Novel CEACAM Antibody Lands FDA Orphan Drug Designation for HCC


YB-200, an investigational CEACAM antibody, has caught the attend of the FDA.

The FDA has granted an orphan drug designation to YB-200 for the treatment of patients with hepatocellular carcinoma (HCC).1

YB-200 is a carcinoembryonic antigen-related cell adhesion molecule (CEACAM) antibody with dual action. The agent acts as an immune checkpoint inhibitor and causes direct immune agonistic reaction on immune cells.

"This designation is an important milestone in our development of CEACAM antibodies as anti-cancer treatments," said Katrin Rupalla, PhD, founder and chief executive officer of Ymmunobio, in a press release. "We're very pleased to continue our investigation into CEACAM antibodies for the treatment of hepatocellular carcinoma."

Research suggests that the use of CEA as a biomarker to watch disease progression is well-established.2 By mainly targeting CEACAM-5 and CEACAM-6, CEA targeted therapy demonstrated preclinical efficacy in colorectal cancer and melanoma2, and the data suggests that it may also have activity in pancreatic cancer, non–small cell lung cancer, acute myeloid leukemia, and multiple myeloma.3

Preclinical research also shows that YB-200 led to statistically significant tumor growth inhibition vs an isotype control. Moreover, there was a 10-fold increase in B cells in the tumor microenvironment following YB-200 treatment vs the isotype control. YB-200 also achieved a statistically significant boost in CD3+ and CD4+ T cells while decreasing granulocytes.4

In terms of safety, YB-200 has shown thrombocytopenia, anemia, and grade 3/4 hematologic toxicity in clinical trials. Multiple trials are ongoing to evaluate the efficacy and safety of YB-200 in gastrointestinal cancers.


1. FDA grants orphan drug designation to Ymmunobio's CEACAM antibody for the treatment of liver cancer. News release. Ymmunobio. March 1, 2023. Accessed March 1, 2023.

2. Janicot M, Rupalla K, Schmidt A, et al. 1384 Novel, immune agonistic CEACAM1/5 antibody (YB-200) demonstrates anti-tumor efficacy and significantly increases B-cell response in syngeneic liver Hepa1-6 tumor microenvironment. J Immunother Cancer. 2022;10: doi:10.1136/jitc-2022-SITC2022.1384

3. Han Z, Lyv Z, Cui B, et al. The old CEACAMs find their new role in tumor immunotherapy. Invest New Drugs. 2020 Dec;38(6):1888-1898. doi:10.1007/s10637-020-00955-w

4. Tsang K, Fantini M, Mavroukakis S, et al. Development and characterization of an anti-cancer monoclonal antibody for treatment of human carcinomas. Cancers (Basel). 2022 Jul; 14(13): 3037. Published online Jun 21, 2022. doi: 10.3390/cancers14133037

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