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 therapy being developed for Mucopolysaccharidosis Type IIIA, also known as Sanfilippo syndrome type A, a rare inherited lysosomal storage disorder. It is a small molecule designed to address the underlying enzyme deficiency associated with this condition. DNL126 has received Orphan Drug and Fast Track designations from the FDA.
DNL126 targets SGSH, the enzyme sulfamidase that is deficient in Mucopolysaccharidosis Type IIIA. By targeting SGSH, DNL126 aims to address the enzyme deficiency that leads to heparan sulfate accumulation in this lysosomal storage disorder. It is a small molecule therapeutic candidate.
DNL126 is being developed by Denali Therapeutics Inc., which trades on the Nasdaq under the ticker symbol DNLI. Denali is a biopharmaceutical company focused on therapies for neurodegenerative and rare diseases, and DNL126 is its small molecule candidate for Mucopolysaccharidosis Type IIIA.
DNL126 is in Phase 1 clinical development. It is an investigational drug and has not been approved by the FDA. The Phase 1 program is evaluating DNL126 in pediatric participants with Mucopolysaccharidosis Type IIIA, and the FDA has granted the program Orphan Drug and Fast Track designations.
DNL126 is being studied in one active clinical trial, NCT06181136, titled Study of DNL126 in Pediatric Participants With Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome Type A). The Phase 1 trial is active but not recruiting, has an enrollment of 20 participants, and is being conducted in the United States.