Approval Probability
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Adjusted LOA
ML Risk
NBI-74788 · 1 trial · 1 indication
Percent changes in 17-OHP were assessed through the collection of samples from 0600 hours to 1000 hours both prior to study drug administration (i.e., at baseline) and after 14 days of study drug dosing. The 3 samples collected during this morning window at each visit were averaged and used to determine the percent change from baseline.
| Arm | Type | Description |
|---|---|---|
| Cohort 1 (50 mg QHS) | EXPERIMENTAL | NBI-74788 50 mg once daily at bedtime (QHS) administered orally for 14 consecutive days. |
| Cohort 2 (100 mg QHS) | EXPERIMENTAL | NBI-74788 100 mg once daily at bedtime (QHS) administered orally for 14 consecutive days. |
| Cohort 3 (100 mg QPM) | EXPERIMENTAL | NBI-74788 100 mg once daily in the evening (QPM) administered orally for 14 consecutive days. |
| Cohort 4 (100 mg BID) | EXPERIMENTAL | NBI-74788 100 mg twice daily (BID) administered orally for 14 consecutive days. |
| Name | Type | Description |
|---|---|---|
| NBI-74788 | DRUG | Capsule, administered daily. |
Inclusion Criteria: 1. Be in good general health. 2. Have a medically confirmed diagnosis of classic 21-hydroxylase deficiency CAH. 3. Be on a stable regimen of steroidal treatment for CAH that is expected to remain stable throughout the study. 4. Subjects of childbearing potential must be instruct...
NBI-74788 is an investigational small molecule being developed for the treatment of congenital adrenal hyperplasia (CAH), an endocrine disorder. It is currently in Phase 2 clinical development and has not been approved by regulatory authorities.
The specific molecular target of NBI-74788 has not been disclosed in available information. It is a small molecule being studied for its effects on congenital adrenal hyperplasia, but its mechanism of action is not publicly detailed.
NBI-74788 is being developed by Neurocrine Biosciences, Inc., a biopharmaceutical company traded on NASDAQ under the ticker NBIX. The company is conducting clinical research on this compound for congenital adrenal hyperplasia.
NBI-74788 is in Phase 2 clinical development. It is an investigational drug, meaning it has not been approved by the FDA or other regulatory bodies. Its safety and efficacy are still being evaluated in clinical trials.
NBI-74788 has one completed Phase 2 clinical trial, NCT03525886, which evaluated its safety, tolerability, pharmacokinetics, and pharmacodynamics in 18 adults with congenital adrenal hyperplasia in the United States. The trial was controlled but not randomized or double-blinded.