pharma · Acute Myeloid Leukemia · Narcolepsy · JAZZ
Jazz Pharmaceuticals Ireland Limited
Jazz Pharmaceuticals Ireland is a pharma organization headquartered in Dublin, IE. It trades on NYSE under ticker JAZZ. Primary therapeutic focus areas include Acute Myeloid Leukemia, Narcolepsy, Epilepsy, Acute Lymphobl
Phase 2 · small molecule · Meningioma
ONC201 is a small-molecule therapeutic candidate developed by Jazz Pharmaceuticals Ireland Limited for the treatment of meningioma, currently in Phase 2 clinical development. The program was terminated as of January 26, 2026, marking the end of its clinical advancement. Meningioma represents a significant unmet medical
Internal code 0137-24-FB
ONC201 is a small-molecule therapeutic candidate developed by Jazz Pharmaceuticals Ireland Limited for the treatment of meningioma, currently in Phase 2 clinical development. The program was terminated as of January 26, 2026, marking the end of its clinical advancement. Meningioma represents a significant unmet medical need, particularly for recurrent or treatment-resistant cases where therapeutic options remain limited. Jazz Pharmaceuticals' decision to discontinue ONC201 reflects a strategic portfolio reassessment, though the specific rationale for termination has not been disclosed. The program was supported by clinical trial NCT06012929, which evaluated the candidate in the meningioma indication. Prior to termination, ONC201 was positioned within a competitive landscape that includes several Phase 2 programs targeting meningioma, including Jazz's own JZP3507, B10 L-BPA Injection from Heron Therapeutics, and other investigational approaches. The termination of ONC201 does not affect Jazz Pharmaceuticals' broader meningioma pipeline strategy, as evidenced by the continued development of alternative candidates.
Meningioma represents a significant clinical challenge with substantial unmet medical need. While many meningiomas are benign, recurrent and atypical variants pose serious treatment challenges, with limited effective systemic therapeutic options beyond surgery and radiation therapy. The disease affects thousands of patients annually, and the lack of targeted medical therapies drives continued investment in novel approaches. ONC201's termination underscores the competitive and clinical hurdles in meningioma drug development, where efficacy and safety profiles must demonstrate clear advantages over existing standard-of-care approaches. The meningioma market remains attractive for pharmaceutical development due to the significant patient population and limited therapeutic alternatives, particularly for advanced or recurrent disease. Jazz Pharmaceuticals' continued investment in meningioma through alternative programs such as JZP3507 indicates the company's commitment to this indication despite ONC201's discontinuation. The competitive landscape includes multiple Phase 2 programs, suggesting active industry recognition of the therapeutic opportunity. ONC201's failure to advance highlights the challenges in translating preclinical promise into clinical efficacy and the rigorous evaluation required for CNS-targeted therapies in rare tumor indications.
ONC201 is a small-molecule therapeutic candidate classified within the broader category of investigational oncology agents. The specific mechanism of action, molecular target, and route of administration have not been disclosed in available information. As a small-molecule modality, ONC201 would be expected to be orally bioavailable or administered via parenteral routes, though this remains unconfirmed. The candidate was developed specifically for meningioma, a central nervous system tumor, suggesting potential CNS penetration properties were considered in its design.
Also known as: meningioma (disease), meningothelial cell tumour, primary meningeal tumour
Prevalence: Annual incidence: 1-9 / 100 000 (Germany) — source: Orphanet, validated.
A generally slow growing tumor attached to the dura mater. It is composed of neoplastic meningothelial (arachnoidal) cells. It typically occurs in adults, often women and it has a wide range of histopathological appearances. Of the various subtypes, meningothelial, fibrous and transitional meningiomas are the most common. Most meningiomas are WHO grade I tumors, and some are WHO grade II or III tumors. Most subtypes share a common clinical behavior, although some subtypes are more likely to recur and follow a more aggressive clinical course. (Adapted from WHO)
ClinicalTrials.gov lists 112 registered studies for Meningioma (AACT aggregate).
Phase breakdown: NA (63), PHASE2 (25), PHASE1/PHASE2 (8), EARLY_PHASE1 (5), PHASE1 (5), PHASE3 (3), PHASE4 (3)
Common investigational therapies:
Disease data sourced from MONDO Disease Ontology (MONDO:0016642), Orphanet — meningioma, NCT00003483, NCT00004483, NCT00589784, NCT00706810, NCT00859040, AACT (ClinicalTrials.gov aggregate), ClinicalTrials.gov, Open Targets Platform (CC BY 4.0).
Phase 2 enrollment and conduct
ONC201 Phase 2 clinical trial NCT06012929 was initiated to evaluate efficacy and safety in meningioma patients.
Program termination
ONC201 development program was terminated as of January 26, 2026, ending clinical advancement.
