PALO ALTO, Calif.--(BUSINESS WIRE)-- Immune-Onc Therapeutics, Inc. (“Immune-Onc”), a clinical-stage cancer immunotherapy company today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation status for its first-in-class antagonist antibody IO-202 for treatment of acute myeloid leukemia (AML). In addition, the company announced acceptance of its “Trials in Progress” poster presentation for IO-202 at the 62nd American Society of Hematology (ASH) Annual Meeting and Exposition. The meeting is taking place virtually December 5-8, 2020.

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, to advance the evaluation and development of products that demonstrate promise for the diagnosis and treatment of rare diseases or conditions that affect fewer than 200,000 people in the United States. Orphan Drug Designation can also convey seven years of marketing exclusivity for a drug approved to treat an orphan disease in the United States.

“Receiving orphan drug designation for IO-202 in AML is another important milestone for Immune-Onc and underscores the need for effective new treatments for this aggressive and hard-to-treat cancer,” said Charlene Liao, Ph.D., chief executive officer of Immune-Onc. “We are pleased with the progress we are making in advancing IO-202. As outlined in our ASH poster presentation, IO-202 holds promise for AML patients because it demonstrates novel mechanisms of action in overcoming immune suppression. IO-202 is one of several programs in our pipeline that target the LILRB family of immune inhibitory receptors. We are excited to continue our momentum in evaluating IO-202 and Immune-Onc’s preclinical candidates in other cancers, including solid tumors, in the near future.”

ASH “Trials in Progress” Poster

Based on highly selective criteria, “Trials in Progress” abstracts are reviewed for the most innovative science. The abstract (#2867), “A First-in-Human (FIH) Phase 1 Study of the Anti-LILRB4 Antibody IO-202 in Relapsed/Refractory (R/R) Myelomonocytic and Monocytic Acute Myeloid Leukemia (AML) and R/R Chronic Myelomonocytic Leukemia (CMML)”, was selected for poster presentation in the 2020 ASH Annual Meeting Program. Lead investigator Courtney D. DiNardo, M.D., MSc, associate professor, Department of Leukemia, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center, will share the poster via a brief PowerPoint presentation with accompanying audio.

The poster (Abstract #2867) will be presented at Session: 616. Acute Myeloid Leukemia: Novel Therapy, excluding Transplantation: Poster III, on Monday, December 7, 2020, 7:00 a.m. - 3:30 p.m. Pacific Time.

In addition to the presentation, the abstract was published online in the November supplemental issue of Blood and in the online meeting program on November 5, 2020.

ABOUT LILRB4 (also known as ILT3)

LILRB4, also known as ILT3, is an immune-modulatory transmembrane protein found on antigen presenting cells (APCs). It inhibits APC activation and induces immune tolerance via T-suppressor cells. It is expressed on certain hematologic cancer cells and immune suppressive myeloid cells in the solid tumor microenvironment. Immune-Onc and The University of Texas published pioneering research in Nature illuminating the role of LILRB4 in immune suppression and tumor infiltration in AML.

ABOUT IO-202

IO-202 is a first-in-class monoclonal antibody that antagonizes LILRB4 with high binding affinity and specificity. It has broad potential in both blood cancers and solid tumors. In hematologic malignancies, preclinical studies showed that IO-202 converts a “don’t kill me” to a “kill me” signal by activating T cell killing and converts a “don’t find me” to a “find me” signal by inhibiting infiltration of hematologic cancer cells.

IO-202 is being evaluated in a Phase I trial in two forms of blood cancer, AML and CMML. The U.S. Food and Drug Administration granted IO-202 Orphan Drug Designation status for treatment of AML in October 2020.

In solid tumors, IO-202 has potential to be combined with anti-PD-(L)1, other immunotherapies, and/or immunogenic chemotherapy. The company plans to evaluate IO-202 in solid tumors and in other forms of blood cancer in the near future.

ABOUT AML and CMML

AML, the most common acute leukemia (blood and bone marrow cancer) in adults, is characterized by the proliferation of abnormal myeloblasts (a type of white blood cell) in the bone marrow. Nearly 20,000 new cases are expected in the U.S. in 2020. Despite advances in treatment, less than 30 percent of acute myeloid leukemia patients are alive five years after initial diagnosis.

CMML is a cancer that starts in blood-forming cells in the bone marrow and invades the blood. The condition is rare, with about 1,100 cases in the U.S. each year.

ABOUT IMMUNE-ONC THERAPEUTICS

Immune-Onc Therapeutics, Inc. (“Immune-Onc”) is a clinical-stage cancer immunotherapy company dedicated to the discovery and development of novel myeloid checkpoint inhibitors for cancer patients. 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 biotechnology companies.

The company aims to translate unique scientific insights in myeloid cell biology and immune inhibitory receptors to discover and develop first-in-class biotherapeutics that disarm immune suppression in the tumor microenvironment. Immune-Onc has a promising pipeline with a current focus on targeting the Leukocyte Immunoglobulin-Like Receptor subfamily B (LILRB) of myeloid checkpoints. The company has strategic research collaborations with The University of Texas, Albert Einstein College of Medicine, and Memorial Sloan Kettering Cancer Center, and has invested in proprietary models, assays and tools to interrogate the biology and translate this cutting-edge research into the development of novel therapies.

In addition to IO-202, Immune-Onc’s pipeline includes IO-108, an antibody targeting LILRB2 (also known as ILT4) in the IND-enabling stage of development. Additional preclinical candidates focused on myeloid checkpoints include an anti-LAIR1 antibody and multiple undisclosed programs for solid tumors and hematologic malignancies. For more information, please visit www.immune-onc.com and follow us on Twitter and LinkedIn.

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