Our Science – Disease Therapy
Currently, there are no approved drug treatments for celiac disease (CD). The only option available for celiac sufferers is a strict, lifelong adherence to a gluten-free diet (GFD). Total exclusion of dietary gluten is difficult because gluten is one of the most common food ingredients. Even highly motivated patients who attempt to adhere to a GFD are affected due to inadvertent or background exposure to gluten (Green 2007), and a majority of patients with CD who are in clinical remission and who claim to be following a GFD have persistent abnormalities in small bowel biopsy specimens (Lee, Lo et al. 2003). Greater than 60% of celiac disease patients continue to experience persistence of CD symptoms even on an attempted GFD. Thus, there is an acute need for non-dietary therapies for celiac disease.
Active and Safe Drug IMGX003
ImmunogenX is developing IMGX003 (formerly Alvine Pharmaceuticals ALV003), a mixture of two gluten-specific recombinant proteases that degrades gluten proteins into small physiologically irrelevant fragments, and is to be administered as an adjunct to an attempted gluten-free diet for the potential treatment of celiac disease. The technology is backed by over 50 issued or pending patents, has been extensively studied in Phase 1 and Phase 2 clinical trials, and is the only CD treatment that has been shown to mitigate gluten-induced intestinal mucosal injury as well as effect symptomatic improvements in clinical trials.
Alvine successfully completed two separate ALV003 phase 1 pharmacokinetic and pharmacodynamic studies. These studies enrolled healthy volunteers as well as celiac disease patients. In these studies, ALV003 was shown to be active in the gastric compartment and to degrade gluten ingested in a complex meal. A randomized, placebo-controlled phase 2a study conducted in Finland demonstrated that ALV003 administered daily for 6 weeks to celiac patients, well controlled on a GFD, could attenuate small intestinal mucosal injury induced by a daily gluten challenge under the controlled conditions of the study.
Patient Reported Outcome (PRO) Tool
ImmunogenX also acquired three celiac disease-specific patient reported outcome (PRO) tools developed by Alvine Pharmaceuticals. The Celiac Disease Symptom Diary© (CDSD©) can be used to quantify the effects that a potential treatment has on the most common symptoms affecting patients with celiac disease. The Impact of Celiac Disease Symptoms Questionnaire© (ICDSQ©) and the Impact of the Gluten-Free Diet Questionnaire© (IGFDQ©) are each quality-of-life instruments that were designed to measure the effects of symptoms associated with celiac disease and the effects of adherence to a gluten-free diet on a person’s quality of life. Each of these PRO tools, developed in compliance with FDA guidances has been rigorously tested in large clinical studies of patients with celiac disease.