The randomized, double-blind, placebo-controlled clinical trial enrolled 86 patients who were confirmed biopsy positive for celiac disease and in compliance with a gluten-free diet for at least six months prior to enrollment. Patients were randomized into seven drug-treated and placebo groups and challenged three times a day with gluten during a 14-day period. Four doses of the enteric coated oral formulation of AT-1001, all less than 10 mg, were given prior to each gluten challenge. Study endpoints included intestinal permeability (IP) -- a marker of disease state in celiac disease -- as well as patient symptoms and outcomes, measured by two validated tests of gastrointestinal disease outcome: the Gastrointestinal Symptoms Rating Scale (GSRS) and the Psychological General Well-Being Index (PGWBI).
Preliminary analysis revealed the following:
-- At day 14, IP, as measured by the change in urinary lactulose-to-
mannitol (LA/MA) ratio, exhibited a dose dependent response. On day
21, one week after the final drug dosing and gluten challenge, the dose
dependent trend continued to statistically significant levels.
-- The GSRS and PGWBI provided additional efficacy signals that further
support the IP observations. Patients on the AT-1001 drug arms
performed better than those on the gluten/placebo arm. Analyses
demonstrated that several symptoms and outcomes improved at
statistically significant levels.
-- Safety and tolerability of multiple oral doses of AT-1001 in the
patient population was demonstrated. There were no Severe Adverse
Events and all Adverse Events were reported as mild or moderate.
"We are very encouraged by the preliminary data and look forward to applying the extensive knowledge gained in this Phase IIa exploratory clinical trial to a larger, highly powered Phase IIb gluten challenge study later this year" said Blake Paterson, M.D., Chief Executive Officer of Alba Therapeutics. Using the highly complex and ambitious seven arm study design for the Phase IIa trial, we repeated the proof of concept from the Phase Ib study, showed a statistically significant effect across a variety of measures and are well prepared to move the celiac program forward."
Based on these results, Alba will advance AT-1001 into a Phase IIb clinical study in celiac disease subjects during the third quarter of 2007. The Phase IIb study, to be performed in multiple centers in the United States and Canada, will assess the efficacy of AT-1001 utilizing multiple endpoints, including a composite index of disease activity. The first patient is expected to be enrolled into this study in the third quarter of 2007, and the study should conclude in early 2008.
About Celiac Disease
Celiac Disease is a T-cell mediated autoimmune disease that occurs in genetically susceptible individuals and is characterized by small intestinal inflammation, injury and intolerance to gluten. According to the National Institutes of Health, celiac disease affects approximately 3 million Americans. The only current treatment for celiac disease is complete elimination of gluten from the diet, which results in remission for some patients.
Alba Therapeutics Corporation is a privately held biopharmaceutical company based in Baltimore, Maryland dedicated to the development and commercialization of disease modifying therapeutics to treat autoimmune and inflammatory diseases based upon the regulation of tight junctions. Albas lead compound, AT-1001, is targeted towards the treatment of Celiac Disease, Inflammatory Bowel Disease and Type 1 Diabetes
Contact: Stuart Sedlack, SVP, Corporate Development