On April 19, 2021, Biogen announced initial findings from DEVOTE at the America Academy of Neurology Annual Meeting. Here is the relevant section from our news release summarizing these initial findings:
“An analysis of the higher loading and maintenance dosing regimen in Part A (n=6; 28 mg) showed no new safety concerns in study participants who were followed for up to approximately five months (64-158 days). There were no adverse events (AEs) reported that were considered related to the higher dose study drug and there were no severe or serious AEs. Four patients reported mild or moderate AEs, including AEs considered related to the treatment administration procedure. This emerging safety profile supports Biogen’s continued development of a higher dose of SPINRAZA, including ongoing enrollment of patients in the pivotal Part B of the DEVOTE study. This part will evaluate the higher-dose regimen (2 loading doses of 50 mg two weeks apart followed by 28 mg maintenance doses every four months) compared to the approved 12 mg dose of SPINRAZA: four loading doses, followed by maintenance doses every four months.
More information about DEVOTE is available at www.ClinicalTrials.gov (NCT04089566).”
Initial findings that Biogen announced and presented on Feb 19, 2021 from DEVOTE at the American Academy of Neurology Annual Meeting can be found here.
Also in April, the first person with spinal muscular atrophy (SMA) was successfully treated in Biogen’s ONWARD (NCT04729907) study. ONWARD is an open label extension (OLE) study for participants who successfully complete participation in the DEVOTE study.
The DEVOTE study (NCT04089566) is designed to evaluate the safety, tolerability, and potential for even greater efficacy of SPINRAZA® (nusinersen) when it is administered at a higher dose than currently approved for the treatment of SMA. All participants enrolled in DEVOTE receive treatment with nusinersen, either through a higher dose regimen or the currently approved dosing regimen. There is no sham or placebo group in the study. Enrollment in the ONWARD extension study is available to individuals who successfully complete participation in Biogen’s DEVOTE study. The objectives of ONWARD are to further evaluate the long-term safety, tolerability, and efficacy of nusinersen when administered at a higher dose than currently approved for the treatment of SMA. Regardless of which treatment group they were in during DEVOTE, all participants enrolled in ONWARD will receive the investigational higher dose of nusinersen.
Biogen is actively recruiting individuals with SMA into the DEVOTE study (Part B through a global network of enrollment sites. As of today May 5, 2021, there are open enrollment sites in Estonia (1), France (2), Germany (1), Greece (1), Hungary (2), Ireland (1), Italy (1), Republic of Korea (1), Latvia (1), Poland (2), Spain (4), Taiwan (2) and the United States (5). Biogen is also working to open enrollment at additional sites in additional countries. A full list of enrollment sites and their status is maintained online at www.clinicaltrials.gov (NCT04089566)
The initiation of ONWARD is an important milestone in the exploration of the impact that a higher dose of nusinersen may have on clinical outcomes in people with SMA.