On March 2, 2017 CTI BioPharma Corp. (NASDAQ and MTA: CTIC) reported financial results for the fourth quarter and full year ended December 31, 2016 (Press release, CTI BioPharma, MAR 2, 2017, View Source [SID1234517959]). Schedule your 30 min Free 1stOncology Demo! "The presentation of detailed results from the Phase 3 PERSIST-2 trial of pacritinib at the ASH (Free ASH Whitepaper) Annual Meeting in December demonstrated that pacritinib may have potential to address a critical unmet need for myelofibrosis patients with low blood platelets or those ineligible to receive, intolerant of or which have insufficient response to the approved JAK1/JAK2 inhibitor," said Richard Love, Interim President and CEO of CTI BioPharma. "We are committed to advancing pacritinib for these patients with no other treatment option and look forward to discussions with the regulatory agencies worldwide this year. We are very pleased to have Adam Craig, M.D., Ph.D., join us as President and CEO. We expect that his experience in hematology-oncology drug development will be a tremendous asset for the company. We believe that we are well positioned to make significant progress in 2017 and it should be a transformative year for the company."
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Recent Events
Research and Development
The Marketing Authorization Application (MAA) for pacritinib was submitted to the European Medicines Agency (EMA) in February 2016 with an indication statement based on the PERSIST-1 trial data. In its initial assessment report, the Committee for Medicinal Products for Human Use (CHMP) determined that the current application is not approvable because of major objections in the areas of efficacy, safety (hematological and cardiovascular toxicity) and the overall risk-benefit profile of pacritinib. Subsequent to the filing of the MAA, data from the second phase 3 trial of pacritinib, PERSIST-2, were reported. These data suggest that pacritinib may show clinical benefit in patients who have failed or are intolerant to ruxolitinib therapy, a population for which there is no approved therapy. Following discussions with the EMA about how PERSIST-2 data might address the major objections and how to integrate the data into the current application, the Company has decided to withdraw the MAA. The Company is preparing a new MAA that seeks to address the major objections by including data from PERSIST-2. The new application will focus on patients who have failed or are intolerant to ruxolitinib. The Company plans to submit this new application in the second quarter of 2017.
In January 2017, the CTI BioPharma received a €7.5 million milestone payment from the Company’s partner Servier following achievement of a milestone associated with patient enrollment in the Phase 3 PIX306 clinical trial of PIXUVRI.
In January 2017, the FDA removed the full clinical hold following review of CTI BioPharma’s complete response submission which included, among other items, final Clinical Study Reports for both PERSIST-1 and 2 trials and a dose-exploration clinical trial protocol that the FDA requested. At that time, the Company announced that it would conduct a new trial, PAC203, that 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 12 and 24 weeks) of three dose regimens: 100 mg once-daily, 100 mg twice-daily (BID) and 200 mg BID. The 200 mg BID dose regimen was used in PERSIST-2.
In December 2016, CTI BioPharma announced the presentation of data from PERSIST-2, a randomized Phase 3 clinical trial comparing pacritinib with physician-specified best available therapy (BAT), for the treatment of high risk, thrombocytopenic myelofibrosis patients (platelet counts <100,000/µL ) in in a late-breaking oral session at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting. Data presented at ASH (Free ASH Whitepaper) show that in myelofibrosis patients a statistically significant response rate in spleen volume reduction (SVR) with pacritinib therapy was observed compared to BAT that included use of the approved JAK1/JAK2 inhibitor ruxolitinib. The co-primary endpoint of reduction of Total Symptom Score (TSS) was not achieved but trended toward improvement in TSS. The results from the trial demonstrated that efficacy and safety in the combined patient group with <50,000/µL or had been exposed to a prior JAK2 inhibitor was generally consistent with the overall ITT results.
