Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
ION717 · 1 trial · 1 indication
| Arm | Type | Description |
|---|---|---|
| ION717 + Placebo, Regimen 1 | EXPERIMENTAL | Participants will receive multiple doses of study drug (ION717 and placebo) during the 30-week treatment period; the order of doses is blinded. Participants will then receive multiple doses of ION717 during the 142-week open-label extension period. |
| ION717 + Placebo, Regimen 2 | EXPERIMENTAL | Participants will receive multiple doses of study drug (ION717 and placebo) during the 30-week treatment period; the order of doses is blinded. Participants will then receive multiple doses of ION717 during the 142-week open-label extension period. |
| ION717, Regimen 3 | EXPERIMENTAL | Participants will receive multiple doses of ION717 during the 30-week treatment period and the 142-week open-label extension period. |
| Name | Type | Description |
|---|---|---|
| ION717 | DRUG | ION717 will be administered by IT injection. |
| Placebo | DRUG | Placebo-matching ION717 will be administered by IT injection. |
Key Inclusion Criteria * A confirmed diagnosis of probable or definite prion disease. * Early-stage prion disease at the time of Screening. * Willing to meet all study requirements, including travel to Study Center, procedures, measurements and visits. * Patients must have a caregiver who is ≥ 18 y...
ION717 is an investigational drug being developed for the treatment of prion disease, a rare and fatal neurodegenerative condition. It is currently in Phase 1 clinical development and has not been approved by regulatory authorities.
ION717 targets the PRNP gene, which encodes the prion protein. By targeting this gene, the drug aims to reduce the production of the prion protein, which is central to the pathology of prion disease.
ION717 is being developed by Ionis Pharmaceuticals, Inc., a biopharmaceutical company. The company's stock is traded under the ticker symbol IONS on the NASDAQ exchange.
ION717 is in Phase 1 clinical development. It is an investigational drug, meaning it has not yet been approved for commercial use and is still undergoing clinical trials to assess its safety and efficacy.
ION717 is being studied in a Phase 1 clinical trial with the identifier NCT06153966. This trial, known as PrProfile, is assessing the safety, tolerability, pharmacokinetics, and pharmacodynamics of ION717 in patients with prion disease. The trial is active but not recruiting participants.
ION717 is the primary name for this investigational drug. No alternative names have been reported for this compound in the available information.