Atiprimod is an orally bio-available small molecule drug that displays multiple mechanisms of action. The drug has been shown to be anti-angiogenic, inhibit secretion of VEGF and IL-6, elicit an apoptotic response (programmed cellular death), and inhibit phosphorylation of key kinases involved in tumor progression and survival including Akt and STAT3.

Clinical development

Atiprimod is presently in three clinical trials: a Phase II trial in advanced carcinoid cancer patients, a Phase II extension trial in advanced carcinoid cancer patients and a Phase I/IIa clinical trial in relapsed or refractory multiple myeloma patients.

The open label, multi-center, single-agent Phase II trial with Atiprimod in advanced carcinoid cancer patients was initiated in November, 2006. In October 2007 this trial was extended to allow patients who successfully completed 12 treatment cycles to continue to receive Atiprimod treatment. This extension is limited to patients who have shown at least a 20% decrease in symptoms. In addition, a Phase I/IIa multi-center clinical trial of Atiprimod in relapsed or refractory multiple myeloma patients is in progress. Atiprimod has been given orphan drug designation to treat both carcinoid cancer and multiple myeloma.

Carcinoid cancer

Carcinoid tumors, or carcinoids, originate in hormone-producing cells of the gastrointestinal (GI) tract, the respiratory tract, the hepatobiliary (liver) system and the reproductive glands. The most common site of origin is the GI tract, with tumors often developing in the rectum, and other sections of the small intestine. Approximately 7,000 cases of carcinoid cancer are diagnosed in the U.S. annually, with the number increasing over the past 20-30 years. Carcinoid tumors that metastasize to the liver have a poor prognosis. Traditionally, chemotherapy relieves symptoms in less than 30% of cases of metastatic carcinoid tumors, usually for less than 1 year. Carcinoid tumors typically produce a condition called “carcinoid syndrome” which is caused by the release of hormones by the tumors into the blood stream. The symptoms vary depending on which hormones are released by the tumors, but typically include diarrhea, facial flushing, wheezing, abdominal pain and valvular heart disease.