Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
ION356 · 1 trial · 1 indication
| Arm | Type | Description |
|---|---|---|
| Cohort A: ION356 Dose A | EXPERIMENTAL | Participants will receive ION356 intrathecally at Dose A in the MAD Period, followed by ION356 Dose A in the LTE Period. |
| Cohort B: ION356 Dose B | EXPERIMENTAL | Participants will receive ION356 intrathecally at Dose B in the MAD Period, followed by ION356 Dose B in the LTE Period. |
| Name | Type | Description |
|---|---|---|
| ION356 | DRUG | Administered as intrathecal (IT) injection. |
Key Inclusion Criteria 1. Participant's parent or legally accepted representative can provide informed consent, attend all scheduled study visits, provide feedback regarding the participant's symptoms, and can comply with all study requirements. 2. Diagnosis of PMD with genetic confirmation of PLP1...
ION356 is an investigational small molecule being developed for Pelizaeus-Merzbacher Disease (PMD), a rare genetic disorder. It is administered intrathecally and is currently in Phase 1 clinical development. The drug is designed to target the PLP1 gene, which is implicated in the condition.
ION356 targets PLP1, the gene associated with Pelizaeus-Merzbacher Disease. By modulating PLP1, the drug aims to address the underlying genetic cause of the disease. It is administered intrathecally to deliver the therapy directly to the central nervous system.
ION356 is being developed by Ionis Pharmaceuticals, Inc., a biopharmaceutical company traded on NASDAQ under the ticker IONS. The company is conducting a Phase 1 clinical trial to evaluate the drug's safety, pharmacokinetics, and pharmacodynamics in participants with Pelizaeus-Merzbacher Disease.
ION356 is in Phase 1 clinical development. It is an investigational drug and has not been approved by regulatory authorities. The ongoing Phase 1 trial, known as the Orbit Study, is currently recruiting participants with Pelizaeus-Merzbacher Disease.
ION356 is being evaluated in the Orbit Study (NCT06150716), a Phase 1 trial assessing the safety, pharmacokinetics, and pharmacodynamics of intrathecally administered ION356 in participants with Pelizaeus-Merzbacher Disease. The trial is recruiting 24 male participants aged 2 years and older across the United States, France, Germany, Japan, and the Netherlands.