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ION440 · 1 trial · 1 indication
| Arm | Type | Description |
|---|---|---|
| Cohort 1: ION440 Dose A | EXPERIMENTAL | Participants will receive ION440 intrathecally at Dose A during Part 1/MAD, followed by ION440 Dose A during Part 2/LTE. |
| Cohort 2: ION440 Dose B | EXPERIMENTAL | Participants will receive ION440 intrathecally at Dose B during Part 1/MAD, followed by ION440 Dose B during Part 2/LTE. |
| Cohort 3: ION440 Dose C | EXPERIMENTAL | Participants will receive ION440 intrathecally at Dose C during Part 1/MAD, followed by ION440 Dose C during Part 2/LTE. |
| Sham Procedure | SHAM_COMPARATOR | During the Part 1/MAD period, a lumbar procedure (LP) will be performed at the same frequency as ION440 administration. Participants will not receive ITB injections during this period. It will be followed by the open-label Part 2/LTE period, where participants will receive ION440 at the same dose as their enrolled cohort (e.g. Dose A, Dose B or Dose C). |
| Name | Type | Description |
|---|---|---|
| ION440 | DRUG | ION440 will be administered by intrathecal bolus (ITB) injection. |
| Sham procedure | PROCEDURE | An LP will be performed with CSF collection but will not be followed by the administration of study treatment by ITB injection. |
Key Inclusion criteria for Part 1: 1. Males aged ≥ 2 to ≤ 65 years, depending on specific cohort and group, at the time of informed consent. 1. Group A: ≥ 8 to ≤ 65 years old 2. Group B: 2 to 7 years old, inclusive 2. Participant has at least one parent or caregiver ≥ 18 years old capable of...
ION440 is an investigational drug being developed for Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome, a rare genetic disorder. It is administered intrathecally and is currently in Phase 1 clinical development. The drug is not yet approved and remains under study for safety, tolerability, pharmacokinetics, and pharmacodynamics.
ION440 targets MECP2, the Methyl CpG Binding Protein 2 gene. By targeting MECP2, the drug aims to address the underlying cause of MECP2 Duplication Syndrome. The drug is a small molecule designed for intrathecal administration to reach the central nervous system.
ION440 is 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 in patients with MECP2 Duplication Syndrome.
ION440 is in Phase 1 clinical development. It is an investigational drug, not yet approved by regulatory authorities. The ongoing Phase 1 trial, NCT06430385, is recruiting participants to assess the drug's safety, tolerability, pharmacokinetics, and pharmacodynamics in patients with MECP2 Duplication Syndrome.
ION440 is being studied in a Phase 1 clinical trial with the identifier NCT06430385, titled ATTUNE. This randomized, double-blind, sham-controlled study is recruiting 48 male participants aged 2 years and older with MECP2 Duplication Syndrome. The trial is conducted in the United States, Austria, France, and Spain.