Erivedge (vismodegib) / Curis, Roche - LARVOL DELTA

Developmental Sacral Fusion Defines a Biologically Relevant Model for Studying Pathological Ankylosis in Axial Spondyloarthritis (ACR Convergence 2026) - "Developmental sacral fusion recapitulates key structural features of pathological ankylosis and provides a biologically relevant platform for studying new bone formation in axSpA. Our findings suggest that Hedgehog-regulated fibrocartilaginous remodeling and progenitor-cell dynamics contribute to tissue-intrinsic programs driving spinal ankylosis."

Ankylosing Spondylitis • Fibrosis • Immunology • Inflammatory Arthritis • Seronegative Spondyloarthropathies • Spondylarthritis

Vismodegib-loaded ufasomes inhaler for treatment of non-small cell lung cancer. (PubMed, Naunyn Schmiedebergs Arch Pharmacol) - "Additionally, toxicity and histopathological studies confirmed the efficacy and safety of the inhaled VLU. The inhaled VLU shows promise as an effective and safe treatment option for NSCLC."

Journal • Lung Cancer • Non Small Cell Lung Cancer • Oncology • Solid Tumor • AFP • CEACAM5

Vismodegib hedgehog-signaling inhibition and treatment of basal cell carcinomas in Gorlin-Goltz syndrome (EADV 2026) - No abstract available

Basal Cell Carcinoma • Non-melanoma Skin Cancer • Oncology

Improving public cancer care by implementing precision medicine in Norway (clinicaltrialsregister.eu) - P1/2 | N=1000 | Recruiting | Sponsor: Oslo University Hospital HF | N=6000 ➔ 1000

Enrollment change • Oncology

INVASIVE MELANOMA ARISING ON A PREVIOUS BASAL CELL CARCINOMA SCAR TREATED WITH VISMODEGIB: A CASE REPORT (EADV 2026) - No abstract available

Case report • Clinical • Basal Cell Carcinoma • Melanoma • Non-melanoma Skin Cancer • Oncology • Solid Tumor

Structure-based discovery of potent SMO inhibitors guided by MM/GBSA binding free energies in hedgehog-driven cancers. (PubMed, Comput Biol Chem) - "Virtual screening of the ZINC database highlighted ZINC000299767263 (ZINC7263) as a top candidate, exhibiting a Glide score of -10.86 and MM/GBSA binding energy of -65.3 kJ/mol, comparable to the FDA-approved inhibitor Vismodegib...These findings position ZINC7263 as a novel, potent SMO inhibitor with a stable binding affinity, dynamic interaction stability, and promising preclinical potential for targeting Hh-driven cancers. Its unique scaffold and optimized pharmacokinetic profile advocate for advanced in vitro and in vivo studies to translate these computational insights into therapeutic applications."

Journal • Oncology

Treating challenging basal cell carcinoma with Vismodegib: A Real-World prospective experience (EADV 2026) - No abstract available

Clinical • Real-world • Real-world evidence • Basal Cell Carcinoma • Non-melanoma Skin Cancer • Oncology

Patient-Derived Xenograft Models of Pediatric Medulloblastoma Enable Therapeutic Evaluation of Vismodegib (EANO 2026) - "Patient-derived xenograft models of pediatric medulloblastoma represent robust and biologically relevant platforms for translational research. Their ability to preserve tumor characteristics supports their application in precision oncology. The antitumor activity observed with vismodegib reinforces the utility of these models for preclinical evaluation of targeted therapies."

Preclinical • Brain Cancer • Medulloblastoma • Oncology • Solid Tumor

N2M2/NOA-20: Phase I/IIa umbrella trial of molecularly matched targeted therapies plus radiotherapy in patients with newly diagnosed glioblastoma without MGMT promoter hypermethylation. (ASCO 2024) - P1/2 | "Background: Patients with glioblastoma without MGMT promoter hypermethylation are unlikely to benefit from temozolomide (TMZ)...The alectinib and vismodegib subtrials were closed since no molecularly matching patients were accrued; the idasanutlin subtrial was closed prior to the optimal dose at nine patients at discretion of the company providing the drug... N 2 M 2 allows for elaborate molecular testing being integrated into the treatment decision and efficient determination of treatment activity for patients with newly diagnosed glioblastoma. There is clinical activity of temsirolimus in patients with tumors harboring an activated mTOR pathway although this is not positively prognostic without mTOR inhibition; there is no clinical activity for asunercept and atezolizumab in not molecularly selected patients and also palbociclib in molecularly selected patients."

