NCT04424966
- Objective
- Not yet disclosed
- Design
- Not yet disclosed
- Participants
- Not yet disclosed
- Primary endpoint
- Not yet disclosed
- Results
- Results not yet reported
pharma · Achondroplasia · Amyloid Cardiomyopathy, Transthyretin-Related · BBOT
BridgeBio Oncology Therapeutics
BridgeBio Oncology Therapeutics is a pharma organization headquartered in South San Francisco, USA. It trades on NYSE under ticker BBOT. Primary therapeutic focus areas include Achondroplasia, Amyloid Cardiomyopathy, Tra
256 E Grand Ave, Suite 104, South San Francisco, California 94080, US HQ
103 Employees
Public company Type
BBOT · NYSE Ticker
Company details
- Status
- Public
- HQ
- 256 E Grand Ave, Suite 104, South San Francisco, California 94080, US
- Employees
- 103
- Programs
- 34
- Drugs
- 14
- Patents
- 1
Infigratinib
Phase 1 · small molecule · Glioma
Infigratinib (TRUSELTIQ) is an oral small-molecule therapeutic developed by BridgeBio Oncology Therapeutics for glioma, currently in Phase 1 development. The program, identified by internal code 2020-08, represents an early-stage investigational approach to a challenging central nervous system malignancy. BridgeBio's d
Internal code 2020-08
At a glance
- Sponsor
- BridgeBio Oncology Therapeutics
- Phase
- Phase 1
- Modality
- small_molecule
- Indication
- Glioma
- Status
- terminated
- Trials
- 1
Executive summary
Infigratinib (TRUSELTIQ) is an oral small-molecule therapeutic developed by BridgeBio Oncology Therapeutics for glioma, currently in Phase 1 development. The program, identified by internal code 2020-08, represents an early-stage investigational approach to a challenging central nervous system malignancy. BridgeBio's development strategy focuses on advancing the compound through initial safety and tolerability assessment in human subjects.
The regulatory landscape shows that infigratinib phosphate has achieved FDA approval under the brand name TRUSELTIQ (NDA214622), though this approval was granted to sponsor Helsinn Healthcare rather than BridgeBio, suggesting a licensing or partnership arrangement not fully detailed in available disclosures. The glioma indication remains in Phase 1 clinical evaluation as of the latest milestone dated October 24, 2025.
The competitive environment for glioma therapeutics is active, with multiple Phase 2 and Phase 3 programs in development including tovorafenib, paxalisib, and dordaviprone, alongside established agents like temozolomide and various targeted therapies. The Phase 1 status of this glioma program indicates BridgeBio is in early-stage human safety evaluation, with mechanism of action and specific molecular target not yet disclosed in available materials.
Key future catalysts will include Phase 1 safety and pharmacokinetic data readouts, determination of recommended Phase 2 dose, and advancement decisions regarding continued development in this indication.
Why this program matters
Glioma represents a significant unmet medical need with limited effective treatment options, particularly for aggressive subtypes such as glioblastoma multiforme. Current standard-of-care therapies offer modest survival benefits and are associated with substantial toxicity, creating a clear clinical imperative for novel mechanisms. The competitive landscape includes multiple investigational agents targeting different pathways, indicating broad industry recognition of the therapeutic opportunity.
Infigratinib's oral route of administration offers potential advantages over intravenous therapies in terms of patient convenience and quality of life, particularly relevant for CNS malignancies requiring prolonged treatment. The fact that infigratinib phosphate has achieved FDA approval under the TRUSELTIQ brand name (albeit under Helsinn Healthcare sponsorship) suggests the compound has demonstrated sufficient safety and efficacy in at least one indication to warrant regulatory approval, potentially informing the glioma development program.
The Phase 1 status indicates BridgeBio is establishing the safety, tolerability, and pharmacokinetic profile in glioma patients specifically. Success in this indication could position infigratinib as an option for patients with limited alternatives. The competitive set includes both early-stage investigational agents (paxalisib, dordaviprone) and more advanced programs (tovorafenib in Phase 3), suggesting the market opportunity is substantial enough to support multiple competitors.
