CTI BioPharma Announces Phase 3 Data from the PERSIST-2 Trial of Pacritinib to be Presented in Late-Breaking Session at ASH Annual Meeting

Myelofibrosis is associated with significantly reduced quality of life and shortened survival. Spleen enlargement (splenomegaly) is a common and debilitating symptom of myelofibrosis. 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 increase significantly.

Details of the PERSIST-2 presentation, two additional poster presentations regarding pacritinib, as well as poster presentations highlighting PIXUVRI^® (pixantrone) and tosedostat, are below. Full abstracts can be accessed on the ASH website at www.hematology.org.

PERSIST-2 Oral Presentation

Results of the PERSIST-2 Phase 3 Study of Pacritinib (PAC) Versus Best Available Therapy (BAT), Including Ruxolitinib (RUX), in Patients with Myelofibrosis (MF) and Platelet Counts Less Than 100,000/µL
First Author: John Mascarenhas, M.D., Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai, New York, NY
Date/Time: Tuesday, December 6 at 8:30 a.m. PT
Location: Hall AB
Oral Session: Late-Breaking Abstracts
Abstract #LBA-5

Poster Presentations

Pacritinib

Relationship of JAK2^V617F Allelic Burden (AB) to Demographics, Disease Characteristics, and Response to Therapy in PERSIST-1, A Randomized Phase 3 Study of Pacritinib (PAC) Versus Best Available Therapy (BAT) in Patients (Pts) with Primary and Secondary Myelofibrosis (MF)First Author: Alessandro M. Vannucchi, M.D., CRIMM, AOU Careggi, University of Florence, Florence, Italy
Date/Time: Sunday, December 4 at 6:00-8:00 p.m. PT
Location: Hall GH
Poster Session: 634. Myeloproliferative Syndromes: Clinical: Poster II
Abstract #3131

Pacritinib Targets IRAK1 and Shows Synergy with HDAC and BET Inhibitors in Acute Myeloid Leukemia
First Author: Anupriva Agarwal, Ph.D., Knight Cancer Institute, Division of Hematology and Medical Oncology, Oregon Health & Science University, Portland, OR
Date/Time:  Sunday, December 4 at 6:00-8:00 p.m. PT
Location: Hall GH
Poster Session: 802. Chemical Biology and Experimental Therapeutics: Poster II
Abstract #3514

Pixantrone

The Combination of Pixantrone, Etoposide, Bendamustine and, in CD20+ Tumors, Rituximab (PREBEN) Shows Promising Feasibility/Efficacy in Heavily Pre-Treated Aggressive Lymphomas of B- And T-Cell Phenotype – Results of the Pre-Trial Experience Leading to a Nordic Phase 1/2 Study (the PREBEN Trial)
First Author: Michael R. Clausen, M.D., Hematology, Aarhus University Hospital, Aarhus, Denmark
Date/Time: Saturday, December 3 at 5:30-7:30 pm PT
Location: Hall GH
Poster Session: 623. Mantle Cell, Follicular, and Other Indolent B-Cell Lymphoma – Clinical Studies: Poster I
Abstract #1782

Tosedostat

A Phase 1 Dose-Escalation Study of the Class 1 Selective Histone Deacetylase Inhibitor CHR-3996 in Combination with Tosedostat for Patients with Relapsed, Refractory Multiple Myeloma: Results of the MUK Three Trial
First Author: Rakesh Popat, MBBS, Ph.D., University College London Hospitals NHS Foundation Trust, London, UK
Date/Time: Sunday, December 4 at 6:00-8:00 p.m. PT
Location: Hall GH
Poster Session: 653. Myeloma: Therapy, excluding Transplantation: Poster II
Abstract #3321

About the Phase 3 Development Program of Pacritinib

Pacritinib was evaluated in two Phase 3 clinical trials, known as the PERSIST program, for patients with myelofibrosis, with one trial in a broad set of patients without limitations on platelet counts, the PERSIST-1 trial; and the other in patients with low platelet counts, the PERSIST-2 trial. In August 2014, pacritinib was granted Fast Track designation by the FDA for the treatment of intermediate and high risk myelofibrosis including, but not limited to, patients with disease-related thrombocytopenia (low platelet counts); patients experiencing treatment-emergent thrombocytopenia on other JAK2 inhibitor therapy; or patients who are intolerant of, or whose symptoms are not well controlled (sub-optimally managed) on other JAK2 therapy.

