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NIO752

Phase 1

Alzheimer Disease | Small molecule | Neurology |Novartis AG|Last Updated: Jun 1, 2026

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLED
Total Trials1
Total Enrollment38

FDA Designations

No designations recorded

Clinical trial landscape

NIO752 · 1 trial · 2 indications

Phase 1 1
NCT05469360Study of Safety, Tolerability, Pharmacodynamics and Pharmacokinetics of NIO752 in Early Alzheimer's Disease ParticipantsAlzheimer Disease
ACTIVE NOT_RECRUITING38 Analytics
PHASE1ACTIVE NOT_RECRUITING
Study of Safety, Tolerability, Pharmacodynamics and Pharmacokinetics of NIO752 in Early Alzheimer's Disease Participants
Alzheimer DiseaseUnlock trial analytics

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 to 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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Frequently asked questions about NIO752

What is NIO752 used for in Alzheimer Disease?

NIO752 is an investigational small molecule being studied for the treatment of early Alzheimer's Disease, including mild cognitive impairment. It is currently in Phase 1 clinical development and has not been approved by regulatory authorities.

What does NIO752 target?

The molecular target of NIO752 has not been disclosed. It is a small molecule being developed by Novartis for Alzheimer's Disease, but its specific mechanism of action is not publicly available.

Who makes NIO752?

NIO752 is being developed by Novartis AG, a global pharmaceutical company traded on the New York Stock Exchange under the ticker symbol NVS. The company is conducting a Phase 1 clinical trial to evaluate the drug in early Alzheimer's Disease.

What phase is NIO752 in?

NIO752 is in Phase 1 clinical development. It is an investigational drug and has not been approved by the FDA or any other regulatory agency. The ongoing Phase 1 trial is active but not recruiting participants.

What clinical trials is NIO752 in?

NIO752 is being studied in a single Phase 1 clinical trial with the identifier NCT05469360. This randomized, double-blind, placebo-controlled study is evaluating the safety, tolerability, pharmacodynamics, and pharmacokinetics of NIO752 in participants with early Alzheimer's Disease.

Is NIO752 the same as any other drug?

No alternative names for NIO752 have been disclosed. It is a distinct investigational small molecule being developed by Novartis for Alzheimer's Disease, and no other names or aliases are currently associated with it.