Agios Announces New Data from Ongoing Phase 1 Dose Escalation and Expansion Trial of AG-221 Showing Durable Clinical Activity in Patients with Advanced Hematologic Malignancies

On June 12, 2015 Agios Pharmaceuticals reported new data from the dose-escalation phase and expansion cohorts from the ongoing Phase 1 study evaluating single agent AG-221, a first-in-class, oral, selective, potent inhibitor of mutant isocitrate dehydrogenase-2 (IDH2), in advanced hematologic malignancies (Press release, Agios Pharmaceuticals, JUN 12, 2015, View Source;p=RssLanding&cat=news&id=2058805 [SID:1234505404]). The data will be presented at the 20th Congress of the European Hematology Association (EHA) (Free EHA Whitepaper) taking place June 11-14, 2015 in Vienna.

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Data as of May 1, 2015 from 177 patients (104 in dose escalation and 73 from the first four expansion cohorts) with advanced hematologic malignancies treated with single agent AG-221 showed durable clinical activity and a favorable safety profile. More than half of the 177 patients remain on treatment. The study had an overall response rate of 40 percent (63 of 158 response-evaluable patients, using the criteria below) and a complete remission rate of 16 percent (26 of 158 response-evaluable patients). Patients responding to AG-221 continue to show durable clinical activity on treatment for more than 15 months, with an estimated 76 percent of responders staying on treatment for six months or longer. The overall safety profile observed was consistent with previously reported data with more than 100 additional patients treated as of the last analysis.

This new data reflects responses in the evaluable population, which includes all patients with a pre-AG-221 screening assessment and day 28 or later response assessment or an earlier discontinuation for any reason. Patients with a screening assessment who were still on treatment, but had not reached the day 28 disease assessment, were excluded.

"The clinical profile of AG-221 continues to be impressive from the perspectives of response rate, durability, safety and unique mechanism of action," said Courtney DiNardo, M.D., lead investigator and assistant professor, leukemia at University of Texas MD Anderson Cancer Center. "Additionally, it is encouraging to see early proof-of-concept in myelodysplastic syndrome (MDS) and untreated acute myeloid leukemia (AML) given the need for more effective therapies for these patients."

"As the data from the AG-221 study continue to mature, we are compiling a robust dataset to quickly move this program into global registration studies later this year in collaboration with Celgene," said Chris Bowden, M.D., chief medical officer of Agios. "We are excited about the speed of enrollment we’ve seen to date in our four expansion cohorts and are on track to enroll our recently announced fifth expansion cohort of 125 patients with relapsed and/or refractory AML. With this progress, we are executing on our strategy to combine speed and breadth to reach people with hematologic malignancies in urgent need of better treatments."

About the Ongoing Phase 1 Trial for AG-221 in Advanced Hematologic Malignancies

AG-221 is currently being evaluated in an ongoing Phase 1 trial that includes a dose-escalation phase and four expansion cohorts of 25 patients each, evaluating patients with relapsed or refractory AML who are 60 years of age and older and transplant ineligible; relapsed or refractory AML patients under age 60; untreated AML patients who decline standard of care chemotherapy; and patients with other IDH2-mutant positive hematologic malignancies. Data reported here are from patients receiving AG-221 administered from 60 mg to 450 mg total daily doses in the dose escalation arm and 100 mg once daily in the first four expansion arms, as of May 1, 2015. The median age of these patients is 69 (ranging from 22-90). Treatment with AG-221 showed substantial reduction in the plasma levels of the oncometabolite 2-hydroxglutarate (2HG) to the level observed in healthy volunteers.

Safety Data

A safety analysis was conducted for all 177 treated patients as of May 1, 2015.

The majority of adverse events reported by investigators were mild to moderate, with the most common being nausea, fatigue, increased blood bilirubin and diarrhea.

The majority of serious adverse events (SAE) were disease related; SAEs possibly related to study drug were reported in 27 patients.
A maximum tolerated dose (MTD) has not been reached.
The all-cause 30-day mortality rate was 4.5 percent.

Efficacy Data

Sixty-three out of 158 response-evaluable patients achieved investigator-assessed objective responses for an overall response rate of 40 percent as of May 1, 2015.

Of the 63 patients who achieved an objective response, there were 26 (16 percent) complete remissions (CR), three CRs with incomplete platelet recovery (CRp), 14 marrow CRs (mCR), two CRs with incomplete hematologic recovery (CRi) and 18 partial remissions (PR).
Of the 111 patients with relapsed or refractory AML, 46 (41 percent) achieved an objective response, including 20 (18 percent) CRs, one CRp, 16 PRs, eight mCRs and one CRi.

Of the 22 patients with AML that had not been treated, seven achieved an objective response, including three CRs, two PRs, one mCR and one CRi.

Of the 14 patients with myelodysplastic syndrome (MDS), seven achieved an objective response, including two CRs, one CRp and four mCRs.

Responses were durable, with duration on study drug more than 15 months and ongoing. As of the analysis date, an estimated 88 percent of responses lasted three months or longer, and 76 percent of responses lasted six months or longer.

Upcoming Milestones for AG-221

Agios studies in IDH2-mutated solid and hematologic tumors are ongoing or planned for 2015 to further support development of AG-221.

Continue to enroll patients in the fifth expansion cohort of 125 patients with IDH2 mutant-positive AML who are in second or later relapse, refractory to second-line induction or re-induction treatment, or have relapsed after allogeneic transplantation.

Initiate combination trials to evaluate AG-221 as a potential frontline treatment for patients with AML and a broad range of hematologic malignancies in the second half of 2015.

