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    DRUG:

    Erivedge (vismodegib)

    i
    Other names: GDC 0449, GDC-0449, GDC0449, R 3616, R3616, RG3616, RG 3616, RO5450815
    Company:
    Curis, Roche
    Drug class:
    Hedgehog cell-signalling pathway inhibitor
    Related drugs:
    20d
    A novel TCGA-validated programmed cell-death-related signature of ovarian cancer. (PubMed, BMC Cancer)
    Our model could precisely predict the prognosis, immune status and drug sensitivity of OC patients.
    Journal • Tumor mutational burden • PD(L)-1 Biomarker • IO biomarker
    |
    PD-L1 (Programmed death ligand 1) • TMB (Tumor Mutational Burden) • RB1 (RB Transcriptional Corepressor 1) • PD-1 (Programmed cell death 1) • LAG3 (Lymphocyte Activating 3) • IDO1 (Indoleamine 2,3-dioxygenase 1) • CLTC (Clathrin Heavy Chain) • IGF2BP1 (Insulin Like Growth Factor 2 MRNA Binding Protein 1) • OGG1 (8-Oxoguanine DNA glycosylase) • PPP1R15A (Protein Phosphatase 1 Regulatory Subunit 15A) • UBR5 (Ubiquitin Protein Ligase E3 Component N-Recognin 5) • CD3E (CD3 Epsilon Subunit Of T-Cell Receptor Complex)
    |
    cisplatin • gefitinib • paclitaxel • Erivedge (vismodegib)
    1m
    Testing GDC-0449 (Vismodegib) as Potentially Targeted Treatment in Cancers With Smoothened or Patched 1 Mutant Tumors (MATCH - Subprotocol T) (clinicaltrials.gov)
    P2, N=35, Active, not recruiting, National Cancer Institute (NCI)
    New P2 trial
    |
    Erivedge (vismodegib)
    1m
    To Assess the Safety and Efficacy of SP-002 With Vismodegib for the Treatment of Locally Advanced Basal Cell Carcinoma (clinicaltrials.gov)
    P2, N=80, Recruiting, Stamford Pharmaceuticals, Inc. | Not yet recruiting --> Recruiting
    Enrollment open • Metastases
    |
    Erivedge (vismodegib)
    1m
    To Assess the Safety and Efficacy of SP-002 With Vismodegib for the Treatment of Locally Advanced Basal Cell Carcinoma (clinicaltrials.gov)
    P2, N=80, Not yet recruiting, Stamford Pharmaceuticals, Inc.
    New P2 trial
    |
    Erivedge (vismodegib)
    2ms
    GDC-0449 and Erlotinib Hydrochloride With or Without Gemcitabine Hydrochloride in Treating Patients With Metastatic Pancreatic Cancer or Solid Tumors That Cannot Be Removed by Surgery (clinicaltrials.gov)
    P1, N=55, Active, not recruiting, National Cancer Institute (NCI)
    Trial completion date • Surgery • Metastases
    |
    BCL2 (B-cell CLL/lymphoma 2)
    |
    erlotinib • gemcitabine • Erivedge (vismodegib)
    2ms
    A sterol analog inhibits hedgehog pathway by blocking cholesterylation of smoothened. (PubMed, Cell Chem Biol)
    Q29 exhibits an additive inhibitory effect on medulloblastoma with vismodegib, a clinically used SMO-7TM inhibitor for treating basal cell carcinoma (BCC). Importantly, Q29 overcomes resistance caused by SMO mutants against SMO-7TM inhibitors and inhibits the activity of SMO oncogenic variants. Our work demonstrates that the SMO-CRD inhibitor can be a new way to treat Hh pathway-driven cancers.
