Jazz Pharmaceuticals plc

JAZZ

Held by 7 specialist biotech funds

High Convergence

7 funds opened new positions. Next phase3 readout (Brincidofovir): Jun 2016. Short interest: 5.7% of float.

BiotechnologyCEO: Renee D. GalaIPO: 2007Website ↗

Jazz Pharmaceuticals plc, a biopharmaceutical company, identifies, develops, and commercializes pharmaceutical products for various unmet medical needs in the United States, Europe, and internationally. The company has a portfolio of products and product candidates with a focus in the areas of neuroscience, including sleep medicine and movement disorders; and in oncology, including hematologic and solid tumors. Its lead marketed products include Xyrem, an oral solution for the treatment of cataplexy and excessive daytime sleepiness (EDS) in narcolepsy patients seven years of age and older; Sunosi for the treatment of EDS in patients with narcolepsy and obstructive sleep apnea; Erwinaze to treat acute lymphoblastic leukemia; Defitelio for the treatment of adult and pediatric patients with hepatic veno-occlusive disease; Vyxeos liposome for injection, a product for the treatment of adults with newly-diagnosed therapy-related acute myeloid leukemia; and Zepzelca for the treatment of adult patients with metastatic small cell lung cancer. The company also offers Xywav, an oxybate product candidate, to treat EDS and cataplexy with narcolepsy and idiopathic hypersomnia; JZP-324, a low sodium oxybate formulation with the potential to provide a clinically meaningful option for narcolepsy patients; JZP385, a T-type calcium channel modulator, for the treatment of essential tremor; JZP458, a recombinant Erwinia asparaginase, for use as a component of a multi-agent chemotherapeutic regimen in the treatment of pediatric and adult patients; and JZP150 for treatment of post-traumatic stress disorder. The company has licensing and collaboration agreements with ImmunoGen, Inc.; Codiak BioSciences, Inc.; Pfenex, Inc.; XL-protein GmbH; and Redx Pharma plc. Jazz Pharmaceuticals plc was incorporated in 2003 and is headquartered in Dublin, Ireland.

Catalysts

Phase 3Q3 2002GW-1000-02Multiple SclerosisNCT01610713

Primary: Change From Mean Part A Primary Impairment Visual Analogue Scale Score (After 6 Weeks) at the End of Four Weeks of Open-label Treatment (10 Weeks Total) (End of Part A (week 6) - end of Part B (week 10 [4 weeks total open-label treatment]))Enrollment:154/154Sites: 1NA, TREATMENT

Phase 3Q1 2004PlaceboPalliative Care, Pain, CancerNCT00674609

Primary: The Change in Mean Pain Numerical Rating Scale (NRS) Score From Baseline to the End of the Treatment. (2 weeks: baseline - end of week 2 (last 3 days of treatment))Enrollment:177/177Sites: 1RANDOMIZED, QUADRUPLE, SUPPORTIVE_CARE

Phase 3PositiveQ1 2004Sativex®Spasticity, Multiple SclerosisNCT00711646

Primary: Assessment of Change From Baseline in the Mean Spasticity 0-10 Numerical Rating Scale Score. (0-52 days)Enrollment:189/189Sites: 1RANDOMIZED, QUADRUPLE, SUPPORTIVE_CARE

Phase 3Q1 2005GW-1000-02Multiple Sclerosis, SpasticityNCT01610687

Primary: Incidence of Adverse Events as a Measure of Patient Safety (up to1206 days)Enrollment:137/137Sites: 1NA, TREATMENT

Phase 3PositiveQ3 2006SativexPain, CancerNCT00675948

Primary: The Incidence of Adverse Events as a Measure of Subject Safety (0 - 657 days)Enrollment:43/43Sites: 1NON_RANDOMIZED, SUPPORTIVE_CARE

Phase 3PositiveQ2 2007Sativex®Pain, Peripheral NeuropathyNCT00713323

Primary: Change From Parent Study Baseline in Mean Pain 0-10 Numerical Rating Scale (NRS) Score During the Last 4 Weeks of Open-label Treatment (38 weeks)Enrollment:380/380Sites: 1NA, SUPPORTIVE_CARE

