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Cancer Chemother Pharmacol. 2016 Sep;78(3):577-84. doi: 10.1007/s00280-016-3108-5. Epub 2016 Jul 23.

A phase 1 dose-escalation study of NEO-102 in patients with refractory colon and pancreatic cancer.

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Division of Hematology/Oncology, University of Texas Southwestern Medical Center, 5323 Harry Hines Boulevard, Dallas, TX, 75390-8852, USA.
Sidney Kimmel Comprehensive Cancer Center, Johns Hopkins University, 401 N. Broadway, Baltimore, MD, 21231, USA.
Division of Hematology/Oncology, University of California San Diego Health System, 3855 Health Sciences Drive, La Jolla, CA, 92093, USA.
Department of Pathology, University of Texas Southwestern Medical Center, 5323 Harry Hines Boulevard, Dallas, TX, 75390-8852, USA.
Precision Biologics, Inc., 9700 Great Seneca Hwy, Suite 321, Rockville, MD, 20850, USA.
Division of Hematology/Oncology, Duke Cancer Institute, Duke University Medical Center, DUMC 384, Durham, NC, 27710, USA.



NEO-102 is a novel chimeric IgG1 monoclonal antibody which recognizes a variant form of MUC5AC expressed specifically by human pancreatic and colorectal tumors. Preclinical models have demonstrated encouraging signs of anti-tumor activity through antibody-dependent cell-mediated cytotoxicity.


This is a phase 1, dose-escalation trial of NEO-102 (Ensituximab) for patients with refractory pancreatic and colorectal cancer. The primary objective was to determine safety and tolerability of escalating doses of NEO-102. Secondary objectives were to assess pharmacokinetics, anti-tumor activity and biologic correlates. Patients whose tumors express NPC-1 antigen were eligible. Dose-escalation was performed in a 3 + 3 design at doses of 1.5, 2, 3 and 4 mg/kg.


A total of 19 patients (4 pancreatic and 15 colon cancer) were enrolled at participating institutions in the treatment phase. Most common treatment-related adverse events included anemia, fatigue, fevers, chills and flushing. There was no detectable hemolysis. Of twelve patients evaluable for disease response, the response rate at week 8 included 5 patients with stable disease and 8 patients with progressive disease (PD). Treatment-related grade 3/4 hyperbilirubinemia and anemia were observed at 4 mg/m2. Reversible hypoxia at 3 mg/kg was a dose-limiting toxicity. The maximum tolerated dose was established at 3 mg/kg. Of 74 patients who underwent tissue screening, positive NPC-1 expression was 47 % in colon and 59 % in pancreatic cancer.


Treatment with the NEO-102, in this first-in-human study, is well tolerated with a manageable safety profile. A maximum tolerated dose of 3 mg/kg has been established. Toxicity profile is typical for this therapeutic class and allows for combination with conventional cytotoxic therapies.


Clinical trial; Colon cancer; Immunotherapy; Monoclonal antibody; Pancreatic cancer

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