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Immune-Onc Therapeutics Receives Orphan Drug Designation from US FDA for IO-202 (Anti-LILRB4) Targeting Chronic Myelomonocytic Leukemia (CMML)

Thursday, February 22, 2024

Immune-Onc Therapeutics, Inc. ("Immune-Onc"), a biopharmaceutical company focusing on immunology and oncology treatments targeting myeloid cell inhibitory receptors, announced today that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to IO-202 for chronic myelomonocytic leukemia (CMML). IO-202 received Fast Track Designation for relapsed or refractory CMML in 2023, and previously, Fast Track and Orphan Drug Designations for acute myeloid leukemia (AML) in 2022 and 2020, respectively.

IO-202 is a novel antagonist antibody binding to Leukocyte Immunoglobulin-Like Receptor subfamily B4 (LILRB4), offering targeted therapy for various blood cancers, autoimmune, and inflammatory diseases. Currently, IO-202 is in a Phase 1 dose expansion clinical trial (NCT04372433), in combination with azacitidine (AZA), for newly diagnosed CMML patients who haven't received hypomethylating agents (HMA). Clinical trials have demonstrated IO-202's efficacy in relapsed or refractory AML and CMML, either as a monotherapy or combined with AZA, with a favorable safety profile in all treated patients.

Immune-Onc, emphasized the significant unmet need for effective CMML treatments and expressed pride in the FDA's Orphan Drug Designation for IO-202. Liao reiterated Immune-Onc's commitment to collaborating with investigators and the FDA to bring this promising therapy to patients with challenging blood cancers.

Orphan Drug Designation, granted by the FDA to drugs addressing rare diseases affecting fewer than 200,000 individuals in the U.S., provides sponsors with various development incentives under the Orphan Drug Act. These include FDA application fee exemptions, tax credits for qualified clinical testing, and seven years of marketing exclusivity upon approval for treating an orphan disease in the United States.

CMML is a rare blood cancer, with approximately 1,100 cases reported annually in the United States, affecting 4 out of every 1,000,000 people. It is characterized by elevated monocyte counts and dysplastic features in the bone marrow. Currently, FDA-approved therapies for CMML predominantly consist of hypomethylating agents like azacitidine, aiming to manage the disease without significant improvements in overall survival.

LILRB4, also identified as ILT3, is an immune-modulatory transmembrane protein found on monocytes and their derivatives. It is expressed on certain hematologic cancer cells, including myelomonocytic leukemia blasts, and certain pathogenic cells implicated in autoimmunity and inflammation.

IO-202, a first-in-class antagonist antibody, exhibits specific, high-affinity binding to LILRB4. It is a humanized IgG1 antibody with Fc effector function, enabling the killing of LILRB4hi cells via antibody-dependent cellular cytotoxicity (ADCC) and antibody-dependent cellular phagocytosis (ADCP). Consequently, IO-202 represents a targeted therapy with broad potential across blood cancers, autoimmune, and inflammatory diseases.

The Phase 1 trial of IO-202, which completed its dose escalation part and presented data at the 2023 European Hematology Association (EHA) Congress, has progressed to the dose expansion stage. This stage evaluates IO-202 in combination with azacitidine (NCT04372433) in newly diagnosed CMML patients who have not received HMAs.

IO-202 has received Fast Track Designations from the FDA for treating relapsed or refractory AML and CMML. Additionally, the FDA granted Orphan Drug Designations to IO-202 for treating both AML and CMML.



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