Report scope. This 2026 Drug Asset Due Diligence Report evaluates Vocimagene amiretrorepvec as a potential licensing, partnership, acquisition, or option asset. The review separates verified evidence from open diligence questions across clinical development, intellectual property, market positioning, and transaction precedent.
Executive recommendation: CONDITIONAL GO
Vocimagene amiretrorepvec is a Gene therapy associated with CDA and a global highest development stage of Phase 2. The lead disclosed indication is High grade glioma. The current evidence supports a CONDITIONAL GO posture, subject to confirmation of study-level data, ownership, patent coverage, manufacturing readiness, and regional rights.
Decision memo
- Asset: Vocimagene amiretrorepvec
- Target: CDA
- Lead indication: High grade glioma
- Highest phase: Phase 2
- Matched trials: 11
- Matched result records: 16
- Matched asset deals: 2
Use PatSnap Life Sciences MCP servers to validate the Vocimagene amiretrorepvec diligence evidence.
1. Asset identity and development status
The first diligence task is entity resolution. Teams should reconcile development codes, nonproprietary names, salts, formulations, combinations, sponsors, and predecessor owners before relying on portfolio counts. For Vocimagene amiretrorepvec, the MCP profile identifies CDA as the primary target context and High grade glioma as the lead indication. Global phase labels are useful orientation points, but investment decisions require country- and indication-level status verification.
Confirm the exact molecule, formulation, route, dose, biomarker definition, and current sponsor. Review discontinuations separately from active development so that an historical high phase is not mistaken for a live program.
Use PatSnap Life Sciences MCP servers to validate the Vocimagene amiretrorepvec diligence evidence.
2. Clinical program: design and endpoint audit
| Registry | Phase | Status | Enrollment | Lead primary endpoint |
|---|---|---|---|---|
| NCT06504381 | Phase 1/2 | Recruiting | Not disclosed | Not disclosed |
| NCT06264388 | Phase 2 | Recruiting | Not disclosed | Not disclosed |
| NCT04327011 | Phase 1 | Terminated | Not disclosed | Not disclosed |
| NCT04105374 | Phase 2/3 | Withdrawn | Not disclosed | Not disclosed |
| NCT04089163 | Phase 1 | Withdrawn | Not disclosed | Not disclosed |
| NCT02598011 | Phase 1 | Withdrawn | Not disclosed | Not disclosed |
| NCT02576665 | Phase 1 | Terminated | Not disclosed | Not disclosed |
| NCT02414165 | Phase 2/3 | Terminated | Not disclosed | Not disclosed |
| NCT01985256 | Phase 1 | Completed | Not disclosed | Not disclosed |
| NCT01470794 | Phase 1 | Completed | Not disclosed | Not disclosed |
| NCT01156584 | Phase 1 | Completed | Not disclosed | Not disclosed |
The clinical program should be audited for randomization, comparator relevance, dose selection, endpoint hierarchy, multiplicity, analysis population, geographic mix, and readout timing. Early-stage programs require attention to pharmacokinetics, pharmacodynamics, adverse events, dose-limiting toxicities, and the rationale for the expansion dose.
Use PatSnap Life Sciences MCP servers to validate the Vocimagene amiretrorepvec diligence evidence.
3. Clinical readouts: what is known
Effect of Vocimagene Amiretrorepvec in Combination With Flucytosine vs Standard of Care on Survival Following Tumor Resection in Patients With Recurrent High-Grade Glioma
Study: NCT02414165 (TOCA 5, Pubmed | JAMA Oncol). Phase: Phase 2/3. Enrollment: 403. Published: 2020-12-01.
mOS = 11.1 Month | mOS = 12.22 Month
Immune modulation after Toca 511 and Toca FC treatment of colorectal cancer patients.
Study: NCT02576665 (Toca 6, ASCO_GI2020). Phase: Phase 1. Enrollment: 17. Published: 2020-02-04.
mOS = 9.4 Month
P347 Immunomodulation in tumor and peripheral blood following Toca 511 & Toca FC treatment in patients with solid tumors
Study: NCT02576665 (Toca 6, SITC2019). Phase: Phase 1. Enrollment: 21. Published: 2019-11-01.
