SEATTLE, Jan. 9, 2017 /PRNewswire/ — CTI BioPharma Corp. (CTI BioPharma) (NASDAQ and MTA: CTIC) today announced positive progress on its lead programs in addition to key business priorities for 2017.
“Throughout 2016 we maintained our commitment to bringing new therapies to patients with unmet medical needs, and were successful in working with the FDA to remove the full clinical hold on pacritinib and get it back on the development track for the benefit of myelofibrosis patients,” said Richard Love, Interim President and Chief Executive Officer of CTI BioPharma. “The PERSIST-2 clinical trial of pacritinib was highlighted as one of six late-breaking data presentations at the American Society of Hematology conference in December. We believe this oral presentation was well received by the hematology/oncology community, which recognizes the unmet need for myelofibrosis patients who are ineligible to receive or are not benefitting from the approved JAK1/JAK2 inhibitor, ruxolitinib. Additionally, the PIX306 confirmatory trial of our commercial product PIXUVRI (R)(pixantrone) continues to progress toward an announcement of top-line results later this year. If positive, this trial could provide the opportunity for full approval and label expansion by EMA, and discussions with the FDA about accelerated PIXUVRI approval in the US for the treatment of patients with relapsed or refractory aggressive B-cell non-Hodgkin lymphoma. We have also made significant effort at reducing our expenses and believe we are well positioned moving into 2017.”
Recent Progress Update
In January 2017, CTI BioPharma announced the U.S. Food and Drug Administration (FDA) removed the full clinical hold on studies being conducted under the Investigational New Drug (IND) application for pacritinib.
In December 2016, data from the randomized Phase 3 PERSIST-2 clinical trial comparing pacritinib with physician-specified best available therapy (BAT), including ruxolitinib, for treatment of patients with myelofibrosis whose baseline platelet counts are less than 100,000 per microliter was one of six late-breaking oral presentations at the American Society of Hematology Annual Meeting. Patients in the trial were randomized to receive 200 mg pacritinib twice daily (BID), 400 mg pacritinib once daily (QD), or BAT. In those patients who had a chance to reach Week 24 (the primary analysis time point) at the time the clinical hold was imposed, the trial showed a statistically significant response rate in spleen volume reduction (SVR) in patients treated with pacritinib compared to BAT irrespective of prior treatment with ruxolitinib. The co-primary endpoint of reduction of Total Symptom Score (TSS) was not achieved but trended toward improvement in TSS. Although secondary objectives could not be evaluated formally due to the study not achieving one of the primary objectives, when the two pacritinib dosing arms were evaluated separately versus BAT, pacritinib BID showed a higher percent of SVR and TSS responses compared to BAT; whereas, pacritinib given QD showed only a higher percent SVR responses compared to BAT. There was no significant difference in overall survival (OS) across treatment arms, censored at the time of clinical hold. The most common treatmentemergent adverse events (AEs), occurring in 20 percent or more of patients treated with pacritinib within 24 weeks, of any grade, were gastrointestinal (generally manageable diarrhea, nausea and vomiting) and hematologic (anemia and thrombocytopenia) and were generally less frequent for BID versus QD administration. The most common serious treatment-emergent AEs (incidence of ≥5 percent reported in any treatment arm irrespective of grade) were anemia, thrombocytopenia, pneumonia and acute renal failure none of which exceeded 8 percent individually in any arm. The presentation was also selected to be part of the “2017 Highlights of ASH” program designed to review significant scientific updates presented at ASH with hematologists/oncologists at five locations across the U.S.
In January 2017, CTI BioPharma received a €7.5 million milestone payment from its partner Servier following achievement of a milestone associated with patient enrollment in the Phase 3 PIX306 clinical trial of PIXUVRI. The trial is a postauthorization trial as part of the conditional marketing authorization of PIXUVRI in the European Union (E.U.) The PIX306 is comparing PIXUVRI and rituximab with gemcitabine and rituximab in the setting of aggressive B-cell non-Hodgkin lymphoma (NHL). The trial continues to enroll patients.
