Nusinersen sodium

Pictured: Biogen's signage at its headquarters in Massachusetts/iStock, JHVEPhoto With the global immunology market projected to grow from nearly $103.2 billion in 2024 to $257.4 billion by 2032, second only to oncology in terms of the largest therapy area by value, it’s not surprising that immunology has attracted major investment recently from biopharma companies. Add the fact that drugmakers are facing losses from looming patent expirations and immunology drugs have a relatively high chance of success in clinical trials, it’s clear to see why the market is so attractive. In April, Vertex acquired Alpine Immune Sciences for $4.9 billion, the largest acquisition so far in 2024, giving it access to potential treatments for autoimmune and inflammatory diseases. That same month, Bristol Myers Squibb announced a potential $1.8 billion, multi-year collaboration with Repertoire Immune Medicines to create up to three vaccines for autoimmune diseases. Biogen is the latest big player to join the fray with its acquisition of privately held Human Immunology Biosciences, also known as HI-Bio. Last month, Biogen announced it would acquire the company, which is focused on severe immune-mediated diseases, paying $1.15 billion upfront and with the potential for $650 million in milestone payments. Analysts see the deal as part of CEO Chris Viehbacher’s plan to move the company away from riskier plays in the neurological space and expand Biogen’s immunology and rare diseases pipeline. Specifically, Biogen is looking to expand its pipeline beyond its current crop of drugs, such as the Friedreich’s ataxia drug Skyclarys and the Alzheimer’s disease treatment Leqembi, Myles Minter, a research analyst at William Blair, told BioSpace. The move aims to “de-risk” their pipeline and avoid clinical trials with higher bars for success, he said. “They want to create a pipeline that is more de-risked. I think you get that in immunology. They don’t want to be this company where they’re relying on a pivotal trial in stroke or [a] very, very risky study in relapsing MS.” The buy gives Biogen access to drug candidate felzartamab, an anti-CD38 antibody being developed by HI-Bio for several chronic kidney conditions with large unmet medical needs, including IgA nephropathy, primary membranous nephropathy and antibody-mediated rejection in kidney transplant recipients. In a note to investors last month on Biogen’s HI-Bio deal, Bank of Montreal Capital Markets analysts made the case that felzartamab’s mechanism of selectively depleting CD38+ cells could give Biogen “applicability across a range of immune-mediated diseases.” Indeed, immunology and inflammation (I&I) is a particularly attractive market because of its “pipeline-in-a-product” potential of expanding into “adjacent indications and build a monster brand,” biotech entrepreneur Andrew Pannu told BioSpace in February. Other Moves in the I&I Space For other large biopharma companies, the role model in the immunology space is AbbVie’s blockbuster Humira, one of the highest grossing drugs of all time. Used to treat Crohn’s disease, plaque psoriasis and rheumatoid arthritis, among other inflammatory conditions, it brought in about $200 billion over the last two decades. Minter sees the I&I market, which attracts relatively high valuations, as an appropriate sector to target as part of Biogen’s and other companies’ de-risking strategies as they look to hedge their bets on more uncertain pipelines. While Biogen has been focused on its Alzheimer’s medicines, it has not been an easy path. Aduhelm faced intense scrutiny over its efficacy and pricing, making it perhaps one of the most controversial FDA approvals. Ultimately, Biogen in January discontinued Aduhelm and halted a post-approval study, deciding to focus its efforts instead on Leqembi with partner Eisai. The loss of patent protections is another factor fueling Biogen’s pipeline realignment. Minter contends that Biogen is being “forced” into de-risking its business strategy as the oral therapy for relapsing-remitting multiple sclerosis Tecfidera, a multibillion-dollar earner, is teetering on the patent cliff. Tecfidera’s patent will expire in the E.U. in 2025 and efforts to restore patent protections in the U.S. have been defeated. At the same time, Biogen’s combined expertise in immunology and rare diseases with products such as Skyclarys and the spinal muscular atrophy drug Spinraza, are a big leg up for the company as it targets the I&I market, according to Minter. New product launches, such as Skyclarys and Leqembi, are part of Biogen’s plan to bring revenue back up as well as investments in expanding markets. Startups are also looking to challenge the big players in the I&I space. Inflammatory disease-focused Mirador Therapeutics landed the largest early-stage venture capital investment in biopharma last quarter, launching with $400 million in financing. Alumis recently raised $259 million in a Series C round, while AltruBio and Attovia secured $225 million and $105 million in Series Bs, respectively. Alumis, a potential rival to Bristol Myers Squibb and Takeda in autoimmune diseases, in an SEC filing last week for an initial public offering said it plans to start Phase III trials of a TYK2 inhibitor in plaque psoriasis. Tyler Patchen is a staff writer at BioSpace. You can reach him at tyler.patchen@biospace.com. Follow him on LinkedIn.

Roche unveiled five-year results Friday showcasing sustained efficacy and a favourable safety profile for its oral spinal muscular atrophy (SMA) treatment Evrysdi (risdiplam), allowing it to keep the pressure on Biogen's first-mover Spinraza (nusinersen) as well as Novartis' one-time gene therapy Zolgensma (onasemnogene abeparvovec)."This is the final readout of the FIREFISH study, which has provided a wealth of insights and data, helping to firmly establish Evrysdi as an important treatment option," said chief medical officer Levi Garraway.Evrysdi was first approved in 2020 to treat SMA in adults and children two months and older based on the FIREFISH and SUNFISH trials. The small molecule's label was expanded in 2022 to include infants under two months.Newly released results from the FIREFISH study extension showed that after five years of Evrysdi treatment, 91% of children with Type 1 SMA – the most severe, infantile-onset form – were alive, whereas historically these patients would not have been expected to survive past two years of age. Additionally, 81% were alive without needing permanent ventilation.The majority also achieved or maintained major motor milestones like sitting – 59% could do so without support for 30 seconds – as well as standing and walking. Beyond mobility gains, 96% of participants could swallow, while 80% could feed orally without a feeding tube. Safety data were also reassuring, with no treatment discontinuations due to adverse events and a 66% reduction in overall event rates between years one and five.Industry observers see room for Evrysdi to stake its claim in the SMA space. In a recent investor note, Baird analysts highlighted the crowded but lucrative landscape, stating that "while progressive, fatal, ultra orphan diseases have often provided blockbuster opportunities for many individual products, the emergence of three highly effective treatments in such rapid succession should likely continue to lead to pressure on the first."Spinraza was approved by the FDA in 2016, while Zolgensma entered the market over two years later. In 2023, Biogen's drug generated sales of $1.7 billion, while Evrysdi generated CHF 1.4 billion ($1.6 billion) and Novartis recorded $1.2 billion from Zolgensma.Baird analysts expect Spinraza's commercial dominance to face "moderate pressure" from Evrysdi going forward, but anticipate the Biogen drug will still add "a nice cash stream for the next decade." They also touted well-entrenched Spinraza as "one of the most effective drugs we have ever seen."