What Is Efineptakin alfa?
Efineptakin alfa, also known by its investigational code NT-I7, is a drug currently under study in clinical trials. Based on the available trial data, the specific mechanism by which efineptakin alfa works is not detailed. However, trial descriptions indicate it is administered via intramuscular injection.
Efineptakin alfa is being investigated for its potential therapeutic effects in a range of complex conditions. These include various cancers such as Non-Small Cell Lung Cancer, Kaposi Sarcoma, High Grade Astrocytic Tumor, and Recurrent Diffuse Large B-Cell Lymphoma. It is also being studied for HIV Infection. Research is exploring its use both as a standalone treatment and in combination with other established therapies, including pembrolizumab, atezolizumab, and following CAR-T cell infusions.
There have been a total of 5 clinical trials involving efineptakin alfa, with a combined enrollment of 166 participants. The earliest trial began on October 19, 2020, and the most recent started on July 20, 2022. Key sponsors conducting these studies include NeoImmuneTech, Fred Hutchinson Cancer Center, Hyunseok Kang, MD, and Mayo Clinic.
Uses and Conditions Under Study
Efineptakin alfa is being investigated across several serious medical conditions, primarily focusing on various cancers and immune system disorders.
One significant area of study is Non-Small Cell Lung Cancer (NSCLC). This type of cancer accounts for the majority of all lung cancers. Efineptakin alfa is being explored for its potential role in treating different forms of NSCLC, including Carcinoma, Non-Small-Cell Lung; Non-Small Cell Lung Cancer; Non-Small Cell Lung Carcinoma; Non Small Cell Lung Cancer; and Nonsmall Cell Lung Cancer. These related conditions are being studied in a total of 1 trial for each specific listing, indicating a focus on this broad category.
Another condition under investigation is Kaposi Sarcoma, including AIDS-Related Kaposi Sarcoma. This is a type of cancer that forms lesions on the skin, in lymph nodes, organs, or mucous membranes, often associated with immune suppression. Efineptakin alfa is being studied in 1 trial for AIDS-Related Kaposi Sarcoma and 1 trial for Kaposi Sarcoma, suggesting its potential utility in immune-compromised cancer patients.
Efineptakin alfa is also being studied for its effects in HIV Infection, a condition that weakens the immune system. A single trial is exploring the drug's role in this context. Additionally, it is being investigated for High Grade Astrocytic Tumor, a type of aggressive brain cancer, in 1 trial. Finally, Recurrent Diffuse Large B-Cell Lymphoma, an aggressive form of non-Hodgkin lymphoma that has returned after initial treatment, is another condition where efineptakin alfa is being studied in 1 trial.
Dosing
Efineptakin alfa, also known as NT-I7, has been studied exclusively as an intramuscular injection in clinical trials. The specific dosage forms mentioned in the trials describe it as a 'Treatment' rather than traditional forms like tablets or oral solutions, indicating its administration as an injectable therapy.
One detailed dosing regimen observed in trials involves administering 1200 μg/kg of NT-I7. This dose is given intramuscularly (IM) once every 6 weeks (Q6W), starting on Cycle 1. This treatment schedule is designed to continue for an extended period, up to a maximum of 35 cycles, which is approximately 2 years.
In other investigational settings, efineptakin alfa has been administered via intramuscular injection after CAR-T cell infusion, specifically on Day 21. It has also been studied in combination with other drugs, such as pembrolizumab and atezolizumab, as part of a broader treatment strategy. The available data does not distinguish between adult and pediatric dosing, focusing on the described regimens for participants in the studies.
Side Effects
In a clinical trial (NCT04893018) investigating Efineptakin alfa (NT-I7) for Kaposi Sarcoma, all participants in both dose levels experienced at least one adverse event. Specifically, 100% of participants in the Dose Level 1 group and 100% of participants in the Dose Level 2 group reported adverse events. The available data did not provide specific details on the types or frequencies of individual side effects experienced by participants in this study.
Clinical Trial Results
The efficacy of Efineptakin alfa (NT-I7) was evaluated in a completed clinical trial (NCT04893018) for Kaposi Sarcoma in patients with or without HIV. This study investigated the drug's impact on immune cell counts and tumor response.
