New data presented at Cure SMA reveal residual unmet needs in young SMA patients treated with gene therapy and suggest further potential of using Spinraza.

“The data we are presenting at this year’s Cure SMA conference – including the latest updates from the RESPOND and DEVOTE studies – reinforce Biogen’s commitment to evaluating the potential of Spinraza to further improve clinical outcomes for individuals with SMA,” said Maha Radhakrishnan, M.D., Chief Medical Officer at Biogen. “There are key unmet needs within the SMA community and we are committed to addressing these through our ongoing research that includes active enrollment in three global clinical studies.”

SMA Research Updates: Growing enrollment in the RESPOND study indicate there are residual unmet clinical needs in infants and toddlers with SMA following treatment with Zolgensma (onasemnogene abeparvovec). The Phase IV study is evaluating the clinical benefit and safety of Spinraza in infants and toddlers with SMA who have unmet needs following treatment with the gene therapy. Since initial findings from nine patients were shared in March 2022, baseline and safety data from 16 patients enrolled in RESPOND (as of November 2021) are being presented. 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 (median duration of 132.5 days) show three participants experienced a serious adverse event (AE) during the study period; none of these events were considered related to Spinraza.

The RESPOND study (NCT04488133) is currently enrolling participants at 20 sites worldwide; more information about the eligibility criteria is available at clinicaltrials.gov. Biogen will also share final data from Part A of the ongoing, three-part DEVOTE study evaluating the safety and tolerability of investigational, higher doses of nusinersen. Results from Part A, an open-label safety evaluation period in children and teens with later-onset SMA, suggest that a higher dosing regimen (28 mg) of nusinersen leads to higher levels of the drug in the cerebrospinal fluid and is generally well-tolerated, with most AEs reported considered to be mild in severity. The most common AEs reported were headache and procedural pain. Two serious AEs (fall, femur fracture) were reported in one participant during the study period. No AEs were considered related to nusinersen and some were related to treatment administration. The totality of Part A data supports further development of a higher dose of nusinersen.

Currently, Part B and Part C of DEVOTE evaluating an investigational, higher dose of nusinersen are enrolling at 52 sites worldwide. Information on the DEVOTE trial (NCT04089566) is available at clinicaltrials.gov.

Featured Spinraza Data Presentations Include; .i. Results From the End of Part A of the Ongoing 3-Part DEVOTE Study to Explore Higher Doses of Nusinersen in SMA – Friday, June 17, 2022 at 9:40 a.m. PT. ii. Baseline Characteristics and Initial Safety Results in RESPOND: A Phase IV Study of Nusinersen in Children With SMA Who Received Onasemnogene Abeparvovec – Friday, June 17, 2022 at 10 a.m. PT.

Nusinersen is currently commercialized under the brand name Spinraza and the FDA-approved dose is 12 mg. As a foundation of care in SMA, more than 13,000 individuals have been treated with Spinraza worldwide.