Clinical studies under the CTI BioPharma investigational new drug (IND) for pacritinib are currently subject to a full clinical hold issued by the U.S. Food and Drug Administration in February 2016.

About Pacritinib

Pacritinib is an investigational oral kinase inhibitor with specificity for JAK2, FLT3, IRAK1 and CSF1R. 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. In addition to myelofibrosis, the kinase profile of pacritinib suggests its potential therapeutic utility in conditions such as acute myeloid leukemia, or AML, myelodysplastic syndrome, or MDS, chronic myelomonocytic leukemia, or CMML, and chronic lymphocytic leukemia, or CLL, due to its inhibition of c-fms, IRAK1, JAK2 and FLT3.

About PIXUVRI® (pixantrone)

PIXUVRI is a novel aza-anthracenedione with unique structural and physiochemical properties. PIXUVRI was structurally designed so that it cannot bind iron and perpetuate oxygen radical production or form a long-lived hydroxyl metabolite -- both of which are the putative mechanisms for anthracycline induced acute and chronic cardiotoxicity. 

In May 2012, the European Commission granted conditional marketing authorization for PIXUVRI as a monotherapy for the treatment of adult patients with multiply relapsed or refractory aggressive NHL. The benefit of PIXUVRI treatment has not been established in patients when used as fifth line or greater chemotherapy in patients who are refractory to last therapy. The Summary of Product Characteristics (SmPC) has the full prescribing information, including the safety and efficacy profile of PIXUVRI in the approved indication. The SmPC is available at www.pixuvri.eu. PIXUVRI does not have marketing approval in the United States.

About Tosedostat

Tosedostat is an investigational oral aminopeptidase inhibitor that has demonstrated anti-tumor responses in blood-related cancers and solid tumors in Phase 1-2 clinical trials. Tosedostat is currently being evaluated in multiple Phase 2 clinical trials for the treatment of patients with AML or high-risk MDS. Tosedostat is not approved or commercially available.

About CTI BioPharma

CTI BioPharma Corp. 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 a commercial presence in Europe with respect to PIXUVRI^® and a late-stage development pipeline, including pacritinib for the treatment of patients with myelofibrosis. CTI BioPharma is headquartered in Seattle, Washington, with offices in London and Milan under the name CTI Life Sciences Limited. For additional information and to sign up for email alerts and get RSS feeds, please visit www.ctibiopharma.com.

Forward-Looking Statements

This press release includes forward-looking statements, 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, but are not limited to, expectations with respect to our ability to be able to interpret clinical trial data and results despite not satisfying the pre-specified minimum evaluable patient goal and expectations with respect to the potential therapeutic utility of pacritinib, including pacritinib's potential to achieve treatment goals across patients with myelofibrosis, regardless of baseline characteristics, such as starting platelet count and in particular, its potential to reduce spleen volume and symptom burden and improve HRQoL. Investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date of this release. In particular, this press release addresses select preliminary clinical trial data and results, and should be evaluated together with information regarding primary and secondary endpoints, safety and additional data once such data has been more fully analyzed and is made publicly available. In addition, meaningful interpretation of PERSIST-2 may not be possible because the pre-specified minimum evaluable patient goal was not met. The statements are based on assumptions about many important factors and information currently available to us to the extent we have thus far had an opportunity to fully and carefully evaluate such information in light of all surrounding facts, circumstances, recommendations and analyses. A number of results and uncertainties could cause actual results to differ materially from those in the forward-looking statements, including: satisfaction of regulatory and other requirements; that trial results observed to date may differ from future results or that different conclusions or considerations may qualify such results once existing data has been more fully evaluated; actions of regulatory bodies and other governmental authorities; other clinical trial results; changes in laws and regulations; product quality, product efficacy, study protocol, data integrity or patient safety issues; product development risks; and other risks identified in each of the issuer's most recent filings on Forms 10-K and 10-Q and other Securities and Exchange Commission filings. Except as required by law, CTI Biopharma does not intend to update any of the statements in this press release upon further developments.

PIXUVRI is a registered trademark of CTI BioPharma Corp.

CTI BioPharma Contact:

Ed Bell
+1 206-272-4345
[email protected]

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SOURCE CTI BioPharma Corp.

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