Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
DNL952 · 1 trial · 1 indication
| Arm | Type | Description |
|---|---|---|
| Cohort A1 | EXPERIMENTAL | Participants with LOPD |
| Cohort A2 | EXPERIMENTAL | Participants with LOPD |
| Cohort A3 (Optional) | EXPERIMENTAL | Participants with LOPD |
| Cohort A4 (Optional) | EXPERIMENTAL | Participants with LOPD |
| Cohort B1 (Optional) | EXPERIMENTAL | Participants with LOPD |
| Cohort B2 (Optional) | EXPERIMENTAL | Participants with LOPD |
| Name | Type | Description |
|---|---|---|
| DNL952 | DRUG | Intravenous repeating dose |
Key Inclusion Criteria: * Body weight ≥40 kg * Diagnosis of LOPD * Upright FVC ≥ 30% of predicted normal value * Able to ambulate ≥ 40 meters (use of assistive devices is acceptable) * \[Cohorts A1-A4 only\] Have received avalglucosidase alfa or cipaglucosidase alfa at a dose of 20 mg/kg every 2 we...
DNL952 is an investigational small molecule being developed for the treatment of late-onset Pompe disease, a rare genetic disorder. It is currently in Phase 1 clinical development and has not been approved by regulatory authorities. The drug is being studied in adult participants with this condition.
DNL952 targets GAA, which is an enzyme. In late-onset Pompe disease, deficiencies in GAA activity lead to glycogen accumulation. By targeting this enzyme, DNL952 aims to address the underlying pathology of the disease. The drug is a small molecule designed to modulate GAA function.
DNL952 is being developed by Denali Therapeutics Inc., a biopharmaceutical company. Denali is publicly traded under the ticker symbol DNLI. The company is conducting clinical trials to evaluate the safety, pharmacokinetics, and pharmacodynamics of DNL952 in patients with late-onset Pompe disease.
DNL952 is in Phase 1 clinical development. It is an investigational drug and has not yet been approved by the FDA or other regulatory bodies. The ongoing Phase 1 trial is recruiting participants to assess the drug's safety, pharmacokinetics, and pharmacodynamics in adults with late-onset Pompe disease.
DNL952 is being evaluated in a Phase 1 clinical trial with the identifier NCT07354724. This study is titled 'A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of DNL952 in Adult Participants With Late-Onset Pompe Disease.' The trial is currently recruiting 32 participants in the United States and is controlled but not randomized or double-blinded.