CTI BioPharma to Submit a New Drug Application (NDA) for the Accelerated Approval of Pacritinib for the Treatment of Myelofibrosis Patients with Severe Thrombocytopenia
SEATTLE, Sept. 29, 2020 /PRNewswire/ -- CTI BioPharma Corp. (Nasdaq: CTIC) today announced that following a recent Pre-NDA meeting with the U.S. Food and Drug Administration ("FDA" or "the Agency"), the Company has reached an agreement to submit an NDA for the potential accelerated approval of pacritinib as a treatment for myelofibrosis patients with severe thrombocytopenia (platelet count less than 50 x 109/L). The NDA will be based on the available data from the Company's completed Phase 3 PERSIST-1 and PERSIST-2 trials and the Phase 2 PAC203 dose-ranging trial. The FDA has agreed to a rolling NDA submission which is expected to commence within a few weeks, with completion of the NDA submission anticipated in the first quarter of 2021. The ongoing Phase 3 PACIFICA trial is expected to be completed as a post-marketing commitment.
"Since the completion of the PAC203 Phase 2 dose-ranging trial, we have been working collaboratively with the FDA to identify an expeditious approval pathway for pacritinib in myelofibrosis patients with severe thrombocytopenia, a patient population with an important unmet medical need due to reduced survival and limited therapeutic options. During a recent Pre-NDA meeting, we identified a data package from the PERSIST-1, PERSIST-2 and PAC203 Phase 2 trials that will serve as the basis for an accelerated approval application. In particular, we discussed risk mitigation measures to address the FDA's prior concerns regarding safety," said Adam R. Craig, M.D., Ph.D., President and Chief Executive Officer of CTI Biopharma. "In myelofibrosis patients, severe thrombocytopenia occurs as a result of disease or drug-related toxicity from current therapies. There is no approved drug that specifically addresses the unmet need of the myelofibrosis patients who have severe thrombocytopenia. Pacritinib has demonstrated clinical benefit in treating these patients in multiple trials and now has the potential to become a new treatment option for treatment-naïve and second-line myelofibrosis patients in 2021."
Conference Call and Webcast
CTI will host a conference call and webcast to discuss this announcement tomorrow, September 30, 2020 at 8:30 AM ET. To access the live call by phone please dial (877) 735-2860 (domestic) or (602) 563-8791 (international); the conference ID is 9275344. A live audio webcast of the event may also be accessed through the "Investors" section of CTI's website at www.ctibiopharma.com. A replay of the webcast will be available for 30 days following the event.
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. Myelofibrosis patients with severe thrombocytopenia have limited treatment options, creating a significant area of 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.
In March 2008, pacritinib received orphan drug designation for the treatment of primary myelofibrosis (MF), post-polycythemia vera MF, and post-essential thrombocythemia MF.
In August 2014, pacritinib was granted Fast Track designation by the FDA for the treatment of intermediate and high risk myelofibrosis, including, but not limited to, patients with disease-related thrombocytopenia (low platelet counts); patients experiencing treatment-emergent thrombocytopenia on other JAK2 inhibitor therapy; or patients who are intolerant of or whose symptoms are not well controlled (sub-optimally managed) on other JAK2 therapy.
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Company Codes: NASDAQ-SMALL:CTIC