bioRxiv Science⌕ Search

bioRxiv · 10.64898/2026.02.18.706707

Mutation-Resolved Drug Sensitivity Atlas Reveals Broad RAS(ON) Inhibitor Vulnerabilities and a STAT3 Co-Dependency in NRAS-Mutant Melanoma

Abstract

NRAS-mutated melanoma remains a major unmet clinical need, with no approved targeted therapy and rapid progression on standard treatment. Tri-complex RAS(ON) inhibitors such as daraxonrasib (RMC-6236) and RMC-7977 have shown early clinical activity, but the mutation-specific sensitivity landscape and adaptive resistance programs in melanoma remain undefined. To address this, we generated an isogenic 3D melanoma platform and performed a saturation mutagenesis screen across 95 NRAS missense variants (>99% of clinically recurrent variants), profiling oncogenic fitness and responses to six RAS-targeting agents in spheroids and xenografts. RMC-6236 and RMC-7977 showed the broadest activity and stratified recurrent NRAS mutants into hypersensitive (G12 variants and Q61R/K/L; [~]95% of cases), moderately sensitive (G13D/R/V; [~]4%), and resistant (G60E and Q61P; [~]1%) classes. Structural analyses supported distinct mechanisms underlying reduced susceptibility in a restricted subset of variants. In sensitive genotypes, RAS(ON) inhibition elicited an adaptive cytokine- and RTK-associated survival program converging on STAT3. Co-inhibition of STAT3 enhanced apoptosis, suppressed MYC, and induced tumor regression in NRAS-mutant melanoma models. Together, these findings define a mutation-resolved therapeutic landscape for NRAS-mutant melanoma and identify adaptive STAT3 signaling as a rational target for combination therapy. Statement of Translational SignificanceNRAS-mutated melanoma lacks effective targeted treatments, and clinical responses to immunotherapy are suboptimal. This study presents the first comprehensive drug sensitivity map across 95 NRAS mutations in melanoma, identifying the pan-RAS(ON) inhibitors RMC-6236 and RMC-7977 as broadly effective agents. Multiple mutants with reduced susceptibility are identified, providing mutation-informed guidance for patient selection and clinical trial stratification. Mechanistic analyses reveal that RTK/cytokine-driven STAT3 activation functions as a key survival pathway under RAS(ON) blockade, and its inhibition markedly enhances the efficacy of pan-RAS(ON) inhibitors. These findings support mutation-guided use of RAS(ON) inhibition and highlight STAT3 co-targeting as a rational strategy to strengthen and prolong therapeutic responses in NRAS-mutated melanoma. Highlights- A functional and therapeutic atlas defines 95 recurrent and nonrecurrent NRAS missense variants in melanoma - RMC-6236 and RMC-7977 show broad but genotype-selective activity across major NRAS mutations - A restricted subset of recurrent NRAS mutants shows reduced susceptibility to RAS(ON) inhibition - RAS(ON) inhibition induces an adaptive STAT3 survival program that is therapeutically targetable

Explore related subjects

Keep this discovery

Explore connections, maps & timelines

BibTeXRIS

Yeung, S. F., Chen, J. X., Law, C. T. Y., Law, A. C. H., Lee, C., Leung, A. M. F., Chau, M. P. K., Ko, B. C.-B., Wu, Y., Liang, K., Cho, W. C., Siu, M. K. Y., Chan, K. K. L., Tsui, S. K. W.. 2026-02-20. Mutation-Resolved Drug Sensitivity Atlas Reveals Broad RAS(ON) Inhibitor Vulnerabilities and a STAT3 Co-Dependency in NRAS-Mutant Melanoma. https://doi.org/10.64898/2026.02.18.706707

Cite the original work for its findings. Save a collection to share your selection of sources.

KEEP EXPLORING

Related preprints

EWSR1::ATF1 hyperactivation via TRIM11 depletion triggers lethal metabolic rewiring in clear cell sarcoma

