rugonersen (RG6091) / Roche, Oak Hill Bio - LARVOL DELTA

Comparison of Angelman Syndrome Candidate Therapeutics Using a Novel Humanized Ube3a-ATS Mouse Model Highlights Major Differences in Efficacy, Tolerability, and Biodistribution (ASGCT 2025) - "Similarly, ASOs in clinical trial including RO7248824 (Roche), 1273062 (Ionis), and 4.4.PS.L (Ultragenyx) significantly upregulated patUbe3a in primary cortical neurons prepared from hsATS mice but not wild-type littermates...Overall, these studies highlight the power of the humanized Ube3a-ATS model for preclinical studies of potential Angelman syndrome therapeutics. Disease Focus of Abstract:Central Nervous System Disorders"

Preclinical • CNS Disorders • Developmental Disorders • Psychiatry • CLSPN, • SNHG14 • UBE3A

Oak Hill Bio Enters into Exclusive License Agreement with Roche to Obtain Global Rights for a Phase 3-Ready, Potential Best-in-Class Treatment for Individuals with Angelman Syndrome (Businesswire) - "Oak Hill Bio...has entered into an exclusive license agreement with Roche...to obtain global rights for rugonersen (RO7248824). Rugonersen is an ASO designed as a potential best-in-class treatment for individuals with Angelman syndrome, a rare, genetic neurodevelopmental condition for which there is no approved disease-modifying treatment. Oak Hill plans to initiate a Phase 3 study in early 2026."

Commercial • New P3 trial • Genetic Disorders • Rare Diseases

A Study To Investigate The Safety, Tolerability, Pharmacokinetics And Pharmacodynamics Of RO7248824 In Participants With Angelman Syndrome (clinicaltrials.gov) - P1 | N=74 | Active, not recruiting | Sponsor: Hoffmann-La Roche | Trial completion date: Aug 2024 ➔ Jul 2025 | Trial primary completion date: Aug 2024 ➔ Jul 2025

Trial completion date • Trial primary completion date • UBE3A

A Study To Investigate The Safety, Tolerability, Pharmacokinetics And Pharmacodynamics Of RO7248824 In Participants With Angelman Syndrome (clinicaltrials.gov) - P1 | N=74 | Active, not recruiting | Sponsor: Hoffmann-La Roche | Recruiting ➔ Active, not recruiting

Enrollment closed • UBE3A

A Study To Investigate The Safety, Tolerability, Pharmacokinetics And Pharmacodynamics Of RO7248824 In Participants With Angelman Syndrome (clinicaltrials.gov) - P1 | N=74 | Recruiting | Sponsor: Hoffmann-La Roche | Active, not recruiting ➔ Recruiting

Enrollment open • UBE3A

"@Roche ends research on rugonersen in Angelman syndrome https://t.co/usxRjHavhM" (@ThePharmaLetter)

A Study To Investigate The Safety, Tolerability, Pharmacokinetics And Pharmacodynamics Of RO7248824 In Participants With Angelman Syndrome (clinicaltrials.gov) - P1 | N=74 | Active, not recruiting | Sponsor: Hoffmann-La Roche | Recruiting ➔ Active, not recruiting | Trial completion date: Dec 2023 ➔ Aug 2024 | Trial primary completion date: Dec 2023 ➔ Aug 2024

Enrollment closed • Trial completion date • Trial primary completion date • UBE3A

A Study To Assess Distribution Of RO7248824 In The Central Nervous System Following Single Intrathecal Doses Of [89zr] Labeled RO7248824 In Healthy Male Participants (clinicaltrials.gov) - P1 | N=21 | Completed | Sponsor: Hoffmann-La Roche | Active, not recruiting ➔ Completed

Trial completion

A Study To Assess Distribution Of RO7248824 In The Central Nervous System Following Single Intrathecal Doses Of [89zr] Labeled RO7248824 In Healthy Male Participants (clinicaltrials.gov) - P1 | N=26 | Active, not recruiting | Sponsor: Hoffmann-La Roche | Recruiting ➔ Active, not recruiting

Enrollment closed

A Study To Assess Distribution Of RO7248824 In The Central Nervous System Following Single Intrathecal Doses Of [89zr] Labeled RO7248824 In Healthy Male Participants (clinicaltrials.gov) - P1 | N=26 | Recruiting | Sponsor: Hoffmann-La Roche | Trial completion date: Mar 2022 ➔ Jul 2022 | Trial primary completion date: Mar 2022 ➔ Jul 2022

Trial completion date • Trial primary completion date

A Study To Investigate The Safety, Tolerability, Pharmacokinetics And Pharmacodynamics Of RO7248824 In Participants With Angelman Syndrome (clinicaltrials.gov) - P1 | N=74 | Recruiting | Sponsor: Hoffmann-La Roche | N=140 ➔ 74 | Trial completion date: Sep 2024 ➔ Dec 2023 | Trial primary completion date: Sep 2024 ➔ Dec 2023

Enrollment change • Trial completion date • Trial primary completion date

A Study To Investigate The Safety, Tolerability, Pharmacokinetics And Pharmacodynamics Of RO7248824 In Participants With Angelman Syndrome (clinicaltrials.gov) - P1 | N=140 | Recruiting | Sponsor: Hoffmann-La Roche | N=66 ➔ 140 | Trial completion date: Sep 2022 ➔ Sep 2024 | Trial primary completion date: Sep 2022 ➔ Sep 2024

Enrollment change • Trial completion date • Trial primary completion date

A Study To Assess Distribution Of RO7248824 In The Central Nervous System Following Single Intrathecal Doses Of [89zr] Labeled RO7248824 In Healthy Male Participants (clinicaltrials.gov) - P1; N=26; Recruiting; Sponsor: Hoffmann-La Roche; Trial primary completion date: Nov 2021 ➔ Mar 2022

Clinical • Trial primary completion date

A Study To Investigate The Safety, Tolerability, Pharmacokinetics And Pharmacodynamics Of RO7248824 In Participants With Angelman Syndrome (clinicaltrials.gov) - P1; N=66; Recruiting; Sponsor: Hoffmann-La Roche; Trial completion date: Dec 2022 ➔ Sep 2022; Trial primary completion date: Dec 2022 ➔ Sep 2022

Clinical • Trial completion date • Trial primary completion date

A Study To Assess Distribution Of RO7248824 In The Central Nervous System Following Single Intrathecal Doses Of [89zr] Labeled RO7248824 In Healthy Male Participants (clinicaltrials.gov) - P1; N=26; Recruiting; Sponsor: Hoffmann-La Roche

Clinical • New P1 trial

A Study To Investigate The Safety, Tolerability, Pharmacokinetics And Pharmacodynamics Of RO7248824 In Participants With Angelman Syndrome (clinicaltrials.gov) - P1; N=66; Recruiting; Sponsor: Hoffmann-La Roche; Not yet recruiting ➔ Recruiting

Clinical • Enrollment open