CTI BioPharma Establishes Accelerated Approval Pathway for Pacritinib in Treating Myelofibrosis Patients With Severe Thrombocytopenia
SEATTLE, Feb. 3, 2020 /PRNewswire/ -- CTI BioPharma Corp. (Nasdaq: CTIC) today announced that following a meeting with the U.S. Food and Drug Administration ("FDA" or "the Agency"), CTI has reached agreement on an accelerated approval pathway for pacritinib for the treatment of myelofibrosis patients with severe thrombocytopenia (platelet counts <50,000/µL). CTI will be amending the PACIFICA pivotal Phase 3 trial protocol to allow for the primary analysis of SVR rates on the first 168 patients, with an end-of-study analysis of TSS and OS following the full enrollment of 348 patients. If the primary endpoint of SVR is met following the planned review of data from the first 168 patients, CTI intends to submit a New Drug Application (NDA) under the FDA's subpart H regulations, subject to review of all available efficacy and safety data. Conversion to a regular approval of pacritinib would be anticipated following the successful end-of-study assessment of the secondary efficacy endpoints, and the completion of post-marketing requirements.
"Since the initiation of the PACIFICA trial in September 2019, we have been working diligently with the FDA to identify an expedited approval pathway for pacritinib for the treatment of myelofibrosis patients with severe thrombocytopenia," said Adam R. Craig, M.D., Ph.D., President and Chief Executive Officer of CTI Biopharma. "Severely thrombocytopenic myelofibrosis patients (platelet counts <50,000/µL) have reduced survival and very limited therapeutic options. Pacritinib has now demonstrated clinical benefit in this population in three clinical trials, including two prior randomized Phase 3 studies, so we believe that pacritinib has the potential to change the treatment paradigm in this area of serious unmet medical need."
Based on the new trial design, CTI expects to report primary SVR data by the end of 2021, with a potential NDA filing in early 2022 if the SVR data is positive. Final study efficacy data is expected in 2023.
Concurrent with this press release, CTI is announcing a $60 million rights offering. For further details, see the concurrent press release relating to the rights offering.
About Myelofibrosis and Severe Thrombocytopenia
Myelofibrosis is a type of bone marrow cancer that results in formation of fibrous scar tissue and can lead to severe anemia, weakness, fatigue and an enlarged spleen and liver. Patients with severe thrombocytopenia are estimated to make up more than one-third of patients treated for myelofibrosis, or approximately 17,000 people. Severe thrombocytopenia, defined as blood platelet counts of less than 50,000 per microliter, has been shown to result in overall survival rates of just 15 months. Thrombocytopenia in patients with myelofibrosis is associated with the underlying disease but has also been shown to correlate with treatment with ruxolitinib, which can lead to dose reductions, and as a result, may potentially reduce clinical benefit. Survival in patients who have discontinued ruxolitinib therapy is further compromised, with an average overall survival of seven to 14 months. There are currently no approved therapies available to treat myelofibrosis patients with severe thrombocytopenia or patients who have failed ruxolitinib treatment, thereby making this a significant unmet medical need.
Pacritinib is an investigational oral kinase inhibitor with specificity for JAK2, FLT3, 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. (Article from : www.drugs.com)