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ALN-SNCA

Phase 1

Parkinsons Disease | Small molecule | Neurology |Regeneron Pharmaceuticals, Inc.|Last Updated: Apr 14, 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-BlindPLACEBO_CONTROLLED
Total Trials1
Total Enrollment46
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT07216066First-in-Human Study of ALN-SNCA in Adult Participants With Early Parkinson's Disease (PD)PHASE1 RECRUITING 46Dec 5, 2025Jun 7, 2029Apr 14, 20262 Canada, Netherlands
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Study Endpoints
Primary Endpoints
Occurrence of Treatment-Emergent Adverse Events (TEAEs)
Through Week 80
Severity of TEAEs
Through Week 80
Secondary Endpoints
Concentration of alpha (a)-synuclein protein in CSF
Through Week 48
Change from baseline in concentration of a-synuclein protein in CSF
Through Week 48
Concentration of ALN-SNCA in plasma
Through Week 48
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelSEQUENTIAL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
ALN-SNCA Dose EscalationEXPERIMENTAL -
Matching PlaceboPLACEBO_COMPARATOR -
Interventions
NameTypeDescription
ALN-SNCADRUGAdministered per the protocol
PlaceboDRUGAdministered per the protocol
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Eligibility Criteria
Age Range50 Years — 80 Years
SexALL
Healthy VolunteersNo
Study Sites2

Key Inclusion Criteria: 1. Diagnosis of Parkinson's disease according to the Movement Disorder Society (MDS) criteria, as assessed by the investigator, with bradykinesia plus at least one of the other cardinal signs of Parkinson's disease (resting tremor, rigidity), without any other known or suspe...

Countries:CanadaNetherlands
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT07216066primaryCompletionDate: changed
LOWMay 24, 2026NCT07216066studyFirstPostDate: changed