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Biogen's (BIIB) Tofersen Reports Mixed Data From ALS Study

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Biogen (BIIB - Free Report) reported mixed results from the phase III VALOR study and ongoing open-label extension (“OLE") study evaluating its investigational antisense drug tofersen in patients having amyotrophic lateral sclerosis (“ALS”) with superoxide dismutase 1 (“SOD1”) mutation.

The VALOR study evaluated the efficacy, safety, tolerability and pharmacodynamic effects of tofersen in comparison with placebo in adult patients with SOD1-ALS across a 28-week treatment period.

The study did not achieve statistical significance in its primary endpoint measured by change from baseline to week 28 in the Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R).

However, the study observed favorable trends across multiple secondary and exploratory endpoints. Data from the VALOR study and OLE study showed that patients administered tofersen exhibited signs of reduced disease progression across multiple measures including reduction in SOD1 protein, reduction of neurofilament as well as improvement in respiratory function as compared with placebo.

Shares of the company were down in pre-market trading on Oct 18 following the above news. However, shares of the company have risen 14.8% so far this year against the industry’s 11% decline.

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ALS is a neurodegenerative disease whose progression leads to a steady decline in the ability to move, speak, eat and eventually breathe. The average life expectancy of people with ALS ranges from three to five years. SOD1-mutated ALS is a genetic form of ALS that currently accounts for 2% of the ALS population.

Currently, there are no genetically targeted treatment options for ALS. While the study did not achieve statistical significance in primary endpoints, data from both the VALOR study and the OLE study demonstrated that patients who started tofersen earlier reported signs of reduced disease progression.

Per the press release, the company also plans to expand the ongoing early access program in countries allowing such programs to cater to a larger SOD1-ALS patient population to receive tofersen free of charge.

Please note that Biogen in-licensed rights to tofersen from Ionis Pharmaceuticals (IONS - Free Report) under a collaborative development and license agreement.

Zacks Rank & Stocks to Consider

Biogen currently carries a Zacks Rank #3 (Hold). A couple of better-ranked stocks in the same sector are Horizon Therapeutics (HZNP - Free Report) and Regeneron Pharmaceuticals (REGN - Free Report) , each sporting a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.

Horizon’s earnings per share estimates for 2021 have increased from $4.46 to $4.68 in the past 60 days. The same for 2022 has risen from $5.84 to $6.24 over the same period. The stock has rallied 53.2% in the year so far.

Regeneron’s earnings per share estimates for 2021 have increased from $54.15 to $62.31 in the past 60 days. The same for 2022 has risen from $44.11 to $46.72 over the same period. The stock has rallied 14.5% in the year so far.