Clinical • P1/2 data • Brain Cancer • CNS Tumor • Glioblastoma • Oncology • Solid Tumor • CDK4 • CDKN2A • CDKN2B

NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines): Basal Cell Skin Cancer, Version 1.2027 (NCCN)

NCCN guideline • Basal Cell Carcinoma

Establishment of a patient-derived malignant peripheral nerve sheath tumor organoid model reveals SHH pathway activation and therapeutic vulnerability. (PubMed, Hum Cell) - "Consistent with these findings, treatment with the Smoothened inhibitor vismodegib significantly suppressed organoid growth and downregulated SHH-related gene expression. These results indicate that SHH pathway activation may represent a therapeutically actionable vulnerability in a subset of MPNST. Our study establishes a patient-derived organoid model of MPNST and highlights its utility as a preclinical platform for investigating tumor biology and evaluating targeted therapies in this rare malignancy."

Journal • Brain Cancer • Neurofibrosarcoma • Oncology • Sarcoma • Soft Tissue Sarcoma • Solid Tumor • NF1 • PTCH1 • SUZ12

Proton Pump Inhibitor (PPI) use and outcomes of Vismodegib therapy in advanced basal cell carcinoma: a post-hoc analysis of the ERIVANCE-BCC trial (EADO 2026) - "Conclusions Concomitant PPI use did not affect vismodegib efficacy but was linked to higher risk of neurological AEs. Age and ECOG PS emerged as key prognostic factors for response and survival outcomes."

Metastases • Retrospective data • Basal Cell Carcinoma • Non-melanoma Skin Cancer • Oncology

Fatal Outcome of Widespread Cranial Basal Cell Carcinoma Due to Intraparenchymal Brain Extension in a Setting of Local Recurrences. (PubMed, Cureus) - "She subsequently underwent re-excision, multiple reconstructive procedures with grafts from the bilateral thighs, left forearm, and back, and immunotherapy with vismodegib...This is a unique case of recurrent invasive and locally advanced BCC that supports the notion that this is an aggressive form of the more common indolent cancer seen in daily practice. Emphasis on early detection and intervention is vital in these particularly aggressive cases."

Journal • Basal Cell Carcinoma • Infectious Disease • Non-melanoma Skin Cancer • Oncology • Pain

Vismodegib in Treating Patients With Advanced Chondrosarcomas (clinicaltrials.gov) - P2 | N=45 | Active, not recruiting | Sponsor: National Cancer Institute (NCI) | Trial completion date: Jul 2026 ➔ Jul 2027

Trial completion date • Oncology • Sarcoma • Solid Tumor

Precision medicine directed therapy enabling long-term survival in medulloblastoma: a case report. (PubMed, Front Oncol) - P=N/A | "A molecular tumor board, using precision medicine analysis, recommended vorinostat, vismodegib, curcumin, and palbociclib as targeted therapy. A cell line was derived from the subject's tumor and used for in vitro testing. Combined molecular therapies demonstrated synergistic efficacy compared with single-agent treatment."

Journal • Brain Cancer • Medulloblastoma • Oncology • Solid Tumor • Transplantation

Use of Hedgehog Pathway Inhibitors in Combination Therapy for Basal Cell Carcinoma: A Review and Narrative Synthesis. (PubMed, J Clin Aesthet Dermatol) - "Combination strategies for HHI therapy represent a promising avenue to optimize efficacy, improve tolerability, and expand therapeutic options for patients with BCC."

Journal • Review • Basal Cell Carcinoma • Dermatology • Non-melanoma Skin Cancer • Oncology

FINPROVE - The Finnish National Study to Facilitate Patient Access to Targeted Anti-cancer Drugs (clinicaltrialsregister.eu) - P1/2 | N=250 | Recruiting | Sponsor: HUS-Yhtymae | Not yet recruiting ➔ Recruiting

Enrollment open • Hematological Malignancies • Oncology

Pulmonary Metastases From a Long-Standing Untreated Facial Basal Cell Carcinoma: A Rare Case Report. (PubMed, Cureus) - "The patient received broad-spectrum intravenous antibiotics for the superimposed infection and was evaluated for systemic therapy with vismodegib or cemiplimab. This case illustrates how prolonged treatment delay can transform a highly curable malignancy into locally destructive and metastatic disease."

Journal • Basal Cell Carcinoma • Infectious Disease • Non-melanoma Skin Cancer • Oncology • Pain • Skin Cancer

Vismodegib Exerts a Pleotropic Effect on Topotecan-Resistant and Sensitive Ovarian Cancer Cell Lines in 2D and 3D Cell Culture Models. (PubMed, Molecules) - "Our results suggest that Vismodegib re-sensitizes TOP-resistant cells in both 2D and 3D models. However, further research is needed to evaluate its usefulness in ovarian cancer treatment."

