Biogen Advances Spinal Muscular Atrophy (SMA) Clinical Research with New Study, DEVOTE, Evaluating a Higher Dose of SPINRAZA and Additional Data in a Broad Range of Patients

Biogen today announced updates to the SPINRAZA (nusinersen) clinical development program including the initiation of a new global clinical trial, DEVOTE. The DEVOTE study will evaluate if a higher dose of SPINRAZA can provide even greater efficacy in the treatment of spinal muscular atrophy (SMA) across a broad patient population. In addition, new data further demonstrating the safety and efficacy of treatment with SPINRAZA in individuals with later-onset SMA will be featured in a podium presentation at the 13th Congress of the European Paediatric Neurology Society (EPNS) in Athens (September 17-21).

New study, DEVOTE, to evaluate if SPINRAZA can offer even greater efficacy in treating SMA

Building on the demonstrated long-term safety profile and proven efficacy of SPINRAZA in a broad range of patients, the DEVOTE trial will examine the potential for even greater efficacy, as well as the safety and tolerability of SPINRAZA, when administered at a higher dose. The trial is a Phase 2/3 randomized, controlled dose-escalating study that will be conducted at 50 sites around the world with a projected enrollment of 126 individuals with SMA of all ages, including adults.

The three-part trial will include an open-label safety evaluation and a pivotal, double-blind, active control randomized treatment period followed by an open-label treatment period. After the safety evaluation, the trial will compare two loading doses of 50 milligrams (mg) 15 days apart followed by a maintenance dose of 28 mg every four months with the current U.S. Food and Drug Administration-approved administration of SPINRAZA, which is four loading doses with 12 mg maintenance doses every four months. The third part of the trial will be an open-label evaluation to determine how to safely and efficiently transition patients from the currently approved dose of SPINRAZA to the higher dose being tested in the study.

More information on the trial (NCT04089566) is available at

Data to be presented at EPNS demonstrate improvements or stabilization in motor function following longer-term treatment

An integrated analysis from SHINE (NCT02594124), an open-label extension study for patients with SMA who participated in prior SPINRAZA studies, found that children with later-onset SMA (Type 2 or Type 3) experienced improvements or stabilization in one or more measures of motor function for up to nearly six years, in contrast to the expected decline observed in natural history cohorts. SHINE is following 24 patients aged 2-15 at treatment initiation (SMA Type 2; n=10 and Type 3; n=14) who transitioned from the CS2/CS12 studies, which previously showed that individuals with later-onset SMA who were treated with SPINRAZA demonstrated improvements in motor function and disease stabilization over approximately three years, that were not observed in natural history cohorts.2  Patients in the study were between 7 and 21 years old at the last study visit.

Motor function measures in this analysis of the SHINE study included the Hammersmith Functional Motor Scale–Expanded (HFMSE), Upper Limb Module (ULM), and Six-Minute Walk Test (6MWT). No participants discontinued treatment due to adverse events, and no new safety concerns were identified during the nearly six-year follow-up period.

Cure SMA Provides Seed Funding for Spinraza

From 2003 to 2006, Cure SMA provided the very first research funding needed to begin investigation into the therapeutic approach behind SPINRAZA. We would like to thank and acknowledge Cold Spring Harbor Laboratory (CSHL) and the University of Massachusetts Medical School for generating critical intellectual property for the program that was licensed to Ionis Pharmaceuticals. We specifically thank Drs. Adrian Krainer, Yimin Hua and colleagues at CSHL for years of dedication to and hard work on the preclinical development of Spinraza for SMA, and Drs. Ravindra Singh and Elliot Androphy for their work funded by Cure SMA in originally identifying the ISSN1 gene sequence, which is the sequence targeted in Spinraza.

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