Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as NIO752
antisense oligonucleotide · 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...
It is an investigational RNA therapy from Novartis AG (ticker NVS) being studied for Progressive Supranuclear Palsy (PSP), a neurological disorder. It is an antisense oligonucleotide, a type of RNA therapy designed to act on genetic messages inside cells. It remains in clinical development and is not an approved treatment.
It is being studied as a treatment for Progressive Supranuclear Palsy (PSP), a neurological condition. The clinical program enrolled adults aged 40 years and older with PSP across the United States, Canada, Germany, and the United Kingdom. It is investigational and has not been approved for PSP or any other indication.
Novartis AG, which trades under the ticker NVS, is the developer of this antisense oligonucleotide for Progressive Supranuclear Palsy. The company sponsored the Phase 1 clinical program evaluating the drug in adults with PSP. Novartis is a large pharmaceutical company with operations in neurology and other therapeutic areas.
It is in Phase 1 development for Progressive Supranuclear Palsy. The single Phase 1 trial, NCT04539041, has been completed, and no trials are currently listed as active. The drug is investigational and has not received FDA approval for PSP or any other condition.
One Phase 1 trial has been conducted: NCT04539041, titled Safety, Tolerability and Pharmacokinetics of Multiple Ascending Doses of NIO752 in Progressive Supranuclear Palsy. It was a randomized, double-blind, placebo-controlled study that enrolled 59 participants across the United States, Canada, Germany, and the United Kingdom. The trial is completed.
Yes. NIO752 is the trial code name used for this antisense oligonucleotide in its Phase 1 study in Progressive Supranuclear Palsy. The completed trial, NCT04539041, evaluated multiple ascending doses of NIO752 in adults with PSP. Searches for NIO752 refer to the same investigational Novartis RNA therapy.