In short
NIO752 is an investigational nucleic acid-based substance being studied for progressive supranuclear palsy (PSP), a rare brain disorder. It has no marketing authorisation and is given by intrathecal injection. One Phase 3 trial with 287 participants is currently authorised to evaluate its efficacy and safety in PSP with Richardson syndrome.
Key points
- NIO752 is an investigational nucleic acid substance with no marketing authorisation.
- It is being studied in a Phase 3 trial for progressive supranuclear palsy with Richardson syndrome (PSP-RS).
- The Phase 3 trial plans to enrol 287 participants and is currently authorised.
- NIO752 is given by intrathecal injection (into the spine).
- The main measure is the change in the rPSPRS-10 score at week 72.
- An earlier study looked at safety, tolerability and pharmacokinetics in 64 participants.
What is NIO752?
NIO752 is an investigational nucleic acid-based substance being developed by Novartis Pharma AG. It has no marketing authorisation in the product records and is therefore not yet an approved medicine. It has received orphan designation, which means it is intended for a rare disease. The substance is also known by the development code NIO752.
How does NIO752 work?
The registry data classify NIO752 as a nucleic acid substance, meaning it is based on genetic material rather than a traditional chemical drug. The exact mechanism of action in progressive supranuclear palsy is not described in the available sources. The therapeutic area is nervous system diseases.
Which conditions is NIO752 being studied for?
- Progressive supranuclear palsy with Richardson syndrome (PSP-RS) — a rare brain disorder that affects movement, balance and eye movements. The Phase 3 trial focuses specifically on participants with this form of PSP.
How is NIO752 given?
NIO752 is prepared as a concentrate for solution for injection and is given by intrathecal use, meaning it is injected directly into the fluid around the spinal cord. An earlier study also described the treatment as an intrathecal injection into the spine.
What do the clinical trials show?
One Phase 3 trial is currently authorised. It is a randomised, placebo-controlled, parallel-group, double-blind study in 287 participants with progressive supranuclear palsy with Richardson syndrome. The trial compares NIO752 with a placebo (isotonic saline solution) given by intrathecal injection. The main outcome is the change from baseline at week 72 in the rPSPRS-10 score, a rating scale that measures the severity of PSP symptoms. After the double-blind phase, participants can continue into an open-label extension, where everyone receives NIO752.
An earlier Phase 1 study, described by the UK Health Research Authority, evaluated the safety, tolerability and pharmacokinetics (how the drug moves through the body) of multiple ascending doses of intrathecally administered NIO752 in people with PSP. That study planned to enrol approximately 64 participants aged 40 to 75 years across 5 countries, testing 6 different doses against placebo.
Who can take part in these trials?
The Phase 3 trial enrols participants with progressive supranuclear palsy with Richardson syndrome. The earlier Phase 1 study included participants with PSP aged 40 to 75 years inclusive. In that study, 3 out of 4 participants received NIO752 and 1 out of 4 received placebo, with neither participants nor study doctors knowing which treatment was given.
What side effects are known?
The available sources do not report specific side effects of NIO752. The Phase 3 trial is designed to evaluate safety, and the earlier Phase 1 study also assessed safety and tolerability, but no results on side effects are provided in the data.