FDA Grants Orphan Drug Designation Status to NeoImmuneTech’s Hyleukin-7 for Idiopathic CD4+ Lymphocytopenia Treatment
April 16, 2019 — ROCKVILLE, Md.– (BUSINESS WIRE)–NeoImmuneTech, Inc. (NIT), a T cell-focused therapeutics company, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to Hyleukin-7TM, a T cell amplifier, in development for the treatment of Idiopathic CD4+ Lymphocytopenia (ICL). Hyleukin-7 also received ODD from the European Medicines Agency in 2017, and it is the first and only agent that has obtained ODD for ICL.
ICL was first defined in 1992 by the Centers for Disease Control and Prevention, and is a rare disease in which patients present persistently low CD4+ T lymphocyte counts without human immunodeficiency virus (HIV) infection or any other cause of immunodeficiency.
"Patients with ICL frequently suffer from severe and recurrent opportunistic infections and are at high risk for developing certain types of cancer. Currently, no specific treatment for ICL exists. As such, there is high medical need for therapies that can increase CD4+ T cells in ICL patients," said NgocDiep (Diep) Le, M.D., Ph.D., NIT's Executive Vice President and Chief Medical Officer. "We are delighted that the FDA recognized the potential of Hyleukin-7 as an innovative and transformative treatment for ICL and look forward to conducting a clinical trial in this patient population."
In the phase 1 trial in healthy subjects and multiple ongoing dose-escalation trials in cancer patients, Hyleukin-7 showed a well-tolerated safety profile and dose-dependent increases of CD4+ and CD8+ T lymphocyte counts. NIT has been also actively conducting and planning multiple proof-of-concept clinical trials to develop Hyleukin-7 as an immune-oncology (IO)-enabling drug in combination with other IO therapeutics.
The FDA grants ODD status to medicines intended for the treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the US. Receiving ODD may help to expedite and reduce the cost of development, approval and commercialization of a therapeutic agent.
Hyleukin-7TM (rhIL-7-hyFc, NT-I7), an immuno-oncology agent, is a T cell growth factor composed of a covalently linked homodimer of engineered Interleukin-7 (IL-7) molecule, biologically fused with the proprietary long-acting platform - hyFc™. IL-7 is known to be a critical factor for T cells homeostasis, acting to increase both the number and functionality of T cells. Hyleukin-7 amplifies and reinvigorates persistent T cell immunity in the treatment of patients with cancer and lymphopenia, thus providing unique opportunities for immuno-oncology (IO) combination strategies. Hyleukin-7 is being developed as an "IO enabling" therapy to harness T cell immunity in combination with current cancer treatments such as anti-PD-(L)1 agents or chemo/radiotherapy as well as next generation IO therapeutics. 
1. FDA Grants Orphan Drug Designation Status to NeoImmuneTech’s Hyleukin-7 for Idiopathic CD4+ Lymphocytopenia Treatment. http://neoimmunetech.com/board/list.html?Ncode=b1. Accessed April 22th, 2019.
https://news.bioon.com/article/6737146.html. Accessed April 22, 2019.