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CTI BioPharma and Baxter Announce Phase 3 Data of Pacritinib in Patients with Myelofibrosis to be Presented in Late-Breaking Session at ASCO
-- Pacritinib Abstract #LBA7006 also to be Highlighted in the Official ASCO Press Program --
SEATTLE and DEERFIELD, Ill., April 22, 2015 /PRNewswire/ -- CTI BioPharma Corp. (CTI BioPharma) (NASDAQ and MTA: CTIC) and Baxter International Inc. (NYSE:BAX) today announced that data from the randomized Phase 3 PERSIST-1 trial evaluating the investigational agent pacritinib in patients with myelofibrosis will be highlighted in a late-breaking oral presentation at the upcoming American Society of Clinical Oncology (ASCO) 2015 Meeting (May 29-June 2, 2015 in Chicago, Ill). These data have also been selected as part of this year's official ASCO press briefing – titled "Targeted Therapy" – to be held on Saturday, May 30, 2015 at 8:00 a.m. CT.

Myelofibrosis (a type of myeloproliferative neoplasm) is a rare, but serious and life-threatening chronic bone marrow disorder 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. Pacritinib is a next generation JAK2/FLT3 inhibitor under investigation for the treatment of patients with myelofibrosis.

The details of the oral presentation are:

Title: Results of the PERSIST-1 phase III study of pacritinib (PAC) versus best available therapy (BAT) in primary myelofibrosis (PMF), post-polycythemia vera myelofibrosis (PPV-MF) or post-essential thrombocythemia-myelofibrosis (PET-MF)
First Author: Ruben Mesa, M.D., Deputy Director, Mayo Clinic Cancer Center, Chair of the Division of Hematology & Medical Oncology, Mayo Clinic Cancer Center, Scottsdale, AZ
Date/Time: Saturday, May 30 at 2:37 p.m. CT
Session: Leukemia, Myelodysplasia, and Transplantation
Abstract #: LBA7006
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Source: press release, 4/22/15. http://investors.ctibiopharma.com/phoenix.zhtml?c=92775&p=irol-newsArtic...

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CTI BioPharma Completes Recruitment in PERSIST-1 Pivotal Phase 3 Trial of Pacritinib for Patients with Myelofibrosis
-Top-line Results Expected in Early 2015-
-CTI Expects to Receive $20 Million Development Milestone Payment from Baxter-
SEATTLE, July 1, 2014 /PRNewswire/ -- CTI BioPharma Corp. (CTI or the Company) (NASDAQ and MTA: CTIC) announced today that it has completed recruitment in the PERSIST-1 pivotal Phase 3 clinical trial of pacritinib, a novel oral JAK2/FLT3 inhibitor that is being evaluated for the treatment of myelofibrosis. Under the development and commercialization agreement for pacritinib with Baxter International, Inc. (Baxter), CTI expects to receive a $20 million development milestone payment in connection with the first treatment dosing of the last patient enrolled in PERSIST-1. CTI expects to achieve this milestone and receive payment by mid-third quarter 2014.

"The PERSIST-1 clinical trial evaluating pacritinib, a novel JAK2/FLT3 inhibitor, is the most inclusive study of myelofibrosis patients seen in routine clinical practice to date," said Claire Harrison, M.D., Consultant Hematologist, Guy's and St. Thomas' NHS Foundation Trust, Guy's Hospital, London, United Kingdom and one of the principal investigators for PERSIST-1. "Previous randomized clinical trials have excluded patients with low platelet counts (below 50,000-100,000 per microliter (uL)) despite almost 30 percent of all myelofibrosis patients having disease-related thrombocytopenia. Currently available JAK1/JAK2 inhibitors are associated with treatment-emergent myelosuppression, including thrombocytopenia as a side effect of their therapy requiring reduced doses and sometimes early cessation of treatment when used in patients with disease-related thrombocytopenia. These patients represent an unmet medical need; a non-myelosuppressive JAK2 inhibitor would represent a significant advancement in the treatment of this chronic disease."

