The U.S. FDA has cleared InDex Pharmaceuticals AB’s Investigational New Drug (IND) application to launch a Phase 2b clinical trial for cobitolimod (Kappaproct) in patients with moderate to severe ulcerative colitis (UC).

Cobitolimod is the the Stockholm, Sweden-based company’s lead drug candidate, now in late-stage clinical development for moderate to severe ulcerative colitis. UC is a chronic inflammation of the large intestine which can often be debilitating. Cobitolimod is a first-in-class TLR (Toll-like receptor) 9 agonist that mimics microbial DNA, the natural ligand of the receptor, through immunomodulation. The drug candidate provides local anti-inflammatory relief by healing the colonic mucosa and improving clinical symptoms.

In January, the World Health Organization (WHO) recommended the International Nonproprietary Name (INN) “cobitolimod,” also known as Kappaproct and DIMS0150.

Cobitolimod has been successful at its clinical proof-of-concept studies in moderate to severe ulcerative colitis, with a very favorable safety profile. Data from four placebo-controlled clinical trials suggested the product has statistically significant effects on the most relevant endpoints for the disease, either from a regulatory or clinical perspective. Endpoints comprise pivotal clinical symptoms, such as existence of blood in stool, number of stools, and mucosal healing.

The upcoming Phase 2b clinical trial will be a randomized, double-blind, placebo-controlled study to assess the safety and efficacy of cobitolimod in inducing clinical remission in patients with chronic moderate to severe ulcerative colitis. The study will also assess higher dose levels and more frequent dosing than previously used in other studies, aiming to provide substantial increased efficacy while sustaining the drug’s higher safety profile.

“The clearance of the IND is a major milestone for InDex Pharmaceuticals in our efforts to develop cobitolimod as a new therapy for patients with ulcerative colitis, a disease with a high unmet medical need,” InDex Pharmaceuticals CEO Peter Zerhouni said in a press release.

“We are very pleased about this validation by the FDA, which is based on the extensive preclinical and clinical data package available for cobitolimod, and we look forward to initiating the study later this year. Having clearance from the FDA is key for our ongoing discussions with potential licensing partners and investors. We will continue to work with the FDA as the development of this product progresses,” Zerhouni said.