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• W2891571710 abstract "Spinal muscular atrophy (SMA) is characterized by motor neuron loss and muscle atrophy, due to reduced levels of survival of motor neuron (SMN) protein from loss of function of the SMN1 gene. While SMN1 produces full-length SMN protein, a second gene, SMN2, produces only low levels of functional SMN protein. RG7916 (RO7034067) is an investigational, orally administered, centrally and peripherally distributed small molecule that modulates SMN2 pre-mRNA splicing towards the production of full-length SMN2 mRNA, resulting in an increase of SMN protein. SUNFISH (NCT02908685) is an ongoing multicenter, double-blind, placebo-controlled, seamless study of RG7916 (randomized 2:1, RG7916:placebo) in patients aged 2-25 years with Type 2 or 3 SMA. Part 1 (n=51) is principally assessing safety, tolerability, pharmacokinetics and pharmacodynamics of different RG7916 dose levels. Pivotal Part 2 (n=168) assesses safety and efficacy of the RG7916 dose level selected based on the results from Part 1. An interim analysis of SUNFISH Part 1 showed that RG7916 resulted in a dose-dependent increase in SMN protein levels in whole blood up to a median of 2.5-fold. To date, adverse events have been mostly mild, resolved despite ongoing treatment and were reflective of the underlying disease. There have been no drug-related safety findings leading to withdrawal. Effects on exploratory clinical outcome measures, biomarker results, and a recent update of safety data from SUNFISH Part 1 patients will be presented. Based on the differences in SMN protein levels between SMA severity types, an up to 2.5-fold increase in SMN protein is expected to lead to clinical efficacy. The clinical benefit of RG7916 is being assessed in SUNFISH Part 2, which is ongoing." @default.
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• W2891571710 date "2018-10-01" @default.
• W2891571710 modified "2023-09-27" @default.
• W2891571710 title "SMA THERAPIES II AND BIOMARKERS" @default.
• W2891571710 doi "https://doi.org/10.1016/j.nmd.2018.06.303" @default.
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