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Biogen reports progress in SMA treatment with higher nusinersen dose

Published 10/08/2024, 07:41 AM
BIIB
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CAMBRIDGE, Mass. - Biogen Inc. (NASDAQ:BIIB) today shared results from its DEVOTE study, indicating that a higher dose regimen of nusinersen may offer clinical benefits for individuals with spinal muscular atrophy (SMA), including those previously treated with the drug and treatment-naïve patients. The study involved an investigational dosage that includes an initial rapid loading phase with two 50 mg doses 14 days apart, followed by a 28 mg maintenance dose every four months.

The results, set to be presented at the World Muscle Society 2024 Congress, suggest that the increased dose of nusinersen could more quickly lower neurofilament levels, a marker of neurodegeneration, compared to the currently approved 12 mg regimen of the drug, marketed as SPINRAZA®. This finding is significant as slowing neurodegeneration is crucial for individuals living with SMA.

In the pivotal Part B cohort of the study, treatment-naïve infants demonstrated significant improvements in motor function when treated with the higher dose regimen. These infants saw a 94% reduction in plasma neurofilament light chain from baseline to Day 183, a stark contrast to the 30% reduction in the sham control group. Additionally, there was a 67.8% relative reduction in the risk of death or permanent ventilation compared to the sham group and a 29.9% reduction when compared to the 12 mg regimen.

The DEVOTE study also included Part C, where participants aged 4-65 transitioned to the higher dose regimen after a median of 3.9 years on the approved 12 mg dose. These participants experienced improvements in motor function, with mean increases on various motor function scales.

Across the study, the higher dose regimen was generally well-tolerated, with a safety profile consistent with the approved 12 mg regimen. Biogen plans to submit regulatory applications globally for approval of the higher dose regimen of nusinersen.

The DEVOTE study is a Phase 2/3 trial designed to evaluate the safety and potential efficacy of a higher dose of nusinersen compared to the currently approved regimen for treating SMA. The study enrolled 145 participants across different ages and SMA types at approximately 42 sites worldwide.

The information in this article is based on a press release statement from Biogen.

In other recent news, Biogen Inc. has seen a flurry of activity with various developments. The company reported a total revenue of $2.5 billion in its second-quarter results. BMO Capital, Baird, TD Cowen, RBC Capital Markets, and Mizuho Securities have all maintained an Outperform rating for Biogen, with varying price targets.

Biogen, in collaboration with UCB, reported successful results from a Phase III trial of their drug Dapirolizumab pegol (DZP) for treating Systemic Lupus Erythematosus, and plans for a second trial in 2024 are underway. The company also achieved a significant regulatory milestone with Samsung (KS:005930) Bioepis, as their biosimilar, OPUVIZ™, was endorsed for marketing authorization by the European Medicines Agency's Committee for Medicinal Products for Human Use.

Further, Biogen expanded its Board of Directors with the appointment of Lloyd B. Minor, M.D., and Sir Menelas (Mene) Pangalos, Ph.D. These recent developments underscore Biogen's commitment to enhancing treatment options for diseases with unmet medical needs and expanding its product offerings in the biotechnology sector.

InvestingPro Insights

As Biogen (NASDAQ:BIIB) unveils promising results from its DEVOTE study, it's worth examining the company's current financial position and market performance. According to InvestingPro data, Biogen has a market capitalization of $26.71 billion, reflecting its significant presence in the biotechnology sector.

Despite the positive news from the DEVOTE study, Biogen's stock has faced challenges recently. InvestingPro Tips indicate that the stock is trading near its 52-week low and the RSI suggests it may be in oversold territory. This could present an opportunity for investors who see potential in the company's pipeline, including the higher dose regimen of nusinersen.

Financially, Biogen remains solid with a P/E ratio of 15.26 (adjusted for the last twelve months as of Q2 2024), which is lower than its current P/E of 22.94. This suggests that the stock might be undervalued relative to its earnings. The company's revenue for the last twelve months stands at $9.67 billion, with a high gross profit margin of 76.45%, indicating strong pricing power for its products.

An InvestingPro Tip highlights that Biogen is a prominent player in the Biotechnology industry, which aligns with the company's ongoing research and development efforts, such as the DEVOTE study. Additionally, the company's liquid assets exceed short-term obligations, providing financial flexibility to pursue further clinical studies and potential commercialization of new treatments.

It's worth noting that Biogen does not pay a dividend to shareholders, instead likely reinvesting profits into research and development, which is crucial for biotechnology companies to maintain their competitive edge.

For investors seeking a deeper understanding of Biogen's prospects, InvestingPro offers 8 additional tips that could provide valuable insights into the company's potential. These tips, along with real-time metrics, can help investors make more informed decisions about Biogen's stock in light of its recent clinical developments.

This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.

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