Initiate a global Phase 3 registration-enabling study in relapsed/refractory AML patients that harbor an IDH2 mutation in the second half of 2015.

Continue dose escalation in the Phase 1/2 trial in patients with advanced solid tumors, including glioma and angioimmunoblastic T-cell lymphoma (AITL) that carry an IDH2 mutation in 2015.

Pacritinib Phase 3 Study Shows Positive Results In Patient Reported Outcomes Measuring Quality Of Life In Patients With Myelofibrosis

On June 12, 2015 CTI BioPharma and Baxter International reported new patient-reported outcome (PRO) data for pacritinib – an investigational oral multikinase inhibitor with specificity for JAK2 and FLT3 – from the Phase 3 PERSIST-1 study (Press release, CTI BioPharma, JUN 12, 2015, View Source;p=RssLanding&cat=news&id=2058786 [SID:1234505405]). As recently reported at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) annual meeting, results show a significant reduction in the Total Symptom Score (TSS) (the proportion of patients with a 50 percent or greater reduction in TSS from baseline to Week 24), and in each individual common disease-related symptom, from baseline to Week 24, in patients treated with pacritinib compared to best available therapy (exclusive of a JAK inhibitor) (BAT). These PROs, as well as other quality of life measures, will be presented at the 20th Congress of European Hematology Association (EHA) (Free EHA Whitepaper) by Adam Mead, M.D., Guy’s and St. Thomas’ NHS Foundation Trust, Guy’s Hospital, London, United Kingdom in an oral presentation on Sunday, June 14, 2015 at 12:15 CEST (abstract #LB2072). These data were also selected for inclusion in the official EHA (Free EHA Whitepaper) Press Briefing which occurred today (Friday, June 12, 2015) at 08:30 CEST. As previously reported, the PERSIST-1 trial met its primary endpoint of spleen volume reduction of 35 percent or greater from baseline to Week 24 as measured by MRI/CT scan.

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Myelofibrosis is a rare blood cancer associated with significantly reduced quality of life and shortened survival. As the disease progresses, the body slows production of important blood cells and within one year of diagnosis the incidence of disease-related thrombocytopenia (very low blood platelet counts), severe anemia, and red blood cell transfusion requirements increases significantly. Among other complications, most patients with myelofibrosis present with enlarged spleens (splenomegaly), as well as many other potentially devastating physical symptoms such as abdominal discomfort, bone pain, feeling full after eating little, severe itching, night sweats, and extreme fatigue.

"Patient-reported outcomes are an important measure for understanding the potential benefit of a therapy on patients’ lives – particularly for a disease such as myelofibrosis where the symptoms have such a tremendous impact on the quality of patients’ daily lives," stated James A. Bianco, M.D., President and CEO of CTI BioPharma. "These new data from the PERSIST-1 study further support our belief, not only in the activity of pacritinib, but also the potential to positively impact patients’ daily lives by relieving the symptoms that accompany myelofibrosis."

"The PERSIST-1 trial has continued to generate positive and important findings for the hematology community," said David Meek, Head of Oncology at Baxter BioScience. "We look forward to advancing the clinical trial program of pacritinib as we work to realize the full potential of this investigational compound to help patients with serious blood cancers, such as myelofibrosis."

Study Details and Findings Presented at EHA (Free EHA Whitepaper)
PERSIST-1 is a randomized (2:1), controlled Phase 3 registration-directed trial comparing the efficacy and safety of pacritinib to BAT – which included a range of currently utilized off-label treatments – in 327 patients with myelofibrosis, regardless of the patients’ platelet counts. As previously reported at ASCO (Free ASCO Whitepaper), the trial met its primary endpoint of spleen volume reduction (35 percent or greater reduction from baseline to Week 24 by MRI/CT scan) in the intent-to-treat (ITT) population; these results included patients with severe or life-threatening thrombocytopenia. The study also measured patient-reported outcomes (PROs), the proportion of patients with a 50 percent or greater reduction in TSS from baseline to Week 24, which have become important for approval of new therapies and was one of the secondary endpoints of the study. As previously reported, patients treated with pacritinib experienced greater improvement in their disease-related symptoms (ITT patient population: 24.5 percent of pacritinib-treated patients vs 6.5 percent of BAT-treated patients, p<0.0001; evaluable patient population: 40.9 percent of pacritinib-treated patients vs 9.9 percent of BAT-treated patients, p<0.0001).

New data presented at EHA (Free EHA Whitepaper), which included results from multiple PROs measurement tools, showed:

Myeloproliferative Neoplasm Symptom Assessment (MPN-SAF TSS and MPN-SAF TSS 2.0)

When using the MPN-SAF TSS and MPN-SAF TSS 2.0, each of the six common disease-related symptoms from the TSS results showed improvements in abdominal discomfort (46 percent improvement with pacritinib vs no improvement with BAT); bone pain (32 percent improvement with pacritinib vs 8 percent improvement with BAT); feeling of early fullness (45 percent improvement with pacritinib vs 1 percent worsening with BAT); itching (48.5 percent improvement with pacritinib vs 10 percent improvement with BAT); night sweats (69.5 percent improvement with pacritinib vs no improvement with BAT); and fatigue (27.5 percent improvement with pacritinib vs 4 percent worsening with BAT). MPN-SAF TSS and MPN-SAF TSS 2.0 are specific sets of questions patients answer daily (via electronic diary) and which are based on a questionnaire originally developed by Ruben A. Mesa, M.D., Deputy Director of the Mayo Clinic Cancer Center in Scottsdale, Arizona, USA.

