumar, Antonio Palumbo, Thierry Facon, Gareth Morgan, Robert Orlowski, Michele Cavo, Hermann Einsele, Frank
Neumann, Richard Labotka, Sagar Lonial, Paul Richardson

Location: Poster Area, 1st Floor

Format/Timing: Poster Display; Friday, September 25, 2015 (6:40-7:40 p.m. CEST)
Abstract No: 0112
Abstract Title: The Current Unmet Medical Needs in the Treatment and Management of Multiple Myeloma (MM)
Authors: Paul Richardson, Antonio Palumbo, Maria-Victoria Mateos, Helgi van de Velde, Tomas Skacel, Sagar Lonial
Location: Poster Area, 1st Floor

Format/Timing: Poster Discussion; Thursday, September 24, 2015 (6:40-7:40 p.m. CEST)
Abstract No: 0167
Abstract Title: Phase 3 Study of the Oral Proteasome Inhibitor Ixazomib for Relapsed/Refractory AL Amyloidosis: TOURMALINE-AL1
Authors: Giampaolo Merlini, Angela Dispenzieri, Deborah Berg, Douglas V. Faller, Ai-Min Hui, Raymond L. Comenzo
Location: Poster Area, 1st Floor

Format/Timing: Poster Discussion; Friday, September 25, 2015 (6:40 – 7:40 p.m. CEST)
Abstract No: 0177
Abstract Title: Safety Profile of Oral Ixazomib: Experience from 761 Patients Across 14 Phase 1 or Phase 1/2 Clinical Studies
Authors: Sagar Lonial, Kathleen Colson, R. Donald Harvey, Shaji Kumar, Ai-Min Hui, Guohui Liu, Deborah Berg, Paul Richardson
Location: Poster Area, 1st Floor

About Ixazomib and the TOURMALINE Trials
Ixazomib is an investigational oral proteasome inhibitor which is being studied in multiple myeloma, systemic light-chain (AL) amyloidosis, and other malignancies. Ixazomib was granted orphan drug designation in multiple myeloma in both the United States and Europe in 2011 and for AL amyloidosis in both the U.S. and Europe in 2012. Ixazomib received Breakthrough Therapy status by the U.S. FDA for relapsed or refractory AL amyloidosis in 2014. It is also the first oral proteasome inhibitor to enter Phase 3 clinical trials.

Ixazomib’s clinical development program further reinforces Takeda’s ongoing commitment to developing innovative therapies for people living with multiple myeloma worldwide and the healthcare professionals who treat them. Five global Phase 3 trials are ongoing:

TOURMALINE-MM1, investigating ixazomib vs. placebo, in combination with lenalidomide and dexamethasone in relapsed and/or refractory multiple myeloma
TOURMALINE-MM2, investigating ixazomib vs. placebo, in combination with lenalidomide and dexamethasone in patients with newly diagnosed multiple myeloma
TOURMALINE-MM3, investigating ixazomib vs. placebo as maintenance therapy in patients with newly diagnosed multiple myeloma following induction therapy and autologous stem cell transplant (ASCT)
TOURMALINE-MM4, investigating ixazomib vs. placebo as maintenance therapy in patients with newly diagnosed multiple myeloma who have not undergone ASCT
TOURMALINE-AL1, investigating ixazomib plus dexamethasone vs. physician choice of selected regimens in patients with relapsed or refractory AL amyloidosis

For additional information on the ongoing Phase 3 studies please visit www.clinicaltrials.gov.

About Multiple Myeloma
Multiple myeloma is a cancer of the plasma cells, which are found in the bone marrow. In multiple myeloma, a group of plasma cells, or myeloma cells, becomes cancerous and multiplies, increasing the number of plasma cells to a higher than normal level. Because plasma cells circulate widely in the body, they have the potential to affect many bones in the body, possibly resulting in compression fractures, lytic bone lesions and related pain. Multiple myeloma can cause a number of serious health problems affecting the bones, immune system, kidneys and red blood cell count, with some of the more common symptoms including bone pain and fatigue, a symptom of anemia. Multiple myeloma is a rare form of cancer with approximately 39,000 new cases in the EU and 114,000 new cases globally per year.

