Delving into the Latest Updates on Vismodegib with Synapse

This phase Ib trial tests the safety, side effects, and best dose of the combination of vismodegib and atezolizumab in treating patients with non-small cell lung cancer (NSCLC) that has come back after a period of improvement (recurrent) or has spread from where it first started (primary site) to other places in the body (metastatic). Vismodegib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Immunotherapy with monoclonal antibodies, such as atezolizumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread. Giving a combination of vismodegib and atezolizumab may be safe, tolerable and/or effective than either drug alone in treating patients with recurrent or metastatic NSCLC.

Start Date31 Dec 2024

Sponsor / Collaborator

Ohio State University Comprehensive Cancer Center

[+1]

NCT06344052

/ RecruitingPhase 2

A Phase 2 Study to Assess the Efficacy of SP-002 with Vismodegib for the Treatment of Locally Advanced Basal Cell Carcinoma

The goal of this clinical trial is to evaluate the efficacy of using SP-002 in participants with locally advanced Basal cell carcinoma. The main question it aims to answer is what the objective response rate for a basal cell carcinoma tumor is following 1 or 3 cycles of SP-002 treatment given as an add-on to hedgehog pathway inhibitor therapy.
Researchers will compare the objective response rate for treated Basal cell carcinoma tumors between 3 treatment Arms.
* Arm 1 participants will receive daily hedgehog pathway inhibitor, and 3 cycles of SP-002 treatment.
* Arm 2 participants will receive daily hedgehog pathway inhibitor, and 1 cycle of SP-002 treatment.
* Arm 3 participants will receive daily hedgehog pathway inhibitor only.

Start Date09 Apr 2024

Sponsor / Collaborator

Stamford Pharmaceuticals, Inc.

NCT05561634

/ RecruitingPhase 2

Evaluation of Radiotherapy After Complete Response to Sonic Hedgehog Pathway Inhibitors in Patients With Locally Advanced Basal Cell Carcinoma: a Prospective Multicenter Study

Locally advanced basal cell carcinoma (BCC) are large BCCs or BCCs located in areas subject to functional and aesthetic risk following surgery or radiotherapy. In these particular situations, surgery and radiotherapy are sometimes not appropriate, and Sonic Hedgehog inhibitors (SHHi) (Vismodegib and Sonidegib) can be proposed. SHHi are effective treatments in laBCC but most CR patients discontinue treatment because of tolerability. Approximately 65% of the population experience a relapse after discontinuation. A few cases of patients treated concomitantly by radiotherapy and vismodegib have been reported in the literature, suggesting that combining vismodegib and concomitant radiotherapy results in an improved overall response compared to a single modality treatment. There is no study evaluating a "consolidation radiotherapy" after complete response to SHHi. We carry out a prospective multicenter study in order to evaluate consolidation radiotherapy in patients with laBCC after achieving complete response with SHHi, with the hypothesis of reducing recurrence after discontinuation of SHHi.

Start Date20 Jun 2023

Sponsor / Collaborator

Centre Hospitalier Universitaire de Lille

[+1]

100 Clinical Results associated with Vismodegib

Login to view more data

100 Translational Medicine associated with Vismodegib

Login to view more data

100 Patents (Medical) associated with Vismodegib

Login to view more data

1,271

Literatures (Medical) associated with Vismodegib

31 Dec 2025·ANNALS OF MEDICINE

Prevalence of genetic alterations in basal cell carcinoma patients resistant to Hedgehog pathway inhibitors: a systematic review

Review

Author: Ponvilawan, Ben ; Limwongse, Chanin ; Nampipat, Nichanant ; Untaaveesup, Suvijak ; Techataweewan, Gynna ; Pongphaew, Chanamon ; Srichana, Pornteera ; Dendumrongsup, Wichapol

INTRODUCTION:

Basal cell carcinoma (BCC) is a prevalent form of skin cancer that can be localized or metastatic. Current evidence supports the use of Hedgehog (Hh) pathway inhibitors for locally advanced or metastatic BCC with resistance due to genetic alterations in the Hh pathway. This systematic review evaluated the prevalence of genetic alterations in Hh pathway genes in BCC.

