Currently, the only available treatment for a patient with celiac disease is a strict, gluten-free diet. A lifelong diet completely free of gluten can be very costly and challenging. Many patients’ intestines don’t heal completely and they continue to suffer from symptoms, most often due to imperfect adherence to the gluten-free diet. Despite patients’ best efforts, accidental ingestion of gluten through cross-contamination continues to jeopardize their health and well-being. Future drug therapies are currently in development with the hope of reducing the burden of living with celiac disease and improving long-term health outcomes. Find out more below.

Pre-Clinical

AMYRA Biotech AG

AMYRA is a biotech company that is developing a highly promising, novel, enzyme-based treatment for celiac disease, targeted to inhibit the toxic potential of gluten. Read more about AMYRA Biotech AG.

Avaxia Biologics

Current research efforts at Avaxia are focused primarily on inflammatory diseases of the gastrointestinal tract. Avaxia has identified potential targets for antibodies that may improve celiac disease. Read more about Avaxia Biologics.

Bioniz Therapeutics, BNZ-2

BNZ-2 selectively inhibits IL-15 and IL-21 and has showed potent in vivo activity. IL-15 is central in celiac disease and many other inflammatory diseases of the gut and recent research has demonstrated that IL-21 functions in tandem/synergistically with IL-15. Read more about Bioniz Therapeutics.

Calypso Biotech SA, CALY-002

Calypso Biotech SA is a biopharmaceutical company developing CALY-002, an antibody that inhibits the cytokine, Interleukin-15 (IL-15), which plays multiple roles in the immune system, especially in the gastrointestinal tract. CALY-002 may treat several gastrointestinal symptoms that currently have no approved treatment, including celiac disease. Read more about Calypso Biotech SA.

ExPharma Limited/Nemysis Limited

ExPharma Limited is a medical foods development company that recently acquired Nemysis Limited. Nemysis has patented compounds potentially useful for the treatment of symptoms associated with celiac disease. Read more about Nemysis and ExPharma Limited.

Intrexon

Intrexon’s ActoBiotics® platform delivers therapeutic molecules to the oral and gastrointestinal tracts for the treatment of oral, gastrointestinal, metabolic, allergic, and autoimmune diseases. Using ActoBiotics® technology, Intrexon is working to develop safe, effective therapeutic solutions for celiac disease, oral mucositis, inflammatory bowel disease, allergy, type 1 diabetes, porcine growth, and nutrition. Read more about Intrexon.

James Cook University, Hookworm Larvae

James Cook University hopes to find a drug derived from hookworm larvae to treat gluten sensitivity. Hookworms may secrete anti-inflammatory proteins, which could be put in a pill and ultimately restore gluten tolerance. For more information, Read more about James Cook University.

Provid Pharmaceuticals

Provid focuses on synthetic and medicinal chemistry and is developing inhibitors of DQ2 and DQ8, the two genes associated with celiac disease. Read more about Provid Pharmaceuticals.

Selecta Biosciences and Sanofi, SVP

Selecta Biosciences and Sanofi are collaborating on a new immune tolerance program that will use Selecta’s proprietary Synthetic Vaccine Particle (SVP™) platform to develop an immunotherapy for the treatment of celiac disease. Read more about Selecta Biosciences.

Sitari Pharma

Sitari Pharma is developing treatments for celiac disease that target Transglutaminase 2 (TG2), an enzyme that is thought to play a role in celiac disease. TG2 is the primary human protein that is attacked as part of an autoimmune response in patients with celiac disease. Sitari is developing inhibitors of TG2 with the potential to minimize the autoimmune response that results in intestinal inflammation and disease development in celiac disease patients. Read more about Sitari Pharma.

TEC Edmonton, The University of Alberta, IGY Inc. and Vetanda Group Ltd.

