Biogen presents data on the RESPOND and DEVOTE Trials
23 June 2022
RESPOND is a phase 4 study evaluating the benefit and safety of Spinraza™
(nusinersen) in people treated with Zolgensma™
(onasemnogene abeparvovec). The study includes children who have unmet clinical needs after receiving the gene therapy (as identified by a doctor). Growing enrolment in this study indicates there are residual unmet clinical needs in infants and toddlers with SMA following treatment with the gene therapy.
Data presented showed that all enrolled study participants reported suboptimal clinical status across a variety of measures at baseline, with 13 of 16 showing this in multiple areas, including motor and respiratory functions and swallowing / feeding ability.
After beginning Spinraza™ treatment, initial safety findings showed three participants experienced a serious adverse event during the study period; none of these events were considered related to Spinraza™.
The DEVOTE Trial
Biogen also presented final data from Part A of the ongoing, three-part DEVOTE study evaluating the safety and tolerability of investigational, higher doses of nusinersen.
Part A of DEVOTE is an open-label safety evaluation period in children and teens with later-onset SMA. Results suggest that a higher dosing regimen of nusinersen leads to higher levels of the drug in the cerebrospinal fluid and is generally well-tolerated, with most reported adverse events considered to be mild in severity. None were considered related to nusinersen, but some were related to treatment administration. The totality of Part A data supports further development of a higher dose of nusinersen.
Part B and Part C of DEVOTE are currently enrolling at 52 sites worldwide.