The PERSIST-1 trial is the first of two Phase 3 trials in the pacritinib development program in myelofibrosis. The second Phase 3 trial, PERSIST-2, is currently evaluating pacritinib for the treatment of patients with low platelet counts compared to best available therapy, including approved JAK2 inhibitors at their recommended dose and schedule for myelofibrosis patients with thrombocytopenia. The two clinical trials are intended to support an anticipated New Drug Application (NDA) regulatory submission in the U.S. in late 2015, followed by an anticipated Marketing Authorization Application (MAA) in Europe in 2016.

"Completing recruitment in the PERSIST-1 trial is a significant milestone in our development program for pacritinib, and we look forward to reporting top-line results in early 2015," said James A. Bianco, M.D., President and CEO of CTI BioPharma. "The high physician and patient interest in participating in this trial underscores the need for new, effective and less toxic treatment options for patients with myelofibrosis."

The PERSIST-1 trial was designed to enroll approximately 320 patients and is a randomized, open-label, multicenter trial comparing the efficacy and safety of pacritinib with that of best available therapy, other than JAK inhibitors, in patients with primary myelofibrosis, post-polycythemia vera myelofibrosis or post-essential thrombocythemia myelofibrosis, without exclusion for low platelet counts. The primary endpoint is the percentage of patients achieving a greater than or equal to 35 percent reduction in spleen volume measured by MRI or CT at 24 weeks of treatment.
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Source: press release, 7/01/14. http://investors.ctibiopharma.com/phoenix.zhtml?c=92775&p=irol-newsArtic...

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James A. Bianco, M.D., CEO, Director, commented on the progress being made for enrollment of the PERSIST-1 trial. He stated, "We are on track to complete patient accrual in the PERSIST-1 Phase III trial of Pacritinib, early 2014, and initiate the second Phase III trial for Pacritinib in the second half of this year."
Source: Q1 2013 earnings conference call, 5/02/13.

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Steven E. Benner, M.D., M.H.S., EVP, CMO, commented on the progress being made with enrollment of the PERSIST-1 trial. He stated, "The trial plans to enroll at approximately 80 clinical sites in Europe, Australia and the United States. We've recently held investigative meetings in Australia and Europe. I can tell you that the sites are enthusiastic about the trial and are identifying patients. We anticipate fully enrolling the trial within 12 to 14 months."
Source: Q4 2012 earnings conference call, 2/28/12.

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Cell Therapeutics Begins Enrollment in Phase 3 PERSIST-1 Trial of Pacritinib for the Treatment of Myelofibrosis

SEATTLE, Jan. 9, 2013 /PRNewswire/ -- Cell Therapeutics, Inc. (CTI) (Nasdaq and MTA: CTIC) today announced that the Company has initiated clinical trial sites and began enrolling patients in a Phase 3 clinical trial, known as PERSIST-1 or PAC325, for pacritinib, CTI's investigational JAK2 inhibitor, which is being evaluated for the treatment of patients with myelofibrosis. The randomized trial is expected to enroll 270 patients and will evaluate the safety and efficacy of pacritinib compared to best available therapy, excluding JAK inhibitors, in patients with myelofibrosis. Pacritinib is a selective oral JAK2 inhibitor that demonstrated meaningful clinical benefits and good tolerability in myelofibrosis patients in Phase 2 clinical trials, without apparent drug-related thrombocytopenia or anemia. Myelofibrosis patients will be enrolled in the PERSIST-1 trial without exclusion for low platelet counts.
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Source: press release, 1/09/13. http://investors.celltherapeutics.com/phoenix.zhtml?c=92775&p=irol-newsA...

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Steven E. Benner., M.D., M.H.S. EVP, Chief Medical Officer, gave guidance for the Pacritinib for Myelofibrosis program. He stated, "We anticipate that the first patient will be enrolled before year end. Based on a feasibility study, we estimate enrollment will take 12 months after enrollment is initiated. Our second study of Pacritinib, we anticipated initiating in the second quarter of 2013 after having received feedback from both the FDA and EMA. As currently planned, this study will target patients with Myelofibrosis, with study entry of platelet counts of less than or equal to 100,000, which is a population not included in the COMFORT trials."
Source: Q3 2012 earnings conference call, 11/01/12.

