On May 24, 2021, biopharmaceutical company NeoImmuneTech, Inc. shared that the first patient was dosed in a pilot study centered around NT-I7 (efineptakin alfa). Altogether, the study is designed to evaluate NT-I7 for patients with progressive multifocal leukoencephalopathy (PML). Currently, no approved treatment options exist for PML. Thus, NT-I7 offers an opportunity to fill an unmet need within this patient population.
NT-I7
In this early Phase 1 clinical trial, researchers will explore NT-I7 as a potential therapeutic option for patients with PML. Overall, an estimated 12 patients will enroll. According to the study details, participants must be aged 18+. During the trial:
Participants will have a 7-day inpatient stay, outpatient visits, [follow-up phone calls], a medical history and physical exam, [and will provide] urine [and blood] samples. Participation will last for 12 to 19 months.
During the course of the trial, patients will receive up to 3 NT-I7 doses via intramuscular injection. Ultimately, the study goal is to determine whether NT-I7 administration raises lymphocyte levels. Lymphocytes, a type of white blood cell, help the body to control infections. Since PML is caused by the John Cunningham virus (JCV), increased lymphocytes could help control symptoms and manage outcomes.
NT-I7 is a long-acting human interleukin-7 (IL-7) which helps prompt T-cell development. T cells are a type of lymphocyte and also play a role in immune response. According to NeoImmuneTech, NT-I7 is:
a more potent, stable, and long-acting human IL-7 engineered to overcome its structural instability and to enable mass production of NT-I7. The hyFc® domain extends the half-life of IL-7, thereby enhancing bioavailability which provides better potency and stability.
Thus far, NT-I7 has been explored in a number of clinical trials. During these trials, researchers have determined that NT-I7 is relatively safe and well-tolerated, both alone and in conjunction with other therapies.
Progressive Multifocal Leukoencephalopathy (PML)
Progressive multifocal leukoencephalopathy (PML) is a progressive neurological disorder caused by the John Cunningham virus (JCV), named after the first patient with this condition. Typically, people become infected with JCV during childhood. In fact, an estimated 85% of the adult population has this virus. In healthy individuals, the virus causes no issues or complications. Rather, it remains inactive, typically in the kidneys and lymphoid organs. But when the immune system becomes weakened, the virus causes a host of health complications. The virus begins to target and destroy myelin, the protective covering of nerve cells. As a result, the brain’s white matter is damaged. Although some individuals have only one brain lesion, many people with PML have multiple brain lesions.
Altogether, an estimated 1 in every 200,000 people has PML. Conditions like HIV/AIDS, cancer, immune deficiencies, and other disorders increase the risk of PML. Because lesions can occur throughout the central nervous system (CNS), symptoms vary between individuals. However, some symptoms include:
- Seizures
- Note: An estimated 1/3 of patients with PML experience seizures.
- Problems with gait, such as difficulty walking
- Difficulty speaking
- Vision loss
- Clumsiness or loss of coordination
- Facial drooping
- Progressive muscle weakness
- Changes in mood, behavior, or personality
- Sensory impairment
- Headaches
- Memory problems