8-K – Current report

On September 23, 2015 Provectus Biopharmaceuticals, Inc. (NYSE MKT: PVCT, http://www.pvct.com), a development-stage oncology and dermatology biopharmaceutical company ("Provectus" or the "Company"), reported it has completed development of the protocol for Phase1b/2 testing of its investigational cancer drug PV-10 in combination with pembrolizumab in patients with Stage IV melanoma (Filing, 8-K, Provectus Pharmaceuticals, SEP 23, 2015, View Source [SID:1234507527]). Pembrolizumab (also known as Keytruda, a product of Merck and Co. Inc., NYSE: MRK) is an immune checkpoint inhibitor approved for treatment of patients with advanced or unresectable melanoma. PV-10 is Provectus’s novel investigational drug for cancer that is injected into solid tumors (intralesional administration); it is currently undergoing Phase 3 clinical testing in patients with Stage III melanoma. Clinical testing under the new Phase 1b/2 protocol is expected to commence before the end of the year.

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The combination protocol enables initial clinical testing of concepts at the center of a patent held by Provectus, U.S. Patent number 9,107,887, which Pfizer, Inc. (NYSE: PFE) jointly owns. Specifically, the patent covers the use of PV-10 in combination with systemic inhibitors of immune system down-regulation, such as anti-CTLA-4, PD-1 and PD-L1 immune checkpoint inhibiting antibodies. Pembrolizumab is an anti-PD-1 antibody. Pre-clinical testing of PV-10 used in combination with these important classes of drugs demonstrated potential importance for treatment of advanced cancers.

The FDA granted accelerated approval to pembrolizumab in September 2014, making it the first FDA-approved anti-PD-1 immune checkpoint inhibitor. Because pembrolizumab is already FDA-approved, Provectus can commence this study with or without assistance of a partner.

The Phase 1b/2 study will incorporate a modest sized single arm Phase 1b component of 24 subjects with expedited safety and efficacy end points. This is designed to support expansion to a larger randomized Phase 2 component. Combined, these two arms will enable assessment of the potential safety and clinical benefit of PV-10 when used with pembrolizumab for treatment of advanced melanoma.

Dr. Eric Wachter, CTO of Provectus, stated, "The primary end point of tolerability in the Phase 1b portion of the study, combined with assessment of progression free survival (PFS) and objective response rate (ORR) by RECIST criteria as key secondary endpoints, assessed over a 15 week treatment interval, establish a basis for determining whether to proceed to the larger, randomized Phase 2 portion of the study. We will use an adaptive design for powering Phase 2 based on preliminary results from Phase 1, and estimate this portion of the study to require at least 120 subjects, with a primary endpoint of PFS and key secondary endpoint of ORR. In both portions of the study, pembrolizumab will be administered every three weeks for up to 24 months, as is standard of care; PV-10 will be administered on the same schedule for the first 15 weeks to all of the subject’s skin lesions. Subjects in Phase 1b will receive both PV-10 and pembrolizumab, whereas in Phase 2 subjects will be randomized to PV-10 + pembrolizumab or pembrolizumab alone."

Pete Culpepper, CFO and COO of Provectus, noted, "This study is both scientifically and commercially important to Provectus. Scientifically, combination therapy in cancer treatment is a rapidly maturing area, where rational combination of agents is replacing the empirical approaches of the past. Commercially, this is the second of three steps that we hope will significantly strengthen our hand in negotiating a co-development transaction with an immunotherapy-focused partner. Our joint patent with Pfizer was the first; this study is the second; and the third is our immune mechanism of action clinical study, which is underway at the Moffitt Cancer Center and which has completed recruitment."

The mechanism of action study’s preliminary clinical findings, reported last year, showed that the immunologic effects of tumor ablation with PV-10 may be complementary to immune checkpoint inhibition. Companion pre-clinical testing of PV-10 in murine models of melanoma, also reported last year, showed that the therapeutic effects of PV-10 and immune checkpoint inhibition are increased when the two are used in combination.

The cost of pembrolizumab is reimbursed so it is not paid for by Provectus and the remaining cost of this study is budgeted with existing cash on hand of the Company.

For further details on the protocol visit View Source

Clovis Oncology Announces Data Presentations at 2015 European Cancer Congress

On September 23, 2015 Clovis Oncology (NASDAQ:CLVS) reported that three oral presentations and four scientific posters highlighting updated results from clinical studies of the company’s two compounds in advanced clinical development are being presented at the 2015 European Cancer Congress (ECC), which will take place Sept. 25-29 in Vienna, Austria (Press release, Clovis Oncology, SEP 23, 2015, View Source;p=RssLanding&cat=news&id=2089808 [SID:1234507525]).

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"We are pleased to have the opportunity to share significant clinical progress across our product pipeline at ECC this year," said Patrick J. Mahaffy, CEO and President of Clovis Oncology. "Some key highlights include updated results from our rucaparib studies in the treatment of advanced ovarian cancer, including ARIEL2, for which we intend to file our initial regulatory submissions in the US in mid-2016."

