Celiac disease is a genetic autoimmune disorder that affects approximately 1% of the global population. In celiac disease, ingestion of gluten leads to damage of the villi in the small intestine causing malabsorption of nutrients. This can lead to long-term health conditions such as nutritional and vitamin deficiencies often precipitating early onset of osteoporosis or osteopenia. Furthermore, it can lead to more malignant conditions such as Non-Hodgkin lymphoma and small intestinal adenocarcinoma.
There is no approved drug for the treatment of celiac disease, or medication to prevent the immune attack or repair the damage to the villi resulting from exposure to gluten. Current treatment is limited to maintaining a gluten-free diet. Failure to comply with a gluten-free diet will typically lead to relapse as the underlying pathology of celiac disease remains unaddressed.
COUR has developed CNP-101, a biodegradable nanoparticle encapsulating gliadin proteins – the major component of dietary gluten. CNP-101 is the first agent to induce antigen-specific immune tolerance in any autoimmune disease.
In a Phase 2, double-blind, placebo-controlled clinical trial, patients with celiac disease were treated with CNP-101 and, starting one week later, were fed wheat gluten for 14 days. While patients receiving placebo developed severe immune responses to gliadin and damaging inflammation in their small intestine, patients treated CNP-101 showed significantly less inflammation. In addition, CNP-101 showed a trend toward protecting the intestines from gluten-related injury when compared to untreated patients.
Based on these Phase 2 clinical results, Takeda acquired the license to CNP-101 in October 2019 and will lead further clinical development.