Adverse Event: Grade 4 adverse events = no related Grade 4 adverse events
Tocagen Reports Results of Toca 5 Phase 3 Trial in Recurrent Brain Cancer
Study: NCT02414165 (Toca5, Biospace). Phase: Phase 3. Enrollment: Not disclosed. Published: 2019-09-12.
OS = 11.1 Month | OS = 12.2 Month
Durable complete responses in some recurrent high-grade glioma patients treated with Toca 511 + Toca FC
Study: NCT01470794 (Pubmed | Neuro Oncol). Phase: Phase 1. Enrollment: 56. Published: 2018-09-03.
DRR = 21.7 %
ATIM-02. DURABLE RESPONSES OBSERVED IN IDH1 WILDTYPE AND MUTANT RECURRENT HIGH GRADE GLIOMA (rHGG) WITH TOCA 511 & TOCA FC TREATMENT
Study: NCT01470794 (ATIM-02, SNO2017). Phase: Phase 1. Enrollment: 24. Published: 2017-11-06.
DRR = 20.8 %
ATIM-02. SUCCESSFUL CANCER-SELECTIVE GENE DELIVERY FOLLOWING INTRAVENOUS TOCA 511 DELIVERY IN PATIENTS WITH RECURRENT HIGH GRADE GLIOMA (HGG)
Study: NCT01985256 (ATIM-02, SNO2016). Phase: Phase 1. Enrollment: 17. Published: 2016-11-07.
Adverse Event: low grade pyrexia = n=2, 18.2%
Prior Radiation in Subjects Who Were Treated With Toca 511 and Toca FC Across 3 Toca 511 Phase 1 Trials
Study: ASTRO2016. Phase: Not Applicable. Enrollment: Not disclosed. Published: 2016-10-01.
AE = 40.2 % | AE = 46.9 %
Phase 1 trial of vocimagene amiretrorepvec and 5-fluorocytosine for recurrent high-grade glioma
Study: NCT02414165 (Pubmed | Sci Transl Med). Phase: Phase 1. Enrollment: 45. Published: 2016-06-01.
mOS = 13.6 Month (95%CI, 10.8 - 20.0) | -
Encouraging survival with Toca 511 and Toca FC compared to external lomustine control.
Study: ASCO 12016 | ASCO 22016. Phase: Phase 1. Enrollment: Not disclosed. Published: 2016-05-20.
AE(Discontinuations) = 0 % | AE(Discontinuations) = 4.8 %
SURG-09 RESULTS OF A DOSE ESCALATION TRIAL OF TOCA 511 WITH TOCA FC IN RECURRENT HGG UNDERGOING REPEAT RESECTION
Study: NCT01470794 (SURG-09, SNO2015). Phase: Phase 1. Enrollment: 44. Published: 2015-11-09.
Adverse Event: Grade 3 adverse event of asthenia = 1 Grade 3 adverse event of asthenia considered related to Toca 511 | Adverse Event: Grade 3 adverse event of asthenia = 1 Grade 3 adverse event of asthenia considered related to Toca 511
SURG-10 TRANSCRANIAL TOCA 511 FOLLOWED BY TOCA FC IN SUBJECTS WITH RECURRENT HIGH GRADE GLIOMA (HGG)
Study: NCT01156584 (SURG-10, SNO2015). Phase: Phase 1. Enrollment: Not disclosed. Published: 2015-11-09.
Median survival = 13.8 Month ( 7.1 - 22.2) | Median survival = 10.6 Month ( 4.3 - 13.1)
IT-05 ADMINISTRATION OF TOCA 511 TO SUBJECTS WITH RECURRENT HGG UNDERGOING REPEAT RESECTION
Study: NCT01470794 (IT-05, SNO2014). Phase: Phase 1. Enrollment: 36. Published: 2014-11-01.
Adverse Event: grade 3 possibly treatment related AE of transient generalized weakness = 1 grade 3 possibly treatment related AE of transient generalized weakness
Ascending dose trials of a retroviral replicating vector (Toca 511) in patients with recurrent high-grade glioma.
Study: NCT01156584 | NCT01985256 | NCT01470794 (ASCO2014). Phase: Phase 1. Enrollment: Not disclosed. Published: 2014-05-20.
MRI changes = consistent with tumor regression | MRI changes = consistent with tumor regression
Interim Report from Two Ongoing Ascending Dose Trials of a Retroviral Replicating Vector (Toca 511) in Patients with Recurrent High Grade Glioma
Study: NCT01156584 | NCT01470794 (Toca 511, ASGCT2014). Phase: Phase 1. Enrollment: Not disclosed. Published: 2014-05-01.
