Pipeline

Pipeline

PRE-CLINICAL
Undisclosed targets
Neuroscience
CombiPlex
Exploratory activities
JZP341
(Long-acting Erwinia asparaginase)
ALL/LBL
JZP815
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
JZP898 (WTX-613)2
Conditionally-activated IFNα
Undisclosed targets
Oncology
Undisclosed targets
Cannabinoids
PHASE 1
JZP3243
Oxybate extended-release formulation
JZP441 (DSP-0187)2,4
Orexin-2 receptor agonist
JZP351 (Vyxeos)
Low Intensity Dosing for higher risk MDS5
JZP351+ other approved therapies
  • R/R AML or HMA Failure MDS5
  • 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
Suvecaltamide (JZP385)
Essential tremor
JZP1506
PTSD
Lurbinectedin (Zepzelca)
Basket trial: advanced urothelial cancer, large cell neuroendocrine tumor of the lung, HRD+ cancers
JZP351
  • HR-MDS (EMSCO)7
  • Newly diagnosed older adults with HR-AML7
JZP351 + venetoclax
de novo or R/R AML5
Additional Cannabinoids

Autism spectrum disorders
PHASE 3
Lurbinectedin1
1L treatment SCLC in combination with Tecentriq® (atezolizumab)
JZP351
  • AML or HR-MDS >60 yrs (AML18)7
  • Newly diagnosed adults with standard- and HR-AML (AMLSG)7
  • Newly diagnosed <22 yrs with AML (COG)5
Cannabidiol (Epidiolex)3
EMAS

Nabiximols
MS spasticity

Nabiximols3
Spinal cord injury spasticity

REGULATORY
JZP458 (Rylaze)8
(recombinant Erwinia asparaginase)
ALL/LBL
Neuroscience
Oncology
Cannabinoids
1Partnered collaboration;
2Recently acquired;
3Planned;
4Phase 1 trial ongoing in Japan – expect to rapidly advance development in U.S.;
5Jazz & MD Anderson Cancer Center collaboration study;
6JZP150 is a fatty acid amide hydrolase inhibitor which modulates the endocannabinoid anandamide;
7Cooperative group study;
8FDA approval on June 30, 2021; submitted additional data to support U.S. label update.

1L = first line, ALL/LBL = acute lymphoblastic leukemia / lymphoblastic lymphoma, AML = acute myeloid leukemia, COG = Children’s Oncology Group, HMA = hypomethylating agents, HR = high-risk, HRD+ = homologous recombinant deficient positive, MDS = myelodysplastic syndromes, MS = multiple sclerosis, PTSD = post-traumatic stress disorder, R/R = relapsing/refractory, SCLC = small cell lung cancer, SG = study group

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

Suvecaltamide (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