CTI BioPharma Provides Program Update Following Regulatory Feedback from the U.S. FDA on Pacritinib Development

On December 18, 2018 CTI BioPharma Corp. (NASDAQ:CTIC) reported that it has received input from the U.S. Food and Drug Administration (FDA) at a recent Type C meeting on key elements of the design of a new randomized Phase 3 study of pacritinib in adult patients with myelofibrosis (primary myelofibrosis, post-polycythemia vera myelofibrosis, or post-essential thrombocythemia myelofibrosis) and who have severe thrombocytopenia (as defined by patients with platelet counts of less than 50,000 per microliter), an indication that has been recognized by the medical community as an important unmet medical need (Press release, CTI BioPharma, DEC 18, 2018, View Source [SID1234532119]).

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The planned Phase 3 study is designed to evaluate the effects of pacritinib as compared to physician’s choice of treatment. The primary efficacy endpoint will be the proportion of patients achieving a greater than or equal to 35% spleen volume reduction (SVR) between baseline and Week 24. Secondary efficacy endpoints of the study include total symptom score reduction and overall survival.

Before commencing the Phase 3 study, CTI plans to meet with the FDA to discuss the final optimal dose analysis from the PAC203 Phase 2 study. To expedite the transition to Phase 3, CTI intends to amend the PAC203 protocol to include a Phase 3 component. The PAC203 Phase 3 component is designed to enroll approximately 200 patients with enrollment expected to commence in the third quarter of 2019. The anticipated cost of the Phase 3 study is approximately $25 million. Taking into account the impact of recently-announced cost saving efforts and the anticipated cost of the new Phase 3 trial, the current CTI financial analysis projects a cash runway that extends into 2020.