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INO-5401

Phase 1

Glioblastoma | Monoclonal antibody | Oncology |Inovio Pharmaceuticals, Inc.|Last Updated: Sep 2, 2026

Success Probability

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Trial Design

CONTROLLED
Total Trials1
Total Enrollment52

FDA Designations

No designations recorded

Clinical trial landscape

INO-5401 · 2 trials · 2 indications

Phase 1 2
NCT03502785INO-5401 + INO-9012 in Combination With Atezolizumab in Locally Advanced Unresectable or Metastatic/Recurrent Urothelial CarcinomaUrothelial Carcinoma
COMPLETED35 Analytics
NCT03491683INO-5401 and INO-9012 Delivered by Electroporation (EP) in Combination With Cemiplimab (REGN2810) in Newly-Diagnosed Glioblastoma (GBM)Glioblastoma
ACTIVE NOT_RECRUITING52 Analytics
PHASE1COMPLETED
INO-5401 + INO-9012 in Combination With Atezolizumab in Locally Advanced Unresectable or Metastatic/Recurrent Urothelial Carcinoma
Urothelial CarcinomaUnlock trial analytics
PHASE1ACTIVE NOT_RECRUITING
INO-5401 and INO-9012 Delivered by Electroporation (EP) in Combination With Cemiplimab (REGN2810) in Newly-Diagnosed Glioblastoma (GBM)
GlioblastomaUnlock trial analytics

Study Endpoints

Primary Endpoints

Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Adverse Events of Special Interest (AESIs) Treatment
Up to approximately 71 months

An adverse event (AE) is defined as any untoward medical occurrence in a participant or clinical investigation participant administered a pharmaceutical product and which does not necessarily have to have a causal relationship with this treatment. TEAEs were defined as any AEs that occurred on or after Day 0. AESIs were toxicities and immune-mediated AEs that may have occurred up to 90 days after the last dose of trial treatment. AESIs were reported by the investigator to the Sponsor within 24 hours after learning of the event.

Number of Participants With Clinically Significant Changes in Hematological Parameters
Up to approximately 71 months

Clinically significant changes in hematological parameters were determined based on the investigator's discretion.

Number of Participants With Clinically Significant Changes in Serum Chemistry Parameters
Up to approximately 71 months

Clinically significant changes in serum chemistry parameters were determined based on the investigator's discretion.

Number of Participants With Clinically Significant Changes in Urinalysis Parameters
At baseline, Weeks 3, 6, 9, then every 6 weeks thereafter, up to 71 months

Clinically significant changes in urinalysis parameters were determined based on the investigator's discretion.

Antigen-Specific Immune Response
Up to approximately 71 months

Blood and tissue samples were collected to evaluate the antigen-specific immune response to INO-5401 + INO-9012 in combination with atezolizumab. Planned assessments included: Enzyme Linked Immunosorbent Spot-forming (ELISpot) Assay, Flow cytometry, T cell receptor (TCR) sequencing, ELISA and/or gene expression analysis.

Objective Response Rate (ORR) as Assessed by Response Evaluation Criteria in Solid Tumors (RECIST) Version 1.1 by Investigator Review in Cohort A
From date of treatment start until date of first PD or death (whichever occurred first), up to approximately 71 months

ORR was defined as the percentage of participants who had a confirmed complete response (CR) or a partial response (PR). CR was defined as disappearance of all target lesions; any pathological lymph nodes (whether target or non-target) must have reduction in short axis to \< 10 millimeters (mm). PR was defined as at least a 30% decrease in the sum of diameters of target lesions taking as reference the baseline sum of diameters) per RECIST 1.1.

Percentage of Participants with Adverse Events (AEs)
From Day 0 to 30 days after the last dose of study treatment (non-serious AEs) and to 6 months after the last dose of study treatment (immune-related AEs, AEs of special interest and serious AEs) up to approximately 24 months

Secondary Endpoints

ORR as Assessed by RECIST Version 1.1 by Investigator Review in Cohort B
From date of treatment start until date of first PD or death (whichever occurred first), up to approximately 71 months
Percentage of Participants With ORR by Immune Response Evaluation Criteria in Solid Tumors (iRECIST)
From date of treatment start until date of first PD or death (whichever occurred first), up to approximately 71 months
Duration of Response (DoR)
From first documented confirmed CR or PR until first documentation of PD or death, whichever occurred first (approximately 71 months)
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Study Design & Arms

