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)
ALL/LBL
Pan-RAF Inhibitor program
Raf & Ras mutant tumors
Undisclosed targets
Ras/Raf/MAP kinase pathway1
Exosome targets
NRAS and up to 4 others)1
Hematological malignancies/solid tumors
Undisclosed targets
Oncology
Undisclosed targets
Cannabinoids
PHASE 1
JZP3243
Oxybate extended-release formulation
JZP351
Low Intensity Dosing for higher risk MDS2
JZP351 + 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
JZP385
Essential tremor
JZP1503
PTSD
Lurbinectedin3
Basket trial: advanced urothelial cancer, large cell neuroendocrine tumor of the lung, HRD+ cancers
JZP351
  • HR-MDS (EMSCO)4
  • Newly diagnosed older adults with HR-AML4
JZP351 + venetoclax
de novo or R/R AML2
Additional Cannabinoids
Schizophrenia
Additional Cannabinoids
Autism spectrum disorders

PHASE 3
Lurbinectedin1
1L treatment SCLC in combination with atezolizumab
JZP351
  • AML or HR-MDS >60 yrs (AML18)4
  • Newly diagnosed adults with standard- and HR-AML (AMLSG)4
  • Newly diagnosed <22 yrs with AML (COG)4
Cannabidiol3
EMAS

Nabiximols
MS spasticity

Nabiximols3
Spinal cord injury spasticity

REGULATORY
JZP4585
(recombinant Erwinia asparaginase)
ALL/LBL
Neuroscience
Hematology/Oncology
Cannabinoids
1Partnered collaboration.
2Jazz & MD Anderson Cancer Center collaboration study.
3Planned.
4Cooperative group study.
5FDA approval on June 30, 2021; plan to submit additional data to support U.S. label update.
Molecule
Phase

JZP458

  • Acute lymphoblastic leukemia and lymphoblastic lymphoma
Regulatory

Cannabidiol

  • Epilepsy with myoclonic-atonic seizures (EMAS)
3

Nabiximols

  • MS spasticity
3
  • Spinal cord injury spasticity
3

Lurbinectedin

  • 1L treatment SCLC in combination with atezolizumab
3
  • Basket trial: advanced urothelial cancer, large cell neuroendocrine tumor of the lung, HRD+ cancers
2

JZP351

  • AML or HR-MDS >60 yrs (AML18)
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

JZP351 + venetoclax

  • De novo AML
2
  • Relapsed refractory AML
2

Jazz and MD Anderson Cancer Center Collaboration Study

Study Sponsor – MD Anderson Cancer Center

JZP351 + other approved therapies

  • R/R AML or HMA Failure MDS2
1
  • First-line, fit AML (Phase 1b)
1
  • 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

CombiPlex

  • Exploratory activities
Pre-clinical

Exosome candidates

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

JZP341

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

Pan-RAF Inhibitor Program

  • RAF & RAS mutant tumors
Pre-clinical