VIENNA, June 12, 2015 /PRNewswire/ — CTI BioPharma Corp. (CTI BioPharma) (NASDAQ and MTA: CTIC) and Baxter International’s BioScience business (NYSE: BAX) today announced Brand-new patient-reported outcome (PRO) data for pacritinib – an investigational oral multikinase inhibitor along 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 prove to a substantial reduction in the Total Symptom Score (TSS) (the proportion of patients along with a 50 percent or higher reduction in TSS from baseline to Week 24), and in each specific common disease-related symptom, from baseline to Week 24, in patients treated along with pacritinib compared to ideal available therapy (exclusive of a JAK inhibitor) (BAT). These PROs, as well as others quality of life measures, will certainly 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 likewise picked 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 higher from baseline to Week 24 as measured by MRI/CT scan.
Myelofibrosis is a rare blood cancer associated along with significantly reasonable quality of life and shortened survival. As the disease progresses, the physique slows production of essential blood cells and within one year of diagnosis the incidence of disease-related thrombocytopenia (quite reasonable blood platelet counts), severe anemia, and red blood cell transfusion requirements boosts significantly. Among others complications, most patients along with myelofibrosis present along with enlarged spleens (splenomegaly), as well as lots of others potentially devastating physical symptoms such as abdominal discomfort, bone pain, feeling full after consuming little, severe itching, night sweats, and extreme fatigue.
“Patient-reported outcomes are an essential measure for understanding the potential benefit of a therapy on patients’ lives – particularly for a disease such as myelofibrosis where the symptoms have actually such a tremendous impact on the quality of patients’ day-to-day lives,” stated James A. Bianco, M.D., President and CEO of CTI BioPharma. “These Brand-new data from the PERSIST-1 study further support our belief, not only in the activity of pacritinib, however likewise the potential to positively impact patients’ day-to-day lives by relieving the symptoms that accompany myelofibrosis.”
“The PERSIST-1 trial has actually continued to generate positive and essential 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 job to realize the full potential of this investigational compound to advice patients along 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 along 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 higher reduction from baseline to Week 24 by MRI/CT scan) in the intent-to-treat (ITT) population; these results included patients along with severe or life-threatening thrombocytopenia. The study likewise measured patient-reported outcomes (PROs), the proportion of patients along with a 50 percent or higher reduction in TSS from baseline to Week 24, which have actually become essential for approval of Brand-new therapies and was one of the secondary endpoints of the study. As previously reported, patients treated along with pacritinib suffered higher 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)
As quickly as using the MPN-SAF TSS and MPN-SAF TSS 2.0, each of the 6 common disease-related symptoms from the TSS results showed improvements in abdominal discomfort (46 percent improvement along with pacritinib vs no improvement along with BAT); bone pain (32 percent improvement along with pacritinib vs 8 percent improvement along with BAT); feeling of early fullness (45 percent improvement along with pacritinib vs 1 percent worsening along with BAT); itching (48.5 percent improvement along with pacritinib vs 10 percent improvement along with BAT); night sweats (69.5 percent improvement along with pacritinib vs no improvement along with BAT); and fatigue (27.5 percent improvement along with pacritinib vs 4 percent worsening along with BAT). MPN-SAF TSS and MPN-SAF TSS 2.0 are individual sets of questions patients answer day-to-day (via electronic diary) and which are based on a questionnaire originally created by Ruben A. Mesa, M.D., Deputy Director of the Mayo Clinic Cancer Focus in Scottsdale, Arizona, USA.
Patient Global Impression of Modification (PGIC)
Based on the PGIC assessment – which measures a patient’s assessment of overall health on a 7-point scale ranging from “quite much worse” to “quite much improved” – approximately 80 percent of evaluable patients treated along with pacritinib rated their condition as improved compared to approximately 20 percent along with BAT.
European Organization for Research and Treatment of Cancer Quality-of-Life 30 Questionnaire (EORTC QLQ-C30)
A higher improvement was likewise reported by evaluable patients treated along 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 edge events occurring along with pacritinib within 24 weeks, of any grade, were mild to moderate diarrhea (53.2 percent vs 12.3 percent along with BAT), nausea (26.8 percent vs 6.6 percent along with BAT), anemia (22.3 percent vs 19.8 percent along with BAT), thrombocytopenia (16.8 percent vs 13.2 percent along with BAT), and vomiting (15.9 percent vs 5.7 percent along with BAT). Of the patients treated along with pacritinib, 3 discontinued therapy and 13 patients needed dose interruption (standard one week) for diarrhea. Patients received a day-to-day full dose of pacritinib over the duration of treatment. Gastrointestinal symptoms typically lasted for approximately one week and few patients discontinued treatment as a result of adverse effects. There were no Grade 4 gastrointestinal events reported.