    Journal
    |
    SMO (Smoothened Frizzled Class Receptor)
    |
    SMO mutation
    |
    Erivedge (vismodegib)
    2ms
    Plasma ctDNA Monitoring of a PTCH1-Mutant Metastatic Adult Medulloblastoma Showing a Durable Benefit With Vismodegib. (PubMed, Oncologist)
    Several small studies demonstrate objective but short-lived responses to SMO inhibitors such as vismodegib or sonidegib. We present the case of a 26-year-old patient with a recurrent MB, in which next-generation sequencing (FoundationOne CDx) revealed a mutation in PTCH1, allowing the patient to be treated with vismodegib in second line, resulting in a durable benefit lasting for 1 year. Using an in-house digital PCR probe, the PTCH1 mutation could be tracked in ctDNA during treatment with first-line chemotherapy and while on treatment with vismodegib, demonstrating a precise correlation with the radiological and clinical behavior of the disease.
    Journal • Circulating tumor DNA • Metastases
    |
    TP53 (Tumor protein P53) • TERT (Telomerase Reverse Transcriptase) • PTCH1 (Patched 1) • SMO (Smoothened Frizzled Class Receptor)
    |
    TP53 wild-type • PTCH1 mutation • SMO mutation
    |
    FoundationOne® CDx
    |
    Erivedge (vismodegib) • Odomzo (sonidegib)
    2ms
    Serial Measurements of Molecular and Architectural Responses to Therapy (SMMART) PRIME Trial (clinicaltrials.gov)
    P1, N=2, Terminated, OHSU Knight Cancer Institute | Active, not recruiting --> Terminated; Low accrual
    Trial termination
    |
    Keytruda (pembrolizumab) • Opdivo (nivolumab) • Avastin (bevacizumab) • Venclexta (venetoclax) • Lynparza (olaparib) • Mekinist (trametinib) • erlotinib • Gilotrif (afatinib) • Yervoy (ipilimumab) • Ibrance (palbociclib) • dasatinib • Zelboraf (vemurafenib) • Tafinlar (dabrafenib) • carboplatin • Imfinzi (durvalumab) • sorafenib • Rozlytrek (entrectinib) • imatinib • sunitinib • everolimus • Nerlynx (neratinib) • Iclusig (ponatinib) • Kadcyla (ado-trastuzumab emtansine) • Cotellic (cobimetinib) • Lorbrena (lorlatinib) • Lenvima (lenvatinib) • bortezomib • doxorubicin hydrochloride • capecitabine • Verzenio (abemaciclib) • Xtandi (enzalutamide capsule) • Cabometyx (cabozantinib tablet) • albumin-bound paclitaxel • Stivarga (regorafenib) • abiraterone acetate • oxaliplatin • Aliqopa (copanlisib) • Vizimpro (dacomitinib) • Zydelig (idelalisib) • daunorubicin • Zolinza (vorinostat) • Idhifa (enasidenib) • Farydak (panobinostat) • Erivedge (vismodegib) • Nubeqa (darolutamide) • bicalutamide • leucovorin calcium • cabazitaxel • Vesanoid (tretinoin) • fluorouracil topical
    2ms
    Beyond cyclopamine: Targeting Hedgehog signaling for cancer intervention. (PubMed, Arch Biochem Biophys)
    Inhibiting Hh signaling is an important oncological strategy in which inhibitors of the ligands SMO or GLI have been looked at. This review briefly narrates the Hh ligands, signal transduction, the target genes involved and comprehensively describes the numerous inhibitors that have been evaluated for use in various neoplastic settings.
