13 September 2017 - - US-based biotech company Cancer Prevention Pharmaceuticals, Inc has received a total of USD 9.5m from its North America commercialization partner Sucampo Pharmaceuticals, Inc. (NASDAQ: SCMP), the company said.

Sucampo paid CPP USD4.5 m in option fees and invested USD 5m in CPP via a convertible note, all in accordance with the terms of the agreements that Sucampo and CPP entered into in January 2016.

The payments were triggered by recent positive results from a planned interim futility analysis of CPP's pivotal Phase 3 trial, CPP FAP-310, evaluating CPP-1X/sul for adults with familial adenomatous polyposis.

An Independent Data Monitoring Committee recently recommended continuation of the Phase 3 trial, which is fully enrolled and expected to be completed in 2018 unless there are extensions.

The US Food and Drug Administration and European Medicines Agency both granted CPP-1X/sul orphan drug status for treatment of FAP.

CPP received USD 8m from Sucampo in January 2016 upon signing a collaboration agreement that grants Sucampo the sole option to acquire an exclusive license to commercialize CPP-1X/sul in North America.

USD 3m of that initial payment was a one-time option fee; the remaining USD 5m was an investment in the form of a convertible note.

In connection with the original agreement, Sucampo, which is headquartered in Rockville, MD, agreed to provide another USD 9.5m, in the form of option payments and a convertible note investment, upon completion of the positive futility analysis milestone.

FAP is a rare genetic disease that if left untreated progresses to colorectal cancer in nearly 100% of patients. For most FAP patients, current medical practice recommends a lifetime of periodic monitoring as well as surgeries (FAP-related events).

These FAP-related events include surgical removal of the colon, rectum, surgical pouch, duodenum, and/or high risk adenomas.

The purpose of this randomized, double-blind, Phase 3 trial is to determine if the combination of eflornithine plus sulindac is superior to eflornithine or sulindac as single agents in delaying time to the first occurrence of any FAP-related event.

Cancer Prevention Pharmaceuticals, Inc. is developing therapeutics designed to reduce the risk of cancer and other diseases. CPP's pharmaco-prevention approach has been used with success in other disease categories such as cardiovascular, neurovascular, and infectious disease.

In addition to the CPP FAP-310 trial, CPP is co-sponsoring with the National Cancer Institute and SWOG a large Phase 3 trial in colon cancer survivors. CPP is also working collaboratively with nonprofit groups to support their clinical trials in neuroblastoma, gastric cancer, and early-onset type 1 diabetes. CPP is located in Tucson, Arizona.

Sucampo Pharmaceuticals is a biopharmaceutical company focused on the development and commercialisation of specialised medicines.

The company has a late-stage pipeline of product candidates in clinical development for orphan disease areas, including VTS-270, a mixture of 2-hydroxypropyl-B-cyclodextrins with a specific compositional fingerprint that has been granted orphan designation in the US and Europe and is in a pivotal Phase 2/3 clinical trial for the treatment of Niemann-Pick Disease Type C-1, a rare progressive genetic disorder.

VTS-270 has also been granted breakthrough therapy designation in the US Sucampo has an exclusive option for the North American rights to CPP-1X/sulindac, which is in Phase 3 development for the treatment of familial adenomatous polyposis and has been granted orphan drug designation in the US.