2017 Key Objectives
Advance Marketing Authorization Application in E.U. and define regulatory pathway in U.S. for pacritinib. CTI BioPharma continues to have dialogue with the European Medicines Authority (EMA) on the Marketing Authorization Application (MAA) for pacritinib that had been previously filed by its former partner, Baxalta. At the time of the filing only data from the first Phase 3 clinical trial of pacritinib, PERSIST-1, was available. With the availability of results from the PERSIST-2 clinical trial and the recent completion of the PERSIST-2 clinical study report, CTI BioPharma believes that the best strategy currently to achieve marketing authorization is to utilize the combined clinical evidence from both Phase 3 trials. Accordingly, CTI BioPharma is evaluating whether to update the current application with the additional data from PERSIST-2 or to resubmit the MAA. Under either plan, CTI BioPharma would expect to pursue marketing authorization for the treatment of patients with myelofibrosis who are ineligible to receive, intolerant of or have insufficient response to the approved JAK1/JAK2 inhibitor, ruxolitinib.
CTI BioPharma also intends to discuss with the FDA the future development of pacritinib.
Initiate PAC203 trial. CTI BioPharma expects to initiate the PAC203 trial in the second quarter of 2017. The trial plans to enroll up to approximately 105 patients with primary myelofibrosis who have failed prior ruxolitinib therapy to evaluate the safety and the dose response relationship for efficacy (spleen volume reduction at 24 weeks) of three dose regimens: 100 mg once-daily, 100 mg twice-daily (BID) and 200 mg BID.
Secure ex-U.S. partner for pacritinib. CTI BioPharma intends to secure a partnership for the development and commercialization of pacritinib in certain territories outside the U.S.
Release top-line results of PIX306. CTI BioPharma expects to complete enrollment in the ongoing PIX306 trial of PIXUVRI and release top-line results by the end of 2017.
CTI BioPharma’s preliminary, unaudited estimates of its cash and cash equivalents balance as of December 31, 2016 is approximately $44.0 million. In January 2017, we received a €7.5 million milestone payment from Servier. CTI BioPharma expects that its cash burn (a non-GAAP financial measure), excluding cash inflows from future business development activities and proceeds from capital markets financing activities, would be approximately $65-75 million for 2017. The Company expects to meet its cash requirements for 2017 with existing cash and by partnering one or more product assets during the course of the year.
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.
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.
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. The PERSIST-1 trial met its primary endpoint of spleen volume reduction (35 percent or greater from baseline to Week 24 by MRI/CT scan). The PERSIST-2 trial met one of its coprimary endpoints, that of spleen volume reduction. The co-primary endpoint of reduction of Total Symptom Score (TSS) was not achieved but trended toward improvement in TSS.
Clinical studies under the investigational new drug (IND) for pacritinib were subject to a full clinical hold issued by the FDA in February 2016. In January 2017, the FDA removed the full clinical hold and stated that clinical trials may be resumed.
About PIXUVRI® (pixantrone)
PIXUVRI is a novel aza-anthracenedione with unique structural and physiochemical properties. 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 B-cell 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.
In September 2014, CTI BioPharma entered into an exclusive license and collaboration agreement, with Servier with respect to the development and commercialization of PIXUVRI. Under the agreement, CTI BioPharma retains full commercialization rights to PIXUVRI in Austria, Denmark, Finland, Germany, Israel, Norway, Sweden, Turkey, the United Kingdom and the U.S. while Servier has exclusive rights to commercialize PIXUVRI in all other countries.
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.
This press release includes forward-looking statements 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 CTI BioPharma’s 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, 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, and statements regarding CTI BioPharma’s expectations with respect to the development of CTI BioPharma, its financial position and its product and product candidate portfolio, including strategies and plans for achieving marketing authorization and other approvals, partnerships, and the initiation or completion of clinical strials. 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, 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. Risks that contribute to the uncertain nature of the forward-looking statements include, among others: risks associated with the biopharmaceutical industry in general and with CTI BioPharma and its product and product candidate portfolio in particular, risks associated with the pace or geography of enrollment of its clinical trials, risks associated with the commencement or outcome of preclinical and clinical studies, 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, that CTI BioPharma may not obtain favorable determinations by other regulatory, patent and administrative governmental authorities or will not be in a position to submit regulatory submissions as or when projected, risks related to the actions of regulatory bodies and other governmental authorities, risks related to changes in laws and regulations, product quality, product efficacy, study protocol, data integrity or patient safety issues, risks related to the costs of developing, producing and selling PIXUVRI, pacritinib and CTI BioPharma’s other product candidates, and other risks, including, without limitation, competitive factors, technological developments, and that CTI BioPharma may not achieve previously announced goals and objectives as or when projected as well as other risks listed or described from time to time in CTI BioPharma’s most recent filings with the SEC on Forms 10-K, 10-Q and 8- K. 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.