    Review • Journal
    |
    GLI1 (GLI Family Zinc Finger 1)
    |
    Erivedge (vismodegib) • Odomzo (sonidegib) • cyclopamine
    3ms
    IMPRESS-Norway: Improving Public Cancer Care by Implementing Precision Medicine in Norway (clinicaltrials.gov)
    P2, N=3000, Recruiting, Oslo University Hospital | N=1000 --> 3000
    Enrollment change
    |
    Avastin (bevacizumab) • Herceptin (trastuzumab) • Tecentriq (atezolizumab) • Zelboraf (vemurafenib) • Rozlytrek (entrectinib) • Alecensa (alectinib) • Perjeta (pertuzumab) • Zejula (niraparib) • Zykadia (ceritinib) • Jemperli (dostarlimab-gxly) • Erivedge (vismodegib) • Phesgo (pertuzumab/trastuzumab/hyaluronidase-zzxf)
    3ms
    My Pathway: A Study Evaluating Herceptin/Perjeta, Tarceva, Zelboraf/Cotellic, Erivedge, Alecensa, and Tecentriq Treatment Targeted Against Certain Molecular Alterations in Participants With Advanced Solid Tumors (clinicaltrials.gov)
    P2, N=673, Completed, Genentech, Inc. | Phase classification: P2a --> P2
    Phase classification
    |
    EGFR (Epidermal growth factor receptor) • HER-2 (Human epidermal growth factor receptor 2) • BRAF (B-raf proto-oncogene) • ALK (Anaplastic lymphoma kinase) • TMB (Tumor Mutational Burden) • PTCH1 (Patched 1) • SMO (Smoothened Frizzled Class Receptor)
    |
    EGFR mutation • HER-2 overexpression • BRAF mutation • HER-2 amplification • BRAF V600 • ALK rearrangement • ALK mutation • SMO mutation
    |
    Herceptin (trastuzumab) • Tecentriq (atezolizumab) • erlotinib • Zelboraf (vemurafenib) • Alecensa (alectinib) • Perjeta (pertuzumab) • Cotellic (cobimetinib) • Erivedge (vismodegib)
    3ms
    Ablative fractional laser treatment reduces hedgehog pathway gene expression in murine basal cell carcinomas. (PubMed, Lasers Med Sci)
    In conclusion, a single AFL treatment significantly reduced hedgehog gene expression in murine BCCs mimicking the effects of eight topical applications of vismodegib. Further studies are needed to assess whether AFL can be utilized for BCC treatment, either as monotherapy or in combination with other drugs.
    Preclinical • Journal
    |
    PTCH1 (Patched 1) • GLI1 (GLI Family Zinc Finger 1) • GLI2 (GLI Family Zinc Finger 2)
    |
    GLI1 expression
    |
    Erivedge (vismodegib)
    3ms
    Laser-assisted topical delivery of vismodegib reduces hedgehog gene expression in human basal cell carcinomas in vivo. (PubMed, Lasers Surg Med)
    A single AFL-assisted topical application of vismodegib resulted in clinically relevant intratumoral drug concentrations and significant reductions in hedgehog pathway gene expressions.
    Preclinical • Journal
    |
    PTCH1 (Patched 1) • GLI1 (GLI Family Zinc Finger 1) • GLI2 (GLI Family Zinc Finger 2)
    |
    GLI1 expression
    |
    Erivedge (vismodegib) • Odomzo (sonidegib)
    3ms
    Photobiomodulation Recovers the Submandibular Gland in Vismodegib-Treated Rats. (PubMed, Photobiomodul Photomed Laser Surg)
    Also, 850 nm high-power LED recovered the damaged structure of SMG and increased SHH-related proteins and salivary functional markers. The study results suggest that PBM can restore SMG structure and function through SHH signaling.
    Preclinical • Journal
    |
    PTCH1 (Patched 1) • GLI1 (GLI Family Zinc Finger 1) • KRT5 (Keratin 5) • SHH (Sonic Hedgehog Signaling Molecule)
    |
    Erivedge (vismodegib)
    4ms
    Dynamic optical coherence tomography evaluation in locally advanced basal cell carcinoma during sonidegib treatment. (PubMed, J Eur Acad Dermatol Venereol)
    Sonidegib can be considered an effective treatment option in cases where surgery or radiotherapy would be unfeasible or has previously failed, although pigmented lesions did not show complete clearance, suggesting that there are factors other than the SHH pathway involved in tumour growth. Videodermoscopy and D-OCT were useful in the quick and seamless follow-up of lesions and added valuable information in assessing efficacy.
