Drug intelligence / Profile preview

JBZ-001

Development stage
Phase 1
Lead developer
JBZ
Modality
Nucleic Acid-Directed Small Molecules → Small Molecules, Covalent Small Molecules → Small Molecules, Classical Binding Small Molecules → Small Molecules
Administration
Oral
01

Overview

JBZ-001 is an orally bioavailable small-molecule inhibitor of dihydroorotate dehydrogenase (DHODH), the rate-limiting enzyme in the de novo pyrimidine nucleotide biosynthesis pathway. By inhibiting DHODH, JBZ-001 disrupts nucleotide production essential for cancer cell proliferation, leading to cytotoxic effects in tumor cells. The drug is being developed by Jabez Biosciences as a next-generation targeted therapy for advanced solid tumors and hematological malignancies, including non-Hodgkin lymphoma and acute myeloid leukemia (AML). Preclinical studies have shown robust anti-cancer activity and favorable safety profiles. As of mid-2025, JBZ-001 is undergoing Phase 1 clinical trials to assess its safety, tolerability, pharmacokinetics/pharmacodynamics (PK/PD), and preliminary efficacy in patients with advanced cancers[1][2][3][5][6][7].

Other names
JBZ 001JBZ001JBZ-001
02

Targets

DHODH (Dihydroorotate dehydrogenase (quinone), mitochondrial)

Beyond the preview

Go deeper on JBZ-001.

Explore the evidence, development activity, and competitive landscape with Gosset’s full data platform.

Clinical trials

Full profile access

Follow clinical development from study design and recruitment through results.

  • Trial phase
  • Status
  • Readouts

Indications & development

Full profile access

Explore development by indication, patient population, and geography.

  • Indications
  • Development status
  • Countries

Licensing & deals

Full profile access

Trace asset ownership, licensing agreements, and commercial partnerships.

  • Partners
  • Deal terms
  • Milestones

Patents & exclusivity

Full profile access

Explore the patent landscape and regulatory exclusivity around an asset.

  • Patents
  • Expiration dates
  • Exclusivity

Competitive landscape

Full profile access

Compare development programs by target, modality, and indication.

  • Competing assets
  • Targets
  • Development stage

Research & analysis

Full profile access

Connect source evidence and development news to your research questions.

  • Publications
  • News
  • Analysis

Bring the full picture into focus.

See how Gosset can support your research on JBZ-001.

Explore the full profile

Gosset Free

Get started with Gosset.

Enter your work email and we’ll be in touch with next steps.

Work email preferred.

Book a call