Patient Global Impression of Change (PGIC)
Based on the PGIC assessment – which measures a patient’s assessment of overall health on a 7-point scale ranging from "very much worse" to "very much improved" – approximately 80 percent of evaluable patients treated with pacritinib rated their condition as improved compared to approximately 20 percent with BAT.

European Organization for Research and Treatment of Cancer Quality-of-Life 30 Questionnaire (EORTC QLQ-C30)
A greater improvement was also reported by evaluable patients treated with pacritinib vs BAT across all components of the EORTC QLQ-C30 questionnaire, a well-validated measure of quality of life in cancer patients.

The most common adverse events occurring with pacritinib within 24 weeks, of any grade, were mild to moderate diarrhea (53.2 percent vs 12.3 percent with BAT), nausea (26.8 percent vs 6.6 percent with BAT), anemia (22.3 percent vs 19.8 percent with BAT), thrombocytopenia (16.8 percent vs 13.2 percent with BAT), and vomiting (15.9 percent vs 5.7 percent with BAT). Of the patients treated with pacritinib, 3 discontinued therapy and 13 patients required dose interruption (average one week) for diarrhea. Patients received a daily full dose of pacritinib over the duration of treatment. Gastrointestinal symptoms typically lasted for approximately one week and few patients discontinued treatment due to side effects. There were no Grade 4 gastrointestinal events reported.

About Pacritinib
Pacritinib is an investigational oral multikinase inhibitor with specificity for JAK2 and FLT3. The JAK family of enzymes is a central component in signal transduction pathways, which are critical to normal blood cell growth and development, as well as inflammatory cytokine expression and immune responses. Mutations in these kinases have been shown to be directly related to the development of a variety of blood-related cancers, including myeloproliferative neoplasms, leukemia, and lymphoma.

About Myelofibrosis
Myelofibrosis is a serious and life-threatening chronic blood cancer caused by the accumulation of malignant bone marrow cells that triggers an inflammatory response and scars the bone marrow. The replacement of bone marrow with scar tissue limits its ability to produce red blood cells, prompting the spleen and liver to take over this function. Symptoms that arise from this disease include enlargement of the spleen, anemia, extreme fatigue, and pain. Myelofibrosis is a one of three main types of myeloproliferative neoplasms (MPN), which are a closely related group of hematological blood cancers.1 The estimated prevalence of MPNs suggest there are approximately 300,000 people living with the disease in the U.S. of which myelofibrosis accounts for approximately 18,000 patients.2 In Europe, there is a wide variation of prevalence observed across data sources. Myelofibrosis has a median age of 64 at the time of diagnosis2 and is a progressive disease with approximately 20 percent of patients eventually developing acute myeloid leukemia.3 The median survival for high-risk patients is less than one and a half years; median survival for myelofibrosis patients overall is approximately six years.4

About the PERSIST Phase 3 Development Program of Pacritinib
Pacritinib is currently being evaluated in two Phase 3 clinical trials, known as the PERSIST program, for patients with myelofibrosis. PERSIST-1 is a randomized (2:1), open-label, multinational Phase 3 clinical trial comparing the efficacy and safety of pacritinib with that of BAT, in 327 enrolled patients with primary and secondary myelofibrosis (PMF), post-polycythemia vera myelofibrosis (PPV-MF), or post-essential thrombocythemia myelofibrosis (PET-MF), without exclusion for low platelet counts. PERSIST-2 is a randomized (2:1), open-label, multinational Phase 3 clinical trial evaluating pacritinib compared to BAT, including the approved JAK1/JAK2 inhibitor dosed according to product label for patients with myelofibrosis whose platelet counts are less than or equal to 100,000 per microliter. The trial is designed to enroll up to 300 patients in North America, Europe, Australia, New Zealand, and Russia.

Additional details are available at www.clinicaltrials.gov or www.PERSISTprogram.com.

CTI BioPharma and Baxter BioScience, which is expected to become Baxalta Incorporated in mid-2015, entered into a worldwide license agreement in November 2013 to develop and commercialize pacritinib. CTI BioPharma and Baxter will jointly commercialize pacritinib in the U.S. while Baxter has exclusive commercialization rights for all indications outside the U.S.

Targovax and Oncos Therapeutics join forces to create a Nordic leader within immuno-oncology

On June 11, 2015 Targovax AS ("Targovax" or the "Company") reported that it has entered into an agreement with the shareholders of Oncos Therapeutics Oy ("Oncos") to acquire the shares of Oncos with settlement in Targovax shares (the "Transaction") (Press release, Oncos Therapeutics, JUN 11, 2015, View Source [SID:1234512624]). The combined company with multiple assets in research and development will be a Nordic leader within immuno-oncology. The new company will have a strong senior management team and be financially backed by reputable institutional investors with sector specialist HealthCap as the largest shareholder. After the Transaction, the shareholders of Oncos will own 50 per cent of Targovax.

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To fund the combined company, Targovax contemplates to raise NOK 125-150 million through a private placement directed towards Norwegian and international investors (the "Private Placement") and has already received indications from leading institutional investors for a substantial amount, including but not limited to a NOK 26 million commitment from HealthCap, NOK 25 million from Datum Invest AS and NOK 5 million from the Norwegian Radium Hospital Research Foundation. In addition, the Private Placement is directed towards the 24 employees of Targovax and Oncos and their respective subsidiaries, and companies controlled by any such employees ("Eligible Employees").

Rationale for the Transaction
Targovax and Oncos—based in Oslo and Helsinki, respectively—are both clinical-stage biotechnology companies with broad pipelines addressing the immuno-oncology market, which currently is quite modest, but is expected to grow to USD 30-35 billion over the next decade.