    Journal • Metastases
    |
    PTCH1 (Patched 1) • SMO (Smoothened Frizzled Class Receptor)
    |
    PTCH1 mutation • SMO mutation
    |
    Erivedge (vismodegib) • Odomzo (sonidegib)
    4ms
    SMMART Adaptive Clinical Treatment (ACT) Trial (clinicaltrials.gov)
    P1, N=0, Withdrawn, OHSU Knight Cancer Institute | N=25 --> 0 | Not yet recruiting --> Withdrawn
    Enrollment change • Trial withdrawal
    |
    HER-2 (Human epidermal growth factor receptor 2) • BRCA1 (Breast cancer 1, early onset) • BRCA2 (Breast cancer 2, early onset)
    |
    BRCA2 mutation • BRCA1 mutation • HR positive • HER-2 negative • EGFR positive
    |
    Avastin (bevacizumab) • Herceptin (trastuzumab) • Lynparza (olaparib) • Tecentriq (atezolizumab) • Ibrance (palbociclib) • Zelboraf (vemurafenib) • carboplatin • Rozlytrek (entrectinib) • Alecensa (alectinib) • Kadcyla (ado-trastuzumab emtansine) • Cotellic (cobimetinib) • capecitabine • Piqray (alpelisib) • Zejula (niraparib) • albumin-bound paclitaxel • fulvestrant • irinotecan • Halaven (eribulin mesylate) • letrozole • vinorelbine tartrate • Herzuma (trastuzumab-pkrb) • anastrozole • Erivedge (vismodegib) • Trazimera (trastuzumab-qyyp) • Phesgo (pertuzumab/trastuzumab/hyaluronidase-zzxf)
    4ms
    A Clinical and Molecular Risk-Directed Therapy for Newly Diagnosed Medulloblastoma (clinicaltrials.gov)
    P2, N=660, Active, not recruiting, St. Jude Children's Research Hospital | Trial primary completion date: Jan 2025 --> Sep 2024
    Trial primary completion date
    |
    cisplatin • gemcitabine • cyclophosphamide • pemetrexed • vincristine • Erivedge (vismodegib)
    5ms
    The HEDGEHOG-GLI1 pathway is important for fibroproliferative properties in keloids and as a candidate therapeutic target. (PubMed, Commun Biol)
    Moreover, the HH signal inhibitor vismodegib reduced keloid reconstituted tumor size and keloid-related gene expression in nude mice and the collagen bundle and expression of cytokines characteristic for keloids in ex vivo culture of keloid tissues. These results implicate the HH-GLI1 pathway in keloid pathogenesis and suggest therapeutic targets of keloids.
    Journal
    |
    SPP1 (Secreted Phosphoprotein 1) • GLI1 (GLI Family Zinc Finger 1)
    |
    Erivedge (vismodegib)
    5ms
    The Sonic hedgehog pathway inhibitor GDC0449 induces autophagic death in human Medulloblastoma Daoy cells. (PubMed, Ultrastruct Pathol)
    It was observed that inhibiting autophagy using Beclin1 siRNA significantly blocked the apoptosis-inducing effects of GDC0449, suggesting that GDC0449 mediates its apoptotic effects by inducing autophagy.Our data suggests that GDC0449 inhibits the growth of human MB Daoy cells by autophagy-mediated apoptosis. The mechanism of GDC0449-induced autophagy in Daoy cells may be related to the inhibition of the PI3K/AKT/mTOR signaling pathway.
