Benzinga | Dec 6, 2020 04:09PM EST

CTI BioPharma Announced Presentation of Encouraging Data Supporting Pacritinib's Potential Benefit in Preventing Acute GVHD at ASH 2020

CTI BioPharma Corp. (NASDAQ:CTIC) today announced an oral presentation supporting the Company's pacritinib development program in the prevention of acute graft versus host disease (GVHD) at the 62nd American Society of Hematology (ASH) Annual Meeting & Exposition, being held virtually December 5-8, 2020. The results are from an investigator-sponsored Phase 1/2 study conducted at the Mayo Clinic and Moffit Cancer Center.

"The data presented today at ASH highlights the potential for the expanded use of pacritinib. Acute GVHD following allogeneic hematopoietic cell transplantation results in higher rates of morbidity and mortality, compromises the anti-leukemic response of the graft and occurs despite standard immunosuppressive prophylaxis, rendering it an area where new therapeutic options are needed," said Adam R. Craig, M.D., Ph.D., President and Chief Executive Officer of CTI Biopharma. "These data demonstrate that in this Phase 1/2 study, adding pacritinib to the standard prophylaxis of sirolimus and low-dose tacrolimus resulted in a significant reduction in the expected acute GVHD rates in patients within the first 100 days of therapy as compared to historical data, without compromising transplantation outcomes and without any new safety concerns. We are encouraged by these data, and look forward to further evaluating pacritinib's efficacy and safety in this indication in the on-going Phase 2 clinical trial."

All presentation materials will be available at ctibiopharma.com following the presentations.

Biological and Clinical Impact of JAK2/mTOR Blockade in GVHD Prevention: Preclinical and Phase 1 / 2 Study ResultsResults from an investigator-sponsored (Mayo Clinic and Moffit Cancer Center) Phase 1/2 study of GVHD prophylaxis for allogeneic hematopoietic cell transplantation (alloHCT) using pacritinib, sirolimus, low-dose tacrolimus are being presented today, Sunday, December 6 at 10 a.m. PT in an oral presentation session. Abstract:No. 355Summary:This single-arm Phase 1/2 study (NCT02891603) tested the safety, pharmacodynamics and efficacy of pacritinib when administered with sirolimus plus low-dose tacrolimus (PAC/SIR/TAC) after alloHCT. A 3+3 dose escalation design was planned using three doses of pacritinib (100 mg QD, 100 mg BID, 200 mg BID) in combination with standard sirolimus and low-dose tacrolimus immunosuppressive therapy. Efficacy endpoints included acute GVHD (aGVHD) rate at day 100.

PAC 100 mg twice daily (BID), dose level 2, was selected as the recommended Phase 2 dose based on its biological activity and safety profile. Preliminary evidence of the benefit of adding pacritinib to standard immunosuppressive therapy in reducing the rates of aGVHD was reported, with a 25% rate of grade 2-4 aGVHD across all dose cohorts, and 17% at the recommended Phase 2 dose of 100 mg BID, as compared to grade 2-4 aGVHD incidence rate of 43% expected with sirolimus plus low-dose tacrolimus administered alone1. No compromise in alloHCT outcome or major safety concerns were observed. Futher, there was no evidence of cytopenias, impaired immune reconstitution, or cytomegalovirus (CMV) reactivation. A Phase 2 clinical trial to fully evaluate efficacy in GVHD prevention is ongoing (NCT02891603