Delving into the Latest Updates on Sonidegib Phosphate with Synapse

Overview

Basic Info

Drug Type

Small molecule drug

Synonyms

Erismodegib, Erismodegib-phosphate, sonidegib

+ [9]

Target

SMO

Action

antagonists

Mechanism

SMO antagonists(Smoothened receptor antagonists)

Therapeutic Areas

Neoplasms

Active Indication

Metastatic basal cell carcinomaBasal Cell Carcinoma

Inactive Indication

Advanced breast cancerAdvanced Gastroesophageal Junction AdenocarcinomaAdvanced Malignant Solid Neoplasm+ [59]

Originator Organization

Novartis Pharma AG

Active Organization

Jemincare Therapeutics Corp.Sun Pharmaceutical Industries (Europe) BVSun Pharma ANZ Pty Ltd.

+ [2]

Inactive Organization

Novartis Pharmaceuticals Corp.

Novartis Pharmaceuticals Canada, Inc.

Novartis AG

+ [1]

License Organization

Novartis AG

Sun Pharmaceutical Industries Ltd.

Jiangxi Jemincare Group Co., Ltd.

Drug Highest PhaseApproved

First Approval Date

United States (24 Jul 2015),

Basal Cell Carcinoma

RegulationOrphan Drug (United States), Overseas New Drugs Urgently Needed in Clinical Settings (China)

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Structure/Sequence

Molecular FormulaC26H32F3N3O11P2

InChIKeyRWIVSVMMGFFZIJ-VWDRLOGHSA-N

CAS Registry1218778-77-8

This study will test the hypothesis that a novel combination of three drugs (sorafenib, sonidegib, and irinotecan), in conjunction with individually optimized doses, can be safely administered and lead to improved clinical outcomes in patients with hepatocellular carcinoma compared to standard of care. The main objective of this study is to establish safe dose ranges for the coadministration of sorafenib, sonidegib, and irinotecan in patients with hepatocellular carcinoma. Furthermore, we will collect data to inform the application of an artificial intelligence/computational approach to individual dosing of combination chemotherapy. Individualization of dosing will be achieved by using Phenotypic Personalized Medicine (PPM) to maximize treatment efficacy in patients with hepatocellular carcinoma, while minimizing toxicity. Drug efficacy will be assessed by measuring plasma circulating tumor DNA (ctDNA). Toxicity will be assessed by quantitating organ injury and patient tolerability. Recommended dosing for future studies will be based on the totality of the data.

Start Date02 Dec 2024

Sponsor / Collaborator

University of Florida

[+1]

ACTRN12623001216606

/ RecruitingPhase 2IIT

Single-institution, single-arm, open-label phase II basket study investigating the effect of mutation and/or elevated hedgehog (Hh) expression-directed smoothened inhibitor sonidegib in patients with advanced solid malignancies

Start Date12 Dec 2023

Sponsor / Collaborator

Northern Sydney Local Health District

100 Clinical Results associated with Sonidegib Phosphate

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100 Translational Medicine associated with Sonidegib Phosphate

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100 Patents (Medical) associated with Sonidegib Phosphate

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469

Literatures (Medical) associated with Sonidegib Phosphate

01 Oct 2026·JOURNAL OF THE AMERICAN ACADEMY OF DERMATOLOGY

Comparison of efficacy and tolerability of hedgehog pathway inhibitors for locally advanced basal cell carcinoma: An expert opinion in the new age of immunotherapy

Review

Author: Sun, Kennedy H ; Migden, Michael R ; Millan, Sarah H ; Patel, Vishal A ; Tan, Marcus

Basal cell carcinoma (BCC) is the most diagnosed malignancy. While most cases are curable, a subset progresses to unresectable locally advanced (laBCC) or metastatic BCC (mBCC). The Smoothened inhibitors (SMOi) vismodegib and sonidegib have significantly improved outcomes in these cases. However, differences in trial design, pharmacokinetics, adverse effects (AEs), and real-world application warrant clinical comparison. This narrative review examines key clinical trials (ERIVANCE, BOLT, STEVIE, MIKIE), FDA labeling, and real-world data. Efficacy outcomes, AE profiles, pharmacokinetics, and resistance mechanisms are analyzed alongside expert recommendations for optimizing treatment. In expert opinion, sonidegib demonstrates a longer median duration of response and more durable disease control compared to vismodegib. Its delayed-onset AEs and deeper tissue penetration make it favorable for infiltrative or high-risk disease. Vismodegib, though more widely prescribed, is associated with earlier-onset AEs and challenges with treatment interruptions due to its shorter half-life. Tailored dosing and AE mitigation strategies as well as neoadjuvant applications of SMOi improve tolerability and outcomes for both agents. Both vismodegib and sonidegib remain essential tools in the management of advanced BCC. Understanding their pharmacologic and clinical differences enables more personalized treatment planning, enhancing long-term outcomes and expanding their use in neoadjuvant and maintenance settings.

