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NIO752

Phase 1

Alzheimer Disease | Small molecule | Neurology |Novartis AG|Last Updated: Jun 1, 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_CONTROLLEDBiomarker
Total Trials1
Total Enrollment38
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT05469360Study of Safety, Tolerability, Pharmacodynamics and Pharmacokinetics of NIO752 in Early Alzheimer's Disease ParticipantsPHASE1 ACTIVE NOT_RECRUITING 38Feb 23, 2023Sep 24, 2027Jun 1, 202610 Finland, France +2
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Study Endpoints
Primary Endpoints
Change in cerebrospinal total tau from baseline to Day 85
Baseline, Day 85

Total tau protein levels in cerebrospinal fluid. More frequent timepoints might be added as deemed necessary per the site Investigator's judgment.

Secondary Endpoints
Number of adverse events and serious adverse events
Cohort 1 & 2: Baseline up to 170 days (placebo-controlled part) and up to 589 days (OLE part), Cohort 3: Baseline up to 169 days (placebo-controlled part) and up to 507 days (OLE part)
Concentration of NIO752 in cerebrospinal fluid (CSF)
Cohorts 1 & 2: Pre-dose, Days 57, 85, 170 (placebo-controlled part) and Days 252, 420, and 588 (OLE part), Cohort 3: Pre-dose, day 1, 57, 85, 169 (placebo-controlled part) and Days 388 and 506 (OLE part)
Cmax, Ctrough in plasma
Cohorts 1, 2 & 3: Pre-dose, 0.5, 1, 2, 3 4, 5, 6, 24 hours post first dose. Cohort 1 & 2: Days 14, 57, 85 and 170 (pbo-controlled part) and 252, 253, 420, 421, and 588 (OLE) Cohort 3: Days 14, 57, 85 and 169 (pbo-controlled part) and 338 and 506 (OLE)
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Study Design & Arms
AllocationRANDOMIZED
MaskingDOUBLE
ModelSEQUENTIAL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
NIO752 - Dose A - Cohort 1EXPERIMENTALA single intrathecal injection of Dose A
Matching placebo - Cohort 1PLACEBO_COMPARATORA single intrathecal injection of artificial cerebrospinal fluid (CSF)
NIO752 Dose B - Cohort 2EXPERIMENTALA single intrathecal injection of Dose B
Matching placebo - Cohort 2PLACEBO_COMPARATORA single intrathecal injection of artificial cerebrospinal fluid (CSF)
NIO752 OLE - Cohorts 1 and 2EXPERIMENTALMultiple intrathecal injections of Dose A
NIO752 - Dose C - Cohort 3EXPERIMENTALTwo intrathecal injections of Dose C
Matching Placebo - Cohort 3PLACEBO_COMPARATORTwo intrathecal injections of artificial cerebrospinal fluid (CSF)
NIO752 OLE - Cohort 3EXPERIMENTALSingle intrathecal injection of Dose C
Interventions
NameTypeDescription
NIO752DRUGA single intrathecal (cerebrospinal) injection of NIO752 of Dose A
Matching placeboDRUGA single intrathecal injection of matching placebo
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Eligibility Criteria
Age Range30 Years — 74 Years
SexALL
Healthy VolunteersNo
Study Sites10

Main Inclusion Criteria (placebo-controlled part): * Between 30 to 74 years old (both inclusive) at the time of informed consent. * A diagnosis of mild Alzheimer's Disease (AD) or mild cognitive impairment (MCI) due to AD at screening with at least a 6-month decline in cognitive function prior to s...

Countries:FinlandFranceSpainSweden
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Recent Changes (Last 90 Days)
MEDIUMJun 2, 2026NCT05469360Status: RECRUITING → ACTIVE_NOT_RECRUITING
MEDIUMJun 2, 2026NCT05469360Status: RECRUITING → ACTIVE_NOT_RECRUITING
MEDIUMJun 2, 2026NCT05469360Status: RECRUITING → ACTIVE_NOT_RECRUITING
LOWMay 26, 2026NCT05469360primaryCompletionDate: changed
LOWMay 24, 2026NCT05469360studyFirstPostDate: changed