- The stock price of Biogen Inc (NASDAQ: BIIB) increased by over 2% during intraday trading today. This is why.
The stock price of Biogen Inc (NASDAQ: BIIB) increased by over 2% during intraday trading today. Investors are responding positively to Biogen announcing it 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. And Biogen is a presenting sponsor of Cure SMA’s 2022 Annual SMA Conference, the world’s largest meeting dedicated to SMA research and care.
SMA Research Updates
The 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). And the Phase 4 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 9 patients were shared in March 2022, baseline and safety data from 16 patients enrolled in RESPOND (as of November 2021) are being presented. And 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. And the RESPOND study (NCT04488133) is currently enrolling participants at 20 sites worldwide.
Biogen is also sharing final data from Part A of the ongoing, three-part DEVOTE study evaluating the safety and tolerability of investigational, higher doses of nusinersen. And the 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. And 2 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.
“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. 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.”
— Maha Radhakrishnan, M.D., Chief Medical Officer at Biogen
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