    Journal
    |
    CASP3 (Caspase 3) • EIF4EBP1 (Eukaryotic translation initiation factor 4E binding protein 1) • BECN1 (Beclin 1)
    |
    Erivedge (vismodegib)
    6ms
    Vismodegib Combined With Atezolizumab in Platinum Resistant Ovarian, Fallopian Tube, and Primary Peritoneal Cancer (clinicaltrials.gov)
    P2, N=48, Recruiting, Ronald Buckanovich | Trial completion date: Mar 2026 --> Apr 2028 | Trial primary completion date: Mar 2025 --> Apr 2028
    Trial completion date • Trial primary completion date
    |
    BRCA (Breast cancer early onset) • CD4 (CD4 Molecule)
    |
    Tecentriq (atezolizumab) • Erivedge (vismodegib)
    6ms
    Signaling Pathways and Therapeutic Strategies in Advanced Basal Cell Carcinoma. (PubMed, Cells)
    The discovery of mutations in the Hh signaling pathway has paved the way for the development of Hh pathway inhibiting agents, such as vismodegib and sonidegib, which have represented a breakthrough in the aBCC management. However, the use of these agents is limited by the frequent occurrence of adverse events or the development of drug resistance. In this review, we thoroughly describe the current knowledge regarding the available options for the pharmacological management of aBCCs and provide a forward-looking update on novel therapeutic strategies that could enrich the therapeutic armamentarium of BCC in the near future.
    Review • Journal • Metastases
    |
    SMO (Smoothened Frizzled Class Receptor)
    |
    Erivedge (vismodegib) • Odomzo (sonidegib)
    6ms
    Turning enemy into ally: hijack brain tumor cells to break down the blood-tumor barrier (SNO 2023)
    Using a genetically engineered mouse model of SHH MB, we show that tumor-specific knockout of volume-regulated ion channel, Lrrc8a, significantly increases BTB permeability and enhances the therapeutic response of MB-bearing mice to vismodegib, an FDA-approved antagonist of the SHH pathway...Furthermore, tumor-specific LRRC8A knockdown enhances BTB permeability in a xenograft human Group 3 MB model. Collectively, our findings establish Lrrc8a/LRRC8A as a therapeutic target to increase the therapy response of MB and provide evidence that inducing EndMT could be a strategy for enhancing BTB permeability to treat brain cancer.
    Tumor cell
    |
    LRRC8A (Leucine Rich Repeat Containing 8 VRAC Subunit A) • TGFB2 (Transforming Growth Factor Beta 2)
    |
    Erivedge (vismodegib)
    6ms
    Whole exome sequencing of intracranial epidermoid cysts (IECs) reveals potential somatic drivers (SNO 2023)
    Digital droplet PCR (ddPCR) of these samples is underway to validate the low tumor allele frequencies ( < 4%). The discovery of activating mutations in SMO is significant because it potentiates FDA-approved SMO-inhibitors like Vismodegib and Sonidegib as viable treatment options for patients with IECs.
    Whole exome sequencing
    |
    SMO (Smoothened Frizzled Class Receptor) • STIM1 (Stromal Interaction Molecule 1)
    |
    SMO mutation
    |
    Erivedge (vismodegib) • Odomzo (sonidegib)
    6ms
    Increasing Ciliary ARL13B Expression Drives Active and Inhibitor-Resistant Smoothened and GLI into Glioma Primary Cilia. (PubMed, Cells)
    ARL13B-driven increases in ciliary SMO and GLI2 are resistant to SMO inhibitors, GDC-0449, and cyclopamine. Collectively, our data suggest that factors increasing ARL13B expression in glioma cells may promote both changes in ciliary membrane characteristics and IFT proteins, leading to the accumulation of drug-resistant SMO and GLI. The downstream targets and consequences of these ciliary changes require further investigation.
    Journal
    |
    GLI2 (GLI Family Zinc Finger 2)
    |
    Erivedge (vismodegib) • cyclopamine
    7ms
    Co-targeting FAK and Gli1 inhibits the tumor-associated macrophages-released CCL22-mediated esophageal squamous cell carcinoma malignancy. (PubMed, MedComm (2020))
    Furthermore, inhibition of FAK activity by VS-4718, the FAK inhibitor, enhanced antitumor effect of GDC-0449, the HH inhibitor, both in xenografted models and in vitro assays. Clinically, CCL22/Gli1 axis is used to evaluate ESCC prognosis. Overall, our study establishes the communication of FAK with HH pathway and offers the novel mechanism related to Gli1 activation independent of Smoothened as well as the rationale for the anti-ESCC combination treatment.
