NIO752 / Novartis - LARVOL DELTA

Exploratory Results From a Phase 1 Study of NIO752 in Progressive Supranuclear Palsy Supporting Further Clinical Studies (MDS Congress 2026) - No abstract available

Clinical • P1 data • CNS Disorders • Movement Disorders • Progressive Supranuclear Palsy

Novartis Enrolled First Participant in Phase 3 PRESERVE Clinical Trial for Experimental PSP-RS Treatment (AFTD) - "The experimental drug NIO752 is designed to reduce levels of tau in the hopes of slowing or stopping the progression of PSP."

Enrollment open • CNS Disorders • Progressive Supranuclear Palsy

NIO752, a tau-targeting antisense oligonucleotide, in progressive supranuclear palsy: Phase I results and phase III design (EAN 2026) - P1 | "NIO752 is the first MAPT ASO investigated as a potential PSP treatment. Phase I results support proceeding to Phase III to establish the potential of NIO752 as a disease-modifying therapy in PSP-RS."

First-in-human • P3 data • CNS Disorders • Movement Disorders • Progressive Supranuclear Palsy • MAPT

NIO752 Silk: Participant Experience After A Phase 1b Randomised, Double-blinded, Placebo-controlled, Multicentre Trial (AAIC 2026) - No abstract available

Clinical • P1 data

CNIO752A12301: A Phase III, randomized, placebo-controlled, parallel group, double-blind study to evaluate the efficacy and safety of NIO752 in participants with Progressive Supranuclear Palsy followed by an Open Label Extension (clinicaltrialsregister.eu) - P2/3 | N=138 | Not yet recruiting | Sponsor: Novartis Pharma AG

New P2/3 trial • CNS Disorders • Movement Disorders • Progressive Supranuclear Palsy

Study of Safety, Tolerability, Pharmacodynamics and Pharmacokinetics of NIO752 in Early Alzheimer's Disease Participants (clinicaltrials.gov) - P1 | N=38 | Active, not recruiting | Sponsor: Novartis Pharmaceuticals | Recruiting ➔ Active, not recruiting | Trial completion date: Jan 2028 ➔ Sep 2027

Enrollment closed • Trial completion date • Alzheimer's Disease • CNS Disorders • Cognitive Disorders • APP • Plasma T-Tau

CNIO752A12301: A Phase III, randomized, placebo-controlled, parallel group, double-blind study to evaluate the efficacy and safety of NIO752 in participants with Progressive Supranuclear Palsy followed by an Open Label Extension (clinicaltrialsregister.eu) - P2/3 | N=138 | Not yet recruiting | Sponsor: Novartis Pharma AG | N=76 ➔ 138

Enrollment change • CNS Disorders • Movement Disorders • Progressive Supranuclear Palsy

A Study to Evaluate the Efficacy of NIO752 in Participants With Progressive Supranuclear Palsy (clinicaltrials.gov) - P3 | N=300 | Recruiting | Sponsor: Novartis Pharmaceuticals | Not yet recruiting ➔ Recruiting

Enrollment open • CNS Disorders • Movement Disorders • Progressive Supranuclear Palsy

CNIO752A12301: A Phase III, randomized, placebo-controlled, parallel group, double-blind study to evaluate the efficacy and safety of NIO752 in participants with Progressive Supranuclear Palsy followed by an Open Label Extension (clinicaltrialsregister.eu) - P2/3 | N=76 | Not yet recruiting | Sponsor: Novartis Pharma AG

New P2/3 trial • CNS Disorders • Movement Disorders • Progressive Supranuclear Palsy

A Study to Evaluate the Efficacy of NIO752 in Participants With Progressive Supranuclear Palsy (clinicaltrials.gov) - P3 | N=300 | Not yet recruiting | Sponsor: Novartis Pharmaceuticals

New P3 trial • CNS Disorders • Movement Disorders • Progressive Supranuclear Palsy

NIO752 IN PROGRESSIVE SUPRANUCLEAR PALSY (PSP): RESULTS OF A PHASE I, RANDOMIZED, PLACEBO-CONTROLLED STUDY (ADPD 2026) - P1 | "NIO752 is the first MAPT ASO studied in patients with PSP. These results support further clinical development of NIO752 in this debilitating disorder."

Clinical • First-in-human • CNS Disorders • Movement Disorders • Progressive Supranuclear Palsy • CSF T-tau • MAPT • NEFL

Quantifying tau knockdown in individuals with Alzheimer's Disease receiving intrathecal tau MAPT antisense oligonucleotide NIO752 using Stable Isotope Labelling Kinetics (SILK) – interim analysis of a phase 1B randomised, double-blinded, placebo-controlled, multicentre trial (CTAD 2025) - No abstract available

Clinical • P1 data • Alzheimer's Disease • CNS Disorders • MAPT

Study of Safety, Tolerability, Pharmacodynamics and Pharmacokinetics of NIO752 in Early Alzheimer's Disease Participants (clinicaltrials.gov) - P1 | N=36 | Recruiting | Sponsor: Novartis Pharmaceuticals | Trial primary completion date: Nov 2027 ➔ Sep 2026

Trial primary completion date • Alzheimer's Disease • CNS Disorders • Cognitive Disorders • APP • Plasma T-Tau

NIO-SILK: A Trial Evaluating the Effect of NIO752 on Tau Synthesis Measured by a Process Known as SILK (clinicaltrials.gov) - P1 | N=10 | Recruiting | Sponsor: University College, London | Not yet recruiting ➔ Recruiting | Trial completion date: Aug 2025 ➔ Jul 2026 | Trial primary completion date: Feb 2025 ➔ Jul 2026