Pacritinib is an investigational oral multikinase inhibitor along 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 actually been shown to be directly related to the development of a variety of blood-related cancers, including myeloproliferative neoplasms, leukemia, and lymphoma.
Myelofibrosis is a serious and life-threatening chronic blood cancer caused by the accumulation of malignant bone marrow cells that causes an inflammatory response and scars the bone marrow. The replacement of bone marrow along with scar tissue limits its ability to develop 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 individuals living along 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 actually a median age of 64 at the time of diagnosis2 and is a progressive disease along with approximately 20 percent of patients eventually making acute myeloid leukemia.3 The median survival for high-risk patients is much less compared to one and a half years; median survival for myelofibrosis patients overall is approximately 6 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 along with myelofibrosis. PERSIST-1 is a randomized (2:1), open-label, multinational Phase 3 clinical trial comparing the efficacy and safety of pacritinib along with that of BAT, in 327 enrolled patients along with primary and secondary myelofibrosis (PMF), post-polycythemia vera myelofibrosis (PPV-MF), or post-necessary thrombocythemia myelofibrosis (PET-MF), devoid of exclusion for reasonable 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 along with myelofibrosis whose platelet counts are much less compared to 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 in to a global license agreement in November 2013 to produce and commercialize pacritinib. CTI BioPharma and Baxter will certainly jointly commercialize pacritinib in the U.S. while Baxter has actually exclusive commercialization rights for all indications outside the U.S.
About CTI BioPharma Corp.
CTI BioPharma Corp. (NASDAQ and MTA: CTIC) is a biopharmaceutical company focused on the acquisition, development, and commercialization of novel targeted therapies covering a spectrum of blood-related cancers that offer a unique benefit to patients and healthcare providers. CTI BioPharma has actually a commercial presence in Europe and a late-stage development pipeline, including pacritinib, CTI BioPharma’s lead product candidate, which is currently being studied in a Phase 3 program for the treatment of patients along with myelofibrosis. CTI BioPharma is headquartered in Seattle, Washington, along with offices in London and Milan under the name CTI Life Sciences Limited. For additional article and to sign up for email alerts and grab RSS feeds, please visit www.ctibiopharma.com.
About Baxter Global Inc.
Baxter Global Inc., through its subsidiaries, develops, manufactures and markets products that save and sustain the lives of individuals along with hemophilia, immune disorders, cancer, infectious diseases, kidney disease, trauma and others chronic and acute medical conditions. As a global, diversified healthcare company, Baxter applies a unique combination of expertise in medical devices, pharmaceuticals and biotechnology to produce products that advance patient care worldwide.
Forward Looking Statements
This press release includes forward-looking statements related to pacritinib and related clinical trials conducted pursuant to a collaboration between Baxter Global Inc. and CTI BioPharma Corp., as well as statements concerning the planned separation of Baxter’s biopharmaceutical and medical products businesses, which are within the meaning of the Safe Harbor provisions of the Private Securities Litigation Reform Act of 1995. Such statements are subject to a number of risks and uncertainties, the outcome of which could materially and/or adversely affect actual future results and the trading price of the issuers’ securities. Such statements include, however are not limited to, statements concerning expectations along with respect to the potential therapeutic utility of pacritinib, the ability of the PERSIST-1 and PERSIST-2 trials to support a potential regulatory submission, the anticipated completion of enrollment, the ability of pacritinib to meet unmet medical requires and future regulatory, development and commercialization plans. Investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date of this release and are based on assumptions Concerning lots of essential factors and article currently available to us to the extent we have actually thus far had an opportunity to evaluate such article in light of all surrounding facts, circumstances, guidance and analyses. A number of results and uncertainties could cause actual results to differ materially from those in the forward-looking statements: satisfaction of regulatory and others requirements; clinical trial results; changes in laws and regulations; product quality, product efficacy, study protocol, data integrity or patient safety issues; product development risks; and others risks identified in each issuer’s most recent filings on Form 10-K and others Securities and Exchange Commission filings as well as the Form 10 filed by Baxalta Incorporated. Neither Baxter nor CTI BioPharma undertakes to update its forward-looking statements.
1. MPN Research Foundation website, www.mpnresearchfoundation.org.
2. Based on Mesa R, ASH 2012 poster.
3. Cervantes F, et al., Brand-new prognostic scoring system for primary myelofibrosis based on a study of the Global Working Group for Myelofibrosis Research and Treatment. Blood. 2009; 113:2895-2901.
4. Vannucchi, A. Management of Myelofibrosis. ASH Education Book. 2011; 1:222-230.
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