CytRx Corporation (Nasdaq:CYTR), a biopharmaceutical company specializing in oncology, today announced that an advanced form of acute promyelocytic leukemia, or APL, has been eradicated in former NBA player Ray Johnston following treatment with CytRx’s experimental cancer drug tamibarotene. Mr. Johnston was afflicted with a particularly aggressive type of APL called chloromas and had previously failed other approved therapies. More than 30 tumors were detected throughout his body prior to treatment with tamibarotene, which was administered in tablet form on a compassionate use protocol. “Following four months of treatment with tamibarotene, the disease was totally eliminated,” said Mr. Johnston. “In January, my PET scan showed a significant decrease in the disease, and in April it confirmed that the leukemia was completely gone. Last week marked my six-month anniversary of being cancer-free.” Mr. Johnston will continue taking orally administered tamibarotene every other month as an added measure given his history of relapses. Mr. Johnston, 31, was diagnosed with APL six years ago, ending his brief career as a Dallas Maverick. He previously was treated with FDA-approved therapies for APL, as well as with a blood stem cell transplant. He has experienced several remissions of his leukemia, but has always relapsed. In December 2009, his physician, Dr. Robert Collins at the University of Texas Southwestern Medical Center, requested tamibarotene from CytRx on a compassionate use basis, and the request was granted. “We wanted to give tamibarotene a try for Ray given the fact that his APL has relapsed several times following treatment with all trans-retinoic acid (ATRA), anthracycline chemotherapy and arsenic trioxide (ATO), the current first- and second-line therapies. There currently is no approved third-line therapy for APL,” said Dr. Collins. CytRx is conducting the Phase 2 STAR-1 registration trial under a Special Protocol Assessment to evaluate the efficacy and safety of tamibarotene in patients with third-line APL. In Japan, tamibarotene was approved in 2005 and is marketed for the treatment of second-line APL.
“I’ve regained my life following treatment with tamibarotene,” added Mr. Johnson. “It is the best I have felt since I joined the Dallas Mavericks.” Following his diagnosis, Mr. Johnston formed the band: The Ray Johnston Band, and has been featured on Dallas Mavericks’ owner Mark Cuban’s HDNet reality series – The Ray Johnston Band Road Diaries. Currently, the Ray Johnston Band is on tour in North America and has opened for Jimmy Buffet, The Cure, The Fray, and Los Lonely Boys, all internationally renowned performers.Last week, CytRx announced that a 44-year-old female patient with advanced APL showed no evidence of disease in the blood cells and/or bone marrow following treatment with tamibarotene. This patient is enrolled in CytRx's Phase 2 STAR-1 registration trial, which is evaluating the efficacy and safety of this drug candidate as a third-line treatment for APL. She is one of three previously reported advanced APL patients to achieve a hematologic complete response with tamibarotene treatment in the STAR-1 trial. A detailed report of this patient, who also had failed first- and second-line APL treatments as well as a bone marrow transplant, was published in the July online issue of the peer-reviewed British Journal of Haematology with publication in the print edition expected later this year. Steven A. Kriegsman, CytRx President and CEO, said, “We are thrilled with Ray’s response to tamibarotene. As important, we are highly encouraged about tamibarotene’s prospects now that several patients afflicted with advanced APL who have failed multiple other treatments are reporting total elimination of this disease with our drug. We estimate tamibarotene’s market opportunity in refractory APL in the U.S. alone to approach $20 million annually and, with an expanded label to include refractory, maintenance and front-line therapy, to increase to $150 million annually in the U.S. and Europe. Furthermore, in order to reach blockbuster status, we are evaluating the development of tamibarotene in other deadly cancers with large market opportunities.”
About TamibaroteneTamibarotene is an orally available, rationally designed, synthetic retinoid compound that was developed to potentially avoid toxic side effects of ATRA by binding to its molecular target more selectively than ATRA. CytRx holds the North American and European rights to tamibarotene as a treatment for APL. The efficacy and safety of tamibarotene as a third-line treatment for APL is currently being evaluated in the Phase 2 STAR-1 registration trial, which is being conducted under a Special Protocol Assessment (SPA). In June 2009 CytRx reported that, of the 11 patients enrolled in the STAR-1 trial at that time, three (27%) achieved a hematologic complete response, and four (36%) a morphologic leukemia-free state. In December 2009, favorable preliminary results from the STAR-1 registration trial were presented at the Annual Meeting of the American Society of Hematology (ASH). The U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for APL and Fast Track Designation for the use of tamibarotene in patients with relapsed or refractory APL following treatment with ATRA and ATO. In addition, tamibarotene has been granted orphan medicinal product status by the European Medicines Agency for the treatment of APL. The efficacy of orally administered tamibarotene was demonstrated in two Phase 2 studies conducted in Japan in a total of 63 Japanese subjects with APL. The overall complete response rate in these subjects was 60%. In subjects experiencing their first relapse, the overall complete response rate was 81%. A dose escalation study with tamibarotene combined with ATO in patients with relapsed APL is currently being conducted by Northwestern University under the leadership of Dr. Jessica Altman, Assistant Professor of Medicine, at the Northwestern University Feinberg School of Medicine. In this multi-center Phase 1 trial, between 16 and 22 relapsed APL subjects in three dose groups will be treated with two to three six-week cycles of intravenous ATO and self-administered oral tamibarotene tablets with two to six weeks between cycles. A total of 10 subjects will be enrolled at the maximum dose for one or two additional cycles of therapy and evaluated for disease remission. CytRx plans to conduct a subsequent Phase 2 trial with tamibarotene at the dose at which at least five of six subjects tolerate the treatment or the maximum dose used in this trial.
