New England Journal of Medicine DECEMBER 06, 2016

AURA3: Osimertinib versus Platinum-Based Chemotherapy in EGFR T790M-Positive Advanced NSCLC

Vassiliki A. Papadimitrakopoulou et al.

Bottom Line

The AURA3 trial demonstrated that third-generation EGFR inhibitor osimertinib significantly improves progression-free survival compared to platinum-pemetrexed chemotherapy in patients with T790M-positive non-small cell lung cancer who progressed on prior EGFR-TKI therapy.

Key Findings

1. Osimertinib demonstrated superior progression-free survival (PFS) with a median of 10.1 months compared to 4.4 months for chemotherapy (Hazard Ratio [HR] 0.30; 95% CI: 0.23–0.41; P<0.001).
2. The objective response rate (ORR) was significantly higher in the osimertinib arm at 71% versus 31% in the chemotherapy arm.
3. In patients with CNS metastases, osimertinib showed a significant PFS benefit over chemotherapy (HR 0.32; 95% CI: 0.21–0.49).
4. No statistically significant improvement in overall survival was observed (HR 0.87; 95% CI: 0.67–1.12), likely confounded by a high crossover rate (approximately 73%) from the chemotherapy group to osimertinib upon progression.
5. Osimertinib showed a more favorable safety profile with a lower incidence of grade 3 or higher adverse events (23%) compared to the chemotherapy group (47%).

Study Design

Design
RCT
Open-Label
Sample
419
Patients
Duration
Data cutoff 15 March 2019
Median
Setting
Multicenter, International
Population Adult patients with locally-advanced or metastatic EGFR T790M-positive non-small cell lung cancer (NSCLC) who had disease progression on or after previous EGFR-TKI therapy.
Intervention Oral osimertinib 80 mg once daily.
Comparator Platinum-based doublet chemotherapy (pemetrexed plus carboplatin or cisplatin) for up to 6 cycles, with optional pemetrexed maintenance.
Outcome Progression-free survival (PFS) assessed by blinded independent central review.

Study Limitations

The open-label design could introduce performance or assessment bias, though the primary endpoint was determined by blinded independent central review.
The study did not demonstrate an overall survival benefit, largely due to the allowance of crossover upon disease progression, which complicates survival analysis.
The study focused exclusively on patients with T790M-positive mutations, limiting direct application to patients whose acquired resistance mechanism is independent of the T790M mutation.

Clinical Significance

AURA3 established osimertinib as the standard of care for patients with advanced EGFR-mutated NSCLC who develop the T790M resistance mutation after initial EGFR-TKI therapy, replacing platinum-based chemotherapy in this setting.

Historical Context

Before AURA3, acquired resistance to first-generation EGFR-TKIs was a major clinical challenge, with the T790M mutation accounting for approximately 60% of such resistance cases. The trial served as a confirmatory Phase III study to support the accelerated approval of osimertinib, moving the paradigm away from cytotoxic chemotherapy for patients with identifiable T790M mutations.

Guided Discussion

High-yield insights from every perspective

Med Student
Medical Student

Explain the biochemical mechanism by which the T790M mutation confers resistance to first-generation EGFR inhibitors like erlotinib and how osimertinib overcomes this.

Key Response

The T790M 'gatekeeper' mutation replaces a threonine residue with a bulkier methionine in the ATP-binding pocket of the EGFR kinase domain, sterically hindering the binding of first-generation reversible TKIs. Osimertinib is a third-generation, irreversible inhibitor that forms a covalent bond with the Cys797 residue, allowing it to maintain high affinity for the receptor even in the presence of the T790M mutation.

Resident
Resident

A patient with EGFR-mutated NSCLC progresses on gefitinib. According to the paradigm established by AURA3, what is the necessary diagnostic workflow before switching to osimertinib?

Key Response

The patient must undergo molecular testing specifically for the T790M resistance mutation. This is typically done via a 'plasma-first' approach using liquid biopsy (ctDNA). If the liquid biopsy is negative, a tissue re-biopsy is required to rule out a false negative, as AURA3 demonstrated that osimertinib's superior efficacy over chemotherapy is contingent upon T790M-positive status.

