Pipeline

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

Pipeline

PRE-CLINICAL
Oxybate
Once Nightly Formulation

CombiPlex
Solid tumors candidate

CombiPlex
Hem/Onc exploratory activities

Recombinant Crisantaspase-HLE2
ALL/other hematological malignancies

Recombinant Pegaspargase1
Hematological malignancies

Defibrotide
Exploratory activities

Exosome NRAS candidate2
Hematological malignancies

Exosome STAT3 candidate2
Hematological malignancies

Exosome-based candidates2
Solid tumors/ Hematological malignancies

Pan-RAF Inhibitor Program
RAF & RAS mutant tumors

PHASE 1
CPX-351 + gemtuzumab3
R/R AML or HMA Failure
MDS

CPX-351 + venetoclax
Low Intensity Therapy for first-line, unfit AML (Phase 1b)

CPX-3513
Low Intensity Dosing for higher risk MDS

IMGN6321
CD123+ Hematological malignancies5

PHASE 2
Defibrotide
Prevention of aGvHD

Defibrotide4
Treatment of TA-TMA

Defibrotide4
Prevention of CAR-T associated neurotoxicity

CPX-351 + venetoclax3
de novo or R/R AML

CPX-3516
HR-MDS

CPX-3516
R/R AML (COG)

CPX-3514,6
Newly diagnosed older adults with HR-AML

CPX-351 + venetoclax4,6
HR-AML

JZP-458 (Recombinant Crisantaspase)4
ALL (Phase 2/3)

CX-8998
Essential Tremor

PHASE 3
JZP-258
Cataplexy & EDS in narcolepsy

JZP-258
Idiopathic hypersomnia

Defibrotide
Prevention of VOD

CPX-3516
AML or HR-MDS (AML19)

CPX-3516
AML or HR-MDS (AML18)

CPX-3516
Newly diagnosed adults with standard- and HR-AML

CPX-3514,6
Newly diagnosed pediatric patients

REGULATORY
Solriamfetol EU
EDS in OSA and Narcolepsy

Sleep / Neuroscience
Hematology / Oncology
1Opt-in opportunity
2Partnered collaboration
3Jazz & MD Anderson Cancer Center collaboration study
4Planned
5Including AML and BPDCN
6Cooperative group study

Molecule
Phase

Solriamfetol

  • Excessive daytime sleepiness in OSA or narcolepsy
Regulatory (EU)

A selective dopamine and norepinephrine reuptake inhibitor (DNRI) in development for the potential treatment of EDS in adult patients with narcolepsy, OSA, and Parkinson’s disease.

In 2014, Jazz Pharmaceuticals acquired a license to develop and commercialize solriamfetol from Aerial Biopharma. Jazz Pharmaceuticals has worldwide development, manufacturing, and commercialization rights to solriamfetol, excluding certain jurisdictions in Asia. SK Biopharmaceuticals, the discoverer of the compound, maintains rights in 12 Asian markets, including Korea, China and Japan. Solriamfetol has orphan drug designation in the United States for narcolepsy.

Status — European Medicines Agency (EMA) Marketing Authorization Application (MAA) submitted

Clinicaltrials.gov

Full prescribing information, Medication Guide and additional important safety information, please visit:
www.jazzpharma.com/medicines/our-medicines

CPX-351

  • AML or HR-MDS (AML18)
3
  • AML or HR-MDS (AML19)
3
  • MDS
2
  • De novo or Relapsed Refractory AML
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

  • Low intensity dosing for unfit AML
1

Jazz and MD Anderson Cancer Center Collaboration Study

Study Sponsor – MD Anderson Cancer Center

CPX-351 + gemtuzumab

  • Relapsed refractory AML (ages ≥18)
1

  • HMA Failure MDS (ages ≥18)
1

Jazz and MD Anderson Cancer Center Collaboration Study

Study Sponsor – MD Anderson Cancer Center

JZP-258

  • Cataplexy and EDS in Narcolepsy
3

  • EDS in Idiopathic Hypersomnia
3

Oral solution that contains a mixture of oxybate salts, resulting in 90 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.

Status – Narcolepsy trial: enrollment complete near year-end 2018; Idiopathic Hypersomnia: Phase 3 study initiated in Fall 2018

Clinicaltrials.gov

Defibrotide

  • Prevention of VOD
3

  • Prevention of acute GvHD
2

  • CAR-T associated neurotoxicity
2

  • TA-TMA
1

  • 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.

Status – prevention of VOD trial began enrollment 1Q17; prevention of acute GvHD began enrollment 1Q18; TA-TMA study start anticipated late-2019

Clinicaltrials.gov

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

JZP-458

  • ALL/LBL
2/3

IMGN632

  • Hematologic malignancies*
1

*including AML and BPDCN
Opt-in opportunity through ImmunoGen collaboration

Combiplex

  • Solid tumors
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

Recombinant Crisantaspase-HLE

  • ALL/other hematologic malignancies
Pre-clinical

Opt-in based on Pfenex partnership

Pan-RAF Inhibitor Program

  • RAF & RAS mutant tumors
Pre-clinical