NeoImmuneTech, a clinical-stage T cell-focused biopharmaceutical company, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to NeoImmuneTech’s NT-I7 (efineptakin alfa), the only clinical-stage long-acting human IL-7, for the treatment of Progressive Multifocal Leukoencephalopathy (PML).

PML is a rare, aggressive, opportunistic brain infection caused by the reactivation of John Cunningham virus (JCV) in immunocompromised individuals.

“The greatest barrier between PML patients and recovery is a weakened immune system,” said NgocDiep Le, M.D., Ph.D., Executive Vice President and Chief Medical Officer of NeoImmuneTech. “NT-I7 has demonstrated its ability to reverse immunosuppression by increasing T cell counts and functionality, paving a potential path to recovery for the currently underserved PML patient population.”

PML is characterized by progressive damage of the white matter of the brain at multiple locations resulting from lytic infection of glia cells, leading to permanent damage of the affected nervous system, and in some cases can be fatal. Common conditions predisposing PML are hematologic and solid malignancies, rheumatologic disorders, primary immune deficiencies, and HIV infection. Currently, there is no specific prophylaxis for PML and no effective anti-JCV treatment.

“Despite its detrimental effects on patients, there remains no designated treatment for PML, and the outcome is dependent on each patient’s capability to recover immune system function. NT-I7’s receipt of Orphan Drug Designation for the treatment of PML underscores its potential to bolster the immune response and reduce severe risks associated with this infection,” said Se Hwan Yang, Ph.D., President and Chief Executive Officer of NeoImmuneTech. “In addition to exploring a new indication for NT-I7, we are grateful to provide much needed hope to the patients and families affected by PML by developing an innovative treatment option.”

The Orphan Drug Act allows the FDA to advance the evaluation and development of products that demonstrate promise for the diagnosis and/or treatment of rare diseases or conditions that affect fewer than 200,000 people in the U.S., incentivizing sponsors to develop products for rare diseases.

NT-I7 (efineptakin alfa) is the only clinical-stage long-acting human IL-7, and is being developed for oncologic and immunologic indications, in which T-cell amplification and increased functionality may provide clinical benefit. IL-7 is a fundamental cytokine for na?ve and memory T-cell development and for sustaining immune response to chronic antigens (as in cancer) or foreign antigens (as in infectious diseases). NT-I7 exhibits favorable PK/PD and safety profiles, making it an ideal combination partner. NT-I7 is being studied in multiple clinical trials in solid tumors and as a vaccine adjuvant. Studies are being planned for testing in hematologic malignancies, additional solid tumors and other immunology-focused indications.