ImmusanT's celiac disease immunotherapy passes first clinical test

Phase 2 trial will test Nexvax2 alongside a gluten-free diet

People with celiac disease could be freed from the gluten-free aisle of the supermarket if a therapeutic approach developed by ImmusanT proves effective in trials.

The Massachusetts biotech says it has completed its first phase 1b trial of Nexvax2, designed to protect celiac sufferers from the effects of exposure to gluten and the gastrointestinal symptoms that can result such as diarrhea, abdominal pain and bloating.

Celiac disease is remarkable among chronic diseases as it is very common but has no approved treatment beyond dietary restriction. It affects around 1% of Americans—although more than 80% are undiagnosed—and in nine of out of 10 cases is thought to be caused by a mutation in the HLA-DQ2 gene which is involved in immune regulation.

Developing effective treatments is important as around a third of people with celiac disease still suffer damage to the cells lining the intestines—sometimes without realizing it—and symptoms whilst on a gluten-free diet. It can also be hard to stick to gluten-free as some products won't explicitly list it on the label.

ImmusanT's big idea is to use a trio of three peptides as an immunotherapy that it hopes will encourage the T cells involved in the inflammatory reaction in celiac disease to become tolerant to gluten. After a first course to induce tolerance, the company hopes that it can be maintained by periodic re-injection with the vaccine.

It's a case of so far, so good, with the phase 1b trial in 38 patients revealing no concerns about safety or tolerability and showing that the immunotherapy seemed to have the desired effects on the immune system. 

The study also allowed ImmusanT to select a dosing regimen for planned phase 2 trials that will see if Nexvax2 can be used alongside a gluten-free diet to protect patients when they are inadvertently exposed to gluten, which ImmusanT sees as the quickest route to approval.

Depending on the results, a follow-up program is planned will focus on an immunotherapy that could do away with the need for a restricted diet entirely—something that could be more challenging to get past the FDA. The biotech is also developing a companion diagnostic for the vaccine which could guide its use and help improve diagnosis rates.

It's taken many years, but at long last there seems to be an emerging crop of treatment candidates for celiacs.

Switzerland's Anokion teamed up with Japanese pharma Astellas in 2015 to create a spinout called Kanyos that is working on an immunotherapy for celiac disease along with type 1 diabetes, and Sanofi is also working with Selecta on a similar approach.