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Biogen's Phase II/III SMA Study for Higher Spinraza Dose Meets Goals
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Biogen (BIIB - Free Report) announced positive initial data from the pivotal cohort (Part B) of its mid to late-stage study evaluating the safety and efficacy of a higher dose regimen of Spinraza (nusinersen) in previously untreated, symptomatic infants with spinal muscular atrophy (SMA).
The investigational higher dosing regimen of Spinraza, an antisense oligonucleotide targeting the root cause of SMA, includes two 50 mg doses 14 days apart, and a higher maintenance regimen, 28 mg, every four months. Please note that the 12 mg dosage strength of Spinraza is already approved in the United States to treat SMA in pediatric and adult patients.
BIIB’s Higher Spinraza Dose Improves Motor Functions in SMA Patients
Biogen reported that the phase II/III DEVOTE study achieved the primary endpoint, demonstrating a statistically significant improvement in motor function in infants after six months of treatment with the higher dosing regimen of Spinraza compared with a prespecified matched sham (untreated) control group from the pivotal phase III ENDEAR study. The 2016 FDA approval of Spinraza 12mg for the SMA indication was based on the success of the ENDEAR study and another pivotal study.
Per management, the higher dose regimen of Spinraza has the potential to slow neurodegeneration faster through greater reductions in neurofilament as early as day 64 relative to the approved dose. Biogen believes that the maintenance of the higher dose over time could lead to meaningful clinical benefit in infants with symptomatic SMA. Detailed results from the phase II/III DEVOTE study will be shared at upcoming medical conferences.
Year to date, shares of Biogen have plunged 20.8% compared with the industry’s 0.9% decline.
Image Source: Zacks Investment Research
Additionally, the higher dose regimen showed better results than the sham group across secondary endpoints and demonstrated a trend of improvement over the currently approved 12mg dose on key biomarkers and efficacy measures. The candidate was generally well tolerated, with adverse events aligning with those typically seen in SMA and Spinraza's known safety profile. Interestingly, the percentage of serious adverse events was lower in the higher dose group compared with the 12mg group.
Based on such encouraging results, Biogen plans to file for regulatory approval of this investigational higher dose regimen of Spinraza to treat SMA in infants soon.
Declining Sales Trend for Spinraza a Woe for Biogen
Spinraza faces competition from Novartis’ gene therapy, Zolgensma, and Roche and PTC Therapeutics’ Evrysdi (risdiplam), which are hurting sales of Spinraza in the United States.
Spinraza sales declined almost 3% year over year in 2023 and 2% in the first half of 2024 due to increased competitive pressure.
Spinraza was discovered and developed jointly by Biogen and Ionis Pharmaceuticals (IONS - Free Report) . Post approval for the SMA indication, Biogen in-licensed the drug’s worldwide rights from Ionis and assumed responsibility for its future development, manufacturing and commercialization.
Please note that the two companies also have an ongoing collaboration agreement for another candidate,tofersen, which was initially developed by Ionis. Last year, tofersen, an antisense medicine, received accelerated approval in the United States and EU under the brand name Qalsody to treat adult patients with superoxide dismutase 1 amyotrophic lateral sclerosis. Biogen enjoys worldwide commercial rights for Qalsody.
In the past 60 days, estimates for Illumina’s 2024 earnings per share have moved up from $1.18 to $3.62. Earnings per share estimates for 2025 have improved from $2.93 to $4.50. Year to date, shares of ILMN have lost 7.6%.
ILMN’s earnings beat estimates in each of the trailing four quarters, the average surprise being 463.46%.
In the past 60 days, estimates for Fulcrum Therapeutics’ 2024 loss per share have narrowed from $1.24 to 48 cents. Loss per share estimates for 2025 have narrowed from $1.71 to $1.51. Year to date, shares of FULC have jumped 22.2%.
FULC’s earnings beat estimates in each of the trailing four quarters, the average surprise being 393.18%.
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Biogen's Phase II/III SMA Study for Higher Spinraza Dose Meets Goals
Biogen (BIIB - Free Report) announced positive initial data from the pivotal cohort (Part B) of its mid to late-stage study evaluating the safety and efficacy of a higher dose regimen of Spinraza (nusinersen) in previously untreated, symptomatic infants with spinal muscular atrophy (SMA).
