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Research and Development

As the field of science has grown, so have our R&D capabilities. We actively explore revolutionary drug options ranging from small molecule advancements to biologics, and novel compounds within hematology/oncology and sleep medicine. This dynamic approach has allowed us to shepherd new medicines to market — from late-stage discovery through clinical development and regulatory approval.

All of these advancements are a reflection of our continued investment in an evolving and growing research and development focus.

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
Defibrotide
  • Prevention of CAR-T associated neurotoxicity
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

Through Jazz’s patented CombiPlex® platform, Jazz is evaluating oncology combinations to support identification of clinical product candidate(s) and is leading a collaborative approach with partners to research and develop new therapies.