Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as antisense oligonucleotide
NIO752 · 3 trials · 7 indications
Number of participants with TEAEs including SAEs, AESIs, and AEs leading to treatment interruption/discontinuation or dose reduction.
Total tau protein levels in cerebrospinal fluid. More frequent timepoints might be added as deemed necessary per the site Investigator's judgment.
Adverse events will be collected at clinical visits and other contacts. All abnormalities from safety assessments (physical exams and neurological exams and clinical safety labs) considered clinically significant will be recorded as adverse events
The Columbia-Suicide Severity Rating Scale (C-SSRS) is a questionnaire that prospectively assesses Suicidal Ideation and Suicidal Behavior. The C-SSRS must be administered at visits. If, at any time after "screening and/or baseline" version, the score is "yes" on item 4 or item 5 of the Suicidal Ideation section of the C-SSRS or "yes" on any item of the Suicidal Behavior section, the participant must be referred to a mental health care professional for further assessment and/or treatment.
CSF safety labs measure levels of proteins, glucose, lactate and white blood cell counts with differential indicating infections.
| Arm | Type | Description |
|---|---|---|
| NIO752 - Cohort 1 (PSP non-RS) | EXPERIMENTAL | NIO752 solution |
| Placebo - Cohort 1 (PSP non-RS) | PLACEBO_COMPARATOR | Placebo solution |
| NIO752 - Cohort 2 (CBS non-AD) | EXPERIMENTAL | NIO752 solution |
| Placebo - Cohort 2 (CBS non-AD) | PLACEBO_COMPARATOR | Placebo solution |
| NIO752 - Cohort 3 (MAPT-FTLD) | EXPERIMENTAL | NIO752 solution |
| Placebo - Cohort 3 (MAPT-FTLD) | PLACEBO_COMPARATOR | Placebo solution |
| NIO752 - Dose A - Cohort 1 | EXPERIMENTAL | A single intrathecal injection of Dose A |
| Matching placebo - Cohort 1 | PLACEBO_COMPARATOR | A single intrathecal injection of artificial cerebrospinal fluid (CSF) |
| NIO752 Dose B - Cohort 2 | EXPERIMENTAL | A single intrathecal injection of Dose B |
| Matching placebo - Cohort 2 | PLACEBO_COMPARATOR | A single intrathecal injection of artificial cerebrospinal fluid (CSF) |
| NIO752 OLE - Cohorts 1 and 2 | EXPERIMENTAL | Multiple intrathecal injections of Dose A |
| NIO752 - Dose C - Cohort 3 | EXPERIMENTAL | Two intrathecal injections of Dose C |
| Matching Placebo - Cohort 3 | PLACEBO_COMPARATOR | Two intrathecal injections of artificial cerebrospinal fluid (CSF) |
| NIO752 OLE - Cohort 3 | EXPERIMENTAL | Single intrathecal injection of Dose C |
| Cohort A NIO752 | EXPERIMENTAL | 4 injections of NIO752 at dose A |
| Cohort B NIO752 | EXPERIMENTAL | 4 injections of NIO752 at dose B |
| Placebo | PLACEBO_COMPARATOR | 4 injections of placebo |
| Cohort C NIO752 | EXPERIMENTAL | 4 injections of NIO752 at dose C |
| Cohort D NIO752 | EXPERIMENTAL | 4 injections of NIO752 at dose D |
| Cohort E NIO752 | EXPERIMENTAL | 4 injections of NIO752 at dose E |
| Name | Type | Description |
|---|---|---|
| NIO752 | DRUG | Antisense Oligonucleotide Solution |
| Placebo | DRUG | Placebo solution |
| Matching placebo | DRUG | A single intrathecal injection of matching placebo |
| antisense oligonucleotide | DRUG | solution of antisense oligonucleotide injected intrathecally (spine tap) at multiple dose levels |
Inclusion Criteria: Common across all cohorts: 1. Signed informed consent must be obtained prior to participation in the study. 2. Participants between the ages of ≥41 and ≤81 years. 3. Able to walk at least 10 steps with minimal assistance (stabilization of one arm or use of cane/walker). 4. Docu...
NIO752 is an investigational small molecule being developed by Novartis AG for neurological conditions, specifically Alzheimer disease and progressive supranuclear palsy. It is in clinical development and has not been approved by the FDA. Novartis is studying it in primary tauopathies, a group of disorders characterized by abnormal tau protein accumulation in the brain.
NIO752 is being studied for the treatment of Alzheimer disease and progressive supranuclear palsy, as well as other primary tauopathies including corticobasal degeneration and frontotemporal lobar degeneration. It is an investigational therapy and is not approved for any indication. Clinical trials are evaluating its safety, tolerability, and pharmacodynamic effects in these patient populations.
NIO752 is being developed by Novartis AG, which trades on the New York Stock Exchange under the ticker symbol NVS. Novartis is a multinational pharmaceutical company headquartered in Switzerland. The company is sponsoring clinical trials of NIO752 in Alzheimer disease and primary tauopathies including progressive supranuclear palsy.
NIO752 is in Phase 1 and Phase 2 clinical development. A Phase 1 trial in early Alzheimer disease has been completed for enrollment and is active but not recruiting. A Phase 2 trial in primary tauopathies, including progressive supranuclear palsy, is not yet recruiting. NIO752 has not been approved by the FDA.
NIO752 is being evaluated in two clinical trials. NCT05469360 is a Phase 1 study of safety, tolerability, pharmacodynamics, and pharmacokinetics in early Alzheimer disease participants, conducted in Finland, France, Spain, and Sweden. NCT07868328 is a Phase 2 study of safety, tolerability, and pharmacodynamics in primary tauopathies, including progressive supranuclear palsy, corticobasal degeneration, and frontotemporal lobar degeneration.