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BIIB's Next-Gen SMA Therapy Salanersen Wins FDA's Breakthrough Tag
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Key Takeaways
BIIB's salanersen received FDA Breakthrough Therapy designation for spinal muscular atrophy.
Phase Ib data showed motor function gains, lower neurofilament levels and a favorable safety profile.
Salanersen may allow once-yearly dosing and is being tested in three global phase III studies.
Biogen (BIIB - Free Report) recently announced that the FDA has granted Breakthrough Therapy designation to Salanersen, an investigational next-generation antisense oligonucleotide (ASO) for the treatment of patients with spinal muscular atrophy (SMA).
The FDA’s Breakthrough Therapy designation is intended to facilitate drug development and expedite the review of therapies for serious diseases with unmet medical needs.
Breakthrough Therapy Tag Based on BIIB’s Phase Ib Data
The FDA's Breakthrough Therapy Designation for salanersen was supported by data from the phase Ib study in children with SMA who had an inadequate response to prior gene therapy. The study demonstrated that treatment with once-yearly dosing of salanersen led to meaningful improvements in motor function, reduced neurodegeneration as measured by lower neurofilament levels and a favorable safety profile.
SMA is a rare genetic neuromuscular disorder caused by insufficient production of the survival motor neuron (SMN) protein, leading to the progressive loss of motor neurons in the spinal cord. It represents one of the leading genetic causes of infant mortality and can result in varying degrees of disability in adolescents and adults.
Biogen currently markets Spinraza, the first FDA-approved treatment for SMA. Salanersen and Spinraza are ASOs designed to increase SMN protein production by modifying SMN2 gene splicing. However, Salanersen has several potential advantages. The biggest differentiator is dosing frequency. Spinraza requires four loading doses followed by maintenance doses every four months, while Salanersen has been engineered for enhanced potency, which may allow once-yearly administration, significantly reducing the treatment burden for patients.
The FDA approved a higher dose of Spinraza in March, which offers two initial doses spaced 14 days apart, followed by a maintenance dose every four months. However, the dosing regimen has not yet been launched.
Year to date, shares of Biogen have risen 11.7% against the industry’s 3.4% decline.
Image Source: Zacks Investment Research
More on Biogen’s Salanersen
Biogen acquired worldwide rights to develop, manufacture and commercialize Salanersen from Ionis Pharmaceuticals (IONS - Free Report) in January 2022. The candidate was originally discovered by IONS.
Salanersen is being studied in a broad global phase III program comprising three studies: STELLAR-1, SOLAR and STELLAR-2.
STELLAR-1 is assessing salanersen in treatment-naïve, presymptomatic infants younger than six weeks with a genetic diagnosis of SMA.
SOLAR is evaluating the therapy in adolescents and adults aged 15 to 60 years with SMA, including both treatment-naïve patients and those previously treated with Evrysdi (risdiplam), Roche's (RHHBY - Free Report) approved SMA therapy.
Meanwhile, STELLAR-2 will study salanersen in presymptomatic infants who received Zolgensma (onasemnogene abeparvovec-xioi) gene therapy at six weeks of age or younger, with Salanersen treatment initiated approximately six months later. The study is expected to begin this month. Zolgensma is a one-time gene replacement therapy developed by Novartis (NVS - Free Report) .
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BIIB's Next-Gen SMA Therapy Salanersen Wins FDA's Breakthrough Tag
Key Takeaways
Biogen (BIIB - Free Report) recently announced that the FDA has granted Breakthrough Therapy designation to Salanersen, an investigational next-generation antisense oligonucleotide (ASO) for the treatment of patients with spinal muscular atrophy (SMA).
The FDA’s Breakthrough Therapy designation is intended to facilitate drug development and expedite the review of therapies for serious diseases with unmet medical needs.
Breakthrough Therapy Tag Based on BIIB’s Phase Ib Data
The FDA's Breakthrough Therapy Designation for salanersen was supported by data from the phase Ib study in children with SMA who had an inadequate response to prior gene therapy. The study demonstrated that treatment with once-yearly dosing of salanersen led to meaningful improvements in motor function, reduced neurodegeneration as measured by lower neurofilament levels and a favorable safety profile.
SMA is a rare genetic neuromuscular disorder caused by insufficient production of the survival motor neuron (SMN) protein, leading to the progressive loss of motor neurons in the spinal cord. It represents one of the leading genetic causes of infant mortality and can result in varying degrees of disability in adolescents and adults.
Biogen currently markets Spinraza, the first FDA-approved treatment for SMA. Salanersen and Spinraza are ASOs designed to increase SMN protein production by modifying SMN2 gene splicing. However, Salanersen has several potential advantages. The biggest differentiator is dosing frequency. Spinraza requires four loading doses followed by maintenance doses every four months, while Salanersen has been engineered for enhanced potency, which may allow once-yearly administration, significantly reducing the treatment burden for patients.
The FDA approved a higher dose of Spinraza in March, which offers two initial doses spaced 14 days apart, followed by a maintenance dose every four months. However, the dosing regimen has not yet been launched.
Year to date, shares of Biogen have risen 11.7% against the industry’s 3.4% decline.
Image Source: Zacks Investment Research
More on Biogen’s Salanersen
Biogen acquired worldwide rights to develop, manufacture and commercialize Salanersen from Ionis Pharmaceuticals (IONS - Free Report) in January 2022. The candidate was originally discovered by IONS.
Salanersen is being studied in a broad global phase III program comprising three studies: STELLAR-1, SOLAR and STELLAR-2.
STELLAR-1 is assessing salanersen in treatment-naïve, presymptomatic infants younger than six weeks with a genetic diagnosis of SMA.
SOLAR is evaluating the therapy in adolescents and adults aged 15 to 60 years with SMA, including both treatment-naïve patients and those previously treated with Evrysdi (risdiplam), Roche's (RHHBY - Free Report) approved SMA therapy.
Meanwhile, STELLAR-2 will study salanersen in presymptomatic infants who received Zolgensma (onasemnogene abeparvovec-xioi) gene therapy at six weeks of age or younger, with Salanersen treatment initiated approximately six months later. The study is expected to begin this month. Zolgensma is a one-time gene replacement therapy developed by Novartis (NVS - Free Report) .
BIIB’s Zacks Rank
Biogen currently carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.