Accelerating research for treatments and a cure for celiac disease is the top priority of the Celiac Disease Foundation.
We are optimistic that the current surge of research will produce the first FDA-approved treatment for celiac disease. Our optimism comes from the variety of approaches biopharmaceutical companies are taking to treat the disease to make it easier for patients to manage and thrive. I want to highlight for you these approaches. The Celiac Disease Foundation is proud to be a partner with each of the companies, supporting their work with our powerful research tools, iCureCeliac® and iQualifyCeliac.
At Digestive Disease Week (DDW) this past May, the Anokion research team, led by Celiac Disease Foundation Medical Advisory Board Member and recipient of the first inaugural Celiac Disease Foundation Prize for Excellence in Celiac Disease Research, Dr. Joseph Murray of Mayo Clinic, announced the findings of a successful Phase 1 trial.
Anokion’s approach is to engineer immune tolerance. Patients suffering from autoimmune diseases harbor immune cells that mistakenly recognize and attack the body’s own cells as foreign entities. When people with celiac disease ingest gluten, these immune cells produce inflammation, damaging the body’s organs and tissues and causing symptoms and long-term health complications. KAN-101 works by delivering a gluten antigen to the liver to educate the immune system to ignore gliadin, the component of gluten that many researchers believe triggers the gluten response in celiac disease patients. The goal of this education process is to create immune tolerance while also leaving the rest of the immune system untouched. On the heels of the promising results for KAN-101 in its Phase 1 trial, Anokion has received permission from the FDA to begin a Phase 2 trial to more specifically test efficacy and dosage.
Latiglutenase is a dual enzyme drug designed to break down gluten in the stomach into small, harmless fragments, reducing the gliadin immune response in people with celiac disease. It is a drink-based medication intended to be taken at meals to help relieve common symptoms suffered by celiac disease patients after accidental gluten exposure. In Phase 2 trials, self-reported relief for abdominal pain, bloating, tiredness, and constipation was pronounced in celiac disease patients who continued to test positive for gluten-induced antibodies in their blood (seropositive).
Provention Bio: PRV-015
The novel drug PRV-015 is an antibody designed to block an immune system protein called interleukin-15, also known as IL-15. Gluten ingestion in people with celiac disease results in the production of IL-15, which triggers intestinal inflammation that leads to the destruction of intestinal cells. It is being co-developed with Amgen for the treatment of gluten-free diet non-responsive celiac disease (NRCD) by Provention Bio with the aims of reducing both symptoms and intestinal inflammation and damage caused by accidental gluten exposure.
Already tested in approximately 250 people in six completed research studies, PRV-015 has shown to be well-tolerated, and it may reduce inflammation and improve the symptoms of celiac disease.The Celiac Disease Foundation is currently recruiting for the Proactive Celiac Study Phase 2b trial in the United States and Canada.
Takeda: TAK-101, TAK-062, and the Virtual Celiac Symptoms Study
Takeda Pharmaceuticals has emerged as one of the major players in the celiac disease drug space. They have procured rights for two potential therapies from smaller biopharmaceutical companies, with both heading into Phase 2 clinical trials this fall.
Takeda licensed TAK-101 (formerly CNP-101 or TIMP-GLIA) from Cour Pharmaceuticals in 2019. TAK-101 is an immune modifying nanoparticle containing gliadin proteins designed to bind with immune cells called monocytes and load them with gliadin proteins. The cells travel to the spleen where the gliadin is released, reprogramming the immune system to tolerate gluten as a non-threatening substance and negating or reversing the signs and symptoms of celiac disease without using immune suppressing drugs.
TAK-062 (formerly Kuma062) is a highly potent super glutenase developed by PvP Biologics that Takeda acquired after a successful Phase 1 proof-of-mechanism study. TAK-062 is designed to degrade the immune-reactive parts of gluten before they exit the stomach in order to prevent an immune response. Unlike other glutenase drugs that have been tested for celiac disease, TAK-062 is specifically engineered to better target gluten and to survive the acidity of the stomach. TAK-062 has shown robust gluten degradation in humans and holds great promise to alleviate suffering among celiac patients.
Virtual Celiac Symptoms Study
To better understand the symptom patterns and impacts of celiac disease to inform drug development, Takeda, together with the Celiac Disease Foundation, is launching a study where participants will download an app to their smartphone and answer daily questionnaires about their symptoms and life with celiac disease for 12 weeks. Patients ages 12 and up with a confirmed diagnosis of celiac disease will be eligible to participate in this study, launching late summer.
The Celiac Disease Foundation is, or will soon be, supporting seven clinical trials, two Phase 1 and five Phase 2. Recruiting celiac disease patients is challenging and expensive – often prohibitively so.
There will be no approved treatments or a cure for celiac disease without clinical trials. That means there will be no approved treatments or a cure for celiac disease if we can’t find patients willing to participate in clinical trials.
iCureCeliac®, the Celiac Disease Foundation Patient Registry, is the best tool we have to identify and recruit patients for all types of celiac disease research, including for clinical trials. Currently, we have more than 9,000 patients in iCureCeliac®; we need to grow the database dramatically to more effectively support critical research in celiac disease.
The variety and quality of approaches to treating celiac disease from our biopharmaceutical companies is incredibly encouraging. This is the kind of work your generous donations to the Celiac Disease Foundation support. If you are able, please make a tax-deductible gift today. Your sustained support inspires us.
If you have not registered for iCureCeliac® already, please do. And if you have ideas on how to best recruit celiac patients to participate in trials and studies, or any questions about iCureCeliac®, please don’t hesitate to reach out.
To Our Health,
Marilyn G. Geller