The U.S. Food and Drug Administration (FDA) has lifted the full clinical hold placed on all clinical trials of pacritinib – an oral kinase JAK2/FLT3 inhibitor – for treatment for myelofibrosis (MF).
The FDA placed the hold on pacritinib in February 2016 after two phase III trials (PERSIST-1 and PERSIST-2) resulted in deaths related to intracranial hemorrhage, cardiac failure, and cardiac arrest. The FDA suggested that the drug’s manufacturer (CTI BioPharma) conduct dose-exploration studies, submit final study reports and data sets for PERSIST-1 and PERSIST-2, make certain modifications to protocols and study-related documents, and request a meeting with the FDA prior to submitting a response to the full clinical hold.
As a result, CTI BioPharma is planning the PAC203 trial, which will evaluate the safety and dose-response relationship for efficacy (defined as spleen volume reduction at 24 weeks) of pacritinib at three doses: 100 mg once daily, 100 mg twice daily, and 200 mg twice daily (this was the dose used in PERSIST-2). Investigators plan to enroll approximately 105 patients with primary MF who have failed prior ruxolitinib therapy.
Source: CTI BioPharma news release, January 6, 2017.