Genenta Announces Long-Term Follow-Up Observations in Brain Tumor (GBM) Study with Emerging Survival Signals

Genitourinary Tumor Study Ongoing with Active Screening and Enrollment

MILAN and NEW YORK, Genenta Science (Nasdaq: GNTA), a pioneer in immuno-oncology, today announced that a total of 38 patients were enrolled in newly diagnosed glioblastoma multiforme (TEM-GBM) study, with 25 patients receiving Temferon. Two patients have been enrolled in the TEM-LT long-term follow-up study, surviving three years from the time of 1^st surgery. One of these long-term survivors has not experienced disease progression following Temferon administration and has not required any second-line therapies. The other showed initial signs of disease progression that subsequently stabilized without additional therapeutic intervention. Both these cases suggest possible Temferon-mediated control of disease progression, which warrants further investigation in larger studies.

As of the April data cutoff, the survival rate at two years in the GBM trial for unmethylated MGMT (uMGMT) patients remained consistent at 29% with median overall survival holding steady at 17 months. In historical cohorts, uMGMT patients receiving standard of care have shown a two-year survival rate of approximately 14% and a median overall survival of 13 to 15 months.

In parallel, the TEM-GU Phase 1 study—designed to enroll 12 patients with genitourinary tumors—has begun recruitment. In this trial, Temferon is administered at a fixed dose of 4 million genetically modified cells per kilogram of body weight—a level previously shown to be safe and well tolerated in the TEM-GBM dose-ranging study. Genenta aims to demonstrate the safety and tolerability of Temferon in patients with Metastatic Renal Cell Carcinoma by year-end. The study is designed to evaluate Temferon in combination with immune checkpoint inhibitors or tyrosine kinase inhibitors to assess the potential for immunologic synergy in this patient population. Further clinical updates will be shared once sufficient patient experience has been gained to support meaningful interpretation.

Temferon’s mechanism of action is based on the reprogramming of the tumor microenvironment, which promotes the activation and durability of adaptive immune responses. A scientific manuscript demonstrating Temferon’s potential to enhance and prolong the durability of CAR-T activity in preclinical murine models of solid tumors has been accepted for publication in Science Translational Medicine.

“For the first time, we show that hematopoietic stem cells can be engineered to durably give rise to myeloid cells that localize to the tumor and reprogram its immune environment. In glioblastoma, this strategy induced a pro-inflammatory shift in macrophages and the emergence of tumor-reactive T cells, offering a promising new avenue for immune engagement against one of the most resistant cancers,” said prof. Luigi Naldini, co-founder of Genenta Science.
“We are encouraged by the consistent clinical signals emerging from our glioblastoma trial,” said Pierluigi Paracchi, CEO of Genenta Science. “These findings reinforce our confidence in Temferon’s differentiated mechanism and support our commitment to advancing the platform.”

About Genenta Science
Genenta Science (Nasdaq: GNTA) is a clinical stage immuno-oncology company developing a proprietary hematopoietic stem cells therapy for the treatment of a variety of solid tumor cancers. Genenta’s first in class product candidate is Temferon™, which is designed to allow the expression of immune-therapeutic payloads within the tumor microenvironment by bone marrow derived myeloid cells and enable a durable and targeted response. Genenta has completed the Phase 1 trial for newly diagnosed Glioblastoma Multiforme (GBM) patients with an unmethylated MGMT gene promoter, which suggests the potential reprogramming of the tumor microenvironment and inhibiting of myeloid induced tolerance, while allowing the induction of T cell responses, potentially breaking immune tolerance. Genenta has initiated a Phase 1/2a metastatic Renal Cell Carcinoma study that will also include a combination with immune checkpoint inhibitors. Genenta’s treatments are designed as one-time monotherapies, but with the additional potential, when used in combination, to significantly enhance the efficacy of other approved therapeutics.