CTI BioPharma Corp, a biopharmaceutical company, announced positive progress of its lead clinical programme in addition to several key business priorities for 2016.

"After a productive 2015, we have entered 2016 well capitalized and focused on preparing for the potential accelerated approval and launch of a new treatment option for people with intermediate and high-risk myelofibrosis with low platelet counts," said James A. Bianco, M.D., CTI BioPharma's president and chief executive officer.

"With the recently completed NDA submission for pacritinib, interim data indicating the potential therapeutic utility for tosedostat in AML and high-risk MDS, and a strong financial position, we believe the stage is set for a transformational year for our company."

In January 2016, CTI BioPharma announced the completion of the rolling New Drug Application (NDA) to the US Food and Drug Administration (FDA) for pacritinib, an investigational oral kinase inhibitor with specificity for JAK2, FLT3, IRAK1 and CSF1R. CTI BioPharma and Baxalta Incorporated (Baxalta) are seeking US marketing approval of pacritinib for the treatment of patients with intermediate and high-risk myelofibrosis with low platelet counts of less than 50,000 per microliter (<50,000/µL).

In December 2015, researchers presented 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 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.

In December 2015, researchers presented an update on results from an investigator-sponsored phase 2 trial of tosedostat – CTI BioPharma's investigational oral selective aminopeptidase inhibitor – in elderly patients with either primary (de novo) acute myeloid leukemia (AML) or secondary AML. The data showed a complete response (CR) of 48.5 per cent with tosedostat in combination with low dose cytarabine/Ara-C (LDAC) – 33 per cent of these patients were still responding after a median of 506 days.

In November 2015, CTI BioPharma announced that the United Kingdom's National Cancer Research Institute (NCRI) Haematological Oncology Clinical Studies Group has chosen to advance tosedostat to the second stage of a randomized clinical trial of low-dose cytarabine plus or minus tosedostat in older patients with AML or high risk myelodysplastic syndrome (MDS). The AML Less Intensive (LI-1) trial is designed as a "Pick-a-Winner" trial to be able to simultaneously test a number of promising agents added to standard therapy with low-dose cytarabine in older patients with AML or MDS who are unfit for standard aggressive induction therapy. Nine regimens have been tested in the Pick-a-Winner program, of which only four, including tosedostat, have passed the initial hurdle for progression (which requires evidence of an improvement in remission rate with acceptable safety). CTI BioPharma plans to discuss potential approval pathways with regulators in the US and EU in 2016.

In December 2015, the company announced receipt of a $10 million milestone payment from Teva Pharmaceutical Industries Ltd. related to the achievement of sales milestones for Trisenox (arsenic trioxide).

In September 2015, the company completed a registered direct offering and in October and December 2015, the company completed two underwritten public offerings resulting in aggregate net proceeds of approximately $115 million. CTI BioPharma's current cash balance is anticipated to fund operations for at least the next two years based on our current budget expectations and development plans.

CTI BioPharma plans to provide 2016 financial guidance in its fourth quarter and year-end 2015 financial results announcement.

CTI BioPharma seeks US marketing approval of pacritinib for the treatment of patients with intermediate and high-risk myelofibrosis with low platelet counts of <50,000/µL.

CTI BioPharma expects to complete enrollment in the PERSIST-2 phase 3 trial of pacritinib for patients with myelofibrosis whose platelet counts are less than or equal to 100,000 per microliter in the first quarter 2016 and unblind and report top-line data late in the fourth quarter of 2016.

Initiate trial in second indication for pacritinib. CTI BioPharma intends to advance a pacritinib development programme in other hematologic malignancies in the fourth quarter of 2016.

Initiate registration-directed trial for tosedostat. CTI BioPharma plans to consult with the FDA and European Medicines Agency (EMA) regarding a registration-directed strategy for tosedostat, including the potential to utilize the results from the ongoing investigator-sponsored LI-1 trial to support registration and approval. If positive, these discussions could help enable the start of a pivotal program in 2016.

Secure ex-US partner for tosedostat. CTI BioPharma intends to secure a partnership for the development and commercialisation of tosedostat in certain territories outside the US.

CTI BioPharma expects to complete enrollment in the ongoing PIX306 post-marketing commitment phase 3 study of Pixuvri in the fourth quarter of 2016.