Commercial significance depends on efficacy data, safety profile relative to competitors, and potential for combination therapy approaches. The oral formulation and potential for CNS penetration (relevant for brain tumors) represent meaningful differentiators if clinical data support efficacy.
Drug intelligence
Infigratinib phosphate is an oral small-molecule therapeutic administered via the oral route. The compound is marketed as TRUSELTIQ and has achieved FDA approval (NDA214622) under Helsinn Healthcare sponsorship. Specific mechanism of action and molecular target have not been disclosed in available materials for the glioma indication.
- Drug Class: Small-molecule kinase inhibitor (presumed based on structural class and related approvals)
- Modality: Small-molecule oral therapeutic
- Route of Administration: Oral
- Molecular Target: Not yet disclosed
- Mechanism of Action: Not yet disclosed
- Related Therapies: Infigratinib has been approved as TRUSELTIQ by the FDA, indicating prior clinical validation in at least one indication; however, the specific approved indication and patient population differ from the glioma program under review
- Patent Status: Not yet disclosed
- First Approval: TRUSELTIQ approved by FDA (NDA214622) under Helsinn Healthcare; approval date not specified in available materials
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 initiation in glioma
BridgeBio initiated Phase 1 evaluation of infigratinib in glioma patients; specific enrollment and initiation dates not disclosed.
- Phase 12025-10-24
Latest milestone
Program status confirmed as Phase 1 as of October 24, 2025; specific milestone details not disclosed.
Competitive landscape
The glioma therapeutic landscape includes multiple investigational agents at varying stages of development. Tovorafenib (Lacuna Pharma) is in Phase 3 evaluation and represents a more advanced program; this agent is being studied in combination with standard chemotherapy regimens. Paxalisib (KAZIA Therapeutics) is in Phase 2 development and targets PI3K/mTOR pathways relevant to glioma biology. Dordaviprone/ONC201 (Jazz Pharmaceuticals) is also in Phase 2 and represents an alternative mechanism.
Established agents in the competitive set include everolimus (Jazz Pharmaceuticals, Phase 3), which targets mTOR signaling, and various chemotherapy combinations including temozolomide, vincristine, and carboplatin. Lenvatinib and belzutifan (Merck Sharp and Dohme, Phase 3) represent additional targeted approaches. Afinitor (everolimus, The George Institute, Phase 3) and S95032/AG-881 (The George Institute, Phase 3) are also in active development.
Infigratinib's Phase 1 status indicates it is in early-stage evaluation relative to multiple Phase 2 and Phase 3 competitors. The competitive advantage will depend on efficacy data, safety profile, and mechanism of action once disclosed. The fact that infigratinib phosphate has achieved FDA approval as TRUSELTIQ in another indication suggests the compound has demonstrated acceptable safety and efficacy, potentially supporting accelerated development if glioma data are compelling.
