Product Description
PTC857, discovered through PTC's Bio-e platform, is an investigational small molecule drug that inhibits 15-Lipoxygenase, an enzyme that is a key regulator of the oxidative stress, protein aggregation and inflammation response pathways that underpin neurodegenerative disease pathology. The first indication planned for PTC857 is Parkinson's disease caused by a mutation in the GBA gene ("GBA Parkinson's disease"), which is one of the most common genetic subtypes of Parkinson's disease. (Sourced from: https://ir.ptcbio.com/news-releases/news-release-details/ptc-therapeutics-announces-first-patient-dosed-phase-1-trial)
Mechanisms of Action: ALOX15 Inhibitor
Novel Mechanism: No
Modality: Small Molecule
Route of Administration: Oral
FDA Designation: None *
Approval Status: Not Approved
Approved Countries: None
Approved Indications: None
Known Adverse Events: None
Company: PTC Therapeutics
Company Location: SOUTH PLAINFIELD NJ 07080-2449
Company CEO: Stuart W. Peltz
Additonal Commercial Interests: None
Clinical Description
Countries in Clinic: Argentina, Australia, Belgium, Brazil, Canada, Czech Republic, France, Germany, Ireland, Italy, Japan, Mexico, Netherlands, Norway, Poland, Spain, Sweden, United States
Active Clinical Trial Count: 5
Highest Development Phases
Phase 2: Amyotrophic Lateral Sclerosis
Trial |
Phase |
Trial Status |
Disease |
Primary Completion Date |
Probability of Success |
|---|---|---|---|---|---|
| PTC857-CNS-001-ALS | P2 |
Unknown Status |
Amyotrophic Lateral Sclerosis |
2027-06-07 |
|
| jRCT2011230043 | P2 |
Recruiting |
Amyotrophic Lateral Sclerosis |
2025-07-31 |
|
| jRCT2011230040 | P2 |
Recruiting |
Amyotrophic Lateral Sclerosis |
2025-07-31 |
|
| CARDINALS | P2 |
Active, not recruiting |
Amyotrophic Lateral Sclerosis |
2025-04-26 |