CTI BioPharma
CTI BioPharma develops novel therapies for rare blood-related cancers, focusing on myelofibrosis treatment with their approved drug VONJO® (pacritinib). Now a subsidiary of Sobi AB, CTI continues to evaluate pacritinib in various clinical trials for its unique JAK2/IRAK1 inhibition mechanism.
Overview of CTI BioPharma
CTI BioPharma focuses on developing novel therapies aimed at treating patients with rare blood-related cancers. The company's approach involves uncovering the scientific drivers underlying the disease to formulate effective treatments. Their key area of specialization is addressing significant unmet needs within blood-related cancers, particularly myelofibrosis. CTI BioPharma operates as a wholly-owned subsidiary of Sobi AB, which emphasizes the company's commitment to advancing in the pharmaceutical industry.
Pacritinib and VONJO
Pacritinib, marketed under the brand name VONJO®, is a novel oral kinase inhibitor developed by CTI BioPharma. It is approved in the United States for treating adults with intermediate or high-risk primary or secondary myelofibrosis with severe thrombocytopenia (platelet count below 50 x 10^9/L). VONJO is specifically tailored to inhibit JAK2 and IRAK1 without affecting JAK1, distinguishing it from other treatments. Pacritinib’s unique mechanism of action helps in reducing splenomegaly and constitutional symptoms and may mitigate the risks of cytopenias and immune dysfunction.
Clinical Trials and Studies
CTI BioPharma has conducted several significant clinical trials to evaluate the efficacy and safety of pacritinib. Notable trials include PERSIST-1, PERSIST-2, PAC203, and the PACIFICA trial. PERSIST-1 and PERSIST-2 were Phase 3 trials that assessed pacritinib’s performance compared to Best Available Therapy for myelofibrosis patients. The PACIFICA trial is a randomized Phase 3 study comparing pacritinib versus Physician’s Choice therapy. Additionally, the PRE-VENT study looked into pacritinib’s use in hospitalized patients with severe COVID-19. These trials highlight CTI BioPharma’s commitment to rigorous scientific evaluation.
Myelofibrosis Treatment Landscape
Within the United States, there are about 21,000 patients diagnosed with myelofibrosis, with approximately 7,000 experiencing severe thrombocytopenia. Pacritinib by CTI BioPharma stands out as the only drug to include patients with cytopenic myelofibrosis in Phase 3 trials, demonstrating its clinical activity in this subgroup. This focus on a niche patient population underscores the company’s dedication to innovating in blood-related cancer therapies where there is a significant unmet need.
Subsidiary Relationship with Sobi AB
CTI BioPharma operates as a wholly-owned subsidiary of Sobi AB. This relationship supports CTI BioPharma’s mission of acquiring, developing, and commercializing novel therapies for blood-related cancers. Being part of Sobi AB enables CTI BioPharma to leverage broader resources and expertise to advance their research and development initiatives, aiming to deliver impactful treatment options for patients.