Pharmaceutical Business review

CTI BioPharma, Baxter report positive results from Phase III trial of pacritinib in patients with myelofibrosis

As recently reported at the American Society of Clinical Oncology (ASCO) annual meeting, results show a significant reduction in the Total Symptom Score (TSS) (the proportion of patients with a 50 percent or greater reduction in TSS from baseline to Week 24), and in each individual common disease-related symptom, from baseline to Week 24, in patients treated with pacritinib compared to best available therapy (exclusive of a JAK inhibitor) (BAT).

These PROs, as well as other quality of life measures, will be presented at the 20th Congress of European Hematology Association (EHA) by Adam Mead, M.D., Guy’s and St. Thomas’ NHS Foundation Trust, Guy’s Hospital, London, United Kingdom in an oral presentation on Sunday, June 14, 2015 at 12:15 CEST (abstract #LB2072). These data were also selected for inclusion in the official EHA Press Briefing which occurred today (Friday, June 12, 2015) at 08:30 CEST.

As previously reported, the PERSIST-1 trial met its primary endpoint of spleen volume reduction of 35 percent or greater from baseline to Week 24 as measured by MRI/CT scan.

Myelofibrosis is a rare blood cancer associated with significantly reduced quality of life and shortened survival. As the disease progresses, the body slows production of important blood cells and within one year of diagnosis the incidence of disease-related thrombocytopenia (very low blood platelet counts), severe anemia, and red blood cell transfusion requirements increases significantly.

Among other complications, most patients with myelofibrosis present with enlarged spleens (splenomegaly), as well as many other potentially devastating physical symptoms such as abdominal discomfort, bone pain, feeling full after eating little, severe itching, night sweats, and extreme fatigue.

"Patient-reported outcomes are an important measure for understanding the potential benefit of a therapy on patients’ lives – particularly for a disease such as myelofibrosis where the symptoms have such a tremendous impact on the quality of patients’ daily lives," stated James A. Bianco, M.D., President and CEO of CTI BioPharma.

"These new data from the PERSIST-1 study further support our belief, not only in the activity of pacritinib, but also the potential to positively impact patients’ daily lives by relieving the symptoms that accompany myelofibrosis."

"The PERSIST-1 trial has continued to generate positive and important findings for the hematology community," said David Meek, Head of Oncology at Baxter BioScience.

"We look forward to advancing the clinical trial program of pacritinib as we work to realize the full potential of this investigational compound to help patients with serious blood cancers, such as myelofibrosis."