Benzinga | Mar 22, 2021 01:00PM EDT

CTI BioPharma's Pacritinib Demonstrates Positive Safety Profile In Graft-Versus-Host-Disease Study

CTI BioPharma Corp (NASDAQ: CTIC) has announced that Clinical Cancer Research, a journal of the American Association for Cancer Research, has published results from a Phase 1 study evaluating pacritinib, to prevent acute graft-versus-host disease (GVHD).

* Pacritinib showed an encouraging safety profile and exhibited preliminary therapeutic activity in preventing acute GVHD after allogeneic hematopoietic cell transplantation.

* The Phase 2 portion of the trial is designed to evaluate the therapeutic effect of pacritinib in combination with sirolimus and low-dose tacrolimus for GVHD prevention.

* The Phase 1 portion of the trial evaluated the safety of pacritinib when administered with sirolimus, plus low-dose tacrolimus after allogeneic hematopoietic cell transplantation.

* A 3+3 dose escalation design identified Pacritinib 100 mg twice a day as the minimum biologically active and well-tolerated dose for further study.

* The new manuscript reports that dual JAK2/mTOR inhibition suppresses pathogenic Th1 and Th17 cells, enhances the potency of immunosuppressive regulatory T cells (Tregs) and maintains natural killer cells and CD8+ cytotoxic T lymphocytes required for graft-versus-leukemia.

* Notably, PAC/SIR/TAC was observed to preserve donor cytomegalovirus (CMV) immunity, permit timely engraftment, and avoid cytopenias observed with nonselective JAK inhibitors.

* Pacritinib is an oral kinase inhibitor with specificity for JAK2, IRAK1, and CSF1R. The JAK family of enzymes is a central component in signal transduction pathways, which are critical to normal blood cell growth and development and inflammatory cytokine expression and immune responses.

* Price Action: CTIC shares trading 2.03% higher at $3.27 in market hours on the last check Monday.