Cylene Pharmaceuticals has initiated a single-agent Phase II clinical trial of its lead Ribosomal RNA Biogenesis Inhibitor, quarfloxin (CX-3543), in patients with chronic lymphocytic leukemia (CLL), a cancer of the white blood cells and bone marrow.

Quarfloxin (CX-3543) is a small molecule that selectively targets a DNA protein complex that is amplified in cancer cells, leading to inhibition of ribosomal RNA biogenesis and induction of apoptotic cell death. In vitro tests have shown CLL cells taken from patients to be very sensitive to the apoptotic effects of Quarfloxin. In this open-label trial, CX-3543 will be administered to patients with CLL whose leukemia has progressed on prior treatment with a purine analog and a monoclonal antibody. This study is expected to enroll up to 25 patients at several leading cancer centers, including the Tower Oncology Group in Los Angeles, California, South Texas Accelerated Research Therapeutics (START) in San Antonio, Texas, and the Mayo Clinic Arizona in Scottsdale, Arizona.

"We are very pleased with the progress of our proprietary Ribosomal RNA Biogenesis Inhibition program. The initiation of the Company's first Phase II trial with our lead molecule is an important milestone for Cylene," said Dr. William Rice, President and Chief Executive Officer of Cylene Pharmaceuticals.

"Because quarfloxin has demonstrated potent in vivo efficacy against a broad range of tumors and a considerable therapeutic window in animal xenograft models, we also plan on initiating additional Phase II studies in other cancer indications," added Dr. Daniel Von Hoff, Cylene's Co-Founder and Vice President, Medical Affairs. "Moreover, quarfloxin appears to have promise in combination with other compounds, and it has been well-tolerated in patients with no substantial toxicities."

About Quarfloxin (CX-3543), a Ribosomal RNA Biogenesis Inhibitor (RBI)

Quarfloxin is a ground-breaking small-molecule targeted cancer therapeutic derived from the validated fluoroquinolone class of drugs. Rationally designed to selectively inhibit ribosomal RNA (rRNA) Biogenesis in cancer cells, quarfloxin disrupts the interaction between the Nucleolin protein and a G-quadruplex DNA structure in the ribosomal DNA (rDNA) template, a critical interaction for rRNA Biogenesis and one that is amplified in cancer cells. As a result, quarfloxin, selectively induces apoptotic cell death in cancers. Many commercialized cancer therapeutics act indirectly on rRNA Biogenesis through upstream modulators, but quarfloxin is the first agent to directly target this cancer-specific aberrant cell function.

About Cylene Pharmaceuticals, Inc.

Cylene Pharmaceuticals is a pharmaceutical company dedicated to the discovery, development and commercialization of targeted small-molecule drugs to treat life-threatening cancers. Using its proprietary Ribosomal RNA Biogenesis Inhibition Technology (RABIT(TM)) to design molecules that combat drug-resistant forms of cancer, Cylene has created multiple product candidates in different stages of development. Cylene's most advanced cancer agent, Quarfloxin (CX-3543), is a Ribosomal RNA Biogenesis Inhibitor (RBI) in Phase II clinical development in chronic lymphocytic leukemia and late-stage Phase I development in solid tumors. More information can be found at cylenepharma.

Cylene cautions you that statements included in this press release that are not a description of historical facts, including implied statements relating to future outcomes of clinical trials, may be forward-looking statements that are subject to risks and uncertainties. Actual results may differ materially from those set forth in this release due to the risks and uncertainties inherent in Cylene's business including, without limitation, risks related to difficulties or delays in, testing, obtaining regulatory approval for, producing and marketing its products; unexpected adverse side effects or inadequate therapeutic efficacy of its products that could delay or prevent product development or commercialization, the scope and validity of patent protection for its products; competition from other pharmaceutical or biotechnology companies; and its ability to obtain additional financing to support its operations. All forward-looking statements are qualified in their entirety by this cautionary statement and Cylene undertakes no obligation to revise or update this news release to reflect events or circumstances after the date hereof.

Cylene Pharmaceuticals
cylenepharma

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