CTI BioPharma Announces Topline Data from Final Analysis of the PRE-VENT Clinical Trial Evaluating Pacritinib as a Treatment for Severe COVID-19

CTI BioPharma Corp. (Nasdaq: CTIC) today announced topline results from the PRE-VENT trial (NCT04404361) of pacritinib in hospitalized patients with severe COVID-19. PRE-VENT, a randomized, double-blind, placebo-controlled multicenter study, compared pacritinib/standard of care versus placebo/standard of care in hospitalized patients with severe COVID-19, including patients with and without cancer. The primary endpoint of the trial was the proportion of patients who progressed to invasive mechanical ventilation and/or extracorporeal membrane oxygenation or died by Day 28. The study was recently amended to a Phase 2 design due to the availability of COVID-19 vaccines and an evolving COVID-19 environment.

The final PRE-VENT analysis was conducted following the randomization of 200 patients. In patients with severe COVID-19 treated with pacritinib/standard of care, compared to placebo/standard of care, a statistically significant improvement in the primary endpoint of progression to invasive mechanical ventilation and/or extracorporeal membrane oxygenation or death by Day 28 was not demonstrated (26.3% vs. 24.8% [OR: 1.01 (95% CI: 0.51-1.99)], P=0.98).

Pacritinib was generally well-tolerated by patients with severe COVID-19, with lower rates of grade 3+ events and serious adverse events being reported in patients treated with pacritinib/standard of care compared to placebo/standard of care (29.2% vs. 40.6% and 20.8% vs. 32.7%, respectively). The most common adverse events comparing pacritinib/standard of care to placebo/standard of care were increased alanine aminotransferase (15.6% vs. 16.8%), anemia (12.5% vs. 17.8%), bradycardia (15.6% vs. 10.9%), constipation (12.5% vs. 12.9%), hypokalemia (12.5% vs. 12.9%), and hyperkalemia (9.4% vs. 10.9%).

“Last year, we launched the PRE-VENT study of pacritinib in patients with severe COVID-19 in response to the pandemic and an unprecedented medical need to improve outcomes for these patients,” said Adam R. Craig, M.D., Ph.D., President and Chief Executive Officer of CTI BioPharma. “Although pacritinib was well tolerated on study, it did not demonstrate a clinical benefit in treating the sickest patients with COVID-19. We would like to thank all the patients, families, physicians, and medical teams who participated in this study during such a difficult time. Based on the results of this study, we have decided not to pursue further development of pacritinib for the treatment of severe COVID-19. We will continue to focus our efforts on launch preparedness activities for pacritinib in myelofibrosis, which is currently under Priority Review with a PDUFA date of November 30, 2021.”

The Company expects to publish a full report on the study in a medical journal.