Future Therapies for Celiac Disease

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The only 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, most often due to a failure to adhere to the gluten-free diet strictly enough. Future therapies currently in the pipeline hope to make living with celiac disease less of a burden.

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. For more information, click here.

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. For more information, click here.

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. For more information, click here.

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. For more information, click here.

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. For more information, click here.

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, click here.

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

PvP Biologics, 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. For more information, click here.

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. For more information, click here.

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. For more information, click here.

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. For more information, click here.

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. For more information, click here.

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. For more information, click here.

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. For more information, click here.

Glenmark Pharmaceuticals Ltd., GBR 830
Glenmark Pharmaceuticals Ltd. (GPL) developed GBR 830, a novel antibody for the treatment of celiac disease. GBR 830 targets activated T cells, which is the type of cell that causes the issues in most autoimmune diseases. The phase I studies for GBR 830 were well tolerated and its safety supports the transition into clinical phase II studies. For more information, click here.

Phase 2:

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. For more information, click here.

ImmunogenX, IMGX003
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. For more information, click here.

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. For more information, click here.

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. For more information, click here.


Summary of Clinical Trial Phases
Phase Primary goal Dose Patient monitor Typical number of participants Notes
Preclinical Testing of drug in non-human subjects, to gather efficacy, toxicityand pharmacokinetic information unrestricted A graduate level researcher (Ph.D.) not applicable (in vitro and in vivo only)
Phase 0 Pharmacokinetics particularly oral bioavailability and half-life of the drug very small, subtherapeutic clinical researcher 10 people often skipped for phase I
Phase I Testing of drug on healthy volunteers for dose-ranging often subtherapeutic, but with ascending doses clinical researcher 20-100 determines whether drug is safe to check for efficacy
Phase II Testing of drug on patients to assess efficacy and safety therapeutic dose clinical researcher 100-300 determines whether drug can have any efficacy; at this point, the drug is not presumed to have any therapeutic effect whatsoever
Phase III Testing of drug on patients to assess efficacy, effectiveness and safety therapeutic dose clinical researcher and personal physician 1000-2000 determines a drug’s therapeutic effect; at this point, the drug is presumed to have some effect
Phase IV Postmarketing surveillance – watching drug use in public therapeutic dose personal physician anyone seeking treatment from their physician watch drug’s long-term effect

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Current Treatment

Currently, the only treatment for celiac disease and non-celiac wheat sensitivity is lifelong adherence to a strict gluten-free diet. People living gluten-free must avoid foods with wheat, rye and barley, such as bread and beer. Ingesting small amounts of gluten, like crumbs from a cutting board or toaster, can trigger intestinal damage.
Learn about the Gluten-Free Diet