Biogen Presents New Data from its DEVOTE Study of Spinraza (nusinersen)

22 April 2021

Biogen presents new data from its DEVOTE study of SpinrazaTM (nusinersen) as well as an update on biomarkers and digital tools to enhance disease monitoring, at the 73rd American Academy of Neurology (AAN) Annual Meeting.

The AAN meeting is being held virtually between 17th and 22nd April 2021.
 

DEVOTE

DEVOTE is a three-part study designed to evaluate the safety, tolerability and potentially improved efficacy of nusinersen when administered at a higher dose than currently approved (12mg) for the treatment of SMA. The Phase 2/3 randomised, controlled, dose-escalating study is to be conducted at approximately 50 sites around the world and aims to enrol individuals of all ages with SMA.

The three parts consist of:

  • An open-label safety evaluation cohort (part A)
  • A pivotal, double-blind, active control randomised treatment cohort (part B) and
  • An open-label treatment cohort (part C).

These new data show that:

  1. The higher loading and maintenance dosing regimen of 28mg showed no new safety concerns in study participants who were followed for up to approximately five months (part A).
     
  2. No adverse events (AEs) reported were considered related to the higher dose study drug and there were no severe or serious AEs.

This emerging safety profile supports Biogen’s continued development of a higher dose of SPINRAZA, including ongoing enrolment of patients in the pivotal Part B of the study.

Part B will evaluate the higher-dose regimen (two loading doses of 50mg two weeks apart, followed by 28mg maintenance doses every four months) compared to the approved 12mg dose of SPINRAZA: four loading doses, followed by maintenance doses every four months.
 

Biomarkers and digital tools to enhance disease monitoring

Biogen is advancing research to evaluate biomarkers and digital tools that expand on traditional clinical assessments and incorporate more sensitive measures to help better predict and monitor the course of SMA.

New data in 75 patients from the CHERISH/SHINE studies of SPINRAZA build upon the body of evidence that neurofilament levels in blood warrant further evaluation as a biomarker for assessing treatment response in SMA.

Data show that higher neurofilament levels at baseline were, on average, associated with greater improvements in motor function scores among SPINRAZA-treated individuals with later-onset SMA.

The use of biomarkers could improve the understanding of disease mechanisms and interventions for SMA. Therefore, measuring neurofilament levels has been integrated as an exploratory endpoint in the DEVOTE and RESPOND studies.

Additionally and in consultation with SMA experts, Biogen has developed a conceptual clinical framework to evaluate the potential value of Konectom™.

Konectom™ is a mobile application designed to enable adults living with SMA to quantitatively and remotely self-assess motor function in their daily lives.

Currently used only in research settings, Konectom™ leverages smart sensing technologies like touchscreen and accelerometry, to capture tangible data when studying neurological diseases.

In SMA, monitoring fatigue and smartphone typing skills may be useful to assess functional impact across a broad range of patients with varying levels of condition severity. It is hoped that this new technology will provide a more accurate and complete picture of how SMA impacts a person’s daily life.
 

Further information:

DEVOTE study

RESPOND study

The press release

About nusinersen