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Cell Therapeutics Completes Acquisition of Pacritinib a Highly Selective JAK2 Inhibitor
Phase 3 Study in Patients with Myelofibrosis and Low Platelet Counts Targeted to Start in Q4-2012

SEATTLE, June 4, 2012 /PRNewswire/ -- Cell Therapeutics, Inc. ("CTI") (NASDAQ and MTA: CTIC), announced today that it has completed its acquisition gaining world-wide rights to S*BIO Pte Ltd.'s ("S*BIO") pacritinib. Pacritinib is a highly selective oral JAK2 (Janus Associated Kinase 2) inhibitor that demonstrated encouraging clinical activity in phase 1 and 2 clinical studies of patients with primary myelofibrosis ("MF") and MF secondary to other myeloproliferative neoplasms ("MPN"). Pacritinib has orphan drug designation in the United States and Europe for myelofibrosis.
Source: press release, 6/04/12. http://investors.celltherapeutics.com/phoenix.zhtml?c=92775&p=irol-newsA...

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A Randomized Controlled Phase 3 Study of Oral Pacritinib Versus Best Available Therapy in Patients With Primary Myelofibrosis, Post-Polycythemia Vera Myelofibrosis, or Post-Essential Thrombocythemia Myelofibrosis
Estimated Enrollment: 270
Study Start Date: December 2012
Estimated Study Completion Date: August 2017
Estimated Primary Completion Date: August 2014 (Final data collection date for primary outcome measure)
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Source: clinical trials.gov. http://clinicaltrials.gov/ct2/show/NCT01773187?term=pacritinib&rank=1

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Compound/DeviceSpecialtyIndicationCompound ClassTarget
PacritinibOncology HematologicMyelofibrosisJanus-associated Kinase InhibitorsJanus-associated Kinase 2 (JAK2)

Mechanism of action: Pacritinib is an orally bioavailable inhibitor of Janus kinase 2 (JAK2) and the JAK2 mutant JAK2V617F with potential antineoplastic activity. Pacritinib competes with JAK2 for ATP binding, which may result in inhibition of JAK2 activation, inhibition of the JAK-STAT signaling pathway, and so caspase-dependent apoptosis. JAK2 is the most common mutated gene in bcr-abl-negative myeloproliferative disorders; the JAK2V617F gain-of-function mutation involves a valine-to-phenylalanine modification at position 617. The JAK-STAT signaling pathway is a major mediator of cytokine activity.

Phase of Development: III

Event Type: Data: Phase III trial results

Dates: 2015-05-30

Results:

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Long-Term Follow Up Data For Pacritinib Presented At 2016 ASCO Annual Meeting

-72 week follow up of patients with myelofibrosis, including patients with baseline thrombocytopenia, show pacritinib treatment led to durable reductions in spleen volume and symptom burden-

SEATTLE, June 7, 2016 /PRNewswire/ -- CTI BioPharma Corp. (CTI BioPharma) (NASDAQ and MTA:CTIC) today announced long-term safety and efficacy results from the pivotal Phase 3 PERSIST-1 trial evaluating pacritinib versus best available therapy, excluding treatment with JAK2 inhibitors (BAT), in patients with myelofibrosis. As previously reported, the PERSIST-1 trial met its primary endpoint in the intent-to-treat population with statistically significant reduction in spleen volume when compared to patients receiving BAT. The results represent an update on the efficacy and safety for all patients regardless of their initial platelet count, including patients with very low platelet counts at study entry, a condition known as severe or life-threatening thrombocytopenia. The most frequently occurring adverse events with pacritinib were gastrointestinal events and incidence decreased over time. For patients crossing over to receive pacritinib treatment (84 percent of BAT patients), less than 5 percent of patients had diarrhea with only one patient experiencing grade 3. Patients in the BAT arm that crossed over to receive pacritinib treatment had a similar rate of events as patients initially randomized to BAT or pacritinib. A planned analysis of the study up to 72 weeks demonstrated treatment with pacritinib led to durable reductions in spleen volume and symptom burden, two key measures of disease control, in patients with myelofibrosis, including patients with low platelets at baseline (less than 50,000 per microliter and less than 100,000 per microliter). Patients who crossed over to pacritinib from BAT experienced similar reductions in spleen volume and symptom burden as patients initially randomized to pacritinib, including patients with low platelets. Pacritinib is an investigational oral kinase inhibitor with specificity for JAK2, FLT3, IRAK1 and CSF1R, which are kinases found to be involved in the growth and spread of myelofibrosis and other blood-related cancers.

