Immune-Onc Therapeutics gets FDA orphan drug status for IO-202 in CMML

Immune-Onc Therapeutics, Inc., a clinical-stage biopharmaceutical company focused on the development of innovative therapies in immunology and oncology, has achieved a significant milestone with the U.S. Food and Drug Administration’s (FDA) recent Orphan Drug Designation for IO-202. This designation is a crucial step forward in the treatment of chronic myelomonocytic leukemia (CMML), a rare form of blood cancer. Furthermore, IO-202 had previously received Fast Track Designation for the treatment of relapsed or refractory CMML in 2023, underscoring its potential as a significant advancement in oncology.

IO-202 is at the forefront of targeted cancer therapy, being a first-in-class antagonist antibody that specifically binds with high affinity to the Leukocyte Immunoglobulin-Like Receptor subfamily B4 (LILRB4). This targeted approach has broad implications not only for blood cancers but also for autoimmune and inflammatory diseases. Currently, IO-202 is undergoing a Phase 1 dose expansion clinical trial in combination with azacitidine (AZA), targeting newly diagnosed CMML patients who have not yet been treated with hypomethylating agents (HMA).

Charlene Liao, CEO and board chair of Immune-Onc, emphasized the urgent need for effective disease-modifying treatments for CMML. Despite existing therapies that can improve quality of life, a gap remains for treatments that offer a potential cure. Liao’s statement, “We are very proud that the FDA has granted IO-202 Orphan Drug Designation for the treatment of CMML,” highlights the company’s commitment to addressing this unmet need and their ongoing collaboration with the FDA and medical investigators.

The FDA’s Orphan Drug Designation is reserved for promising new therapies intended to treat, diagnose, or prevent rare diseases affecting fewer than 200,000 people in the U.S. annually. This designation brings several development incentives, including exemption from FDA application fees and tax credits for clinical testing, alongside seven years of marketing exclusivity upon drug approval. For IO-202, this designation marks an important step toward bringing a new, potentially life-altering therapy to patients with CMML.

CMML presents a unique challenge in oncology, characterized by a high monocyte count and dysplastic features in the bone marrow. The current standard of care, hypomethylating agents like azacitidine, has limited impact on overall survival, underscoring the need for more effective treatments. IO-202’s targeting of LILRB4, an immune-modulatory protein expressed on certain cancer and pathogenic cells, offers a novel approach to therapy that could significantly impact treatment paradigms.

The progression of IO-202 through clinical trials, including the completion of a dose escalation trial and the ongoing Phase 1 dose expansion trial, demonstrates significant clinical efficacy and tolerability in patients. With Fast Track and Orphan Drug Designations for both CMML and acute myeloid leukemia (AML), IO-202 stands as a beacon of hope for patients with these challenging conditions. Immune-Onc’s dedication to advancing IO-202 through clinical development is a testament to their commitment to addressing hard-to-treat blood cancers and improving patient outcomes.

The FDA’s recognition of IO-202 through Fast Track and Orphan Drug Designations underscores the potential impact of this therapy on the treatment landscape for CMML and AML. As Immune-Onc continues to explore the full potential of IO-202, the oncology community watches with keen interest, anticipating further developments that could revolutionize treatment options for patients with these rare blood cancers.