Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
PTC923 · 3 trials · 3 indications
A TEAE is any unfavorable or unintended sign (including an abnormal laboratory finding), symptom or disease in a study participant who is administered study drug in this study
Phe tolerance is defined as the total amount of dietary Phe (milligrams \[mg\]/kilogram \[kg\] per day) ingested while maintaining blood Phe levels within the range of 40 to 360 micromoles (μmol)/liter (L) (defined as ≥40 to \<360 μmol/L).
Baseline was defined as the average of Day -1 and Day 1 predose blood Phe levels in Part 2, and mean level at Weeks 5 and 6 was calculated as the average of blood Phe levels collected during the Week 5-6 analysis visit window. Least square (LS) mean and standard error (SE) were calculated using mixed model repeated measures (MMRM) method.
Baseline was defined as the average of Day -1 and Day 1 predose blood Phe levels in Part 2, and mean level at Weeks 5 and 6 was calculated as the average of blood Phe levels collected during the Week 5-6 analysis visit window. LS mean and SE were calculated using MMRM method.
An adverse event (AE) was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Serious adverse event (SAE) was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. AEs included both SAEs and non-serious AEs. TEAEs were defined as AEs that commenced or worsened after the first dose of study drug. A summary of other non-serious AEs and all SAEs, regardless of causality is located in the 'Reported AE section'.
| Arm | Type | Description |
|---|---|---|
| PTC923 | EXPERIMENTAL | Participants will receive PTC923 7.5 mg/kg (participants 0 to \<6 months of age), 15 mg/kg (participants 6 to \<12 months of age), 30 mg/kg (participants 12 months to \<2 years of age), or 60 mg/kg (participants ≥2 years of age) orally once daily for a minimum of 12 months or until participant experiences lack of efficacy, adverse events (AEs) that lead to discontinuation, withdraws from treatment, or PTC923 is authorized and commercially available in the specific country. |
| Part 1: PTC923 | EXPERIMENTAL | Participants will receive PTC923 7.5 milligrams (mg)/kilogram (kg) (participants 0 to \<6 months of age), 15 mg/kg (participants 6 to \<12 months of age), 30 mg/kg (participants 12 months to \<2 years of age), or 60 mg/kg (participants ≥2 years of age) orally once daily for 14 days. |
| Part 2: PTC923 | EXPERIMENTAL | Participants will receive PTC923 20 mg/kg daily for Weeks 1 and 2, then PTC923 40 mg/kg daily for Weeks 3 and 4, then PTC923 60 mg/kg daily for Weeks 5 and 6. |
| Part 2: Placebo | PLACEBO_COMPARATOR | Participants will receive equivalent quantities of placebo to match the 20 to 40 to 60 mg/kg dose escalation of the PTC923 treatment arm. |
| Cohort 1: PTC923 2.5 and 10 mg/kg/day | EXPERIMENTAL | Participants will receive PTC923 suspension 2.5 milligrams (mg)/kilogram (kg)/day (dose divided in 2 for twice daily administration) orally for 7 days in Period 1, undergo a 3 (±1) day washout period, then escalate to a dose of 10 mg/kg/day (dose divided in 2 for twice daily administration) administered orally for 7 days in Period 2 (14 days total treatment). |
| Cohort 2: PTC923 5 and 20 mg/kg/day | EXPERIMENTAL | Participants will receive PTC923 suspension 5 mg/kg/day (dose divided in 2 for twice daily administration) orally for 7 days in Period 1, undergo a 3 (±1) day washout period, then escalate to a dose of 20 mg/kg/day (dose divided in 2 for twice daily administration) administered orally for 7 days in Period 2 (14 days total treatment). |
| Name | Type | Description |
|---|---|---|
| PTC923 | DRUG | PTC923 powder for oral use will be suspended in water or apple juice prior to administration. |
| Placebo | DRUG | Placebo matching to PTC923 |
Inclusion Criteria: * Clinical diagnosis of PKU with hyperphenylalaninemia (HPA) documented by past medical history of at least 2 blood Phe measurements ≥600 μmol/L. * Women of childbearing potential must have a negative pregnancy test at screening and agree to abstinence or the use of at least one...
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| BioMarin Pharmaceutical Inc. | BMRN | 5 | PHASE3 | Pegvaliase |
| PTC Therapeutics, Inc. | PTCT | 2 | PHASE3 | PTC923 |
| Sanofi SA Sponsored ADR | SNY | 1 | PHASE1 | SAR444836 |
| Agios Pharmaceuticals, Inc. | AGIO | 1 | PHASE1 | AG-181 |
| Illumina, Inc. | ILMN | 1 | - | Undisclosed |
| Q32 Bio Inc | QTTB | 1 | - | HMI-102 |
PTC923 is an investigational small molecule being developed for phenylketonuria (PKU) and BH4 deficiency, a rare genetic disorder causing hyperphenylalaninemia. It is in clinical development and has not been approved by the FDA. The drug is being studied in Phase 1 and Phase 3 trials for these conditions.
PTC923 targets sepiapterin reductase, an enzyme involved in the synthesis of tetrahydrobiopterin (BH4), a cofactor essential for phenylalanine metabolism. By modulating this enzyme, the drug aims to address the underlying metabolic defect in phenylketonuria and BH4 deficiency.
PTC923 is being developed by PTC Therapeutics, Inc., a biopharmaceutical company traded on NASDAQ under the ticker PTCT. The company is conducting clinical trials to evaluate the drug's safety and efficacy in patients with phenylketonuria and BH4 deficiency.
PTC923 is in Phase 1 clinical development for BH4 deficiency and has completed Phase 3 trials for phenylketonuria. It is an investigational drug, meaning it is not yet approved by regulatory authorities. A long-term safety study is ongoing, and the drug remains under clinical investigation.
PTC923 has been studied in three clinical trials. NCT03519711 was a completed Phase 1 study in BH4 deficiency with 8 participants. NCT05099640 was a completed Phase 3 trial in phenylketonuria with 157 participants. NCT05166161 is an active, not recruiting Phase 3 long-term safety study in phenylketonuria with 200 participants.
Yes, PTC923 is also known as CNSA-001. In the Phase 1 trial NCT03519711, the drug was referred to as PTC923 (CNSA-001). This alternative name may appear in earlier research literature or clinical trial records, but both names refer to the same investigational compound.