17 Aug 2026·CLINICAL AND EXPERIMENTAL DERMATOLOGY

Real-world efficacy and safety of sonidegib in locally advanced and multiple basal cell carcinomas: a monocentric prospective observational study

Article

Author: Filastro, Vincenzo ; Fasano, Gaia ; De Giacomo, Pierfrancesco ; Valenti, Giancarlo ; Del Re, Chiara

Abstract:

This monocentric study evaluated the real-world efficacy and safety of sonidegib in 17 patients with locally advanced (laBCC) or multiple (mBCC) basal cell carcinomas, characterized by complex surgical histories and comorbidities. The objective response was that 53% of patients achieved complete response, 29% partial response and 18% stable disease, with no documented progression during treatment (mean 7.3 months). Sonidegib showed significant efficacy in mBCC, with a mean reduction of 3.89 lesions per patient. Key findings indicate that prior surgery correlates with poorer response, while sonidegib effectively ‘clears the field’ of subclinical lesions. The safety profile was favourable, with predominantly mild and manageable adverse events. Despite the limited sample size, our findings confirm the significant efficacy of sonidegib in a frail pretreated population with multiple comorbidities. This study supports the use of sonidegib as a valuable therapeutic strategy for complex patients with high tumour burden or those ineligible for further surgery.

03 Aug 2026·Expert Opinion On Drug Safety

Comparative safety analysis of Hedgehog inhibitor preparations: insights from the FAERS database

Article

Author: Liu, Yijiang ; Chen, Jia ; Li, Gen ; Wang, Chengliang ; Chen, Li

BACKGROUND:

Vismodegib and sonidegib are currently the only Hedgehog inhibitors (HHIs) approved by the U.S. Food and Drug Administration (FDA) for the treatment of locally advanced BCC (laBCC). However, there is limited systematic research comparing the adverse drug events (ADEs) associated with these two inhibitors.

RESEARCH DESIGN AND METHODS:

Data were extracted from the Food and Drug Administration Adverse Event Reporting System (FAERS) for the period from 2019 to the second quarter of 2024. Disproportionality analysis was conducted to compare ADEs between vismodegib and sonidegib.

RESULTS:

The occurrence of ADEs was notably higher among patients aged ≥ 65 years (72.98%) and males (60.17%). The most frequently reported ADEs associated with HHIs included skeletal muscle and joint diseases and nervous system disorders, particularly muscle spasms and dysgeusia. Differences in adverse reactions between vismodegib and sonidegib were observed, mainly in nervous system, gastrointestinal system, skin and subcutaneous tissue, and renal and urinary disorders.

CONCLUSIONS:

This analysis, using the FAERS database, enhances understanding of the ADE profiles associated with both vismodegib and sonidegib. Further research is needed to validate the hypotheses generated by this study.

