IGV-001 Offers Hope for Prolonged OS in Newly Diagnosed Glioblastoma

IGV-001, an investigational autologous cell immunotherapeutic combination product, may offer clinically meaningful overall survival (OS) benefits for patients with newly diagnosed glioblastoma (GBM), according to topline data from a randomized phase 2b trial (NCT04485949).¹

The objective of the trial was to assess the safety and efficacy of IGV-001 in this patient population.^2,3 While the trial did not meet statistical significance for its primary end point of progression-free survival, these latest findings show a notable improvement in its key secondary end point of OS after a 22-month follow-up period. Specifically, those treated with IGV-001 and standard-of-care radiation therapy and temozolomide (Temodar) following surgical resection had a median OS of 20.3 months compared with 14.0 months among those receiving placebo and standard of care, extending survival by 6.3 months.

Additionally, the therapy demonstrated a promising safety profile consistent with that observed in its phase 1b pilot study (NCT02507583), with no drug-related serious adverse events observed in the treatment arm. In the phase 1b study (n = 33), IGV-001 was well-tolerated by patients.⁴

“The data from this trial are highly encouraging and suggest both a clinically meaningful improvement in [OS] for [patients with newly diagnosed] GBM and a benign safety profile for the therapy,” said J. Bradley Elder, MD, Department of Neurological Surgery at The Ohio State University Wexner Medical Center, in a news release.¹ “These results represent a potential watershed moment for the treatment of this deadly disease.”

About IGV-001: Mechanisms and Future Development

Standard of care for newly diagnosed GBM consists of surgical resection as first-line treatment, concurrent radiotherapy and temozolomide, and temozolomide maintenance therapy. Despite these established care standards, GBM remains one of the most challenging malignancies to treat, with a dismal prognosis of only 14 months.⁵

IGV-001 is a first-in-class biologic-device product developed to meet this need by combining personalized tumor-derived cells with antisense oligonucleotide targeting insulin-like growth factor 1 receptor (IGF-1R), which is overexpressed in malignant cells.⁴ The product utilizes the proprietary Goldspire^® platform by Imvax, sponsor of the IGV-001 trials.

“What they're inserting [in] the cells is called antisense oligonucleotide. [This is] a chemical that's very similar to RNA. So, it's blocking a particular type of signaling mechanism called [IGF-1R] … and the idea is that by blocking this, you could stimulate the immune system to go after [tumor cells],” explained Deric Park, MD, neuro-oncologist at Hackensack University Medical Center, in a 2024 interview with Targeted Oncology.

A preclinical study confirmed the therapeutic potential of the product, finding that it induced anticancer immune responses in animal models.⁶

These positive phase 2 results reinforce IGV-001’s 2 previous FDA designations, including fast track and orphan drug designations, in recognition of its therapeutic promise in GBM.¹ Looking ahead, Imvax has announced they intend to meet with the FDA in the coming months to discuss the regulatory pathway for IGV-001. Such meetings will be critical to defining the trajectory of this therapeutic candidate and potentially the future of GBM treatment.

REFERENCES

1. Imvax announces positive top-line data from phase 2b clinical trial of IGV-001 in newly diagnosed GBM. News release. Imvax. December 2, 2025. Accessed December 2, 2025. https://tinyurl.com/ke2rz58h

2. A phase 2b clinical study with a combination immunotherapy in newly diagnosed patients with glioblastoma. ClinicalTrials.gov. Updated September 11, 2025. Accessed December 2, 2025. https://clinicaltrials.gov/study/NCT04485949

3. Lee IY, Hanft S, Schulder M, et al. Autologous cell immunotherapy (IGV-001) with IGF-1R antisense oligonucleotide in newly diagnosed glioblastoma patients. Future Oncol. 2024;20(10):579-591. doi:10.2217/fon-2023-0702

4. Andrews DW, Judy KD, Scott CB, et al. Phase Ib clinical trial of IGV-001 for patients with newly diagnosed glioblastoma. Clin Cancer Res. 2021;27(7):1912-1922. doi:10.1158/1078-0432.CCR-20-3805

5. Mohammed S, Dinesan M, Ajayakumar T. Survival and quality of life analysis in glioblastoma multiforme with adjuvant chemoradiotherapy: a retrospective study. Rep Pract Oncol Radiother. 2022;27(6):1026-1036. Published 2022 Dec 29. doi:10.5603/RPOR.a2022.0113

6. Cultrara C, Uhl C, Kirby K, et al. A biologic-device combination product delivering tumor-derived antigens elicits immunogenic cell death-associated immune responses against glioblastoma. J Immunother Cancer. 2023;11(8):e006880. doi:10.1136/jitc-2023-006880