Prospects and treatment options for UC patients that do not respond to conventional or biologic treatments are limited, and leading experts in the area have encouraged the development of new therapies that can offer better healing of the large intestine and improved long-term effects without causing severe side effects.
InDex is currently in late-stage clinical development with its lead drug candidate cobitolimod, a first in class treatment for moderate to severe ulcerative colitis. Cobitolimod is locally administered in the large intestine, providing a local anti-inflammatory effect which can lead to healing of the mucosa and improvement of clinical symptoms. Cobitolimod also has very limited systemic absorption, and an excellent safety profile in sharp contrast to many approved treatments.
A new and unique mechanism of action
Cobitolimod is part of InDex Pharmaceuticals’ DNA-based ImmunoModulatory Sequence (DIMS) platform. It is administered locally in the large intestine, where the substance binds to Toll-like receptor 9 (TLR9) present inside immune cells as well as on the surface of epithelial cells. The binding triggers the cells to product anti-inflammatory cytokines, agents that play an important role in reducing the inflammation and healing the wounds in the large intestine caused by UC.
Clinical studies evaluating cobitolimod have shown a competitive clinical efficacy in treating ulcerative colitis combined with a superior safety profile. This safety profile is in stark contrast to current systemic treatments that weaken the immune system and therefore are associated with serious side effects such as infection and cancer. Read more about cobitolimod’s clinical trials here.
An outstanding combination of efficacy and safety
Right now, more than 2 million people have ulcerative colitis in the US, Europe and Japan. The prevalence of UC is expected to increase by 0.8 percent each year, and the risk of getting inflammatory bowel disease has increased tenfold over the past 75 years.
The United States is the largest single market, representing more than 50 percent of the global market for ulcerative colitis. Biological drugs are currently the largest market segment in terms of value with annual sales reaching over 5 billion USD in 2016 and more than 200,000 patients are currently treated with biological drugs.
Of all patients who suffer from UC, about 60 percent are estimated to suffer from moderate to severe ulcerative colitis. Approximately 55 percent of this group are considered to have left-sided colitis and more than half do not respond to conventional treatment and need other alternatives. With an assumed price in the US, Europe and Japan in line with the most recently launched products, this corresponds to an estimated addressable market of over USD 9 billion for cobitolimod in moderate to severe left-sided ulcerative colitis.
The qualitative market research studies that InDex has performed among specialist physicians and experts from pricing authorities and insurance agencies confirm the significant medical need for new, safe, and effective drugs to treat moderate to severe ulcerative colitis. Specialist physicians stated that they would be likely to prescribe cobitolimod to a large number of their patients. The results also provided support for pricing in line with modern therapies.
With an outstanding combination of efficacy and safety, cobitolimod has significant market potential. The annual sales of cobitolimod – given a successful commercialization – are estimated to reach over 1 billion USD.