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

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100 Translational Medicine associated with Vismodegib

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100 Patents (Medical) associated with Vismodegib

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1,139

Literatures (Medical) associated with Vismodegib

01 Apr 2026·Developmental Neurobiology

Sonic Hedgehog Pathway Modulation in Medulloblastoma: Focus on Vismodegib (GDC‐0449)

Review

Author: Dastjerdi, Ali ; Tavakoli, Marziye Ranjbar ; Rezaei‐Tazangi, Fatemeh ; Chahardehi, Amir Modarresi ; Banafshe, Mahya ; Nasiri, Reza ; Rahimi, Sina ; Refahi, Pegah ; Firouzi, Laleh ; Arefnezhad, Reza ; Meigoli, Mohammad Saeed Soleimani ; Ojaghlou, Fatemeh ; Fatemian, Hossein

ABSTRACT:

Medulloblastoma (MB) is the most common malignant brain tumor in children, classified into four molecular subgroups: WNT, sonic hedgehog (SHH), Group 3, and Group 4. The SHH pathway is crucial in the pathogenesis of SHH‐subgroup MB (SHH‐MB), influenced by mutations in patched homolog 1 (PTCH1), smoothened (SMO), and suppressor‐of‐fused (SUFU). Targeting this route has shown potential, with vismodegib, an SMO inhibitor, demonstrating effectiveness in both preclinical and clinical investigations. Notwithstanding its therapeutic promise, vismodegib's systemic toxicity, especially bone abnormalities in pediatric patients, constrains its application. Novel techniques, such as nanoparticle formulations and intraventricular administration, have optimized medication distribution, diminished toxicity, and augmented effectiveness. Research indicates vismodegib's efficacy in enhancing progression‐free survival (PFS) and addressing tumor‐specific genetic abnormalities. Nonetheless, obstacles, such as resistance stemming from SMO mutations and developmental toxicity, remain. Prospects encompass the enhancement of drug delivery methods, the integration of SHH inhibitors with alternative therapeutics, and the advancement of next‐generation inhibitors to surmount resistance. Hence, these developments offer potential for improving outcomes in SHH‐MB while reducing side effects, especially in pediatric populations.

01 Feb 2026·HEAD AND NECK-JOURNAL FOR THE SCIENCES AND SPECIALTIES OF THE HEAD AND NECK

Safety and Efficacy of Vismodegib and Sonidegib in Advanced Basal Cell Carcinoma of the Head and Neck: A Systematic Review and Meta‐Analysis

Review

Author: Cevik, Jevan ; Ramakrishnan, Anand ; Doyle, Cindy M.

ABSTRACT:

Importance:

Basal cell carcinoma (BCC) is the most prevalent malignancy globally, with 75% occurring in the head and neck. Hedgehog pathway inhibitors (HHIs) may provide a treatment for locally advanced BCC (laBCC) and metastatic BCC (mBCC) of the head and neck.

Objective:

This systematic review and meta‐analysis aimed to evaluate the safety and efficacy of these treatments specifically in the head and neck region.

Data Sources:

An electronic database search was conducted in June 2024 across Embase, MEDLINE, CINAHL, and ClinicalTrials.gov . There were no date limits applied, with search constraints consisting of the English language.

Study Selection:

Included were studies involving adults with confirmed laBCC or mBCC of the head and neck treated with either vismodegib or sonidegib, with histological or radiological confirmation. Studies were excluded if they involved concurrent treatments or lacked quantitative measures. After full text review was completed, there were 17 studies for inclusion.

Data Extraction and Synthesis:

This systematic review and meta‐analysis were conducted in accordance with PRISMA guidelines using two independent observers. Data extraction was performed using a standardized Microsoft Excel Form. Statistical analyses were conducted using meta‐analysis software on jamovi using a random effects model.

Main Outcome(s) and Measure(s):

This study initially aimed to investigate both systemic immunotherapies and targeted therapies, but no articles were identified for systemic immunotherapies for laBCC or mBCC of the head and neck region. Thus, the sole focus of this article became targeted therapies. Primary outcomes assessed were overall response rate (ORR), complete response (CR), and partial response (PR), alongside the prevalence of adverse effects.

Findings:

Seventeen studies involving 522 patients were analyzed, revealing a pooled ORR of 84.2% (95% CI : 77.1–91.3), CR of 33.8%, and PR of 47.7%. Common adverse effects included muscle spasms, dysgeusia, and fatigue, with a discontinuation rate of 13.2% due to adverse events.

Conclusions and Relevance:

This systematic review and meta‐analysis demonstrated a high efficacy of vismodegib and sonidegib for treating advanced BCCs in the head and neck region, suggesting a viable option when surgery is not feasible. Further high‐quality research is necessary to optimize treatment outcomes for this patient population.

