Temferon Yields Preliminary Survival in Newly Diagnosed Glioblastoma Trial

Early findings related to the tumor microenvironment showed bone marrow–derived myeloid cells can reach the tumor site and administer immunotherapeutic payloads in situ that is consistent with temferon’s intended mechanism.

Treatment with temferon produced consistent survival outcomes in a small cohort of patients with newly diagnosed glioblastoma multiforme and unmethylated MGMT gene promoter status, according to a press release on updated findings from the phase 1/2a TEM-GBM study (NCT03866109).1

The 18-month survival rate was 44% among 25 evaluable patients, exceeding the rate of 38% based on the last report in April 2025. Additionally, data showed a 2-year survival rate of 29% and a median overall survival (OS) of 17 months, which appeared consistent with prior reports in the trial. Developers noted a historical 2-year survival rate of 14% and a median OS of 13 to 15 months for patients with unmethylated MGMT disease following treatment with standard of care.

Investigators observed 1 patient who achieved 3 years of survival at 39 months following initial surgery; this patient did not undergo treatment with any subsequent interventions or agents. Furthermore, this patient is the second in the study who did not need additional therapeutic intervention during follow-up.

Early findings related to the tumor microenvironment showed bone marrow–derived myeloid cells can reach the tumor site and administer immunotherapeutic payloads in situ that is consistent with temferon’s intended mechanism. According to the developers, these observations may inform additional development of temferon as part of combinatorial therapeutic strategies.

The developers noted that these trial findings were preliminary in nature and were gathered directly from clinical sites. Quality control review or independent verification of the data via clinical research organization has not yet been completed.

Investigators of the non-randomized, open-label, multicenter phase 1/2a TEM-GBM trial aimed to assess treatment with a single injection of temferon, a novel advanced therapy medicinal product including autologous CD34-positive–enriched hematopoietic stem and progenitor cells exposed to transduction via third-generation lentiviral vector-related myeloid-specific interferon alpha-2 expression.2 Part A of the trial was designed to evaluate the tolerability and safety of temferon at 5 escalating doses in combination with 3 different conditioning regimens across a maximum of 27 patients.

The trial’s primary end point was the tolerability and safety of temferon in the first 90 days following its administration based on the incidence of adverse effects (AEs). Secondary end points included the long-term tolerability and safety of the agent based on AEs, the maximum tolerated dose, clinical response per Immunotherapy Response Assessment in Neuro-Oncology (iRANO) guidelines, progression-free survival, OS, changes in ECOG performance status, and quality of life.

Patients 18 to 70 years old with histologically confirmed, newly diagnosed supratentorial glioblastoma with unmethylated MGMT gene promoter status and complete or partial tumor resection were eligible for enrollment on the study. Other eligibility criteria included having a life expectancy of at least 6 months at screening, a Karnofsky performance status of 70 or higher, and suitability to receive radiotherapy. Having adequate cardiac, renal, hepatic, and pulmonary functions were additional requirements for enrollment.

Those with receipt of other investigational agents or procedures within 4 weeks of study entry; treatment with any oral or parenteral chemotherapy or immunotherapy within 2 years of screening; previous allogenic bone marrow transplantation, kidney, or liver transplant; or clinical evidence of persistently raised intracranial pressure after surgical resection were ineligible to enroll on the trial. Patients were also unable to enroll if they had a history of sarcoidosis; a history of severe cardiovascular conditions including prior stroke, coronary artery disease, or unresolved arrhythmias in the last 6 months; evidence of any hematological neoplasm; or known bleeding diathesis or history of abnormal bleeding.

References

1. Genenta reports long-term survival trends and immune findings in brain tumor (GBM) trial. News release. Genenta Science. November 24, 2025. Accessed November 25, 2025. https://tinyurl.com/yj74tf5
2. A study evaluating temferon in patients with glioblastoma & unmethylated MGMT (TEM-GBM). ClinicalTrials.gov. Updated January 6, 2025. Accessed November 25, 2025. https://tinyurl.com/5cdxuh8k