By combining Targovax and Oncos, a larger and more visible Nordic immuno-oncology player with a more extensive portfolio will be created. Furthermore, the combination of two highly competent and complementary organizations will promote more efficient execution and accelerate the development of ongoing and future programs.

Clinical results to date confirm the safety and the mechanisms of action for both technology platforms. Safety data from Targovax and Oncos programs have been collected on approximately 250 and 290 patients, respectively. Both companies have completed Phase I and are now in Phase II development, or about to enter Phase II development, with broad pipelines, and both utilize an immuno-oncology approach that is highly suitable for combination therapies. Clinical programs in several indications will provide increased opportunities for newsflow over the next 18 months.

"The combination of Targovax and Oncos creates a major Nordic player within immuno-oncology. We will have a wide array of programs in the pipeline and multiple shots at goal. The companies’ complementary technologies will provide a unique platform for the development of cutting-edge immunotherapies, and we will have a higher chance of success, thus offering investors an even more compelling opportunity. I am excited to lead an organization of highly skilled professionals with deep industry experience", said Gunnar Gårdemyr, Chief Executive Officer of Targovax.

Senior Executives and Board of Directors
After the Transaction, the highly experienced senior management team will consist of the following members:

Gunnar Gårdemyr (Chief Executive Officer) has more than 30 years of international experience in the pharmaceutical and biotech industry from companies such as Nycomed and Takeda
Dr. Magnus Jäderberg (Chief Medical Officer) has more than 25 years of experience from R&D functions and was previously CMO at Bristol-Myers Squibb (Europe)
Øystein Soug (Chief Financial Officer) has prior experience as the Chief Financial Officer of Algeta and in positions with the Orkla Group
Jon Amund Eriksen (Chief Operating Officer) was the co-founder of Targovax and has 35 years of R&D experience in the pharmaceutical and biotech industry, of which 25 years were within immuno-oncology
After the Transaction, the proposed Board of Directors will consist of the following members:

Jónas Einarsson (Chairman of the Board of Directors), Chief Executive Officer of the Norwegian Radium Hospital Research Foundation
Johan Christenson (Board Member), Partner at HealthCap
Per Samuelsson (Board Member), Partner at HealthCap
Lars Lund-Roland (Board Member), CEO of Bringwell, with more than 25 years of experience from various executive positions within Merck
Bente-Lill Romøren (Board Member), Board Member of the Norwegian Radium Hospital Research Foundation and Chairman of Farmastat and Photocure
Robert Burns (Board Member), Chairman of Haemostatix, previously CEO of 4-Antibody, Affitech, and Celldex Therapeutics, all three being early companies in the immuno-oncology space

Bristol-Myers Squibb Demonstrates Commitment to Hematology and Advancing Research and Development Across Multiple Blood Cancers Through Immuno-Oncology Leadership at the 20th Congress of the European Hematology Association

On June 11, 2015 Bristol-Myers Squibb reported the presentation of clinical research from its hematology portfolio at the 20th Congress of the European Hematology Association (EHA) (Free EHA Whitepaper) in Vienna, Austria from June 11-14 (Press release, Bristol-Myers Squibb, JUN 11, 2015, View Source [SID:1234505393]). Bristol-Myers Squibb will present data for elotuzumab, an investigational immunostimulatory antibody, in relapsed or refractory multiple myeloma; Opdivo (nivolumab), in patients with relapsed or refractory lymphoid malignancies; and Sprycel (dasatinib), in chronic myeloid leukemia.

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Data to be presented at EHA (Free EHA Whitepaper) exemplify Bristol-Myers Squibb’s commitment to advancing the treatment of blood cancers through its experience in hematology and its transformative science of Immuno-Oncology.

Key oral presentations include:

ELOQUENT-2: A Phase 3, open-label study [Abstract #S471] comparing elotuzumab in combination with lenalidomide and dexamethasone (ELd) versus lenalidomide and dexamethasone alone (Ld) in patients with relapsed or refractory multiple myeloma, will be featured in the EHA (Free EHA Whitepaper) press briefing on Friday, June 12 at 8:30 a.m. CEST and will be presented during the Presidential Symposium, also on June 12, at 3:45 p.m. CEST. The ELOQUENT-2 study was published in the New England Journal of Medicine on June 2.

Study 009: A Phase 2, open-label study [Abstract #S103] comparing elotuzumab in combination with bortezomib (a proteasome inhibitor) and dexamethasone versus bortezomib and dexamethasone in patients with relapsed or refractory multiple myeloma will be presented in an oral session on June 12 at 12:00 p.m. CEST.

PREAMBLE: A preliminary analysis of an ongoing, multinational, observational study [Abstract #S148] evaluating the real-world clinical effectiveness of standard treatments, including immunomodulatory drugs (IMiDs) and proteasome inhibitors (PIs), in patients with relapsed or refractory multiple myeloma will be presented in an oral session on June 12 at 12:00 p.m. CEST.

CheckMate -039: Updated data from a Phase 1 study [Abstract #S808] evaluating the safety, tolerability and potential efficacy of Opdivo in several hematologic malignancies, including classical Hodgkin Lymphoma will be presented in an oral session on Sunday, June 14 at 8:45 a.m. CEST.

"Bristol-Myers Squibb is leveraging its broad experience in oncology and leading Immuno-Oncology science to develop a portfolio of innovative therapies, including a novel modality for multiple myeloma, because we believe patients with blood cancers deserve more," said Michael Giordano, senior vice president, Head of Development, Oncology, Bristol-Myers Squibb. "These data at EHA (Free EHA Whitepaper) illustrate our commitment to transforming survival expectations for more patients with a variety of hematologic malignancies."

