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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 being developed for the treatment of Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome, also known as MDS. This is a rare neurodevelopmental disorder caused by extra copies of the MECP2 gene. ION440 is an investigational therapy and has not been approved for any indication.
ION440 targets MECP2, the gene and protein product that is duplicated in MECP2 Duplication Syndrome. By targeting MECP2, the drug aims to address the underlying genetic cause of the condition. ION440 is a small molecule designed to modulate MECP2 expression.
ION440 is being developed by Ionis Pharmaceuticals, Inc., which trades on the Nasdaq under the ticker symbol IONS. Ionis is a biopharmaceutical company focused on RNA-targeted and other novel therapies for rare and serious diseases.
ION440 is in Phase 1 clinical development. The only trial to date is a Phase 1 study that is currently recruiting participants. ION440 is investigational and has not received FDA approval.
ION440 is being evaluated in a single Phase 1 trial, NCT06430385, also called the ATTUNE study. This is a randomized, double-blind, sham-controlled trial testing intrathecally administered ION440 in participants with MECP2 Duplication Syndrome. It plans to enroll 48 male participants aged 2 years and older across the United States, Austria, Canada, France, and Spain.