Real-World Data Demonstrate a 49% Response Rate for Commercial Amtagvi® in Patients with Advanced Melanoma

Rhea-AI Summary

Iovance Biotherapeutics (NASDAQ: IOVA) reported positive real-world data for its commercial Amtagvi® (lifileucel) therapy in advanced melanoma patients. The study showed a 48.8% overall response rate among 41 evaluable patients across four treatment centers. Notably, patients receiving treatment in third-line or earlier demonstrated a higher response rate of 60.9%, while those with three or more prior lines of therapy showed a 33.3% response rate.

Amtagvi, approved by the FDA in February 2024, is the first one-time T cell therapy for solid tumor cancer and the first approved treatment for advanced melanoma after anti-PD-1 and targeted therapy. The company is currently conducting the TILVANCE-301 Phase 3 trial in frontline advanced melanoma to confirm clinical benefit.

Positive

• Strong 48.8% overall response rate in real-world settings
• Higher 60.9% response rate in less heavily treated patients (third-line or earlier)
• First-of-its-kind approved one-time T cell therapy for solid tumors
• Demonstrated efficacy across multiple treatment centers

Negative

• Lower 33.3% response rate in heavily pretreated patients (3+ prior lines)
• Still requires confirmatory Phase 3 trial for full approval

Insights

Oncology Specialist

Amtagvi shows impressive 49% real-world response rate in advanced melanoma, particularly effective (61%) in less pre-treated patients.

The real-world data for Amtagvi (lifileucel) demonstrates remarkable efficacy that validates its February 2024 FDA accelerated approval. The overall 48.8% response rate across 41 evaluable patients is clinically meaningful for advanced melanoma patients who have progressed after standard therapies. What's particularly striking is the 60.9% response rate in less heavily pre-treated patients (third-line or earlier), compared to 33.3% in those with three or more prior treatments.

This data carries significant clinical implications. The 27.6% higher response rate in earlier-treated patients strongly suggests that Amtagvi should be positioned earlier in treatment sequencing, potentially as a standard third-line option following immune checkpoint inhibitor failure. This pattern of diminishing returns with increased prior therapies is consistent with what we observe with many cancer immunotherapies, where less exhausted immune systems typically respond better.

As the first FDA-approved one-time T-cell therapy for solid tumors, these real-world results confirm that Amtagvi's efficacy translates from clinical trials to everyday practice settings. The pending TILVANCE-301 Phase 3 trial in frontline melanoma could potentially expand its indication dramatically if positive. For advanced melanoma patients with limited options after PD-1 inhibitor and BRAF/MEK targeted therapy failure, these response rates represent a significant therapeutic advancement that could establish TIL therapy as a foundational treatment approach.

Biotechnology Investment Analyst

Iovance's Amtagvi shows robust real-world effectiveness with 49% response rate, validating commercial potential and positioning for expanded market share.

This real-world data substantially derisks Iovance's commercial launch of Amtagvi, providing compelling evidence that the therapy's clinical trial efficacy translates into real-world settings. The 48.8% overall response rate across 41 patients essentially confirms the efficacy seen in the pivotal C-144-01 trial that led to approval. More importantly, the superior 60.9% response rate in less heavily pretreated patients creates a clear value proposition for earlier use.

From a market perspective, this data strategically positions Amtagvi for broader and earlier adoption, potentially accelerating revenue growth. The therapy addresses a significant unmet need in post-PD-1 advanced melanoma, with approximately 7,000 eligible patients annually in the US alone. With a premium one-time treatment price point likely in the $450,000-$500,000 range, achieving significant market penetration could translate to substantial revenue.

The physician quote highlighting "remarkable" responses in more than half of less pretreated patients suggests growing confidence among key opinion leaders, which typically drives community oncologist adoption. Additionally, Iovance's ongoing TILVANCE-301 Phase 3 trial in frontline melanoma represents a significant market expansion opportunity that could multiply the addressable patient population. As the first approved TIL therapy for solid tumors, Amtagvi establishes Iovance as the leader in this emerging therapeutic class, with potential applications across multiple tumor types in their pipeline.