Rucaparib, the Company’s oral, potent, small molecule inhibitor of PARP1 and PARP2 being developed for the treatment of ovarian cancer, specifically in patients with tumors with BRCA mutations and other DNA repair deficiencies beyond BRCA such as high genomic LOH (also referred to as "BRCA-like"), is the subject of two oral presentations and two posters:

Abstract #2700 – Final results of ARIEL2 (Part 1): a Phase 2 trial to prospectively identify ovarian cancer (OC) responders to rucaparib using tumor genetic analysis

R Kristeleit
Tuesday, Sept. 29, 9-9:15am CEST
Room: Hall A2

Abstract #2701 – Quantification of genomic loss of heterzygosity enables prospective selection of ovarian cancer patients who may derive benefit from the PARP inhibitor rucaparib

A Oza
Tuesday, Sept. 29, 9:15-9:30am CEST
Room: Hall A2

Poster # P409 – A phase 2 open-label, multicenter study of single-agent rucaparib in the treatment of patients with relapsed ovarian cancer and a deleterious BRCA mutation

R Shapira-Frommer
Monday, Sept. 28, 2015, 9:15-11:15am CEST
Room: Hall C

Rociletinib, the Company’s oral, potent, mutant-selective inhibitor of epidermal growth factor receptor (EGFR) under investigation for the treatment of EGFR-mutated non-small cell lung cancer (NSCLC), is the subject of one oral and three posters presentations:

Abstract #3009 – Activity of rociletinib in EGFR mutant NSCLC patients with a history of CNS involvement

A Varga
Monday, Sept. 28, 10:10-10:25am CEST
Room: Strauss

Poster # P356 – Rociletinib treatment and outcomes in non-small cell lung cancer (NSCLC) patients with negative central testing for T790M

B Soloman
Sunday, Sept. 27, 9:15-11:15am CEST
Room: Hall C

Poster # P356 – Efficacy of rociletinib (CO-1686) in EGFR-mutant non-small cell lung cancer (NSCLC) patients assessed with a plasma EGFR test

S Gadgeel
Sunday, Sept. 27, 9:15-11:15am CEST
Room: Hall C

Poster # P357 – Dose optimization of rociletinib for EGFR mutated NSCLC: Benefit/risk analysis from the TIGER-X trial

JC Soria
Sunday, Sept. 27, 9:15-11:15am CEST
Room: Hall C

About Rucaparib

Rucaparib is an oral, potent small molecule inhibitor of PARP1 and PARP2 being developed for the treatment of ovarian cancer, specifically in patients with tumors with BRCA mutations and other DNA repair deficiencies beyond BRCA, such as high genomic LOH, which is commonly referred to as "BRCA-like." Rucaparib was granted Breakthrough Therapy designation by the U.S. FDA in April 2015.

About Rociletinib

Rociletinib is an oral, potent, mutant-selective inhibitor of epidermal growth factor receptor (EGFR) under investigation for the treatment of EGFR-mutated non-small cell lung cancer (NSCLC). Rociletinib targets the activating mutations of EGFR (L858R and Del19), while also inhibiting the dominant acquired resistance mutation, T790M, which develops in approximately 60 percent of patients treated with first- and second-generation EGFR inhibitors, while sparing wild-type, or "normal" EGFR at anticipated therapeutic doses. Rociletinib was granted Breakthrough Therapy designation by the U.S. FDA in May 2014.

Amgen To Present New Kyprolis® (carfilzomib) Data At 15th International Myeloma Workshop

On September 23, 2015 Amgen (NASDAQ:AMGN) reported the presentation of several studies evaluating Kyprolis (carfilzomib) for Injection, a next-generation proteasome inhibitor, at the 15th International Myeloma Workshop (IMW), from Sept. 23-26, 2015, in Rome (Press release, Amgen, SEP 23, 2015, View Source;p=RssLanding&cat=news&id=2089855 [SID:1234507524]). Kyprolis is approved in the United States (U.S.) for use in combination with lenalidomide and dexamethasone for the treatment of relapsed multiple myeloma, an incurable blood cancer. In the European Union (EU), Kyprolis is under accelerated assessment with the European Medicines Agency (EMA).

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"Multiple myeloma has historically been one of the most difficult to treat diseases because of the inherent complexities related to the recurring pattern of remission and relapse," said Sean E. Harper, M.D., executive vice president of Research and Development at Amgen. "Data to be presented at IMW will help provide further insight into the potential of Kyprolis as an important treatment option for patients living with relapsed multiple myeloma."