About Elotuzumab

Elotuzumab is an investigational immunostimulatory antibody targeted against Signaling Lymphocyte Activation Molecule (SLAMF7), a cell-surface glycoprotein that is highly and uniformly expressed on myeloma cells and Natural Killer (NK) cells, but is not detected on normal solid tissues or on hematopoietic stem cells. Elotuzumab is being investigated to determine whether the compound may selectively target myeloma cells. It is believed that elotuzumab works through a dual mechanism of action: binding to SLAMF7 on NK cells, directly activating them and binding to SLAMF7 on myeloma cells, flagging them for NK cell recognition and destruction.

In May 2014, the U.S. Food and Drug Administration (FDA) granted elotuzumab Breakthrough Therapy Designation for use in combination with one of the chemotherapy treatments for multiple myeloma (lenalidomide, used in combination with dexamethasone) in patients who have received one or more prior treatments. Elotuzumab is an investigational compound, and its safety and efficacy have not been evaluated by the FDA or any other health authority.

Bristol-Myers Squibb and AbbVie are co-developing elotuzumab, with Bristol-Myers Squibb solely responsible for commercial activities.

About Opdivo

Bristol-Myers Squibb has a broad, global development program to study Opdivo in multiple tumor types consisting of more than 50 trials – as monotherapy or in combination with other therapies – in which more than 8,000 patients have been enrolled worldwide.

In May 2014, the FDA granted Opdivo Breakthrough Therapy Designation for the treatment of patients with Hodgkin Lymphoma after failure of autologous stem cell transplant and brentuximab. Opdivo became the first PD-1 immune checkpoint inhibitor to receive regulatory approval anywhere in the world on July 4, 2014 when Ono Pharmaceutical Co. announced that it received manufacturing and marketing approval in Japan for the treatment of patients with unresectable melanoma. In the U.S., the (FDA) granted its first approval for Opdivo for the treatment of patients with unresectable or metastatic melanoma and disease progression following Yervoy (ipilimumab) and, if BRAF V600 mutation positive, a BRAF inhibitor. On March 4, 2015, Opdivo received its second FDA approval for the treatment of patients with metastatic squamous non-small cell lung cancer with progression on or after platinum-based chemotherapy.

OPDIVO (nivolumab) IMPORTANT SAFETY INFORMATION

Immune-Mediated Pneumonitis

Severe pneumonitis or interstitial lung disease, including fatal cases, occurred with OPDIVO treatment. Across the clinical trial experience in 691 patients with solid tumors, fatal immune-mediated pneumonitis occurred in 0.7% (5/691) of patients receiving OPDIVO; no cases occurred in Trial 1 or Trial 3. In Trial 1, pneumonitis, including interstitial lung disease, occurred in 3.4% (9/268) of patients receiving OPDIVO and none of the 102 patients receiving chemotherapy. Immune-mediated pneumonitis occurred in 2.2% (6/268) of patients receiving OPDIVO; one with Grade 3 and five with Grade 2. In Trial 3, immune-mediated pneumonitis occurred in 6% (7/117) of patients receiving OPDIVO, including, five Grade 3 and two Grade 2 cases. Monitor patients for signs and symptoms of pneumonitis. Administer corticosteroids for Grade 2 or greater pneumonitis. Permanently discontinue OPDIVO for Grade 3 or 4 and withhold OPDIVO until resolution for Grade 2.

Immune-Mediated Colitis

In Trial 1, diarrhea or colitis occurred in 21% (57/268) of patients receiving OPDIVO and 18% (18/102) of patients receiving chemotherapy. Immune-mediated colitis occurred in 2.2% (6/268) of patients receiving OPDIVO; five with Grade 3 and one with Grade 2. In Trial 3, diarrhea occurred in 21% (24/117) of patients receiving OPDIVO. Grade 3 immune-mediated colitis occurred in 0.9% (1/117) of patients. Monitor patients for immune-mediated colitis. Administer corticosteroids for Grade 2 (of more than 5 days duration), 3, or 4 colitis. Withhold OPDIVO for Grade 2 or 3. Permanently discontinue OPDIVO for Grade 4 colitis or recurrent colitis upon restarting OPDIVO.

Immune-Mediated Hepatitis

In Trial 1, there was an increased incidence of liver test abnormalities in the OPDIVO-treated group as compared to the chemotherapy-treated group, with increases in AST (28% vs 12%), alkaline phosphatase (22% vs 13%), ALT (16% vs 5%), and total bilirubin (9% vs 0). Immune-mediated hepatitis occurred in 1.1% (3/268) of patients receiving OPDIVO; two with Grade 3 and one with Grade 2. In Trial 3, the incidences of increased liver test values were AST (16%), alkaline phosphatase (14%), ALT (12%), and total bilirubin (2.7%). Monitor patients for abnormal liver tests prior to and periodically during treatment. Administer corticosteroids for Grade 2 or greater transaminase elevations. Withhold OPDIVO for Grade 2 and permanently discontinue OPDIVO for Grade 3 or 4 immune-mediated hepatitis.

Immune-Mediated Nephritis and Renal Dysfunction

In Trial 1, there was an increased incidence of elevated creatinine in the OPDIVO-treated group as compared to the chemotherapy-treated group (13% vs 9%). Grade 2 or 3 immune-mediated nephritis or renal dysfunction occurred in 0.7% (2/268) of patients. In Trial 3, the incidence of elevated creatinine was 22%. Immune-mediated renal dysfunction (Grade 2) occurred in 0.9% (1/117) of patients. Monitor patients for elevated serum creatinine prior to and periodically during treatment. For Grade 2 or 3 serum creatinine elevation, withhold OPDIVO and administer corticosteroids; if worsening or no improvement occurs, permanently discontinue OPDIVO. Administer corticosteroids for Grade 4 serum creatinine elevation and permanently discontinue OPDIVO.