61% Response Rate Observed in Third-Line or Earlier Patients

SAN CARLOS, Calif., July 14, 2025 (GLOBE NEWSWIRE) -- 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, today announced that a real-world, retrospective study demonstrates the benefit of commercial Amtagvi^® (lifileucel) in real-world clinical settings for patients with advanced (unresectable or metastatic) melanoma previously treated with immune checkpoint inhibitor (ICI) therapy and, if appropriate, targeted therapy.

Among 41 evaluable patients treated at four authorized treatment centers, the physician-assessed objective response rate (ORR) was 48.8% (20/41). Response rates with Amtagvi were higher in third-line or earlier patients (two or fewer prior lines of therapy) with an ORR of 60.9% (14/23). An ORR of 33.3% (6/18) was observed in patients following three or more prior lines of therapy. All evaluable patients received commercial Amtagvi according to the U.S. prescribing information and completed at least one follow-up physician assessment.

Additional results and follow-up from the real-world clinical study will be presented at an upcoming medical meeting this year.

Lilit Karapetyan, MD, MS of H. Lee Moffitt Cancer Center & Research Institute stated, “Lifileucel demonstrates a robust response rate in real-world clinical settings. I am particularly encouraged by the higher response rate in less heavily treated patients. It is remarkable to observe a response in more than half of those patients, supporting consideration of lifileucel as soon as possible after ICI therapy. I am confident in the potential for positive outcomes in more patients as we continue to adopt lifileucel.”

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 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.

About Iovance Biotherapeutics, Inc.
Iovance Biotherapeutics, Inc. aims to be the global leader in innovating, developing, and delivering tumor infiltrating lymphocyte (TIL) therapies for patients with cancer. We are pioneering a transformational approach to cure cancer by harnessing the human immune system’s ability to recognize and destroy diverse cancer cells in each patient. The Iovance TIL platform has demonstrated promising clinical data across multiple solid tumors. Iovance’s Amtagvi^® is the first FDA-approved T cell therapy for a solid tumor indication. We are committed to continuous innovation in cell therapy, including gene-edited cell therapy, that may extend and improve life for patients with cancer. For more information, please visit http://www.iovance.com/.

Amtagvi^® and its accompanying design marks, Proleukin^®, Iovance^®, and IovanceCares™ are trademarks and registered trademarks of Iovance Biotherapeutics, Inc. or its subsidiaries. All other trademarks and registered trademarks are the property of their respective owners.

