9th Aug 2011, 10:47 am by Brad Lemaire
Drug maker Lexicon Pharmaceuticals (NASDAQ:LXRX) reported positive data from a phase two study of its experimental drug LX1032, designed to treat a chronic condition that causes severe diarrhea.
Carcinoid syndrome is caused by neuroendocrine tumors that usually originate from the gastrointestinal tract, and is characterized by severe diarrhea and flushing episodes, with long-term consequences including malnutrition, heart disease, and death.
Symptoms of the disease have been linked to excess production of serotonin by metastatic tumor cells. The company’s drug, taken orally, is designed to reduce serotonin production.
The phase two trial was carried-out as a randomized, placebo-controlled study made up of 23 patients with carcinoid syndrome, in the United States.
Patients in the study averaged six bowel movements a day, with subjects receiving either a placebo or one of four doses of the drug daily for 28 days.
The study’s endpoint was safety and tolerability. Efficacy measures included change in bowel movements, relief of symptoms and reduction in serotonin production.
Data from the study showed five patients taking the drug reduced bowel movements per day by 30% for two weeks or more during the study.
Further, nine patients had a complete biochemical response of a 50% reduction in urinary 5-HIAA, a biomarker of serotonin production. at the end of the study, six patients also reported sufficient relief of carcinoid symptoms.
According to the results, the drug was also well tolerated and adverse events were mild to moderate, the company said.
Additionally, preliminary data from a separate, single-arm study of the drug in Europe reported five out of six patients with carcinoid syndrome showed sustained reductions o of at least 30% in bowel movement frequency.
Two of these responses occurred in the first four weeks of therapy, while three more occurred in the fourth and eighth week of treatment, under the 12-week protocol.
“The phase two data clearly show a reduction in bowel movements and symptom relief in a population that has exhausted all standard-of-care treatment options,” Chief Medical Officer of Lexicon, Pablo Lapuerta, said in a statement.
“The phase two data are also consistent with preliminary results from the European study, where there appear to be some early responses and additional benefit with continued treatment.”
The LX1032 drug is being developed under fast track designation from the FDA and orphan drug designation from the European Medicines Agency (EMA).
Currently, Lexicon is planning a phase two trial of the drug for mild to moderate ulcerative colitis, an inflammatory bowel disease that affects the lining of the large intestine.
Shares in the biopharmaceutical company rose one penny to $1.41 on Tuesday morning.