Viral dose levels = 6 level | Viral dose levels = 5 level
Replicating Retrovirus Encoding a Prodrug Activator (Toca 511) Enters Clinic for the Treatment of Glioblastoma Multiforme (GBM)
Study: Toca 511 (ESGCT2010). Phase: Phase 1/2. Enrollment: Not disclosed. Published: 2010-10-22.
Median survival = at least 6 Month | Median survival = approximately 30 Month
For every reported endpoint, obtain the protocol, statistical analysis plan, patient disposition, baseline characteristics, subgroup definitions, exposure duration, and full safety tables. Conference abstracts and registry summaries can support screening, but they should not replace source data review in a binding transaction.
Use PatSnap Life Sciences MCP servers to validate the Vocimagene amiretrorepvec diligence evidence.
4. Market and competitive position
The commercial case depends on differentiated efficacy, tolerability, convenience, biomarker reach, treatment setting, and access. A target-level market may be attractive while an individual asset remains undifferentiated. Benchmark Vocimagene amiretrorepvec against approved therapies and clinical competitors in High grade glioma, including likely standards of care at the expected launch date.
Build a scenario model around addressable patients, diagnosis and biomarker rates, line of therapy, duration, net price, uptake, displacement, combination costs, and probability-adjusted timelines. Test slower enrollment, delayed readout, narrower labels, safety restrictions, and competitor launches.
Use PatSnap Life Sciences MCP servers to validate the Vocimagene amiretrorepvec diligence evidence.
5. Transaction precedent and economics
| Date | Transaction | Status | Type | Disclosed value |
|---|---|---|---|---|
| 2020-04-17 | Denovo plans to purchase Tocagen's RRV technology, Toca-511, PD-L1 inhibitors and other immunooncology targets. | Active | Asset Acquisition | Not disclosed |
| 2018-04-17 | ApolloBio to collaborate with Tocagen in the development and commercialization of Toca-511 for HGG in Greater China. | Active | License | 131 |
Transaction records should be normalized for territory, asset scope, phase at signing, option mechanics, development obligations, cost sharing, royalties, milestones, and change-of-control terms. Undisclosed economics must not be converted into invented benchmarks.
6. IP and freedom-to-operate screen
Complete a family-level patent review covering composition of matter, sequence or construct claims, polymorph and salt claims, formulation, dosing, combinations, biomarkers, manufacturing, and method of use. Verify priority, ownership, assignments, prosecution status, term adjustments, extensions, oppositions, and regional coverage. Freedom to operate is a separate legal analysis and should include competitor claims and platform dependencies.
The diligence room should reconcile inventorship, employee and contractor assignments, university or platform licenses, sublicensing restrictions, government rights, and obligations triggered by development milestones or commercialization.
Use PatSnap Life Sciences MCP servers to validate the Vocimagene amiretrorepvec diligence evidence.
7. Principal risks and diligence gates
- Identity risk: resolve all aliases, formulations, combinations, and owners.
- Clinical risk: confirm source data, protocol deviations, endpoint definitions, and safety follow-up.
- Regulatory risk: review agency correspondence, meeting minutes, holds, commitments, and indication-specific pathways.
- IP risk: validate enforceable coverage, remaining life, ownership, and freedom to operate.
- CMC risk: assess process control, analytical methods, comparability, scale-up, supply, and cost of goods.
- Commercial risk: pressure-test differentiation, market access, competitive timing, and realistic adoption.
- Transaction risk: map rights, encumbrances, royalties, options, consent requirements, and change-of-control provisions.
8. Final go/no-go memorandum
CONDITIONAL GO
Proceed only through staged diligence. Before exclusivity or a binding offer, require a verified asset identity package, complete clinical data room, regulatory correspondence, patent-family schedule, freedom-to-operate opinion, CMC package, ownership chain, regional rights map, and risk-adjusted valuation. Any material mismatch between public records and source documents should pause the process until reconciled.
Method note: This report uses PatSnap Drug & Asset MCP, Clinical Trials MCP, and Company & Deal Intelligence MCP data retrieved on 2026-10-14. It is a screening and diligence framework, not medical, legal, regulatory, or investment advice.
