NeoImmuneTech has received orphan drug designation (ODD) from the US Food and Drug Administration (FDA) for its investigational drug, NT-I7 (efineptakin alfa) (rhIL-7-hyFc), to treat Acute Radiation Syndrome (ARS), an acute illness.
The clinical-stage long-acting human IL-7, NT-I7 is being developed in oncologic and immunologic indications.
In these indications, T cell amplification and increased functionality are expected to provide clinical benefit.
The therapy exhibits favourable PK/PD and safety profiles and is being studied in multiple clinical trials in solid tumours and as a vaccine adjuvant.
The company is planning studies for testing the therapy in additional solid tumours, haematologic malignancies, and other immunology-focused indications.
NeoImmuneTech president and CEO Dr Se Hwan Yang said: “Obtaining the orphan drug designation from the FDA is an important acknowledgment of the immense potential that NT-I7 holds in providing a beacon of hope for the treatment of ARS.”
ARS is caused by irradiation of the entire body by a high dose of penetrating radiation in a short period of time and leads to severe damage to the bone marrow and the immune system.
In the clinical setting, NT-I7 has demonstrated positive results by boosting T cell counts consistently, while maintaining a good level of tolerance and safety.
The ODD is aimed at advancing treatments for rare diseases, including ARS, which has limited treatment options at present.