CTI BioPharma Announces Presentation of Data Supporting Pacritinib's Potential Benefit in Preventing Acute GVHD at the 62nd American Society of Hematology Meeting
"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
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 Results
Results from an investigator-sponsored (
Abstract: No. 355
Summary: 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).
Pacritinib is an investigational 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, as well as inflammatory cytokine expression and immune responses. Mutations in these kinases have been shown to be directly related to the development of a variety of blood-related cancers, including myeloproliferative neoplasms, leukemia and lymphoma. In addition to myelofibrosis, the kinase profile of pacritinib suggests its potential therapeutic utility in conditions such as acute myeloid leukemia (AML), myelodysplastic syndrome (MDS), chronic myelomonocytic leukemia (CMML), and chronic lymphocytic leukemia (CLL), due to its inhibition of c-fms, IRAK1, JAK2 and FLT3.
We are a biopharmaceutical company focused on the acquisition, development and commercialization of novel targeted therapies for blood-related cancers that offer a unique benefit to patients and their healthcare providers. We concentrate our efforts on treatments that target blood-related cancers where there is an unmet medical need. In particular, we are focused on evaluating pacritinib, our sole product candidate currently in active late-stage development, for the treatment of adult patients with myelofibrosis. In addition, we have recently started developing pacritinib for use in the prevention of acute graft versus host disease and in hospitalized patients with severe COVID-19, in response to the COVID-19 pandemic. We are headquartered in
Statements included in this press release that are not historical in nature are forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, Section 21E of the Securities Exchange Act of 1934 and the Private Securities Litigation Reform Act of 1995. These forward-looking statements are based on current assumptions that involve risks, uncertainties and other factors that may cause the actual results, events or developments to be materially different from those expressed or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to: our ability to successfully demonstrate the safety and efficacy of pacritinib in various indications; our ability to receive regulatory approval for pacritinib pursuant to the accelerated approval pathway or at all; our planned commercialization of pacritinib; our ability to enter into potential partnerships relating to our commercial launch of pacritinib; and those risks more fully discussed in the section entitled "Risk Factors" in our Annual Report on Form 10-K for the year ended
CTI BioPharma Investor Contacts:
1. Pidala et al, Oral Presentation, Abstract 355, ASH 2020.
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