"From the time a patient is diagnosed with myelofibrosis, the incidence of disease-related thrombocytopenia increases with time," stated Claire Harrison, M.D., Consultant Hematologist, Guy's and St. Thomas' NHS Foundation Trust, Guy's Hospital, London, United Kingdom and one of the principal investigators for PERSIST-1. "Patients with thrombocytopenia have significantly greater symptom burden, distinct clinical characteristics and shorter overall survival. There is currently a significant unmet need for patients with myelofibrosis who are unable to tolerate or control their disease on other treatments due to low platelet counts.

Dr. Harrison added, "It is encouraging to see that patients with baseline thrombocytopenia who were treated with pacritinib, had stable mean platelet counts and hemoglobin levels through the end of treatment, and that some patients with very low platelets increased their platelet counts while receiving pacritinib treatment. In this intermediate- to high-risk patient group, pacritinib was generally well tolerated."

Myelofibrosis is a rare, but serious and life-threatening chronic leukemia that disrupts the normal production of blood cells and results in scarring of the bone marrow, limiting the ability to produce new blood cells and prompting the spleen and other organs to take over this function. The disease often leads to an enlarged spleen and lower than normal counts of blood cells – including red blood cells and platelets, which are essential for blood clotting. Frequent causes of death among patients with myelofibrosis include leukemic transformation (31 percent), disease progression (18 percent), and thrombosis and cardiovascular complications (13 percent) 1.

Below are highlights presented at the 52nd Annual Meeting of the American Society of Clinical Oncology (ASCO) being held June 3–June 7, 2016 in Chicago, Ill. The poster presentations are available at www.ctibiopharma.com.

Highlights of Data Presented

Mesa, R, et al. Pacritinib (PAC) vs. best available therapy (BAT) in myelofibrosis (MF): 72 week follow-up of the phase III PERSIST-1 trial. Abstract #7065
In this poster, an update on efficacy and safety of the PERSIST-1 clinical trial up to Week 72 is provided for all patients in the study (pacritinib n=220; BAT n=107), regardless of platelet count. Ninety patients (84 percent) randomized to BAT crossed over to receive pacritinib at a median of 27.2 weeks.

Responses to pacritinib were durable and rates of 35 percent or greater spleen volume reduction (SVR) were maintained from Weeks 24 to 72 (25 percent vs. 24 percent). Patients who crossed over to receive pacritinib achieved similar responses to those receiving initial treatment with pacritinib. Overall survival, although not a primary or secondary endpoint of the trial, did not show a statistically significant difference between the pacritinib and BAT arms. This was primarily due to an imbalance between the two arms, with higher risk patients in the pacritinib arm vs. BAT. The result was potentially confounded by a high percentage of patients who crossed over at Week 24 to receive pacritinib therapy. Pacritinib-treated patients who achieved SVR greater or equal to 20 percent had statistically significant longer overall survival vs. patients who did not achieve SVR at this level.

The most frequently occurring adverse events with pacritinib were gastrointestinal events and the incidence decreased over time. Incidence of grade 3/4 treatment-emergent diarrhea with initial pacritinib treatment was highest in Weeks 1-8 (3 percent) and decreased in Weeks 8-16 (1.4 percent), Weeks 16-24 (1.5 percent) and in the final period analyzed, Weeks 64-72 (0.9 percent). Up to 24 weeks, prior to the majority of patients in the BAT arm crossing over, there was no statistically significant difference in the incidence of cardiac and bleeding adverse events between the pacritinib and BAT arms; following crossover to pacritinib, BAT patients had a similar rate of all grades of adverse events. The incidence of all grade cardiac AEs was similar between pacritinib and BAT arms between Weeks 1 and 24; incidence of cardiac AEs was greater for pacritinib between Weeks 24 and 72 vs. BAT, but was low overall (5 percent or less at any time). Among all patients, incidence of grade 3/4 bleeding events was 3 percent or less during any 8-week time interval.

