ABX464, a small and first-in-class molecule demonstrating strong anti-inflammatory activity

Preclinical data generated with ABX464 demonstrate a strong anti-inflammatory effect of the compound.

This effect was shown to be mediated by a 50-fold increase of the expression of IL-22 in macrophages. IL-22 is a cytokine known as a potent suppressor of inflammatory processes. Furthermore, in peripheral blood mononuclear cells, the expression of miR124, an anti-inflammatory micro-RNA, was enhanced >10 fold by ABX464. Inflammation is a cornerstone of numerous pathologies, not only in HIV, but also in a number of other diseases, such as inflammatory bowel disease (IBD, including ulcerative colitis and Crohn’s disease).

When evaluated in a mouse model of IBD, ABX464 demonstrated a long-lasting effect in preventing the typical symptoms of inflammatory colitis, including histological changes. Based on these encouraging results, the company intends to launch a proof of concept clinical study in patients with IBD in 2017.

IBD – a chronic disease with no cure

Inflammatory bowel disease (IBD) is an idiopathic disease caused by a dysregulated immune response to host intestinal microflora. The two major types of inflammatory bowel disease are ulcerative colitis (UC), which is limited to the colon, and Crohn’s disease (CD), which can affect any segment of the gastrointestinal tract from the mouth to the anus, involves "skip lesions," and is transmural. There is a genetic predisposition for IBD, and patients with this condition are more prone to the development of malignancy.

The main symptoms of ulcerative colitis and Crohn’s disease are similar. They include:

  • Pain, swelling or cramping in the abdomen
  • Recurring or bloody diarrhea
  • Weight loss
  • Extreme tiredness

Prevalence (number of existing cases per 100,000 population)

Crohn's disease

Ulcerative colitis

26 to 199 cases per 100,000 persons[1]

37 to 246 cases per 100,000 persons

201 per 100,000 adults[2]

238 per 100,000 adults









[1] Loftus EV, Jr. Clinical epidemiology of inflammatory bowel disease: Incidence, prevalence, and environmental influences. Gastroenterology. 2004; 126:1504-17.

[2] Kappelman MD, Rifas-Shiman SL, Kleinman K, Ollendorf D, Bousvaros A, Grand RJ, Finkelstein JA. The prevalence and geographic distribution of Crohn's disease and ulcerative colitis in the United States. Clin Gastroenterol Hepatol. 2007; 5:1424-9.