Company News: InDex Pharmaceuticals Receives FDA Clearance of IND for Cobitolimod (Kappaproct®) Phase IIb Trial

InDex Pharmaceuticals AB today announced that the U.S. Food and Drug Administration (FDA) has cleared the Company’s Investigational New Drug (IND) application to initiate a phase IIb study with its lead drug candidate cobitolimod (Kappaproct®) in patients with moderate to severe ulcerative colitis (UC).

“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,” said Peter Zerhouni, CEO of InDex Pharmaceuticals. “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 on-going discussions with potential licensing partners and investors. We will continue to work with the FDA as the development of this product progresses.”

Cobitolimod (Kappaproct®) is InDex Pharmaceutical’s lead drug candidate in late-stage clinical development for moderate to severe ulcerative colitis, a debilitating, chronic inflammation of the large intestine. Cobitolimod is a first-in-class Toll-like receptor (TLR) 9 agonist that functions as an immunomodulatory agent by mimicking microbial DNA, the natural ligand of the receptor. Cobitolimod provides local anti-inflammatory effects, leading to healing of the colonic mucosa and improvement of clinical symptoms. In January 2016, WHO recommended the INN name cobitolimod. The substance is also known as Kappaproct® and DIMS0150.

The product has achieved clinical proof-of-concept in moderate to severe ulcerative colitis, with a very favorable safety profile. Data from four placebo-controlled clinical trials show that cobitolimod has statistically significant effects on those endpoints that are most relevant in this disease, both from a regulatory and clinical perspective. These endpoints include the key clinical symptoms such as blood in stool, number of stools, and mucosal healing, respectively.

The planned phase IIb study will be a randomized, double-blind, placebo-controlled trial to evaluate the safety and efficacy of cobitolimod in inducing clinical remission in patients with chronic active moderate to severe ulcerative colitis as compared to placebo. The study will evaluate higher doses and more frequent dosing than those used in previous studies with the goal to provide substantially higher efficacy, while maintaining the compound’s superior safety profile.