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ION440

Phase 1

Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome | Small molecule | Other |Ionis Pharmaceuticals, Inc.|Last Updated: Mar 24, 2026

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
RandomizedDouble-BlindSHAM_CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment48
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT06430385ATTUNE: 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)PHASE1 RECRUITING 48 — — Oct 21, 2024Apr 1, 2030Mar 24, 202611 United States, Austria +2
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Study Endpoints
Primary Endpoints
Part 1: Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
Up to approximately 36 weeks
Part 1: Number of Participants With Clinically Significant Change From Baseline in Vital Signs
Baseline up to approximately 36 weeks
Part 1: Number of Participants With Clinically Significant Change From Baseline in Physical and Neurological Examination Findings
Baseline up to approximately 36 weeks
Part 1: Number of Participants With Clinically Significant Change from Baseline in Laboratory Assessments
Baseline up to approximately 36 weeks
Part 1: Number of Participants With Clinically Significant Change From Baseline in Electrocardiogram (ECG)
Baseline up to approximately 36 weeks
Part 2: Number of Participants With TEAEs
Up to approximately 192 weeks
Part 2: Number of Participants With Clinically Significant Change From Baseline in Vital Signs
Baseline up to approximately 192 weeks
Part 2: Number of Participants With Clinically Significant Change From Baseline in Physical and Neurological Examination Findings
Baseline up to approximately 192 weeks
Part 2: Number of Participants With Clinically Significant Change from Baseline in Laboratory Assessments
Baseline up to approximately 192 weeks
Part 2: Number of Participants With Clinically Significant Change From Baseline in ECG
Baseline up to approximately 192 weeks
Secondary Endpoints
Part 1: Maximum Observed Concentration (Cmax) of ION440 in Plasma
Pre-dose and at multiple points post-dose up to Week 36
Part 1: Area Under the Concentration-time Curve (AUC) of ION440 in Plasma
Pre-dose and at multiple points post-dose up to Week 36
Part 1: Plasma Terminal Elimination Half-life (t½) of ION440
Pre-dose and at multiple points post-dose up to Week 36
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelSEQUENTIAL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Cohort 1: ION440 Dose AEXPERIMENTALParticipants will receive ION440 intrathecally at Dose A during Part 1/MAD, followed by ION440 Dose A during Part 2/LTE.
Cohort 2: ION440 Dose BEXPERIMENTALParticipants will receive ION440 intrathecally at Dose B during Part 1/MAD, followed by ION440 Dose B during Part 2/LTE.
Cohort 3: ION440 Dose CEXPERIMENTALParticipants will receive ION440 intrathecally at Dose C during Part 1/MAD, followed by ION440 Dose C during Part 2/LTE.
Sham ProcedureSHAM_COMPARATORDuring 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).
Interventions
NameTypeDescription
ION440DRUGION440 will be administered by intrathecal bolus (ITB) injection.
Sham procedurePROCEDUREAn LP will be performed with CSF collection but will not be followed by the administration of study treatment by ITB injection.
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Eligibility Criteria
Age Range2 Years — 65 Years
SexMALE
Healthy VolunteersNo
Study Sites11

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...

Countries:United StatesAustriaFranceSpain
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT06430385primaryCompletionDate: changed
LOWMay 24, 2026NCT06430385studyFirstPostDate: changed