ONC201 operated within a competitive meningioma therapeutic landscape that includes multiple Phase 2 investigational programs. Heron Therapeutics is advancing B10 L-BPA Injection, a small-molecule candidate also in Phase 2 development for meningioma. Ningbo Cancer Hospital is conducting the EORTC 2334-BTG trial, another Phase 2 small-molecule program in the same indication. Jazz Pharmaceuticals itself maintains a parallel meningioma program with JZP3507, a small-molecule candidate also in Phase 2, suggesting the sponsor's diversified approach to the indication. Istituto Gentili S.r.l. is evaluating Stivarga 40 mg film-coated tablets (regorafenib), a small-molecule multi-kinase inhibitor, in Phase 2 meningioma studies. The competitive field demonstrates active industry engagement with meningioma as a therapeutic target, with multiple small-molecule approaches under investigation. ONC201's termination does not eliminate Jazz Pharmaceuticals' meningioma pipeline presence, as JZP3507 continues development. The Phase 2 status of all competing programs indicates that no approved meningioma-specific small-molecule therapies have yet emerged from this cohort, underscoring the developmental challenges in this indication.
| Therapy | Company | Mechanism | Status |
|---|---|---|---|
| B10 L-BPA Injection | Heron Therapeutics | small_molecule | phase_2 |
| EORTC 2334-BTG | Ningbo Cancer Hospital | small_molecule | phase_2 |
| JZP3507 | Jazz Pharmaceuticals Ireland Limited | small_molecule | phase_2 |
| Stivarga 40 mg film-coated tablets | Istituto Gentili S.r.l. | small_molecule | phase_2 |
| TRANEXAMIC ACID | — | Plasminogen inhibitor | Phase 3 |
| MIFEPRISTONE | — | Progesterone receptor antagonist | Phase 3 |
| VISTUSERTIB | — | Serine/threonine-protein kinase mTOR inhibitor | Phase 2 |
| VISMODEGIB | — | Smoothened homolog inhibitor | Phase 2 |
| REGORAFENIB | — | Fibroblast growth factor receptor 1 inhibitor | Phase 2 |
| PEMBROLIZUMAB | — | Programmed cell death protein 1 inhibitor | Phase 2 |
| PASIREOTIDE | — | Somatostatin receptor 1 agonist | Phase 2 |
| OCTREOTIDE | — | Somatostatin receptor agonist | Phase 2 |
| NIVOLUMAB | — | Programmed cell death protein 1 inhibitor | Phase 2 |
| LUTETIUM OXODOTREOTIDE LU-177 | — | Somatostatin receptor binding agent | Phase 2 |
| LUTETIUM OXODOTREOTIDE | — | Somatostatin receptor binding agent | Phase 2 |
| IRINOTECAN HYDROCHLORIDE | — | DNA topoisomerase I inhibitor | Phase 2 |
Additional associated therapies sourced from Open Targets Platform (CC0), linked to NovaPharmaNews drug profiles where matched.
Regulatory approval status and interactions for ONC201 have not been disclosed. The program's Phase 2 status prior to termination indicates it had not advanced to Phase 3 or regulatory submission stages. FDA, EMA, PMDA (Japan), and NMPA (China) regulatory pathways and any designations (such as orphan drug status or breakthrough therapy designation) are not yet disclosed. The termination of the program on January 26, 2026, concluded clinical development before any regulatory filing could occur.
ONC201 is a small-molecule investigational therapeutic candidate developed by Jazz Pharmaceuticals Ireland Limited for the treatment of meningioma, a central nervous system tumor. The program was terminated in January 2026 while in Phase 2 clinical development.
No, ONC201 is not approved. The program was terminated during Phase 2 clinical development and never advanced to regulatory submission or approval.
The specific mechanism of action for ONC201 has not been disclosed in available information.
ONC201 is developed by Jazz Pharmaceuticals Ireland Limited. The program was terminated in January 2026.
ONC201 was evaluated in clinical trial NCT06012929, a Phase 2 study in meningioma patients. Results from this trial have not yet been reported, and the program was terminated before completion.
ONC201 development was terminated as of January 26, 2026. The program is no longer in active clinical development.
ONC201 was in Phase 2 clinical development at the time of program termination in January 2026.
Yes, Jazz Pharmaceuticals is developing JZP3507, another small-molecule candidate for meningioma that remains in Phase 2 clinical development.
Competing Phase 2 meningioma programs include B10 L-BPA Injection (Heron Therapeutics), EORTC 2334-BTG (Ningbo Cancer Hospital), JZP3507 (Jazz Pharmaceuticals), and Stivarga 40 mg tablets (Istituto Gentili S.r.l.).
The specific rationale for ONC201's termination in January 2026 has not been disclosed by Jazz Pharmaceuticals.
Meningioma is a central nervous system tumor that can be benign or malignant. Recurrent and atypical meningiomas present significant clinical challenges with limited systemic therapeutic options beyond surgery and radiation, creating substantial unmet medical need.
No, ONC201 is not available for patients. The program was terminated during Phase 2 development and never received regulatory approval.
ONC201 is a small-molecule therapeutic candidate, a class of drugs typically designed for oral or parenteral administration.
The route of administration for ONC201 has not been disclosed in available information.
The date of first disclosure for ONC201 has not been specified in available information.
Orphan drug designation status for ONC201 has not been disclosed in available information.
ONC201 → Drug → Target → Indication → Company → Trials → Competitors
ONC201's termination in January 2026 reflects the substantial clinical and commercial challenges inherent in meningioma drug development. Despite the significant unmet medical need in this indication, the program did not advance beyond Phase 2, suggesting either insufficient efficacy signals, safety concerns, or strategic portfolio prioritization by Jazz Pharmaceuticals. The sponsor's continued investment in JZP3507 indicates that ONC201's discontinuation represents a selective portfolio decision rather than abandonment of the meningioma indication.
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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.