Researchers from the University of Alberta and IGY Incorporated have been developing immune powered eggs. For baby chicks to fight off diseases, mother hens produce natural antibodies known as immunoglobulin yolk. In collaboration with IGY, the University of Alberta researchers developed egg yolk antibodies targeted at wheat proteins. Anti-gluten immunoglobulin yolk antibodies bind to problematic wheat proteins, making them harmless to those with celiac disease. A Canadian human safety trial has been completed. An efficacy trial is underway to test the natural health product’s ability to reduce gluten intolerance and possibly eliminate celiac disease symptoms in those following a gluten-free diet. Read more about TEC Edmonton’s work.

VU University Medical Centre, AN-PEP

VU University Medical Centre is assessing the safety and efficacy of Aspergillus niger prolyl endoprotease (AN-PEP) to lessen the problematic effects of gluten in celiac disease patients. In their pilot study, AN-PEP appeared to be well tolerated. However, additional studies with more patients and a gluten challenge for a longer period of time are required to confirm whether the results have clinical significance. Read more about VU University Medical Centre.

Phase 1:

Cour Pharmaceuticals, TIMP-GLIA

With Cour Pharmaceuticals’ investigational new drug, TIMP-GLIA, the body’s immune system is reprogrammed to tolerate gluten as a non-threatening substance and then reverses the signs and symptoms of celiac disease without using immune suppressing drugs. By encapsulating a component of wheat in a small particle, Cour developed a “back door” approach, where the gluten is not recognized by the body’s immune system until it reaches the spleen, where immune tolerance can be generated. Read more about Cour Pharmaceuticals.

Mayo Clinic, National Cancer Institute, University of Chicago Medicine, Hu-Mik-Beta 1

Hu-Mik Beta-1 is a monoclonal antibody that targets the cytokine receptor subunit IL-2/IL-15Rβ (CD122) as well as blocks IL-15 transpresentation. Hu-Mik Beta-1 is in an open label, phase 1 clinical trial titled “Phase I Study of the Humanized Mik-Beta-1 Monoclonal Antibody Directed Toward IL-2/IL-15R Beta (CD122) That Blocks IL-15 Action In Patients With Refractory Celiac Disease” {NCT01893775}. The antibody is being tested for the treatment of refractory celiac disease.

PvP Biologics & Takeda Pharmaceuticals, KumaMax

PvP Biologics is a pre-clinical biotechnology company developing a therapeutic enzyme for the treatment of celiac disease, called KumaMax. KumaMax is an enzyme that has the potential to recognize and break down the parts of gluten that trigger the immune reaction before those parts exit the stomach and reach the small intestine. Therefore, the immune response to gluten is expected to decrease, reducing the symptoms and intestinal damage associated with celiac disease. Read more about PvP Biologics.

Phase 2:

Amgen/Celimmune, AMG 714

Celimmune licensed AMG 714 from Amgen in 2015. AMG 714 is an antibody that targets IL-15, a cytokine that has shown to be one of the key factors in the loss of tolerance to food antigens and is believed to be involved in celiac disease, refractory celiac disease (RCD), and other autoimmune conditions. Celimmune has initiated phase II studies of AMG 714 for the treatment of diet non-responsive celiac disease and RCD-II, and in small bowel T cell lymphoma. Amgen Inc. acquired Celimmune, LLC in 2017. Celimmune, LLC remains a wholly owned subsidiary of Amgen Inc. Read more about Celimmune.

Dr. Falk Pharma and Zedira, ZED1227

ZED1227 is the first direct acting transglutaminase inhibitor in clinical development. The recently completed phase 1b clinical trial of ZED1227 confirmed safety and tolerability in female and male healthy volunteers, meeting the study objectives. Dr. Falk Pharma and Zedira now intend to investigate efficacy of ZED1227 in celiac disease patients in a phase 2a clinical trial. Read more about Dr. Falk and Zedira.

