Therapeutic discovery for Inflammatory Bowel Disease

Introduction

Inflammatory Bowel Disease (IBD) – a major cause of colorectal cancer (CRC) and a huge socioeconomic burden in Europe and beyond. IBD comprises diseases that are characterized by chronic inflammation in the gastrointestinal tract and includes ulcerative colitis (UC) and Crohn’s disease (CD).

Disease Characteristics

These diseases are lifelong, causing debilitating symptoms and can be life-threatening. IBD is caused by an interplay between the patient’s genetic predisposition, immune responses, and environmental factors. Chronic inflammation with fibrosis is the key driver of disease complications such as:

• Stenosis
• Abscess formation
• Colon cancer

Prevalence and Risks

IBD affects about 3.2 million people in Europe. IBD patients have an increased risk of CRC caused mainly by chronic intestinal inflammation leading to pro-neoplastic effects. In fact, IBD patients have an alarming 2- to 5-fold increased risk of developing CRC compared to the general population.

Economic Burden

Despite many treatment options, there is no cure for IBD. The IBD health economic burden in Europe is high and estimated at over 5B €/year.

Need for Novel Therapies

It is widely accepted that to overcome the challenges and to break through the “therapeutic ceiling,” combination therapies or completely novel approaches will be needed. Due to the increasing incidence and prevalence and associated burden of IBD, the lack of effective long-term treatment options, and the associated risks including CRC, IBD remains an important unmet medical need with an urgent need for novel therapies that can improve patient outcomes.

Proposed Research

Here we will explore the use of a completely novel class of molecules exploiting a unique first-in-class mechanism-of-action and validate their efficacy as a therapeutic for IBD. Proof-of-concept studies will be performed in state-of-the-art mouse models.

Expected Outcomes

The data obtained will represent a robust data package towards IND for this indication. We propose a novel therapeutic option for IBD that has the potential to address this important unmet medical need.