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VCN-01

Phase 1

Pancreatic Cancer | Monoclonal antibody | Oncology |Theriva Biologics, Inc.|Last Updated: Jun 25, 2025

Success Probability

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Market & Valuation

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Trial Design

ACTIVE_CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment13

FDA Designations

FAST_TRACKORPHAN_DRUGRARE_PEDIATRIC_DISEASE

Clinical trial landscape

VCN-01 · 3 trials · 6 indications

Phase 1 3
NCT05057715huCART-meso + VCN-01 in Pancreatic and Ovarian CancerPancreatic Cancer
ACTIVE NOT_RECRUITING13 Analytics
NCT02045589A Phase I Dose Escalation Study of Intratumoral VCN-01 Injections With Gemcitabine and Abraxane® in Patients With Advanced Pancreatic CancerPancreatic Adenocarcinoma
COMPLETED8 Analytics
NCT02045602Phase I Dose Escalation Study of Intravenous VCN-01 With or Without Gemcitabine and Abraxane® in Patients With Advanced Solid TumorsLocally Advanced Solid Tumors
COMPLETED42 Analytics
PHASE1ACTIVE NOT_RECRUITING
huCART-meso + VCN-01 in Pancreatic and Ovarian Cancer
Pancreatic CancerUnlock trial analytics
PHASE1COMPLETED
A Phase I Dose Escalation Study of Intratumoral VCN-01 Injections With Gemcitabine and Abraxane® in Patients With Advanced Pancreatic Cancer
Pancreatic AdenocarcinomaUnlock trial analytics
PHASE1COMPLETED
Phase I Dose Escalation Study of Intravenous VCN-01 With or Without Gemcitabine and Abraxane® in Patients With Advanced Solid Tumors
Locally Advanced Solid TumorsUnlock trial analytics

Study Endpoints

Primary Endpoints

Type, frequency, severity, and attribution of AEs/SAEs as assessed by CTCAE v 5.0
2 years
Occurrence of dose-limiting toxicities.
2 years
Safety and Tolerability by means of Adverse Events (AEs) and laboratory data
At least 6 months
Recommended Phase 2 Dose (RP2D) of VCN-01 in combination with Gemcitabine and Abraxane® by determination of highest feasible dose (MFD) and any Dose Limiting Toxicities
At least 6 months
Recommended Phase 2 Dose (RP2D) by determination of highest feasible dose (MFD) and any Dose Limiting Toxicities
At least 6 months

Secondary Endpoints

Proportion of subjects enrolled who receive one or both of the intended study infusions
2 years
Overall Response Rate (ORR)
15 years
Best Overall Response (BOR)
15 years
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Study Design & Arms

AllocationNON_RANDOMIZED
MaskingNONE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Cohort 1ACTIVE_COMPARATORSingle dose of 3.3x10(12) vp of VCN-01 on Day 0, followed by a single dose of 5x10(7) of huCART-meso cells on Day 14.
Cohort 2ACTIVE_COMPARATORSingle dose of 1x10(13) vp of VCN-01 on Day 0, followed by a single dose of 5x10(7) of huCART-meso cells on Day 14.
Cohort -1ACTIVE_COMPARATORIn the event that 2 DLTs occur in Cohort 1, then enrollment in Cohort 1 will be stopped and Cohort -1 will be opened for evaluation. Enrolled subjects will receive a single dose of huCART-meso cells on Day 0 followed by a single dose of 3.3x10(12) vp of VCN-01 on Day 14.
Dose Escalation, CombinationEXPERIMENTALThree intratumoral administrations of VCN-01 oncolytic adenovirus every 28 days in combination with Abraxane® and Gemcitabine.
Part I: Dose Escalation, Single AgentEXPERIMENTALSingle intravenous injection of VCN-01 oncolytic adenovirus
Part II: Dose Escalation, CombinationEXPERIMENTALSingle intravenous injection of VCN-01 oncolytic adenovirus in combination with Abraxane®/Gemcitabine
Part III: Dose Escalation, Combination, "delayed" scheduleEXPERIMENTALSingle intravenous injection of VCN-01 oncolytic adenovirus in combination with Abraxane®/Gemcitabine

Interventions

NameTypeDescription
VCN-01BIOLOGICALIntravenous administration of VCN-01
huCART-meso CellsBIOLOGICALIntravenous administration of huCART-meso cells
GemcitabineDRUG1000 mg/m2 intravenous administration
Abraxane®DRUG125 mg/m2 intravenous administration
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Eligibility Criteria

Age Range18 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites1

Inclusion Criteria: 1. Patients with one of the following diagnoses: 1. Histologically confirmed unresectable or metastatic pancreatic adenocarcinoma; OR 2. Persistent or recurrent serous epithelial ovarian cancer 2. Progression or intolerance to at least one prior standard of care chemother...

