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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
JZP341
(Long-acting Erwinia asparaginase)1
ALL/other hematological malignancies
Pan-RAF Inhibitor program
Raf & Ras mutant tumors
Undisclosed targets
Ras/Raf/MAP kinase pathway1
Exosome targets
(NRAS and 3 others)1
Hematological malignancies/solid tumors
Defibrotide
Exploratory activities
Undisclosed targets
Cannabinoids
PHASE 1
JZP324
Oxybate extended-release formulation
CPX351
Low Intensity Dosing for higher risk MDS2
CPX351 + other approved therapies
  • R/R AML or HMA Failure MDS2
  • First-line, fit AML (Phase 1b)
  • Low Intensity Therapy for first-line, unfit AML (Phase 1b)
Additional Cannabinoids
Neonatal hypoxic-ischemic encephalopathy
Additional Cannabinoids
Neuropsychiatry targets

PHASE 2
JZP3853
Essential tremor (Phase 2b)
JZP1503
PTSD
CPX351
  • HR-MDS (EMSCO)4
  • Newly diagnosed older adults with HR-AML4,5
CPX351 + venetoclax
de novo or R/R AML2
Nabiximols3
PTSD
Additional Cannabinoids
Schizophrenia
Additional Cannabinoids
Autism spectrum disorders

PHASE 3
CPX351
  • AML or HR-MDS >60 yrs (AML18)4
  • AML or HR-MDS >18 yrs (AML19)4
  • Newly diagnosed adults with standard- and HR-AML (AMLSG)4
  • Newly diagnosed <22 yrs with AML (COG)4
Nabiximols
MS spasticity

Nabiximols3
Spinal cord injury spasticity

REGULATORY
JZP258
Idiopathic hypersomnia
JZP458 (recombinant Erwinia asparaginase)
ALL/LBL (pivotal Phase 2/3)
Neuroscience
Hematology/Oncology
Cannabinoids
1Partnered collaboration.
2Jazz & MD Anderson Cancer Center collaboration study.
3Planned.
4Cooperative group study.
5Subject to FDA approval.
Molecule
Phase

JZP258

  • 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

JZP458

  • Acute lymphoblastic leukemia/lymphoblastic lymphoma (ALL/LBL)
Regulatory Pivotal 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 under U.S. FDA Real-Time Oncology Review pilot program

Clinicaltrials.gov

CPX351

  • 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

CPX351 + venetoclax

  • De novo AML
2
  • Relapsed refractory AML
2

Jazz and MD Anderson Cancer Center Collaboration Study

Study Sponsor – MD Anderson Cancer Center

CPX351 + 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

JZP385

  • Essential tremor (Phase 2b)
2

JZP150

  • PTSD
2

JZP324

  • 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 candidates

  • Exosome NRAS
  • Exosome STAT3
  • Solid tumors/Hematological malignancies
Pre-clinical
Jazz & Codiak BioSciences collaboration

JZP341

  • 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