Pennsylvania-based Locus Pharmaceuticals has submitted an investigational new drug (IND) application to the FDA to begin a Phase I human clinical study of the company's lead compound, LP-261, an orally administered small molecule for the treatment of cancer.

LP-261 has been shown to be effective in cancer cells that are resistant to taxol, vinblastine and Gleevec. LP-261 does not appear to be a substrate for MDR pumps that pump out drugs from the tumor cell. In preclinical studies, LP-261 demonstrates broad anti-tumor and anti-angiogenic activity both in vitro and in vivo, including tumor regression in solid tumor xenograft models (e.g., non-small cell lung cancer and colon). It is anticipated that the dual effects on cell growth and proliferation could allow broad application of LP-261, including its use in combination therapy.

"All of the approved tubulin drugs target either the taxane or vinca sites, are parenterally administered and are natural products which can present manufacturing or toxicity issues," said H. Joseph Reiser, chairman and CEO of Locus. "We believe that LP-261, as an oral, synthetic compound targeting the colchicine site, could have a very exciting profile across various solid and blood-borne cancer types."