Drugs / DNL126
Trials 1
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/2 | NCT06181136 | Dec 2023 → Aug 2028 expected | mucopolysaccharidosis type 3A | Denali Therapeutics Inc. | Active not recruiting | No outcome recorded |
News releases announcing trial results or a regulatory action · 6
| Date | Issuer | Release |
|---|---|---|
| 2026-02-05 | Denali Therapeutics Inc. | Results Denali Therapeutics Presents Enzyme TransportVehicle™ Progress Across Three Clinical Programs for Treatment of Lysosomal Storage Disorders at 2026 WORLDSymposium™ denalitherapeutics.com ↗
Preliminary Phase 1/2 study data show treatment with DNL126 (ETV:SGSH) substantially reduced disease biomarkers in cerebrospinal fluid (CSF) and peripheral tissues, including an 80% mean reduction in CSF heparan sulfate |
| 2025-01-13 | Denali Therapeutics Inc. | Results Denali Therapeutics Announces Key Anticipated 2025 Milestones and Priorities to Further Advance Its Therapeutics Portfolio for Neurodegeneration and Lysosomal Storage Diseases denalitherapeutics.com ↗
Preliminary data from the ongoing Phase 1/2 study of DNL126 have demonstrated a substantial reduction in cerebrospinal fluid heparan sulfate (CSF HS) levels from baseline, including normalization. |
| 2024-11-06 | Denali Therapeutics Inc. | Results Denali Therapeutics Reports Third Quarter 2024 Financial Results and Business Highlights denalitherapeutics.com ↗
Today, we are also pleased to share that preliminary data from our Phase 1/2 study of DNL126 in MPS IIIA demonstrate a robust reduction from baseline in CSF heparan sulfate levels, including normalization. |
| 2024-06-03 | Denali Therapeutics Inc. | Regulatory Denali Therapeutics Announces FDA Has Selected DNL126 (ETV:SGSH) for MPS IIIA (Sanfilippo Syndrome Type A) for START Pilot Program Intended to Accelerate Development of Rare Disease Therapies denalitherapeutics.com ↗
DNL126 was selected as one of three CDER-regulated products based on eligibility criteria including the potential for clinical benefit for a rare neurodegenerative disease and the drug sponsor’s ability to advance development towards a marketing application |
| 2024-02-07 | Denali Therapeutics Inc. | Results Denali Therapeutics Announces New Data and Expansion of Its Blood-Brain Barrier (BBB)-Crossing Enzyme Replacement Therapy Programs at WORLDSymposium™ denalitherapeutics.com ↗
Announcing initiation of dosing with DNL126 (ETV:SGSH) in Phase 1/2 study in MPS IIIA and new supportive data with MPS IIIA mice showing reductions in heparan sulfate correlated with improvements in cognitive function |
| 2023-08-08 | Denali Therapeutics Inc. | Regulatory Denali Therapeutics Reports Second Quarter 2023 Financial Results and Business Highlights denalitherapeutics.com ↗
Today, Denali announced that the Investigational New Drug (IND) application for DNL126 in MPS IIIA has been cleared, and plans remain on track to initiate recruiting activities for the Phase 1/2 study in the second half of 2023. |
All press releases naming this drug 8 releases
Evidence & citations 2 cited values
Every value below carries the sentence it was read from. 1 source stands behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | DNL126 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT06181136 ↗ |
| Route | Intravenous | “intravenous repeating dose” NCT06181136 ↗ |