Drug to treat celiac disease gets to furthest-ever stage of study

A trio of firsts for celiac disease treatment

By Amy Ratner, Medical and Science News Analyst

The first patient in first Phase 3 celiac disease clinical trial has been given the first dose of a drug to treat celiac disease.

Innovate Biopharmaceuticals today announced that is has given the first dose of larazotide acetate to a study participant. Larazotide acetate is the first celiac disease drug to get to a critical, large-scale Phase 3 clinical trial.

Phase 3: The stage of clinical trial in which researchers demonstrate whether a drug offers a treatment benefit to a specific population. This phase involves 300 to 3,000 volunteers who have the disease or condition and typically lasts one to four years. It is part of the Food and Drug Administration approval process.

“This brings us closer in our mission of gaining approval for the first-ever drug for celiac disease,” said Sandeep Laumas, MD, Innovate chief executive officer.

Patrick Griffin, MD, Innovate chief medical officer, noted there is no drug to treat celiac disease and said larazotide acetate could provide the first therapy for the condition. Currently, the only treatment for celiac disease is the gluten-free diet, which is challenging to follow, especially since cross-contact with gluten is difficult to eliminate. Additionally, some patients to continue to have symptoms even when they are following a strict gluten-free diet.

Larazotide acetate is designed to work by decreasing intestinal permeability and regulating tight junctions between intestinal cells. This so-called “leaky gut” is thought to be the gateway to many autoimmune diseases, including celiac disease. The drug would help restore the leaky junctions to a normal state, according to Innovate.

The Phase 3 study is a national, multi-center trial that is expected to include about 600 patients being recruited at multiple sites in the United States and Canada. The primary goal of the clinical trial is to evaluate larazotide acetate for patients who are on the gluten-free diet but continue to have symptoms. It is taken before meals, up to three times a day, to reduce the inflammatory intestinal response triggered by the gluten protein in wheat, barley and rye in those who have celiac disease. Innovate anticipates the study will conclude at the end of 2021, according to information filed with the National Institutes of Health clinical trial website.

Randomized, double-blind, placebo controlled trial: a study in which researchers randomly decided which participants will receive the new drug and which will be in the control group and receive a substance that looks just like a regular treatment or medicine but is actually inactive. Neither the study participants nor researchers know who is in which group.

The study is a randomized, double-blind, placebo-controlled trial. In previous phases of clinical study in nearly 800 patients, larazotide acetate was found to be safe compared to a placebo, according to the company. The drug has a fast track designation from the FDA, which is designed to get important new drugs to patients more quickly, particularly when there is an unmet need.

A 2018 study by the Massachusetts Institute of Technology Sloan School of Management found that 14 percent of all drugs in clinical trials are eventually approved by the FDA, higher than the nine to 10 percent previously thought. Rates vary depending on the condition the drug is designed to treat.

Other treatments for celiac disease are also in the drug pipeline, though all are in Phase 2 or earlier. Nexvax2, a vaccine to treat celiac disease being developed by ImmusanT, was progressing through Phase 2 until earlier this year when study was discontinued due to disappointing results.