Immune-Mediated Hypothyroidism and Hyperthyroidism

In Trial 1, Grade 1 or 2 hypothyroidism occurred in 8% (21/268) of patients receiving OPDIVO and none of the 102 patients receiving chemotherapy. Grade 1 or 2 hyperthyroidism occurred in 3% (8/268) of patients receiving OPDIVO and 1% (1/102) of patients receiving chemotherapy. In Trial 3, hypothyroidism occurred in 4.3% (5/117) of patients receiving OPDIVO. Hyperthyroidism occurred in 1.7% (2/117) of patients, including one Grade 2 case. Monitor thyroid function prior to and periodically during treatment. Administer hormone replacement therapy for hypothyroidism. Initiate medical management for control of hyperthyroidism.

Other Immune-Mediated Adverse Reactions

In Trial 1 and 3 (n=385), the following clinically significant immune-mediated adverse reactions occurred in <2% of OPDIVO-treated patients: adrenal insufficiency, uveitis, pancreatitis, facial and abducens nerve paresis, demyeliniation, autoimmune neuropathy, motor dysfunction, and vasculitis. Across clinical trials of OPDIVO administered at doses 3 mg/kg and 10 mg/kg, additional clinically significant, immune-mediated adverse reactions were identified: hypophysitis, diabetic ketoacidosis, hypopituitarism, Guillain-Barré syndrome, and myasthenic syndrome. Based on the severity of adverse reaction, withhold OPDIVO, administer high-dose corticosteroids, and, if appropriate, initiate hormone- replacement therapy.

Embryofetal Toxicity

Based on its mechanism of action, OPDIVO can cause fetal harm when administered to a pregnant woman. Advise pregnant women of the potential risk to a fetus. Advise females of reproductive potential to use effective contraception during treatment with OPDIVO and for at least 5 months after the last dose of OPDIVO.

Lactation

It is not known whether OPDIVO is present in human milk. Because many drugs, including antibodies, are excreted in human milk and because of the potential for serious adverse reactions in nursing infants from OPDIVO, advise women to discontinue breastfeeding during treatment.

Serious Adverse Reactions

In Trial 1, serious adverse reactions occurred in 41% of patients receiving OPDIVO. Grade 3 and 4 adverse reactions occurred in 42% of patients receiving OPDIVO. The most frequent Grade 3 and 4 adverse drug reactions reported in 2% to <5% of patients receiving OPDIVO were abdominal pain, hyponatremia, increased aspartate aminotransferase, and increased lipase.

In Trial 3, serious adverse reactions occurred in 59% of patients receiving OPDIVO. The most frequent serious adverse drug reactions reported in ≥2% of patients were dyspnea, pneumonia, chronic obstructive pulmonary disease exacerbation, pneumonitis, hypercalcemia, pleural effusion, hemoptysis, and pain.

Common Adverse Reactions

The most common adverse reactions (≥20%) reported with OPDIVO in Trial 1 were rash (21%) and in Trial 3 were fatigue (50%), dyspnea (38%), musculoskeletal pain (36%), decreased appetite (35%), cough (32%), nausea (29%), and constipation (24%).
Please see U.S. Full Prescribing Information for OPDIVO.

About Sprycel

Sprycel is a prescription medicine used to treat adults who have newly diagnosed Philadelphia chromosome–positive (Ph+) chronic myeloid leukemia (CML) in chronic phase. The effectiveness of Sprycel in these patients is based on a study that measured two types of response to treatment (cytogenetic and molecular) by one year. The study is ongoing to find out how Sprycel works over a longer period of time. Sprycel is also indicated for adults with Ph+ CML who are no longer benefitting from, or did not tolerate, other treatment including Gleevec (imatinib mesylate).

SPRYCEL (dasatinib) INDICATIONS & IMPORTANT SAFETY INFORMATION

INDICATIONS

SPRYCEL (dasatinib) is indicated for the treatment of adults with:

Newly diagnosed Philadelphia chromosome-positive chronic myeloid leukemia (Ph+ CML) in chronic phase. The effectiveness of SPRYCEL is based on cytogenetic and major molecular response rates. The trial is ongoing and further data will be required to determine long-term outcome

Chronic, accelerated, or myeloid or lymphoid blast phase Ph+ CML with resistance or intolerance to prior therapy including imatinib
Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL) with resistance or intolerance to prior therapy

IMPORTANT SAFETY INFORMATION

Myelosuppression:

Treatment with SPRYCEL (dasatinib) can cause severe (NCI CTC Grade 3/4) thrombocytopenia, neutropenia, and anemia. Their occurrence is more frequent in patients with advanced phase CML or Ph+ ALL than in chronic phase CML. Myelosuppression was reported in patients with normal baseline laboratory values as well as in patients with pre-existing laboratory abnormalities.

Perform complete blood counts (CBCs) weekly for the first 2 months and then monthly thereafter, or as clinically indicated
Myelosuppression was generally reversible and usually managed by dose interruption, dose reduction, or discontinuation
Hematopoietic growth factor has been used in patients with resistant myelosuppression

Bleeding Related Events:

SPRYCEL caused platelet dysfunction in vitro and thrombocytopenia in humans. In all clinical trials, severe central nervous system (CNS) hemorrhage, including fatalities, occurred in 1% of patients receiving SPRYCEL. Severe gastrointestinal hemorrhage, including fatalities, occurred in 4% of patients and generally required treatment interruptions and transfusions. Other cases of severe hemorrhage occurred in 2% of patients.