The investigational higher dosing regimen of Spinraza, an antisense oligonucleotide targeting the root cause of SMA, includes two 50 mg doses 14 days apart, and a higher maintenance regimen, 28 mg, every four months. Please note that the 12 mg dosage strength of Spinraza is already approved in the United States to treat SMA in pediatric and adult patients.
BIIB’s Higher Spinraza Dose Improves Motor Functions in SMA Patients
Biogen reported that the phase II/III DEVOTE study achieved the primary endpoint, demonstrating a statistically significant improvement in motor function in infants after six months of treatment with the higher dosing regimen of Spinraza compared with a prespecified matched sham (untreated) control group from the pivotal phase III ENDEAR study. The 2016 FDA approval of Spinraza 12mg for the SMA indication was based on the success of the ENDEAR study and another pivotal study.
Per management, the higher dose regimen of Spinraza has the potential to slow neurodegeneration faster through greater reductions in neurofilament as early as day 64 relative to the approved dose. Biogen believes that the maintenance of the higher dose over time could lead to meaningful clinical benefit in infants with symptomatic SMA. Detailed results from the phase II/III DEVOTE study will be shared at upcoming medical conferences.
Year to date, shares of Biogen have plunged 20.8% compared with the industry’s 0.9% decline.
Image Source: Zacks Investment Research
Additionally, the higher dose regimen showed better results than the sham group across secondary endpoints and demonstrated a trend of improvement over the currently approved 12mg dose on key biomarkers and efficacy measures. The candidate was generally well tolerated, with adverse events aligning with those typically seen in SMA and Spinraza's known safety profile. Interestingly, the percentage of serious adverse events was lower in the higher dose group compared with the 12mg group.
Based on such encouraging results, Biogen plans to file for regulatory approval of this investigational higher dose regimen of Spinraza to treat SMA in infants soon.
Declining Sales Trend for Spinraza a Woe for Biogen
Spinraza faces competition from Novartis’ gene therapy, Zolgensma, and Roche and PTC Therapeutics’ Evrysdi (risdiplam), which are hurting sales of Spinraza in the United States.
Spinraza sales declined almost 3% year over year in 2023 and 2% in the first half of 2024 due to increased competitive pressure.
Biogen Inc. Price and Consensus
Biogen Inc. price-consensus-chart | Biogen Inc. Quote
Biogen’s Ongoing Collaborations With Ionis
Spinraza was discovered and developed jointly by Biogen and Ionis Pharmaceuticals (IONS - Free Report) . Post approval for the SMA indication, Biogen in-licensed the drug’s worldwide rights from Ionis and assumed responsibility for its future development, manufacturing and commercialization.
Please note that the two companies also have an ongoing collaboration agreement for another candidate,tofersen, which was initially developed by Ionis. Last year, tofersen, an antisense medicine, received accelerated approval in the United States and EU under the brand name Qalsody to treat adult patients with superoxide dismutase 1 amyotrophic lateral sclerosis. Biogen enjoys worldwide commercial rights for Qalsody.
Zacks Rank & Stocks to Consider
Biogen currently carries a Zacks Rank #3 (Hold).
Some better-ranked stocks in the biotech sector are Illumina, Inc. (ILMN - Free Report) and Fulcrum Therapeutics, Inc. (FULC - Free Report) , each sporting a Zacks Rank #1 (Strong Buy) at present. You can see the complete list of today’s Zacks #1 Rank stocks here.
In the past 60 days, estimates for Illumina’s 2024 earnings per share have moved up from $1.18 to $3.62. Earnings per share estimates for 2025 have improved from $2.93 to $4.50. Year to date, shares of ILMN have lost 7.6%.
ILMN’s earnings beat estimates in each of the trailing four quarters, the average surprise being 463.46%.
In the past 60 days, estimates for Fulcrum Therapeutics’ 2024 loss per share have narrowed from $1.24 to 48 cents. Loss per share estimates for 2025 have narrowed from $1.71 to $1.51. Year to date, shares of FULC have jumped 22.2%.
FULC’s earnings beat estimates in each of the trailing four quarters, the average surprise being 393.18%.