EMA Awards Orphan Drug Status to NeoImmuneTech's NT-I7 for Acute Radiation Syndrome

NeoImmuneTech, Inc. (NIT), a biopharmaceutical company specializing in T cell-focused therapies, has achieved a significant regulatory milestone with the European Medicines Agency (EMA) granting Orphan Drug Designation (ODD) to NT-I7 (efineptakin alfa) for Acute Radiation Syndrome (ARS). This follows a similar designation by the U.S. FDA in November 2023.

Acute Radiation Syndrome is a severe condition caused by high doses of radiation exposure, leading to critical damage to the bone marrow and immune system, often resulting in fatality. Currently, there are no effective treatments available to restore T cell function post-exposure. NT-I7, a novel long-acting human interleukin-7 (IL-7), has shown potential in nonclinical studies to counteract the immunosuppressive effects of ARS by accelerating T cell recovery and enhancing immune response.

NeoImmuneTech is advancing NT-I7 development in collaboration with the National Institute of Allergy and Infectious Diseases (NIAID) and Duke University. Duke University recently secured a $6 million grant from NIAID to further this research. The company is optimistic about NT-I7's potential, with President and CEO Dr. Luke Oh emphasizing the importance of the EMA's ODD in accelerating their development program.

Orphan Drug Designation is awarded to medicines aimed at treating rare diseases, providing several development incentives such as reduced fees and protocol assistance. This is the second ODD granted by the EMA for NT-I7, the first being for Idiopathic CD4+ Lymphocytopenia (ICL) in May 2017.

ARS is an acute illness caused by whole-body irradiation from high doses of penetrating radiation within a short period. Historical instances include the Hiroshima and Nagasaki atomic bombings and the Chernobyl disaster. The condition severely damages bone marrow and the immune system, with no currently effective treatments to promote T cell recovery.

NT-I7, also known as efineptakin alfa, is the only clinical-stage long-acting IL-7 designed to enhance T cell functionality. It plays a crucial role in the development of naïve and memory T cells, sustaining immune responses to chronic and foreign antigens. NT-I7 has demonstrated favorable pharmacokinetic and pharmacodynamic profiles along with safety, making it suitable for combination with other therapies. It is currently under investigation in multiple clinical trials for solid tumors and as a vaccine adjuvant, with future studies planned for hematologic malignancies and other immunology-related conditions.

NeoImmuneTech, led by the scientific founder of NT-I7, is rapidly expanding and collaborating with academic and industry leaders to develop NT-I7 both as a standalone treatment and in combination with other immunotherapies. The company aims to broaden the scope of immuno-oncology and enhance immune responses to infectious diseases.