Clear cell sarcoma (CCS) is an ultra-rare and aggressive soft tissue sarcoma driven by the EWSR1::ATF1 fusion oncoprotein. Currently, no effective and well-tolerated treatment options are available to CCS patients. Direct inhibition of fusion transcription factors is precluded by the absence of defined ligand-binding pockets, whereas their abundance is under the control of the ubiquitin-proteasome system (UPS), offering an alternative avenue for biology-guided treatment. Here we show the abundance of EWSR1::ATF1 is tightly regulated by the UPS, and that release of this constraint is lethal to CCS cells. A reporter-based CRISPR/Cas9 knockout screen of UPS components identified TRIM11 as an E3 ligase that regulates turnover of EWSR1::ATF1, which it recognises as a neosubstrate without altering wild-type EWSR1 or ATF1. Depletion of TRIM11 increased nuclear fusion protein abundance, amplified the fusion-driven transcriptional programs and reduced viability. Proteomic analysis identified the cataplerotic enzyme phosphoenolpyruvate carboxykinase 1 (PCK1), a direct EWSR1::ATF1 target, as the key mediator of lethal effects of fusion hyperactivation. Extracellular flux analysis, isotope tracing and quantitative metabolomics revealed excessive cataplerotic carbon withdrawal and impaired oxidative phosphorylation as the underlying mechanism. In patient tumors, high PCK1 expression associated with metastatic transcriptome programs and disease presentation, as well as shorter survival. Taken together, we report that hyperactivation of EWSR1::ATF1 opens a metabolic vulnerability, establishing TRIM11 as an actionable dependency and PCK1 as a candidate prognostic biomarker in CCS.

cancer biology↗

Glycolysis and interferon transcription in melanoma immunotherapy

Glycolysis and interferon signalling have both been associated with responses to PD-1 blockade, but their joint transcriptional relationship with treatment outcome remains uncertain. We examined whether a state with high glycolysis and low interferon- response was associated with reduced disease control in melanoma. Fixed Hallmark gene sets were scored by single-sample gene set enrichment analysis in GSE91061, GSE78220 and an additional Liu cohort. Disease control analyses included 47, 26 and 100 patients. Conditional odds ratios for the joint state were 6.06 (exact 95% confidence interval, 1.04 to 65.61), 0.35 (0.026 to 3.11) and 1.03 (0.39 to 2.73), respectively. Holm-adjusted P values for the two initial comparisons were 0.073 and 0.365. The additional exploratory P value was 1.000. Continuous models and analyses that omitted each patient in turn did not provide consistent evidence of an adverse association. IFN scores were negatively correlated with DNA-derived tumour purity in 102 patients (Spearman {rho} = -0.671). Among 40 patients with paired biopsies, disease control was associated with higher on-treatment IFN scores after baseline adjustment, although the paired subset had lower baseline glycolysis than patients without paired profiles. In patient-level single-cell analyses, the baseline CD8 T cell correlation weakened when an alternative rank score used a common gene background. The defined joint state was not consistently associated with poorer disease control across cohorts. Bulk IFN scores were related to tumour purity, and cellular correlations were sensitive to scoring choices. These transcriptomic results do not measure pyruvate concentration, glycolytic flux or STAT1 pyruvylation.

cancer biology↗

Psychoneuroimmunology of Viral Hepatitis and Hepatocellular Carcinoma: A Systematic Review and Meta-Analysis

Hepatocellular carcinoma (HCC) and viral hepatitis (VH) are recognized primarily as hepatic diseases, yet they impose a substantial neuropsychiatric disorders (NPDs) encompassing depression, anxiety, cognitive impairment, and fatigue. Whether these manifestations reflect psychological reactions or biologically grounded psychoneuroimmune dysfunction remains poorly understood. Here, we present a pre-registered systematic review and meta-analysis (PROSPERO 2026 CRD420251081757) synthesizing evidence from 24 studies combining psychosocial outcomes with immune dysregulation markers across HCC and VH populations. Bibliometric and network analyses identified a growing psychoneuroimmunology research hub linking cytokine signaling, microglial activation, blood-brain barrier (BBB) disruption, and gut-liver-brain axis dysregulation to the neuropsychiatric phenotype of these diseases. Meta-analysis revealed a pooled hazard ratio (HR) of 1.92 (95% CI: 1.09-3.39) for adverse outcomes in HCC and a pooled HR of 2.28 (95% CI: 1.48-3.52) for depression associated to genetic variants in VH. TNF-a, but not IL-6, showed a tendency toward lower levels in VH patients with versus without NPDs, suggesting differential inflammatory roles. The literature, however, remains fragmented by heterogeneous designs and a paucity of longitudinal data. A psychoneuroimmunological approach, integrating immune, endocrine, neural, and psychosocial dimensions, is essential for capturing the full systemic impact of HCC and VH and should inform future mechanistic research and multidisciplinary clinical strategies.

cancer biology↗