Phase 2Q3 2008Sodium OxybateEssential TremorNCT00598078

Primary: Modified FTM(Fahn-Tolosa-Marin) Essentials Tremor Rating Scale, Sum of All Essential Rating Tremor Scales Including Voice Tremor (Hour 1)Enrollment:19/19Sites: 1RANDOMIZED, DOUBLE, TREATMENT

Phase 2Q4 2008CPX-1 (Irinotecan HCl:Floxuridine) Liposome InjectionColorectal NeoplasmsNCT00361842

Primary: Objective Response Rate (ORR) Per Response Evaluation Criteria in Solid Tumors (RECIST) Version 1.0 (From date of first dose until the date of first documented progression or date of death from any cause, whichever came first, assessed up to 48 months)Enrollment:65/65Sites: 13NON_RANDOMIZED, TREATMENT

Phase 2Q4 2008ClonazepamEpilepsyNCT00594945

Primary: Number of Spikes and Sharp Waves, Relative Change From Baseline to Treatment Day (%). (Change from baseline to treatment day)Enrollment:45/45Sites: 6NON_RANDOMIZED, TREATMENT

Phase 2NegativeQ3 2009Open-LabelChronic Obstructive Pulmonary DiseaseNCT00457951

Primary: Incidence of Treatment Failure (Time to hospital discharge and 21 days post-treatment, up to 31 days)Enrollment:158/158Sites: 38RANDOMIZED, DOUBLE, TREATMENT

Phase 2PositiveQ2 2010CPX-351Acute Myeloid LeukemiaNCT00788892

Primary: Number of Participants With Complete Remission (Within 6 weeks of the last induction treatment)Enrollment:126/126Sites: 28RANDOMIZED, TREATMENT

Phase 2PositiveQ4 2011CPX-351Acute Myeloid LeukemiaNCT00822094

Phase 2PositiveQ1 2012BrincidofovirCytomegalovirus InfectionNCT00942305

Phase 2NegativeQ4 2012Nab paclitaxelMetastatic Pancreatic CancerNCT01461915

Primary: Number of Patients who Achieved Progression-Free Survival (9 months)Enrollment:60/60Sites: 16RANDOMIZED, TREATMENT

Phase 2PositiveQ2 2015CPX-351Acute Myeloid Leukemia (AML), Acute Lymphoblastic Leukemia (ALL), Myelodysplastic Syndrome (MDS)NCT02238925

Primary: Effect of CPX-351 on Cardiac Ventricular Repolarization (QTcF) (21 days)Enrollment:26/26Sites: 5NA, TREATMENT

Phase 2Q2 2015Erwinaze® (Asparaginase Erwinia Chrysanthemi)/Erwinase® (Crisantaspase)Acute Lymphoblastic Leukemia (ALL)/Lymphoblastic Lymphoma (LBL)NCT02150928

Phase 3PositiveQ4 2015CPX-351High Risk Acute Myeloid LeukemiaNCT01696084

Phase 3Q1 2016NabiximolsPain, Advanced CancerNCT01337089

Primary: Percent Of Participants With Treatment-emergent Adverse Events (Baseline, Day 183)Enrollment:660/660Sites: 128NA, TREATMENT

Phase 2Q1 2016GWP42004Diabetes Mellitus, Type 2NCT02053272

Phase 3NegativeQ2 2016BrincidofovirAdenovirus InfectionNCT02087306

Phase 3PositiveQ3 2016DefibrotideHepatic Veno-Occlusive DiseaseNCT00628498

Primary: Survival by Day+100 Post Stem Cell Transplant or Chemotherapy (Day +100 from HSCT or 100 days from start of chemotherapy)Enrollment:1,206/1,206Sites: 125NA, TREATMENT

Phase 3PositiveQ1 2017JZP-110NarcolepsyNCT02348593

Phase 3PositiveQ1 2017XyremNarcolepsy With CataplexyNCT02221869

Phase 2Q2 2017CPX-351Acute Myeloid Leukemia (AML), Acute Lymphoblastic Leukemia (ALL), Myelodysplastic Syndrome (MDS)NCT02269579

Primary: Total Drug Plasma PK (Pre-dose and up to Day 21 during first induction only)0Sites: 5NA, TREATMENT

