New England Journal of Medicine JULY 14, 2022

Adagrasib in Non-Small-Cell Lung Cancer Harboring a KRASG12C Mutation

Pasi A. Jänne, Gregory J. Riely, Shirish M. Gadgeel, et al.

Bottom Line

In patients with previously treated, KRASG12C-mutated non-small-cell lung cancer, the selective KRASG12C inhibitor adagrasib demonstrated durable clinical activity with a manageable safety profile.

Key Findings

1. The primary objective response rate (ORR) was 43.0% (95% CI, 34.7% to 51.6%) by blinded independent central review.
2. The median duration of response (DOR) was 12.4 months (95% CI, 6.3 to 17.5 months).
3. Median progression-free survival (PFS) was 6.9 months (95% CI, 5.4 to 9.8 months).
4. Median overall survival (OS) was 14.1 months (95% CI, 9.2 to 19.2 months).
5. Disease control rate (DCR) reached 80.0%, indicating stabilization or regression in the majority of patients.

Study Design

Design
Phase 1/2 Clinical Trial
Open-Label
Sample
132
Patients
Duration
15.6 mo
Median
Setting
Multicenter, global
Population Patients with advanced or metastatic non-small-cell lung cancer harboring a KRAS G12C mutation who had previously received platinum-based chemotherapy and anti-PD-(L)1 therapy.
Intervention Adagrasib 600 mg administered orally twice daily.
Comparator None (single-arm study).
Outcome Objective response rate (ORR) as assessed by blinded independent central review.

Study Limitations

Non-randomized, single-arm study design precludes direct comparison to standard-of-care therapies.
The cohort consists of a highly selected, previously treated population, limiting generalizability to first-line settings.
Accelerated approval was based on surrogate endpoints (ORR/DOR), requiring verification through ongoing Phase 3 trials.
Adverse events, including gastrointestinal toxicities and serum creatinine elevation, occurred in a high proportion of patients, necessitating dose modifications.

Clinical Significance

Adagrasib provides a targeted therapeutic option for a historically 'undruggable' mutation, KRASG12C, in NSCLC patients who have progressed on platinum-based chemotherapy and checkpoint inhibitors, addressing a critical area of unmet medical need.

Historical Context

KRAS mutations have long been considered 'undruggable' due to their high affinity for GTP and lack of clear binding pockets; however, the discovery of a druggable cysteine residue in the KRASG12C variant allowed for the development of covalent inhibitors like adagrasib following the initial proof-of-concept established by sotorasib.

Guided Discussion

High-yield insights from every perspective

Med Student
Medical Student

Explain the molecular mechanism by which the KRAS G12C mutation leads to constitutive signaling and how adagrasib specifically interrupts this cycle.

Key Response

The G12C mutation replaces glycine with cysteine, impairing the intrinsic GTPase activity and preventing GAP-mediated hydrolysis, which locks KRAS in the active GTP-bound state. Adagrasib is a covalent inhibitor that binds specifically to the cysteine residue in the switch II pocket of the inactive GDP-bound form, trapping it and preventing its reactivation.

Resident
Resident

A patient with KRAS G12C-mutated NSCLC is starting adagrasib; what are the most common treatment-related adverse events (TRAEs) you must monitor for, and what are the specific criteria for managing hepatotoxicity?

Key Response

The most frequent TRAEs are gastrointestinal (diarrhea, nausea, vomiting) and fatigue. Hepatotoxicity is also common; per the study and prescribing labels, Grade 3 ALT/AST elevations require treatment interruption until recovery to Grade 1 or baseline, followed by a dose reduction or permanent discontinuation if recovery does not occur or recurs.

Fellow
Fellow

Considering the intracranial response data from the KRYSTAL-1 trial, how does the pharmacokinetic profile of adagrasib influence its utility in patients with untreated or progressing CNS metastases compared to other KRAS inhibitors?

Key Response

Adagrasib has demonstrated a high brain-to-plasma concentration ratio and clinically meaningful intracranial activity (intracranial ORR ~33% in sub-studies). This is attributed to its long half-life (~23 hours) and dose-dependent PK which maintains concentrations above the IC50, making it a viable option for patients with CNS involvement where other agents might have limited penetration.