    Journal
    |
    GLI1 (GLI Family Zinc Finger 1) • SOX2 • POU5F1 (POU Class 5 Homeobox 1) • CCL2 (Chemokine (C-C motif) ligand 2) • SUFU (SUFU Negative Regulator Of Hedgehog Signaling) • YBX1 (Y-Box Binding Protein 1) • CCL22 (C-C Motif Chemokine Ligand 22) • NANOG (Nanog Homeobox)
    |
    Erivedge (vismodegib) • VS-4718
    8ms
    NCT Neuro Master Match - N²M² (NOA-20) (clinicaltrials.gov)
    P1/2, N=228, Completed, University Hospital Heidelberg | Recruiting --> Completed | N=350 --> 228 | Trial completion date: Sep 2024 --> Feb 2023 | Trial primary completion date: Sep 2023 --> Feb 2023
    Trial completion • Enrollment change • Trial completion date • Trial primary completion date
    |
    BRAF (B-raf proto-oncogene)
    |
    Tecentriq (atezolizumab) • Ibrance (palbociclib) • Alecensa (alectinib) • Torisel (temsirolimus) • Erivedge (vismodegib) • idasanutlin (RG7388) • Apocept (asunercept)
    8ms
    Intraventricular SHH inhibition proves efficient in SHH medulloblastoma mouse model and prevents systemic side effects. (PubMed, Neuro Oncol)
    We conclude that intraventricular application of a SHH pathway inhibitor combines the advantages of a specific treatment agent with precise drug delivery and might evolve as a promising new way of targeted treatment for SHH MB patients.
    Preclinical • Journal • Adverse events
    |
    PTCH1 (Patched 1)
    |
    Erivedge (vismodegib)
    8ms
    Surgery after sonidegib treatment achieves complete response in locally advanced basal cell carcinoma of the face. (PubMed, J Dermatol)
    Vismodegib as a neoadjuvant treatment before surgery has been investigated in a single, multicentre, open-label, phase II trial (VISMONEO); however, sonidegib has not yet been evaluated in this setting. We report the cases of two patients with locally advanced basal cell carcinoma of the face who achieved complete remission with sonidegib followed by a more limited surgical excision than would have been needed without target therapy.
    Journal • Surgery • Metastases
    |
    Erivedge (vismodegib) • Odomzo (sonidegib)
    8ms
    European consensus-based interdisciplinary guideline for diagnosis and treatment of basal cell carcinoma-update 2023. (PubMed, Eur J Cancer)
    Hedgehog inhibitors (HHIs), vismodegib or sonidegib, should be offered to patients with locally advanced and metastatic BCC. Immunotherapy with anti-PD1 antibodies (cemiplimab) is a second-line treatment in patients with a progression of disease, contraindication, or intolerance to HHI therapy...In Gorlin patients, regular skin examinations are required to diagnose and treat BCCs at an early stage. Long-term follow-up is recommended in patients with high-risk BCC, multiple BCCs, and Gorlin syndrome.
    Review • Journal
    |
    Libtayo (cemiplimab-rwlc) • Erivedge (vismodegib) • Odomzo (sonidegib)
    8ms
    Polymeric micelles for cutaneous delivery of the hedgehog pathway inhibitor TAK-441: formulation development and cutaneous biodistribution in porcine and human skin. (PubMed, Int J Pharm)
    TAK-441 is a potent inhibitor of the hedgehog pathway (IC 4.4 nM) developed for the treatment of basal cell carcinoma that is active against the vismodegib-resistant Smoothened receptor D473H mutant...The cutaneous biodistribution profile demonstrated that TAK-441 was predominantly delivered to the viable epidermis and upper dermis. Delivery from the HPMC hydrogel formulation resulted in TAK-441 epidermal concentrations that were several thousand-fold higher than the IC, with almost negligible transdermal permeation, thereby decreasing the risk of systemic side effects in vivo.
