WOBURN, Mass.–(BUSINESS WIRE)–ImmunoMolecular Therapeutics, Inc. (“IM Therapeutics”), a leading innovator in the field of precision medicine for autoimmune diseases, today announced it has launched a Phase 1b study as a multiple ascending dose (MAD) trial. The study is in patients who have type 1 diabetes (T1D), an as yet incurable autoimmune disorder that affects nearly 1.6 million Americans, and who also have a specific genetic trait known as HLA-DQ8, present in about 60% of T1D patients. Variants in certain genes called human leukocyte antigens (HLA) are known to play a strong role in activating an autoimmune response.
The Phase 1b trial will enroll 30 patients between the ages of 18 and 45 into four groups, each of which will have six subjects receiving varying doses of IMT-002 over a two-week duration. The remaining six subjects will receive placebo. The study will compare drug versus placebo for safety, tolerability and pharmacokinetic (PK) profiles over the two-week duration of dosing and a follow-up one week after dose completion. In addition, the study will assess pharmacodynamic (PD) activity with a blood-based assay that measures the ability of IMT-002 to block DQ8 presentation of “self-antigens” or the body’s own proteins, such as insulin or gluten, that can result in autoimmunity.
In conjunction with the Phase 1b study, IM Therapeutics announced the completion of a Phase 1a study of IMT-002 in healthy subjects. The study recruited 28 volunteers in four different groups, each of which had six subjects on a single ascending dose of IMT-002 and one subject on placebo. The study showed no adverse events in any of the groups. In addition, pharmacokinetic (PK) profiles followed over a 24-hour period after drug administration indicated an average drug half-life of about five hours and measurable levels in blood up to 12 hours. The completion of Phase 1a has helped inform frequency and levels of dosing in the Phase 1b study.
“We are encouraged by the favorable safety findings in our first study,” said Nandan Padukone, Ph.D., CEO of IM Therapeutics. “The PK profile of our lead drug candidate indicates a strong possibility of eventually having a once-daily oral therapy for patients with recent diagnosis of T1D, more than half of whom are pediatric patients,” he added. “We will continue to focus on expanding our pipeline of therapeutic candidates for patients impacted by autoimmune disease including celiac, lupus, and rare diseases.”
“We look forward to not only showing further safety and tolerability of our drug candidate, but also its potential as a selective inhibitor of DQ8-related autoimmunity,” said Sarah Bird, Ph.D., VP of Clinical Development at IM Therapeutics. “Tailored therapies against known genetic targets can significantly improve patient-centered care in autoimmune disease.”
IMT-002 has been developed as a selective HLA-DQ8 blocker based on extensive computational work, in vitro and in vivo characterization. Previous studies of a tool drug, L-methyldopa, which is FDA-approved for treating hypertension, in a Phase 1b study, showed effective inhibition of DQ8 activity in new onset type 1 diabetes patients who had the DQ8 gene variant. Several in vivo IND studies indicate that IMT-002, which unlike the tool drug, is not metabolized physiologically, has more potency to block DQ8 activity and a favorable safety profile. Extensive preclinical studies of IMT-002 have been completed leading to IND clearance prior to initiation of human clinical trials.
About IM Therapeutics
IM Therapeutics (http://imtherapeutics.com/) is a clinical stage company developing personalized medicines for autoimmune diseases by building oral drug therapies against human leukocyte antigen (HLA) variants known to confer high risk of disease. The Company’s technology platform develops small molecule drugs using in silico docking of millions of compounds into pockets of an HLA protein where self-antigens may bind to trigger autoimmunity. Selected drug hits are then optimized using proprietary structure-based design and activity screening with cell-based assays for specificity of HLA inhibition. Lead drugs developed against an HLA variant have the ability to block a series of self-antigens and therefore the potential to treat several autoimmune indications related to a selected HLA. The Company is building a broad HLA-targeted pipeline in autoimmune disorders including celiac disease. In June 2019, IM Therapeutics raised $10 million in Series A financing led by Morningside Ventures and the JDRF T1D Fund.