Erivedge (vismodegib) / Curis, Roche - LARVOL DELTA

P023 Metastatic basal cell carcinoma with bone marrow involvement: a rare case report. (PubMed, Br J Dermatol) - "The patient's presentation with pancytopenia underscores the importance of considering metastatic BCC in cases of unexplained haematological abnormalities in patients with a history of aggressive BCC. Systemic therapies such as vismodegib offer a targeted treatment option and represent a significant advancement in managing metastatic BCC."

Journal • Basal Cell Carcinoma • Hematological Disorders • Hematological Malignancies • Non-melanoma Skin Cancer • Oncology

Cutaneous squamous cell carcinoma while on vismodegib therapy for locally advanced basal cell carcinoma. (PubMed, Actas Dermosifiliogr) - No abstract available

Journal • Basal Cell Carcinoma • Non-melanoma Skin Cancer • Oncology • Squamous Cell Carcinoma • Squamous Cell Skin Cancer

Advanced CYLD Cutaneous Syndrome (CCS) Treated With the Hedgehog Signaling Pathway Inhibitor Vismodegib Results in a Limited Clinical Response. (PubMed, APMIS) - "Furthermore, we observed no possible biomarkers of progression and/or treatment response. Hopefully, further studies will contribute to the genetic understanding of BSS and identify patients at high risk of developing severe disease, thereby supporting the stratification of patients who will benefit from early surgical treatment or treatment with Vismodegib."

Journal • Dermatology • Genetic Disorders • Oncology • Skin Cancer

Vismodegib in locally advanced basal cell carcinoma: Nine-year real-world experience. (PubMed, J Eur Acad Dermatol Venereol) - No abstract available

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

A Clinical and Molecular Risk-Directed Therapy for Newly Diagnosed Medulloblastoma (clinicaltrials.gov) - P2 | N=660 | Active, not recruiting | Sponsor: St. Jude Children's Research Hospital | Trial completion date: Jan 2028 ➔ Oct 2031 | Trial primary completion date: Nov 2025 ➔ Oct 2028

Trial completion date • Trial primary completion date • Brain Cancer • Medulloblastoma • Oncology • Solid Tumor

Rational Design of Non-Toxic Multidrug Combinations Demonstrates Durable and Hypoxia-Enhanced Efficacy Against Renal Cell Carcinoma. (PubMed, Pharmaceutics) - " From an initial panel of 10 drugs, either approved or undergoing clinical trial, the optimized drug combinations (ODCs) contained crizotinib, telaglenastat, U-104, and vismodegib at clinical and subtherapeutic doses. Moreover, chronic exposure of 786O and UOK276 cells led to durable responses, suggesting a prolonged effect in responders. Our findings demonstrate the potential of optimized, non-toxic drug combinations as a highly selective and effective strategy for accelerating the development of precision RCC treatment."

Journal • Clear Cell Renal Cell Carcinoma • Genito-urinary Cancer • Oncology • Renal Cell Carcinoma • Sarcoma • Solid Tumor

Identification of Exosome-Related G6PD in Breast Cancer and Investigation of Its Prognostic Significance (COSA 2025) - "Our multi-omics approach elucidates G6PD's critical role in breast cancer exosomes, linking its overexpression to metabolic reprogramming, immune evasion, and poor prognosis. G6PD expression predicts chemotherapy/immunotherapy responses, offering a rationale for targeting G6PD in combination therapies."

IO biomarker • Breast Cancer • Oncology • Solid Tumor • BCL2 • CD4 • CD8 • FASN • G6PD • PDK4

Mechanistic insights into pharmacokinetic interactions between simvastatin and vismodegib: Implications for optimization of combination therapy in medulloblastoma. (PubMed, Biochem Biophys Res Commun) - "Taken together, these findings establish a mechanistic foundation for a proposed therapeutic optimization strategy: Reducing vismodegib dosing while leveraging its inhibition-driven elevation of simvastatin systemic/tissue exposure, thereby mitigating dose-limiting skeletal toxicity while maintaining anti-tumor efficacy. This mechanism-based strategy provides a clinically actionable framework for pediatric medulloblastoma with urgent unmet therapeutic needs."

