DEVOTE study initiated of Spinraza for potential greater efficacy when administered at a higher dose than currently approved for Spinal Muscular Atrophy.- Biogen

Biogen announced that the first patient has been treated in the global clinical study, DEVOTE . The study is designed to evaluate the safety, tolerability and potential for even greater efficacy of Spinraza (nusinersen) when administered at a higher dose than currently approved for the treatment of spinal muscular atrophy (SMA). The Phase II/III randomized, controlled, dose-escalating study will be conducted at approximately 50 sites around the world and aims to enroll individuals of all ages with SMA.

The three-part study will include an open-label safety evaluation cohort and a pivotal, double-blind, active control randomized treatment cohort followed by an open-label treatment cohort. After the safety evaluation cohort completes, the pivotal cohort will begin and include a treatment group receiving two loading doses of 50 milligrams (mg) 14 days apart, followed by a maintenance dose of 28 mg every four months. A second treatment group will receive the current FDA-approved administration of Spinraza, which is four loading doses with 12 mg maintenance doses every four months. The third cohort will be an open-label evaluation to assess the safety and tolerability of transitioning patients from the currently approved dose of Spinraza to the higher dose being tested in the study.