Insert Therapeutics, Receives FDA Approval for IT-101 Phase I Clinical Trial

Arrowhead Research Corporation announced has announced that that the U.S. Food and Drug Administration has approved the Investigational New Drug ("IND") application submitted by its majority-owned subsidiary Insert Therapeutics, Inc. Under the IND, Insert will conduct a Phase I study for IT-101, its first anti-cancer therapeutic, at the City of Hope in Duarte, California.
IT-101 is a nanotechnology-based therapeutic that combines Insert's patented Cyclosert™ technology and the anti-cancer compound camptothecin. Cyclosert is a linear cyclodextrin polymer that was invented at Caltech in the lab of Dr. Mark Davis, a professor in Chemical Engineering and founder of Insert. Insert holds an exclusive license to Cyclosert and has conducted numerous animal studies showing the safety of the Cyclosert delivery platform and the efficacy of IT-101.

"FDA clearance to begin clinical trials is another significant milestone for Insert on the road to becoming a leader in nanotechnology-based therapeutics," said R. Bruce Stewart, Arrowhead's Chairman. "This accomplishment reflects Insert's capability and commitment to commercialize the Cyclosert platform for cancer and other diseases."

Analogues of camptothecin, such as irinotecan and topotecan, realize nearly $1 billion in worldwide sales annually, despite the numerous unwanted side effects of these medications. In preclinical animal studies conducted by Insert, treatments with IT-101 appear to be substantially more effective, resulting in protracted anti-tumor activities, at significantly lower doses than all other treatments, including those with irinotecan. In some tumor types, IT-101 has caused complete remission of tumors.

The Phase I trial will be an open-label, dose-escalation clinical trial of IT-101 in patients with all types of cancer. The trial is expected to enroll between 24 and 48 patients and has been designed to determine safety and tolerability of IT-101.