The following Amgen-sponsored abstracts will be presented at the meeting:

Kyprolis

Efficacy and Safety of Carfilzomib, Lenalidomide, and Dexamethasone (KRd) vs Lenalidomide and Dexamethasone (Rd) in Patients (Pts) With Relapsed Multiple Myeloma (RMM) Based on Age: Secondary Analysis From the Phase 3 Study ASPIRE (NCT01080391)
A. Palumbo, Abstract BP-051, Friday, Sept. 25, 6:40 p.m. – 7:40 p.m. CEST (Poster Area)
Superior Health-Related Quality of Life with Carfilzomib, Lenalidomide, and Dexamethasone versus Lenalidomide and Dexamethasone in Patients With Relapsed Multiple Myeloma (MM): Results From the ASPIRE Trial
A.K. Stewart, Abstract BP-052, Friday, Sept. 25, 6:40 – 7:40 p.m. CEST (Poster Area)

Observational

Survival Analysis in Newly Diagnosed Multiple Myeloma Patients in the United States Medicare Database
A. A. Yusuf, Abstract PO-171, Thursday, Sept. 24, 6:40 – 7:40 p.m. CEST (Poster Area)
Hospitalization Rates for Newly Diagnosed Multiple Myeloma Patients in the United States Medicare Database
A.A. Yusuf, Abstract PO-179, Thursday, Sept. 24, 6:40 – 7:40 p.m. CEST (Poster Area)
In the U.S., Kyprolis is approved as a monotherapy and in combination with lenalidomide and dexamethasone. In Mexico, Israel, Argentina and Thailand, Kyprolis is approved as monotherapy for relapsed, refractory multiple myeloma. In the EU, Kyprolis is under accelerated assessment with the EMA. The EMA application for Kyprolis is based on data from the Phase 3 ASPIRE (CArfilzomib, Lenalidomide, and DexamethaSone versus Lenalidomide and Dexamethasone for the treatment of PatIents with Relapsed Multiple MyEloma) trial and other relevant data. The study showed that patients treated with Kyprolis in combination with Revlimid (lenalidomide) and low-dose dexamethasone (regimen referred to as KRd) lived 50 percent longer (8.7 months) without their disease worsening compared to patients treated with lenalidomide and low-dose dexamethasone alone (regimen referred to as Rd). The median progression free survival was 26.3 months in those treated with KRd compared to 17.6 months in those treated with Rd (HR=0.69; 95 percent CI: 0.57-0.83; p<0.0001). The most common adverse events in the Kyprolis arm included pneumonia (1 percent), myocardial infarction (0.8 percent) and upper respiratory tract infection (0.8 percent).

About Multiple Myeloma
Multiple myeloma is the second most common hematologic cancer.1 Worldwide, more than 230,000 people are living with multiple myeloma with approximately 114,000 new cases diagnosed and 80,000 people dying of the disease each year.2,3 In the U.S., there are nearly 96,000 people living with, or in remission from, multiple myeloma.4 The estimated number of new cases of multiple myeloma in 2014 was more than 24,000 and the estimated number of deaths was 11,090.4 In Europe, it is estimated that more than 89,000 people are living with multiple myeloma. Approximately 39,000 new cases were diagnosed and 24,000 people died in 2012.3

About ASPIRE
The international, randomized Phase 3 ASPIRE (CArfilzomib, Lenalidomide, and DexamethaSone versus Lenalidomide and Dexamethasone for the treatment of PatIents with Relapsed Multiple MyEloma) trial evaluated Kyprolis in combination with lenalidomide and low-dose dexamethasone, versus lenalidomide and low-dose dexamethasone alone, in patients with relapsed multiple myeloma following treatment with one to three prior regimens. The primary endpoint of the trial was progression-free survival (PFS), defined as the time from treatment initiation to disease progression or death. Secondary endpoints included overall survival (OS), overall response rate (ORR), duration of response (DOR), disease control rate, health-related quality of life (HR-QoL) and safety. Patients were randomized to receive Kyprolis (20 mg/m2 on days 1 and 2 of cycle one only, escalating to 27 mg/m2 subsquently), in addition to a standard dosing schedule of lenalidomide (25 mg per day for 21 days on, 7 days off) and low-dose dexamethasone (40 mg per week in 4 week cycles), versus lenalidomide and low-dose dexamethasone alone. In the Kyprolis arm, patients were given a 10 minute infusion on days 1, 2, 8, 9, 15 and 16. Kyprolis was omitted on days 8 and 9 during cycles 13-18 and not administered beyond 18 cycles.The study randomized 792 patients at sites in North America, Europe and Israel.

