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Pipeline

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Pipeline

PRE-CLINICAL
Undisclosed targets
Neuroscience
CombiPlex
Exploratory activities
Undisclosed target
Ras/Raf/MAP kinase pathway1
Exosome targets
(Up to 3 targets)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
Orexin-2 receptor agonist
JZP815
Pan-Raf Inhibitor program
Raf & Ras mutant tumors
Zanidatamab2
HER2-targeted bispecific antibody
Breast cancer
JZP341
(Long-acting Erwinia asparaginase)
Solid tumors
JZP351 (Vyxeos)
Low Intensity Dosing for higher risk MDS4
JZP351 + other approved therapies
  • R/R AML or HMA Failure MDS4
  • 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
Suvecaltamide (JZP385)
Parkinson’s Disease Tremor
JZP1505
PTSD
Zanidatamab2
HER2-targeted bispecific antibody
  • 2L zani monotherapy for BTC6 (Pivotal trial)
  • Additional trials ongoing in BTC, GEA and CRC
  • Multiple trials ongoing in breast cancer
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 AML4
JZP541

Irritability associated with autism spectrum disorders
Additional Cannabinoids

Autism spectrum disorders
PHASE 3
Zanidatamab2
HER2-targeted bispecific antibody
1L zani + chemo ± tislelizumab for GEA8
(Pivotal trial)

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)7
Cannabidiol (Epidiolex)
EMAS

Cannabidiol (Epidyolex)
LGS, DS, TSC in Japan

REGULATORY
JZP458 (Rylaze)9
(recombinant Erwinia asparaginase)
ALL/LBL
Neuroscience
Oncology
Cannabinoids
1Partnered collaboration;
2Recently acquired;
3Planned;
4Jazz & MD Anderson Cancer Center collaboration study;
5JZP150 is a fatty acid amide hydrolase inhibitor which modulates the endocannabinoid anandamide;
6HERIZON-BTC-01;
7Cooperative group study;
8HERIZON-GEA-01;
9FDA approval on June 30, 2021 and FDA approval of sBLA for MWF IM dosing on November 18, 2022. In February 2023, received a complete response letter from FDA requesting additional clinical data on the IV administration sBLA; currently evaluating complete response letter to determine next steps. Submitted Marketing Authorization Application to EMA.

1L = first line, ALL/LBL = acute lymphoblastic leukemia / lymphoblastic lymphoma, AML = acute myeloid leukemia, COG = Children’s Oncology Group, BTC = biliary tract cancer, CRC = colorectal cancer, DS = Dravet syndrome, EMAS = epilepsy with myoclonic-atonic seizures, GEA = gastroesophageal adenocarcinoma, HMA = hypomethylating agents, HR = high-risk, HRD+ = homologous recombinant deficient positive, LGS = Lennox-Gastaut syndrome, MDS = myelodysplastic syndromes, PTSD = post-traumatic stress disorder, R/R = relapsing/refractory, SCLC = small cell lung cancer, SG = study group, TSC = Tuberous sclerosis complex.

Molecule
Phase

JZP458

  • Acute lymphoblastic leukemia and lymphoblastic lymphoma
Regulatory

Cannabidiol

  • Epilepsy with myoclonic-atonic seizures (EMAS)
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

JZP541

  • Irritability associated with autism spectrum disorders
Pre-clinical