ION440 / Ionis - LARVOL DELTA

ATTUNE: A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intrathecally-Administered ION440 in Participants With Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome (MDS) (clinicaltrials.gov) - P1/2 | N=48 | Recruiting | Sponsor: Ionis Pharmaceuticals, Inc. | Trial primary completion date: May 2026 ➔ Sep 2027

Trial primary completion date • Myelodysplastic Syndrome

ATTUNE: A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intrathecally-Administered ION440 in Participants With Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome (MDS) (clinicaltrials.gov) - P1/2 | N=48 | Recruiting | Sponsor: Ionis Pharmaceuticals, Inc. | Not yet recruiting ➔ Recruiting

Enrollment open • Myelodysplastic Syndrome

ATTUNE: A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intrathecally-Administered ION440 in Participants With Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome (MDS) (clinicaltrials.gov) - P1/2 | N=48 | Not yet recruiting | Sponsor: Ionis Pharmaceuticals, Inc.

New P1/2 trial • Myelodysplastic Syndrome