MATERIALS AND METHODS:

We conducted a comprehensive search across four databases: PubMed, EMBASE, SCOPUS and the Cochrane Library. We included articles reporting genetic alterations in patients with locally advanced or metastatic BCC resistant to Hh pathway inhibitors.

RESULTS:

We included three prospective cohort studies encompassing 27 samples, all of which were resistant to vismodegib treatment. The most prevalent genetic mutations in the Hh pathway were in PTCH1, SMO and TP53, with a pooled prevalence of 44.44%.

CONCLUSIONS:

This systematic review highlights the prevalence of genetic alterations in the Hh pathway in BCC and offers insights into the mechanisms involved in treatment resistance. Understanding the high resistance rates of these genes may facilitate the development of more effective targeted therapies for BCC.

01 Nov 2025·ARCHIVES OF ORAL BIOLOGY

GDC-0449 suppresses odontogenic keratocyst aggressiveness in fibroblasts by upregulating SPARC via Hedgehog pathway inhibition

Article

Author: Wu, Shian ; Shen, Jing ; Gao, Jing ; Hong, Yingying ; Qu, Jiafei ; Zhai, Jiemei ; Chen, Xiwen ; Jiang, Yongqi ; Zhou, Yuanzhu ; Chen, Defu

OBJECTIVE:

Odontogenic keratocysts (OKCs) are aggressive jaw lesions, either linked to Gorlin-Goltz syndrome or sporadic. We aimed to investigate the impact of the Hedgehog (Hh) pathway inhibitor GDC-0449 on OKC fibroblasts (OKC-Fs). Additionally, we explored whether secreted protein acidic and rich in cysteine (SPARC) was a novel Hh target and its association with OKC aggressiveness.

DESIGN:

Fibroblasts from four syndromic and four non-syndromic OKCs were isolated, with patched 1 (PTCH1) mutations found only in syndromic ones. Syndromic OKC-Fs with PTCH1 mutations were treated with GDC-0449, using mutation-free non-syndromic OKC-Fs as controls. Wound healing and transwell assays assessed the migration and invasion of OKC-Fs. Real-time polymerase chain reaction (PCR), alkaline phosphatase staining, and tartrate-resistant acid phosphatase staining investigated osteogenic differentiation and osteoclastogenic effects. Real-time PCR and western blotting examined the correlation between SPARC and Hh signaling. SPARC siRNA treatment tested its effect on cell migration and invasion.

RESULTS:

GDC-0449 inhibited the migration, invasion, and osteoclastogenic ability, while promoting their osteogenic differentiation of syndromic OKC-Fs. Moreover, GDC-0449 inhibited Hh pathway by decreasing glioma-associated oncogene 1 (GLI1) expression in syndromic OKC-Fs harboring PTCH1 mutations. Additionally, by suppressing Hh pathway, GDC-0449 upregulated SPARC expression, suggesting that Hh-GLI1 signaling exerts a negative regulatory effect on SPARC. Knockdown of SPARC promoted the migration and invasion of OKC-Fs, which could be reversed by GDC-0449.

CONCLUSION:

GDC-0449 suppresses aggressiveness and osteoclastogenesis of OKCs while promoting osteogenesis. GDC-0449 treats OKCs by inhibiting Hh signaling and upregulating SPARC. SPARC could be a potential therapeutic target for OKCs.

