Isis begins clinical study of ISIS-STAT3 in patients with metastatic liver cancer

Isis Pharmaceuticals, Inc. (NASDAQ: ISIS) announced the initiation of a Phase 1b/2a clinical study of ISIS-STAT3_Rx in patients with advanced metastatic hepatocellular carcinoma (HCC).  HCC is a tumor of the liver and is one of the most common cancers worldwide with at least one million new cases diagnosed each year.  ISIS-STAT3_Rx is an antisense drug discovered by Isis and being developed to treat patients with cancers with a strong link to STAT3, such as HCC.  Isis licensed ISIS-STAT3_Rx to AstraZeneca as part of a strategic alliance in cancer. AstraZeneca is conducting this newly initiated study in patients with HCC.

 "We are pleased to be working with AstraZeneca on the development of ISIS-STAT3_Rx.  AstraZeneca's considerable resources, expertise and development capabilities to develop anti-cancer drugs strengthen substantially our ability to broadly evaluate ISIS-STAT3_Rx across a range of cancer types. Given the role of STAT3 in the growth and survival of tumors, we believe that ISIS-STAT3_Rx could be beneficial in the treatment of many different types of cancers.  As such, together with AstraZeneca, we have designed a broad development program to explore the effects of ISIS-STAT3_Rx in a number of different cancers," said B. Lynne Parshall , chief operating officer at Isis.  "Our early work showed that ISIS-STAT3_Rx treatment resulted in clear responses in patients with advanced cancer who were refractory to prior chemotherapy treatment, with an acceptable safety profile.  The new study builds on this early work and expands our evaluation of ISIS-STAT3_Rx into liver cancer where STAT3 inhibition could also provide therapeutic benefit."

The Phase 1b/2a study will evaluate the safety and efficacy of ISIS-STAT3_Rx in patients with HCC who have failed first-line therapy.  The endpoints for the study include measurements of anti-tumor activity, STAT3 protein levels in tumor biopsies and STAT3-related biomarkers.  The study will also evaluate safety.   Although HCC is common, it is considered a very difficult to treat cancer that does not respond well to chemotherapy. 

"STAT3 is important for the growth, invasiveness and metastases of many types of cancer, which makes it a very attractive target. The potency we have observed in our preclinical cancer studies and the early evidence of clinical activity we observed in our Phase 1 study suggest that ISIS-STAT3_Rx could be an effective anti-tumor agent that is broadly applicable to many different types of cancer," said Brett Monia , Ph.D., senior vice president, antisense drug discovery at Isis.