Alba Therapeutics Announces Positive Results of Phase IIb Trial in Celiac Disease

BALTIMORE, Feb. 11, 2014 /PRNewswire/ -- Alba Therapeutics Corporation announced today the positive results of their Phase IIb trial evaluating its investigational product, larazotide acetate, a first-in-class tight junction regulator, intended for the treatment of patients with celiac disease (CeD).  The study met its primary endpoint and, based on these results, Alba has initiated planning for Phase III clinical trials for the definitive assessment of the oral peptide's efficacy and safety.  The final data will be submitted for publication in the near future.

The double-blind, placebo-controlled study evaluated the efficacy and safety of larazotide acetate in the treatment of 342 patients with CeD who had persistent symptoms despite being on a gluten-free diet.ⁱ This is the seventh study in a broad clinical trial program for larazotide acetate that, to date, has included 828 patients with CeD.^ii,iii  Larazotide acetate has been granted "Fast Track" designation from the FDA. 

"Tight junction regulators represent a paradigm shift in the treatment of immune mediated and inflammatory disorders such as CeD.  Recently published data suggest 70% of patients continue to be exposed to gluten while on a gluten-free diet, highlighting the need for additional treatments for patients with celiac disease," said Joseph A. Murray, MD, the Phase IIb study's lead investigator and a gastroenterologist at the Mayo Clinic. "These promising trial results contribute to a growing body of data supporting the development of larazotide acetate, and the medical community looks forward to its continued development in an effort to provide an option to patients who struggle to manage the disease through diet alone."

Celiac disease is an autoimmune disorder that is triggered by the ingestion of gluten, which is primarily found in bread, pasta, cookies, pizza crust and other foods containing wheat, barley or rye.ⁱⁱ  Signs and symptoms of celiac disease include abdominal pain and cramping, bloating, diarrhea, gas and failure to thrive (infants and small children).  Celiac disease has also been associated with a heightened risk of cancer, including cancers of the esophagus, small and large intestines and T-cell lymphoma.^iv  People with celiac disease who are exposed to gluten experience an immune reaction in their small intestines, causing damage to the inner surface (villi) of the small intestine, and an inability to absorb certain nutrients.  Larazotide acetate has a novel mechanism of action that may help inhibit this immune reaction.^ii,iii 

"There is a high unmet medical need for CeD as many patients are experiencing painful and often debilitating symptoms despite following a gluten-free diet," said Wendy Perrow, MBA, Chief Executive Officer of Alba. "We are very encouraged by the data observed in this Phase IIb study as well as the other studies in the larazotide acetate program and look forward to advancing its development."

About the Investigational Larazotide Acetate

Larazotide acetate, Alba's leading product candidate for CeD, is a novel agent that belongs to a new class of drug called tight junction regulators. Tight junctions, which are located in the bowel, should remain closed except to shed dead cells. However, in patients with CeD, the presence of gluten causes the tight junctions to remain open, thus starting an inflammatory cascade within the bowel that eventually destroys the intestinal villa. Early research suggests larazotide acetate may help keep the tight junctions closed when ingested prior to a meal, thus reducing the inflammatory process in response to gluten.^ii,v,vi

About Celiac Disease

Celiac disease is one of the most common autoimmune disorders, affecting 1% of the population in the United States and Europe (3 million and 3.5 million people, respectively)^vii and approximately 15 million individuals worldwide.^viii  Celiac disease is emerging as a significant public health concern, as it is associated with increased co-morbidities and mortality, and has dramatically increased in prevalence during the past 50 years. ^ix,x  Currently, there are no pharmacologic therapies available to treat this disease. The only management for CeD is to follow a gluten-free diet by eliminating foods that contain gluten. Unfortunately, the ability to maintain a strict gluten-free diet can be difficult,ⁱⁱ as many CeD patients reported being either accidentally or purposefully exposed to gluten-laden foods.^xi For patients who do adhere to a strict gluten-free diet, up to 30 percent respond poorly.^iv For these patients, therapeutic modalities beyond dietary modification could be beneficial.ⁱⁱ

About Alba Therapeutics Corporation

Alba Therapeutics Corporation ("Alba") is a Baltimore, Maryland privately held, clinical-stage biopharmaceutical company focused on the development and commercialization of pharmaceutical products to treat celiac disease and inflammatory diseases.

For more information about Alba's clinical trials, please visit the www.clinicaltrials.gov web site and search for Alba Therapeutics.

[i] ClinicalTrials.gov. A Double-blind Placebo-controlled Study to Evaluate Larazotide Acetate for the Treatment of Celiac Disease.  http://clinicaltrials.gov/ct2/show/NCT01396213?term=clin1001-012&rank=1.  Accessed December 9, 2013.

[ii] Leffler D, et al. A Randomized, Double-Blind Study of Larazotide Acetate to Prevent the Activation of Celiac Disease During Gluten Challenge. Am J Gastroenterol. 2012 Oct; 107(10): 1554-62. Epub 2012 Jul 24.

[iii] Kelly C, et al. Larazotide acetate in patients with coeliac disease undergoing a gluten challenge: a randomised placebo-controlled study. Ailment Pharmacol Ther. 2013 Jan; 37(2): 252-62. Epub 2012 Nov. 19.

[iv] Green et al. Celiac Disease. The New England Journal of Medicine. 2007;357:1731-43.

[v] Gopalakrishnan et al.  Larazotide acetate regulates epithelial tight junctions in vitro and in vivo.  Peptides. 2012 May;35(1):86-94. Epub 2012 Feb 27.

[vi] Gopalakrishnan et al.Larazotide acetate promotes tight junction assembly in epithelial cells.  Peptides. 2012 May;35(1):95-101. Epub 2012 Feb 28.

[vii] Fasano A, et al. Prevalence of Celiac Disease in At-Risk and Not-At-Risk Groups in the United States. Arch Intern Med. 2003;163:286-292.

[viii] Rewers M, Epidemiology of Celiac Disease: What Are the Prevalence, Incidence, and Progression of Celiac Disease? Gastroenterology. 2005 Apr; 128(4 Suppl 1): S47-51.

[ix] Ludvigsson J, et al. Increasing Incidence of Celiac Disease in a North American Population. Am J Gastroenterol. 2013; 108:818-824.  Epub 2013 Mar 19.

[x] Rubio-Tapia A, et al. Increased Prevalence and Mortality in Undiagnosed Celiac Disease. Gastroenterology. 2009 Jul; 137(1):88-93. Epub 2009 Apr 10.

[xi] Hall N, et al. Intentional and inadvertent non-adherence in adult coeliac disease. A cross-sectional survey. Appetite. 2012;68:56-62. Epub 2013 Apr 24.

SOURCE Alba Therapeutics Corporation