Pipeline

OVERVIEW

Zanidatamab, or Ziihera® (zanidatamab-hrii) in the United States and the EU, is a bispecific HER2-directed antibody that binds to two extracellular sites on HER2. Zanidatamab has been granted two Fast Track designations by the U.S. Food and Drug Administration (FDA): one as a single agent for refractory biliary tract cancers (BTC) and one in combination with standard-of-care (SOC) chemotherapy for first-line gastroesophageal adenocarcinoma (1L GEA). Additionally, zanidatamab has received Orphan Drug designations from the FDA for the treatment of BTC, and gastric (including gastroesophageal junction) cancer, and esophageal cancer, as well as Orphan Drug designations from the European Medicines Agency for the treatment of BTC, gastric/gastroesophageal junction cancer and oesophageal cancer.

CLINICAL TRIALS

Phase 3

• The Phase 3 HERIZON-GEA-01 trial (NCT05152147) evaluates zanidatamab in combination with chemotherapy and with or without the checkpoint inhibitor tislelizumab as a first-line treatment for metastatic HER2-positive (HER2+) GEA.
• The Phase 3 HERIZON-BTC-302 trial (NCT06282575) evaluates zanidatamab and CisGem (cisplatin plus gemcitabine) with or without the addition of a programmed death protein 1/ligand 1 (PD-1/L-1) inhibitor versus CisGem with or without a PD-1/L1 inhibitor in adult participants.
• The Phase 3 EmpowHER trial (NCT06435429) evaluates zanidatamab compared to trastuzumab, each in combination with physician's choice of chemotherapy, for the treatment of participants with metastatic HER2-positive breast cancer who have progressed on, or are intolerant to, previous T-DXd treatment.

Phase 2

• The Phase 2 DiscovHER-Pan-206 trial (NCT06695845) evaluating zanidatamab monotherapy in previously-treated patients with HER2+ cancers including breast, gastric, esophageal, gastroesophageal, colorectal, endometrial, non-small cell lung, ovarian, urothelial, salivary and pancreatic.
• The Phase 2 EmpowHER-BC-208 trial (NCT07102381) evaluating zanidatamab in patients with HER2 neoadjuvant and adjuvant breast cancer.
• Phase 2 trial (NCT06043427) evaluating zanidatamab in combination with usual care, paclitaxel and ramucirumab, in HER2+ advanced GEA, in collaboration with the Canadian Cancer Trials Group.
• The Phase 2 ZANGEA trial (NCT07176312) evaluating zanidatamab in combination with pembrolizumab and chemotherapy in patients with HER2+ and PD-L1 positive metastatic GEA, conducted in collaboration with Institut für Klinische Krebsforschung IKF GmbH.
• Phase 2 trial (NCT03929666) evaluating zanidatamab in addition to SOC chemotherapy in patients with BTC, GEA and CRC.
• Phase 2 investigation of serial studies to predict the therapeutic response with imaging and molecular analysis 2 (I-SPY 2) (NCT01042379) trial assessing the efficacy of novel drugs in sequence with standard chemotherapy to advance approaches to personalized medicine. This trial is conducted in collaboration with QuantumLeap Healthcare Collaborative.
• Phase 2 single-arm open-label pilot trial (NCT05035836) evaluating zanidatamab in patients with early-stage, low-risk, HER2-positive breast cancer, conducted in collaboration with MD Anderson Cancer Center.

Zanidatamab is also being investigated in Phase 1 studies, including the I-SPY Phase 1/1b platform trial (NCT05868226) with multiple ongoing drug regiment arms, evaluating single agents or combinations for breast cancer patients. Promising treatment regiments are to be transferred into the I-SPY 2 SMART Design Trial (NCT01042379).

OVERVIEW

Lurbinectedin, or Zepzelca^® in the United States and Canada, is an alkylating drug that binds guanine residues within DNA, which triggers a cascade of events that can affect the activity of DNA binding proteins, including some transcription factors, and DNA repair pathways, resulting in disruption of the cell cycle and eventual cell death.

CLINICAL TRIALS

In addition to being approved for extensive-stage small cell lung cancer (SCLC), the effectiveness of lurbinectedin is being evaluated in pediatric and young adults with relapsed/​refractory ewing sarcoma.

• The Phase 3 LAGOON (NCT05153239) trial evaluating and comparing the activity and safety of lurbinectedin as a single agent and in combination with irinotecan in SCLC patients. This trial is being conducted by PharmaMar as part of Jazz’s partnership with the company.
• The ongoing Phase 1/2 (NCT05734066) trial evaluating the effectiveness and safety of lurbinectedin monotherapy in pediatric and young adult participants with recurrent/refractory Ewing sarcoma.

OVERVIEW

Dordaviprone, or Modeyso™️ in the United States, (formerly known as ONC201) is a protease activator of the mitochondrial caseinolytic protease P (ClpP) and also inhibits dopamine D2 receptor (DRD2). In vitro, dordaviprone activates the integrated stress response, induces apoptosis, and alters mitochondrial metabolism, leading to restored histone H3 K27 trimethylation in H3 K27M-mutant diffuse glioma. Dordaviprone was developed by Chimerix prior to its acquisition by Jazz Pharmaceuticals in April 2025.

CLINICAL TRIALS

The Phase 3 ACTION confirmatory trial (NCT05580562) evaluating the safety and clinical benefit of Modeyso in newly diagnosed patients with H3 K27M-mutant diffuse glioma following radiotherapy.

