New celiac disease drug candidate, KAN-101, in Phase 1 clinical trials

New potential treatment for celiac disease aims to re-educate the immune system so it does not react to gluten

Kate Avery, MPH, Director of Research & Patient Engagement

Kanyos Bio, Inc. (a wholly-owned subsidiary of Anokion SA) launched a Phase 1 trial for a new potential celiac disease treatment in February. The Cambridge, Massachusetts-based biotechnology company is focused on developing new treatments for autoimmune diseases, including celiac disease and multiple sclerosis. The trial, called the ACeD (Assessment of KAN-101 in Celiac Disease) Study, is taking place at up to 12 sites across the US.

A Phase 1 clinical trial is the first step in the clinical trial process all drug candidates must go through before they are approved by the FDA and available to patients. The goal of the ACeD Study is to test the safety and tolerability of KAN-101 in people with celiac disease and to learn how the body processes the drug (called pharmacokinetics).

Kanyos’ (and Anokion’s) approach is focused on restoring normal immune tolerance by targeting specific receptors on the liver. This sets off a cascade of events that drive the re-education of the immune system. Unlike the broadly suppressive therapies (called immunosuppressants) often used for other autoimmune diseases, Kanyos’ platform specifically targets parts of the immune system responsible for driving disease while leaving the majority of the immune system untouched so that it can perform its natural protective functions. KAN-101 is built on this platform technology, originally developed by Stephan Kontos, PhD, Anokion’s chief scientific officer and co-founder, during his graduate and post-doctoral work at the Swiss Federal Institute of Technology (EPFL) in Lausanne.

In celiac disease, the immune system reacts to gliadin, a component of gluten. Kontos explained that the liver-targeting platform technology he developed “delivers proteins into an immune tolerance pathway located in the liver. In doing so, [it] leverages a natural process the liver routinely performs. By using our platform, we retrain the immune pathway to induce tolerance to almost any protein of interest, in this case gliadin (gluten).”

Inducing immune tolerance to gliadin (gluten) is one of several approaches in development to treat celiac disease. ‘Currently, there are no approved treatments for celiac disease and instead, affected people are required to adhere to a strict and burdensome gluten-free diet to manage their symptoms,’ said Dr. Simon Cooper, Anokion’s chief medical officer, when asked why Kanyos chose celiac disease as its first indication to enter the clinic.

ACeD Study participants will be assigned to one of two groups. The first group will receive one dose of KAN-101 while the second group will receive three doses of either KAN-101 or placebo. The second group will also fill out daily questionnaires about their symptoms and complete a three-day gluten challenge to assess their immune system’s response to gluten after receiving KAN-101 or placebo. As is the case with many ongoing clinical trials, COVID-19 has affected the ACeD trial, with many sites pausing recruitment over the summer due to COVID-19 studies or site limitations. Recently, according to Dr. Cooper, all study locations have begun to restart the ACeD study, as well as other trials, taking precautions to ensure participant safety. These safety measure include participant and staff mask-wearing, social distancing, and increased facility cleanings in addition to various state and local precautions mandated by public health requirements.

Beyond Celiac is helping Kanyos Bio recruit for this study as part of our commitment to accelerating research. “It is very exciting to see new potential treatments for celiac disease advancing to clinical development. We at Beyond Celiac are looking forward to partnering with Kanyos Bio to expedite patient recruitment in this trial,” Salvo Alesci, Beyond Celiac Chief Scientist and Strategy Officer noted. To learn more about the ACeD trial and to contact a site near you to see if you qualify, visit Anokion.com/ACeD.