Journal • PK/PD data • Brain Cancer • Medulloblastoma • Oncology • Pediatrics • Solid Tumor • SLCO1B1

A review of the safety, efficacy, and administration of hedgehog inhibitors for the treatment of advanced basal cell carcinoma: an expert consensus panel. (PubMed, Dermatol Online J) - "Sonidegib and vismodegib have similar efficacy in treating advanced BCC, but sonidegib has lower rates and a greater delay in onset of AEs. Sonidegib has a significantly greater volume of distribution and half-life than those of vismodegib. Dosing interruptions have not been shown to reduce the efficacy of HHIs, and L-carnitine supplementation can help reduce the incidence of muscle spasms."

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

Disproportionality analysis comparing safety profiles of sonidegib and vismodegib based on the FAERS database (Nature) - "This study provides an overview of HPIs-associated suspected AEs based on real-world data from the FAERS database and reveals divergent reporting patterns between sonidegib and vismodegib. We identified 25 SOCs affected by AEs associated with HPIs, predominantly in general disorders and administration site, musculoskeletal and connective tissue, gastrointestinal and nervous system. The analysis results were consistent with findings of previous observation and clinical trials. Additionally, we discovered unexpected significant AEs, such as second primary malignancies, disorders of special sensory organs, complete atrioventricular block, and so on. HPIs are a novel class of anticancer drugs, and more research is needed to characterize their safety profile."

Adverse events • Basal Cell Carcinoma • Medulloblastoma • Melanoma • Small Cell Lung Cancer

A case of basal cell nevus syndrome with a SUFU mutation. (PubMed, Dermatol Online J) - "Rarely, this condition is related to a suppressor of fused gene mutation, which occurs downstream from Smoothened, and is unresponsive to Smoothened inhibitors including vismodegib and sonidegib. Genetic testing was negative for patched 1 and patched 2 mutations but positive for a heterozygous suppressor of fused mutation. Patients with basal cell nevus syndrome should be treated with surgical excision, counseled on sun protection, screened and monitored for complications, and treated with vismodegib (if associated with patched 1 mutation) or itraconazole (if associated with suppressor of fused mutation)."

Journal • Basal Cell Carcinoma • Brain Cancer • Dermatopathology • Medulloblastoma • Oncology • Rare Diseases • Solid Tumor • PTCH1 • PTCH2 • SUFU

Disproportionality analysis comparing safety profiles of sonidegib and vismodegib based on the FAERS database. (PubMed, Sci Rep) - "However, as a spontaneous reporting system, FAERS cannot establish causal relationships. These findings should therefore be considered hypothesis-generating and warrant further research."

Clinical • Journal • Alopecia • Basal Cell Carcinoma • Breast Cancer • Eye Cancer • Immunology • Non-melanoma Skin Cancer • Oncology • Otorhinolaryngology • Solid Tumor • Squamous Cell Carcinoma • Triple Negative Breast Cancer

Long-term outcomes and molecularly-guided management of adult medulloblastoma: Data from a single-institution experience (ESMO 2025) - "All pts received craniospinal-RT; adjuvant CT included cisplatin etoposide±cyclophosphamide (62%), cisplatin-lomustine±vincristine (21%), or intensive pediatric regimens (10%); 7% had no chemotherapy due to comorbidities...Three pts received vismodegib at recurrence: one (M0 stage, high-risk) received 3 cycles without toxicity, had PD at 3ms from vismodegib start and died at 30ms from diagnosis; one (M2, high-risk) received 5 cycles without toxicity, had PD at 4ms and died at 64ms...Long-term toxicities highlight the need for de-intensified strategies. Legal entity responsible for the study The authors."

Clinical • Brain Cancer • Medulloblastoma • Oncology • Solid Tumor

Medulloblastoma diagnosis in adults: A monocentric retrospective experience (ESMO 2025) - "Patients were treated with different CT regimens, most frequent were 6 pts (46%) with cisplatin-etoposide-cyclophosphamide and 3 pts (23%) with cisplatin-etoposide; 9 complete responses were achieved (69%). 4 pts (31%) experienced progression of disease and were treated with cisplatin-temozolomide (1 pt) or vismodegib (2 pt)...Rare diagnoses would benefit from regional or national registries to increase the number cases and standardize approaches, possibly tailoring treatments on new molecular knowledges. Legal entity responsible for the study Veneto Institute of Oncology."

