FDA Fast Tracks Avenge Bio’s Ovarian Cancer Cell Therapy AVB-001


AVB-001 is currently being evaluated in a phase 1/2 clinical trial (NCT05538624) that was initiated in January 2023.

Avenge Bio has received fast track designation from the FDA for AVB-001, an investigational allogeneic encapsulated cell product, for the treatment of platinum-resistant, high grade serous adenocarcinoma of the ovary, primary peritoneum, or fallopian tube.1

AVB-001 is currently being evaluated in a phase 1/2 clinical trial (NCT05538624) that was initiated in January 2023 following clearance of an investigational new drug (IND) application by the FDA in August 2022.1,2 The company noted that the study, which is currently in its dose-escalation portion, is anticipated to advance to the dose-expansion portion within the first half of 2024. The first-in-human, multicenter, open-label clinical trial is seeking to enroll an estimated 44 participants in total, with recruitment taking place at locations in Maryland, Massachusetts, Rhode Island, and Texas.

"We are extremely pleased to receive the FDA Fast Track designation for AVB-001 based on FDA’s review of our preclinical and emerging clinical data,” Michael Heffernan, the chief executive officer of Avenge Bio, said in a statement.1 “The Fast Track designation has been provided for platinum-resistant, refractory ovarian cancer, and acknowledges the potential for AVB-001 to treat this significant unmet medical need.”

AVB-001, which is administered intraperitoneally, is based on the company’s LOCOcyte platform and is intended to generate native human interleukin-2 (hIL-2) on a continuous basis in order to produce an immune response. The local administration approach is expected to circumvent toxic effects seen with immunotherapies that are given systemically. Avenge Bio is also working to apply the LOCOcyte platform in the treatment of other solid tumor types.

“Our first cancer target is ovarian cancer which is a prevalent, deadly disease with not a lot of innovation over the past 20 years,” Heffernan said in an interview with CGTLive held shortly after the IND clearance last year. “Patients who don't do well on first-line platinum therapy or PARP inhibitors don't have a lot of additional options other than clinical trials. So, we see this as an option, at least early on, for a later-stage patient who doesn't have a lot of other treatment options where we can attempt to use the patient's immune system to eradicate the cancer.”

In June 2023, the FDA granted orphan drug designation to AVB-001 for one of the other indications Avenge Bio is developing it for, mesothelioma.3 Earlier in the year, in March, the company reported that it had received positive feedback from the FDA in a pre-IND meeting regarding its plans to clinically develop the cell therapy for the treatment of pleural malignant mesothelioma.4 At the time, the company stated it was on track to submit an IND for this indication before the end of 2023.

Preclinical research on AVB-001's potential to treat mesothelioma was previously published in Clinical Cancer Research in December 2022.5 The study looked at effects of “IL2 cytokine factories”, consisting of engineered polymer encapsulated human retinal pigmented epithelial cells, that were implanted into a malignant mesothelioma mouse model.

“IL2 cytokine factories led to the eradication of aggressive mouse malignant mesothelioma tumors and protection from tumor recurrence, and increased the therapeutic efficacy of a PD1 checkpoint therapy...” first author Amanda M. Nash of the Department of Bioengineering at Rice University, and colleagues wrote.5 “Although previous studies have demonstrated that IL2 is highly efficacious, our cytokine factory is the first approach that requires minimal access to the target site. This advantage overcomes current clinical drawbacks regarding catheter infection and may alleviate overall adverse events. The continued occurrence of malignant mesothelioma necessitates the clinical assessment of new, effective treatments. Thus, the potential of the IL2 cytokine factory described here to ease the global burden of malignant mesothelioma highlights the urgency of its evaluation in clinical trials.”

1. Avenge Bio receives FDA fast track designation for AVB-001, a novel cell therapy leveraging the LOCOcyte™ immunotherapy platform. News release. Avenge Bio, Inc. October 2, 2023. Accessed October 9, 2023. https://avengebio.com/wp-content/uploads/2023/09/Avenge-Bio-Receives-FDA-Fast-Track-Designation-for-AVB-001-a-Novel-Cell-Therapy-Leveraging-the-LOCOcyte%E2%84%A2-Immunotherapy-Platform.pdf
2. Avenge Bio announces FDA clearance of the AVB-001 IND for the treatment of ovarian cancer, a novel cellular therapy leveraging the LOCOcyte™ immunotherapy platform. News release. Avenge Bio. August 3, 2022. https://www.prnewswire.com/news-releases/avenge-bio-announces-fda-clearance-of-the-avb-001-ind-for-the-treatment-of-ovarian-cancer-a-novel-cellular-therapy-leveraging-the-lococyte-immunotherapy-platform-301598446.html
3. Avenge Bio receives FDA orphan drug designation for AVB-001 for the treatment of mesothelioma. News release. Avenge Bio, Inc. June 15, 2023. Accessed October 9, 2023.https://avengebio.com/wp-content/uploads/2023/06/Avenge-Bio-Receives-FDA-Orphan-Drug-Designation-for-AVB-001-for-the-Treatment-of-Mesothelioma.pdf
4. Avenge Bio receives positive feedback from pre-INDFDA meeting on development path for AVB-001 mesothelioma program and provides pipeline update. News release. Avenge Bio, Inc. March 9, 2023. Accessed October 9, 2023. https://avengebio.com/wp-content/uploads/2023/03/Avenge-Bio-Receives-Positive-Feedback-from-Pre-IND-FDA-Meeting-on-Development-Path-for-AVB-001-Mesothelioma-Program-and-Provides-Pipeline-Update.pdf
5. Nash AM, Aghlara-Fotovat S, Castillio B, et al. Activation of adaptive and innate immune cells via localized il2 cytokine factories eradicates mesothelioma tumors. Clinical Cancer Research. 2022;28(23):5121-5135.
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