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    DRUG CLASS:

    SMO protein inhibitor

    Related drugs:
    2ms
    Dual Targeting of Smoothened, a Key Regulator in the Hedgehog Pathway, and BCR-ABL1 Effectively Eradicates Drug-Insensitive Stem/Progenitor Cells in Chronic Myeloid Leukemia. (PubMed, Cells)
    Inhibition of SMO through genetic knockdown or with a potent, selective SMO inhibitor, Glasdegib, reduces the survival of cells from nonresponder patients. Notably, SMO inhibition also sensitizes TKI-nonresponder stem/progenitor cells to Bostutinib, a second-generation TKI, both in vitro and in a patient-derived xenotransplantation (PDX) model. These findings present a promising therapeutic target and a model for curative combination therapies in stem-cell-driven cancers.
    Journal
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    ABL1 (ABL proto-oncogene 1) • BCR (BCR Activator Of RhoGEF And GTPase) • CD34 (CD34 molecule) • GLI2 (GLI Family Zinc Finger 2)
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    Daurismo (glasdegib)
    3ms
    Efficacy and Safety of Patidegib Gel 2% for Preventing Basal Cell Carcinomas on the Face of Adults With Gorlin Syndrome (clinicaltrials.gov)
    P3, N=140, Active, not recruiting, Sol-Gel Technologies, Ltd. | Recruiting --> Active, not recruiting | Trial completion date: Dec 2025 --> Aug 2026 | Trial primary completion date: Dec 2025 --> Aug 2026
    Enrollment closed • Trial completion date • Trial primary completion date
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    PTCH1 (Patched 1)
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    patidegib topical (SGT-610)
    6ms
    A Phase 2 Trial of ENV-101 in Patients With Lung Fibrosis (WHISTLE-PF Trial) (clinicaltrials.gov)
    P2, N=200, Recruiting, Endeavor Biomedicines, Inc. | Trial completion date: Jun 2026 --> Oct 2026 | Trial primary completion date: Jun 2026 --> Oct 2026
    Trial completion date • Trial primary completion date
    |
    taladegib (ENV 101)
    7ms
    Smoothened Inhibitor, PF-04449913 Inhibits the Development of Myelofibrosis in a JAK2V617F Transgenic Mouse Model by Reducing TGF-β and MAPK Signaling Pathways. (PubMed, Res Sq)
    Treatment of JAK2V617F driven myeloproliferative neoplasms (MPNs) with Ruxolitinib (JAK inhibitor, JAKi) has shown limited disease-modifying benefits and has led to the search for other pathways as potential therapeutic targets for this disease. Finally, we found that SMO inhibitor blocks bone marrow fibrosis by reducing TGF-β signaling. In conclusion, this report provides critical insight into the mechanism of action of SMO inhibitors in JAK2V617F associated MPN.
    Preclinical • Journal
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    TGFB1 (Transforming Growth Factor Beta 1)
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    Jakafi (ruxolitinib) • Daurismo (glasdegib)
    1year
    A Phase 2 Trial of ENV-101 in Patients With Lung Fibrosis (WHISTLE-PF Trial) (clinicaltrials.gov)
    P2, N=200, Recruiting, Endeavor Biomedicines, Inc. | Not yet recruiting --> Recruiting | N=320 --> 200
    Enrollment open • Enrollment change
    |
    taladegib (ENV 101)
    1year
    Phase 2 Trial of Topical Application of the Hedgehog Inhibitor Patidegib in Patients With Gorlin Syndrome. (PubMed, Br J Dermatol)
    Patidegib Topical Gel warrants further clinical development.
    P2 data • Journal
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    PTCH1 (Patched 1)
    |
    PTCH1 mutation
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    cyclopamine • saridegib oral (IPI 926 oral) • patidegib topical (SGT-610)
    over1year
    CPX-351 and Glasdegib for Newly Diagnosed Acute Myelogenous Leukemia With MDS Related Changes or Therapy-related Acute Myeloid Leukemia (clinicaltrials.gov)
    P2, N=30, Active, not recruiting, University of California, Irvine | Recruiting --> Active, not recruiting | Trial completion date: Sep 2025 --> Jun 2027 | Trial primary completion date: Jun 2024 --> Jun 2025
    Enrollment closed • Trial completion date • Trial primary completion date
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    NPM1 (Nucleophosmin 1) • CEBPA (CCAAT Enhancer Binding Protein Alpha)
    |
    Vyxeos (cytarabine/daunorubicin liposomal formulation) • Daurismo (glasdegib)
    over1year
    Late-onset NPM1 mutation in a MYC-amplified relapsed / refractory acute myeloid leukemia patient treated with gemtuzumab ozogamicin and glasdegib. (PubMed, Haematologica)
    Not available.
    Journal
    |
    MYC (V-myc avian myelocytomatosis viral oncogene homolog) • NPM1 (Nucleophosmin 1)
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    Mylotarg (gemtuzumab ozogamicin) • Daurismo (glasdegib)
    over1year
    A Phase 2 Trial of ENV-101 in Patients With Lung Fibrosis (WHISTLE-PF Trial) (clinicaltrials.gov)
    P2, N=320, Not yet recruiting, Endeavor Biomedicines, Inc.
    New P2 trial
    |
    taladegib (ENV 101)
    almost2years
    A Study Evaluating the Safety and Efficacy of ENV-101 in Subjects With Idiopathic Pulmonary Fibrosis (IPF) (clinicaltrials.gov)
    P2, N=41, Completed, Endeavor Biomedicines, Inc. | Active, not recruiting --> Completed
    Trial completion
    |
    taladegib (ENV 101)
    almost2years
    Efficacy and Safety of Patidegib Gel 2% for Preventing Basal Cell Carcinomas on the Face of Adults With Gorlin Syndrome (clinicaltrials.gov)
    P3, N=140, Recruiting, Sol-Gel Technologies, Ltd. | Not yet recruiting --> Recruiting
    Enrollment open
    |
    PTCH1 (Patched 1)
    |
    PTCH1 mutation
    |
    patidegib topical (SGT-610)
    almost2years
    BRIGHT 1012: A Combination Study of PF-04449913 (Glasdegib) and Azacitidine In Untreated MDS, AML and CMML Patients (clinicaltrials.gov)
    P1, N=73, Completed, Pfizer | Phase classification: P1b --> P1
    Phase classification • Combination therapy
    |
    azacitidine • Daurismo (glasdegib)