^
    3d
    MIRV: Phase 1/2 Study of Mirdametinib + Vinblastine for Newly Diagnosed/Previously Untreated PLGG + Activation of MAPK (clinicaltrials.gov)
    P1/2, N=50, Recruiting, St. Justine's Hospital | Not yet recruiting --> Recruiting
    Enrollment open
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    BRAF (B-raf proto-oncogene) • NF1 (Neurofibromin 1) • KIAA1549
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    BRAF V600E • BRAF V600 • BRAF fusion
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    Gomekli (mirdametinib) • vinblastine
    12d
    A Window of Opportunity Trial of Mirdametinib Plus Vorinostat for NF1 Associated, H3K27 Trimethylation Deficient Malignant Peripheral Nerve Sheath Tumor [MPNST] (clinicaltrials.gov)
    P1, N=8, Recruiting, University of Minnesota | Trial completion date: Jan 2026 --> Jan 2027 | Trial primary completion date: Jan 2026 --> Jan 2027
    Trial completion date • Trial primary completion date
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    Gomekli (mirdametinib) • Zolinza (vorinostat)
    12d
    Study of the Safety and Pharmacokinetics of BGB-283 (Lifirafenib) and PD-0325901 (Mirdametinib) in Participants With Advanced or Refractory Solid Tumors (clinicaltrials.gov)
    P1, N=91, Completed, BeiGene | Trial completion date: Feb 2026 --> Oct 2025 | Active, not recruiting --> Completed
    Trial completion • Trial completion date
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    NRAS (Neuroblastoma RAS viral oncogene homolog)
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    NRAS mutation
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    Gomekli (mirdametinib) • lifirafenib (BGB-283)
    13d
    Head-to-head preclinical treatment design prioritizes promising therapies for neurofibromatosis type 1 optic glioma clinical translation. (PubMed, Neurooncol Adv)
    Nf1 OPG mice were treated with standard of care (SOC; carboplatin), clinically evaluated (everolimus, mirdametinib), and investigational (pexidartinib, HBS-101, lamotrigine) drugs during the period of most rapid tumor growth (6-12 weeks of age). This referential preclinical study design affords direct head-to-head comparisons of investigational therapies relative to SOC treatment using clinically meaningful outcomes (OPG growth and RNFL thickness). Using this strategy, lamotrigine emerged as the most promising therapy for limiting tumor progression and vision loss in Nf1-OPG mice, relevant to clinical translation for children with NF1-OPG.
    Preclinical • Journal • Head-to-Head
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    NF1 (Neurofibromin 1) • CCL2 (Chemokine (C-C motif) ligand 2) • CCL3 (C-C Motif Chemokine Ligand 3)
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    carboplatin • everolimus • Gomekli (mirdametinib) • Turalio (pexidartinib)
    14d
    High-throughput drug screening identifies EGFR/MAPK pathway targeting sensitivities in organoid models of ovarian carcinosarcoma. (PubMed, J Exp Clin Cancer Res)
    OCS is the most aggressive, drug-resistant gynaecological malignancy and eribulin-based combination therapies, particularly triple combination therapies, have the potential to improve patient outcomes.
    Journal
    |
    KRAS (KRAS proto-oncogene GTPase) • TP53 (Tumor protein P53) • MYC (V-myc avian myelocytomatosis viral oncogene homolog) • MYCN (MYCN Proto-Oncogene BHLH Transcription Factor) • ABCB1 (ATP Binding Cassette Subfamily B Member 1) • HMGA2 (High mobility group AT-hook 2)
    |
    TP53 mutation • KRAS mutation • EGFR mutation
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    cisplatin • erlotinib • Halaven (eribulin mesylate) • Gomekli (mirdametinib)
    24d
    Testing the Effectiveness of the Anti-cancer Drug, Mirdametinib, in Treating Relapsed, Refractory Chronic Lymphocytic Leukemia (clinicaltrials.gov)
    P2, N=20, Recruiting, National Cancer Institute (NCI) | Not yet recruiting --> Recruiting | Initiation date: Jan 2026 --> Sep 2026
    Enrollment open • Trial initiation date
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    Gomekli (mirdametinib)
    1m
    To Assess the Enzyme Inducing Effects of Carbamazepine on the PK of Mirdametinib in Healthy Participants (clinicaltrials.gov)
    P1, N=36, Not yet recruiting, SpringWorks Therapeutics, Inc.
    New P1 trial
    |
    Gomekli (mirdametinib)
    1m
    Testing the Effectiveness of the Anti-cancer Drug, Mirdametinib, in Treating Relapsed, Refractory Chronic Lymphocytic Leukemia (clinicaltrials.gov)
    P2, N=20, Not yet recruiting, National Cancer Institute (NCI) | Trial completion date: Dec 2027 --> Feb 2030 | Trial primary completion date: Dec 2027 --> Feb 2030
    Trial completion date • Trial primary completion date
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    Gomekli (mirdametinib)
    1m
    Testing the Effectiveness of the Anti-cancer Drug, Mirdametinib, in Treating Relapsed, Refractory Chronic Lymphocytic Leukemia (clinicaltrials.gov)
    P2, N=20, Not yet recruiting, National Cancer Institute (NCI) | Initiation date: Oct 2025 --> Jan 2026
    Trial initiation date
    |
    Gomekli (mirdametinib)
    1m
    Systematic pan-cancer analysis reveals the prognostic and immunological roles of ectonucleoside triphosphate diphosphohydrolase 6. (PubMed, World J Clin Oncol)
    This pan-cancer study elucidates the pivotal role of ENTPD6 in tumor progression and establishes its potential as a therapeutic target for immunotherapeutic approaches in specific malignancies.
    Journal • Tumor mutational burden • IO biomarker • Pan tumor
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    TMB (Tumor Mutational Burden) • MSI (Microsatellite instability)
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    Koselugo (selumetinib) • Gomekli (mirdametinib) • refametinib (BAY86-9766)
    2ms
    Acute Myeloid Leukemia Relapse after Bromodomain Inhibitor Treatment or Chemotherapy is Characterized by Myc-Ras Transcriptional Remodeling. (PubMed, bioRxiv)
    Here we show that the BET inhibitor PLX51107 potently suppresses the growth of NRAS -mutant AML cell lines, and that these activities are enhanced by co-treatment with the MEK inhibitor PD0325901. AMLs that relapsed after frontline chemotherapy showed similar transcriptional remodeling. These studies demonstrate transcriptional plasticity in primary AMLs that relapse following in vivo treatment with either targeted agents or chemotherapy, and support evaluating BET inhibition in leukemias with monocytic differentiation and RAS mutations.
    Journal
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    NRAS (Neuroblastoma RAS viral oncogene homolog) • MYC (V-myc avian myelocytomatosis viral oncogene homolog)
    |
    NRAS mutation • RAS mutation
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    Gomekli (mirdametinib) • PLX51107