Biogen Eyes Wave of Phase III Readouts as It Expands Beyond Neuroscience

Key Points

• Biogen expects a wave of late-stage results: Ten Phase III trials could begin producing data as soon as next quarter, potentially supporting eight launches or new indications across five molecules.
• The company is diversifying beyond neuroscience through acquisitions and development programs in immunology, kidney disease and ophthalmology, including felzartamab, SYFOVRE and EMPAVELI.
• Key upcoming catalysts include lupus and kidney-disease data: Litifilimab’s systemic lupus results are expected in the fourth quarter, while felzartamab data in antibody-mediated rejection are anticipated in the first half of next year.

Biogen NASDAQ: BIIB is reshaping its business around a broader mix of neuroscience, immunology, kidney disease and ophthalmology assets, President and CEO Chris Viehbacher said during a Morgan Stanley event. The company expects a series of late-stage clinical readouts to begin as soon as next quarter and said its recently acquired assets could help accelerate revenue growth.

Viehbacher said the company’s prior strategy was concentrated in neuroscience, with expensive late-stage programs and a narrow commercial footprint focused on neurologists. Biogen now calls on a wider range of specialists, including nephrologists, transplant nephrologists, rheumatologists, dermatologists, epileptologists and retina specialists.

“Internally, we talk about new Biogen,” Viehbacher said, describing the company’s effort to retain its neuroscience roots while expanding into immunology and other areas with potentially shorter, more defined Phase II studies.

Pipeline and business development strategy

Biogen’s business-development activity has included the acquisition of Human Immunology Biosciences, or Hi-Bio, which added felzartamab, and the acquisition of Apellis Pharmaceuticals. Viehbacher said both transactions fit the company’s focus on immunology, including rare and smaller indications.

He said Biogen has 10 Phase III trials that are expected to start producing results as soon as the next quarter. Those trials could support eight product launches or new indications involving five molecules, according to Viehbacher.

Looking ahead, Biogen intends to focus capital deployment on earlier-stage opportunities, including preclinical, Phase I and Phase II assets. Viehbacher said early development is also the most capital-efficient stage for acquisitions, although competition remains high in immunology because disease biology is well defined in some areas. He added that the pool of early-stage assets has expanded with the emergence of China-based drug development.

LEQEMBI access and Alzheimer’s development

Viehbacher said Alzheimer’s treatment uptake for LEQEMBI has been slower than some expected despite substantial unmet need. He attributed the pace in part to operational changes required at neurology practices, including arranging PET scans, MRIs, cognitive assessments, infusion capacity and supporting staff.

Biogen and partner Eisai are working to simplify the treatment pathway, he said. Blood-based diagnostics may reduce reliance on PET scans, which Viehbacher said can cost about $5,000, compared with less than $130 for a blood-based diagnostic. The company has also seen the proportion of eligible patients among those referred improve from 50% to 70% as blood-based testing is increasingly used in primary care settings, he said.

Biogen received approval for a subcutaneous LEQEMBI maintenance formulation last year and an induction formulation this year. Viehbacher said the induction approval could improve the treatment pathway and competitive positioning, but broad uptake depends on Medicare Part D reimbursement.

He said negotiations with plans are under way, with reimbursement decisions effective Jan. 1. More than 80% of requested medical formulary exceptions have been approved, he added.

Biogen’s AHEAD 3-45 study of LEQEMBI in earlier-stage Alzheimer’s disease is fully enrolled and is expected to read out in 2028 after four years of follow-up. Viehbacher said the studies are designed to assess whether earlier intervention against amyloid accumulation can delay or prevent disease progression. Biogen is also advancing diranersen, a tau-targeting program.

Lupus and kidney disease readouts ahead

Biogen expects Phase III data in systemic lupus erythematosus, or SLE, for litifilimab in the fourth quarter. A Phase III study in cutaneous lupus erythematosus, or CLE, is expected to generate data in the first half of 2027, while the company expects one-year CLE data at a European conference later this year.

Viehbacher acknowledged the challenges of SLE trials, citing disease heterogeneity, varying symptoms, flare-and-remission patterns and background steroid use. Still, he said Biogen has applied lessons from previous lupus studies and believes its experience in multiple sclerosis could be commercially relevant.

The company estimates the worldwide lupus market at about $8 billion, although Viehbacher said broader biologic penetration could take time. Biogen is also partnered with UCB on dapirolizumab, which has produced one positive Phase III result, and has an IRAK4 degrader that has entered the clinic.

Felzartamab is being studied initially across four kidney indications: antibody-mediated rejection, or AMR; immunoglobulin A nephropathy, or IgAN; primary membranous nephropathy; and microvascular inflammation. Data in AMR are expected in the first half of next year.

Viehbacher said a Phase II study showed an 80% resolution rate in AMR, though he cautioned that the result must be replicated in Phase III. He said the U.S. AMR patient population is about 11,000 people and that Biogen could be first to market if the program succeeds.

For IgAN, he highlighted the potential for a treatment-free period after nine infusions. Biogen observed an effect maintained for 18 months without treatment in earlier data, he said, though the duration will be tested in Phase III studies.

Apellis products support commercial expansion

Viehbacher said Apellis added growth products SYFOVRE and EMPAVELI while also giving Biogen a commercial nephrology presence ahead of a potential felzartamab launch.

He said SYFOVRE growth accelerated somewhat following the acquisition after Biogen retained the commercial team. Biogen had initially modeled mid-single-digit growth for the product, and Viehbacher said the company is now somewhat more optimistic than it was at the time of the acquisition, while noting the competitive environment.

EMPAVELI has enabled Biogen to build relationships with nephrologists and transplant centers, Viehbacher said. That existing infrastructure could support a faster uptake for felzartamab if the clinical data and eventual label support approval.

About Biogen (NASDAQ:BIIB)

Biogen Inc is a biotechnology company focused on discovering, developing and commercializing therapies for neurological and neurodegenerative diseases. Founded in 1978, the company is headquartered in Cambridge, Massachusetts, and serves patients and healthcare providers in markets around the world.

Biogen's portfolio includes treatments for multiple sclerosis, including Tecfidera, Vumerity, Tysabri, Avonex and Plegridy, as well as Spinraza for spinal muscular atrophy, developed in collaboration with Ionis Pharmaceuticals.

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