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ION356

Phase 1

Pelizaeus-Merzbacher Disease | Small molecule | Other |Ionis Pharmaceuticals, Inc.|Last Updated: Dec 12, 2025

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
RandomizedCONTROLLEDBiomarker
Total Trials1
Total Enrollment24
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT06150716Orbit Study: A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered ION356 in Participants With Pelizaeus Merzbacher Disease (PMD)PHASE1 RECRUITING 24 — — Apr 10, 2024Jun 1, 2028Dec 12, 20257 United States, France +3
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Study Endpoints
Primary Endpoints
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious TEAEs
Up to Week 145
Number of Participants With Clinically Significant Change from Baseline in Laboratory Assessments
Baseline up to Week 145
Number of Participants With Clinically Significant Change From Baseline in Neurological Examination Findings
Baseline up to Week 145
Number of Participants With Clinically Significant Change From Baseline in Vital Signs
Baseline up to Week 145
Number of Participants With Clinically Significant Change From Baseline in Electrocardiography (ECG)
Baseline up to Week 145
Number of Participants With Change From Baseline in Concomitant Medication Use
Baseline up to Week 145
Secondary Endpoints
Maximum Plasma Concentration (Cmax) of ION356
Pre-dose and at multiple points post-dose up to Week 145
Area Under the Concentration-time Curve (AUC) of ION356
Pre-dose and at multiple points post-dose up to Week 145
Plasma Terminal Elimination Half-life (t½) of ION356
Pre-dose and at multiple points post-dose up to Week 145
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Study Design & Arms
AllocationRANDOMIZED
MaskingNONE
ModelSEQUENTIAL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Cohort A: ION356 Dose AEXPERIMENTALParticipants will receive ION356 intrathecally at Dose A in the MAD Period, followed by ION356 Dose A in the LTE Period.
Cohort B: ION356 Dose BEXPERIMENTALParticipants will receive ION356 intrathecally at Dose B in the MAD Period, followed by ION356 Dose B in the LTE Period.
Interventions
NameTypeDescription
ION356DRUGAdministered as intrathecal (IT) injection.
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Eligibility Criteria
Age Range2 Years — 17 Years
SexMALE
Healthy VolunteersNo
Study Sites7

Key Inclusion Criteria 1. Participant's parent or legally accepted representative can provide informed consent, attend all scheduled study visits, provide feedback regarding the participant's symptoms, and can comply with all study requirements. 2. Diagnosis of PMD with genetic confirmation of PLP1...

Countries:United StatesFranceGermanyJapanNetherlands
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT06150716primaryCompletionDate: changed
LOWMay 24, 2026NCT06150716studyFirstPostDate: changed