    Preclinical • Journal
    |
    SMO D473H
    |
    Erivedge (vismodegib)
    8ms
    Multicenter Retrospective Andalusian Study of the Use of Sonidegib for the Treatment of Local Advanced Basal Cell Carcinoma in Real Clinical Practice. (PubMed, J Clin Med)
    Sonidegib is the second iHh authorized for the treatment of adult patients with locally advanced BCC who are not amenable to curative surgery or radiotherapy, based on the results of the phase II clinical trial, BOLT. Sonidegib shows good effectiveness and an acceptable safety profile in routine clinical practice in the sample presented.
    Retrospective data • Journal • Metastases
    |
    Erivedge (vismodegib) • Odomzo (sonidegib)
    9ms
    Metastatic Basal Cell Carcinoma: Treatment with a potentially best in class Hedgehog Inhibitor, Taladegib (EADV 2023)
    Patient was further treated by vismodegib for 12 months followed by Cemiplimab. For the first time we report here that mBCC patients who were refractory to current standard of care treatments responded to taladegib with a duration of response of around one year with fewer and manageable adverse effects.
    PD(L)-1 Biomarker • IO biomarker • Metastases
    |
    PTCH1 (Patched 1)
    |
    PTCH1 mutation
    |
    Libtayo (cemiplimab-rwlc) • Erivedge (vismodegib) • taladegib (ENV 101)
    9ms
    Review of Targeted Therapy, Vismodegib, for the Treatment of Periocular Basal Cell Carcinoma. (PubMed, Ophthalmic Plast Reconstr Surg)
    In patients with POLA-BCC, vismodegib, a hedgehog pathway inhibitor, provided high rates of orbital preservation, reducing exenteration rates to 6%. Neoadjuvant therapy with vismodegib can also be suggested for patients with POLA-BCC. While extremely effective, side effects lead to temporary or permanent discontinuation of vismodegib in small numbers of patients.
    Review • Journal
    |
    Erivedge (vismodegib)
    9ms
    SMMART Adaptive Clinical Treatment (ACT) Trial (clinicaltrials.gov)
    P1, N=25, Not yet recruiting, OHSU Knight Cancer Institute | Phase classification: P2 --> P1 | N=131 --> 25
    Phase classification • Enrollment change
    |
    HER-2 (Human epidermal growth factor receptor 2) • BRCA1 (Breast cancer 1, early onset) • BRCA2 (Breast cancer 2, early onset)
    |
    BRCA2 mutation • BRCA1 mutation • HR positive • HER-2 negative • EGFR positive
    |
    Avastin (bevacizumab) • Herceptin (trastuzumab) • Lynparza (olaparib) • Tecentriq (atezolizumab) • Ibrance (palbociclib) • Zelboraf (vemurafenib) • carboplatin • Rozlytrek (entrectinib) • Alecensa (alectinib) • Kadcyla (ado-trastuzumab emtansine) • Cotellic (cobimetinib) • capecitabine • Piqray (alpelisib) • Zejula (niraparib) • albumin-bound paclitaxel • fulvestrant • irinotecan • Halaven (eribulin mesylate) • letrozole • vinorelbine tartrate • Herzuma (trastuzumab-pkrb) • anastrozole • Erivedge (vismodegib) • Trazimera (trastuzumab-qyyp) • Phesgo (pertuzumab/trastuzumab/hyaluronidase-zzxf) • EG1206A (pertuzumab biosimilar)
    10ms
    Recommendations for Cost-Conscious Treatment of Basal Cell Carcinoma. (PubMed, Dermatol Ther (Heidelb))
    For a primary superficial BCC < 0.5 cm arising on Area M, the cost-conscious algorithm prioritizes C&E or cryosurgery. For a primary nodular BCC 1.1-2 cm arising on Area L, the cost-conscious algorithm prioritizes C&E, cryosurgery, or 5-FU. For a giant BCC, the cost-conscious algorithm identifies superficial radiation therapy as first line.
