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.
BioLineRx is a clinical-stage biopharmaceutical company dedicated to identifying, in-licensing and developing promising therapeutic candidates. Their strategy involves a drug called BL-7010 which is a large, non-absorbable polymer. A Phase I/II study with BL-7010 was successfully completed in 2014. When taken orally, it was demonstrated that BL-7010 prevents the degradation of the protein in gluten, called gliadin, which causes the over-reactive immune response. With gliadin still intact, immune cells are not triggered to form, which prevents the harmful immune response those with celiac disease would usually experience.
BioLine has received confirmation regarding the classification of BL-7010 as a Class IIb medical device in the European Union, and will continue to evaluate the pathway for celiac disease in Europe. In addition, the Company is conducting a number of activities towards the development of BL-7010 as a food supplement, including the development of a suitable product formulation, preparation of the documents necessary for a GRAS (generally regarded as safe) designation submission, and preparations for a relatively small clinical trial to support future marketing efforts.
For more information, email info@BioLineRx.com.
Celimmune, AMG 714
Celimmune is a clinical development-stage immunotherapy company dedicated to developing transformational therapies that will relieve the burden of celiac disease and refractory celiac disease (refractory sprue) and other serious immune-mediated diseases. Celimmune licensed AMG 714 from Amgen in 2015. AMG 714 iis a monoclonal 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, and other autoimmune conditions. AMG 714 has been studied in approximately 200 patients with rheumatoid arthritis and psoriasis, as well as in healthy volunteers. In a Phase Ib study of AMG 714 in rheumatoid arthritis, which has been published by Baslund and collaborators, AMG 714 appeared to have biological and clinical activity, and demonstrated a safety profile comparable to placebo.
The selectivity, safety and pharmacokinetic profile, in conjunction with the evidence of activity in rheumatoid arthritis, supports the testing of AMG 714 in celiac disease and Type II refractory celiac disease. Celimmune has initiated Phase II studies of AMG 714 for the treatment of diet non-responsive celiac disease and RCD-II, an in situ small bowel T cell lymphoma, and is seeking volunteers for this trial.
For more information, email email@example.com.
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ImmusanT is a clinical development-stage biotechnology company focused on an innovative disease-modifying approach to induce immune tolerance in autoimmune diseases. Nexvax2® is the only disease-modifying treatment in clinical development today that has the potential to enable patients to return to a normal diet. It is a combination of three proprietary peptides that elicit an immune response in patients with celiac disease (who carry the immune recognition gene, HLA-DQ2.5, which accounts for the condition in 80% to 90% of patients. Nexvax2® is delivered intradermally as a therapeutic vaccine, on the same principles as “traditional” desensitization therapy for allergic conditions. It reprograms the T-cells that respond to gluten antigens in patients so that they stop responding defensively by triggering a pro-inflammatory response. As a result, by preventing T-cells from continuing to cause inflammation in the small intestine, the injured tissue heals and patients would be able to resume an unrestricted diet and enjoy improved health. 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.
ImmusanT has recently completed two Phase Ib safety, tolerability, and pharmacodynamics clinical trials, which provided unprecedented data supporting proof-of-mechanism and effectiveness. Specifically, Nexvax2® was found to engage antigen-specific target cells, and a distinct immunological signature was discovered. After repeated dosing, patients who received Nexvax2® were able to complete gluten challenge in contrast to placebo. A multi-nation Phase II trial is planned for 2017.
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Innovate Biopharmaceuticals, INN-202 (Larazotide Acetate)
Innovate is a privately held, clinical-stage biotechnology company focused on developing novel gastroenterology medicines. In February 2016, Innovate licensed the assets of Alba Therapeutics relating to larazotide acetate, a tight junction regulator. Larazotide acetate, now named INN-202, is a novel oral peptide that has consistently demonstrated the reduction of clinical symptoms of celiac disease in multiple clinical trials in more than 800 patients with celiac disease. The latest Phase Ib multi-center clinical trial evaluated the efficacy, safety and tolerability of INN-202 in 342 patients with celiac disease.
INN-202 helps restore “leaky” or open junctions to a normal state. In celiac disease patients, ingestion of gluten causes the tight junctions to remain open. The open junctions cause an inflammatory cascade within the intestinal epithelium that eventually destroys the intestinal villa. 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.
Larazotide acetate has the potential to become the first approved medicine to treat celiac disease and has been granted “Fast Track” designation from the FDA. INN-202 will begin Phase III clinical trials in late 2016. Innovate is targeting 2018 for drug approval.
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|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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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.