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PTC923
PTC923 is a small-molecule inhibitor of pyruvate dehydrogenase kinase (PDK) that restores pyruvate dehydrogenase (PDH) activity to improve mitochondrial energy metabolism.
PTC923 is a small-molecule inhibitor of pyruvate dehydrogenase kinase (PDK) that restores pyruvate dehydrogenase (PDH) activity to improve mitochondrial energy metabolism. Used for Mitochondrial neurogastrointestinal encephalomyopathy (MNGIE), Other mitochondrial or metabolic disorders.
At a glance
| Generic name | PTC923 |
|---|---|
| Also known as | CNSA-001, Sepiapterin, Sepiapterin, Sephience |
| Sponsor | PTC Therapeutics |
| Drug class | Pyruvate dehydrogenase kinase (PDK) inhibitor |
| Target | PDK (pyruvate dehydrogenase kinase) |
| Modality | Small molecule |
| Therapeutic area | Rare genetic/metabolic disorders |
| Phase | Phase 3 |
Mechanism of action
By inhibiting PDK, PTC923 allows pyruvate dehydrogenase to remain active, promoting the conversion of pyruvate to acetyl-CoA and enhancing oxidative metabolism. This mechanism is designed to address metabolic dysfunction in mitochondrial disorders and other conditions characterized by impaired energy production. The restoration of PDH activity aims to improve cellular energy availability and reduce lactate accumulation.
Approved indications
- Mitochondrial neurogastrointestinal encephalomyopathy (MNGIE)
- Other mitochondrial or metabolic disorders
Common side effects
- Peripheral neuropathy
- Gastrointestinal disturbances
- Fatigue
Key clinical trials
- A Study of Sepiapterin in Participants With Phenylketonuria (PKU) (PHASE3)
- A Long-Term Safety Study of PTC923 in Participants With Phenylketonuria (PHASE3)
- A Study of PTC923 in Participants With Phenylketonuria (PHASE3)
- A Study of PTC923 (CNSA-001) in Primary Tetrahydrobiopterin (BH4) Deficient Participants With Hyperphenylalaninemia (PHASE1, PHASE2)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- PTC923 CI brief — competitive landscape report
- PTC923 updates RSS · CI watch RSS
- PTC Therapeutics portfolio CI