The OS results did not cross the pre-specified early stopping boundary for the interim analysis. At the time of the interim analysis, there were 143 deaths (36.1 percent) in the KRd group, compared to 162 deaths (40.9 percent) in the Rd group. The ORR was 87 percent with KRd and 67 percent with Rd. In the KRd and Rd groups, 32 percent versus 9 percent of patients achieved a complete response or higher (stringent complete response [sCR] or complete response [CR]), a measurement indicating depth of response. Median DOR was 28.6 months for patients receiving KRd (95 percent CI, 24.9 to 31.3 months) and 21.2 months for patients receiving Rd (95 percent CI, 16.7 to 25.8 months).

The rate of deaths due to adverse events (AEs) within 30 days of the last dose was balanced between the KRd arm and the Rd arm. The most common causes of death occurring in patients in the KRd arm compared to the Rd arm included cardiac disorders (3 percent versus 2 percent), infection (2 percent versus 3 percent), renal (0 percent versus less than 1 percent) and other AEs (2 percent versus 3 percent). Serious AEs were reported in 60 percent of the patients in the KRd arm and 54 percent of the patients in the Rd arm. The most common serious AEs reported in the KRd arm compared to the Rd arm were pneumonia (14 percent versus 11 percent), respiratory tract infection (4 percent versus 1.5 percent), pyrexia (4 percent versus 2 percent) and pulmonary embolism (3 percent versus 2 percent). Discontinuation due to any AE occurred in 26 percent of patients in the KRd arm versus 25 percent of patients in the Rd arm. Adverse events leading to discontinuation of Kyprolis occurred in 12 percent of patients.

The ASPIRE data were presented at the 56th Annual Meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper) and published in The New England Journal of Medicine in December 2014.

About Kyprolis (carfilzomib) for Injection
Kyprolis (carfilzomib) for Injection is indicated in combination with lenalidomide and dexamethasone for the treatment of patients with multiple myeloma who have received one to three prior lines of therapy.

Kyprolis is also indicated under FDA accelerated approval as a single agent for the treatment of patients with multiple myeloma who have received at least two prior therapies including bortezomib and an immunomodulatory agent and have demonstrated disease progression on or within 60 days of completion of the last therapy. Approval is based on response rate. Clinical benefit, such as improvement in survival or symptoms, has not been verified.

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. Kyprolis is also approved for use in Argentina, Israel, Mexico and Thailand. For more information about Kyprolis, visit www.kyprolis.com.

Important Safety Information Regarding Kyprolis (carfilzomib) for Injection U.S. Indication
This safety information is specific to the current U.S. approved indication.

Cardiac Toxicities
New onset or worsening of preexisting 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. Death due to cardiac arrest has occurred within a day of Kyprolis administration.

Withhold Kyprolis for Grade 3 or 4 cardiac adverse events until recovery, and consider whether to restart Kyprolis based on a benefit/risk assessment.

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, and conduction abnormalities may be at greater risk for cardiac complications.

Acute Renal Failure
Cases of acute renal failure and renal insufficiency adverse events (renal impairment, acute renal failure, 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. This risk was greater in patients with a baseline reduced estimated creatinine clearance. 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 and should be based on an assessment of the patient’s underlying risks, treatment regimen, and clinical status.

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. Reduce or withhold dose as appropriate.

Thrombotic Thrombocytopenic Purpura / Hemolytic Uremic Syndrome (TTP/HUS)
Cases of 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 neuroradiological 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 and the potential hazard to the fetus if Kyprolis is used during pregnancy.

ADVERSE REACTIONS
The most common adverse events occurring in at least 20% of patients treated with Kyprolis in monotherapy trials: anemia, fatigue, thrombocytopenia, nausea, pyrexia, decreased platelets, dyspnea, diarrhea, decreased lymphocyte, headache, decreased hemoglobin, cough, edema peripheral.

The most common adverse events occurring in at least 20% of patients treated with Kyprolis in the combination therapy trial: decreased lymphocytes, decreased absolute neutrophil count, decreased phosphorus, anemia, neutropenia, decreased total white blood cell count, decreased platelets, diarrhea, fatigue, thrombocytopenia, pyrexia, muscle spasm, cough, upper respiratory tract infection, decreased hemoglobin, hypokalemia.

Full prescribing information is available at www.kyprolis.com.