Most bleeding events were associated with severe thrombocytopenia. Exercise caution in patients required to take medications that inhibit platelet function or anticoagulants

Fluid Retention:

SPRYCEL is associated with fluid retention. In clinical trials, fluid retention was severe in up to 10% of patients. Severe ascites, pulmonary edema, and generalized edema were each reported in ≤1% of patients.

Patients who develop symptoms suggestive of pleural effusion, such as dyspnea or dry cough, should be evaluated by chest X-ray
Severe pleural effusion may require thoracentesis and oxygen therapy
Fluid retention was typically managed by supportive care measures that included diuretics or short courses of steroids

QT Prolongation:

In vitro data suggest that SPRYCEL has the potential to prolong cardiac ventricular repolarization (QT interval).

In 865 patients with leukemia treated with SPRYCEL in five phase 2 single-arm studies, the maximum mean changes in QTcF (90% upper bound CI) from baseline ranged from 7.0 ms to 13.4 ms
In clinical trials of patients treated with SPRYCEL (N=2440), 16 patients (1%) had QTc prolongation as an adverse reaction. Twenty-two patients (1%) experienced a QTcF>500 ms
Administer SPRYCEL with caution to patients who have or may develop prolongation of QTc, including patients with hypokalemia, hypomagnesemia, or congenital long QT syndrome and patients taking antiarrhythmic drugs, other medicinal products that lead to QT prolongation, and cumulative high-dose anthracycline therapy
Correct hypokalemia or hypomagnesemia prior to SPRYCEL administration

Congestive Heart Failure, Left Ventricular Dysfunction, and Myocardial Infarction:

Cardiac adverse reactions were reported in 7% of 258 patients taking SPRYCEL, including 1.6% of patients with cardiomyopathy, heart failure congestive, diastolic dysfunction, fatal myocardial infarction, and left ventricular dysfunction.

Monitor patients for signs or symptoms consistent with cardiac dysfunction and treat appropriately

Pulmonary Arterial Hypertension (PAH):

SPRYCEL may increase the risk of developing PAH, which may occur any time after initiation, including after more than one year of treatment. Manifestations include dyspnea, fatigue, hypoxia, and fluid retention. PAH may be reversible on discontinuation of SPRYCEL.

Evaluate patients for signs and symptoms of underlying cardiopulmonary disease prior to initiating SPRYCEL and during treatment. If PAH is confirmed, SPRYCEL should be permanently discontinued

Use in Pregnancy:

SPRYCEL may cause fetal harm when administered to a pregnant woman. There are no adequate and well-controlled studies of SPRYCEL in pregnant women.

Women of childbearing potential should be advised of the potential hazard to the fetus and to avoid becoming pregnant when taking SPRYCEL

Nursing Mothers:

It is unknown whether SPRYCEL is excreted in human milk.

Because of the potential for serious adverse reactions in nursing infants, a decision should be made whether to discontinue nursing or to discontinue SPRYCEL

SPRYCEL is a CYP3A4 substrate and a weak time-dependent inhibitor of CYP3A4.

Drugs that may increase SPRYCEL plasma concentrations are:
CYP3A4 inhibitors: Concomitant use of SPRYCEL and drugs that inhibit CYP3A4 should be avoided. If administration of a potent CYP3A4 inhibitor cannot be avoided, close monitoring for toxicity and a SPRYCEL dose reduction should be considered

Strong CYP3A4 inhibitors (eg, ketoconazole, itraconazole, clarithromycin, atazanavir, indinavir, nefazodone, nelfinavir, ritonavir, saquinavir, telithromycin, voriconazole). If SPRYCEL must be administered with a strong CYP3A4 inhibitor, a dose decrease or temporary discontinuation should be considered
Grapefruit juice may also increase plasma concentrations of SPRYCEL and should be avoided

Drugs that may decrease SPRYCEL plasma concentrations are:
CYP3A4 inducers: If SPRYCEL must be administered with a CYP3A4 inducer, a dose increase in SPRYCEL should be considered

Strong CYP3A4 inducers (eg, dexamethasone, phenytoin, carbamazepine, rifampin, rifabutin, phenobarbital) should be avoided. Alternative agents with less enzyme induction potential should be considered. If the dose of SPRYCEL is increased, the patient should be monitored carefully for toxicity
St John’s Wort may decrease SPRYCEL plasma concentrations unpredictably and should be avoided

Antacids may decrease SPRYCEL drug levels. Simultaneous administration of SPRYCEL and antacids should be avoided. If antacid therapy is needed, the antacid dose should be administered at least 2 hours prior to or 2 hours after the dose of SPRYCEL

H2 antagonists/proton pump inhibitors (eg, famotidine and omeprazole): Long-term suppression of gastric acid secretion by use of H2 antagonists or proton pump inhibitors is likely to reduce SPRYCEL exposure. Therefore, concomitant use of H2 antagonists or proton pump inhibitors with SPRYCEL is not recommended

Drugs that may have their plasma concentration altered by SPRYCEL are:
CYP3A4 substrates (eg, simvastatin) with a narrow therapeutic index should be administered with caution in patients receiving SPRYCEL

Adverse Reactions:

The safety data reflect exposure to SPRYCEL in 258 patients with newly diagnosed chronic phase CML in a clinical trial (minimum of 36 months follow up; median duration of therapy was 37 months), and in 2182 patients with imatinib-resistant or -intolerant CML or Ph+ ALL in clinical trials (1520 patients had a minimum of 2 years follow up and 662 patients with chronic phase CML had a minimum of 60 months follow up).

