Genenta Demonstrated Reprogramming of the Tumor Microenvironment in GBM Patients, Paving the Way for Innovative Treatments of Solid Tumors

Rhea-AI Summary

Genenta Science (NASDAQ: GNTA) announces positive preliminary data from the ongoing dose ranging stage of the Phase 1/2 clinical trial for Temferon in newly diagnosed uMGMT Glioblastoma Multiforme patients. The data indicates no dose-limiting toxicities and a 2-year overall survival rate of 25%, surpassing the historically reported rates. The company expects to report top-line Phase 1 dose-ranging data by the end of 2Q24.

Insights

Oncology Doctor

The preliminary data from Genenta Science's clinical trial in treating uMGMT Glioblastoma Multiforme (GBM) with Temferon™ presents a significant development in the field of immuno-oncology. The reported 2-year Overall Survival (OS) rate of 25% is notably higher than the historical data for patients undergoing the current standard of care, which ranges from 14% to 18%. This suggests a potential improvement in treatment outcomes for a particularly aggressive form of brain cancer.

In analyzing the safety profile, the absence of dose-limiting toxicities in the 22 treated patients is a promising indication that Temferon™ might offer a safer alternative to existing therapies that often have significant side effects. The longevity of Temferon-derived differentiated cells in the peripheral blood is also encouraging, as it implies sustained biological activity and possibly a prolonged therapeutic effect.

However, it is crucial to consider that these results are preliminary and based on a small patient cohort. Large-scale studies and peer-reviewed data will be essential to confirm the efficacy and safety of this treatment before it can be considered a new standard of care for GBM patients.

Medical Research Analyst

Analyzing the impact of Genenta's Phase 1/2 clinical trial from a research perspective, the utilization of hematopoietic stem cells to reprogram the Tumor Microenvironment (TME) and promote immune cell infiltration is a notable advancement. The ability to activate the body's own immune system to fight cancer cells without introducing systemic toxicity could revolutionize the way solid tumors are treated.

Furthermore, the anecdotal evidence of a patient surviving over three years with stable disease while on Temferon™ and second-line treatment adds a human element to the data, which may resonate with stakeholders and patients alike. However, anecdotal results should be interpreted with caution, as they do not substitute for statistically significant findings.

It is also worth noting that RNA single-cell analysis techniques have been employed to validate the mechanism of action of Temferon™, aligning the clinical findings with the preclinical models. This congruence between preclinical and clinical data is an encouraging sign for the potential translatability of the treatment's efficacy.

Financial Analyst

From a financial perspective, the advancement of Genenta Science's Temferon™ into the last cohort of the Phase 1 dose-ranging stage holds potential market implications. The increase in Overall Survival rates for uMGMT GBM patients compared to the standard of care could position Temferon™ as a breakthrough therapy, which may lead to significant market share capture upon successful completion of clinical trials and eventual FDA approval.

The company's expectation to report top-line Phase 1 data by the end of the second quarter of 2024 sets a critical timeline for investors and market analysts. Positive data could lead to stock price appreciation and increased investor interest, while any setbacks could have the opposite effect.

Investors should monitor Genenta's progress closely, as the biotech sector is known for its volatility, especially around key milestones such as clinical trial results. The absence of systemic toxicity, if confirmed in further trials, could be a differentiating factor that enhances the company's value proposition to potential partners or acquirers.

Based on Preliminary Data from Ongoing Dose Ranging Stage of the Phase 1/2 Clinical Trial

MILAN, Italy and NEW YORK, Feb. 08, 2024 (GLOBE NEWSWIRE) -- Genenta Science (NASDAQ: GNTA), a clinical-stage immuno-oncology (I/O) company developing a cell-based platform harnessing the power of hematopoietic stem cells to provide durable and safe treatments for solid tumors, today announced that:

We have successfully dosed the first of three patients in Cohort 8 (Temferon™ at 4x10^6/kg), the last cohort of the Phase 1 dose-ranging part of the Phase 1/2 clinical trial in newly diagnosed uMGMT Glioblastoma Multiforme (TEM-GBM) patients. The second patient has been enrolled and the treatment is planned.

• Thus far, the preliminary data indicate no dose-limiting toxicities related to Temferon have been detected in any of 22 treated patients.
• Temferon-derived differentiated cells were evident within the peripheral blood 14 days after infusion and were still detectable at more than 24 months.
• As of December 2023, preliminary data in uMGMT patients, the most aggressive form of GBM, show a 2-year Overall Survival (OS) of 25%; the historically reported data observed in uMGMT and methilated patients undergoing current standard of care is approximately 14% to 18%, respectively.
• We expect reporting top line Phase 1 dose-ranging data by the end of 2Q24.