    Review • Journal
    |
    cisplatin • 5-fluorouracil • Libtayo (cemiplimab-rwlc) • Erivedge (vismodegib) • Odomzo (sonidegib) • Zyclara (imiquimod)
    10ms
    The Efficacy of Sonidegib in Treating Locally Advanced Basal Cell Carcinoma Involving the Periocular Area. (PubMed, Dermatol Ther (Heidelb))
    To the best of our knowledge, this is the first study investigating the effectiveness and safety of sonidegib in the management of BCC localized at the periocular region. Even if limited, our study suggests this drug as a valuable and safe option in periocular BCC management.
    Journal • Metastases
    |
    Erivedge (vismodegib) • Odomzo (sonidegib)
    10ms
    Complete response of a large basosquamous carcinoma following treatment with cemiplimab and vismodegib. (PubMed, BMJ Case Rep)
    After 21 cycles of cemiplimab, the left shoulder ulcerated lesion was completely re-epithelialised. He remains in complete remission after 31 cycles of cemiplimab in addition to vismodegib.
    Journal
    |
    Libtayo (cemiplimab-rwlc) • Erivedge (vismodegib)
    10ms
    Combinations of PRI-724 Wnt/β-Catenin Pathway Inhibitor with Vismodegib, Erlotinib, or HS-173 Synergistically Inhibit Head and Neck Squamous Cancer Cells. (PubMed, Int J Mol Sci)
    Mixtures of PRI-724 and vismodegib affected cell cycle distribution, greatly reduced cell migration, and downregulated the transcript level of CSC markers, especially POU5F1 encoding OCT4. Combinations of PRI-724 with erlotinib or HS-173 were more potent in inducing apoptosis.
    Journal
    |
    POU5F1 (POU Class 5 Homeobox 1) • ANXA5 (Annexin A5)
    |
    erlotinib • Erivedge (vismodegib) • foscenvivint (PRI724)
    10ms
    Long-term strategies for management of advanced basal cell carcinoma with hedgehog inhibitors. (PubMed, Crit Rev Oncol Hematol)
    The hedgehog inhibitors sonidegib and vismodegib are first-line treatments for advanced BCC with a long-lasting response, but long-term treatment with hedgehog inhibitors is often challenged by tolerability issues. However, several strategies for adverse effect management are available, such as dose interruptions, on-label alternate-day dosing and supportive medications. In conclusion, although BCC shows a high tumor mutational burden that favors a response to immunotherapy, experts recommend keeping patients on hedgehog inhibitors limiting immunotherapy to those who developed resistance during hedgehog inhibitor therapy or in case of persisting toxicity despite long-term management of adverse events.
    Review • Journal • Tumor mutational burden • IO biomarker • Metastases
    |
    TMB (Tumor Mutational Burden)
    |
    TMB-H
    |
    Erivedge (vismodegib) • Odomzo (sonidegib)
    10ms
    Sonidegib as a Locally Advanced Basal Cell Carcinoma Therapy in Real-life Clinical Setting: A National Multicentre Study. (PubMed, Actas Dermosifiliogr)
    Sonidegib shows good effectiveness and acceptable safety profile in usual clinical practice.
    Journal • Metastases
    |
    Erivedge (vismodegib) • Odomzo (sonidegib)
    10ms
    Sonidegib as a locally advanced basal cell carcinoma therapy in real-life clinical setting: a national multicentre study. (PubMed, Actas Dermosifiliogr)
    Sonidegib shows good effectiveness and acceptable safety profile in usual clinical practice.
    Retrospective data • Journal • Metastases
    |
    Erivedge (vismodegib) • Odomzo (sonidegib)