Novartis Provides Update on PRESERVE, a Phase 3 Clinical Trial to evaluate NIO752 in PSP

Novartis announced the initiation of a Phase 3 clinical trial, PRESERVE, to evaluate the efficacy and safety of the investigational drug NIO752 in individuals with progressive supranuclear palsy (PSP).

Information about the Phase 3 clinical study, PRESERVE, is now available on Clinical Trials.gov. Key information about the upcoming study, including study design and eligibility criteria, are now publicly available. Participants need to be 41-81 years old and have a probable/possible diagnosis of PSP Richardson syndrome, with symptom onset in the last five years. Participants need a reliable study partner — such as a spouse, sibling or care partner — to participate in study visits and assessments. Full inclusion criteria are available on ClinicalTrials.gov. Trail sites are being added to Clinical Trials.gov as they become activated.

CurePSP will partner closely with Novartis to support the rollout of the trial, and we will provide updates to our community as they become available later this spring.

"The initiation of a Phase 3 trial for PSP is a meaningful step for the field and a reflection of sustained investment in tau-targeted approaches,” says CurePSP Chief Executive Officer, Kristophe Diaz, PhD. “Antisense oligonucleotide therapies have shown real promise across neurodegenerative diseases, and it is encouraging to see this approach advancing in PSP with a therapy targeting tau. While there is still much work ahead, this study represents the kind of progress our community has been waiting for. CurePSP is proud to partner with Novartis to support the successful rollout of this trial and to help ensure that patients and families are informed and engaged as enrolment begins."

NIO752 is an antisense oligonucleotide (ASO), a type of therapeutic that alters protein production. NIO752 is designed to interfere with the translation of tau mRNA to tau protein, and therefore reduce the levels of tau. PSP is a primary tauopathy characterized by accumulation of misfolded tau, and the goal of testing NIO752 as a therapeutic for PSP is that reducing the amount of tau would slow the progression of tau pathology. NIO752 is administered through an intrathecal injection, an injection given through a thin needle placed in the lower back and into the fluid around the spinal cord.

A previous Phase 1 trial tested the safety and pharmacodynamics of NIO752 in people with PSP, and a trial testing NIO752 in Alzheimer’s disease is ongoing.  We encourage anyone who is interested to review the study information posted on ClinicalTrials.gov and to speak to their healthcare provider. If you have any questions about this information, you can also contact us through the email below. To receive future clinical trial updates, join CurePSP's research mailing list below.

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Questions about the trial can be sent to:

Novartis Pharmaceuticals, novartis.email@novartis.com