GenSpera reports positive results from Phase II trial of mipsagargin to treat HCC

The Phase II results showed that mipsagargin appears to be effective and is well-tolerated by HCC patients.

Mipsagargin targets the enzyme prostate-specific membrane antigen (PSMA), which is highly expressed in tumor vasculature and prostate cancer cells.

The results show that the prodrug effectively stabilizes progression of HCC by reducing blood flow within tumors while not affecting blood flow within normal tissues.

GenSpera chief executive officer Craig Dionne said: "We are very encouraged with the positive results from this Phase II trial that demonstrate the tolerability and show indications of effectiveness of mipsagargin in advanced liver cancer patients.

"Mipsagargin is a first-in-class agent with a novel mechanism of action that is unlike any other drug being tested in patients with advanced liver cancer. Based on the results of this study, we intend to move forward with a large, global Phase III trial."

Compared to other vascular targeting agents, mipsagargin’s targeted approach differs by destroying established tumor vasculature rather than slowing the growth of new tumor blood vessels.

The PSMA-targeting agent is designed to reduce side effects commonly observed with other therapies, while maximizing activity against tumors.

The multi-site, single arm Phase II trial was designed to evaluate time to progression and response rate to treatment and it measured disease progression in 25 patients with advanced-stage liver disease and poor liver reserves who had failed first line treatment with sorafenib.