June 25, 2013
/PRNewswire/ -- Cell Therapeutics, Inc. (CTI) (NASDAQ and MTA: CTIC) today issued the following statement regarding the notification of the U.S. Food and Drug Administration (the "FDA") partial clinical hold on tosedostat (IND 075503), the Company's aminopeptidase inhibitor under development for the treatment of blood-related cancers, that is being studied in an investigator-sponsored trial and not by CTI. CTI's primary development programs are the ongoing Phase 3 trial of pacritinib, the Company's JAK2/FLT3 inhibitor being evaluated for patients with myelofibrosis, and the post-approval commitment study of PIXUVRI
This is a
partial clinical hold
of only part of the clinical work requested under the investigational new drug ("IND") application. Under a partial clinical hold, although a specific protocol or part of a protocol is not allowed to proceed, other parts of such protocol or other protocols are allowed to proceed under the IND. With this partial clinical hold, the Company may not enter new patients onto any of the ongoing tosedostat protocols until agreement is reached with the FDA.
After a study has been placed on partial clinical hold, the sponsor provides additional information and discusses the adverse event with the FDA to understand whether or not it was associated with the drug or due to another cause. The Company has begun work to comply with the FDA request and expects to be able to submit these data to the FDA in the coming weeks. For additional information, please refer to the Current Report on Form 8-K filed by the Company on
June 24, 2013
Recent Tosedostat Publication
published results from the OPAL Phase 2 study of tosedostat in elderly patients with relapsed or refractory acute myeloid leukemia (AML). The trial showed that once-daily oral tosedostat resulted in a disease control rate of 51 percent. Subset analyses suggested the greatest benefit occurred in the difficult-to-treat patients with prior myelodysplastic syndrome (MDS) or those that had received prior hypomethylating therapy (HMA). Adverse events were mild, predictable and manageable.
About Acute Myeloid Leukemia (AML) and Myelodysplastic Syndrome (MDS)
Approximately 14,590 new cases of AML were expected to be diagnosed in
the United States
an estimated 30,993 people were living with (or were in remission from) AML.
Although AML can occur at any age, adults aged 60 years and older are more likely to develop the disease than younger people. AML is a cancer characterized by the rapid growth of abnormal white blood cells that accumulate in the bone marrow and interfere with the production of normal blood cells. AML may develop from the progression of other diseases such as myelodysplastic syndrome (MDS), a blood cancer that also affects the bone marrow leading to a decrease in circulating red blood cells. AML is the most common acute leukemia affecting adults, and its incidence increases with age. The symptoms of AML are caused by replacement of normal bone marrow with leukemic cells, which causes a drop in red blood cells, platelets, and normal white blood cells leading to infections and bleeding. AML progresses rapidly and is typically fatal within weeks or months if left untreated. Although a substantial proportion of younger individuals who develop AML can be cured, AML in the elderly typically responds poorly to standard therapy with few complete remissions.
Cell Therapeutics, Inc. (NASDAQ and MTA: CTIC) is a biopharmaceutical company committed to the development and commercialization of an integrated portfolio of oncology products aimed at making cancer more treatable. CTI is headquartered in
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This press release contains "forward-looking" statements that are made pursuant to the Safe Harbor provisions of the Private Securities Litigation Reform Act of 1995, including, but not limited to, statements concerning our anticipated product development and our ability to submit the above-referenced tosedostat data to the FDA in the coming weeks. Such statements are subject to risks and uncertainties, the outcome of which could materially and/or adversely affect actual future results and the trading price of the Company's securities. Specifically, the risks and uncertainties that could affect our ability to address FDA requests or the development of tosedostat more generally include risks associated with preclinical and clinical developments in the biopharmaceutical industry in general and with tosedostat in particular including, without limitation, the potential failure of tosedostat to prove safe and effective as determined by the FDA and/or the European Medicines Agency; determinations by regulatory, patent, and administrative governmental authorities; competitive factors; technological developments; costs of developing and producing tosedostat; the risk that the FDA may expand its information request or initiate a complete clinical hold or take other actions; and the risk factors listed or described from time to time in the Company's filings with the Securities and Exchange Commission, including, without limitation, the Company's most recent filings on Forms 10-K, 10-Q and 8-K. The Company can give no assurances that any results or events projected or contemplated by its forward-looking statements will in fact occur and the Company cautions you not to place undue reliance on these statements. The Company undertakes no duty to update these forward-looking statements to reflect any future events, developments or otherwise.