OVERVIEW

JZP351, or Vyxeos^®/Vyxeos Liposomal in approved markets, is a liposomal combination of daunorubicin, an anthracycline topoisomerase inhibitor, and cytarabine, a nucleoside metabolic inhibitor.

CLINICAL TRIALS

Phase 2

• Phase 2 trial (NCT05564390) evaluating JZP351 as monotherapy or in combination for first-line treatment of adults with high-risk AML (MyeloMATCH), conducted in collaboration with University of Texas MD Anderson Cancer Center.
• Phase 2 trial (NCT04493164) evaluating JZP351 in combination with ivosidenib in adults with IDH1 AML or higher-risk myelodysplastic syndromes (MDS), conducted in collaboration with University of Texas MD Anderson Cancer Center.

Additional Phase 2 and Phase 3 trials of JZP351 are evaluating the agent in various age populations, and are being conducted with cooperative groups such as the AML Study Group (AMLSG) and the European Myelodysplastic Neoplasms Cooperative Group (EMSCO).

OVERVIEW

Rylaze^® (asparaginase erwinia chrysanthemi (recombinant)-rywn)[recombinant Erwinia asparaginase] is approved in the U.S. for use as a component of a multi-agent chemotherapeutic regimen for the treatment of acute lymphoblastic leukemia (ALL) and lymphoblastic lymphoma (LBL) in adult and pediatric patients one month or older who have developed hypersensitivity to E. coli-derived asparaginase. 

CLINICAL TRIALS

The safety and tolerability of asparaginase Erwinia chrysanthemi (asparaginase Erwinia chrysanthemi-recombinant-rywn [recombinant Erwinia asparaginase]; 25 mg/m^2 every 48 hours [hrs]) is being evaluated in a Phase 2 trial as first asparaginase during induction therapy through the assessment of type, frequency, severity, attribution, time course and duration of adverse events. This trial (NCT06918431) is collaboration with City of Hope. Read here.

OVERVIEW

An investigational pan-RAF inhibitor for the treatment of solid tumors and hematologic malignancies that contain mutations in the mitogen-activated protein kinase (MAPK) pathway. JZP815 targets specific components of the MAPK pathway that, when activated by oncogenic mutations, can be a frequent driver of human cancer. JZP815 potentially inhibits both monomer- and dimer-driven RAF signaling (e.g., RAS-induced), prevents paradoxical pathway activation induced by BRAF selective inhabitation and is active against class 1, class 2 and class 3 BRAF mutants as well as BRAF fusions and CRAF mutants.

CLINICAL TRIALS

A Phase 1, open-label, first-in-human study (NCT05557045).

OVERVIEW

Investigational JZP898, previously WTX-613, is a conditionally activated IFN⍺2b cytokine pro-drug.

CLINICAL TRIALS

The safety, tolerability, pharmacokinetics, immunogenicity and preliminary antitumor activity of JZP898 both as a monotherapy and in combination with pembrolizumab is being investigated in Phase 1, first-in-human study (NCT06108050).

OVERVIEW

JZP3507 (formerly ONC206) is a dual targeted investigational therapy that is an agonist of the mitochondrial protease ClpP and an antagonist of the G protein-coupled receptor DRD2.  

Clinical Trials

Phase 2

• Phase 2 trial (NCT07282587) is evaluating the efficacy and safety of JZP3507 in participants with Pheochromocytoma and Paraganglioma (PCPG).

Additional trials include a Phase 1 trials (NCT04541082) evaluating JZP3507 in participants with newly diagnosed or recurrent diffuse midline gliomas and other recurrent primary malignant CNS tumors, conducted in collaboration with UCSF, as well as a Phase 1 trial evaluating JZP3507 in participants with primary central nervous system (CNS) tumors.

OVERVIEW

KRAS (Kirsten rat sarcoma virus) inhibitor program, including G12D selective and pan-KRAS molecules, acquired from Redx Pharma.

OVERVIEW

JZP3508 (formerly ONC212) is a dual agonist of the mitochondrial protease ClpP and the G protein-coupled receptor GPR132. 

OVERVIEW

Evaluating patented CombiPlex^® platform in a wide range of tumor types. Evaluating improvement in cancer therapy by using extremely small (nano-scale) carriers to deliver optimal ratios of multiple anticancer agents directly to cancer cells over a prolonged period of time.

OVERVIEW

Epidiolex^® (cannabidiol), known as Epidyolex^® in the European Union (EU), United Kingdom (U.K.), Australia and Israel, is a prescription, plant-derived cannabis-based medicine administered as an oral solution. In the United States, Epidiolex is indicated for the treatment of seizures associated with Lennox-Gastaut syndrome (LGS), Dravet syndrome (DS), or tuberous sclerosis complex (TSC) in patients 1 year of age and older. In the EU and U.K., Epidyolex is indicated for use as adjunctive therapy of seizures associated with LGS or DS, in conjunction with clobazam, for patients 2 years of age and older. Epidyolex is also indicated for use as adjunctive therapy of seizures associated with TSC for patients 2 years of age and older.

CLINICAL TRIALS

• An ongoing Phase 3 trial in Japan evaluating the efficacy and safety of Epidyolex in patients with LGS, DS and TSC.​
• A Phase 1b trial (NCT07233239) evaluating Epidiolex as an adjunctive treatment in participants with focal-onset seizures (FOS).