AllocationNON_RANDOMIZED
MaskingNONE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Cohort A: Prior Anti-PD-1/PD-L1EXPERIMENTALParticipants with locally advanced unresectable or metastatic/recurrent UCa, who had confirmed disease progression during or following treatment with anti- programmed death receptor-1/ programmed cell death-ligand 1 (PD-1/PD-L1) therapy received INO-5401 9 milligrams (mg) and INO-9012 1 mg, intramuscular (IM) injection, followed by electroporation (EP) by CELLECTRA® 2000 device, every 3 weeks until Week 9, then every 6 weeks for 6 more doses (until Week 45), thereafter every 12 weeks, until confirmed disease progression, unacceptable toxicity, or deemed intolerable by the investigator in combination with atezolizumab 1200 mg, intravenous (IV) infusion, every 3 weeks, until confirmed disease progression, unacceptable toxicity, or deemed intolerable by the investigator.
Cohort B: Naïve Anti-PD-1/PD-L1EXPERIMENTALParticipants with locally advanced unresectable or metastatic/recurrent UCa who are treatment naïve and ineligible for cisplatin-based chemotherapy received INO-5401 9 mg and INO-9012 1 mg, IM injection, followed by EP by CELLECTRA® 2000 device, every 3 weeks until Week 9, then every 6 weeks for 6 more doses (until Week 45), thereafter every 12 weeks, until confirmed disease progression, unacceptable toxicity, or deemed intolerable by the investigator in combination with atezolizumab 1200 mg, IV infusion, every 3 weeks, until confirmed disease progression, unacceptable toxicity, or deemed intolerable by the investigator.
Cohort A: Unmethylated MGMT PromoterEXPERIMENTALCohort A will include participants with a glioblastoma tumor with an unmethylated MGMT promoter. Participants will receive INO-5401 and INO-9012 and cemiplimab as well as radiation and temozolomide (TMZ; only during radiation therapy), if clinically indicated.
Cohort B: Methylated MGMT PromoterEXPERIMENTALCohort B will include participants with a glioblastoma tumor with a methylated MGMT promoter or with indeterminate MGMT status. Participants will receive INO-5401 and INO-9012 and cemiplimab as well as radiation and temozolomide (TMZ), if clinically indicated. Participants will continue to receive TMZ following radiation therapy, for up to six additional cycles, if clinically indicated.

Interventions

NameTypeDescription
INO-5401BIOLOGICALINO-5401: A mixture of 3 synthetic plasmids that target Wilms' tumor gene-1 (WT1) antigen, prostate-specific membrane antigen (PSMA) and human telomerase reverse transcriptase (hTERT) antigen. IM injection
INO-9012BIOLOGICALINO-9012: A synthetic plasmid that expresses human interleukin-12 (IL-12). IM injection.
AtezolizumabDRUGIV-Infusion.
CELLECTRA® 2000DEVICEIM injection.
CemiplimabBIOLOGICALCemiplimab is an antibody to programmed death-1 (PD-1) protein. Starting on Day 0 cemiplimab will be administered intravenously (IV) every three weeks at a dose of 350 mg per dose in the absence of dose holding, until disease progression as defined by iRANO, unacceptable toxicity, withdrawal of consent, or death.
Radiation TherapyRADIATIONRadiation therapy (RT) will begin no later than 42 days after surgical intervention, and should start approximately 2 weeks after Day 0. RT will be given for three weeks.
TemozolomideDRUGTemozolomide (TMZ) will be given daily during radiation therapy (RT) at a dose of 75 milligrams per square meter (mg/m\^2).
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Eligibility Criteria

Age Range18 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites11

Inclusion Criteria: * Sign an Informed Consent Form (ICF); * Have histologically or cytologically documented locally advanced unresectable or metastatic/recurrent urothelial carcinoma (including renal pelvis, ureters, urinary bladder, and urethra); * For Cohort A: Participants who have radiographic...

Countries:United States
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Frequently asked questions about INO-5401

What is INO-5401 used for?

INO-5401 is an investigational immunotherapy being studied for the treatment of glioblastoma and urothelial carcinoma. It is being evaluated in Phase 1 clinical trials, both in combination with other agents. INO-5401 is developed by Inovio Pharmaceuticals, Inc. (ticker: INO) and is not yet approved by the FDA.

What does INO-5401 target?

INO-5401 is a monoclonal antibody, but its specific molecular target has not been disclosed in the available information. It is being studied in oncology indications, specifically glioblastoma and urothelial carcinoma, in combination with checkpoint inhibitors such as cemiplimab and atezolizumab.

Who makes INO-5401?

INO-5401 is developed by Inovio Pharmaceuticals, Inc., a biopharmaceutical company traded on the stock exchange under the ticker symbol INO. The company is conducting clinical trials to evaluate the safety and efficacy of INO-5401 in patients with glioblastoma and urothelial carcinoma.

What phase is INO-5401 in?

INO-5401 is in Phase 1 clinical development. It is an investigational drug and has not been approved by the FDA. There are two Phase 1 trials: one in glioblastoma that is active but not recruiting, and one in urothelial carcinoma that has been completed.

What clinical trials is INO-5401 in?

INO-5401 is being studied in two Phase 1 trials. NCT03491683 evaluates INO-5401 with INO-9012 and cemiplimab in newly-diagnosed glioblastoma, enrolling 52 patients in the United States. NCT03502785 studies INO-5401 with INO-9012 and atezolizumab in urothelial carcinoma, enrolling 35 patients. Both trials are controlled but not randomized or blinded.

Is INO-5401 the same as INO-9012?

No, INO-5401 and INO-9012 are different investigational agents. They are being studied together in combination in clinical trials for glioblastoma and urothelial carcinoma. INO-5401 is a monoclonal antibody, while INO-9012 is a separate component of the combination regimen.