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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 small molecule being developed for Mucopolysaccharidosis II, also known as Hunter syndrome. It is being studied in pediatric, young adult, and adult participants with both neuronopathic and non-neuronopathic forms of the disease. The drug is currently in Phase 2 clinical development.
Tividenofusp alfa is being developed by Denali Therapeutics Inc., a biopharmaceutical company traded on the Nasdaq under the ticker symbol DNLI. The company is conducting multiple clinical trials of the drug in patients with Mucopolysaccharidosis II across numerous countries, including the United States, Canada, and several European nations.
Tividenofusp alfa is currently in Phase 2 clinical development. It has received several FDA designations, including Priority Review, Breakthrough Therapy, Orphan Drug, Fast Track, and Rare Pediatric Disease. The drug is investigational and has not been approved by the FDA, as it is still undergoing clinical trials.
Tividenofusp alfa is being studied in three active clinical trials. NCT04251026 is a Phase 1 study in pediatric participants with Hunter syndrome. NCT05371613 is a Phase 2 study comparing tividenofusp alfa to idursulfase in pediatric and young adult participants. NCT06075537 is a Phase 2 extension study evaluating long-term safety and efficacy.
Yes, tividenofusp alfa is also known as DNL310. Clinical trial records refer to the drug by both names, with DNL310 appearing in study titles and tividenofusp alfa as the official nonproprietary name. Both names refer to the same investigational therapy for Mucopolysaccharidosis II.
Tividenofusp alfa is a small molecule designed to treat Mucopolysaccharidosis II. The drug is being evaluated for its ability to address the underlying enzyme deficiency associated with the condition. However, the specific molecular target of tividenofusp alfa has not been disclosed in the available clinical trial information.