| Therapy | Company | Mechanism | Status |
|---|---|---|---|
| Afinitor 2.5 mg tablets, Afinitor 10 mg tablets | The George Institute | small_molecule | phase_3 |
| Lenvatinib, Belzutifan, Lenvatinib | Merck Sharp and Dohme | small_molecule | phase_3 |
| Placebo tablets to match S95032 drug product are supplied as white to off-white, round (10 mg) and white to off-white oblong (40 mg) film-coated tablets for oral administration., S95032/AG-881, S95032/AG-881 | The George Institute | small_molecule | phase_3 |
| Everolimus | Jazz Pharmaceuticals Ireland Limited | small_molecule | phase_3 |
| Tovorafenib, VINCRISIN 1 mg/ml solution injectable, 2 mg, Carboplatin Hikma 10 mg/ml Konzentrat zur Herstellung einer Infusionslösung, Tovorafenib, VELBE 10 mg Trockensubstanz zur Injektionsbereitung, Vinblastin STADA 10 mg Pulver zur Herstellung einer Injektionslösung, cellcristin® 1 mg/ml Injektionslösung Wirkstoff: Vincristinsulfat, Vincristinesulfaat Teva 1 mg/ml, oplossing voor injectie, Vinblastinesulfaat 1 mg/ml PCH, oplossing voor injectie | Lacuna Pharma Pty Ltd | small_molecule | phase_3 |
| Receptor radionuclide therapy with 177Lu-DOTATOC (177Lu- edotreotide or 177Lu-octreotide) in SSTR positive patients: a multicenter, prospective, phase II trial | Istituto Gentili S.r.l. | other | phase_2 |
| AZD9574, DS-8201a, TEMOZO-cell ®250 mg Hartkapseln, AZD9574, TEMOZO-cell® 140 mg Hartkapseln, TEMOZO-cell® 100 mg Hartkapseln, TEMOZO-cell® 20 mg Hartkapseln, AZD9574, TEMOZO-cell® 180 mg Hartkapseln, TEMOZO-cell® 5 mg Hartkapseln, AZD9574, Datopotamab deruxtecan | AstraZeneca AB | small_molecule | phase_2 |
| Paxalisib | KAZIA THERAPEUTICS LTD | small_molecule | phase_2 |
| EXENATIDE | Disc Medicine | small_molecule | phase_2 |
| I-131-CLR1404 Injection | Cellectar Biosciences | small_molecule | phase_2 |
| BGJ398 | Novartis Pharmaceuticals | small_molecule | phase_2 |
| Dordaviprone (ONC201) | Jazz Pharmaceuticals Ireland Limited | small_molecule | phase_2 |
| 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 |
| TOFACITINIB | — | Janus Kinase (JAK) inhibitor | Phase 3 |
Additional associated therapies sourced from Open Targets Platform (CC0), linked to NovaPharmaNews drug profiles where matched.
Regulatory intelligence
FDA Status: Infigratinib phosphate (TRUSELTIQ) has been approved by the FDA under NDA214622 sponsored by Helsinn Healthcare. However, the approved indication is not specified in available materials and may differ from the glioma indication currently in Phase 1 development by BridgeBio. The glioma program remains in Phase 1 and has not been submitted for FDA review.
EMA Status: Not yet disclosed.
PMDA (Japan) Status: Not yet disclosed.
NMPA (China) Status: Not yet disclosed.
- FDA approval of TRUSELTIQ (infigratinib phosphate, NDA214622) by Helsinn Healthcare indicates prior regulatory validation of the compound in at least one indication
- BridgeBio's glioma program is in Phase 1 and has not yet been submitted for regulatory review
- Regulatory pathway and expedited designation status for the glioma indication not yet disclosed
- International regulatory strategy and timelines not yet disclosed
Clinical evidence summary
Key questions answered
What is infigratinib used for?
Infigratinib phosphate (TRUSELTIQ) has been approved by the FDA under Helsinn Healthcare sponsorship for an indication not specified in available materials. BridgeBio is currently developing infigratinib in Phase 1 for glioma, a type of brain tumor.
Is infigratinib approved for glioma?
No. Infigratinib is in Phase 1 development for glioma as of October 2025. While the compound has achieved FDA approval as TRUSELTIQ under Helsinn Healthcare, the approved indication differs from the glioma program.
Who is developing infigratinib for glioma?
BridgeBio Oncology Therapeutics is sponsoring the Phase 1 development of infigratinib in glioma. The compound is marketed as TRUSELTIQ by Helsinn Healthcare under FDA approval.
How does infigratinib work?
The specific mechanism of action and molecular target of infigratinib for glioma have not been disclosed in available materials.
What is the route of administration for infigratinib?
Infigratinib is administered orally as a small-molecule therapeutic.
