Annals of Oncology October 01, 2025

Neoadjuvant intralesional targeted immunocytokines (daromun) in stage III melanoma

Kähler KC, Hassel JC, Ziemer M, Rutkowski P, Meier F, Flatz L, et al.

Bottom Line

In patients with resectable stage III melanoma, neoadjuvant intralesional administration of daromun (L19IL2 and L19TNF) prior to surgery significantly prolonged recurrence-free and distant metastasis-free survival compared to upfront surgery alone, with a manageable safety profile.

Key Findings

1. Neoadjuvant daromun followed by surgery significantly improved median recurrence-free survival (RFS) to 16.7 months compared to 6.8 months with upfront surgery alone (HR 0.59; 95% CI, 0.41-0.86; P = .005).
2. The estimated 2-year RFS rate was 41.6% (95% CI, 31.4%-55.1%) in the neoadjuvant daromun arm.
3. Daromun significantly prolonged median distant metastasis-free survival (DMFS) to 28.0 months compared to 17.7 months in the control arm (HR 0.60; 95% CI, 0.37-0.95; P = .029), demonstrating that localized treatment can exert a systemic anti-tumor effect.
4. Grade 3 or higher treatment-related adverse events (TRAEs) occurred in 26.3% of patients receiving daromun versus 6.7% in the upfront surgery arm, with the most common being localized injection site reactions.
5. There were no treatment-related deaths in the daromun group, and the incidence of severe systemic immune-related adverse events was notably low.

Study Design

Design
Phase 3 Randomized Controlled Trial
Open-Label
Sample
256
Patients
Duration
21 mo
Median
Setting
Multicenter, Europe
Population Patients with fully resectable, clinically detectable stage IIIB/C (AJCC version 7) melanoma with skin and/or lymph node metastatic lesions amenable to intralesional injection.
Intervention 4 weekly intralesional injections of daromun (13 million IU of L19IL2 and 400 μg of L19TNF) followed by complete surgical resection.
Comparator Upfront complete surgical resection alone. Post-surgical adjuvant systemic therapies were permitted in both arms.
Outcome Recurrence-free survival (RFS) evaluated by Blinded Independent Central Review (BICR).

Study Limitations

The trial utilized an open-label design, which inherently risks introducing performance and ascertainment biases, although this was mitigated by the use of Blinded Independent Central Review (BICR) for the primary endpoint.
Daromun therapy requires direct intratumoral injection, restricting its utility strictly to patients presenting with clinically accessible (injectable) cutaneous, subcutaneous, or lymph node metastases.
The control arm (upfront surgery) does not directly reflect the most modern evolving standard-of-care, as systemic neoadjuvant checkpoint inhibitors (e.g., dual PD-1/CTLA-4 blockade) have rapidly become a preferred approach for high-risk stage III melanoma.

Clinical Significance

The PIVOTAL trial establishes daromun as an active, localized neoadjuvant immunotherapy that yields profound, systemic clinical benefits (reducing distant metastases) in stage III melanoma. Given its robust efficacy and low rates of severe systemic autoimmune toxicity compared to systemic checkpoint inhibitors, daromun offers an essential alternative for patients who are ineligible for, or who have relapsed on, standard systemic immunotherapies. It provides an effective strategy for optimizing locoregional control while minimizing the extensive toxicity footprint typical of widespread systemic immune activation.

Historical Context

The treatment landscape for locoregionally advanced, resectable stage III melanoma has undergone a paradigm shift from upfront surgery to perioperative systemic immunotherapies. Landmark trials (such as SWOG S1801 and NADINA) recently cemented the superiority of neoadjuvant immune checkpoint inhibitors over adjuvant-only approaches. However, systemic checkpoint blockade carries significant risks of lifelong immune-related adverse events. Intralesional therapies attempt to exploit the tumor as an 'in situ' vaccine, generating systemic tumor-specific immunity with limited systemic drug exposure. Daromun combines two potent cytokines—interleukin-2 (IL-2) and tumor necrosis factor (TNF)—fused to the L19 monoclonal antibody, which specifically targets the extra-domain B of fibronectin found in the tumor neovasculature. Following promising phase II results, the PIVOTAL phase III trial was initiated to confirm whether this targeted intralesional approach could alter the natural history of resectable melanoma without the toxicity burden of systemic drugs.

Guided Discussion

High-yield insights from every perspective

Med Student
Medical Student

What is the mechanistic advantage of administering immunocytokines like IL-2 and TNF intralesionally rather than systemically, and how does targeting the extra-domain B of fibronectin enhance this effect?

Key Response

Systemic administration of IL-2 and TNF is severely limited by toxicities such as capillary leak syndrome and severe hypotension. Intralesional administration directly into the tumor microenvironment maximizes local immune activation while minimizing systemic exposure. The L19 antibody targets the extra-domain B (EDB) of fibronectin, a highly conserved marker of tumor angiogenesis, thereby anchoring the cytokines specifically to the tumor stroma to prevent systemic washout.

