On June 2, 2025 Iovance Biotherapeutics, Inc. (NASDAQ: IOVA), a commercial biotechnology company focused on innovating, developing, and delivering novel polyclonal tumor infiltrating lymphocyte (TIL) therapies for patients with cancer, reported that the Journal of Clinical Oncology has published the final analysis from the Phase 2 C-144-01 clinical trial evaluating the individualized T cell therapy Amtagvi (lifileucel) in patients with advanced melanoma (Press release, Iovance Biotherapeutics, JUN 2, 2025, View Source [SID1234653613]). These five-year follow-up results are being simultaneously presented during an oral session today at the 2025 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting.

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This five-year analysis of the C-144-01 trial represents unprecedented durability and duration of follow-up in advanced melanoma patients previously treated with anti-PD-1 and targeted therapy, where applicable. The analysis includes 153 patients from cohorts 2 and 4 of the C-144-01 trial.

One-time Amtagvi treatment demonstrated long-term benefit in heavily pretreated patients, including deep and durable responses, meaningful overall survival without further treatment, and no new or late-onset adverse events at a median follow-up of 57.8 months. The objective response rate was 31.4% with a median time to response of 1.4 months and a median duration of response of 36.5 months. Nearly one third of responders (31.3%) completed the five-year assessment with ongoing responses. The median overall survival (mOS) was 13.9 months with a five-year survival rate of 19.7%. Survival outcomes were consistent among responders, regardless of time of onset of response to Amtagvi therapy.

The observed safety profile was consistent with that of nonmyeloablative lymphodepletion and interleukin-2 administration. The incidence of AEs decreased rapidly within the first two weeks after Amtagvi infusion, and there were no new or late-onset treatment-related AEs.

Theresa Medina, M.D., medical oncologist at the University of Colorado Cancer Center on the Anschutz Medical Campus, stated, "Amtagvi has demonstrated long-term benefit and meaningful overall survival in a difficult-to-treat melanoma patient population resistant to immune checkpoint inhibitor therapy. Five years following one-time Amtagvi treatment, responses persisted or deepened during an extended treatment-free interval for some patients. Amtagvi offers a new standard of care for the advanced melanoma community and sets a new bar for one-time cell therapies with curative intent in solid tumors."

In February 2024, the U.S. Food and Drug Administration granted accelerated approval to Amtagvi for the treatment of adult patients with unresectable or metastatic melanoma previously treated with a PD-1 blocking antibody, and if BRAF V600 mutation positive, a BRAF inhibitor with or without a MEK inhibitor. The approval was based on overall response rate and duration of response from the C-144-01 clinical trial. With this approval, Amtagvi became the first one-time T cell therapy for a solid tumor cancer as well as the first approved treatment option for patients with advanced melanoma after anti-PD-1 and targeted therapy. Iovance is also conducting TILVANCE-301, a Phase 3 trial in frontline advanced melanoma to confirm clinical benefit.

About the C-144-01 Clinical Trial

C-144-01 is a global, multicenter Phase 2 study in which patients received treatment with lifileucel. The study enrolled patients with metastatic melanoma who were previously treated with at least one systemic therapy, including a PD-1 blocking antibody, and if BRAF V600 mutation positive, a BRAF inhibitor or BRAF inhibitor with MEK inhibitor. Efficacy was established on the basis of objective response rate (ORR) and duration of response (DOR) by Independent Review Committee (IRC) per Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1. The pivotal Cohort 4 and supportive Cohort 2 of Study C-144-01 enrolled patients that met the same primary eligibility criteria, had the same assessments, and had received the same regimen and AMTAGVI that was produced using the same manufacturing process, and product formulation. The final five-year analysis of C-144-01 was published in the Journal of Clinical Oncology in 2025.

About Amtagvi

AMTAGVI is a prescription medicine used to treat adults with a type of skin cancer that cannot be removed surgically or has spread to other parts of the body called unresectable or metastatic melanoma.

AMTAGVI is used when your melanoma has not responded or stopped responding to a PD-1 blocking drug either by itself or in a combination, and if your cancer is BRAF mutation positive, a BRAF inhibitor drug with or without a MEK inhibitor drug that has also stopped working.

The approval of AMTAGVI is based on a study that measured response rate. Continued approval for this use may depend on the results of an ongoing study to confirm benefit.

Important Safety Information

What is the most important information that I should know about AMTAGVI?

You will likely be in a hospital prior to and after receiving AMTAGVI.

Before taking AMTAGVI, tell your healthcare provider about all of your medical conditions, including if you:

Have any lung, heart, liver or kidney problems
Have low blood pressure
Have a recent or active infection or other inflammatory conditions including cytomegalovirus (CMV) infection, hepatitis B or C or human immunodeficiency virus (HIV) infection
Are pregnant, think you may be pregnant, or plan to become pregnant
Are breastfeeding
Notice the symptoms of your cancer are getting worse
Have had a vaccination in the past 28 days or plan to have one in the next few months
Have been taking a blood thinner
Tell your doctor about all the medications you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.

How will I receive AMTAGVI?

AMTAGVI is made from your surgically removed tumor. Tumor derived T cells are grown in a manufacturing center at the end of which they number in the billions of cells.
Your tumor tissue is sent to a manufacturing center to make AMTAGVI. It takes about 34 days from the time your tumor tissue is received at the manufacturing center until AMTAGVI is available to be shipped back to your healthcare provider, but the time may vary. Your AMTAGVI will be provided in 1-4 patient-specific infusion bag(s) containing 100 mL to 125 mL of viable (alive) cells per bag.
After your AMTAGVI arrives at your treating institution, your healthcare provider will give you lymphodepleting chemotherapy to prepare your body.
Approximately 30 to 60 minutes before you are given AMTAGVI, you may be given other medicines including:
Medicines for an allergic reaction (anti-histamines)
Medicines for fever (such as acetaminophen)
Your AMTAGVI will be provided in 1 to 4 infusion bag(s) containing 100 mL to 125 mL of viable cells per bag. When your body is ready for AMTAGVI infusion, your healthcare provider will give AMTAGVI to you by intravenous infusion. This usually takes less than 90 minutes.
After getting AMTAGVI

Beginning 3 to 24 hours after AMTAGVI is given, you may be given up to 6 doses of IL-2 (aldesleukin) every 8 to 12 hours via intravenous infusion. Your doctor may discontinue IL-2 (aldesleukin) infusion any time if you have severe side effects.

You will have to stay in the hospital until you have completed the IL-2 (aldesleukin) treatment and you have recovered from any serious side effects associated with the AMTAGVI treatment.

You should plan to stay within 2 hours of the location where you received your treatment for several weeks after getting AMTAGVI. Your healthcare provider will check to see if your treatment is working and help you with any side effects that occur.

What are the possible side effects of AMTAGVI?

The most common side effects of the AMTAGVI treatment include chills, fever, low white blood cell count (may increase risk of infections), fatigue, low red blood cell count (may cause you to feel tired or weak), fast or irregular heartbeat, rash, low blood pressure, and diarrhea.

These are not all the possible side effects of the AMTAGVI treatment. Talk with your healthcare provider for more information about AMTAGVI. You can ask your healthcare provider for information about AMTAGVI that is written for healthcare professionals.

You may report side effects to Iovance at 1-833-400-4682, or to the FDA, at 1-800-FDA-1088 or at www.fda.gov/medwatch.

Please see Full Prescribing Information and Patient Information, including Boxed Warning, for additional Important Safety Information.