Phase 2Q3 2017GWP42006Epilepsy, Focal SeizuresNCT02365610

Phase 3PositiveQ2 2018GWP42003-PEpilepsy, Dravet SyndromeNCT02224703

Phase 2PositiveQ3 2018CX-8998Essential TremorNCT03101241

Phase 2Q3 2018JZP-110Excessive Sleepiness, Parkinson DiseaseNCT03037203

Primary: Number of Participants With Treatment Emergent Adverse Events (TEAEs) Leading to Early Discontinuation (Up to Day 35)Enrollment:66/66Sites: 25RANDOMIZED, DOUBLE, TREATMENT

Phase 2Q3 2018GWP42003-PEpilepsyNCT02607891

Phase 3PositiveQ1 2019JZP-258Narcolepsy With CataplexyNCT03030599

Phase 2Q2 2019JZP-110Narcolepsy, Excessive SleepinessNCT02806908

Phase 2Q2 2019Dociparstat sodiumAcute Myeloid LeukemiaNCT02873338

Phase 2NegativeQ2 2020GWP42006Autism Spectrum DisorderNCT03849456

Phase 3PositiveQ2 2020JZP-258Idiopathic HypersomniaNCT03533114

Phase 2NegativeQ3 2020DefibrotideDLBCL, Neurotoxicity SyndromesNCT03954106

Phase 2Q3 2020CX-8998Parkinson's Disease, TremorNCT03436953

Phase 3NegativeQ2 2022DociparstatAcute Myeloid LeukemiaNCT04571645

Phase 3Q3 2022IM JZP-458Acute Lymphoblastic Leukemia, Lymphoblastic LeukemiaNCT04145531

Phase 2NegativeQ4 2022Dordaviprone (ONC201)GliomaNCT03295396

Phase 2NegativeQ1 2023Dordaviprone (ONC201)Endometrial Cancer RecurrentNCT03485729

Phase 3NegativeQ3 2023GWP42003-PSeizures Associated With EMASNCT05288283

Phase 2Q4 2023JZP150Post Traumatic Stress DisorderNCT05178316

Phase 2NegativeQ4 2023LurbinectedinAdvanced Solid Tumor, Metastatic Solid Tumor, Urothelial CancerNCT05126433

Phase 2Q2 2024JZP385Essential TremorNCT05122650

Phase 2Jun 30, 2024GlasdegibAcute Myelogenous Leukemia (AML) Due to Therapy, Acute Myeloid Leukemia With Myelodysplasia-Related ChangesNCT04231851

Phase 3Jul 29, 2024AtezolizumabSmall-Cell Lung CancerNCT05091567

Phase 2Sep 1, 2024Enasidenib MesylateRecurrent Acute Myeloid LeukemiaNCT03825796

Phase 2Sep 1, 2024DefibrotideCovid19NCT04652115

Phase 2Q4 2024PlaceboParkinson Disease, TremorNCT05642442

Phase 2Apr 10, 2025Liposome-encapsulated Daunorubicin-CytarabineAcute Myeloid Leukemia Arising From Previous Myelodysplastic Syndrome, Acute Myeloid Leukemia With Myelodysplasia-Related Changes, Secondary Acute Myeloid LeukemiaNCT04269213

Phase 2Jun 6, 2025Liposome-encapsulated Daunorubicin-CytarabineAcute Myeloid Leukemia, Myeloid NeoplasmNCT04195945

Phase 2Aug 28, 2025CPX-351Myeloid NeoplasmNCT05656248

Phase 2Q2 2026NabiximolsGlioblastoma, Brain Tumor, CannabisNCT05629702

Phase 2Jun 30, 2026PaclitaxelGastroesophageal AdenocarcinomaNCT06043427

Phase 3Q3 2026ZanidatamabGastric Neoplasms, Gastroesophageal Adenocarcinoma, Esophageal AdenocarcinomaNCT05152147

Phase 2Q3 2026LurbinectedinAdvanced Soft-tissue Sarcoma, Metastatic Soft-tissue Sarcoma, Advanced LeiomyosarcomaNCT05099666

Phase 2Q4 2026CPX-351Acute Myeloid Leukemia (AML) in RemissionNCT04990102

Phase 2Q4 2026ZanidatamabBreast Cancer, Gastric Cancer, Esophageal CancerNCT06695845

