On December 2, 2025 Imvax, Inc., a clinical-stage biotechnology company developing personalized, whole tumor-derived immunotherapies, reported top line results from its randomized, multicenter, double-blind, placebo-controlled Phase 2b clinical trial of IGV-001 in 99 patients with newly diagnosed glioblastoma (ndGBM). Glioblastoma is a highly aggressive brain cancer with an average life expectancy of just 12 to 15 months, and a five-year survival rate of under six percent. It has been 20 years since the last improvement to the standard of care for the treatment of ndGBM.
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In the trial, patients in the IGV-001 arm had a median overall survival (mOS) of 20.3 months, a difference of 6.3 months, or 45%, compared to a mOS of 14.0 months in the placebo arm. The median follow-up time for all patients in the study was 22 months. There were no drug-related serious adverse events in the treatment arm, and the safety profile seen in the Phase 2b trial is favorable, consistent with that observed in a previous Phase 1b study (n=33). To date, approximately 100 ndGBM patients have received treatment with IGV-001 across two clinical studies. In the study, patients in the IGV-001 arm saw measurable patient benefit across multiple metrics compared to the placebo arm. The Company has informed the U.S. Food and Drug Administration (FDA) that it intends to submit a meeting request to discuss the regulatory pathway for IGV-001.
"The data from this trial are highly encouraging and suggest both a clinically meaningful improvement in overall survival for ndGBM patients and a benign safety profile for the therapy," said J. Bradley Elder, M.D., Director, Neurosurgical Oncology, Professor, Department of Neurological Surgery at The Ohio State University Wexner Medical Center and the highest enrolling investigator in the Phase 2b trial. "These results represent a potential watershed moment for the treatment of this deadly disease."
"While treatments for many cancers have come a long way, treatments for glioblastoma have not changed much over the years. It is a heartbreaking diagnosis made even harder by how few treatment options there are," said Kelly Sitkin, President and Chief Executive Officer of the American Brain Tumor Association. "Ultimately, what any patient or family member wants is a chance at more time with loved ones, and new treatments for glioblastoma provide that hope for our community."
"Today marks a pivotal moment for both Imvax and for the people affected by ndGBM. For the past decade, the Imvax team has been dedicated to advancing the development of IGV-001, and the results from this Phase 2b study bring us meaningfully closer to achieving that goal. Thanks to the strong support of our investors, Imvax has the resources and expertise to execute on a clear strategy for IGV-001," said John P. Furey, Executive Chair of the Imvax Board of Directors. "We are preparing to meet with FDA to discuss the regulatory pathway for IGV-001 and what we believe is a strongly positive risk-benefit profile, especially given the large unmet medical need in ndGBM. Finally, we are profoundly grateful to the investigators, patients, and their families for their commitment to this study."
About IGV-001 and Glioblastoma
IGV-001 is an autologous biologic-device combination product candidate derived from Imvax’s proprietary Goldspire immuno-oncology platform for solid tumors, which involves a unique approach to inducing a patient-specific, broad, and durable immune response against tumors. The U.S. Food and Drug Administration (FDA) has granted Fast Track designation and Orphan Drug Designation to IGV-001 for the treatment of ndGBM. IGV-001 is an investigational therapy and has not been approved by the FDA or any regulatory body.
Glioblastoma is both the most common and most aggressive malignant brain cancer and has resisted significant advances in treatment for decades. Approximately 14,000 people are diagnosed with glioblastoma each year in the United States, and their average life expectancy is 12 to 15 months with the current standard of care. Under six percent of patients survive for five years after diagnosis. The last significant advance in the treatment of ndGBM was the Stupp trial in 2005, which demonstrated a 2.5-month improvement in median overall survival. More than 20 years later, the Stupp protocol – maximal safe resection followed by adjuvant radiotherapy with concurrent temozolomide and subsequent maintenance temozolomide – remains the current standard of care for ndGBM patients.
About the Phase 2b Trial
The Phase 2b clinical trial is a randomized, multicenter, double-blind, placebo-controlled study (NCT04485949) designed to assess the safety and efficacy of IGV-001, an autologous biologic-device combination product, in ndGBM patients. The trial assessed several endpoints, including progression-free survival (PFS, the primary endpoint), overall survival and safety. The trial did not reach statistical significance on PFS but demonstrated a 6.3 month increase in median overall survival and a favorable safety profile.
The trial enrolled 99 participants in a 2:1 randomization across 19 sites in the United States. Approximately 48 hours after surgical resection of the patient’s malignant tumor, participants in the IGV-001 arm were implanted with biodiffusion chambers containing a combination of personalized whole tumor-derived cells with an antisense oligonucleotide (IMV-001); in the placebo arm, the chambers contained an inactive solution only. In both arms, the biodiffusion chambers were explanted approximately 48 hours later, and after six weeks all patients were treated with standard of care (adjuvant concomitant radiotherapy and temozolomide followed by maintenance temozolomide).
(Press release, Imvax, DEC 2, 2025, View Source;utm_medium=rss&utm_campaign=imvax-announces-positive-top-line-data-from-phase-2b-clinical-trial-of-igv-001-in-newly-diagnosed-gbm [SID1234661048])