Approval Probability
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antisense oligonucleotide · 1 trial · 1 indication
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 |
|---|---|---|
| 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 |
|---|---|---|
| antisense oligonucleotide | DRUG | solution of antisense oligonucleotide injected intrathecally (spine tap) at multiple dose levels |
| placebo | DRUG | placebo for each dose level |
Inclusion Criteria: 1. Signed informed consent 2. Between 40 to 75 years old (inclusive) 3. Have PSP diagnosed for less than 5 years with a current classification of probable PSP Richardson syndrome, a progressive supranuclear palsy rating scale (PSPRS) score \< 40 and MOCA score \>17 at screening ...
NIO752 is an investigational antisense oligonucleotide being developed for Progressive Supranuclear Palsy (PSP), a rare neurological disorder. It is currently in Phase 1 clinical development and has not been approved by regulatory authorities.
NIO752 is an antisense oligonucleotide, a type of small molecule that is designed to target and modulate the production of specific proteins involved in the pathology of Progressive Supranuclear Palsy. The exact molecular target has not been disclosed.
NIO752 is being developed by Novartis AG (NYSE: NVS), a global pharmaceutical company. The drug is currently in Phase 1 clinical trials for Progressive Supranuclear Palsy.
NIO752 is in Phase 1 clinical development. A Phase 1 trial has been completed, and the drug remains investigational, meaning it has not yet received regulatory approval for any use.
NIO752 has been studied in one completed Phase 1 trial, NCT04539041, which assessed the safety, tolerability, and pharmacokinetics of multiple ascending doses in patients with Progressive Supranuclear Palsy. The trial enrolled 59 participants across the United States, Canada, Germany, and the United Kingdom.
NIO752 is an antisense oligonucleotide, which is a class of therapeutic molecules. The drug itself is referred to as NIO752 in clinical trials, and no other alternative names have been reported.