NCT05045027
- Objective
- Not yet disclosed
- Design
- Not yet disclosed
- Participants
- Not yet disclosed
- Primary endpoint
- Not yet disclosed
- Results
- Results not yet reported
pharma · Breast Cancer · Prostate Cancer · UTHR
United Therapeutics Europe
United Therapeutics Europe Ltd
United Therapeutics is a pharma organization headquartered in Silver Spring, USA. It trades on NYSE under ticker UTHR. Primary therapeutic focus areas include Breast Cancer, Prostate Cancer, Pulmonary Arterial Hypertensi
1000 Spring Street, Silver Spring, Maryland 20910, US HQ
1996 Founded
1,443 Employees
Public company Type
UTHR · NYSE Ticker
Company details
- Status
- Public
- HQ
- 1000 Spring Street, Silver Spring, Maryland 20910, US
- Founded
- 1996
- Employees
- 1,443
- Programs
- 1032
- Drugs
- 612
- Patents
- 3720
Biospecimen Collection
Phase 1 · other · Glioma
SITOIGANAP is an autologous glioma tumor cell lysate (inactivated) immunotherapy developed by United Therapeutics Europe Ltd for the treatment of glioma. The drug represents a personalized cancer immunotherapy approach, utilizing inactivated tumor cell lysates derived from individual patients to stimulate anti-tumor im
Internal code 21-000514
At a glance
- Sponsor
- United Therapeutics Europe Ltd
- Phase
- Phase 1
- Modality
- other
- Indication
- Glioma
- Status
- active
- Trials
- 1
Executive summary
SITOIGANAP is an autologous glioma tumor cell lysate (inactivated) immunotherapy developed by United Therapeutics Europe Ltd for the treatment of glioma. The drug represents a personalized cancer immunotherapy approach, utilizing inactivated tumor cell lysates derived from individual patients to stimulate anti-tumor immune responses. Currently in Phase 1 development, the program (internal code 21-000514) is actively enrolling patients under clinical trial NCT05045027. The therapeutic agent is classified within the antineoplastic and immunomodulating agents category (ATC L01). Regulatory history shows that SITOIGANAP received an application withdrawal in the European Union, with the marketing authorization holder listed as Epitopoietic Research Corporation-Belgium (E.R.C.) under EMEA product number H/C/003693. The most recent program milestone was recorded on 14 November 2025, though specific milestone details remain undisclosed. United Therapeutics Europe Ltd's development strategy appears focused on advancing personalized immunotherapy approaches, with SITOIGANAP representing an investigational candidate in early-stage clinical evaluation for this difficult-to-treat malignancy.
Why this program matters
Glioma represents a significant unmet medical need with limited treatment options and poor prognosis, particularly for high-grade tumors. The global glioma therapeutics market remains dominated by conventional chemotherapy and radiation approaches, with limited immunotherapy penetration compared to other solid tumors. Personalized autologous cell-based therapies offer a mechanistically distinct approach to conventional small-molecule and monoclonal antibody therapeutics, potentially addressing immune evasion mechanisms inherent to gliomas. The competitive landscape includes approved agents such as AFINITOR (everolimus, Novartis), INLYTA (axitinib, Pfizer), and KYPROLIS (carfilzomib, Amgen), though these represent off-label or indirect applications in glioma management. The patient population for glioma immunotherapy remains underserved, with significant mortality and morbidity burden. Commercial significance is substantial given the high unmet need, limited approved immunotherapies specifically indicated for glioma, and potential for premium pricing in personalized medicine segments. The autologous cell therapy modality, if successful, could establish a new treatment paradigm and differentiate United Therapeutics Europe Ltd within the oncology immunotherapy space. However, manufacturing complexity, patient stratification requirements, and regulatory pathway clarity for personalized cell therapies present both technical and commercial challenges to market adoption.
Drug intelligence
Drug Class: Autologous cell therapy; antineoplastic and immunomodulating agent (ATC L01)
Modality: Other (cell-based immunotherapy)
Composition: Autologous glioma tumor cell lysates (inactivated)
Mechanism of Action: Not yet disclosed
Molecular Target: Not yet disclosed
Route of Administration: Not yet disclosed
Therapeutic Class: Antineoplastic and immunomodulating agents (L01)
Related Therapies: Autologous cell therapies and personalized cancer immunotherapies; checkpoint inhibitors (nivolumab, pembrolizumab) represent alternative immunotherapy approaches in oncology, though not specifically approved for glioma monotherapy
First Approval Status: Application withdrawn in European Union; not approved in any jurisdiction based on disclosed facts
Patent Status: Not yet disclosed
glioma
Also known as: glial neoplasm, glial tumor, glial tumour, neoplasm of neuroglia, neoplasm of the neuroglia, neuroglial neoplasm
Prevalence: Point prevalence: 1-5 / 10 000 (Europe) — source: Orphanet, validated.
