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, AMY02

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.

Circle33/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.

ExPharma/Nemysis, E40-02

ExPharma is a medical foods development company that acquired Nemysis. In May 2021, Nemysis announced the issuance of a Canadian patent for E40, a candidate for the development of a first-line treatment opposing gluten toxicity in celiac disease patients and gluten-sensitive conditions. Read more about Nemysis.

 

 

 

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.

Phase 1:

Anokion, KAN-101

Anokion and its wholly owned subsidiary Kanyos Bio, Inc. developed KAN-101, an investigational treatment which targets the immune cells that drive celiac disease and leaves the otherwise healthy components of the immune system intact. KAN-101 acts by re-educating T cells, or tolerizing them, so they do not respond to gluten antigens. The current Phase 1 clinical trial aims to evaluate the safety and tolerability of single or multiple doses of KAN-101 in subjects with celiac disease, as well as measure how the body responds to the study drug. Read more about Anokion.

 

 

Calypso Biotech, CALY-002

Calypso Biotech 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.

GlaxoSmithKline (GSK), GSK3915393

GlaxoSmithKline (GSK) is conducting a 3-part first time into human (FTIH) study for GSK3915393, an inhibitor of the enzyme Transglutamase 2 (TG2) which triggers an inflammatory response to gluten exposure in people with celiac disease. Parts A and B of the study will evaluate the safety, tolerability and pharmacokinetics (PK) of single ascending and repeat oral doses of GSK3915393 in healthy adult participants. Part C will evaluate the impact of co-administration of GSK3915393 with grapefruit juice and itraconazole on the PK of GSK3915393. By inhibiting TG2, this drug has the potential to minimize the autoimmune response that results in intestinal inflammation and disease development in celiac disease patients. Read more about GSK.

 

 

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. The most recently completed phase 1b trial involved applying hookworm larvae to participants’ skin, finding that hookworm-treated participants tended to have improved tolerance to low levels of gluten consumption. Researchers at James Cook University are still investigating the biological samples they collected in this study and continuing their efforts to progress worm molecules as therapies. For more information, read more about James Cook University.

 

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.” The antibody is being tested for the treatment of refractory celiac disease. Read more about Hu-Mik-Beta-1.

Precigen ActoBio, ActoBiotics® AG017

Precigen ActoBio (formerly ActoBio Therapeutics) is an innovative clinical-stage biotechnology company focused on a new class of microbe-based therapeutic agents. Precigen ActoBio’s AG017 (ActoBiotics®) is an antigen-specific immunotherapy candidate based on the safe food-grade bacterium Lactococcus lactis, specifically engineered to express a gliadin peptide (a component of gluten) in combination with a cytokine (a protein that modifies the immune system). This investigational therapy has the potential to reverse gluten sensitivity. Precigen ActoBio is hoping to test AG017 for safety and efficacy in celiac disease patients. Read more about Precigen ActoBio.

 

Takeda Pharmaceuticals, TAK-062

After a successful phase 1 proof-of-mechanism trial, Takeda acquired PvP Biologics and is continuing the development of TAK-062 (also known as Kuma062, formerly KumaMax). TAK-062 is a highly potent enzyme that is designed 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. Takeda is planning a phase 2b efficacy and dose-ranging study of TAK-062 in celiac patients who maintain a gluten-free diet. Read more about Takeda.

 

 

Phase 2:

Amgen and Provention Bio, PRV-015 (formerly AMG 714)

Provention Bio licensed PRV-015 (formerly AMG 714) from Amgen in 2018. PRV-015 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. The Celiac Disease Foundation is recruiting for Provention Bio’s PROACTIVE Celiac Study, a phase 2b study to test the safety and efficacy of PRV-015 in adult patients who are still experiencing celiac disease symptoms while on a gluten-free diet. See if you qualify.

Dr. Falk Pharma and Zedira, ZED1227

ZED1227 is the first direct acting transglutaminase inhibitor in clinical development. The 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 are now investigating efficacy of ZED1227 in celiac disease patients in a phase 2a clinical trial as of spring 2018. Read more about Dr. Falk and Zedira.

 

 

IGY Life Sciences, Vetanda Group Ltd., and The University of Alberta, AGY-010

Researchers from the University of Alberta and IGY have developed oral egg yolk anti-gliadin antibody (AGY), a novel treatment designed to neutralize the hidden gliadin in food. 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 phase 2 trial is currently underway to test the safety and efficacy of an orally-administered capsule containing egg yolk with AGY in patients with celiac disease. Read more about Vetanda.

 

 

 

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 1 and phase 2 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®

TRIAL DISCONTINUED June 25, 2019: Read more about this. ImmusanT is a clinical development-stage biotechnology company which developed Nexvax2®, a therapeutic vaccine candidate which was intended to potentially enable celiac disease patients to return to a normal diet. Booster shots of Nexvax2® were anticipated to offer periodic reinforcements of the treatment to establish a prolonged tolerance to gluten. Unfortunately, results from an interim analysis revealed Nexvax2 did not provide statistically meaningful protection from gluten exposure for celiac disease patients when compared with placebo.

Takeda Pharmaceuticals and Cour Pharmaceuticals, TAK-101

Takeda licensed TAK-101 (formerly CNP-101 or TIMP-GLIA) from Cour Pharmaceuticals in 2019. With TAK-101, 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. Takeda intends to initiate a dose-ranging study in 2021 to further explore the potential of TAK-101 in the treatment of patients with celiac disease on a gluten-free diet. Read more about Takeda.

 

Phase 3:

9 Meters Biopharma, Larazotide

In February 2016, 9 Meters (formerly known as Innovate Biopharmaceuticals) licensed the assets of Alba Therapeutics relating to larazotide, a tight junction regulator. Larazotide is a novel oral peptide that has consistently demonstrated the reduction of symptoms of celiac disease in multiple clinical trials. Larazotide helps restore “leaky” or open junctions to a normal state. Larazotide, 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 9 Meters.

 

 

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