PALO ALTO, Calif.--(BUSINESS WIRE)-- Immune-Onc Therapeutics Inc. (“Immune-Onc”), a clinical-stage biopharmaceutical company advancing novel therapies in immunology and oncology by targeting myeloid cell inhibitory receptors, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for IO-202 for the treatment of chronic myelomonocytic leukemia (CMML). IO-202 received Fast Track Designation for treatment of relapsed or refractory CMML in 2023. In addition, Fast Track and Orphan Drug Designations for IO-202 were granted by the FDA for the treatment of acute myeloid leukemia (AML) in 2022 and 2020, respectively.

IO-202 is a first-in-class antagonist antibody with specific, high affinity binding to Leukocyte Immunoglobulin-Like Receptor subfamily B4 (LILRB4) and serves as a targeted therapy with broad potential in blood cancers, autoimmune and inflammatory diseases. IO-202 in combination with azacitidine (AZA) is currently in a Phase 1 dose expansion clinical trial (NCT04372433), enrolling newly diagnosed patients with CMML who have not received any hypomethylating agents (HMA). A dose escalation trial of IO-202 has been completed, showing clinical efficacy in relapsed or refractory AML and CMML, either as a monotherapy or in combination with AZA. IO-202 has been shown to be well tolerated in all patients treated to date.

“Although current therapeutic options for CMML can improve a patient’s quality of life, there is a high unmet need for effective disease-modifying approaches that are potentially curative,” said Charlene Liao Ph.D., chief executive officer and board chair of Immune-Onc. “We are very proud that the FDA has granted IO-202 Orphan Drug Designation for the treatment of CMML. We look forward to continued collaborations with our investigators and the FDA as we work to bring this potentially important therapy to patients with hard-to-treat blood cancers.”

The FDA grants Orphan Drug Designation to medicines and potential new medicines intended for the treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the U.S. Orphan Drug Designation qualifies the sponsor for various development incentives of the Orphan Drug Act, including exemption of FDA application fees and tax credits for qualified clinical testing, and conveys seven years of marketing exclusivity for a drug approved to treat an orphan disease in the United States.

ABOUT CHRONIC MYELOMONOCYTIC LEUKEMIA (CMML)

CMML is a rare form of blood cancer, occurring in 4 of every 1,000,000 people in the United States each year, or about 1,100 annual cases¹. CMML is characterized by the presence of a high monocyte count (>1x10⁹/L peripheral monocytes with monocytes ≥ 10% of white blood count) along with dysplastic features in the bone marrow². Current FDA approved therapies for CMML are all hypomethylating agents, including azacitidine, that attempt to control CMML without enhancement to overall survival.

ABOUT LILRB4 (also known as ILT3)

LILRB4, also known as ILT3, is an immune-modulatory transmembrane protein found on monocytes and monocyte-derived cells. LILRB4 is expressed on certain hematologic cancer cells, such as myelomonocytic leukemia blasts, and on certain pathogenic cells involved in autoimmunity and inflammatory processes.

About IO-202

IO-202 is a first-in-class antagonist antibody with specific, high affinity binding to LILRB4. IO-202 is a humanized IgG1 antibody with Fc effector function to kill LILRB4^hi cells via antibody-dependent cellular cytotoxicity (ADCC) and antibody-dependent cellular phagocytosis (ADCP). As such, IO-202 is a targeted therapy with broad potential in both blood cancers and autoimmune and inflammatory diseases.

IO-202 has completed the dose escalation part of the first-in-human, multicenter, open-label Phase 1 study in the U.S., and the data was presented at the European Hematology Association (EHA) Congress in 2023. This Phase 1 trial has advanced to the dose expansion stage to evaluate IO-202 in combination with azacitidine (NCT04372433) in patients with newly diagnosed chronic myelomonocytic leukemia (CMML) who have not received any hypomethylating agents (HMA).

The U.S. Food and Drug Administration granted IO-202 Fast Track Designations for treatment for the treatment of patients with relapsed or refractory acute myeloid leukemia (AML) and for the treatment of patients with relapsed or refractory chronic myelomonocytic leukemia (CMML). The FDA has also granted Orphan Drug Designations to IO-202 for the treatment of AML and for the treatment of CMML.

ABOUT IMMUNE-ONC THERAPEUTICS, INC.

Immune-Onc Therapeutics Inc. (“Immune-Onc”) is a private, clinical-stage biopharmaceutical company developing novel therapies in immunology and oncology by targeting myeloid cell inhibitory receptors.

Immune-Onc has a differentiated pipeline with a current focus on targeting the Leukocyte Immunoglobulin-Like Receptor subfamily B (LILRB). Immune-Onc’s focused platform approach has led to the development of several promising therapeutics across various stages of development. Those include IO-108, an antagonist antibody targeting LILRB2 (also known as ILT4), in Phase 1b/2 clinical development for solid tumors, and IO-202, a first-in-class antagonist antibody targeting LILRB4 (also known as ILT3), in Phase 1b clinical development for the treatment of acute myeloid leukemia (AML), and chronic myelomonocytic leukemia (CMML). IO-202 has the potential to be the best-in-class antibody therapy for lupus and can extend to other indications in immunology and inflammation. Additional assets in Immune-Onc’s pipeline include IO-312 (a novel bispecific antibody targeting LILRB4 and CD3), IO-106 (first-in-class antagonist antibody targeting LAIR1), and undisclosed immunology and oncology programs.

Immune-Onc has established agreements with leading biopharmaceutical companies, including BeiGene, Regeneron and Roche, to support its global product development plans for IO-108 and IO-202. It has received research grants from the National Cancer Institute (NCI) of the National Institutes of Health (NIH) and the California Institute for Regenerative Medicine (CIRM) and investment from The Leukemia & Lymphoma Society Therapy Acceleration Program® (LLS TAP®). Headquartered in Palo Alto, California, Immune-Onc has assembled a diverse team with deep expertise in drug development and proven track records of success at leading biopharmaceutical companies. For more information, please visit www.immune-onc.com and follow us on X and LinkedIn.

1. Epidemiology of myelodysplastic syndromes and chronic myeloproliferative disorders in the United States, 2001-2004, using data from the NAACCR and SEER programs. Blood. 2008 Jul 1;112(1):45-52. doi: 10.1182/blood-2008-01-134858
2. Chronic myelomonocytic leukemia: 2022 update on diagnosis, risk stratification, and management. Am J Hematol. 2022 Mar 1;97(3):352-372. doi: 10.1002/ajh.26455.

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