Treatment with KAN-101 was Safe and Tolerated, Demonstrated Immune Responses and Dose-dependent Inhibition of Key Disease Biomarker, Supporting Advancement into Phase 2 Trial in Second Half 2022
KAN-101 Granted FDA Fast Track Designation for the Treatment of Celiac Disease
Cambridge, Mass. and Lausanne, Switzerland, May 23, 2022 – Anokion SA, a clinical-stage biotechnology company focused on treating autoimmune disease by restoring normal immune tolerance, announced positive clinical trial results from the company’s Phase 1 ACeD (Assessment of KAN-101 in Celiac Disease) clinical trial evaluating its lead candidate, KAN-101, for the treatment of individuals with celiac disease, a serious autoimmune disease triggered by ingestion of gluten for which there are no approved therapeutic treatments available. KAN-101 is designed to induce tolerance to gliadin, a core component of gluten, through natural pathways in the liver. Findings from the ACeD trial showed that treatment with KAN-101 was safe and tolerated, and reduced T cell responses following gluten challenge.
The ACeD trial data will be presented in a plenary session during Digestive Disease Week (DDW) 2022, today, Monday, May 23, 2022, at 8:00 a.m. PT. The presentation entitled “Safety, tolerability and pharmacodynamic assessment of KAN-101, a novel liver-targeted therapy to induce immunological tolerance to gliadin in celiac disease,” will be given by Joseph A. Murray, M.D., professor of medicine and consultant, Division of Gastroenterology and Hepatology, Department of Internal Medicine, and Department of Immunology at the Mayo Clinic and lead investigator in the ACeD trial.
“Celiac disease is a chronic autoimmune disorder in which gliadin-specific T cells drive an inappropriate immune response to gluten peptides that can result in severe, life-altering symptoms and long-term gut damage,” said Dr. Murray. “By inducing immune tolerance of these cells in patients, KAN-101 has the potential to offer a durable, disease-modifying treatment for celiac disease. As the data presented at DDW show, treatment with KAN-101 resulted in meaningful responses across several endpoints and disease biomarkers and showed good safety and tolerability across dose levels evaluated. I am highly encouraged by these data and look forward to Anokion’s continued advancement with this novel, selective agent.”
Phase 1 ACeD Trial Detailed Findings
The Phase 1, randomized, double-blind, placebo-controlled trial enrolled a total of 41 individuals with celiac disease on a gluten-free diet in both single-ascending dose (SAD) and multi-ascending dose (MAD) cohorts. The primary endpoint of the ACeD trial is to assess the safety and tolerability of KAN-101, with secondary endpoints to assess KAN-101 serum concentrations and pharmacokinetics. In addition, exploratory endpoints include assessing cytokines critical to both innate and adaptive immunity, T cell responses, and other serum cytokines and celiac disease symptoms.
In the SAD cohort, patients received a single dose of KAN-101 at one of five dose levels: 0.15, 0.3, 0.6, 1.2 and 1.5 mg/kg, and in the MAD cohort, patients were randomized three to one to receive three administrations of KAN-101 at one of three dose levels: 0.15, 0.3, and 0.6 mg/kg or placebo on days 1, 4 and 7 followed by a 3-day oral gluten challenge starting on day 15. Findings demonstrated:
- Administration of KAN-101 led to a reduction of gliadin (a protein present in wheat) specific T cell responses following gluten challenge as measured by both whole blood restimulation with gliadin and by ELISpot analysis (a highly sensitive immunoassay that measures the frequency of antigen-specific T cells), compared to patients who received placebo
- No broad immunosuppression was observed across several immune biomarkers following administration of KAN-101
- Administration of KAN-101 lead to dose-dependent reductions of gluten-induced plasma IL-2, a cytokine that is elevated in patients with celiac disease following gluten ingestion and which has been shown to correlate with severity of acute symptoms
- Statistically significant reductions of IL-2 were observed in patients who received the 0.6mg/kg dose compared to other groups
- Administration of KAN-101 did not increase gut-homing CD8 T cell responses after gluten challenge, an indicator of immune response to gluten exposure in patients with celiac disease
- KAN-101 treatment was safe and tolerated with no deaths, serious adverse events (SAEs) or dose limiting toxicities observed across all dose levels evaluated
- No clinically relevant trends were observed for vital signs, laboratory or ECGs results and no increases in liver function tests
- The most common adverse events were mild to moderate celiac-like reactions, including nausea, vomiting, abdominal pain, and diarrhea that resolved within a few hours of onset
“We are thrilled with the totality of data generated with KAN-101 in the ACeD trial, which demonstrated statistical evidence of biological activity across multiple clinical endpoints and immunological human proof-of-concept, with no safety signals observed and a safe and tolerated profile in patients,” said Deborah Geraghty, Ph.D., chief executive officer of Anokion. “We are particularly struck by the statistically significant reductions in gluten-induced plasma IL-2, which we believe represent the first-ever dose-dependent effect on this important disease biomarker. These positive data, the continued unmet need for celiac disease patients supported by the recent Fast Track designation of KAN-101 and the recently released draft guidance by the FDA on developing adjunctive treatments for celiac disease, are collectively informing our plans to move quickly into a Phase 2 clinical trial with KAN-101 in the second half of the year. We are incredibly thankful to the patients and physicians who participated in the ACeD study, and look forward to initiating our Phase 2 trial, so that we may be able to bring forward a much-needed treatment to patients suffering from celiac disease.”
KAN-101 Granted FDA Fast Track Designation
In addition, Anokion announced today that the U.S. Food and Drug Administration has granted Fast Track designation to KAN-101 for the treatment of celiac disease. Fast Track is a process designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need. The purpose is to get important new drugs to the patient earlier. Fast Track addresses a broad range of serious conditions, and the request can be initiated by the drug company at any time during the development process. FDA will review the request and decide based on whether or not the drug fills an unmet medical need in a serious condition. Once a drug receives Fast Track designation, early and frequent communication between the FDA and the sponsor is encouraged throughout the entire drug development and review process.
Anokion SA is a clinical-stage Swiss biotechnology company that aims to make a meaningful difference in the lives of patients suffering from autoimmune diseases by restoring normal immune tolerance. The company is focused on both prevalent and rare autoimmune diseases, including celiac disease, multiple sclerosis and type 1 diabetes. Anokion’s distinct approach leverages the company’s immune-based platform, which targets natural pathways in the liver to restore immune tolerance and address the underlying cause of autoimmune disease. For more information, please visit anokion.com.
THRUST Strategic Communications