13

News (Medical) associated with Sonidegib Phosphate

30 Sep 2026

·www.fiercebiotech.com

Highlight Therapeutics illuminates positive data in skin cancer

Highlight Therapeutics touted positive topline results in patients with localized high-risk head and neck basal cell carcinoma (BCC). The phase 2b study met its primary endpoint of visual and pathological response at week 24, as well as safety and tolerability, with no serious adverse events. These results, presented today during the European Academy of Dermatology and Venereology (EADV) congress in Vienna, support the advancement of Highlight’s BO-112 to late-stage development for localized primary BCC. "Today marks an important milestone for Highlight Therapeutics as these results represent the first clinical validation of BO-112 in BCC and give us strong conviction to advance into late-stage development,” Mercedes Diz, CEO of Highlight Therapeutics, said in the release. “We are excited to bring this potential non-surgical option a step closer to patients, particularly those with high-risk BCC in the head and neck, where the need is greatest.” Basal cell carcinoma is the most common type of skin cancer, and it appears most frequently on the face, head, neck and arms. This cancer begins in the basal cells that live on the outer layers of the skin. When these cells are exposed to damaging rays from the sun or other UV light, the cells can begin to mutate and multiply, leading to a lesion on the surface of the skin. The phase 2 trial , called Spotlight-204, is a multicenter, single-arm study evaluating the safety and efficacy of intralesional BO-112 in 46 patients with resectable primary low- and high-risk BCC. Out of the 46 patients who received BO-112, 61% achieved visual and pathological response. Stratifying these patients further, 58% of high-risk and 70% of low-risk patients achieved visual and pathological response. In the high-risk head and neck group, 56% reached complete pathological response, and in those with lesions in the face, 62% achieved complete pathological response. BO-112 was well tolerated across all patients, as no serious adverse events or grade 3 or higher treatment-related adverse events were reported. Grade 1 adverse events were noted in 91% of patients and resolved in 88% of patients within three days. BO-112 is a double-stranded RNA nanoparticle designed to convert immunological cold tumors to hot tumors so that the immune system can recognize them. It works by activating the innate antiviral sensing pathways, inducing immunogenic tumor cell death, leading to the development of tumor-specific adaptive immunity, according to the company. The Spanish biotech was founded in 2010 under the original name of Bioncotech Therapeutics. Back in 2019, Bioncotech entered a phase 2 clinical trial collaboration with MSD, a subsidiary of Merck , to try BO-112 with Keytruda in patients with advanced stage solid tumors with liver metastases. Highlight has multiple phase 1 and 2 trials running in advanced and resistant melanoma as well as sarcoma and hepatocellular carcinoma. In the field of BCC immunotherapy, there is Sanofi and Regeneron’s PD-1 targeted antibody, Libtayo, which was approved by the FDA in 2021 for advanced BCC, Novartis’ Odomzo and Genentech’s oral Erivedge.

Phase 2ImmunotherapyClinical ResultDrug Approval

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

02 Apr 2025

·www.prnewswire.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 CX-4945 Monotherapy: Progression-free survival (PFS) exceeded 21 months in two patients. Optimal Treatment Response: Three patients achieved partial response (PR), while ten patients had stable disease (SD). Tolerability & Disease Control Rate: Shows potential superiority over current first- and second-line therapies. Unmet Medical Need: Enrolled patients with refractory or recurrent disease who had no remaining treatment options. TAIPEI and SAN DIEGO, April 2, 2025 /PRNewswire/ -- 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: Three laBCC patients achieved partial response (PR). Ten patients had stable disease (SD), including 2 mBCC and 8 laBCC patients. Disease control rates: mBCC patients: 80% (4 patients with complete/partial response or stable disease). laBCC patients: 65% (11 patients with complete/partial response or stable disease). Further data analysis showed that: Median progression-free survival (PFS): laBCC: 9.2 months mBCC: 3.7 months Median duration of disease control (DDC): laBCC: 10.3 months mBCC: 7.5 months 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, Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Clinical ResultImmunotherapyPhase 1License out/in

100 Deals associated with Sonidegib Phosphate

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External Link

KEGG	Wiki	ATC	Drug Bank
D10729	Sonidegib Phosphate	L01XJ02	DB09143

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Metastatic basal cell carcinoma	Australia	Sun Pharma ANZ Pty Ltd.	10 Aug 2015
Basal Cell Carcinoma	United States	Sun Pharmaceutical Industries Ltd.	24 Jul 2015

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Basal Cell Nevus Syndrome	Phase 3	Belgium	Novartis Pharmaceuticals Corp.	03 Mar 2017
Basal Cell Nevus Syndrome	Phase 3	Canada	Novartis Pharmaceuticals Corp.	03 Mar 2017
Basal Cell Nevus Syndrome	Phase 3	Czechia	Novartis Pharmaceuticals Corp.	03 Mar 2017
Basal Cell Nevus Syndrome	Phase 3	Finland	Novartis Pharmaceuticals Corp.	03 Mar 2017
Basal Cell Nevus Syndrome	Phase 3	Germany	Novartis Pharmaceuticals Corp.	03 Mar 2017
Basal Cell Nevus Syndrome	Phase 3	Greece	Novartis Pharmaceuticals Corp.	03 Mar 2017
Basal Cell Nevus Syndrome	Phase 3	Italy	Novartis Pharmaceuticals Corp.	03 Mar 2017
Basal Cell Nevus Syndrome	Phase 3	Spain	Novartis Pharmaceuticals Corp.	03 Mar 2017
Basal Cell Nevus Syndrome	Phase 3	Switzerland	Novartis Pharmaceuticals Corp.	03 Mar 2017
Basal Cell Nevus Syndrome	Phase 3	United Kingdom	Novartis Pharmaceuticals Corp.	03 Mar 2017