ImmunogenX, IMGX003 (Latiglutenase)

ImmunogenX is developing IMGX003 (formerly Alvine Pharmaceuticals ALV003), a mixture of two gluten-specific enzymes that break down gluten proteins into small, harmless fragments. It is to be administered as a supplement to a gluten-free diet for the potential treatment of celiac disease. The technology has been extensively studied in phase I and phase II clinical trials, and is the only celiac disease treatment that has been shown to lessen gluten-induced intestinal mucosal injury, as well as improve symptoms in clinical trials. Read more about ImmunogenX.

ImmusanT, Nexvax2®

ImmusanT is a clinical development-stage biotechnology company developing Nexvax2®, the only disease-modifying treatment in clinical development today that has the potential to enable celiac disease patients to return to a normal diet. Booster shots of Nexvax2® would offer periodic reinforcements of the treatment to establish a prolonged tolerance to gluten. ImmusanT plans to release Nexvax2® first to protect patients from inadvertent gluten exposure (e.g., cross-contact with gluten when dining out), followed by a vaccine that would allow patients to resume a diet containing gluten. Read more about ImmusanT.

Phase 3:

Innovate Biopharmaceuticals, INN-202 (Larazotide Acetate)

In February 2016, Innovate licensed the assets of Alba Therapeutics relating to larazotide acetate, a tight junction regulator. Larazotide acetate (INN-202) is a novel oral peptide that has consistently demonstrated the reduction of symptoms of celiac disease in multiple clinical trials. INN-202 helps restore “leaky” or open junctions to a normal state. INN-202, when ingested prior to a meal, may help keep the tight junctions closed, thus reducing the intestinal-inflammatory process in response to gluten. Read more about Innovate.

Summary of Clinical Trial Phases
PhasePrimary goalDosePatient monitorTypical number of participantsnumber of participants Notes
PreclinicalTesting of drug in non-human subjects, to gather efficacy, toxicity and pharmacokinetic informationunrestrictedA graduate level researcher (Ph.D.)not applicable (in vitro and in vivo only)
Phase 0Pharmacokinetics particularly oral bioavailability and half-life of the drugvery small, subtherapeuticclinical researcher10 peopleoften skipped for phase I
Phase ITesting of drug on healthy volunteers for dose-rangingoften subtherapeutic, but with ascending dosesclinical researcher20-100determines whether drug is safe to check for efficacy
Phase IITesting of drug on patients to assess efficacy and safetytherapeutic doseclinical researcher100-300determines whether drug can have any efficacy; at this point, the drug is not presumed to have any therapeutic effect whatsoever
Phase IIITesting of drug on patients to assess efficacy, effectiveness and safetytherapeutic doseclinical researcher and personal physician1000-2000determines a drug’s therapeutic effect; at this point, the drug is presumed to have some effect
Phase IVPostmarketing surveillance – watching drug use in publictherapeutic dosepersonal physiciananyone seeking treatment from their physicianwatch drug’s long-term effect

Summary of Clinical Trial Phases

View table data

PhasePrimary goalDosePatient monitorTypical number of participantsnumber of participants Notes
PreclinicalTesting of drug in non-human subjects, to gather efficacy, toxicity and pharmacokinetic informationunrestrictedA graduate level researcher (Ph.D.)not applicable (in vitro and in vivo only)
Phase 0Pharmacokinetics particularly oral bioavailability and half-life of the drugvery small, subtherapeuticclinical researcher10 peopleoften skipped for phase I
Phase ITesting of drug on healthy volunteers for dose-rangingoften subtherapeutic, but with ascending dosesclinical researcher20-100determines whether drug is safe to check for efficacy
Phase IITesting of drug on patients to assess efficacy and safetytherapeutic doseclinical researcher100-300determines whether drug can have any efficacy; at this point, the drug is not presumed to have any therapeutic effect whatsoever
Phase IIITesting of drug on patients to assess efficacy, effectiveness and safetytherapeutic doseclinical researcher and personal physician1000-2000determines a drug’s therapeutic effect; at this point, the drug is presumed to have some effect
Phase IVPostmarketing surveillance – watching drug use in publictherapeutic dosepersonal physiciananyone seeking treatment from their physicianwatch drug’s long-term effect