Attending
Attending

In the context of evolving resistance to KRAS G12C inhibition, how should the findings of KRYSTAL-1 influence the sequencing of therapy and the integration of repeat molecular profiling at the time of progression?

Key Response

While adagrasib is effective, resistance inevitably develops through bypass signaling (e.g., MET amplification) or secondary KRAS mutations (e.g., Y96D). KRYSTAL-1 establishes a benchmark for second-line efficacy, but the high rate of acquired resistance necessitates repeat biopsy or liquid biopsy at progression to determine if a patient is eligible for combination trials or should transition to docetaxel-based regimens.

Scholarly Review

Critical appraisal through the lens of expert reviewers and guideline development

PhD
PhD

Critique the pharmacodynamic rationale for the 600 mg twice-daily dosing of adagrasib used in KRYSTAL-1 versus a once-daily approach, specifically regarding the saturation of the KRAS protein synthesis-degradation cycle.

Key Response

KRAS G12C inhibitors only bind to the GDP-bound (inactive) state. Because new KRAS protein is synthesized continuously, twice-daily dosing maintains steady-state plasma concentrations that exceed the IC50 throughout the entire 24-hour interval, ensuring that newly synthesized KRAS is inhibited immediately upon entering the GDP-bound state, thereby maximizing target occupancy compared to once-daily dosing.

Journal Editor
Journal Editor

While the KRYSTAL-1 Phase 2 cohort showed an objective response rate of 43%, what limitations of this single-arm study design might a reviewer flag when assessing the generalizability of the survival outcomes compared to the current standard of care?

Key Response

Reviewers would flag the lack of a randomized control arm (e.g., vs. Docetaxel), which makes it difficult to account for selection bias in a heavily pre-treated population (median of 2 prior lines). Additionally, the use of a surrogate endpoint (ORR) as the primary outcome requires subsequent Phase 3 validation (like KRYSTAL-12) to confirm that these responses translate into a statistically significant improvement in overall survival.

Guideline Committee
Guideline Committee

How do the results of KRYSTAL-1 compare to the efficacy of Docetaxel plus Ramucirumab, and should current guidelines be updated to recommend adagrasib as the preferred second-line agent for KRAS G12C mutated NSCLC?

Key Response

The ORR for adagrasib (43%) and median PFS (6.5 months) compare favorably to the historical outcomes for docetaxel + ramucirumab (ORR ~23%, PFS ~4.5 months). NCCN guidelines currently list adagrasib as a preferred subsequent therapy (Category 2A). Full endorsement as the definitive 'preferred' agent often awaits Phase 3 head-to-head data, but the superior response rate and manageable safety profile already support its use over traditional chemotherapy in this molecular subset.

Clinical Landscape

Noteworthy Related Trials

2021

CodeBreaK 100 Trial

n = 126 · NEJM

Tested

Sotorasib 960 mg daily

Population

Patients with KRAS G12C-mutated NSCLC previously treated with chemotherapy or PD-1/PD-L1 inhibitors

Comparator

None (single-arm study)

Endpoint

Objective response rate (ORR)

Key result: Sotorasib demonstrated durable clinical activity with an objective response rate of 37.1% and a manageable safety profile.
2023

CodeBreaK 200 Trial

n = 345 · Lancet

Tested

Sotorasib 960 mg daily

Population

Patients with KRAS G12C-mutated NSCLC previously treated with platinum-based chemotherapy and PD-1/PD-L1 inhibitors

Comparator

Docetaxel

Endpoint

Progression-free survival (PFS)

Key result: Sotorasib significantly improved progression-free survival compared to docetaxel, showing a hazard ratio of 0.66.
2024

KRYSTAL-12 Trial

n = 453 · JCO

Tested

Adagrasib 600 mg twice daily

Population

Patients with KRAS G12C-mutated NSCLC previously treated with platinum-based chemotherapy and immunotherapy

Comparator

Docetaxel

Endpoint

Progression-free survival (PFS)

Key result: Adagrasib showed a statistically significant and clinically meaningful improvement in progression-free survival and objective response rate compared to docetaxel.

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