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Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT06880848 (ASCO2026)	Phase 4	Basal Cell Carcinoma	160	Sonidegib 200 mg	dusifnjvee(kuljbeqbsu) = wwjrnihlnm jnapfouzzd (wgkgrzwjuz, 50.1 - 65.9) View more	Positive	29 May 2026
AAD2026	Not Applicable	Basal Cell Carcinoma	9	Vismodegib with PDT	npvbqxollr(opcmuosweg) = Most tolerable combinations included sonidegib with itraconazole, sonidegib with radiation, and vismodegib with intratumoral immunotherapy. hanmcvbygi (dxwfxsbwms )	Positive	27 Mar 2026
AAD2026	Not Applicable	Basal Cell Carcinoma	9	Sonidegib with radiation		Positive	27 Mar 2026
AAD2025	Not Applicable	Basal Cell Carcinoma	13	Sonidegib	mnglkmhdms(stccespxio) = srccbboiga egsubgxrmy (sxjhcjydvj ) View more	Positive	07 Mar 2025
AAD2025	Not Applicable	Basal Cell Carcinoma	13	Vismodegib	mnglkmhdms(stccespxio) = ahfvbpskpg egsubgxrmy (sxjhcjydvj ) View more	Positive	07 Mar 2025
AAD2025	Not Applicable	Basal Cell Carcinoma	3,774	Sonidegib	ufzaiidcqf(xcoxmuwadm) = gpckxtkbty ihgtjwopdv (yumuttzkzp ) View more	Positive	07 Mar 2025
AAD2025	Not Applicable	Basal Cell Carcinoma	3,774	Vismodegib	ufzaiidcqf(xcoxmuwadm) = furymczyvn ihgtjwopdv (yumuttzkzp ) View more	Positive	07 Mar 2025
NCT04066504 (NISSO, Pubmed \| BMC Cancer)	Not Applicable	Basal Cell Carcinoma	321	Sonidegib 200 mg	chawonaeuk(rskigmluvs) = Most TEAEs were ≤ grade 2 and the most common were muscle spasms (43.9%), dysgeusia (37.1%), and alopecia (30.2%) kobjolqtyr (bqjhfltbut ) View more	Positive	14 Nov 2024
BOLT (AAD2024)	Phase 2	Basal Cell Carcinoma	66	Sonidegib 200 mg	zxaugmemev(zwpvhflpqr) = qrzcizsgtl lmieqbcxap (yiaahemjdv, 68 - 100) View more	Positive	08 Mar 2024
BOLT (AAD2024)	Phase 2	Basal Cell Carcinoma	66	Sonidegib 200 mg (CR)	zxaugmemev(zwpvhflpqr) = mihicmrlqh lmieqbcxap (yiaahemjdv, 54 - 94) View more	Positive	08 Mar 2024
AAD2024	Not Applicable	Basal Cell Carcinoma	1,706	Sonic hedgehog inhibitors (SHHis) vismodegib and sonidegib	vhnysagxaf(wnhsrpgbnh) = ytclqhbcyg hxbmtxvhuf (grqbnhdykl )	Positive	08 Mar 2024
BOLT study (EADV2023)	Phase 2	Basal Cell Carcinoma	194	Sonidegib 200 mg daily	dohyjbczyr(lzrbvoxgov) = wwmxptnyuh ertznofgtx (tewvnbmftd, 15.1 - NE)	-	11 Oct 2023
WCD2023	Not Applicable	-	-	Sonidegib	dqftnmgvuz(skekakvzhj) = nzumoajcab enwtscyvpm (nixricfoiz ) View more	-	05 Jul 2023
NCT04007744 (ASCO 12023 \| ASCO 22023) Manual	Phase 1	HR-positive/HER2-low Solid Tumors \| Non-Small Cell Lung Cancer	33	Sonidegib Phosphate+Pembrolizumab	cajbjxzctb(hiugwvcydj) = lxdjbzzeyz ovzmnlttdy (fuzmssezpo ) View more	Positive	26 May 2023