Overview
A benign or malignant brain and spinal cord tumor that arises from glial cells (astrocytes, oligodendrocytes, ependymal cells). Tumors that arise from astrocytes are called astrocytic tumors or astrocytomas. Tumors that arise from oligodendrocytes are called oligodendroglial tumors. Tumors that arise from ependymal cells are called ependymomas.
Treatment landscape
ClinicalTrials.gov lists 517 registered studies for Glioma (AACT aggregate).
Phase breakdown: NA (265), PHASE1 (85), PHASE2 (82), PHASE1/PHASE2 (33), EARLY_PHASE1 (29), PHASE3 (13), PHASE2/PHASE3 (7), PHASE4 (3)
Common investigational therapies:
- Temozolomide
- Bevacizumab
- Chemotherapy
- Placebo
- Vorasidenib
- Gemcitabine
- Cyclophosphamide
- Pembrolizumab
- Irinotecan
- Thalidomide
Disease data sourced from MONDO Disease Ontology (MONDO:0021042), Orphanet — glioma, NCT00001150, NCT00001336, NCT00001341, NCT00001444, NCT00001500, AACT (ClinicalTrials.gov aggregate), ClinicalTrials.gov, NCT00001148, NCT00001171, NCT00001502, NCT00001573, NCT00009035, Open Targets Platform (CC BY 4.0).
Clinical development timeline
- Phase 1TBD
Phase 1 enrollment ongoing
SITOIGANAP Phase 1 trial (NCT05045027) remains active with latest milestone recorded 14 November 2025; specific enrollment or interim data milestones not yet disclosed.
Competitive landscape
The competitive landscape for glioma therapeutics includes multiple approved agents across diverse mechanisms, though few are specifically indicated for glioma. AFINITOR (everolimus, Novartis) is an mTOR inhibitor approved for various malignancies and used off-label in glioma management. INLYTA (axitinib, Pfizer) is a tyrosine kinase inhibitor with broader oncology applications. KYPROLIS (carfilzomib, Janssen/Amgen) is a proteasome inhibitor primarily used in hematologic malignancies. IMBRUVICA (ibrutinib, Janssen-Cilag) is a Bruton tyrosine kinase inhibitor approved for hematologic cancers. LYSODREN (mitotane) is an adrenolytic agent with limited glioma application. OFEV (nintedanib, Boehringer Ingelheim) is a tyrosine kinase inhibitor primarily indicated for idiopathic pulmonary fibrosis. VYXEOS LIPOSOMAL (daunorubicin/cytarabine, Jazz Pharmaceuticals) is approved for acute myeloid leukemia. UNITUXIN (dinutuximab, United Therapeutics Europe Ltd) is a monoclonal antibody for neuroblastoma, representing the sponsor's existing oncology portfolio. PACLITAXEL ACCORD (paclitaxel, Accord Healthcare) is a conventional chemotherapy agent. LYNOZYFIC (linezolid, Regeneron) is an antibiotic with off-label oncology applications. ARX-IMATINIB (imatinib, Alphapharm) is a tyrosine kinase inhibitor. SITOIGANAP's personalized autologous cell therapy approach represents a mechanistically distinct positioning versus these predominantly small-molecule and monoclonal antibody competitors, though clinical efficacy and regulatory approval status remain to be established.
| Therapy | Company | Mechanism | Status |
|---|---|---|---|
| PFIZER AUSTRALIA PTY LTD | Pfizer Australia Pty Ltd | — | approved |
| IMBRUVICA | Janssen-Cilag Pty Ltd | — | approved |
| AFINITOR | Novartis Pharmaceuticals | — | approved |
| LYSODREN | S.A. | — | approved |
| INLYTA | Pfizer Australia Pty Ltd | — | approved |
| LYNOZYFIC | Regeneron UK Limited | — | approved |
| VYXEOS LIPOSOMAL (PREVIOUSLY VYXEOS) | Jazz Pharmaceuticals Ireland Limited | — | approved |
| KYPROLIS | Amgen | — | approved |
| UNITUXIN | United Therapeutics Europe Ltd | — | approved |
| PACLITAXEL ACCORD | Accord Healthcare Pty. | — | approved |
| OFEV | Boehringer Ingelheim Pty Ltd | — | approved |
| ARX-IMATINIB | Alphapharm Pty Ltd | — | approved |
| EVEROLIMUS | — | FK506-binding protein 1A inhibitor | Approved |
| CARMUSTINE | — | Glutathione reductase inhibitor | Approved |
| BEVACIZUMAB | — | Vascular endothelial growth factor A inhibitor | Approved |
| VINCRISTINE SULFATE | — | Tubulin inhibitor | Phase 3 |
| VINCRISTINE | — | Tubulin inhibitor | Phase 3 |
| TRANEXAMIC ACID | — | Plasminogen inhibitor | Phase 3 |
| TRABEDERSEN | — | Transforming growth factor beta-2 mRNA antisense inhibitor | Phase 3 |
| TOVORAFENIB | — | RAF serine/threonine protein kinase inhibitor | Phase 3 |
Additional associated therapies sourced from Open Targets Platform (CC0), linked to NovaPharmaNews drug profiles where matched.