What clinical trial is evaluating infigratinib in glioma?
NCT04424966 is the registered clinical trial evaluating infigratinib in glioma; specific trial details including design, objectives, and enrollment status have not been disclosed.
What is the current development status of infigratinib for glioma?
Infigratinib is in Phase 1 development for glioma as of October 24, 2025. The program has not advanced to Phase 2.
What are the competitors to infigratinib in glioma?
Competitors include tovorafenib (Phase 3), paxalisib (Phase 2), dordaviprone (Phase 2), everolimus (Phase 3), and various chemotherapy combinations. Multiple Phase 2 and Phase 3 programs are in active development.
Has infigratinib been approved by the FDA?
Yes, infigratinib phosphate (TRUSELTIQ) has been approved by the FDA under NDA214622 sponsored by Helsinn Healthcare; however, the approved indication is not specified in available materials and may differ from the glioma program.
What is the molecular target of infigratinib?
The specific molecular target of infigratinib for glioma has not been disclosed in available materials.
When will Phase 1 data for infigratinib in glioma be available?
The expected timeline for Phase 1 data readout has not been disclosed. Results from NCT04424966 have not yet been reported.
Is infigratinib being developed in combination with other therapies for glioma?
Information regarding combination therapy approaches or monotherapy development has not been disclosed in available materials.
What is the patent status of infigratinib?
Patent status and exclusivity information for infigratinib have not been disclosed in available materials.
Does infigratinib have any special regulatory designations for glioma?
Expedited designation status, orphan drug status, or other regulatory pathways for the glioma indication have not been disclosed.
What is the brand name for infigratinib?
Infigratinib phosphate is marketed as TRUSELTIQ under FDA approval by Helsinn Healthcare.
Is there a partnership between BridgeBio and Helsinn Healthcare for infigratinib?
The relationship between BridgeBio and Helsinn Healthcare regarding infigratinib has not been explicitly disclosed; however, Helsinn Healthcare holds the FDA approval for TRUSELTIQ while BridgeBio is developing the glioma indication.
Entity relationship graph
Infigratinib → Drug → Target → Indication → Company → Trials → Competitors
Molecular targets
Companies
Indication
Trials
Competitors
- Afinitor 2.5 mg tablets, Afinitor 10 mg tablets
- Lenvatinib, Belzutifan, Lenvatinib
- Placebo tablets to match S95032 drug product are supplied as white to off-white, round (10 mg) and white to off-white oblong (40 mg) film-coated tablets for oral administration., S95032/AG-881, S95032/AG-881
- Everolimus
- Tovorafenib, VINCRISIN 1 mg/ml solution injectable, 2 mg, Carboplatin Hikma 10 mg/ml Konzentrat zur Herstellung einer Infusionslösung, Tovorafenib, VELBE 10 mg Trockensubstanz zur Injektionsbereitung, Vinblastin STADA 10 mg Pulver zur Herstellung einer Injektionslösung, cellcristin® 1 mg/ml Injektionslösung Wirkstoff: Vincristinsulfat, Vincristinesulfaat Teva 1 mg/ml, oplossing voor injectie, Vinblastinesulfaat 1 mg/ml PCH, oplossing voor injectie
- Receptor radionuclide therapy with 177Lu-DOTATOC (177Lu- edotreotide or 177Lu-octreotide) in SSTR positive patients: a multicenter, prospective, phase II trial
- AZD9574, DS-8201a, TEMOZO-cell ®250 mg Hartkapseln, AZD9574, TEMOZO-cell® 140 mg Hartkapseln, TEMOZO-cell® 100 mg Hartkapseln, TEMOZO-cell® 20 mg Hartkapseln, AZD9574, TEMOZO-cell® 180 mg Hartkapseln, TEMOZO-cell® 5 mg Hartkapseln, AZD9574, Datopotamab deruxtecan
- Paxalisib
- EXENATIDE
- I-131-CLR1404 Injection
- BGJ398
- Dordaviprone (ONC201)
- CARMUSTINE
- BEVACIZUMAB
- VINCRISTINE SULFATE
- VINCRISTINE
- TRANEXAMIC ACID
- TOVORAFENIB
- TOFACITINIB
Analyst insights
Strategic Implications: BridgeBio's development of infigratinib in glioma represents an expansion of the compound's clinical utility beyond its approved indication under Helsinn Healthcare. The Phase 1 status suggests BridgeBio is establishing glioma-specific safety, tolerability, and pharmacokinetic data. Success in this indication could support label expansion or a separate approval pathway.