01 Nov 2025·FOOD SCIENCE AND BIOTECHNOLOGY

Solasodine inhibited the proliferation of gastric cancer cells through suppression of Hedgehog/Gli1 signaling

Article

Author: Zhan, Xiaoman ; Son, Hui Mang ; Lee, Kang Hyuk ; Lee, Hong Jin ; Kim, Jin Tae ; Zhou, Yimeng ; Ghimire, Alina

In this study, we investigated the beneficial effect of solasodine, a steroidal alkaloid from Solanaceae plants, on suppressing the proliferation of gastric cancer cells by targeting Hedgehog (Hh) signaling. 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. As an upstream regulator, solasodine inhibited Hh signaling by down-regulating Gli1 expression and blocking its nuclear localization without affecting Smo levels. Molecular docking revealed stable binding of solasodine to Gli1, with a binding affinity of -7.4 kcal/mol. 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.

90

News (Medical) associated with Vismodegib

09 Dec 2025

·www.businesswire.com

Delphia Therapeutics Appoints Leading Cancer Expert Steve Kelsey, M.D., to Board of Directors

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Delphia Therapeutics, Inc. (Delphia), a biopharmaceutical company pioneering an innovative new area of cancer biology – activation lethality – which targets cancer's surprising vulnerability to oncogene overactivation, today announced the appointment of Stephen (Steve) Kelsey, M.D., MB ChB, FRCP, FRCPath to its board of directors. Dr. Kelsey is a highly-accomplished physician-scientist, oncologist and cancer drug developer and currently serves as president, research & development at Revolution Medicines. “Steve’s deep expertise across oncology research, early clinical development and organizational leadership makes him an exceptional addition to our board,” said Kevin Marks, Ph.D., co-founder, president and CEO of Delphia. “His track record of advancing transformational cancer medicines—from first-in-human studies through approval—will be invaluable as Delphia advances its pipeline and evolves into a clinical-stage company. I look forward to working closely with him as we continue to build a field-defining platform in activation lethality and advance our programs.” “Activation lethality represents a powerful new approach to exploiting the inherent vulnerabilities of oncogene-driven cancers, and Delphia’s scientific foundation and leadership team position the company to make a major impact,” said Dr. Kelsey. “This approach may open the door to novel therapeutic strategies that could reshape how oncogenic pathways are controlled, with the potential to achieve deeper and more sustained responses for patients. I look forward to supporting the team as it advances its programs toward clinical development and continues to build this important new area of cancer medicine.” Prior to joining Revolution Medicines as president, research & development in 2017, Dr. Kelsey served as president of Onkaido Therapeutics, a Moderna Venture focused on oncology mRNA therapeutics. Prior to Onkaido, he served in senior positions at Medivation and Geron, where he led efforts to develop innovative cancer therapeutics. Prior to Geron, he served as vice president of hematology/oncology at Genentech, where he built the exploratory clinical development group and played a significant role in the development of key products Perjeta®, Kadcyla® and Erivedge®. He began his career in life sciences at Pharmacia/SUGEN where he led the initial clinical development and clinical proof-of-concept for sunitinib (Sutent®) in GIST tumors, resulting in its approval after the acquisition by Pfizer. Before his transition to industry, Dr. Kelsey was a senior lecturer in hematology/oncology at St. Bartholomews and the Royal London School of Medicine and Dentistry. He was also director of the hematology/oncology Phase 1/2 clinical trials unit at Barts and the London School of Medicine and Dentistry and head of the Bone Marrow Transplant unit. Dr. Kelsey holds bachelor of medicine and bachelor of surgery degrees (MB ChB), as well as a doctorate of medicine (M.D.) degree from the University of Birmingham, U.K. and is a fellow of the Royal College of Physicians (FRCP) of London and the Royal College of Pathologists (FRCPath), U.K. About Delphia Therapeutics Delphia Therapeutics is a biopharmaceutical company pioneering activation lethality, an innovative new area of cancer therapeutics that target cancer's surprising vulnerability to oncogene overactivation. Delphia’s activation lethality platform offers the potential for first-in-class, targeted cancer medicines that are effective on their own while also combating the emergence of drug resistance to classic targeted therapies. Delphia is headquartered in Cambridge, MA, is supported by leading life sciences investors including GV, Nextech Invest, Polaris Partners, Alexandria Venture Investments and Taiho Ventures. For more information, please visit us at www.delphiatx.com and follow us on LinkedIn.

