French biotech Ipsen announced their phase 3 study examining the efficacy of Somatuline® (lanreotide) to treat neuroendocrine tumors (NETS) in patients with history of carcinoid syndrome met its primary endpoint.
NETs are rare heterogeneous tumors that usually begin in the gastric mucosa, pancreas, small and large intestine. As a result of their origin, symptoms often include diarrhea and flushing. The only orphan drugs currently approved for NETs or carcinoid syndromes are Afinitor® (everolimus) for treating progressive neuroendocrine tumors of pancreatic origin (PNETs) in patients with unresectable, locally advanced or metastatic disease and Sandostatin LAR® (octreotide) for treating symptoms (flushing, diarrhea) associated with carcinoid syndrome. According to the press release, Somatuline is approved for treating NETs in other countries but in the United States, Somatuline Depot® only has orphan drug approval for treating patients with acromegaly. However, Somatuline Depot has orphan designation status with the FDA for NETs.
Results of the study are not expected to be seen until January 2014 when data from the ELECT study is presented at the 2014 Gastrointestinal Cancers Symposium (January 16-18, 2014 in San Francisco, CA).
ELECT® (A doublE-bLind, randomizEd placebo controlled Clinical Trial investigating the efficacy and safety of Somatuline® (lanreotide) injection in the treatment of carcinoid syndrome) is a 48-week Phase III trial in patients with a history of carcinoid syndrome. The study consisted of a 16-week, double-blind, randomized (to either Somatuline® of placebo), placebo-controlled phase followed by a 32-week open-label phase. Throughout the study the patients were allowed to use rescue medication (octreotide) as needed to control their symptoms related to carcinoid syndrome. The primary endpoint was the percentage of days subcutaneous octreotide was required to control symptoms associated with carcinoid syndrome, as rescue medication during the 16-week double-blind phase of the study. Secondary endpoints included frequency of diarrhea and flushing, usage of other rescue medication, Quality of Life, tumor markers and safety. The trial is registered in ClinicalTrials.gov (NCT00774930).
In their press release, the company announced “Treatment with Somatuline® was found to be statistically significantly superior to placebo in decreasing the number of days patients needed to use rescue medication (subcutaneous somatostatin analogues i.e., octreotide) to control symptoms associated with carcinoid syndrome.”
Image of the neuroendocrine tumor of the stomach courtesy of Wikimedia commons.