The European Commission Grants Orphan Drug Designation to Temferon for Treatment of Glioma

MILAN, Italy and NEW YORK, June 29, 2023 (GLOBE NEWSWIRE) -- Genenta Science (NASDAQ: GNTA), a clinical-stage immuno-oncology 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 the European Commission has granted Orphan Drug Designation (ODD) to Temferon for the treatment of glioma. Glioblastoma Multiforme (GBM) is the first clinical indication of Temferon.

ODD is granted by the European Commission for medicines in development to treat rare conditions affecting no more than five in 10,000 people in the European Union (EU), provided there is no other satisfactory treatment option or the medicine can be of significant benefit to those affected by the condition. Sponsors of with ODD designation can benefit from a number of incentives in the EU; for example, ODD medicines benefit from ten years of market exclusivity once they receive a marketing authorization in the EU.

“European Medicine Agency’s Committee reviewed Genenta's ODD application for Temferon and agreed on the potential significant benefit that Temferon could contribute to patients suffering from GBM if approved. The ODD designation supports and facilitates the development of our cell therapy-based technology platform for solid tumors,” said Pierluigi Paracchi, Chief Executive Officer at Genenta. “The EMA ODD designation follows the orphan drug designation granted by the US Food and Drug Administration to Temferon for the treatment of GBM in March 2023. The preliminary interim results of Genenta’s ongoing phase 1/2a trials in newly diagnosed patients with unmethylated MGMT gene promoter reviewed by EMA included an Overall Survival at two years, which is longer than the median Overall Survival described in published reports,” continued Pierluigi Paracchi.

GBM is the most common malignant primary brain tumor and the most aggressive diffuse glioma, with unmethylated MGMT promoter status identified in approximately 60% of the GBM population.

Temferon is Genenta's product at the most advanced stage of development and consists of the patient's own stem progenitor cells modified with Genenta's platform to express interferon alpha (IFNa) within solid tumors. IFNa is a well-known immunomodulatory protein that has been used in the clinic for decades for the treatment of a variety of cancers, but with limited current use because of the systemic toxicity. Genenta's platform is designed to avoid systemic toxicity and selectively deliver therapeutic activity within the solid tumor. From pre-clinical experiments, it has been observed that Temferon breaks tumor-induced tolerance, thus allowing the immune system to recognize the tumor and mount a durable immune response.