Retrospective data • Brain Cancer • Medulloblastoma • Oncology • Solid Tumor

The role of platinum-free interval in advanced endometrial cancer treatment: A real-world study of 843 patients (JADPRO 2025) - "However, PFI's prognostic role is uncertain and may influence treatment decisions.The objective is to use real-world data to assess the PFI's prognostic significance and its relationship with subsequent therapies and treatment-free intervals (TFI) in patients with advanced EC.METHODSData SourcesThe study was a retrospective electronic health record (EHR) database analysis using the Flatiron Health database, a curated de-identified dataset derived from patient-level structured and unstructured data, comprising 280 cancer clinics and 2.4 million patients.Data were extracted for women with EC, diagnosed and treated between January 1, 2011, and August 31, 2020.Study PopulationThe study population included women $ge 18$ years of age with primary or recurrent advanced EC.Patients were required to have received a platinum-based regimen for primary or first-recurrence advanced EC, with subsequent platinum-based chemotherapy for second recurrence/advanced disease.Patients..."

Clinical • IO biomarker • Metastases • Real-world • Real-world evidence • Endometrial Cancer • Gynecologic Cancers • Oncology • Solid Tumor

Solasodine inhibited the proliferation of gastric cancer cells through suppression of Hedgehog/Gli1 signaling. (PubMed, Food Sci Biotechnol) - "Solasodine inhibited the proliferation of AGS and MKN74 gastric cancer cells and regulated cell cycle (Cyclin D1 and p27) and apoptosis (Bax and Bcl-2) markers in a dose-dependent manner, consistent with the Gli1/2 inhibitor Gant61 but not the Smo inhibitor vismodegib...Moreover, solasodine inhibited Gli1 rather than Smo expression in Hh signaling overexpressed Ptch (-/-) MEF cells. Our findings demonstrate that solasodine inhibits gastric cancer proliferation by targeting Hh/Gli1 signaling, underscoring its potential as a potent agent for prevention and/or inhibition of gastric cancer."

IO biomarker • Journal • Gastric Cancer • Oncology • Solid Tumor • BAX • BCL2 • CCND1 • GLI1 • SMO

Phase II trial of Vismodegib in advanced cancers with Hedgehog pathway alterations (ESMO 2025) - P2 | "Conclusions Vismodegib did not demonstrate a signal of clinical activity in this cohort of tumours harbouring PTCH1 or SMO alterations. A pooled analysis of more pts is underway and may clarify molecular determinants of hedgehog pathway inhibition in a pan-cancer setting."

Metastases • P2 data • Basal Cell Carcinoma • Brain Cancer • Head and Neck Cancer • Medulloblastoma • Melanoma • Non-melanoma Skin Cancer • Oncology • Solid Tumor • Squamous Cell Carcinoma • Squamous Cell Skin Cancer • PTCH1 • SMO

Low-dose oral minoxidil for the management of vismodegib-induced alopecia. (PubMed, JAAD Case Rep) - No abstract available

Journal • Alopecia • Basal Cell Carcinoma • Dermatology • Immunology • Oncology

A cell-based Sonic hedgehog signaling transduction system to identify additive and synergistic chemical interactions. (PubMed, Toxicol Sci) - "Compounds reported to target SHH ligand processing (RU-SKI 43, AY 9944, U18666A), SMO-mediated signal transduction (cyclopamine, vismodegib, piperonyl butoxide, cannabidiol), and GLI transcription factors (GANT 61, arsenic trioxide) reduced Shh pathway-driven reporter activity with AC50 values in the low micromolar range or below. In zebrafish embryos, combined exposure to piperonyl butoxide and cyclopamine also produced a synergistic increase in craniofacial dysmorphogenesis. These findings demonstrate the importance of tractable models that recapitulate complex signal transduction pathways to empirically test for additive and synergistic chemical interactions in risk assessment."

Journal

NCI-MATCH: Targeted Therapy Directed by Genetic Testing in Treating Patients With Advanced Refractory Solid Tumors, Lymphomas, or Multiple Myeloma (The MATCH Screening Trial) (clinicaltrials.gov) - P2 | N=6452 | Active, not recruiting | Sponsor: National Cancer Institute (NCI) | Trial completion date: Dec 2025 ➔ Dec 2026 | Trial primary completion date: Dec 2025 ➔ Dec 2026

