PTC299

Identification

Generic Name
PTC299
DrugBank Accession Number
DB05173
Background

PTC299 is a novel, orally administered small-molecule designed to inhibit the production of vascular endothelial growth factor (VEGF) in tumors. Overexpression of VEGF plays a key role in multiple diseases including cancer and macular degeneration. PTC299 was discovered through PTC's GEMS technology by targeting the post-transcriptional processes that regulate VEGF formation, and is currently being developed for the treatment of cancer.

Type
Small Molecule
Groups
Investigational
Synonyms
Not Available

Pharmacology

Indication

Investigated for use/treatment in cancer/tumors (unspecified) and solid tumors.

Pharmacology
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Contraindications & Blackbox Warnings
Contraindications
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Pharmacodynamics

PTC299 demonstrated a broad range of activity in blocking VEGF synthesis in multiple tumor types, including breast, cervical, colorectal, fibrosarcoma, gastric, lung, melanoma, neuroblastoma, ovarian, pancreatic, prostate and renal cell cancer lines. PTC299 as a monotherapy significantly reduced VEGF concentrations in tumors and plasma, reduced tumor blood vessel density, and substantially impeded tumor progression.

Mechanism of action

PTC299 was designed to inhibit VEGF production in tumors by targeting the post-transcriptional control processes that regulate VEGF formation. Because PTC299 inhibits VEGF production, its action occurs at a different point in the VEGF pathway than therapies, such as Avastin® or Sutent®. PTC299 may be active both as a single agent or when used in combination with other anti-angiogenic agents or with chemotherapy agents for the treatment of cancers.

Absorption

Not Available

Volume of distribution

Not Available

Protein binding

Not Available

Metabolism
Not Available
Route of elimination

Not Available

Half-life

Not Available

Clearance

Not Available

Adverse Effects
Medicalerrors
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Toxicity

Not Available

Pathways
Not Available
Pharmacogenomic Effects/ADRs
Not Available

Interactions

Drug Interactions
This information should not be interpreted without the help of a healthcare provider. If you believe you are experiencing an interaction, contact a healthcare provider immediately. The absence of an interaction does not necessarily mean no interactions exist.
Not Available
Food Interactions
Not Available

Categories

Drug Categories
Classification
Not classified
Affected organisms
Not Available

Chemical Identifiers

UNII
Not Available
CAS number
Not Available
InChI Key
Not Available
InChI
Not Available
IUPAC Name
Not Available
SMILES
Not Available

References

General References
Not Available
PubChem Substance
347909999

Clinical Trials

Clinical Trials
PhaseStatusPurposeConditionsCount
2TerminatedTreatmentNeurofibromatosis 21
2, 3RecruitingTreatmentCoronavirus Disease 2019 (COVID‑19) / Infections, Coronavirus / Pneumonia1
1CompletedTreatmentBrain and Central Nervous System Tumors1
1CompletedTreatmentMetastatic Breast Cancer1
1RecruitingTreatmentAcute Myeloid Leukemia (AML)1
1TerminatedTreatmentCancer, Advanced1
1, 2TerminatedTreatmentKaposi’s sarcoma1

Pharmacoeconomics

Manufacturers
Not Available
Packagers
Not Available
Dosage Forms
Not Available
Prices
Not Available
Patents
Not Available

Properties

State
Solid
Experimental Properties
Not Available
Predicted Properties
Not Available
Predicted ADMET Features
Not Available

Spectra

Mass Spec (NIST)
Not Available
Spectra
Not Available

Drug created on October 21, 2007 22:24 / Updated on June 12, 2020 16:52