Forward-Looking Statements
Certain matters discussed in this press release are “forward-looking statements” of Iovance Biotherapeutics, Inc. (hereinafter referred to as the “Company,” “we,” “us,” or “our”) within the meaning of the Private Securities Litigation Reform Act of 1995 (the “PSLRA”). Without limiting the foregoing, we may, in some cases, use terms such as “predicts,” “believes,” “potential,” “continue,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “forecast,” “guidance,” “outlook,” “may,” “can,” “could,” “might,” “will,” “should,” or other words that convey uncertainty of future events or outcomes and are intended to identify forward-looking statements. Forward-looking statements are based on assumptions and assessments made in light of management’s experience and perception of historical trends, current conditions, expected future developments, and other factors believed to be appropriate. Forward-looking statements in this press release are made as of the date of this press release, and we undertake no duty to update or revise any such statements, whether as a result of new information, future events or otherwise. Forward-looking statements are not guarantees of future performance and are subject to risks, uncertainties, and other factors, many of which are outside of our control, that may cause actual results, levels of activity, performance, achievements, and developments to be materially different from those expressed in or implied by these forward-looking statements. Important factors that could cause actual results, developments, and business decisions to differ materially from forward-looking statements are described in the sections titled "Risk Factors" in our filings with the U.S. Securities and Exchange Commission, including our most recent Annual Report on Form 10-K and Quarterly Reports on Form 10-Q, and include, but are not limited to, the following substantial known and unknown risks and uncertainties inherent in our business: the risks related to our ability to successfully commercialize our products, including Amtagvi, for which we have obtained U.S. Food and Drug Administration (“FDA”) approval, and Proleukin, for which we have obtained FDA and European Medicines Agency (“EMA”) approval; the risk that the EMA or other ex-U.S. regulatory authorities may not approve or may delay approval for our marketing authorization application submission for lifileucel in metastatic melanoma; the acceptance by the market of our products, including Amtagvi and Proleukin, and their potential pricing and/or reimbursement by payors, if approved (in the case of our product candidates), in the U.S. and other international markets and whether such acceptance is sufficient to support continued commercialization or development of our products, including Amtagvi and Proleukin, or product candidates, respectively; future competitive or other market factors may adversely affect the commercial potential for Amtagvi or Proleukin; the risk regarding our ability or inability to manufacture our therapies using third party manufacturers or at our own facility, including our ability to increase manufacturing capacity at such third party manufacturers and our own facility, may adversely affect our commercial launch; the results of clinical trials with collaborators using different manufacturing processes may not be reflected in our sponsored trials; the risk regarding the successful integration of the recent Proleukin acquisition; the risk that the successful development or commercialization of our products, including Amtagvi and Proleukin, may not generate sufficient revenue from product sales, and we may not become profitable in the near term, or at all; the risks related to the timing of and our ability to successfully develop, submit, obtain, or maintain FDA, EMA, or other regulatory authority approval of, or other action with respect to, our product candidates; whether clinical trial results from our pivotal studies and cohorts, and meetings with the FDA, EMA, or other regulatory authorities may support registrational studies and subsequent approvals by the FDA, EMA, or other regulatory authorities, including the risk that the planned single arm Phase 2 IOV-LUN-202 trial may not support registration; preliminary and interim clinical results, which may include efficacy and safety results, from ongoing clinical trials or cohorts may not be reflected in the final analyses of our ongoing clinical trials or subgroups within these trials or in other prior trials or cohorts; the risk that enrollment may need to be adjusted for our trials and cohorts within those trials based on FDA and other regulatory agency input; the risk that the changing landscape of care for cervical cancer patients may impact our clinical trials in this indication; the risk that we may be required to conduct additional clinical trials or modify ongoing or future clinical trials based on feedback from the FDA, EMA, or other regulatory authorities; the risk that our interpretation of the results of our clinical trials or communications with the FDA, EMA, or other regulatory authorities may differ from the interpretation of such results or communications by such regulatory authorities (including from our prior meetings with the FDA regarding our non-small cell lung cancer clinical trials); the risk that clinical data from ongoing clinical trials of Amtagvi will not continue or be repeated in ongoing or planned clinical trials or may not support regulatory approval or renewal of authorization; the risk that unanticipated expenses may decrease our estimated cash balances and forecasts and increase our estimated capital requirements; the risk that we may not be able to recognize revenue for our products; the risk that Proleukin revenues may not continue to serve as a leading indicator for Amtagvi revenues; the risks regarding our anticipated operating and financial performance, including our financial guidance and projections; the effects of global pandemic; the effects of global and domestic geopolitical factors; and other factors, including general economic conditions and regulatory developments, not within our control. Any financial guidance provided in this press release assumes the following: no material change in our ability to manufacture our products; no material change in payor coverage; no material change in revenue recognition policies; no new business development transactions not completed as of the period covered by this press release; and no material fluctuation in exchange rates.

CONTACTS 

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FAQ

What is the response rate of Amtagvi in real-world melanoma patients?

Real-world data shows Amtagvi achieved a 48.8% overall response rate in 41 evaluable patients with advanced melanoma across four treatment centers.

How effective is Amtagvi in early-line melanoma treatment?

Patients receiving Amtagvi in third-line or earlier treatment showed a 60.9% response rate, compared to 33.3% in patients with three or more prior lines of therapy.

When did the FDA approve Iovance's Amtagvi for melanoma treatment?

The FDA granted accelerated approval to Amtagvi in February 2024 for treating advanced melanoma in patients previously treated with PD-1 blocking antibody and targeted therapy if BRAF V600 mutation positive.

What makes Amtagvi unique in cancer treatment?

Amtagvi is the first one-time T cell therapy approved for solid tumor cancer and the first approved treatment for advanced melanoma after anti-PD-1 and targeted therapy.

What ongoing trials is Iovance conducting for Amtagvi?

Iovance is conducting the TILVANCE-301 Phase 3 trial in frontline advanced melanoma to confirm clinical benefit.