Optimization of a lead drug candidate often takes a few years, as companies assess hundreds and sometimes thousands of compounds, seeking just one with the best overall pharmacological profile. By contrast, Dynamix Pharmaceuticals Ltd. says its structure-based discovery approach dubbed DynamixFit has let the firm select lead molecules with less than 100 compounds synthesized in under two years. Dynamix aims to apply its techniques on two main tracks: first, to discover drug candidates that intervene with the metabolism of cancer cells; and second, to selectively target specific cellular signaling pathways mediated by Type II kinase inhibitors.

Cornerstone Pharmaceuticals Inc. is saying little about its lead drug candidate, CPI-613, which entered its first Phase I/II human clinical trial in late 2008, but the company and its backers believe they are developing a potentially game-changing class of new molecules and technology for delivering them. Its Altered Energy Metabolism Directed platform specifically utilizes the difference in cancer cells’ metabolic appetite to facilitate selective delivery of a novel small molecule (CPI-613 being the first example) into the cells’ mitochondria. Similarly the company’s Emulsiphan leverages those same metabolic differences to deliver drugs specifically to the cancer cell cytoplasm.

As reseachers uncover linkages between tumor formation and cellular metabolism, start-ups and their venture backers see an opportunity to create a new class of cancer medicines. In this issue, we profile Advanced Cancer Therapeutics, Cornerstone Pharmaceuticals and Dynamix Pharmaceuticals.