For more than two years, former Dallas Maverick basketball player Ray Johnston has been cancer-free. His advanced, highly aggressive acute promyelocytic leukemia (APL) was totally eradicated in 2010, just four months after beginning treatment with CytRx Corporation’s (NASDAQ:CYTR) cancer drug candidate tamibarotene. Since then, Mr. Johnston has continued taking tamibarotene tablets every other month to guard against a relapse. “Every day without leukemia is a celebration,” said Mr. Johnston, who leads a very active lifestyle with the band he formed following his diagnosis. The Ray Johnston Band is booking about 100 shows across the U.S. this year with a sound described by Johnston as halfway between Zac Brown and Dave Matthews. Tamibarotene was developed to be more potent and avoid some of the toxic side effects of all trans-retinoic acid (ATRA)—the current first-line treatment for APL. CytRx is currently testing tamibarotene as a third-line treatment for this form of leukemia. CytRx also is evaluating tamibarotene in combination with other chemotherapeutic agents in a late-stage, global Phase 2b clinical trial as a first-line treatment for non-small cell lung cancer (NSCLC). Positive signs of efficacy in patients with APL such as Ray Johnson further bolster the case for tamibarotene’s drug activity, providing hope for its success in patients with lung cancer, which is responsible for more deaths than breast, prostate and ovarian cancers combined. Last month an independent group of oncologists and biostatistitions who comprise the Data Safety Monitoring Board for this global trial recommended moving forward with clinical testing following an interim review of safety data. The Company expects to report trial results next year. Mr. Johnston was diagnosed with APL at the age of 24, ending his brief career as a professional basketball player with the Dallas Mavericks. For the following six years, he was treated with multiple therapies for APL approved by the U.S. Food and Drug Administration (FDA), including ATRA, as well as anthracycline chemotherapy, Mylotarg® and arsenic trioxide (ATO). He even underwent a blood stem cell transplant. Although his leukemia went into remission several times, it always came back with a vengeance. In fact, a PET scan performed just prior to his treatment with tamibarotene showed more than 30 tumors throughout his body.
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. The FDA and CytRx granted the request.“Two years ago when I was 31, I asked Dr. Collins if I’d reach 33. His answer then was ‘unless we find a new drug, I don’t think so.’ But here I am,” said Mr. Johnston. “We are delighted that Ray continues to show no signs of leukemia,” said Steven A. Kriegsman, CytRx President and CEO. “Our corporate goal is to improve treatments for people with cancer. We are grateful to be a part of the success that Ray has achieved and hope that tamibarotene can help those suffering from NSCLC.” Mr. Johnston has also achieved success with his band. The Ray Johnston Band has been featured on Dallas Mavericks' owner Mark Cuban's HDNet reality series, The Ray Johnston Band Road Diaries, and has opened for world renowned performers such as Jimmy Buffet, Cheap Trick, The Cure, The Fray, and Los Lonely Boys. About Tamibarotene Tamibarotene is an orally available, rationally designed, synthetic retinoid compound. CytRx holds the North American and European rights to tamibarotene as a treatment for APL and certain other cancers. Tamibarotene was approved in Japan in 2005 where it is marketed as a second-line treatment for APL. In December 2010, CytRx initiated a global Phase 2b clinical trial in patients with advanced NSCLC. In this randomized clinical trial, patients with advanced NSCLC are treated with paclitaxel plus carboplatin and either tamibarotene or placebo. The primary objective of this trial is to determine the objective response rate (complete and partial responses) and progression-free survival. Secondarily, the trial will evaluate overall survival, quality-of-life and examine the pharmacokinetics of tamibarotene in this population, among other measures. 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 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.About CytRx Corporation CytRx Corporation is a biopharmaceutical research and development company specializing in oncology. The CytRx oncology pipeline includes three programs in clinical development for cancer indications: aldoxorubicin (formerly known as INNO-206), tamibarotene and bafetinib. With its tumor-targeted doxorubicin conjugate aldoxorubicin, CytRx has initiated an international Phase 2b clinical trial as a treatment for soft tissue sarcomas, has completed its Phase 1b/2 clinical trial primarily in the same indication, recently initiated a Phase 2 trial for patients with advanced pancreatic ductual adenocarcinomas, and plans to meet with the FDA in the second half of 2012 to discuss a potential Phase 3 pivotal trial as a therapy for patients with soft tissue sarcomas whose tumors have progressed following treatment with chemotherapy. CytRx's pipeline also includes tamibarotene, which it is testing in a double-blind, placebo-controlled, international Phase 2b clinical trial in patients with non-small-cell lung cancer, and which is in a Phase 2 clinical trial as a treatment for acute promyelocytic leukemia (APL). The Company completed its evaluation of bafetinib in the ENABLE Phase 2 clinical trial in high-risk B-cell chronic lymphocytic leukemia (B-CLL), and plans to seek a partner for further development of bafetinib. For more information about the Company, visit 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 the patient described 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 in a timely fashion, cost-effectively or in commercial quantities in compliance with stringent regulatory requirements, 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, 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.