Journal • Preclinical • Gynecologic Cancers • Oncology • Ovarian Cancer • Solid Tumor

Ultradeformable liposomal delivery of vismodegib modulates its biological activity in melanoma cells. (PubMed, Front Med Technol) - "Overall, these results show that ultradeformable liposomal delivery potentiates the in vitro anti-melanoma activity of VDG and supports its further exploration as a repurposing strategy for candidate non-metastatic cutaneous melanoma. More broadly, these findings support the rationale for topical nanocarrier-based delivery as an approach that could potentially improve local drug availability while helping to reduce some of the limitations associated with systemic administration."

Journal • Melanoma • Oncology • Solid Tumor

Biomimetic co-delivery nanoplatform overcomes CAF- and TAM-induced immunosuppression to augment therapeutic efficacy against triple-negative breast cancer. (PubMed, Int J Pharm ) - "To address these limitations, we developed an erythrocyte membrane-camouflaged biomimetic co-delivery nanoplatform encapsulating Vismodegib and BMS-1 (designated as E-V/B@NM)...Synergizing with PD-1/PD-L1 inhibition, this strategy significantly enhanced dendritic cells (DCs) maturation and antigen-presenting function, boosted CD8+ T cell infiltration and immune response, and effectively suppressed primary tumor progression and metastasis in TNBC murine models. Our findings highlight that the biomimetic nanoplatform based on E-V/B@NM remodels the TME by reprogramming both CAFs and TAMs, thereby overcoming immunosuppression and improving ICB efficacy in TNBC."

Journal • Breast Cancer • Oncology • Solid Tumor • Triple Negative Breast Cancer • CAFs • CD8

Vismodegib Improved Therapy of Medulloblastoma by Targeting Sonic Hedgehog Signaling Pathway on DAOY Medulloblastoma Cell Line. (PubMed, Basic Clin Neurosci) - "Also, a notable increase in the activation of metastasis-promoting genes (Bax and p53) and a reduction in the activation of the metastasis-inhibiting gene (Bcl2) were observed. The results of the current study confirm that vismodegib is a potent inhibitor of the Shh pathway and may be used in combination with new therapeutic methods to treat medulloblastoma."

IO biomarker • Journal • Preclinical • Brain Cancer • Medulloblastoma • Oncology • Solid Tumor • BCL2 • GLI1 • MYCN • SMO

Merlin deficiency supports an immunosuppressive milieu in breast cancer. (PubMed, Cell Death Discov) - "Treatment with Vismodegib, a pharmacological Hedgehog pathway inhibitor, reduced tumor growth of Merlin-deficient basal-like tumors, accompanied by a decrease in tumor-infiltrating regulatory T cells and immunosuppressive M2-like macrophages, and increased recruitment of pro-inflammatory M1-like macrophages. The data present Hedgehog inhibition as a targetable vulnerability of Merlin-deficient basal-like, triple-negative breast cancer."

Journal • Breast Cancer • Oncology • Solid Tumor • Triple Negative Breast Cancer • EZR • NF2 • RDX

Multimodal CNS-Directed Therapy Incorporating Intraventricular Chemotherapy in High-Risk Medulloblastoma (ISPNO 2026) - "We describe three patients with high‑risk medulloblastoma treated with integrated intraventricular chemotherapy, etoposide daily for 3–5 days and cytarabine twice weekly for two weeks, within 4‑week cycle of systemic chemotherapy...A 34‑year‑old man with localized SHH‑activated medulloblastoma harboring PTCH1, TP53 mutations and GLI2 amplification underwent standard‑dose craniospinal irradiation, then received integrated systemic and intraventricular chemotherapy, followed by maintenance therapy with vismodegib...This approach may enhance antitumor efficacy without disrupting systemic therapy schedule. These promising early observations warrant further evaluation in clinical trials and may represent a potential avenue to improve survival outcomes."

Brain Cancer • Medulloblastoma • Solid Tumor • GLI2 • PTCH1 • TP53

A071401: Vismodegib, FAK Inhibitor GSK2256098, Capivasertib, and Abemaciclib in Treating Patients with Progressive Meningiomas (clinicaltrials.gov) - P2 | N=124 | Recruiting | Sponsor: Alliance for Clinical Trials in Oncology | Trial completion date: Oct 2024 ➔ Jan 2028 | Trial primary completion date: Oct 2024 ➔ Jan 2026

Trial completion date • Trial primary completion date • Brain Cancer • CNS Tumor • Meningioma • Oncology • Solid Tumor • AKT1 • CCND1 • CCND2 • CCND3 • CCNE1 • CDK4 • CDK6 • CDKN2A • NF2 • PIK3CA • PTCH1 • PTEN • SMO