Research Papers:

A phase II study of axalimogene filolisbac for patients with previously treated, unresectable, persistent/recurrent loco-regional or metastatic anal cancer

Cathy Eng _, Marwan Fakih, Manik Amin, Van Morris, Howard S. Hochster, Patrick M. Boland and Hope Uronis

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Oncotarget. 2020; 11:1334-1343. https://doi.org/10.18632/oncotarget.27536

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Cathy Eng1, Marwan Fakih2, Manik Amin3, Van Morris1, Howard S. Hochster4, Patrick M. Boland5 and Hope Uronis6

1 MD Anderson Cancer Center, Houston, TX, USA

2 City of Hope, Duarte, CA, USA

3 Siteman Cancer Center, Washington University School of Medicine, St. Louis, MO, USA

4 Rutgers Cancer Institute, New Brunswick, NJ, USA

5 Roswell Park Cancer Institute, Buffalo, NY, USA

6 Duke Cancer Institute, Durham, NC, USA

Correspondence to:

Cathy Eng,email: [email protected]

Keywords: anal neoplasms; immunotherapy; Listeria monocytogenes; papillomaviridae; phase II clinical trial

Received: November 08, 2019     Accepted: March 14, 2020     Published: April 14, 2020


Squamous cell carcinoma of the anorectal canal (SCCA) is a rare HPV-related malignancy that is steadily increasing in incidence. A high unmet need exists for patients with persistent loco-regional and metastatic disease. Axalimogene filolisbac (ADXS11-001) is an investigational immunotherapy that stimulates tumor-specific responses against HPV-associated cancers, and has demonstrated benefit in metastatic cervical cancer. We conducted this single-arm, multicenter, phase 2 trial in patients with persistent/recurrent, loco-regional or metastatic SCCA. Patients received ADXS11-001, 1 × 109 colony-forming units intravenously every 3 weeks. A Simon 2-stage design was used to test primary co-endpoints of overall response rate (ORR) and 6-month progression-free survival (PFS) rate. Study would proceed to full enrollment if ORR ≥ 10% or 6-month PFS rate ≥ 20%. Thirty-six patients were treated; 29 patients were evaluable for response. One patient had a prolonged partial response (3.4% ORR). The 6-month PFS rate was 15.5%. Grade 3 adverse event were noted in 10 patients, with the majority being cytokine-release symptoms; one grade 4 adverse event was noted. No grade 5 adverse events occurred. ADXS11-001 was safe and well-tolerated in patients with SCCA. However, this study did not meet either primary endpoint. ADXS11-001 may be more beneficial when administered in combination with other cytotoxic or targeted agents.

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