Resident
Resident

Given the findings of this study, how does the neoadjuvant approach with intralesional daromun theoretically improve outcomes in resectable stage III melanoma compared to adjuvant therapy?

Key Response

Neoadjuvant therapy leverages the presence of the macroscopic tumor and its intact draining lymphatics to prime a more robust and diverse systemic anti-tumor T-cell response before the tumor is surgically removed. This leads to better eradication of micrometastatic disease and is reflected in the prolonged recurrence-free and distant metastasis-free survival seen with daromun, highlighting the shift toward earlier intervention.

Fellow
Fellow

How should we contextualize the efficacy of neoadjuvant intralesional daromun against the recent successes of neoadjuvant systemic immune checkpoint inhibitors in stage III melanoma?

Key Response

While daromun shows benefit over surgery alone, the standard of care is rapidly shifting towards neoadjuvant systemic immune checkpoint blockade (e.g., ipilimumab plus nivolumab). Fellows must weigh the pathological complete response rates, logistical challenges of repeated intralesional injections, and lack of head-to-head data to determine if intralesional cytokines could serve as an alternative for checkpoint inhibitor-refractory patients or those with severe autoimmune contraindications.

Attending
Attending

From a multidisciplinary practice standpoint, what logistical and operational challenges must be overcome to implement a neoadjuvant intralesional injection protocol like daromun prior to definitive surgical resection?

Key Response

Implementing intralesional therapy requires tight coordination between surgical oncology, medical oncology, and potentially interventional radiology. Attendings must consider scheduling logistics, managing localized inflammatory reactions that could obscure or complicate surgical planes, and ensuring the interval between the completion of injections and surgery is optimized for both patient safety and peak immunologic effect.

Scholarly Review

Critical appraisal through the lens of expert reviewers and guideline development

PhD
PhD

From an immunobiology and study design perspective, how would you design a correlative translational study to prove that intralesional daromun induces a systemic abscopal effect rather than purely local tumor necrosis?

Key Response

To validate a systemic immune mechanism, researchers would need to perform high-dimensional flow cytometry or single-cell RNA sequencing on peripheral blood and distant draining lymph nodes, track T-cell receptor (TCR) clonality before and after injection, and utilize preclinical bilateral flank models to evaluate tumor regression in distant, uninjected lesions compared to vehicle controls.

Journal Editor
Journal Editor

In evaluating the methodological rigor of this trial, how does the choice of 'surgery alone' as the control arm impact the clinical relevance and editorial significance of the study's primary endpoints?

Key Response

A critical reviewer would flag that 'surgery alone' is no longer the modern standard of care for high-risk stage III melanoma. Given the widespread adoption of highly effective adjuvant and neoadjuvant systemic therapies (like PD-1 and CTLA-4 inhibitors), the editorial board must decide if a trial demonstrating superiority over surgery alone provides sufficient clinical utility or if the results are outdated by shifting standards.

Guideline Committee
Guideline Committee

Based on this trial's data, what level of evidence does daromun provide, and how should it be positioned within current NCCN or ESMO guidelines alongside established neoadjuvant and adjuvant systemic therapies?

Key Response

The committee must evaluate whether the evidence is robust enough to include daromun as a recommended option. Current NCCN and ESMO guidelines strongly favor systemic checkpoint inhibitors or BRAF/MEK inhibitors for high-risk stage III disease. Daromun might receive a lower-tier recommendation due to the control arm not reflecting current standard-of-care systemic therapy, positioning it potentially as a niche option for patients unsuitable for systemic checkpoint blockade.

Clinical Landscape

Noteworthy Related Trials

2015

OPTiM Trial

n = 436 · JCO

Tested

Talimogene laherparepvec (T-VEC)

Population

Stage IIIB to IVM1c melanoma with injectable lesions

Comparator

Subcutaneous GM-CSF

Endpoint

Durable response rate (DRR)

Key result: T-VEC significantly improved the durable response rate compared to GM-CSF, with a manageable safety profile.
2019

OpACIN-neo Trial

n = 86 · Lancet Oncol

Tested

Neoadjuvant ipilimumab plus nivolumab

Population

Macroscopic stage III melanoma

Comparator

Alternative dosing schedules of ipilimumab and nivolumab

Endpoint

Toxicity and pathological response

Key result: Identified an optimal dosing schedule that preserved high pathological response rates while significantly reducing immune-related toxicities.
2023

SWOG S1801 Trial

n = 313 · NEJM

Tested

Neoadjuvant-adjuvant pembrolizumab

Population

Stage III-IV resectable melanoma

Comparator

Adjuvant pembrolizumab alone

Endpoint

Event-free survival (EFS)

Key result: Neoadjuvant-adjuvant pembrolizumab significantly improved event-free survival compared to adjuvant therapy alone.

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