Harrison, C, et al. Pacritinib (PAC) vs. best available therapy (BAT) in myelofibrosis (MF): Outcomes in patients (pts) with baseline (BL) thrombocytopenia. Abstract #7011
This analysis examines outcomes up to 72 weeks among patients in the PERSIST-1 trial with baseline platelets less than 100,000 per microliter treated with pacritinib vs. BAT (pacritinib, n=72; BAT, n=34). For patients receiving pacritinib, a median duration of spleen volume reduction (SVR) of 35 percent or greater was 48 weeks for patients with baseline platelets less than 50,000 per microliter and 57 weeks for patients with baseline platelets less than 100,000 per microliter. For BAT-treated patients who crossed over to receive pacritinib before or after Week 24, duration of SVR was 73 weeks for patients with baseline platelets less than 50,000 per microliter and 46 weeks for patients with baseline platelets less than 100,000 per microliter. At Week 36, 46 percent (6/13) of evaluable pacritinib-treated patients with baseline platelets less than 50,000 per microliter and 48 percent (12/28) of evaluable pacritinib-treated patients with baseline platelets less than 100,000 per microliter achieved 50 percent or greater reduction in Total Symptom Score (TSS). This is an increase from 32 percent and 42 percent, respectively, at Week 24.

Patients treated with pacritinib had stable mean platelet counts and hemoglobin levels through Week 72 and increased platelet counts in patients with platelets less than 50,000 per microliter. At 24 weeks, bleeding events occurred at a similar rate in pacritinib-and BAT-treated patients; following crossover to pacritinib, BAT patients had a similar rate of events. Among patients with baseline thrombocytopenia treated with pacritinib, mean hemoglobin levels remained stable through Week 72. Due to BAT crossover to pacritinib at Week 24, there were no evaluable patients with baseline thrombocytopenia in the BAT arm beyond Week 36. These data suggest pacritinib treatment led to durable reductions in spleen volume and symptom burden.

Harrison, C, et al. Outcomes in patients with myelofibrosis and RBC-transfusion dependence in the phase III PERSIST-1 study of pacritinib vs. best available therapy. Abstract #7066
In this poster, pacritinib-treated patients demonstrated clinically meaningful reductions in spleen volume independent of RBC-transfusion independence (RBC-TI). Among pacritinib-treated patients, 16 percent (36/220) were RBC-transfusion dependent (RBC-TD) at baseline. Eighteen (18) patients were RBC-TD at the time of crossover.

Twenty-two percent (12/54) of RBC-TD patients treated with pacritinib either from study start or after crossover achieved RBC-TI during the course of the study. During the course of the study, 25 percent (9/36) of RBC-TD patients treated with pacritinib at the start of the study achieved RBC-TI vs. 0 percent (0/16) patients treated with BAT (p=0.043). Seventeen percent (3/18 patients) of RBC-TD patients at the time of crossover achieved RBC-TI during the crossover period. Pacritinib treatment was associated with improved patient outcomes for those with baseline RBC-TD.

Mesa, R, et al. Pacritinib (PAC) vs. best available therapy (BAT) in myelofibrosis (MF): Long-term follow-up of patient-reported outcomes (PROs) in the phase III PERSIST-1 trial. Abstract #7067
Patient Reported Outcomes (PRO) data at 24 weeks was previously reported and the poster presented today provided an update at Week 48. The Myeloproliferative Neoplasm Symptom Assessment Form (MPN-SAF) is a PRO assessment tool designed to measure myelofibrosis-related symptom burden resulting in a TSS based on how the patient feels or functions in relation to six common symptoms. The proportion of patients achieving a TSS reduction of 50 percent or greater improved from Weeks 24 to 48 and was greater than observed with BAT showing that reduction in symptoms continued to increase over time. In patients who crossed over from BAT to pacritinib, reductions in TSS improved from Week 24 to Week 36. Mean percentage reductions in common symptoms measured in both MPN-SAF versions at Week 48 were greater than those observed at Week 24 among patients treated with pacritinib.
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Source: press release, 6/07/16. http://investors.ctibiopharma.com/phoenix.zhtml?c=92775&p=irol-newsArtic...

