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Pipeline

Where there’s a will, there’s a way. Patients are our will.

Pipeline

PRE-CLINICAL
Undisclosed targets
Neuroscience
CombiPlex
Exploratory activities
JZP-341 (Long-acting Erwinia asparaginase)2
ALL/other hematological malignancies
Recombinant Pegaspargase1
Hematological malignancies
Pan-RAF Inhibitor program
Raf & Ras mutant tumors
Undisclosed targets
Ras/Raf/MAP kinase pathway2
Exosome targets (NRAS, STAT3 and 3 others)2
Hematological malignancies/solid tumors
Defibrotide
Exploratory activities
PHASE 1
JZP-324
Oxybate extended-release formulation
CPX-351
Low Intensity Dosing for higher risk MDS3
CPX-351 + other approved therapies
  • R/R AML or HMA Failure MDS3
  • First-line, fit AML (Phase 1b)
  • Low Intensity Therapy for first-line, unfit AML (Phase 1b)
PHASE 2
JZP-3854
Essential tremor (Phase 2b)
JZP-1504
PTSD
CPX-351
  • HR-MDS (EMSCO)5
  • Newly diagnosed older adults with HR-AML4,5
CPX-351 + venetoclax
de novo or R/R AML3
PHASE 3
CPX-351
  • AML or HR-MDS >60 yrs (AML18)5
  • AML or HR-MDS >18 yrs (AML19)5
  • Newly diagnosed adults with standard- and HR-AML (AMLSG)5
  • Newly diagnosed <22 yrs with AML (COG)5
REGULATORY
JZP-258
Idiopathic hypersomnia
JZP-458 (recombinant Erwinia asparaginase)
ALL/LBL (pivotal Phase 2/3)
Neuroscience
Hematology/Oncology
1Opt-in opportunity
2Partnered collaboration
3Jazz & MD Anderson Cancer Center collaboration study
4Planned
5Cooperative group study
6Exclusive U.S. license
*PF-04457845

Molecule
Phase

JZP-258

  • EDS in Idiopathic Hypersomnia
Regulatory
Oral solution that contains a mixture of oxybate salts, resulting in 92 percent less sodium content than Xyrem® (sodium oxybate) oral solution. For Xyrem full Prescribing Information, including Boxed WARNING, please visit www.jazzpharma.com/medicines/our-medicines.

Clinicaltrials.gov

JZP-458

  • Acute lymphoblastic leukemia/lymphoblastic lymphoma (ALL/LBL)
2/3
Recombinant Erwinia asparaginase; granted Fast Track designation by the U.S. Food and Drug Administration in October 2019

Status – Pivotal Phase 2/3 study began enrollment 4Q19; Filing initiation began December 2020

Clinicaltrials.gov

CPX-351

  • AML or HR-MDS >60 yrs (AML18)
3
  • AML or HR-MDS >18 yrs (AML19)
3
  • Newly diagnosed adults with standard- and HR-AML (AMLSG)
3
  • Newly diagnosed <22 yrs with AML (COG)
3
  • HR-MDS (EMSCO)
2
  • Newly diagnosed older adults with HRAML
2
  • Low Intensity Dosing for higher risk MDS
1
Status – AML18, AML19, MDS and the R/R AML trial are key cooperative group studies

Study Sponsor – Cardiff University (for AML18, AML19)

Clinicaltrials.gov

For Vyxeos® (daunorubicin and cytarabine) full Prescribing Information, including Boxed WARNING, please visit www.jazzpharma.com/medicines/our-medicines

CPX-351 + venetoclax

  • De novo AML
2
  • Relapsed refractory AML
2

Jazz and MD Anderson Cancer Center Collaboration Study

Study Sponsor – MD Anderson Cancer Center

CPX-351 + other approved therapies

  • R/R AML or HMA Failure MDS3
  • First-line, fit AML (Phase 1b)
  • Low Intensity Therapy for first-line, unfit AML (Phase 1b)
1

JZP-385

  • Essential tremor (Phase 2b)
2

JZP-150

  • PTSD
2

JZP-324

  • Oxybate extended-release formulation
1

Defibrotide

  • Exploratory activities
Pre-clinical
Defibrotide has orphan drug designation from the U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) for the prevention of hepatic VOD. Defibrotide has orphan drug designation from EMA for the prevention of acute GvHD.

Clinicaltrials.gov

For Defitelio® full Prescribing Information, please visit www.jazzpharma.com/medicines/our-medicines.

CombiPlex

  • Exploratory activities
Pre-clinical

Exosome NRAS

  • Hematologic malignancies
Pre-clinical
Jazz & Codiak BioSciences collaboration

Exosome STAT3

  • Hematologic malignancies
Pre-clinical
Jazz & Codiak BioSciences collaboration

Exosome candidates

  • Solid tumors/Hematological malignancies
Pre-clinical
Jazz & Codiak BioSciences collaboration

Recombinant Pegaspargase

  • Hematologic malignancies
Pre-clinical
Opt-in based on Pfenex partnership

JZP-341

  • ALL/other hematologic malignancies
Pre-clinical
Long-acting Erwinia asparaginase; Opt-in based on Pfenex partnership

Pan-RAF Inhibitor Program

  • RAF & RAS mutant tumors
Pre-clinical