Fellow
Fellow

Discuss the clinical significance of osimertinib's CNS penetration as demonstrated in the AURA3 trial compared to the platinum-pemetrexed control arm.

Key Response

In patients with stable CNS metastases at baseline, AURA3 showed that osimertinib significantly prolonged CNS progression-free survival (11.7 months vs 5.6 months). This is due to osimertinib's superior blood-brain barrier penetration compared to chemotherapy and earlier TKIs, making it a preferred agent for preventing or treating intracranial progression in the T790M+ setting.

Attending
Attending

Given the results of the subsequent FLAURA trial, how has the practical application of the AURA3 data shifted in current oncology practice for newly diagnosed EGFR-mutant NSCLC?

Key Response

While AURA3 established osimertinib as the second-line standard for T790M+ patients, the FLAURA trial demonstrated its superiority in the first-line setting regardless of T790M status. Consequently, AURA3 remains relevant primarily for 'legacy' patients who were started on 1st/2nd generation TKIs, though most new patients now receive osimertinib upfront, rendering the T790M resistance pathway less common in later lines.

Scholarly Review

Critical appraisal through the lens of expert reviewers and guideline development

PhD
PhD

AURA3 permitted crossover from the chemotherapy arm to the osimertinib arm. Evaluate how this design affects the analysis of Overall Survival (OS) and describe a statistical approach to mitigate this confounding variable.

Key Response

Crossover frequently leads to an underestimation of the survival benefit (Type II error) in the experimental arm because control-group patients receive the active drug after progression. To adjust for this, researchers often utilize the Rank-Preserving Structural Failure Time (RPSFT) model or Inverse Probability of Censoring Weighting (IPCW) to estimate a crossover-adjusted hazard ratio for OS.

Journal Editor
Journal Editor

In AURA3, the T790M status was determined by local or central testing using various assays. What concerns might a reviewer raise regarding the lack of a single companion diagnostic for the primary analysis of PFS?

Key Response

The use of heterogeneous testing methods (tissue vs. plasma, varying sensitivity of NGS vs. PCR) can introduce selection bias and variability in the study population. A reviewer would demand a subgroup analysis comparing outcomes between patients identified via liquid biopsy versus tissue biopsy to ensure the treatment effect is robust across different diagnostic thresholds.

Guideline Committee
Guideline Committee

How does AURA3 influence the NCCN and ASCO recommendations regarding the management of EGFR-mutant NSCLC patients who progress without a detectable T790M mutation?

Key Response

AURA3 specifically demonstrated benefit in T790M+ patients; therefore, guidelines (e.g., NCCN Category 1) only recommend osimertinib in the second line for T790M+ disease. If a patient is T790M-negative after both plasma and tissue testing, guidelines recommend platinum-based doublet chemotherapy (the control arm of AURA3), as there is no evidence that osimertinib outperforms chemotherapy in T790M-negative resistance.

Clinical Landscape

Noteworthy Related Trials

2009

IPASS Trial

n = 1,217 · NEJM

Tested

Gefitinib

Population

Advanced NSCLC in never- or light-smokers

Comparator

Carboplatin plus paclitaxel

Endpoint

Progression-free survival

Key result: Gefitinib was superior to chemotherapy for patients with EGFR-mutated tumors, establishing EGFR-TKI as a first-line treatment.
2013

LUX-Lung 3 Trial

n = 345 · Lancet Oncol

Tested

Afatinib

Population

EGFR mutation-positive advanced NSCLC

Comparator

Cisplatin plus pemetrexed

Endpoint

Progression-free survival

Key result: Afatinib significantly improved progression-free survival compared to platinum-based chemotherapy in patients with EGFR mutations.
2018

FLAURA Trial

n = 556 · NEJM

Tested

Osimertinib

Population

Untreated EGFR-mutated advanced NSCLC

Comparator

Erlotinib or Gefitinib

Endpoint

Progression-free survival

Key result: Osimertinib showed significantly longer progression-free survival compared to first-generation EGFR TKIs in the first-line setting.

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