CAMBRIDGE, Mass. – Biogen Inc. (NASDAQ:BIIB) announced that the European Commission has granted marketing authorization for QALSODY® (tofersen) for the treatment of adults with amyotrophic lateral sclerosis (ALS) linked to a mutation in the superoxide dismutase 1 (SOD1) gene. This marks QALSODY as the first therapy in the European Union targeting a genetic cause of ALS.
The approval, given under exceptional circumstances due to the rarity of SOD1-ALS, maintains the orphan designation previously granted to the drug. The decision follows a positive benefit/risk assessment and is supported by a body of evidence including the targeted mechanism of action, biomarker, and clinical data from the Phase 3 VALOR study. Although the primary efficacy endpoint in the study did not show statistical significance, there was a favorable trend, and a significant reduction in plasma neurofilament light chain (NfL) levels, a marker of neuronal damage, was observed in treated patients.
The European Academy of Neurology has acknowledged QALSODY as a first-line treatment for patients with SOD1-ALS. Stephanie Fradette, Pharm.D., Head of the Neuromuscular Development Unit at Biogen, emphasized the company's commitment to bringing QALSODY to SOD1-ALS patients throughout Europe as quickly as possible.
QALSODY has also been approved in the United States, and Biogen is in discussions with regulatory authorities in other regions. The drug, an antisense oligonucleotide (ASO), is designed to reduce the production of SOD1 protein, which in its toxic form contributes to the degeneration of motor neurons in ALS. The U.S. Food and Drug Administration granted accelerated approval based on the reduction in plasma NfL levels, with continued approval contingent upon further clinical trials.
The European Organisation for Professionals and People living with ALS (EUpALS) expressed excitement about the availability of QALSODY in Europe, viewing it as a significant development for the ALS community. Through Biogen's early access program, approximately 330 people with SOD1-ALS have already received the treatment across 18 EU countries.
Biogen, which licensed QALSODY from Ionis Pharmaceuticals (NASDAQ:IONS), Inc., is also conducting the Phase 3 ATLAS study to investigate the drug's potential to delay the onset of clinical symptoms in presymptomatic individuals with a SOD1 mutation.
InvestingPro Insights
As Biogen Inc. (NASDAQ:BIIB) celebrates the European Commission's authorization of QALSODY for ALS treatment, investors may be interested in the company's financial health and market performance. Biogen, a notable name in the biotechnology sector, has been profitable over the last twelve months, with a reported revenue of $9.66 billion. Despite a slight downturn in revenue growth of -4.37% during the same period, the company has maintained a robust gross profit margin of 75.49%, showcasing its ability to retain a majority of its revenue as gross profit.
Biogen's current market capitalization stands at $32.04 billion, reflecting investor valuation of the company. With an adjusted P/E ratio of 19.25, Biogen trades at a multiple that suggests investors are confident in its earnings potential relative to its share price. An interesting characteristic of Biogen's stock is its low price volatility, which can be a sign of a stable investment to potential shareholders. Additionally, Biogen's liquid assets have surpassed its short-term obligations, indicating a strong liquidity position.
InvestingPro Tips highlight that while 11 analysts have revised their earnings downwards for the upcoming period, the company's stock price often moves in the opposite direction of the market. This contrarian behavior could be a hedge against market downturns for diversified portfolios. Moreover, Biogen does not pay a dividend, aligning with companies that prefer to reinvest earnings into growth and development. For those interested in deeper analysis and more tips, InvestingPro offers additional insights on Biogen, available with a special offer using the coupon code PRONEWS24 for an extra 10% off a yearly or biyearly Pro and Pro+ subscription. Currently, there are 7 additional InvestingPro Tips available for Biogen, which could provide valuable guidance for investors.
As Biogen progresses with its innovative treatments and clinical studies, investors and analysts alike will be watching closely to see how these developments might influence the company's financial trajectory and stock performance in the dynamic biotech industry.
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