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817 AST-201 (pUMVC3-hIGFBP2 N-terminus) demonstrates anti-tumor effect in an ovarian cancer mouse model
  1. Jinback Lim1,
  2. Hyo-Hyun Park1,
  3. Jin Kyeong Choi2,
  4. Jee Hyun Choi1,
  5. JinHo Kang1,
  6. Seong-Yong Jang3,
  7. Min-Ah Kim3,
  8. Myeong-Kyu Park3,
  9. Mary L Disis4,
  10. Eunkyo Joung1 and
  11. Hun Jung1
  1. 1Aston Sci., Inc., Seoul, Republic of Korea
  2. 2Jeonbuk National University Medical School, Jeonju, Republic of Korea
  3. 3Korea Testing and Research Institute, Hwasun, Republic of Korea
  4. 4University of Washington, Seattle, WA, USA
  • Journal for ImmunoTherapy of Cancer (JITC) preprint. The copyright holder for this preprint are the authors/funders, who have granted JITC permission to display the preprint. All rights reserved. No reuse allowed without permission.


Background IGFBP2, known to enhance the invasion capacity of ovarian cancer cells,1 has been suggested that its inhibition could be potentially a treatment strategy of ovarian cancer. AST-201 (pUMVC3-hIGFBP2) is a therapeutic cancer vaccine using a plasmid DNA encoding IGFBP2 N-terminus. In a phase 1 study (NCT01322802) completed, 100 μg AST-201 (intradermal immunization, id) showed not only a significant efficacy by inducing the Th1-cell immunity against IGFBP2 but also safety and tolerability profiles in ovarian cancer patients. The primary objective of this study is to evaluate whether the administration of AST-201 alone and the combination with pembrolizumab could show anti-tumor efficacy and/or synergic effect in the ID8-Luc2 ovarian cancer mouse model. Also, immunological responses were observed as explorative endpoint

Methods AST-201 (100 μg/animal, id, mixed with mGM-CSF as an immune adjuvant) was immunized to mice (C57BL/6) once a week for a total of 3 times on different days, either alongside pembrolizumab (10 mg/kg, intraperitoneal injection) twice a week for a total of 3 times on different days. Also, AST-201 was immunized was a mono treatment. The efficacy was evaluated by a tumor growth inhibition (TGI) rate at the last day, and immune cell profiling via FACS analysis was conducted with splenocytes and tumor tissues collected at 8 weeks after the first injection.

Results As a primary endpoint, a TGI rate at Day 55 of AST-201 mono treatment was 67%, comparing to a control group (p<0.05). The anti-tumor effect of AST-201 combining with pembrolizumabas better than standard dose pembrolizumab, based on a TGI rate at Day 55 (78% vs 66%, not significant). Immune profiling showed that AST-201 and pembrolizumab combination regimen could inhibit a tumor growth by transforming TME into inflamed-type (‘hot’) form the low immunoreactivity, which was supported by increased CD4+TEM, and CD8+TEM and T helper cells in splenocyte and TIL analysis.

Conclusions A study demonstrated that AST-201 (IGFBP2 cancer vaccine) showed an anti-tumor effect as mono treatment and would be potentially leading to a synergistic effect with a combination regimen of pembrolizumab. Phase 2 randomized-controlled study of AST-201 in ovarian cancer will be initiated under the MRCT strategy (CornerStone-004 study, NCT05794659).


  1. Eun-Ju Lee, Cristian Mircean, Ilya Shmulevich, Huamin Wang, Jinsong Liu, Antti Niemistö, John J Kavanagh, Je-Ho Lee, Wei Zhang. Mol Cancer. 2005 Feb 2;4(1):7. doi: 10.1186/1476-4598-4-7.

Ethics Approval Approval number of Institutional Animal Care and Use Committee : IAC2022–3007

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