The majority of SPRYCEL-treated patients experienced adverse reactions at some time. Patients aged 65 years and older are more likely to experience toxicity. In the newly diagnosed chronic phase CML trial, the cumulative discontinuation rate was 9% with a minimum of 36 months follow up. In patients resistant or intolerant to prior imatinib therapy, the discontinuation rate for SPRYCEL at 2 years for adverse reactions was: 15% of patients in chronic phase CML (all doses), 16% of patients in accelerated phase CML, 15% of patients in myeloid blast phase CML, 8% in lymphoid blast phase CML, and 8% in Ph+ ALL. In patients resistant or intolerant to prior imatinib therapy with chronic phase CML (minimum 60 months follow up), the rate of discontinuation for adverse reactions was 18% in patients treated with 100 mg once daily.

In newly diagnosed chronic phase CML patients:
The most frequently reported serious adverse reactions included pleural effusion (4%), hemorrhage (2%), congestive heart failure (1%), pulmonary hypertension (1%), and pyrexia (1%)

The most frequently reported adverse reactions (reported in ≥10% of patients) included myelosuppression, fluid retention events (pleural effusion and superficial localized edema), diarrhea, headache, musculoskeletal pain, rash, and nausea

Grade 3/4 laboratory abnormalities included neutropenia (24%), thrombocytopenia (19%), anemia (12%), hypophosphatemia (7%), hypocalcemia (3%), elevated bilirubin (1%), and elevated creatinine (1%)

In patients resistant or intolerant to prior imatinib therapy:
The most frequently reported serious adverse reactions included pleural effusion (11%), gastrointestinal bleeding (4%), febrile neutropenia (4%), dyspnea (3%), pneumonia (3%), pyrexia (3%), diarrhea (3%), infection (2%), congestive heart failure/cardiac dysfunction (2%), pericardial effusion (1%), and CNS hemorrhage (1%)

The most frequently reported adverse reactions (reported in ≥20% of patients) included myelosuppression, fluid retention events, diarrhea, headache, dyspnea, skin rash, fatigue, nausea, and hemorrhage

Grade 3/4 hematologic laboratory abnormalities in chronic phase CML patients resistant or intolerant to prior imatinib therapy who received SPRYCEL 100 mg once daily with a minimum follow up of 60 months included neutropenia (36%), thrombocytopenia (24%) and anemia (13%). Other grade 3/4 laboratory abnormalities included: hypophosphatemia (10%) and hypokalemia (2%)

Among chronic phase CML patients with resistance or intolerance to prior imatinib therapy, cumulative Grade 3 or 4 cytopenias were similar at 2 and 5 years including: neutropenia (36% vs 36%), thrombocytopenia (23% vs 24%), and anemia (13% vs 13%)
Grade 3/4 elevations of transaminase or bilirubin and Grade 3/4 hypocalcemia, hypokalemia, and hypophosphatemia were reported in patients with all phases of CML

Elevations in transaminase or bilirubin were usually managed with dose reduction or interruption

Patients developing Grade 3/4 hypocalcemia during the course of SPRYCEL therapy often had recovery with oral calcium supplementation

AVEO Announces FDA Update for Tivozanib in Colorectal Cancer

On June 11, 2015 AVEO Oncology reported that it has received written feedback from the U.S. Food and Drug Administration regarding a potential pivotal study for tivozanib in the treatment of NRP-1 low colorectal cancer (CRC) (Press release, AVEO, JUN 11, 2015, View Source;p=RssLanding&cat=news&id=2058718 [SID:1234505395]). Tivozanib is an oral, potent, selective inhibitor of vascular endothelial growth factor (VEGF) with a long half-life and activity against all three VEGF receptors.

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AVEO recently presented results from the BATON-CRC study, a 265 patient randomized trial exploring the combination of mFOLFOX6 and tivozanib or bevacizumab as first-line treatment in patients with advanced metastatic CRC, at the 2015 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Tumor Angiogenesis and Vascular Normalization Conference. Among the predefined biomarkers explored in this study, neuropilin-1 (NRP-1), a signaling protein known to bind to VEGF-A in serum, was found to be a potential prognostic marker for angiogenesis inhibitor activity and may be predictive of tivozanib activity relative to bevacizumab.

Results from this study and the Company’s ongoing assay development efforts were presented to the FDA. Updated analyses from the Company’s assay development efforts, which are similar to those presented at the AACR (Free AACR Whitepaper) conference, will be presented at the upcoming European Society of Medical Oncology (ESMO) (Free ESMO Whitepaper) 17th World Congress on Gastrointestinal Cancer taking place July 1-4, 2015 in Barcelona Spain.

In response to questions from AVEO regarding a proposed pivotal phase 3 trial of tivozanib in CRC, the FDA suggested that the Company continue work on the development of its biomarker assay to address variability between assays presented, and that, at present, "insufficient data exists to determine the appropriateness of this [NRP-1 low] subgroup" for the proposed phase 3 study. This feedback is consistent with the Company’s current clinical strategy and discussions with cancer research cooperative groups. As such, AVEO plans to evaluate options to confirm the activity of tivozanib and FOLFOX in NRP-1 low CRC through a prospectively defined, randomized Phase 2 study, while continuing to work on the development of a commercially viable assay.

"NRP-1 expression is a biomarker of growing interest for malignancies treated with angiogenesis inhibitors," said Michael Bailey, president and chief executive officer of AVEO. "As part of our effort to develop a commercial companion diagnostic, we continue to evaluate alternative NRP-1 assays. Feedback from the FDA reinforces the importance of these ongoing efforts prior to embarking on a pivotal trial. We look forward to continuing our dialogue with the Agency and cooperative groups toward the design and potential conduct of an appropriate confirmatory study and the development of a companion diagnostic."