Enrollment open • Trial completion date • Trial primary completion date • Alzheimer's Disease • CNS Disorders

Study of Safety, Tolerability, Pharmacodynamics and Pharmacokinetics of NIO752 in Early Alzheimer's Disease Participants (clinicaltrials.gov) - P1 | N=36 | Recruiting | Sponsor: Novartis Pharmaceuticals | Active, not recruiting ➔ Recruiting | N=25 ➔ 36 | Trial completion date: Oct 2025 ➔ Nov 2027 | Trial primary completion date: Oct 2025 ➔ Nov 2027

Enrollment change • Enrollment open • Trial completion date • Trial primary completion date • Alzheimer's Disease • CNS Disorders • Cognitive Disorders • APP • Plasma T-Tau

Safety, Tolerability and Pharmacokinetics of Multiple Ascending Doses of NIO752 in Progressive Supranuclear Palsy (clinicaltrials.gov) - P1 | N=59 | Completed | Sponsor: Novartis Pharmaceuticals | Active, not recruiting ➔ Completed

Trial completion • CNS Disorders • Movement Disorders • Progressive Supranuclear Palsy • COMT

NIO-SILK: A Trial Evaluating the Effect of NIO752 on Tau Synthesis Measured by a Process Known as SILK (clinicaltrials.gov) - P1 | N=10 | Not yet recruiting | Sponsor: University College, London

New P1 trial • Alzheimer's Disease • CNS Disorders

Study of Safety, Tolerability, Pharmacodynamics and Pharmacokinetics of NIO752 in Early Alzheimer's Disease Participants (clinicaltrials.gov) - P1 | N=25 | Active, not recruiting | Sponsor: Novartis Pharmaceuticals | Recruiting ➔ Active, not recruiting | Trial completion date: Oct 2024 ➔ Oct 2025 | Trial primary completion date: Oct 2024 ➔ Oct 2025

Enrollment closed • Trial completion date • Trial primary completion date • Alzheimer's Disease • CNS Disorders • Cognitive Disorders • APP

Safety, Tolerability and Pharmacokinetics of Multiple Ascending Doses of NIO752 in Progressive Supranuclear Palsy (clinicaltrials.gov) - P1 | N=59 | Active, not recruiting | Sponsor: Novartis Pharmaceuticals | Recruiting ➔ Active, not recruiting

Enrollment closed • CNS Disorders • Movement Disorders • Progressive Supranuclear Palsy • COMT

Safety, Tolerability and Pharmacokinetics of Multiple Ascending Doses of NIO752 in Progressive Supranuclear Palsy (clinicaltrials.gov) - P1 | N=66 | Recruiting | Sponsor: Novartis Pharmaceuticals | Trial completion date: Aug 2024 ➔ Nov 2024 | Trial primary completion date: Aug 2024 ➔ Nov 2024

Trial completion date • Trial primary completion date • CNS Disorders • Movement Disorders • Progressive Supranuclear Palsy • COMT

Study of Safety, Tolerability, Pharmacodynamics and Pharmacokinetics of NIO752 in Early Alzheimer's Disease Participants (clinicaltrials.gov) - P1 | N=24 | Recruiting | Sponsor: Novartis Pharmaceuticals | Not yet recruiting ➔ Recruiting | Trial completion date: Nov 2023 ➔ Oct 2024 | Initiation date: Sep 2022 ➔ Feb 2023 | Trial primary completion date: Nov 2023 ➔ Oct 2024

Enrollment open • Trial completion date • Trial initiation date • Trial primary completion date • Alzheimer's Disease • CNS Disorders • Cognitive Disorders • APP

Safety, Tolerability and Pharmacokinetics of Multiple Ascending Doses of NIO752 in Progressive Supranuclear Palsy (clinicaltrials.gov) - P1 | N=66 | Recruiting | Sponsor: Novartis Pharmaceuticals | Trial completion date: May 2024 ➔ Oct 2024 | Trial primary completion date: Oct 2023 ➔ Oct 2024

Trial completion date • Trial primary completion date • CNS Disorders • Movement Disorders • Progressive Supranuclear Palsy

DEVELOPMENT OF NIO752, AN INTRATHECALLY ADMINISTERED MICROTUBULE ASSOCIATED PROTEIN TAU (MAPT) ANTISENSE OLIGONUCLEOTIDE IN PROGRESSIVE SUPRANUCLEAR PALSY AND ALZHEIMER DISEASE (ADPD 2023) - "The preclinical data s upports clinical development of NIO752 in PSP and AD."

Alzheimer's Disease • CNS Disorders • Movement Disorders • Progressive Supranuclear Palsy • MAPT

Study of Safety, Tolerability, Pharmacodynamics and Pharmacokinetics of NIO752 in Early Alzheimer's Disease Participants (clinicaltrials.gov) - P1 | N=24 | Not yet recruiting | Sponsor: Novartis Pharmaceuticals

New P1 trial • Alzheimer's Disease • CNS Disorders • Cognitive Disorders • APP

Safety, Tolerability and Pharmacokinetics of Multiple Ascending Doses of NIO752 in Progressive Supranuclear Palsy (clinicaltrials.gov) - P1 | N=66 | Recruiting | Sponsor: Novartis Pharmaceuticals | Trial completion date: Jan 2025 ➔ May 2024

Trial completion date • CNS Disorders • Movement Disorders • Progressive Supranuclear Palsy