Executive ChangeDrug Approval

14 Nov 2025

·www.prnewswire.com

Curis to Present at Upcoming 30th Annual SNO Meeting

LEXINGTON, Mass., Nov. 14, 2025 /PRNewswire/ -- Curis, Inc. (NASDAQ: CRIS), a biotechnology company focused on the development of emavusertib (CA-4948), an orally available, small molecule IRAK4 inhibitor, today announced that Curis will report emavusertib and BTKi clinical data in Primary CNS Lymphoma (PCNSL) and Secondary CNS Lymphoma (SCNSL) in three presentations and preclinical data on emavusertib in an oral presentation at the 30th Annual Meeting of the Society for Neuro-Oncology (SNO) on November 19-23: November 21, 2025 – 4:30 PM ET (11:30 AM HT) November 22, 2025 – 4:45 PM ET (11:45 AM HT) November 23, 2025 – 3:24 PM ET (10:24 AM HT) About Curis, Inc. Curis is a biotechnology company focused on the development of emavusertib, an orally available, small molecule IRAK4 inhibitor. Emavusertib is currently being evaluated in the TakeAim Lymphoma Phase 1/2 study (CA-4948-101) in patients with relapsed/refractory primary central nervous system lymphoma (PCNSL) in combination with the BTK inhibitor ibrutinib, as a monotherapy in the TakeAim Leukemia Phase 1/2 study (CA-4948-102) in patients with relapsed/refractory acute myeloid leukemia (AML) and relapsed/refractory high risk myelodysplastic syndrome (hrMDS), and as a frontline combination therapy with venetoclax and azacitidine in patents with AML (CA-4948-104). Emavusertib has received Orphan Drug Designation from the U.S. Food and Drug Administration for the treatment of PCNSL, AML and MDS and from the European Commission for the treatment of PCNSL. Curis, through its 2015 collaboration with Aurigene Discovery Technologies Limited, has the exclusive license to emavusertib (CA-4948). Curis licensed its rights to Erivedge® to Genentech, a member of the Roche Group, under which they are commercializing Erivedge® for the treatment of advanced basal cell carcinoma. For more information, visit Curis's website at . SOURCE Curis, Inc. 21% more press release views with Request a Demo

Orphan DrugLicense out/inASCOASH

02 Apr 2025

·pipelinereview.com

Senhwa Biosciences Announces Positive Clinical Data from Phase 1/Expansion Trial of Silmitasertib (CX-4945) in the Treatment of Basal Cell Carcinoma