Countries:United StatesSpain
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Competitive Landscape -Pancreatic Cancer 182 trials

Top 20 of 71 competitors

CompanyTickerTrialsLead PhaseDrugs
Revolution Medicines, Inc.RVMD9PHASE3RMC-6236, Gemcitabine, nab-paclitaxel, Irinotecan, Liposomal irinotecan
Arcus Biosciences, Inc.RCUS3PHASE3Quemliclustat, Nab-paclitaxel, Gemcitabine
Pfizer Inc.PFE7PHASE2tisotumab vedotin, pembrolizumab, carboplatin, cisplatin
AstraZeneca PLCAZN9PHASE2AZD0901, 5-Fluorouracil, Leucovorin, l-leucovorin, Irinotecan
AbbVie, Inc.ABBV4PHASE2TTX-030, nab-paclitaxel and gemcitabine, Nab-Paclitaxel and gemcitabine
AngioDynamics, Inc.ANGO2PHASE3Modified FOLFIRINOX Regimen
Bristol-Myers Squibb CompanyBMY4PHASE2Navlimetostat, Gemcitabine, Nab-paclitaxel
Immuneering Corp. Class AIMRX2PHASE3Atebimetinib, GnP, mGnP
RenovoRx, Inc.RNXT1PHASE3Gemcitabine, nab-paclitaxel
BioNTech SE Sponsored ADRBNTX2PHASE2Pumitamig, Nab-paclitaxel, Gemcitabine, mFOLFIRINOX
Veracyte, Inc.VCYT1PHASE3Tislelizumab
ArriVent BioPharma, Inc.AVBP3PHASE2JAB-21822
Merck & Co., Inc.MRK2PHASE2Belzutifan
Eli Lilly and CompanyLLY7PHASE1LY4101174
Exelixis, Inc.EXEL1PHASE2Zanzalintinib, Everolimus
Agenus Inc.AGEN4PHASE2AGEN1423, Botensilimab, Gemcitabine, Nab-paclitaxel
HUTCHMED (China) Limited Sponsored ADRHCM1PHASE2Surufatinib Combined With Camrelizumab, Nab-paclitaxel, and Gemcitabine, Nab-paclitaxel Plus Gemcitabine, Surufatinib with Nab-paclitaxel, and Gemcitabine
ImmunityBio IncIBRX2PHASE2N-803, Aldoxorubicin, PD-L1 t-haNK, Nab-paclitaxel, Gemcitabine
Incyte CorporationINCY3PHASE2Ruxolitinib, Capecitabine, Regorafenib
Candel Therapeutics, Inc.CADL1PHASE2Aglatimagene besadenovec
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Recent Changes (Last 90 Days)

LOWMay 24, 2026NCT05057715studyFirstPostDate: changed

Frequently asked questions about VCN-01

What is VCN-01 used for?

VCN-01 is an investigational gene therapy being developed for oncology indications, including pancreatic cancer, locally advanced solid tumors, and pancreatic adenocarcinoma. It is currently in Phase 1 clinical development and has not been approved by the FDA.

How does VCN-01 work?

VCN-01 is a gene therapy designed to target cancer cells. It is being studied as a treatment for pancreatic cancer and other solid tumors. The therapy is administered either intratumorally or intravenously, often in combination with chemotherapy agents such as gemcitabine and nab-paclitaxel.

Who is developing VCN-01?

VCN-01 is being developed by Theriva Biologics, Inc., a biopharmaceutical company traded on the NYSE American under the ticker symbol TOVX. The company is conducting clinical trials to evaluate the safety and efficacy of VCN-01 in patients with advanced solid tumors.

What phase is VCN-01 in?

VCN-01 is currently in Phase 1 clinical development. It has received Fast Track, Orphan Drug, and Rare Pediatric Disease designations from the FDA, but it remains investigational and is not yet approved for any indication.

What clinical trials is VCN-01 in?

VCN-01 has been studied in three Phase 1 trials. NCT02045589 evaluated intratumoral VCN-01 with gemcitabine and Abraxane in advanced pancreatic cancer. NCT02045602 tested intravenous VCN-01 with or without gemcitabine and Abraxane in advanced solid tumors. NCT05057715 is an active trial combining huCART-meso and VCN-01 in pancreatic and ovarian cancer.

Is VCN-01 the same as huCART-meso?

No, VCN-01 is not the same as huCART-meso. VCN-01 is a gene therapy developed by Theriva Biologics, while huCART-meso is a chimeric antigen receptor T-cell therapy. They are being studied in combination in a Phase 1 trial for pancreatic and ovarian cancer.