Phase 2Q1 2027CannabidivarinAutism Spectrum DisorderNCT03202303

Phase 3Q2 2027CytarabineAcute Myeloid LeukemiaNCT03897127

Phase 2Jul 30, 2027LurbinectedinEwing Sarcoma, Desmoplastic Small Round Cell Tumor, Pediatric CancerNCT05918640

Phase 2Dec 1, 2027CBD Oral SolutionFragile X SyndromeNCT06261502

Phase 2Dec 15, 2027CBD Oral Solution (eCBD system Target)Fragile X SyndromeNCT06261450

Phase 3Q4 2027XywavNarcolepsy, Idiopathic HypersomniaNCT07625280

Phase 2Dec 31, 2027ONC201Diffuse Intrinsic Pontine Glioma, Diffuse Midline Glioma, H3 K27M-Mutant, Recurrent Diffuse Intrinsic Pontine GliomaNCT05009992

Phase 2Q1 2028ZanidatamabHER2-positive Breast Cancer, Breast CancerNCT07102381

Phase 2Q1 2028LurbinectedinRefractory Ewing Sarcoma, Relapsed Ewing Sarcoma, Ewing SarcomaNCT05734066

Phase 2Jan 31, 2028Asparaginase Erwinia chrysanthemiB Acute Lymphoblastic Leukemia, Philadelphia Chromosome Negative, T Acute Lymphoblastic LeukemiaNCT06738368

Phase 2Q1 2028LurbinectedinExtensive-stage Small-cell Lung CancerNCT07459634

Phase 2Mar 13, 2028ZanidatamabNon-Small Cell Lung Cancer, Sarcoma, Head &Amp; Neck CancerNCT07192068

Phase 3Q2 2028ZanidatamabMetastatic HER2-positive Breast CancerNCT06435429

Phase 2Jun 1, 2028TislelizumabGastroesophageal AdenocarcinomaNCT07290985

Phase 3Q2 2028Dordaviprone (ONC201)H3 K27M, GliomaNCT05580562

Phase 3Q3 2028JZP926 capsuleJuvenile Myoclonic EpilepsyNCT07723963

Phase 2Aug 2, 2028Cytarabine and IdarubicinAcute Myeloid LeukemiaNCT05260528

Phase 3Q3 2028EverolimusDiffuse Intrinsic Pontine Glioma, Diffuse Midline Glioma, H3 K27M-Mutant, Diffuse Midline Glioma, H3K27-alteredNCT05476939

Phase 2Q4 2028JZP3507MeningiomaNCT07533942

Phase 3Q4 2028ZanidatamabBiliary Tract CancerNCT06282575

Phase 2Q4 2028ONC206Advanced Pheochromocytoma and Paraganglioma, PCPGNCT07282587

Phase 2Q1 2029ZanidatamabGastroesophageal Adenocarcinoma, First Line Therapy, HER2 + Gastric CancerNCT07176312

Phase 2Q2 2029LurbinectedinSmall Cell Lung Cancer ( SCLC ), Transformed Small Cell Lung CancerNCT07153055

Phase 3Q3 2029CBD-OSDevelopmental and Epileptic Encephalopathy (DEE)NCT07723976

Phase 3Dec 1, 2029Cannabidiol (CBD)Clinical High Risk for Psychosis (CHR), Clinical High Risk for Psychosis, Clinical High Risk for Developing PsychosisNCT07434973

Phase 2Feb 28, 2030CannabidiolEarly Life Adversity, Trauma, StressNCT07278505

Recent News & Research

PubMed

TP53-mutated Therapy-related Leukemia with the Der(5;17)(p10;q10) Chromosomal Abnormality Following Chemotherapy for Acute Promyelocytic Leukemia Bridged to Allogeneic Hematopoietic Stem Cell Transplantation Using CPX-351.