Basal Cell Carcinoma CSR Highlights TAIPEI, Taiwan and SAN DIEGO, CA, USA I April 2, 2025 I Senhwa Biosciences, Inc. (TPEx: 6492), a new drug development company focusing on first-in-class therapeutics for oncology, rare diseases, and infectious diseases, today announced that the completion of Clinical Study Report (CSR) for Phase 1/Dose Expansion Trial of Silmitasertib (CX-4945) in the Treatment of Basal Cell Carcinoma(BCC), with positive data outcomes. The study included patients who had relapsed after standard therapies and had no other treatment options. Among them, three patients experienced over 30% tumor reduction (PR), and two patients had a progression-free survival (PFS) exceeding 21 months. CX-4945 significantly prolonged survival in advanced cancer patients, marking a major milestone for both the patients and Senhwa. Compared to current first- and second-line therapies, CX-4945 demonstrated superior tolerability and disease control potential. Therefore, Senhwa will actively pursue licensing possibilities while carefully evaluating CX-4945’s potential as a monotherapy or in combination with the second-line immunotherapy Libtayo. The company aims to explore further indications and potential as a first-line treatment, offering better options for patients. Clinical Trial Overview The trial enrolled 25 patients with locally advanced BCC (laBCC, 20 patients) and metastatic BCC (mBCC, 5 patients). The primary objective was to determine the recommended Phase 2 dose (RP2D) and optimal dosing regimen for CX-4945. Among 22 patients eligible for efficacy analysis: Further data analysis showed that: CX-4945 exhibited promising anti-tumor efficacy and disease stabilization potential in this indication. Additionally, CX-4945 has a lower study drug discontinuation rate of 24% due to AEs, compared to first-line treatments including Vismodegib and Sonidegib, suggesting superior tolerability. Future Prospects This trial is particularly noteworthy as it evaluated CX-4945 monotherapy in patients who had already failed first-line HHIs. Notably, 27.3% (6 patients) had also failed second-line PD-1 inhibitors such as Libtayo or Keytruda. Among the enrolled patients, two advanced-stage patients achieved a progression-free survival (PFS) of over 21 months, lasting 653 days and 667 days, respectively. Notably, the primary lesion of the former patient was almost undetectable by visual inspection. Given the significant potential of CX-4945 as a monotherapy, Senhwa is now actively pursuing regional licensing to accelerate commercialization through strategic partnerships, ultimately benefiting more patients. SOURCE: Senhwa Biosciences

Clinical ResultImmunotherapyLicense out/inPhase 1

100 Deals associated with Vismodegib

Login to view more data

External Link

KEGG	Wiki	ATC	Drug Bank
D09992	Vismodegib	L01XX43	DB08828

R&D Status

Approved

10 top approved records.

Login

to view more data

Indication	Country/Location	Organization	Date
Metastatic basal cell carcinoma	European Union	Roche Registration GmbH	12 Jul 2013
Metastatic basal cell carcinoma	Iceland	Roche Registration GmbH	12 Jul 2013
Metastatic basal cell carcinoma	Liechtenstein	Roche Registration GmbH	12 Jul 2013
Metastatic basal cell carcinoma	Norway	Roche Registration GmbH	12 Jul 2013
Basal Cell Carcinoma	United States	Genentech, Inc.	30 Jan 2012

Developing

10 top R&D records.

Login

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Platinum-Resistant Fallopian Tube Carcinoma	Phase 2	United States	Genentech, Inc.	15 May 2023
Platinum-Resistant Primary Peritoneal Carcinoma	Phase 2	United States	Genentech, Inc.	15 May 2023
Odontogenic Tumors	Phase 2	United States	Genentech, Inc.	27 Oct 2015
Chronic graft-versus-host disease	Phase 2	United States	Genentech, Inc.	08 Sep 2015
Steroid Refractory Graft Versus Host Disease	Phase 2	United States	Genentech, Inc.	08 Sep 2015
Idiopathic Pulmonary Fibrosis	Phase 2	-	Hoffmann-La Roche, Inc.	01 Oct 2014
Advanced Malignant Solid Neoplasm	Phase 2	United States	Genentech, Inc.	14 Apr 2014
Biliary Tract Neoplasms	Phase 2	United States	Genentech, Inc.	14 Apr 2014
Bladder Cancer	Phase 2	United States	Genentech, Inc.	14 Apr 2014
Salivary Gland Neoplasms	Phase 2	United States	Genentech, Inc.	14 Apr 2014

