RX-0201 is a first-in-class signal inhibitor that directly blocks the production of Akt, a protein kinase that plays a key role in cancer progression.
The clinical trial of RX-0201 was intended primarily to determine the safety and tolerability of the drug in patients with advanced cancer. The trial has demonstrated that the dose limiting toxicity of RX-0201 occurs in the form of fatigue. No other serious adverse reactions such as hematological toxicities were observed in this study.
Phase II of the RX-0201 clinical trial is expected to begin in early 2007 in patients with advanced renal cell carcinoma who have failed previous treatments.
In January 2005, the company received orphan drug designation from the FDA for RX-0201 for five cancer indications, including renal cell carcinoma, ovarian cancer, glioblastoma, stomach cancer, and pancreatic cancer. Companies that receive orphan drug designation are provided an accelerated review process, tax advantages, and seven years of market exclusivity in the US.
In the future, the Rexahn plans to apply RX-0201 to the treatment of orphan indications and other cancers.