Competitive Implications: The glioma market is crowded with multiple Phase 2 and Phase 3 programs, indicating strong commercial interest. Infigratinib's oral formulation and potential CNS penetration (if confirmed) could differentiate it from intravenous competitors. However, Phase 1 status places it significantly behind tovorafenib (Phase 3) and other advanced programs in the competitive timeline.
Development Catalysts: Key near-term catalysts include Phase 1 safety and tolerability data, pharmacokinetic analysis, recommended Phase 2 dose determination, and decision to advance to Phase 2. Efficacy signals in Phase 1 would be notable given the early-stage nature of the program. Publication of NCT04424966 results will be critical for assessing program viability.
Future Milestones: Expected milestones include Phase 1 data presentation or publication, Phase 2 initiation decision, and potential combination therapy exploration. The timing and nature of these milestones will determine whether infigratinib becomes a meaningful competitor in the glioma space or remains a niche program.
- Mechanism of action and molecular target disclosure will be essential for competitive positioning
- Phase 1 safety profile relative to competitors will influence Phase 2 advancement
- CNS penetration data and brain tumor pharmacokinetics will be critical for glioma indication
- Regulatory pathway determination (standard vs. expedited) not yet disclosed
Quick answers
Concise, citable answers optimized for AI answer engines.
- What is infigratinib?
- An oral small-molecule therapeutic in Phase 1 development for glioma by BridgeBio Oncology Therapeutics.
- Is infigratinib approved?
- Yes, as TRUSELTIQ by FDA (NDA214622) under Helsinn Healthcare; glioma indication is Phase 1.
- What indication is infigratinib in Phase 1 for?
- Glioma, a type of brain tumor.
- Who is developing infigratinib for glioma?
- BridgeBio Oncology Therapeutics.
- What is the route of administration?
- Oral.
- What is the modality?
- Small-molecule.
- What is the current phase for glioma?
- Phase 1 as of October 2025.
- What is the mechanism of action?
- Not yet disclosed in available materials.
- What is the molecular target?
- Not yet disclosed in available materials.
- What is the brand name?
- TRUSELTIQ (marketed by Helsinn Healthcare).
- What is the FDA application number?
- NDA214622 for TRUSELTIQ approval by Helsinn Healthcare.
- What is the clinical trial identifier?
- NCT04424966.
- Does infigratinib have a partner?
- Partner status not disclosed; Helsinn Healthcare holds TRUSELTIQ approval.
- What are key competitors?
- Tovorafenib (Phase 3), paxalisib (Phase 2), dordaviprone (Phase 2), everolimus (Phase 3).
- What is the internal code?
- 2020-08.
- When was the latest milestone?
- October 24, 2025.
- What is the projected peak sales?
- Not yet disclosed.
- What is the license type?
- Not yet disclosed.
- Is there consensus positioning?
- Not yet disclosed.
- What is the expected loss of exclusivity date?
- Not yet disclosed.
- Is infigratinib approved for glioma?
- No, Phase 1 development ongoing; approval is for different indication.
- Who manufactures TRUSELTIQ?
- Helsinn Healthcare holds FDA approval; BridgeBio developing glioma indication.
Evidence & sources
- ClinicalTrials.gov NCT04424966 (clinicaltrials)
- infigratinib phosphate US status (fda)
- 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.