Login to view more data

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


ACTRN12618000281291 (ESMO2025)	Phase 2	PTCH1 mutation \| SMO mutation	16	Vismodegib	veezorknfk(hsswkhakfz) = The most common adverse events (AE) were fatigue, myalgia and dysgeusia (all grade 1-2); 2 grade 3 AEs, and 1 serious AE. pyattdedps (pnqkvijutc )	Negative	17 Oct 2025
NCT02465060 (EAY131, Pubmed \| JCO Precis Oncol)	Phase 2	Neoplasms SMO-Mutated \| PTCH1-Mutated	31	Vismodegib	lsosuavehe(sgvxeylbpm) = qjejnbivko zevoysyobx (ioyujmrssz, 1.4 - 23.1) View more	Negative	01 Oct 2025
NCT02091141 (MyPathway, CTgov)	Phase 2	Advanced Malignant Solid Neoplasm \| Bladder Cancer \| Biliary Tract Neoplasms ... View more	673	Trastuzumab+Pertuzumab (Trastuzumab Plus Pertuzumab)	rcwqllqpuz = wavbuslapf dypocqlnkb (tzbvpxofzs, vmkaodusnp - cazhzchazo) View more	-	23 Jul 2024
NCT02091141 (MyPathway, CTgov)	Phase 2		673	Atezolizumab (Atezolizumab)	rcwqllqpuz = vvlnmonbem dypocqlnkb (tzbvpxofzs, svervbpqvg - vzailwgnmr) View more	-	23 Jul 2024
NCT01835626 (Pubmed) Manual	Phase 2	Locally Advanced Unresectable Breast Carcinoma Induction	19	Vismodegib	syfmpnbdeo(onsvllxptk) = tqyvdvukvn abrjbgvare (ymeqfhlhxs, 68 - 98) View more	Positive	17 Apr 2024
NCT02436408 (VISORB trial, Pubmed \| PLoS One)	Phase 4	Basal Cell Carcinoma hedgehog signaling \| Gli1 positive \| SMO W535L mutation	-	Vismodegib	uavqwbkyhb(hrvbkdxmqf) = ypnmdwuzpb ktzlbbzhnw (coyjuhjuks )	-	01 Dec 2022
NCT02091141 (MyPathway, ESMO2022)	Phase 2	Advanced Malignant Solid Neoplasm HER2 amplification \| tumor mutational burden ≥16 mut/Mb \| ALK rearrangements ... View more	672	pertuzumab + trastuzumab	fqxxibtynq(gnpllmiljh) = crgggeyrja zpfcemjpea (cavrulsbor )	-	11 Sep 2022
NCT02091141 (MyPathway, ESMO2022)	Phase 2		672	atezolizumab	fqxxibtynq(gnpllmiljh) = amyvpxaews zpfcemjpea (cavrulsbor )	-	11 Sep 2022
NCT02465060 (EAY131, ASCO2022) Manual	Phase 2	Neoplasms PTCH1 Mutation \| SMO Mutation	31	vismodegib 150 mg (all analyzable pts)	ifnhrvktou(cxkeufdtaw) = xiyigzajzb icezyrhzqi (bfslctjfcm ) View more	Negative	02 Jun 2022
NCT02465060 (EAY131, ASCO2022) Manual	Phase 2	Neoplasms PTCH1 Mutation \| SMO Mutation	31	vismodegib 150 mg (MATCH-confirmed)	ifnhrvktou(cxkeufdtaw) = oniiktopce icezyrhzqi (bfslctjfcm ) View more	Negative	02 Jun 2022
NCT03052478 (Pubmed \| J Cancer) Manual	Phase 2	Stomach Cancer	19	vismodegib	wgpiamvnot(pypiigfwjr) = ewilmkzdyb srmsetuddy (piqsvehrij ) View more	Positive	09 Jan 2022
NCT02436408 (VISORB \| VISORB trial, CTgov)	Phase 4	Basal Cell Carcinoma	35	Vismodegib	rfwhjeeiyl = nesxmxlzyo mzgpmnymbe (blxewjmodz, fiipasavet - eokfntilbu) View more	-	28 Oct 2021
NCT02337517 (CTgov)	Phase 2	Steroid Refractory Graft Versus Host Disease \| Chronic graft-versus-host disease	6	Laboratory Biomarker Analysis+Vismodegib	lehscatveg = vecuhjbnlr svhqussdzm (kuaxnicgpd, egdgcvirho - hehazmhelv) View more	-	16 Jun 2021