Biogen (BIIB) R&D Day to Provide Overview of Diversified Pipeline and Capabilities with Potential for Multiple Novel Therapies in Neuroscience

Biogen Inc. (Nasdaq: BIIB) will hold a virtual Investor R&D Day today, providing a comprehensive overview of the company’s diversified pipeline in neuroscience. Biogen plans to advance innovative therapies for patients through the work of its world-class researchers and the strength of its global network of collaborators. As part of R&D Day, Biogen’s scientific leaders will discuss ongoing research and highlight key advancements among Biogen’s 33 clinical programs, including 12 programs in Phase 3 or filed.

“Biogen is helping to change the Alzheimer’s disease treatment paradigm by providing the first therapy in 20 years, and we continue to lead the way in neuroscience with our diversified pipeline,” said Michel Vounatsos, Chief Executive Officer at Biogen. “Across our portfolio, we have achieved significant scientific progress, and I believe our specialization in terms of people, capabilities, and programs positions us to deliver multiple novel therapies with the potential to address the most complex neurological diseases.”

Key R&D Highlights “The talented team at Biogen is at the forefront of innovation in neuroscience. We look forward to sharing the assets and approaches we have developed aimed at addressing some of the most difficult challenges in healthcare,” said Alfred Sandrock, Jr., M.D., Ph.D., Head of Research and Development at Biogen. “Scientific advancements have increased our understanding of human genetics and allow us to advance the appropriate treatment modality for the right target for each disease. We believe this will mitigate risk in neuroscience, increase the probability of success for each program, and create opportunities for early and targeted treatments towards prevention.”

Alzheimer's DiseaseBiogen will present an overview of its Alzheimer’s disease pipeline with the recent FDA-approved ADUHELM serving as the foundation of a multi-target, multi-modality Alzheimer’s disease franchise. In addition to ADUHELM, the company will present progress on lecanemab (BAN2401), a new amyloid beta-directed antibody therapy in Phase 3 being developed by Eisai in collaboration with Biogen, and BIIB080 (IONIS-MAPTRx), a tau-directed antisense oligonucleotide (ASO).

Lecanemab received Breakthrough Therapy designation from the FDA in June 2021. In clinical studies, lecanemab has shown potential to slow clinical decline in Alzheimer’s disease and is now being studied in patients with early Alzheimer’s disease as well as the preclinical setting, when individuals are clinically normal and have intermediate or elevated levels of amyloid in their brains.

A Phase 1b study of BIIB080 met the primary objective of safety and tolerability, and demonstrated a durable time and dose dependent reduction of tau protein in cerebrospinal fluid. Growing evidence suggests tau may be a key driver of neurodegeneration in Alzheimer’s disease.NeuropsychiatryIn neuropsychiatry, Biogen and Sage Therapeutics are co-developing zuranolone, and will present an overview of the two-week, once-daily drug being studied in major depressive disorder (MDD) and postpartum depression (PPD). Zuranolone has demonstrated positive Phase 3 results.

In clinical trials, zuranolone, which represents a potential new class of drug, demonstrated rapid-acting efficacy and was generally well-tolerated. Beyond MDD and PPD, zuranolone may have potential for development in other psychiatric disorders, including treatment-resistant depression, bipolar disorder, and generalized anxiety disorder.

Major depressive disorder is a common comorbidity of multiple neurological disorders in Biogen’s core therapeutic areas.

Biogen will also present an overview of BIIB104, currently under investigation in a Phase 2 study for cognitive impairment associated with schizophrenia.

Amyotrophic Lateral Sclerosis (ALS)Biogen has multiple ALS assets in development, which target both genetic subtypes of ALS and the broader population. Tofersen, developed in collaboration with Ionis Pharmaceuticals, is currently in Phase 3 studies. The program targets SOD1, a gene believed to be a genetic driver of disease in two percent of all ALS cases.

In the recently announced ATLAS study, Biogen will be exploring the use of tofersen prior to symptoms in SOD1 carriers with the intent to delay or slow the decline in function associated with ALS.

Based on learnings from the company’s prior and ongoing trials, Biogen is also advancing BIIB078, an ASO targeting C9orf72, another potential genetic driver of ALS, and BIIB105, an ASO targeting ATXN2, a potential treatment for ALS in the broader population supported by human genetics.

StrokeBiogen is progressing its research in the treatment of stroke. The potential to expand the stroke treatment window has been shown in positive proof of concept studies for BIIB131, a next generation thrombolytic agent, and BIIB093, a treatment for brain swelling in large hemispheric infarctions.

BIIB131, a treatment for acute ischemic stroke, was recently acquired based on positive Phase 2 data. BIIB093 is in a Phase 3 study for large hemispheric infarction, and a Phase 2 trial for lesion expansion in brain contusion.

LupusDecades of study by Biogen at the intersection of neurological and immunological pathways provide the company with expertise in immunology. Biogen is advancing two lupus therapies in Phase 3 trials. Dapirolizumab pegol is being developed in collaboration with UCB for systemic lupus erythematosus (SLE). The second, BIIB059, was fully developed in-house at Biogen and is now in Phase 3 for SLE, with plans for further study in cutaneous lupus erythematosus.

Distinct Capabilities and World-Class TalentBiogen has attracted and collaborated with leading scientists from industry and academia to focus on treatments with the highest unmet need. This focus also includes 27 business development transactions and collaborations since 2017, including with Sangamo Therapeutics, Inc., Denali Therapeutics Inc., Sage Therapeutics Inc, and InnoCare Pharma Limited. Biogen will continue to bolster the pipeline through both internal development and external collaborations.

Biogen has refined the early discovery process with new approaches to patient selection, target engagement, and monitoring of disease progression, increasing the probability of success for each program. Biogen centers its R&D methodologies on human biology, employing the predictive effects of genetics to validate potential targets. Utilizing biomarkers to measure modulation of disease early on in the drug development process can contribute to lowering the risk associated with developing novel therapies for neurological diseases.

Biogen Digital Health Today’s technological advances open a new era of opportunities for digital health in neuroscience. In April 2021, the company formed Biogen Digital Health, a global unit that aspires to transform patients’ lives and Biogen by making personalized and digital medicine in neuroscience a reality. In an on-demand presentation, Biogen Digital Health will share key areas of focus and highlights, including progress on digital biomarkers and efforts to develop deep-learning software.

Investor R&D Day Presenters Biogen scientific leaders will be joined by executives from Eisai, Sage Therapeutics, and Denali Therapeutics.

To join today's event, please go to the investors section of the Biogen website at investors.biogen.com or access the event link directly here. An archived version of the webcast and slides, as well as additional video presentations and slides, will be available here.

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