Regulatory intelligence
European Union: SITOIGANAP (autologous glioma tumor cell lysates, inactivated) received an application withdrawal under EMEA product number H/C/003693. Marketing authorization holder: Epitopoietic Research Corporation-Belgium (E.R.C.). No authorization dates are recorded, indicating the application was withdrawn prior to approval. Current regulatory status in the EU is inactive.
United States (FDA): Regulatory status not yet disclosed.
Japan (PMDA): Regulatory status not yet disclosed.
China (NMPA): Regulatory status not yet disclosed.
Development Status: The program remains in Phase 1 clinical development under sponsor United Therapeutics Europe Ltd, with active enrollment in trial NCT05045027 as of the latest milestone (14 November 2025). No regulatory filings, approvals, or label expansions have been disclosed in other jurisdictions.
Clinical evidence summary
Key questions answered
What is SITOIGANAP and what disease does it treat?
SITOIGANAP is an autologous glioma tumor cell lysate (inactivated) immunotherapy in development for the treatment of glioma. It is a personalized cancer immunotherapy derived from inactivated tumor cells from individual patients.
Who is developing SITOIGANAP?
SITOIGANAP is being developed by United Therapeutics Europe Ltd. The marketing authorization holder is listed as Epitopoietic Research Corporation-Belgium (E.R.C.).
What is the current development status of SITOIGANAP?
SITOIGANAP is currently in Phase 1 clinical development with active patient enrollment in trial NCT05045027. The most recent program milestone was recorded on 14 November 2025.
Is SITOIGANAP approved by regulatory agencies?
SITOIGANAP is not currently approved. The application was withdrawn in the European Union. Regulatory status in the United States, Japan, and China has not been disclosed.
How does SITOIGANAP work mechanistically?
The specific mechanism of action for SITOIGANAP has not been disclosed. It is classified as an antineoplastic and immunomodulating agent (ATC L01) based on its therapeutic class.
What is the route of administration for SITOIGANAP?
The route of administration for SITOIGANAP has not been disclosed.
What clinical trial is evaluating SITOIGANAP?
SITOIGANAP is being evaluated in clinical trial NCT05045027. Specific trial design, objectives, endpoints, and enrollment details have not been disclosed.
What is the molecular target of SITOIGANAP?
The molecular target of SITOIGANAP has not been disclosed.
What is the therapeutic class of SITOIGANAP?
SITOIGANAP is classified as an antineoplastic and immunomodulating agent (ATC L01), representing a cell-based immunotherapy approach to cancer treatment.
Are there competing therapies for glioma?
Yes. Approved agents used in glioma management include AFINITOR (everolimus), INLYTA (axitinib), KYPROLIS (carfilzomib), and others, though most are not specifically indicated for glioma and represent off-label use or alternative mechanisms.
What is the internal program code for SITOIGANAP?
The internal program code for SITOIGANAP is 21-000514, and it is also referred to as the 'Biospecimen Collection' program.
Does SITOIGANAP have a development partner?
No development partner has been disclosed for SITOIGANAP. United Therapeutics Europe Ltd is listed as the sole sponsor.
What is the EMEA product number for SITOIGANAP?
The EMEA product number for SITOIGANAP is H/C/003693, associated with the withdrawn application in the European Union.
What are the peak sales projections for SITOIGANAP?
Peak sales projections for SITOIGANAP have not been disclosed.
What is the patent status of SITOIGANAP?
Patent status information for SITOIGANAP has not been disclosed.
What patient population is targeted by SITOIGANAP?
SITOIGANAP targets patients with glioma. Specific patient subpopulations, disease stages, or biomarker criteria have not been disclosed.