Corporate
In February 2017, the CTI BioPharma announced the appointment of Adam Craig, M.D., Ph.D., as President and CEO and as a Director of CTI BioPharma effective March 20, 2017. Dr. Craig has over 20 years of experience in hematology, oncology and drug development in both the US and Europe. Dr. Craig has worked as an independent consultant providing strategic and operational advice and support to CTI BioPharma and other hematology/oncology biotechnology companies since 2016. Prior to consulting, Dr. Craig was Chief Medical Officer (CMO) and Executive Vice President of Development of Sunesis Pharmaceuticals from 2012 to 2016. From 2008 to 2012, Dr. Craig was CMO and Senior Vice President of Chemgenex Pharmaceuticals Ltd, a publicly-traded biotechnology company which was acquired by Cephalon/Teva Pharmaceuticals in 2011. Dr. Craig is a Member of the Royal College of Physicians (UK) and undertook Post-Graduate Training in Pediatrics and Pediatric Oncology.
In January 2017, CTI BioPharma announced that Michael Metzger had been appointed to the Board of Directors. Mr. Metzger has extensive experience leading and growing companies in the biopharmaceutical industry over the last 20 years. Mr. Metzger is currently president and chief operating officer of Syndax Pharmaceuticals, Inc., a publicly traded immuno-oncology biopharmaceutical company. He has served in executive and senior management positions at Regado Biosciences, Mersana Therapeutics, Forest Laboratories and OncoNova Therapeutics.
Fourth Quarter and Full Year Financial Results
Total revenues for the fourth quarter and full year ended December 31, 2016, were $9.1 million and $57.4 million, respectively, compared to $11.3 million and $16.1 million for the respective periods in 2015. The increase in total revenue for full year 2016 is primarily due to recognition of $32 million in milestone payments and reimbursement of development costs from Shire plc related to pacritinib, recognition of $8.0 million (or €7.5 million) in a milestone payment from Servier related to the PIX306 clinical trial of PIXUVRI, and increased net product sales of PIXUVRI. CTI BioPharma had previously received a cash advance for the $32 million in milestone payments from Baxalta in the second quarter of 2015 that was accounted for as long-term debt until the achievement of the associated milestones in the first quarter of 2016. Net product sales of PIXUVRI for the fourth quarter and the full year ended December 31, 2016, were $1.1 million and $4.3 million, respectively, compared to $1.1 million and $3.5 million for the respective periods in 2015.
GAAP operating loss for the fourth quarter and full year ended December 31, 2016, was $5.6 million and $49.2 million, respectively, compared to GAAP operating loss of $26.2 million and $116.7 million for the respective periods in 2015. Non-GAAP operating loss, which excludes non-cash share-based compensation expense, for the fourth quarter and year ended December 31, 2016, was $3.5 million and $35.8 million, respectively, compared to the non-GAAP operating loss of $24.4 million and $101.8 million for the respective periods in 2015. Non-cash share-based compensation expense for the fourth quarter and full year ended December 31, 2016, was $2.1 million and $13.3 million, respectively, compared to $1.8 million and $14.8 million for the respective periods in 2015. For information on CTI BioPharma’s use of non-GAAP operating loss and a reconciliation of such measure to GAAP operating loss, see the section below entitled "Non-GAAP Financial Measures."
Net loss for the fourth quarter of 2016 was $6.4 million, or ($0.23) per share, compared to a net loss of $28.8 million, or ($1.27) per share, for the same period in 2015. Net loss for the full year ended December 31, 2016, was $52.0 million, or ($1.86) per share, compared to a net loss of $122.6 million, or ($6.51) per share, for the same period in 2015. The decrease in net loss for the fourth quarter and the year ended December 31, 2016, compared to the respective periods in 2015 is primarily due to increased net product sales and license and contract revenue and decrease in operating expenses.
As of December 31, 2016, cash and cash equivalents totaled $44.0 million, compared to $128.2 million at December 31, 2015. The cash balance does not include $8.0 million milestone payment that was recognized as revenue in the fourth quarter of 2016 but not received until January 2017.