Tamibarotene also has demonstrated statistically significant anti-tumor activity in combination with glucocorticoids in an animal trial for multiple myeloma - an incurable malignant tumor of the plasma cells of bone marrow. CytRx retains an option to expand its licenses for the use of tamibarotene in other fields in oncology, including multiple myeloma, myelodysplastic syndrome and certain solid tumors in the U.S., and multiple myeloma, myelodysplastic syndromes and solid tumors other than hepatocellular carcinoma in Europe.About Acute Promyelocytic Leukemia (APL) APL is diagnosed in approximately 1,500 new patients in the U.S. annually. It is a subtype of acute myelogenous leukemia, a cancer of the blood and bone marrow. In APL, an abnormal accumulation of immature granulocytes called promyelocytes in the bone marrow results in a reduction in the production of normal red blood cells and platelets, resulting in anemia and thrombocytopenia. Either leukopenia (low white cell count) or leukocytosis (high white cell count) may be observed in the peripheral blood. Symptoms include fatigue, weakness, shortness of breath from anemia, easy bruising and bleeding from thrombocytopenia and coagulopathy, and fever and infection from lack of normal white blood cells. About CytRx Corporation CytRx Corporation is a biopharmaceutical research and development oncology company engaged in the development of high-value human therapeutics. The CytRx oncology pipeline includes three programs in clinical development for cancer indications: INNO-206, bafetinib and tamibarotene. In May 2010, the Company announced initiation of a Phase 2 clinical trial with bafetinib as a treatment for high-risk B-cell chronic lymphocytic leukemia (B-CLL). CytRx also plans to initiate Phase 2 clinical trial in advanced prostate cancer and a Phase 1 trial in glioblastoma multiforme (a common and aggressive type of primary brain tumor). CytRx has announced plans to initiate three Phase 2 clinical trials with its oncology candidate INNO-206 as a treatment for pancreatic cancer, gastric cancer and soft tissue sarcomas. In addition, CytRx is developing two drug candidates based on its industry-leading molecular chaperone technology, which aims to repair or degrade misfolded proteins associated with disease. CytRx also maintains a 17% equity interest in publicly traded RXi Pharmaceuticals, Inc. (NASDAQ:RXII). For more information on the Company, visit http://www.cytrx.com. Forward-Looking Statements This press release contains forward-looking statements within the meaning of Section 21E of the Securities Exchange Act of 1934, as amended. Such statements involve risks and uncertainties that could cause actual events or results to differ materially from the events or results described in the forward-looking statements, including risks relating to possible reappearance of cancer in patients cited in this press release, the failure of other patients to response to our drug candidate or possible unreported cases of adverse patient outcomes, the ability to obtain regulatory approval for clinical testing of tamibarotene, including additional clinical trials for patients with APL, the scope of clinical testing that may be required by regulatory authorities and the timing and outcome of further clinical trials, the risk that any future human testing of tamibarotene might not produce results similar to those seen in animals, risks related to CytRx's ability to manufacture tamibarotene and its other drug candidates, including tamibarotene, in a timely fashion, cost-effectively or in commercial quantities in compliance with stringent regulatory requirements, risks related to CytRx's ability to enter into partnerships or other transactions to advance the clinical development of its portfolio of drug candidates, risks related to CytRx's need for additional capital or strategic partnerships to fund its ongoing working capital needs and development efforts, including any future clinical development of tamibarotene, risks related to the future market value of CytRx's investment in RXi and the liquidity of that investment, and the risks and uncertainties described in the most recent annual and quarterly reports filed by CytRx with the Securities and Exchange Commission and current reports filed since the date of CytRx's most recent annual report. All forward-looking statements are based upon information available to CytRx on the date the statements are first published. CytRx undertakes no obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise.