PAb 001

PAb 001, the First-In-Class Novel Therapeutic Antibody, Targeting OT-MUC1 for Cancer Therapy


Mucin1 (MUC1) is a highly glycosylated transmembrane protein that plays a crucial role in the lubrication and protection of normal epithelial cells. However, MUC1 has emerged as a potential target for cancer therapy because it is overexpressed and functions in several types of cancers.(which is the one of the most promising cancer targets as NCI reported)

Mucin1 (MUC1) is a heterodimeric glycoprotein containing an extracellular N-terminal domain (MUC1-N) and a C-terminal domain (MUC1-C). MUC1-C has an extracellular domain, transmembrane domain and cytoplasmic tail and thus spans the cell membrane. MUC1 C-terminal subunit (MUC1-C) is composed of a 58-amino-acid extracellular domain, 28-amino-acid transmembrane domain, and 72-amino-acid cytoplasmic tail .

MUC1 is overexpressed in a wide range of human epithelial malignancies including breast, prostate, ovarian, pancreatic, and colon cancers and also in malignant plasma cells in multiple myeloma. In these conditions, the MUC1-overexpressing cells lose their polarity and MUC1 is aberrantly distributed throughout the entire surface of the cancerous cells.

Peptron is developing a MUC1-C specific monoclonal antibody (PAb001) that decreases proliferation of breast cancer cells in vitro and efficiently targets MUC1 in breast tumor in a xenograft mouse model.

Pharmacological and Histopathological Profile

PAb001-OT-MUC1 interaction was well characterized by enzyme-linked immunosorbent assay (EC50 ≥ 0.5 nM), flow cytometry (EC50 = 3 nM) and surface plasmon resonance (KD=0.8 nM).

PAb001-OT-MUC1 complex is internalized into MUC1+ cancer cells.

Highly effective in vivo antitumor activity of Antibody-Drug Conjugates (ADC) is shown in the figure below.


  • MUC1+ triple-negative breast cancer (TNBC)
  • 12 other cancers : non-small cell lung, ovarian, prostate, esophagus, gastric, pancreatic, colorectal, kidney, bladder cancers, acute myeloid leukemia, and multiple myeloma


  • Guang Wu, Sony Maharjan, Dongbum Kim, Jung Nam Kim, Byoung Kwon Park, Heeju Koh, Kyungduk Moon, Younghee Lee, Hyung-Joo Kwon. 2018 "A Novel Monoclonal Antibody Targets Mucin1 and Attenuates Growth in Pancreatic Cancer Model" Int. J. Mol. Sci. 2018, 19 https://doi.org/10.3390/ijms19072004
  • Guang Wu, Dongbum Kim, Jung Nam Kim, Sangkyu Park, Sony Maharjan, Heeju Koh, Kyungduk Moon, Younghee Lee, Hyung-Joo Kwon. 2018 "A Mucin1 C-terminal Subunit-directed Monoclonal Antibody Targets Overexpressed Mucin1 in Breast Cancer" Theranostics 2018; 8(1):78-91. doi:10.7150/thno.21278