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Analysis of Pivotal Phase 3 Patient Outcomes by Subgroups Shows Treatment with Pacritinib Resulted in Consistent Rates of Reduction in Spleen Volume and Symptom Burden

Results support the effectiveness of pacritinib across intermediate or high-risk myelofibrosis patient subgroups analyzed

ORLANDO, Fla., Dec. 5, 2015 /PRNewswire/ -- CTI BioPharma Corp. (CTI BioPharma) (NASDAQ and MTA: CTIC) and Baxalta Incorporated (Baxalta) (NYSE: BXLT) today announced results from a new analysis of the pivotal Phase 3 trial, PERSIST-1, evaluating pacritinib versus best available therapy, excluding treatment with JAK2 inhibitors (BAT), in patients with myelofibrosis. Data examining patient outcomes across baseline demographic factors that are associated with prognosis – including age, baseline hemoglobin, baseline platelet count, ECOG status, JAK2 mutation status and red blood cell transfusion dependency – showed that treatment with pacritinib resulted in consistent rates of spleen volume reduction and control of disease-related symptoms across all intermediate or high-risk myelofibrosis subgroups. These findings were presented by Alessandro M. Vannucchi, M.D., associate professor of Hematology, University of Florence, Italy, during an oral presentation at the 57th American Society of Hematology (ASH 2015) Annual Meeting & Exposition in Orlando (Abstract #58).

Pacritinib is an investigational oral multikinase inhibitor with specificity for JAK2, FLT3, IRAK1 and CSF1R, which are kinases found to be involved in the growth and spread of myelofibrosis and other blood-related cancers such as acute myeloid leukemia (AML).

"Reducing the burden of myelofibrosis-related symptoms is an important goal of treatment. However, for patients diagnosed with this rare blood cancer, there are limited therapeutic options – a gap that is even more significant for patients with low platelet counts," said Prof. Vannucchi. "These data presented at ASH 2015 are important and clinically meaningful as they demonstrate pacritinib's potential to achieve treatment goals across intermediate or high-risk patients with myelofibrosis, regardless of baseline characteristics including starting platelet count."

Myelofibrosis is a rare blood cancer associated with significantly reduced quality of life and shortened survival. Most patients with the disease 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.

"The results from this analysis add to the growing body of data for pacritinib suggesting it is a unique JAK inhibitor with a differentiated efficacy and safety profile that is not limited by the baseline characteristics of patients with myelofibrosis," said James Bianco, M.D., President and Chief Executive Officer, CTI BioPharma. "We believe pacritinib has the potential to fill a gap that exists for many patients whose lives are profoundly impacted by myelofibrosis, particularly those patients with low platelet counts."

"We are developing pacritinib with particular focus on targeting the underlying biology of myelofibrosis to improve the treatment landscape for patients with this underserved, progressive disease, including those in intermediate and high-risk subgroups," said David Meek, Executive Vice President and President, Oncology at Baxalta. "We look forward to working with worldwide regulatory authorities to advance treatment options for all patients with myelofibrosis as we begin our registration submissions for pacritinib in the coming months."
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Source: press release, 12/05/15. http://investors.ctibiopharma.com/phoenix.zhtml?c=92775&p=irol-newsArtic...

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

Significant Improvements in Symptom Score were Reported with Pacritinib, Across All Measured Symptoms, Compared to Best Available Therapy

VIENNA, June 12, 2015 /PRNewswire/ -- CTI BioPharma Corp. (CTI BioPharma) (NASDAQ and MTA: CTIC) and Baxter International's BioScience business (NYSE: BAX) today announced 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. As recently reported at the American Society of Clinical Oncology (ASCO) 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) 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 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.

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
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, 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, 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.
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Source: press release, 6/12/15. http://investors.ctibiopharma.com/phoenix.zhtml?c=92775&p=irol-newsArtic...

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CTI BioPharma And Baxter Announce Positive Top-Line Results From Phase 3 Persist-1 Trial Of Pacritinib For Patients With Myelofibrosis
Trial Meets Primary Endpoint in Intent-to-Treat Population
Oral Pacritinib is the First Agent with Clinical Data Supporting its Use in Treating Patients with Myelofibrosis Irrespective of Their Platelet Count, Including Severe and Life-Threatening Thrombocytopenia (low platelets)
CTI BioPharma to Host Conference Call Today at 8:00 am EDT
SEATTLE and DEERFIELD, Ill., March 9, 2015 /PRNewswire/ -- CTI BioPharma Corp. (CTI BioPharma) (NASDAQ and MTA: CTIC) and Baxter International Inc. (NYSE:BAX) today announced positive top-line results for the primary endpoint from PERSIST-1, the randomized, controlled Phase 3 registration clinical trial examining pacritinib, a next generation oral JAK2/FLT3 multikinase inhibitor, for the treatment of patients with primary or secondary myelofibrosis. The PERSIST-1 trial met its primary endpoint in the intent-to-treat population with statistically significant activity observed in patients irrespective of their initial platelet count, including patients with very low platelet counts at study entry, a condition known as severe or life-threatening thrombocytopenia.

