The past decade has heralded an era of dramatic successes among molecular-targeting cancer dugs such as Avastin®, Herceptin®, Gleevec®, Erbitux®, and Rituxan®. Building upon the success of these molecular-targeting agents, the industry has sought to identify new Targeted Therapies that can be exploited to create entirely new classes of cancer drugs.
At Cylene, we pioneered the Ribosomal RNA Biogenesis Inhibitor (RBI) approach that selectively disrupts the association between the Nucleolin protein and a unique G-quadruplex DNA motif in ribosomal DNA (rDNA). Disrupting this Protein:DNA association inhibits the aberrant ribosomal RNA (rRNA) biogenesis in cancer cells and induces apoptotis. This approach maintains selectivity via a targeted approach while providing potentially broad activity against multiple types of cancers in the absence of toxicity to normal cells. Quarfloxin (CX-3543) is the first selective small molecule Ribosomal RNA Biogenesis Inhibitor to begin human clinical trials. The compound is now in early Phase II clinical trials in patients with chronic lymphocytic leukemia (CLL), as well as in late Phase I trials in patients with solid tumors or lymphomas.
Cylene's pipeline also includes CX-4945 which is a member of the CK2 Inhibitor Series that targets the CK2 protein kinase enzyme. CX-4945 is currently in preclinical development. Using its RABIT technology platform, Cylene has also developed the Pol I Inhibitor Series, which is a series of orally bioavailble Ribosomal RNA Biogenesis Inhibitors (the CX-3800 series). In addition, CX-3773 is a Ribosomal RNA Biogenesis Inhibitor which crosses the blood-brain-barrier and has the potential to treat brain metastases. The CX-1300 Series represents Cylene's fifth product discovery program. These antitumor agents block transcription in tumor cells through a mechanism of action that is unrelated to RBI technology.
