CEB-01

Phase 3Small molecule

BioMarin Pharmaceutical

CEB-01 is one of 11 small molecule programs targeting Pancreatic Carcinoma. It is currently in Phase 3.

Indications

Total Trials

Active Trials

Competitors

Pipeline

Status by Indication[3][1]

Indication

Pancreatic CarcinomaActive, not recruiting

PhenylketonuriaActive, not recruiting

PediatricsActive, not recruiting

Pancreatic CarcinomaPhase 1

Active, not recruiting

PhenylketonuriaPhase 3

Active, not recruiting

PediatricsPhase 2

Active, not recruiting

Competition

Competitive Landscape

8 competitors in Phenylketonuria

#DrugCompanyPhaseTrials

Avelox

Bayer

Approved

—2

L-Carnitine

IIperboreal Pharma Srl

Phase 3

1 3

Sephience

PPTC Therapeutics

Phase 3

2 4

CEB-01

BioMarin Pharmaceutical

Phase 3

1 5

Pegvaliase

BioMarin Pharmaceutical

Preclinical

—6

AG-181

AAgios Pharmaceuticals, Inc.

Phase 1

1 7

Repinatrabit

Otsuka

Phase 1

Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.

Timeline

Expected Milestones

Q3 2028

Phase 1Active, not recruiting

Pancreatic Carcinoma

NCT06538857

Q2 2029

Phase 2Active, not recruiting

Pediatrics

NCT06986811

Clinical

Trials by Indication[1]

Pediatrics1 trial

First-in-pediatrics, Open Label, Exploratory, Externally Controlled Clinical Trial to Evaluate Safety, Efficacy and Pharmacokinetics of 7-ethyl-10-hydroxy Camptothecin (SN-38) Formulated as a Biocompatible Polymeric Nanofiber Membrane (CEB-01) for Treatment, in Addition to Standard of Care, of Pediatric Patients From Birth to Less Than 18 Years of Age With a Locally Resectable Tumors in Comparison to Standard of Care

Active, not recruiting·CCEBIOTEX·Phase 2·NCT06986811·80 enrolled

Pancreatic Carcinoma1 trial

Exploratory Clinical Trial to Assess Safety, Tolerability Efficacy and Pharmacokinetics of CEB-01 PLGA Membrane in Participants With Pancreatic Cancer.

Active, not recruiting·CCEBIOTEX·Phase 1·NCT06538857·39 enrolled

Phenylketonuria1 trial

PEGVALIASE-LT: Pegvaliase Long-Term in PKU

Active, not recruiting· BioMarin Pharmaceutical logo

BioMarin Pharmaceutical·Phase 3·NCT04619056·168 enrolled

Sources

References

[1]
ClinicalTrials.gov — 3 trial records for CEB-01. U.S. National Library of Medicine, National Institutes of Health.
[2]
ChEMBL — CEB-01 compound record. European Bioinformatics Institute (EMBL-EBI).
[3]
Open Targets Platform — Drug–disease associations for CEB-01. EMBL-EBI & Wellcome Sanger Institute.
[4]
SEC EDGAR — BioMarin Pharmaceutical company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Modality: Small molecule[2]
Highest Phase: Phase 3[1]
Company: BioMarin Pharmaceutical

CEB-01

Status by Indication[3][1]

Competitive Landscape

Expected Milestones

Q3 2028

Q2 2029

Trials by Indication[1]

Pediatrics1 trial

Pancreatic Carcinoma1 trial

Phenylketonuria1 trial

References

Drug Info

Quick Facts

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