A New and Improved, Safer Alternative to Imatinib

At Inhibikase, we developed IkT-001Pro as an outgrowth of our efforts to improve the safety of the first FDA-approved Abl kinase inhibitor, imatinib (Gleevec®), which is commonly taken for hematological and gastrointestinal cancers that arise from Abl kinase mutations in bone marrow or Kit kinase mutations in the stomach.

IkT-001Pro has shown to be potentially as much as 5 times safer than imatinib in non-human primates, blunting the severe gastrointestinal side effects of imatinib therapy that occur following oral administration. Removing these gastrointestinal side effects has the potential to significantly improve the number of patients that reach and sustain complete cytogenetic response in stable-phase chronic myelogenous leukemia (CML). We believe that removing these side effects will also improve patient adherence to daily therapy and improve the quality of life of patients while on therapy.

IkT-001Pro has received Orphan Drug Designation for stable-phase CML and will follow the development pathway for approval through the 505(b)(2) regulation. This pathway would allow us to rely, in part, on data in the public domain or the FDA’s prior conclusions regarding the safety and effectiveness of an approved compound.

About Chronic Myeloid Leukemia

Chronic myeloid leukemia (CML)1 is a slowly progressing cancer that affects the blood and bone marrow. In CML, a genetic change takes place in an immature version of myeloid cells—the cells that make red blood cells, platelets, and most types of white blood cells. This change creates an abnormal gene product, called BCR-ABL, which transforms the cell into a CML cell. The leukemia cells increasingly grow and divide in an unregulated manner, eventually spilling out of the bone marrow and circulating in the body via the bloodstream. Because they are not fully mature, CML cells are unable to work properly to fight infections. In time, the cells can also settle in other parts of the body, including the spleen. CML is a form of slow-growing leukemia that can change into a fast-growing form of acute leukemia that is difficult to treat.

1. Also known as chronic myelogenous leukemia, chronic myelocytic leukemia, and chronic granulocytic leukemia (CGL)

RAMPTM Drug Innovation Engine

Our RAMP™ (Re-engineering Approach with Metabolism Preserved) drug innovation engine enables us to design and develop novel small-molecule product candidates with enhanced potency and preserved safety profile for the treatment of neurodegenerative diseases. 

Our Technology