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® (nusinersen)
CAMBRIDGE, Mass., June 15, 2022 (GLOBE NEWSWIRE) -- Biogen Inc. (Nasdaq: BIIB) will present new data from clinical studies aimed at assessing remaining unmet needs for people living with spinal muscular atrophy (SMA) and evaluating the potential impact of SPINRAZA® (nusinersen) in different patient populations at the SMA Research & Clinical Care Meeting hosted by Cure SMA this week in Anaheim, Calif. Biogen is a presenting sponsor of Cure SMA’s 2022 Annual SMA Conference, the world’s largest meeting dedicated to SMA research and care.
“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
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 treatment. 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:
*Nusinersen is currently commercialized under the brand name SPINRAZA® and the U.S. Food and Drug Administration-approved dose is 12 mg. As a foundation of care in SMA, more than 13,000 individuals have been treated with SPINRAZA worldwide.1
About SPINRAZA® (nusinersen)
Biogen licensed the global rights to develop, manufacture and commercialize SPINRAZA from Ionis Pharmaceuticals, Inc. (Nasdaq: IONS), the leader in antisense therapeutics. Please click here for Important Safety Information and full Prescribing Information for SPINRAZA in the U.S., or visit your respective country’s product website.
About Spinal Muscular Atrophy (SMA)
SMA impacts approximately 1 in 10,000 live births,5-8 is a leading cause of genetic death among infants9 and causes a range of disability in teenagers and adults.4
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These statements involve risks and uncertainties that could cause actual results to differ materially from those reflected in such statements, including without limitation risks relating to the occurrence of adverse safety events and/or unexpected concerns that may arise from additional data or analysis, including from the DEVOTE, RESPOND and ASCEND studies; the risk that we may not fully enroll our clinical trials, or enrollment will take longer than expected; failure to obtain regulatory approvals in other jurisdictions; risks of unexpected costs or delays; failure to protect and enforce our data, intellectual property and other proprietary rights and uncertainties relating to intellectual property claims and challenges; regulatory authorities may require additional information or further studies; product liability claims; third party collaboration risks; and the direct and indirect impacts of the ongoing COVID-19 pandemic on our business, results of operations and financial condition. The foregoing sets forth many, but not all, of the factors that could cause actual results to differ from our expectations in any forward-looking statement. Investors should consider this cautionary statement as well as the risk factors identified in our most recent annual or quarterly report and in other reports we have filed with the U.S. Securities and Exchange Commission. These statements are based on our current beliefs and expectations and speak only as of the date of this news release. We do not undertake any obligation to publicly update any forward-looking statements, whether as a result of new information, future developments or otherwise.