SEATTLE, July 18, 2018
SEATTLE, July 18, 2018 /PRNewswire/ -- CTI BioPharma Corp. (NASDAQ:CTIC) today announced that it recently conducted a Type B meeting with the U.S. Food and Drug Administration (FDA) for its lead product candidate pacritinib. The purpose of the meeting was to discuss the regulatory pathway for pacritinib. Based on FDA feedback, the Company plans to conduct a randomized Phase 3 study in patients with myelofibrosis. The dosing for the Phase 3 study will be determined using the results of the PAC203 Phase 2 study. PAC203 is expected to complete enrollment by the end of 2018, with full top-line data expected in the second quarter of 2019. The new Phase 3 study is expected to open in 2019.
"We have received the clarity we requested from the FDA on a regulatory path to possible approval in the U.S., and look forward to using all available data, including pharmacokinetic analyses, to select the optimal dose for a new Phase 3 study," said Adam R. Craig, M.D., Ph.D., President and Chief Executive Officer of CTI BioPharma. "The new study is expected to address the unmet medical needs of patients with myelofibrosis, particularly those with severe thrombocytopenia. We plan to request an additional meeting with the FDA, after the second interim analysis, to discuss interim data from PAC203 and the design of the new Phase 3 study."
Following a planned interim data review in June 2018 by the Independent Data Monitoring Committee (IDMC) of PAC203, CTI BioPharma announced that a second interim review will be conducted in the third quarter of 2018. The IDMC did not identify any drug- or dose-related safety concerns and specifically did not identify any concerns about cardiac or bleeding events. The PAC203 study is evaluating the safety and efficacy of three dosing schedules (100 mg once daily, 100 mg twice daily and 200 mg twice daily) over 24 weeks in patients with myelofibrosis previously treated with ruxolitinib. The study is designed to enroll up to 150 patients and is expected to complete enrollment before the end of 2018. More information on the PAC203 trial can be found at ClinicalTrials.gov at https://clinicaltrials.gov/ct2/show/NCT03165734.
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.
About Myelofibrosis and Myeloproliferative Neoplasms
Myelofibrosis is one of three main types of myeloproliferative neoplasms (MPN), which are a closely related group of progressive blood cancers. The three main types of MPNs are primary myelofibrosis (PMF), polycethemia vera (PV) and essential thrombocythemia (ET).
Myelofibrosis is a serious and life-threatening bone marrow disorder caused by the accumulation of malignant bone marrow cells that triggers an inflammatory response and scars the bone marrow. The replacement of bone marrow with scar tissue limits its ability to produce red blood cells, prompting the spleen and liver to take over this function. Symptoms that arise from this disease include enlargement of the spleen, anemia, extreme fatigue and pain.
The estimated prevalence of MPNs suggest there are approximately 300,000 people living with the disease in the U.S., of which myelofibrosis accounts for approximately 18,000 patients. In Europe, there is a wide variation of prevalence observed across data sources. Myelofibrosis has a median age of 64 at the time of diagnosis and is a progressive disease with approximately 20 percent of patients eventually developing acute myeloid leukemia (AML). The median survival for high-risk myelofibrosis patients is less than 1.5 years, while the median survival for patients with myelofibrosis overall is approximately 6 years.
About CTI BioPharma Corp.
CTI BioPharma Corp. is a biopharmaceutical company focused on the acquisition, development and commercialization of novel targeted therapies covering a spectrum of blood-related cancers that offer a unique benefit to patients and healthcare providers. The CTI BioPharma lead product candidate, pacritinib, is being developed for the treatment of patients with myelofibrosis. CTI BioPharma is headquartered in Seattle, Washington.
This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934 and the Private Securities Litigation Reform Act of 1995. These forward-looking statements include statements regarding our expectations regarding, the timing of and results from clinical trials and pre-clinical development activities related to pacritinib, the potential efficacy, safety profile, future development plans, addressable market, regulatory success and commercial potential of pacritinib, the anticipated timing of regulatory submissions , the efficacy of, and potential changes to, our clinical trial designs and anticipated enrollment, our ability to successfully develop and achieve milestones in the development of pacritinib, and the anticipated benefits of pacritinib. 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, many of which are beyond our control, include, but are not limited to: clinical trials may not demonstrate safety and efficacy of any of our or our collaborators' product candidates; our assumptions regarding our planned expenditures and sufficiency of our cash to fund operations may be incorrect; our efforts to advance our pipeline may not be successful; any of our or our collaborators' product candidates may fail in development, may not receive required regulatory approvals, or may be delayed to a point where they are not commercially viable; we may not achieve additional milestones in our proprietary or partnered programs; the impact of competition; the impact of expanded product development and clinical activities on operating expenses; adverse conditions in the general domestic and global economic markets; as well as the other risks identified in our filings with the Securities and Exchange Commission. These forward-looking statements speak only as of the date hereof and we assume no obligation to update these forward-looking statements, and readers are cautioned not to place undue reliance on such forward-looking statements.
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CTI BioPharma Contacts:
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SOURCE CTI BioPharma Corp.