Entity relationship graph
Biospecimen Collection → Drug → Target → Indication → Company → Trials → Competitors
Companies
Indication
Trials
Analyst insights
Strategic Positioning: United Therapeutics Europe Ltd's development of SITOIGANAP reflects a strategic focus on personalized immunotherapy approaches within oncology. The autologous cell therapy modality differentiates the program from the sponsor's existing monoclonal antibody portfolio (UNITUXIN) and positions the company within the emerging personalized medicine segment.
Regulatory Pathway Challenges: The prior application withdrawal in the EU suggests potential regulatory, manufacturing, or efficacy challenges in the personalized cell therapy pathway. The transition from withdrawn application to Phase 1 trial enrollment indicates either a revised development strategy, new clinical data, or regulatory guidance clarification. The lack of disclosed regulatory interactions in other jurisdictions (FDA, PMDA, NMPA) limits visibility into global development strategy.
Competitive Implications: Glioma remains an underserved indication with limited approved immunotherapies. Success of SITOIGANAP could establish a new treatment paradigm and differentiate United Therapeutics Europe Ltd within oncology immunotherapy. However, the competitive landscape includes multiple approved agents used off-label, and the clinical efficacy advantage of autologous cell therapy versus checkpoint inhibitors or conventional chemotherapy remains unproven.
Manufacturing and Scalability: Autologous cell therapies require patient-specific manufacturing, creating operational complexity, cost barriers, and potential supply chain constraints. These factors may limit market penetration despite clinical success.
Future Catalysts: Phase 1 trial completion and interim safety/efficacy data release; advancement to Phase 2; regulatory interactions with FDA or EMA; potential partnerships or licensing agreements; manufacturing process optimization announcements.
Quick answers
Concise, citable answers optimized for AI answer engines.
- What is SITOIGANAP?
- Autologous glioma tumor cell lysate (inactivated) immunotherapy for glioma treatment.
- Who manufactures SITOIGANAP?
- Developed by United Therapeutics Europe Ltd; MAH is Epitopoietic Research Corporation-Belgium.
- What indication does SITOIGANAP treat?
- Glioma
- What is the development phase of SITOIGANAP?
- Phase 1 (active enrollment)
- Is SITOIGANAP approved?
- No; application withdrawn in EU; not approved elsewhere.
- What is the mechanism of action of SITOIGANAP?
- Not yet disclosed
- What is the molecular target of SITOIGANAP?
- Not yet disclosed
- What is the route of administration?
- Not yet disclosed
- What is the therapeutic class?
- Antineoplastic and immunomodulating agent (ATC L01)
- What is the modality?
- Cell-based immunotherapy (autologous)
- What clinical trial evaluates SITOIGANAP?
- NCT05045027
- Does SITOIGANAP have a development partner?
- No partner disclosed
- What is the internal program code?
- 21-000514 (Biospecimen Collection)
- What is the EMEA product number?
- H/C/003693
- When was the latest milestone?
- 14 November 2025
- What are peak sales projections?
- Not yet disclosed
- What is the patent status?
- Not yet disclosed
- Who is the lead investigator?
- Not yet disclosed
- What is the consensus analyst position?
- Not yet disclosed
- What license type applies?
- Not yet disclosed
- Are there competing glioma therapies?
- Yes; AFINITOR, INLYTA, KYPROLIS, and others used off-label.
- What is the regulatory status in FDA?
- Not yet disclosed
Evidence & sources
- ClinicalTrials.gov NCT05045027 (clinicaltrials)
- autologous glioma tumor cell lysates (inactivated) EU status (ema)
- Source: phase (source_attribution)
- MONDO Disease Ontology (MONDO:0021042) (mondo)
- Orphanet — glioma (orphanet)
- NCT00001150 (clinicaltrials_gov)
- NCT00001336 (clinicaltrials_gov)
- NCT00001341 (clinicaltrials_gov)
- NCT00001444 (clinicaltrials_gov)
- NCT00001500 (clinicaltrials_gov)
- AACT (ClinicalTrials.gov aggregate) (aact)
- ClinicalTrials.gov (clinicaltrials_gov)
- NCT00001148 (clinicaltrials_gov)
- NCT00001171 (clinicaltrials_gov)
- NCT00001502 (clinicaltrials_gov)
- NCT00001573 (clinicaltrials_gov)
- NCT00009035 (clinicaltrials_gov)
- Open Targets Platform (opentargets)
Intelligence compiled from public regulatory and clinical sources (FDA, EMA, ClinicalTrials.gov and company disclosures). Figures may be editorial or analyst estimates; verify against primary sources before relying on them.