# Investment Summary: Jazz Pharmaceuticals (JAZZ) This case report describes a therapy-related leukemia (tAML) with TP53 mutation emerging in an acute promyelocytic leukemia (APL) patient treated with CPX-351 (Vyxeos, Jazz's liposomal cytarabine/daunorubicin combination) followed by stem cell transplant. While isolated case reports have limited statistical power, the observation of secondary malignancy in a bridging therapy context warrants monitoring for potential long-term safety signals that could impact Vyxeos' competitive positioning in APL, particularly as the company faces increasing scrutiny on treatment-related toxicity profiles. **Investor Note:** This does not represent new safety data requiring immediate action, but investors should track whether similar cases accumulate in post-marketing surveillance, as secondary malignancy liability could affect reimbursement discussions or label restrictions

Aug 8, 2026

bioRxiv

Synergistic cytotoxicity with Chk1/Chk2-inhibitor prexasertib in small cell lung cancer following lurbinectedin-induced G2/M-checkpoint activation

# Jazz Pharmaceuticals Investment Summary **Key Finding:** The study demonstrates synergistic cytotoxicity when combining prexasertib (a Chk1/Chk2 inhibitor in Jazz's pipeline) with lurbinectedin (a topoisomerase I inhibitor) in small cell lung cancer (SCLC) models, where lurbinectedin triggers G2/M checkpoint activation that prexasertib can exploit. **Clinical/Commercial Implications:** This pre-clinical data supports a potential combination strategy for SCLC, a notoriously difficult-to-treat indication with limited options, which could expand prexasertib's addressable market beyond single-agent applications and improve competitive positioning if validated in clinical trials. However, investor caution is warranted given the early-stage nature (bioRxiv preprint, not peer-reviewed), and the combination's path to development depends on

Aug 4, 2026

bioRxiv

Purified CBD and CBD-rich full-spectrum Cannabis sativa extract potentiate the angiogenic paracrine function of umbilical cord derived mesenchymal stem cells

# Research Summary for JAZZ Investors This preprint demonstrates that cannabidiol (CBD) enhances the angiogenic (blood vessel-forming) paracrine signaling of mesenchymal stem cells in vitro, suggesting potential therapeutic applications in regenerative medicine and tissue repair. The findings are preliminary (bioRxiv preprint) and lack in vivo validation or clinical data, limiting immediate commercial relevance to Jazz's current portfolio. While Jazz holds significant CBD assets through its Epidiolex franchise, this basic science work does not directly advance approved indications or near-term pipeline candidates, and would require substantial development before any commercial application. **Investor note:** Monitor for progression to in vivo models and clinical translation, but this represents exploratory research rather than a near-term value driver for JAZZ.

Jul 9, 2026

PubMed

Transportability of tislelizumab trial outcomes from RATIONALE-315, RATIONALE-303, and RATIONALE-312 to the European population in early-stage non-small cell lung cancer (NSCLC), advanced NSCLC and extensive-stage small cell lung cancer (ES-SCLC).

# Investment Summary: Tislelizumab Transportability Analysis **Clinical Finding:** This analysis examines whether efficacy and safety data from tislelizumab trials (a PD-L1 inhibitor) can be reliably applied to European patient populations across early-stage NSCLC, advanced NSCLC, and ES-SCLC—addressing a key regulatory requirement for European market expansion. **Commercial Implication:** Successful transportability validation would support Jazz's European regulatory submissions and label expansion, potentially unlocking significant market access in these large oncology indications. Conversely, poor transportability could require additional European trials, delaying market entry and increasing development costs in a competitive immunotherapy landscape. **Why It Matters:** Regulatory agencies increasingly scrutinize whether non-European trial populations adequately represent regional patient populations; this analysis directly impacts Jazz's ability to efficiently convert clinical data into approved European ind

Jul 7, 2026

bioRxiv

mTOR inhibition augments antitumor immune effector response by reprogramming the TP53-mutant, immune-cold HNSCC tumor microenvironment.

# Jazz Pharmaceuticals Investment Summary **Clinical/Commercial Relevance:** This preclinical research demonstrates that mTOR inhibition can convert immunologically "cold" head and neck squamous cell carcinomas (HNSCC)—particularly TP53-mutant tumors resistant to checkpoint inhibitors—into immunologically "hot" tumors, potentially enabling combination therapy approaches. Jazz's mTOR inhibitor portfolio (notably everolimus) could be repositioned to address the significant unmet need in TP53-mutant HNSCC, a subset that currently shows poor response to standard immunotherapy and represents a commercially valuable patient stratification opportunity. If validated in clinical studies, this mechanism could expand Jazz's addressable market in oncology and provide rational combination strategies with checkpoint inhibitors or other immunotherapies. **Investor Consideration:** This is early-stage (preprint) research requiring IND enabling studies and clinical validation; success

Jun 8, 2026

Jazz Pharmaceuticals plc

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