Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
DNL126 · 1 trial · 1 indication
| Arm | Type | Description |
|---|---|---|
| Cohort A1 | EXPERIMENTAL | Participants with MPS IIIA |
| Cohort A2 | EXPERIMENTAL | Participants with MPS IIIA |
| Cohort A3 | EXPERIMENTAL | Participants with MPS IIIA |
| Cohort B1 | EXPERIMENTAL | Participants with MPS IIIA |
| Cohort B2 | EXPERIMENTAL | Participants with MPS IIIA |
| Name | Type | Description |
|---|---|---|
| DNL126 | DRUG | intravenous repeating dose |
Key Inclusion Criteria: * Confirmed diagnosis of MPS IIIA * For Cohort A2: No more than 1 participant may have predictors of a slow-progressing phenotype * For Cohort A3: Approximately 2 participants will have predictors of the slow-progressing phenotype * For Cohort B1: Have a severe phenotype bas...
DNL126 is an investigational small molecule being developed for the treatment of Mucopolysaccharidosis Type IIIA, also known as Sanfilippo Syndrome Type A. It is currently in Phase 1 clinical development for this rare disease indication.
DNL126 is being developed by Denali Therapeutics Inc., a biopharmaceutical company traded on the NASDAQ under the ticker symbol DNLI. The company is conducting clinical trials of DNL126 in the United States.
DNL126 is in Phase 1 clinical development. It is an investigational drug and has not been approved by the FDA. The drug has received Orphan Drug, Fast Track, and Accelerated Approval designations from the FDA.
DNL126 is being studied in a Phase 1 clinical trial with the identifier NCT06181136. This trial is titled 'Study of DNL126 in Pediatric Participants With Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome Type A)' and is active but not recruiting participants.
DNL126 is an investigational treatment specifically for Mucopolysaccharidosis Type IIIA, which is also known as Sanfilippo Syndrome Type A. It is being studied in pediatric participants with this condition.