Potential HCC drug stabilized disease progression

Mipsagargin was effective in stabilizing hepatocellular carcinoma progression and was well tolerated among a cohort of patients with hepatocellular carcinoma, according to results of a phase 2 clinical trial presented at the Gastrointestinal Cancers Symposium.

“These results demonstrate the potential that mipsagargin has to treat one of the world’s deadliest cancers,” researcher Devalingam Mahalingam, MD, PhD, of the Cancer Therapy and Research Center, University of Texas Health Science Center at San Antonio, said in a press release. “In addition to observing prolonged disease stabilization in some patients, we noted anecdotal evidence that patients had improved performance status with much less fatigue and side effects associated with other anti-cancer therapeutics.”

Mipsagargin (G-202, GenSpera) is a thapsigargin prodrug that targets the enzyme prostate-specific membrane antigen and aims to reduce blood flow within tumors, without affecting blood flow within normal tissues, according to the release.

A cohort of 22 patients with HCC, whose disease progressed after failing treatment with sorafenib (Nexavar, Bayer and Onyx Pharmaceuticals), were treated with mipsagargin through IV infusion on days 1, 2 and 3 of a 28-day cycle. Thirty-five percent of patients received at least five cycles of mipsagargin for 7 months, according to the abstract. More than 70% of the patients exhibited disease stabilization. Patients who underwent dynamic contrast-enhanced MRI showed an average 56% decrease in tumor values, suggesting mipsagargin helped reduce early arterial blood flow in hepatic lesions, according to the research.

One patient experienced congestive heart failure and three patients experienced a creatinine increase, acute renal failure and acute kidney injury; all serious adverse events were mipsagargin-related.

“We are very encouraged with the positive results from this phase 2 trial that demonstrate the tolerability and show indications of effectiveness of mipsagargin in advanced liver cancer patients,” Craig Dionne, PhD, CEO of GenSpera, said in the release. “Mipsagargin is a first-in-class agent with 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 3 trial.”

For more information:

Mahalingam D. Abstract #301. Presented at: Gastrointestinal Cancers Symposium; Jan. 16-18, 2015; San Francisco.

Disclosure: Relevant financial disclosures were not provided by the researchers.