Dr. Luigi Naldini, co-founder of Genenta and Director at the San Raffaele - Telethon Institute for Gene Therapy, stated, “Several techniques, including RNA single-cell analysis, suggest that Temferon can reprogram the Tumor Microenvironment (TME) in patients similarly to what we had demonstrated in preclinical models and activate the immune cell infiltration towards mounting immune responses against the tumor.”

“We had a patient who, albeit anecdotal, survived for over three years with Temferon and second line treatment, with a stable tumor during that period,” reported Carlo Russo, CMO and Head of Development. “We believe our preliminary data generated so far, including the duration of the cell presence in the patients, combined with our pre-clinical data showing the agnostic efficacy, open up intriguing possibilities for the use of Temferon in a variety of solid tumors.”

“The absence of limiting toxicity observed so far suggests the ability of our technology to prevent systemic toxicity in humans commonly associated with powerful anti-tumor proteins,” noted Pierluigi Paracchi, CEO of Genenta.

About Genenta and Temferon
Genenta (www.genenta.com) is a clinical-stage biotechnology company engaged in the development of a proprietary hematopoietic stem cell therapy for the treatment of a variety of solid tumor cancers. Temferon™ is based on ex-vivo gene transfer into autologous Tie2+ hematopoietic stem/progenitor cells (HSPCs) to deliver immunomodulatory molecules directly via tumor-infiltrating monocytes/macrophages (Tie2 Expressing Monocytes - TEMs). Temferon, which is under investigation in a phase 1/2a clinical trial in newly diagnosed Glioblastoma Multiforme patients who have an unmethylated MGMT gene promoter (uMGMT-GBM), is designed to reach solid tumors, induce a durable immune response not restricted to pre-selected tumor antigens nor type, and avoid systemic toxicity, which are some of the main unresolved challenges in immuno-oncology.

Forward-Looking Statements
Statements in this press release contain “forward-looking statements,” within the meaning of the U.S. Private Securities Litigation Reform Act of 1995, that are subject to substantial risks and uncertainties. All statements, other than statements of historical fact, contained in this press release are forward-looking statements. Forward-looking statements contained in this press release may be identified by the use of words such as “anticipate,” “believe,” “contemplate,” “could,” “estimate,” “expect,” “intend,” “seek,” “may,” “might,” “plan,” “potential,” “predict,” “project,” “suggest,” “target,” “aim,” “should,” "will,” “would,” or the negative of these words or other similar expressions, although not all forward-looking statements contain these words. Forward-looking statements are based on Genenta’s current expectations and are subject to inherent uncertainties, risks and assumptions that are difficult to predict, including risks related to the completion and timing of the phase 1/2a clinical trial or any studies relating to the treatment of glioblastoma multiforme patients who have an unmethylated MGMT gene promoter (uMGMT-GBM). Further, certain forward-looking statements are based on assumptions as to future events that may not prove to be accurate. These and other risks and uncertainties are described more fully in the section titled “Risk Factors” in Genenta's Annual Report on Form 20-F for the year ended December 31, 2022 filed with the Securities and Exchange Commission. Forward-looking statements contained in this announcement are made as of the date of this announcement, and Genenta undertakes no duty to update such information except as required under applicable law.

Genenta Media
Tiziana Pollio, mobile +39 348 23 15 143
tiziana.pollio@genenta.com

FAQ

What is the ticker symbol for Genenta Science?

The ticker symbol for Genenta Science is GNTA.

What is the Phase of the clinical trial mentioned in the press release?

The press release mentions the Phase 1/2 clinical trial for Temferon in newly diagnosed uMGMT Glioblastoma Multiforme patients.

What is the 2-year Overall Survival rate reported in the preliminary data?

The preliminary data reported a 2-year Overall Survival rate of 25% in uMGMT Glioblastoma Multiforme patients.

Who is the co-founder of Genenta and Director at the San Raffaele - Telethon Institute for Gene Therapy mentioned in the press release?

Dr. Luigi Naldini is mentioned as the co-founder of Genenta and Director at the San Raffaele - Telethon Institute for Gene Therapy.

What do the preliminary data suggest about the ability of Temferon in preventing systemic toxicity in humans?

The preliminary data suggest that Temferon has the ability to prevent systemic toxicity in humans commonly associated with powerful anti-tumor proteins.