Biomarker • Trial completion date • Trial primary completion date • Bladder Cancer • Brain Cancer • Breast Cancer • Cervical Cancer • Colon Cancer • Colorectal Cancer • Endometrial Cancer • Esophageal Cancer • Gastric Cancer • Genito-urinary Cancer • Glioblastoma • Glioma • Head and Neck Cancer • Hematological Malignancies • Hormone Receptor Positive Breast Cancer • Kidney Cancer • Liver Cancer • Lung Cancer • Lymphoma • Melanoma • Multiple Myeloma • Oncology • Ovarian Cancer • Pancreatic Cancer • Prostate Cancer • Refractory Ovarian Cancer • Renal Cell Carcinoma • Skin Cancer • Solid Tumor • Thyroid Gland Carcinoma • Uterine Cancer • CD4 • MSI

Comprehensive genomic profiling to guide personalized targeted and immunotherapy in gastrointestinal tumors: Subgroup analysis of the ROME trial (ESMO-GI 2025) - P2 | "Funding: Erlotinib, Pertuzumab, Vemurafenib, Trastuzumab Emtansine, Alectinib, Vismodegib, Cobimetinib, Atezolizumab, Trastuzumab, Ipatasertib (GDC-0068), Entrectinib and Pralsetinib were provided by Roche; Everolimus, Lapatinib, Alpelisib were provided by Novartis, Palbociclib and Talazoparib were provided by Pfizer, Ipilimumab and Nivolumab were provided by Bristol Myers Squibb (BMS); Brigatinib was provided by Takeda Pharmaceutical Co.; Ponatinib, Itacitinib (INCB039110), Pemigatinib (INCB054828) were provided by Incyte; Selpercatinib was provided by Eli Lilly; Tepotinib was provided by the healthcare business of Merck KGaA, Darmstadt, Germany (CrossRef Funder ID: 10.13039/100009945). CGP with MTB-guided TT may identify patients with GI cancer who benefit from targeted therapies not routinely available in clinical practice. The roles of TMB and potential disease-specific thresholds deserve further investigation."

IO biomarker • Tumor mutational burden • Colorectal Cancer • Gastrointestinal Cancer • Oncology • Solid Tumor • HER-2 • PIK3CA • TMB

Gastrointestinal adverse events associated with Hedgehog pathway inhibitors: a disproportionality analysis. (PubMed, Int J Pharm Pract) - "HPIs had triggered various types of GIAEs. Liver injuries were a safety concern for the HPI-treated patient, which was not listed by the USFDA. Further researches are needed to confirm these findings."

Adverse events • Journal • Basal Cell Carcinoma • Liver Failure • Non-melanoma Skin Cancer • Oncology

Long-term outcomes and molecularly-guided management of adult medulloblastoma: data from a single-institution experience (EANO 2025) - "All pts received craniospinal-RT; 18 pts(62%) received adjuvant CT with cisplatin-etoposide±cyclophosphamide, 6(21%) cisplatin-lomustine±vincristine, 3(10%) an intensive pediatric regimen and 2(7%) did not received CT due to comorbidities. Our findings confirm the clinical and biological heterogeneity of adult MB, highlighting the need for a multimodal approach. Long-term survivorship is often achievable, though late toxicities remain a concern. Vismodegib showed a significant impact on outcome."

Clinical • Brain Cancer • Medulloblastoma • Oncology • Solid Tumor

Ligand-dependent activation of sonic hedgehog-Gli1 signaling in chordomas (EANO 2025) - "This current preclinical evidence elucidates the therapeutic potential of Shh-Gli1 signaling pathway targeting for chordoma treatment. Vismodegib may be a promising targeted agent, and further clinical trials are warranted."

Chordoma • Oncology • GLI1 • PTCH1

Additional metronomic cyclophosphamide surmounts Vismodegib resistance in adult SHH-mutated medulloblastoma with bone metastasis: a case report (EANO 2025) - "Given the early progression of the bone lesion despite stable brain imaging, systemic chemotherapy — two cycles of carboplatin-etoposide (03-04/2022), followed by Temozolomide-Topotecan (06-07/2022) — was prioritized over immediate craniospinal irradiation. We report the first case of vismodegib combined with cyclophosphamide for the treatment of bone metastatic SHH-medulloblastoma in an adult. The favorable therapeutic outcome in this patient supports the need for dedicated prospective trials to further evaluate the potential benefits of this strategy."

Case report • Clinical • Brain Cancer • Medulloblastoma • Oncology • Solid Tumor • SHH • SMO