The primary endpoint of the trial was the proportion of patients achieving a 35 percent or greater reduction in spleen volume from baseline to Week 24 as measured by magnetic resonance imaging (MRI) or computerized tomography (CT) when compared with physician-specified best available therapy (BAT), excluding treatment with JAK2 inhibitors. The PERSIST-1 trial demonstrated that pacritinib treatment provided a clinically and statistically significant response rate (p = 0.0003) in spleen volume reduction in patients with myelofibrosis when compared to BAT. Importantly, the trial results also demonstrated a significant difference among patients with platelet counts of less than 100,000 per microliter and less than 50,000 per microliter, both subgroups that were stratified at randomization. The magnitude of treatment effect was consistent with previously reported Phase 2 results, with the greatest reduction observed among the sickest patients (platelet counts <50,000 per microliter). Among 50 patients who were red blood cell (RBC) transfusion dependent at study entry (≥ 6 units of RBC over 90 days pre entry), pacritinib therapy resulted in a clinically meaningful percentage of patients becoming transfusion independent compared to BAT. Seventy-nine percent (79%) of patients in the BAT arm of the study crossed over to pacritinib therapy.

The safety profile in the PERSIST-1 trial was consistent with prior Phase 2 trials. While the most common treatment emergent adverse events were diarrhea, nausea and vomiting, the incidence of grade 3 events was lower than observed in Phase 2 trials. No grade 4 gastrointestinal adverse events were reported. Three patients discontinued therapy and nine patients required dose reduction for diarrhea. Preliminary analysis suggests that very few patients discontinued treatment while on pacritinib or required a dose reduction due to treatment-related anemia or thrombocytopenia. Additional data from ongoing analyses along with top-line results from PERSIST-1 will be submitted for presentation at an upcoming scientific meeting.

"Despite the introduction of JAK2 inhibitors as effective therapies for patients with myelofibrosis, there remains a treatment gap for patients with disease-related or treatment emergent thrombocytopenia. The currently approved drug may require dose titration to less effective doses in this patient population, thus limiting our ability to effectively treat them. Results from the PERSIST-1 randomized trial demonstrate that pacritinib could address this unmet medical need," stated Claire Harrison, M.D., Consultant Hematologist, Guy's and St. Thomas' NHS Foundation Trust, Guy's Hospital, London, United Kingdom and one of the principal investigators for PERSIST-1. "It is encouraging to see that patients were able to receive therapeutic doses of pacritinib over a long period of time irrespective of their baseline platelet or red blood cell count while having therapeutic benefit in reduction in spleen volume and disease-related symptoms and improvement in transfusion dependency."

"PERSIST-1 is the first randomized Phase 3 trial investigating the potential benefit of a JAK2 inhibitor across a patient population with myelofibrosis that is representative of patients that healthcare providers see and treat in clinical practice," said James A. Bianco, M.D., CTI BioPharma's President and CEO. "We are excited by the clinical profile demonstrated in this randomized trial with respect to benefit–risk especially for a segment of MF patients excluded from other randomized trials with JAK2 inhibitors. We are grateful for the support and commitment of the investigators, our steering committee and, most importantly, all the patients who participated in PERSIST-1. We look forward to building on the progress we have made thus far."

"These positive top-line results illustrate the potential of this investigational treatment to become a valuable new treatment option for this challenging disease. Pacritinib is an important component of Baxter's growing oncology portfolio, and we look forward to partnering with CTI BioPharma to share these results with physicians and discussing next steps with regulatory agencies," said David Meek, Head of Oncology at Baxter BioScience.
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Source: press release, 3/09/15. http://investors.ctibiopharma.com/phoenix.zhtml?c=92775&p=irol-newsArtic...

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