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Phase III studies of crenezumab in early (prodromal-to-mild) Alzheimer’s disease (CREAD/CREAD2): Biomarker results

Crenezumab is a humanized anti-amyloid-β (Aβ) monoclonal antibody evaluated in clinical trials in patients with sporadic Alzheimer’s disease (AD). Phase III studies CREAD and CREAD2 investigated the safety and efficacy of crenezumab in patients with early AD and included assessments of imaging and fluid biomarkers to better understand the effects of crenezumab on the underlying pathology of AD. This presentation discusses data from the CREAD and CREAD2 biomarker analyses.

Thirty-six–month amyloid PET results show continued reduction in amyloid burden with gantenerumab

Previous analyses of gantenterumab-treated patients from the SCarlet RoAD (SR) and Marguerite RoAD (MR) open-label extension (OLE) studies showed large mean amyloid reductions on positron emission tomography (PET) over 24 months, with some patients achieving reductions below the amyloid positivity threshold. Updated results at 36 months are presented.

Anchor- and Distribution-based methods to establish clinically meaningful score changes on the Clinical Dementia Rating Scale – Sum of Boxes in patients with prodromal Alzheimer’s Disease

Although change in Clinical Dementia Rating Scale – sum of boxes (CDR-SB) is often the primary endpoint of clinical trials in early AD, there is no clear consensus as to what constitutes a clinically meaningful change on this scale. Here we present findings from a secondary analysis of data from the Alzheimer’s Disease Cooperative Study ADC-008 phase III clinical trial of donepezil and vitamin E in patients with amnestic mild cognitive impairment. Anchor- and distribution-based approaches were used to establish a threshold of score changes on the CDR-SB associated with a clinically meaningful change/decline at the individual patient level.

Phase 1b/2 study of the anti-myostatin adnectin RG6206 in ambulatory boys with Duchenne muscular dystrophy: A 120-week treatment update

A 120-week update from the Phase 1b/2 study of the anti-myostatin adnectin RG6206 in ambulatory boys with Duchenne muscular dystrophy.

Use of Stride Velocity as a wearable-derived, secondary endpoint for real-world ambulation in SPITFIRE

ActiMyo is a wearable, medical device that was easy to use by physicians and patients. Roche is using ActiMyo to measure 95% stride velocity (SV95C) as a secondary digital endpoint in SPITFIRE, an ongoing Phase 2/3 study of RG6206 in ambulatory boys with DMD.

Prevalence of Huntington’s disease in the US

The prevalence of HD in the US is currently uncertain. This poster presents the results of a study which used published prevalence rates of HD to estimate the number of individuals with HD in the US

Reliability, feasibility and validity of a novel digital monitoring platform assessing cognitive and motor symptoms in people with Stage I and II Huntington’s disease (HD)

Longitudinal monitoring of HD signs and symptoms through frequent, home-based testing promises to enhance standard clinical testing methods that are potentially limited by natural symptom fluctuations, inter-rater variability and subjectivity. This poster presents preliminary adherence to demonstrate acceptance and feasibility of the approach in the Roche HD Natural History Study at baseline in patients with early manifest HD.

Preliminary results from an ongoing open-label extension (OLE) study investigating RG6042 huntingtin protein (HTT) antisense oligonucleotide (ASO) in adults with manifest Huntington’s disease (HD)

This poster presents a safety, tolerability and biomarker update on the ongoing open-label extension of the Phase I/IIa study of RG6042 in adults with early manifest HD. This open-label extension study is exploring the long-term safety, tolerability, pharmacokinetics and pharmacodynamics of 120 mg of RG6042 administered intrathecally, monthly or bi-monthly, for 15 months.

Efficacy of satralizumab (SA237) in subgroups of patients in SAkuraSky: a Phase III double-blind, placebo-controlled, add-on study in patients with neuromyelitis optica spectrum disorder (NMOSD)

Satralizumab in combination with baseline immunosuppressants significantly reduced the risk of protocol-defined relapse in patients with NMOSD, particularly those who were seropositive for antibodies against aquaporin-4 (AQP4-IgG).

Efficacy of satralizumab (SA237) in subgroups of patients in SAkuraSky: a Phase III double-blind, placebo-controlled, add-on study in patients with neuromyelitis optica spectrum disorder (NMOSD)

Satralizumab in combination with baseline immunosuppressants significantly reduced the risk of protocol-defined relapse in patients with NMOSD, particularly those who were seropositive for antibodies against aquaporin-4 (AQP4-IgG).

The Roche HD Natural History Study – an external comparator by design

This poster describes the comparability of participants in the HD Natural History Study and ongoing open-label extension of the Phase I/IIa at baseline on key demographics and clinical features. The HD Natural History is a prospective, longitudinal cohort study designed to provide further clinical validation of mHTT protein as a prognostic biomarker of disease outcomes in early manifest HD. This study will be used as an external control group for the ongoing open-label extension of the Phase I/IIa to aid the interpretation of treatment effects of RG6042.

JEWELFISH: Safety and pharmacodynamic data in patients with spinal muscular atrophy (SMA) receiving treatment with risdiplam (RG7916) that have previously been treated with nusinersen

JEWELFISH (NCT03032172) is an ongoing, multicenter, open-label study to assess the safety, tolerability and PK/PD relationship of daily, oral risdiplam in adults, children and infants with SMA previously enrolled in Study BP29420 (MOONFISH) with the splicing modifier RO6885247 or previously treated with nusinersen (SPINRAZA®), olesoxime or onasemnogene abeparvovec-xioi (ZOLGENSMA®). This poster presentation provides an overview of the latest JEWELFISH study enrollment criteria and describes the safety and PK/PD of risdiplam in 12 patients.

RAINBOWFISH: A study of risdiplam (RG7916) in newborns with pre-symptomatic spinal muscular atrophy (SMA)

RAINBOWFISH (NCT03779334) is an open-label, single-arm, multicenter clinical study to investigate the efficacy, safety, PK and PD of risdiplam in infants with genetically diagnosed SMA who are not yet presenting with symptoms. This poster presentation provides an overview of the RAINBOWFISH study design and study objectives.

FIREFISH Part 1: 16-month safety and exploratory outcomes of risdiplam (RG7916) treatment in infants with Type 1 spinal muscular atrophy (SMA)

FIREFISH (NCT02913482) is an ongoing, multicenter, open-label study aiming to asssess the safety and efficacy of risdiplam (RG7916) in infants aged 1-7 months with type 1 SMA and two SMN2 gene copies. This poster presentation describes one-year motor milestone data in infants from FIREFISH Part 1