Type 1 Diabetes Therapy Being Readied for Clinical Trials; Celiac Disease in Drug Development
Autoimmune diseases affect about 4% of the world’s population and comprise over 80 disorders, many of which such as type 1 diabetes (T1D) and celiac disease remain unmet medical needs with no approved therapy.Current treatments for autoimmune diseases with high prevalence such as rheumatoid arthritis, multiple sclerosis and inflammatory bowel disease (IBD) represent a 100 billion+ dollar global market but can have limited effectiveness and leave many autoimmune indications yet in need of a therapy.An approach to develop therapies targeted at the underlying genetic risk factors of disease should lead to potentially high-impact patient-centered treatments.
Several autoimmune conditions are a result of variant human leukocyte antigen (HLA) proteins that are otherwise involved in mediating normal immune response to pathogenic attacks.HLA DQ8, a variant responsible for 60% of T1D and about 10% of celiac binds insulin or α-gliadin peptides to trigger an autoimmune response from T cells leading to T1D and celiac, respectively.HLA DQ2 is involved in 90% of celiac and about 40% of T1D.Other HLA variants have been well established in their role in rheumatoid arthritis, neuromyelitis optica (NMO), Sjogren’s syndrome and narcolepsy.
IM Therapeutics was founded by world-class physician scientists, Drs. Peter Gottlieb and Aaron Michels, at CU’s Barbara Davis Center for Diabetes.The Company has developed an HLA-targeting platform directed at the root cause of autoimmunity to design small molecule drugs that optimally bind the HLA native peptide-binding domain – therefore, arresting the trigger of autoimmune T cell response.By screening hundreds of millions of drug combinations computationally, rational design of drug hits and screening with antigen-specific T cell assays, the Company has developed a lead drug, IMT-002 against HLA DQ8 in T1D and potentially celiac disease.The Company also demonstrated – first such targeted approach in autoimmune disease – clinical effectiveness in a Phase 1b study of recently diagnosed T1D patients pre-selected for positive status of the variant HLA DQ8.
“Our value is in creating personalized oral therapies in a range of autoimmune diseases as high-impact, patient-centered treatments with the potential for disease prevention down the road”
Nandan Padukone, PhD, CEO
The Company recently completed a Series A financing of $10 million led by the JDRF T1D Fund and Morningside Ventures with participation from CU Healthcare Innovation Fund.The funding will advance its lead drug, IMT-002, through IND approval and Phase 1 studies in T1D and potentially celiac disease.A program in DQ2 is underway for drug discovery in celiac disease and the Company plans to develop a broad pipeline of drug candidates against other HLA targets.