The U.S. Food and Drug Administration (FDA) has agreed to Immune-Onc Therapeutics launching a Phase 1 clinical trial of IO-202, its lead antibody treatment candidate, in patients with advanced acute myeloid leukemia (AML) and chronic myelomonocytic leukemia (CMML).
The announcement came after the agency cleared the company’s investigational new drug (IND) application asking that IO-202 be authorized to enter clinical testing.
“The FDA clearance to proceed with the first-in-human trial of IO-202 in AML and CMML is an important milestone for our company,” Charlene Liao, PhD, CEO of Immune-Onc, said in a press release.
“Acute myeloid leukemia is a devastating disease that needs new approaches and better treatment options. We look forward to working with investigators as we advance the first anti-LILRB4 antibody into the clinic,” Liao added.
IO-202 is an antibody that specifically recognizes and blocks the activity of LILRB4, a protein receptor found on the surface of immune T-cells that normally inhibits their activity. By blocking LILRB4, IO-202 is expected to promote the activation of T-cells, enhancing their ability to target and destroy malignant cells.
Preclinical studies in animal models of disease have shown that IO-202 was able to activate and instruct T-cells to eliminate leukemia cancer cells, effectively preventing tumor infiltration.
The Phase 1 clinical trial (NCT04372433), which is not yet recruiting, will assess the safety, tolerability, clinical activity, and pharmacological properties of IO-202 in people with relapsed or refractory AML and CMML. It is expected to open in July, and finish collecting top-line data in December 2023.
The trial will be divided into two phases. The first is an initial dose-escalation phase, in which participants will receive increasing doses of the medication until the optimal dose is found. The second is an expansion phase, in which patients will be treated with the determined optimal dose of IO-202 for a longer period of time.
The study’s protocol may still be amended to allow IO-202 to be given alongside other medications, such as with Vidaza (azacitidine), rather than alone.
In addition to safety and pharmacological assessments, investigators will also evaluate changes in the levels of several biomarkers they hope will support IO-202’s mechanism of action, and be informative for future clinical trials.
Immune-Onc Therapeutics is also planning to explore IO-202’s potential to treat other types of blood and solid cancers.