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.
Reduce drug development failure ratesBuild, train, & validate machine-learning modelswith evidence-based and structured datasets.Build, train, & validate predictive machine-learning models with structured datasets.- Contraindications & Blackbox Warnings
Avoid life-threatening adverse drug eventsImprove clinical decision support with information on contraindications & blackbox warnings, population restrictions, harmful risks, & more.Avoid life-threatening adverse drug events & improve clinical decision support.- 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
Improve decision support & research outcomesWith structured adverse effects data, including: blackbox warnings, adverse reactions, warning & precautions, & incidence rates.Improve decision support & research outcomes with our structured adverse effects data.- 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
- 053QD2I96A
- CAS number
- Not Available
- InChI Key
- Not Available
- InChI
- Not Available
- IUPAC Name
- Not Available
- SMILES
- Not Available
References
- General References
- Not Available
- External Links
- PubChem Substance
- 347909999
Clinical Trials
- Clinical Trials
Phase Status Purpose Conditions Count 2 Terminated Treatment Neurofibromatosis 2 1 2, 3 Recruiting Treatment Coronavirus Disease 2019 (COVID‑19) / Infections, Coronavirus / Pneumonia 1 1 Completed Treatment Brain and Central Nervous System Tumors 1 1 Completed Treatment Metastatic Breast Cancer 1 1 Terminated Treatment Acute Myeloid Leukemia (AML) 1 1 Terminated Treatment Cancer, Advanced 1 1, 2 Terminated Treatment Kaposi’s sarcoma 1
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 at October 21, 2007 22:24 / Updated at June 12, 2020 16:52