Eugene Gerner (left) is a UA professor emeritus and co-founder of Cancer Prevention Pharmaceuticals, Inc. Jeffrey Jacob (right) is the company's CEO. (Photo by Chris Mooney)
Eugene Gerner (left) is a UA professor emeritus and co-founder of Cancer Prevention Pharmaceuticals, Inc. Jeffrey Jacob (right) is the company's CEO. (Photo by Chris Mooney)

Licensing Agreement Brings UA-Developed Drug to Cancer Patients Outside U.S.

A spin-off company founded by a former UA professor and cancer researcher has entered a licensing agreement for Japan and Europe.
Jan. 21, 2014

A drug developed at the University of Arizona will be developed and commercialized in Europe and Japan through a licensing agreement coordinated by Tech Launch Arizona, the UA's research commercialization initiative.

The agreement is between Tucson-based company Cancer Prevention Pharmaceuticals Inc., which was co-founded by UA professor emeritus Eugene Gerner, and Swiss-based Tillotts Pharma AG, a subsidiary of Zeria Pharmaceutical Co. Ltd. in Tokyo. The agreement provides for European and Japanese rights to develop and commercialize an anti-cancer combination drug called CPP-1X/sulindac, which resulted from basic cancer research done at the UA. Doctors use CPP-1X/sulindac to treat familial adenomatous polyposis, or FAP, an orphan disease, and other gastrointestinal conditions. CPP specializes in prevention therapies for people with an elevated risk of cancer.

"This licensing agreement is very important for us and will give us a multiyear runway and fund our Phase III approval clinical trial in familial adenomatous polyposis," said Jeffrey Jacob, CEO of Cancer Prevention Pharmaceuticals. "Tillotts, Zeria's EU subsidiary, is focused on the gastrointestinal area, which is the niche for our product. They will provide funding and lead all the marketing and commercialization efforts in the European Union and Japan for our product."

Familial adenomatous polyposis, or FAP, is a genetic predisposition to colorectal cancer that comes with a nearly 100 percent risk of developing colon cancer before age 40. Patients with the classic presentation of FAP begin to develop multiple benign polyps in the colon in their early teenage years. Eventually, the colon becomes carpeted with hundreds to thousands of polyps and the polyps almost always become cancerous if left untreated. The only option for most people with FAP is to remove the entire colon in their late teens or early 20s, and in many cases a lifetime of surgeries lies ahead to control the condition.

CPP's lead product, CPP-1X/sulindac, is being developed to minimize the occurrence and/or recurrence of polyps and tumors associated with FAP and offers the potential of saving patients from surgery. CPP-1X/sulindac is currently being evaluated in a Phase III clinical trial to test the drug's ability to stop cancerous polyps and tumors from recurring or even occurring in the first place.

CPP-1X/sulindac is a two-drug combination that targets different pathways important in cancer development, according to Gerner, who retired from the Department of Cellular and Molecular Medicine in the UA College of Medicine in 2012. The drug combo was developed over years of research to elucidate the molecular pathways underlying cancer formation and how to target them with those drugs.

CPP was founded in 2008 to apply decades' worth of systematic, basic research led by Gerner and former UA researcher Frank Meyskens to improve clinical practice. The work then was successfully translated to the clinic with the help of the National Cancer Institute (NCI) and various research partners. Sulindac belongs to of the class of non-steroidal anti-inflammatory drugs, with aspirin being a notable member. Sulindac targets the inflammatory pathway.

The agreement can potentially be expanded into additional indications for which CPP-1X/sulindac has shown promise. CPP will be responsible for product development and Tillotts for regulatory and marketing for the respective markets. In addition, Zeria will be responsible for development and marketing in Japan. CPP continues to retain all rights to CPP-1X/sulindac outside of Europe and Japan and outside the gastrointestinal field.

"We are very pleased to partner with Tillotts and Zeria, companies that have shown strength and focus in the gastrointestinal therapeutic area in Europe and Japan," said Jacob. "We are delighted to share the benefits of both their scientific and business expertise in the development and future commercialization of CPP-1X/sulindac. Tillotts and Zeria share our belief in the great potential of this product in familial adenomatous polyposis, an orphan indication with limited treatment options."