Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
tividenofusp alfa · 3 trials · 1 indication
The intensity of IRRs will be assessed following each infusion of DNL310 using the categories of Mild, Moderate and Severe. IRRs will be summarized overall as well as stratified by intensity.
| Arm | Type | Description |
|---|---|---|
| Cohort A2 | EXPERIMENTAL | Participants with nMPS II, aged ≥5 to ≤10 years |
| Cohort B2 | EXPERIMENTAL | Participants with nMPS II or nnMPS II, aged ≥1 to ≤18 years |
| Cohort C2 | EXPERIMENTAL | Participants with nMPS II, aged \<4 years |
| Cohort D2 | EXPERIMENTAL | Participants with nMPS II or nnMPS II, aged ≤18 years with preexisting hepatomegaly who have never taken standard-of-care ERT |
| Cohort E2 | EXPERIMENTAL | Participants with nMPS II, aged ≥6 years; participants with nnMPS II, aged \<6 or ≥17 years; or participants with nMPS II, aged ≥1 to ≤18 years, with a history of prior HSCT or gene therapy and have completed at least 48 weeks in Study DNLI-E-0001 |
| Cohort A7 | EXPERIMENTAL | Participants with nMPS II, aged ≥2 to \<6 years |
| Cohort B7 | EXPERIMENTAL | Participants with nnMPS II, aged ≥6 to \<17 years |
| Cohort A: Participants with nMPS II | EXPERIMENTAL | - |
| Cohort B: Participants with nnMPS II | EXPERIMENTAL | - |
| Open-label Treatment Phase | EXPERIMENTAL | Participants who meet pre-specified criteria may receive DNL310 or idursulfase |
| Cohort A | EXPERIMENTAL | Dose escalation followed by a consistent dose level in participants with neuronopathic MPS II |
| Cohort B | EXPERIMENTAL | A consistent dose level in participants with non-neuronopathic MPS II, neuronopathic MPS II, or unknown phenotype followed by dose escalation in some participants. |
| Cohort C | EXPERIMENTAL | A consistent dose level in participants with neuronopathic MPS II |
| Cohort D | EXPERIMENTAL | A consistent dose level in participants with non-neuronopathic MPS II or neuronopathic MPS II |
| Cohort E | EXPERIMENTAL | A consistent dose level in participants with non-neuronopathic MPS II or neuronopathic MPS II |
| Name | Type | Description |
|---|---|---|
| tividenofusp alfa | DRUG | Intravenous repeating dose |
| idursulfase | DRUG | Intravenous repeating dose |
Key Inclusion Criteria: * For participants from Study DNLI-E-0002 only: Completed at least through the Week 49 visit in Study DNLI-E-0002 and did not discontinue study intervention early * For participants from Study DNLI-E-0007 only: Completed the treatment period of 96 weeks in Cohort A for nMPS ...
Tividenofusp alfa is an investigational therapy being developed for Mucopolysaccharidosis II, also known as Hunter syndrome, a rare inherited disorder. It is in Phase 2 clinical development and is not yet approved for commercial use. Denali Therapeutics is studying it in pediatric and young adult patients with both neuronopathic and non-neuronopathic forms of the disease.
Tividenofusp alfa targets IDS, the iduronate-2-sulfatase enzyme that is deficient in Mucopolysaccharidosis II. By addressing this enzyme target, the therapy aims to treat the underlying cause of the condition. It is designed as a small molecule therapeutic and is being evaluated in Phase 2 trials in patients with MPS II.
Tividenofusp alfa is being developed by Denali Therapeutics Inc., which trades under the ticker DNLI. The company is running multiple clinical studies of the candidate in Mucopolysaccharidosis II, including a Phase 2 comparison against idursulfase and a long-term extension study.
Tividenofusp alfa is in Phase 2 clinical development for Mucopolysaccharidosis II. It is investigational and has not been approved by the FDA. The program includes three active trials: two Phase 2 studies and one Phase 1 study, with a combined enrollment of 211 participants across the program.
Tividenofusp alfa is being studied in three active trials. NCT05371613 is a Phase 2 study comparing it with idursulfase in pediatric and young adult patients. NCT06075537 is a Phase 2 long-term extension study, and NCT04251026 is a Phase 1 study in pediatric participants with Hunter syndrome.
Yes, tividenofusp alfa is also known as DNL310. Both names refer to the same investigational therapy from Denali Therapeutics being developed for Mucopolysaccharidosis II. The DNL310 designation appears in the trial titles for the Phase 1 and Phase 2 studies of the program.