Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Label indication DAYBUE and DAYBUE STIX are indicated for the treatment of Rett syndrome in adults and pediatric patients 2 years of age and older. DAYBUE and DAYBUE STIX are indicated for the treatment of Rett syndrome in adults and pediatric patients 2 years of age and older. ( 1 ) FDA label
Trofinetide · 2 trials · 1 indication
Percentage of subjects with treatment-emergent adverse events (TEAEs), percentage of subjects with serious adverse events (SAEs), and percentage of subjects with withdrawals due to AEs
Potentially clinically important ECG changes were defined in the study protocol as absolute QTcF interval \>500 ms or QTcF interval change from the baseline value of previous study ACP-2566-003 of \>60 ms
Potentially clinically important changes in vital signs were defined in the study protocol as: systolic blood pressure (SBP) ≥180 mmHg and increased ≥20 mmHg from baseline; SBP ≤90 mmHg and decreased ≥20 mmHg from baseline; diastolic blood pressure (DBP) ≥ 105 mmHg and increased ≥15 mmHg from baseline; DBP ≤50 mmHg and decreased ≥15 mmHg from baseline; Pulse ≥120 bpm and increased ≥15 bpm from baseline; Pulse ≤50 bpm and decreased ≥15 bpm from baseline
Potentially clinically important changes in body weight were defined in the study protocol as: Weight increase ≥7% from baseline; Weight decrease ≥7% from baseline
Potentially clinically important changes in laboratory parameters were defined in the study protocol as: Sodium ≤125 mmol/L; Sodium ≥155 mmol/L; Potassium ≤3.0 mmol/L; Potassium ≥5.5 mmol/L; Chloride ≤85 mmol/L; Chloride ≥120 mmol/L; Calcium \<2.0 mmol/L; Calcium \>2.0 mmol/L; Blood urea nitrogen ≥10.71 mmol/L; Creatinine \>1.5 x upper limit of normal (ULN); Uric acid ≥505.75 μmol/L; Lactate dehydrogenase ≥3 x ULN; Glucose ≤2.48 mmol/L; Glucose ≥11 mmol/L; Albumin ≤26 g/L; Albumin ≥60 g/L; Protein ≤50 g/L; Protein ≥100 g/L; Alanine aminotransferase ≥3 x ULN; Aspartate aminotransferase ≥3 x ULN; Gamma glutamyl transpeptidase ≥3 x ULN; Alkaline phosphatase ≥3 x ULN; Bilirubin ≥1.5 x ULN
The RSBQ is a 45-item caregiver-completed rating scale that includes 45 items, 39 of them grouped into 8 subscales, whose ratings reflect the severity and frequency of symptoms. Items are rated as 0 (not true), 1 (somewhat or sometimes true), or 2 (very true). The 8 subscales are general mood, breathing problems, hand behavior, face movements, body rocking/expressionless face, night-time behaviors, fear/anxiety, and walking/standing. Scores for item 31 are reversed in the calculation of the total score. The total score ranges from 0 to 90 and is calculated as the sum of the item scores. Higher scores mean worse behaviour.
To rate how much the subject's illness has improved or worsened relative to a baseline state, a 7-point scale is used from 1=very much improved, 2=much improved, 3=minimally improved, 4=no change, 5=minimally worse, 6=much worse, 7=very much worse.
| Arm | Type | Description |
|---|---|---|
| Trofinetide | EXPERIMENTAL | - |
| Drug - Trofinetide | EXPERIMENTAL | Trofinetide solution of 30-60 mL based on the subject's weight at Baseline, administered twice daily by mouth or gastrostomy tube (G-tube) |
| Placebo | PLACEBO_COMPARATOR | Trofinetide placebo solution of 30-60 mL based on the subject's weight at Baseline, administered twice daily by mouth or gastrostomy tube (G-tube) |
| Name | Type | Description |
|---|---|---|
| Trofinetide | DRUG | Trofinetide solution of 30-60 mL based on subject's weight at Baseline, administered twice daily by mouth or gastrostomy tube (G-tube) |
| Placebo | OTHER | Trofinetide placebo solution administered based on the subject's weight at Baseline, twice daily for 12 weeks |
Inclusion Criteria: 1. Has completed the Week 12/End-of-treatment visit of the antecedent study, Study ACP-2566-003 2. Met all entry criteria for the antecedent study 3. May benefit from long-term treatment with open-label trofinetide in the judgment of the Investigator 4. Can still swallow the stu...
Top 2 of 7 competitors
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Akebia Therapeutics, Inc. | AKBA | 1 | PHASE2 | Vadadustat |
| Rigel Pharmaceuticals, Inc. | RIGL | 1 | PHASE2 | Fostamatinib |
Trofinetide is being developed for the treatment of Rett Syndrome, a rare neurodevelopmental disorder. It is an investigational small molecule studied in girls and women with Rett Syndrome. The clinical program includes two completed Phase 3 trials, and the drug has not been approved by the FDA.
Trofinetide is a small molecule belonging to the -tide (peptide) class. Its target class is described as a peptide, and it was studied with biomarker selection in its clinical program. The specific molecular target is not further characterized in the available reference information.
Trofinetide is being developed by ACADIA Pharmaceuticals Inc., which trades under the ticker ACAD. The company is the sponsor of the Phase 3 clinical program evaluating the drug in Rett Syndrome.
Trofinetide is in Phase 3 development. Two Phase 3 trials have been completed, and no trials are currently active. The drug remains investigational and has not been approved by the FDA for any indication.
Trofinetide has been studied in two completed Phase 3 trials: NCT04181723, known as the LAVENDER trial, which enrolled 187 participants, and NCT04279314, an open-label extension study that enrolled 154 participants. Both trials enrolled girls and women with Rett Syndrome in the United States.
Both Phase 3 trials of Trofinetide were randomized, double-blind, and placebo-controlled, with a total enrollment of 341 participants across the two studies. Participants were female, aged 5 years and older, and had Rett Syndrome. The trials were conducted in the United States.