Immune Cell Kinetics
The study measured changes in various immune cell populations in the blood, expressed as median ratios compared to baseline:
- Absolute Lymphocyte Counts (ALC): In patients receiving Efineptakin alfa Dose Level 1, median ALC ratios ranged from 1.06 to 3.20, indicating an increase in these counts. For Dose Level 2, median ALC ratios ranged from 1.15 to 3.93, also showing an increase.
- CD4+ T Cells: For Dose Level 1, median CD4+ T cell ratios ranged from 1.26 to 4.46. For Dose Level 2, these ratios ranged from 1.19 to 6.30. These results suggest an increase in CD4+ T cell counts in both dose groups.
- CD8+ T Cells: In Dose Level 1, median CD8+ T cell ratios ranged from 0.89 to 3.25. A ratio of 0.89 indicates a slight decrease in some measurements, while other measurements showed increases up to 3.25 times baseline. For Dose Level 2, median CD8+ T cell ratios ranged from 1.29 to 4.12, indicating an increase in these cells.
Objective Response Rate (ORR)
The objective response rate, which measures the proportion of patients whose cancer shrinks or disappears, was reported as the number of participants achieving a response:
- In the Dose Level 1 group, 1 or 2 participants (depending on the specific assessment point) achieved an objective response, while 0 participants achieved a response at another assessment.
- In the Dose Level 2 group, 2 participants achieved an objective response at two different assessment points, while 0 participants achieved a response at another assessment.
Other outcomes such as Duration of Response (DOR), Overall Survival (OS), and Progression-free Survival (PFS) were not available (NA) in the reported data for this trial.
Currently Recruiting Trials
Efineptakin alfa is an immunotherapy drug being studied for its potential to help the immune system fight tumor cells. Clinical trials are currently recruiting participants to evaluate its safety and effectiveness, often in combination with other treatments.
One active trial, NCT05465954, is a Phase 2 study testing efineptakin alfa in combination with pembrolizumab. This trial focuses on patients with glioblastoma that has returned (recurrent), including high-grade astrocytic tumors, IDH-wildtype recurrent glioblastoma, and recurrent gliosarcoma. The study aims to understand the safety and side effects of this combination therapy. Sponsored by the Mayo Clinic, this trial plans to enroll up to 54 patients.
Where to Participate
Participation in the currently recruiting trial for efineptakin alfa is available at a single location in the United States. This study is actively enrolling participants at one site, located in Rochester, Minnesota.
To be eligible for this trial, participants must be between the ages of 18 to 18 years old. The study is open to individuals of all genders, but it does not accept healthy volunteers or children. Patients interested in participating should discuss their eligibility with their healthcare provider.
Development Timeline
The journey of efineptakin alfa in clinical development began on October 19, 2020, with the latest trial starting on July 20, 2022. Since its inception, a total of five clinical trials have been initiated, enrolling a combined total of 166 participants.
Early development primarily involved Phase 1 studies, with three trials exploring initial safety and dosage. The program has since advanced to include two Phase 2 trials, investigating effectiveness in specific conditions. Several organizations have sponsored these studies:
- NeoImmuneTech (2 trials)
- Fred Hutchinson Cancer Center (1 trial)
- Hyunseok Kang, MD (1 trial)
- Mayo Clinic (1 trial)
Efineptakin alfa's pipeline has significantly expanded from its initial focus on conditions such as IBS-C and hyperphosphatemia. Its development now includes a wide range of challenging diseases, particularly various cancers. These include high-grade astrocytic tumors, recurrent glioblastoma, and gliosarcoma, as well as different forms of non-small cell lung cancer. The drug is also being explored for its potential in recurrent and refractory diffuse large B-cell lymphoma and other high-grade B-cell lymphomas. Furthermore, studies have expanded to include recurrent and resectable squamous cell carcinomas of the head and neck, such as those affecting the hypopharynx, larynx, oral cavity, and oropharynx. Efineptakin alfa is also under investigation for HIV infection and Kaposi Sarcoma, reflecting a broad therapeutic exploration.