Biogen reports positive interim data for once-yearly SMA treatment

CAMBRIDGE, Mass. - Biogen Inc. (NASDAQ:BIIB), a prominent biotechnology company with a market capitalization of $18.51 billion and strong financial health metrics according to InvestingPro, announced interim Phase 1 results for salanersen (BIIB115/ION306), an antisense oligonucleotide being developed for spinal muscular atrophy (SMA), showing promising outcomes in children previously treated with gene therapy.

The study evaluated two dose levels (40 mg and 80 mg) administered once yearly. According to the company, which maintains an impressive 75.65% gross profit margin and generates substantial free cash flow, both doses were generally well-tolerated and led to substantial reductions in neurofilament levels, indicating a slowing of neurodegeneration.

In participants with at least one year of follow-up who received the 40 mg dose, half achieved new World Health Organization motor milestones they previously could not accomplish independently, such as sitting, standing, crawling, or walking.

These participants also showed mean improvements of 3.3 points on the Hammersmith Functional Motor Scale-Expanded and 5.3 points on the Revised Upper Limb Module, which the company described as clinically meaningful.

"To see a child dosed with gene therapy at one year of age and still unable to sit without support at age five then gain the ability to sit independently just 3 months after initiating salanersen, that is unexpected," said Dr. Valeria Sansone, a principal investigator for the trial, in the press release.

The most common adverse events reported were pyrexia and upper respiratory tract infection, with most adverse events characterized as mild to moderate.

Salanersen uses the same mechanism of action as Biogen’s approved SMA treatment SPINRAZA (nusinersen) but is designed for greater potency and once-yearly dosing.

Based on these results, Biogen stated it is engaging with global health authorities regarding the design of Phase 3 studies for salanersen. The company, currently trading at an attractive P/E ratio of 12.51 and identified as undervalued by InvestingPro analysts, licensed the global rights for salanersen from Ionis Pharmaceuticals, Inc. Get access to detailed financial analysis and 6 additional ProTips about Biogen’s investment potential with an InvestingPro subscription.

SMA is a rare genetic disease affecting approximately 1 in 10,000 live births and is characterized by progressive muscle weakness due to motor neuron loss. For comprehensive analysis of Biogen’s market position and detailed financial metrics, access the full Pro Research Report available on InvestingPro, covering what really matters for informed investment decisions.

In other recent news, Biogen has initiated a Phase 3 trial for omaveloxolone in children with Friedreich ataxia, aiming to address the unmet needs in younger patients with this rare disorder. This study will involve 255 children and follows the marketing of omaveloxolone as SKYCLARIS for older age groups in over 40 countries. Additionally, Goldman Sachs has maintained its Buy rating for Biogen, with a price target of $196, following strong earnings reports from its partner, Eisai, related to Alzheimer’s treatment Leqembi. Eisai’s fiscal year revenue for Leqembi surpassed its forecast, driven by robust sales in Japan and China.

Meanwhile, UBS has reiterated a neutral rating for Biogen, noting potential in pre-symptomatic Alzheimer’s treatments but expressing caution due to timeline uncertainties. H.C. Wainwright also maintained a Buy rating, highlighting Biogen’s focus on nephrology and the potential of felzartamab in rare kidney diseases. The firm noted promising Phase 2 results for felzartamab, which could establish a nephrology franchise for Biogen. These developments reflect Biogen’s strategic efforts across various therapeutic areas, including neurology and nephrology.

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