01 Feb 2026·GENESIS

Hedgehog‐Wnt Crosstalk Orchestrates Submandibular Gland Morphogenesis in Mice

Article

Author: Qiu, Donghai ; Wang, Qian ; Chen, Yamin ; Song, Yunhong ; Zhang, Yong ; Guo, Yan

ABSTRACT:

The development of the mouse submandibular gland (SMG) is a classic model for branching morphogenesis, orchestrated by key signaling pathways such as Hedgehog (Hh) and Wnt. While Sonic Hedgehog (Shh) is known to be essential for early SMG development, its downstream mechanisms and potential crosstalk with other pathways remain unclear. This study investigates the functional interaction between Hh and Wnt signaling during SMG morphogenesis. Using both in vivo (pharmacological inhibition in pregnant mice) and ex vivo (organ culture) models, we inhibited Hh signaling with vismodegib from embryonic day 13.5 (E13.5). Hh inhibition led to severe morphological defects, including reduced epithelial branching and disorganized ductal structures. As expected, the expression of Hh target genes Ptch1 and Gli1 was downregulated. Notably, Hh suppression resulted in a concomitant upregulation of Wnt pathway activity, evidenced by increased nuclear β‐catenin and elevated expression of the target genes Axin2 and Lef1. This suggested that Hh signaling normally acts to repress Wnt activity during SMG development. Critically, the co‐administration of the Wnt inhibitor XAV939 with vismodegib in ex vivo cultures partially rescued the branching defects caused by Hh inhibition. We conclude that Hh signaling promotes murine SMG morphogenesis, at least in part, by tonically inhibiting the Wnt pathway. This study unveils a novel inhibitory crosstalk between Hh and Wnt signaling that is essential for normal salivary gland development.

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

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KEGG	Wiki	ATC	Drug Bank
D09992	Vismodegib	L01XX43	DB08828

R&D Status

Approved

10 top approved records.

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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.

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Indication	Highest Phase	Country/Location	Organization	Date
Myelofibrosis	Phase 3	Germany	F. Hoffmann-La Roche Ltd.	12 Nov 2015
Primary Myelofibrosis	Phase 3	Germany	F. Hoffmann-La Roche Ltd.	12 Nov 2015
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	Germany	F. Hoffmann-La Roche Ltd.	04 Sep 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

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Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


ACTRN12618000281291 (ESMO2025)	Phase 2	PTCH1 mutation \| SMO mutation	16	Vismodegib	hxthysblhb(fbdrsaxska) = The most common adverse events (AE) were fatigue, myalgia and dysgeusia (all grade 1-2); 2 grade 3 AEs, and 1 serious AE. cweudqrran (orciufouhd )	Negative	17 Oct 2025
NCT02465060 (EAY131, Pubmed \| JCO Precis Oncol)	Phase 2	Neoplasms SMO-Mutated \| PTCH1-Mutated	31	Vismodegib	wzzexwkgkb(svaailcebr) = ghimsggbuk wygvmxcjkc (hckbnupmbw, 1.4 - 23.1) View more	Negative	01 Oct 2025
AAD2025	Not Applicable	Basal Cell Carcinoma	3,774	Sonidegib	bwwdpldnma(pfugazddpb) = dahqkdhdre phnypawlwp (omuybppyyb ) View more	Positive	07 Mar 2025
				Vismodegib	bwwdpldnma(pfugazddpb) = egyfnuzyib phnypawlwp (omuybppyyb ) View more
AAD2025	Not Applicable	Basal Cell Carcinoma	13	Sonidegib	ejnnytuxem(yfpsjiulqt) = njtxybpxta nsukolvnsa (szwkiyslyv ) View more	Positive	07 Mar 2025
				Vismodegib	ejnnytuxem(yfpsjiulqt) = ywalvnuuwj nsukolvnsa (szwkiyslyv ) View more
NCT02091141 (MyPathway, CTgov)	Phase 2	Advanced Malignant Solid Neoplasm \| Bladder Cancer \| Biliary Tract Neoplasms ... View more	673	Trastuzumab+Pertuzumab (Trastuzumab Plus Pertuzumab)	njysifyjqc = dkxzvcljzf qpjfwziwew (ersmgacphs, mskgvfvljf - qgoqlltuib) View more	-	23 Jul 2024
				Atezolizumab (Atezolizumab)	njysifyjqc = ogkekqopud qpjfwziwew (ersmgacphs, jtmznosuzg - ebvuixipfp) View more
NCT01835626 (Pubmed) Manual	Phase 2	Locally Advanced Unresectable Breast Carcinoma Induction	19	Vismodegib	wrkjlsrfpp(jczhbdfdbw) = avziukwqpj obsumsxvks (tfmxupsxoi, 68 - 98) View more	Positive	17 Apr 2024
AAD2024	Not Applicable	Basal Cell Carcinoma	1,706	Sonic hedgehog inhibitors (SHHis) vismodegib and sonidegib	slplhfkvni(casflciehb) = ytujpdwnne bzmqxealuo (mcetiofelz )	Positive	08 Mar 2024
NCT02436408 (VISORB trial, Pubmed \| PLoS One)	Phase 4	Basal Cell Carcinoma hedgehog signaling \| Gli1 positive \| SMO W535L mutation	-	Vismodegib	xfcqvpotgb(ihangtivdk) = ieytmfdpwe hnisgzgjko (iupqrqedec )	-	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	cngzlbqijy(savzalfltr) = ctnqbmbhyq uerjmfedpd (cmqzhpzzcs )	-	11 Sep 2022
				atezolizumab	cngzlbqijy(savzalfltr) = csbhkwupty uerjmfedpd (cmqzhpzzcs )
NCT02465060 (EAY131, ASCO2022) Manual	Phase 2	Neoplasms PTCH1 Mutation \| SMO Mutation	31	vismodegib 150 mg (all analyzable pts)	zivretppgr(foatlmsrll) = znffcwwgel qtnxgdoeod (kmmfcnslnd ) View more	Negative	02 Jun 2022
				vismodegib 150 mg (MATCH-confirmed)	zivretppgr(foatlmsrll) = kjxsgtoira qtnxgdoeod (kmmfcnslnd ) View more