Message from the CEO

 

 

2019 was a remarkable year for Biogen as we delivered strong operating performance across all of our core business areas, double-digit earnings growth versus a year ago and strong execution of our strategy. We strengthened our pipeline by adding seven new clinical programs, which we believe will help us further expand our multi-franchise portfolio and drive mid- and long-term growth.

2019 was also marked by changing developments for aducanumab, an investigational treatment for early Alzheimer’s disease. In October 2019, together with our collaboration partner Eisai Co., Ltd. (Eisai), we announced plans to pursue regulatory approval for aducanumab in the U.S. This decision was based on a new analysis, conducted in consultation with the U.S. Food and Drug Administration (FDA), of a larger dataset from the Phase 3 EMERGE and ENGAGE studies of aducanumab that had been discontinued in March 2019 following a pre-specified futility analysis.


The extraordinary events surrounding aducanumab and the progress across our portfolio is a testament to Biogen’s commitment to follow the science – one that was made possible by fearless, dedicated colleagues.


Before turning to 2019 in more detail, we want to acknowledge the health and economic challenges facing all of us as a result of the current COVID-19 pandemic. Many of our communities as well as a number of our colleagues have been directly affected by COVID-19. We are committed to doing all we can to ensure the health and safety of all our employees and provide an uninterrupted supply of our medicines to patients around the world. We are grateful to everyone at Biogen who has helped us maintain our manufacturing and business operations so that patients can continue to receive our therapies. We are closely monitoring the ongoing and ever-changing developments and the impact it may have on our business operations, including our sales, manufacturing and clinical trials.

 

 

We work with purpose to advance science to address the urgent and long-term challenges facing humanity.

— Michel Vounatsos
Chief Executive Officer

 

 

Our purpose

At Biogen we pioneer science with the goal of better understanding and preserving the underlying qualities of our essential human nature. We strongly believe that neuroscience is the next frontier that will see real scientific progress and breakthrough, and we believe that our diverse, talented workforce, with more than 7,700 employees worldwide, is uniquely positioned to take on some of the most challenging healthcare needs and to move Biogen forward. As we work to improve patients’ lives, we also care deeply about making a difference in our society as a whole through science that may have the potential to, among other things, improve brain health, mobility and vision. We focus on science that seeks to solve societal problems and create access to innovation. We work with purpose to advance science to address the urgent and long-term challenges facing humanity.

 

 

Delivering sustainable performance

In 2019, we generated $14.4 billion in full-year total revenues, a 7% increase versus the prior year, and we generated net cash flows from operations of approximately $7.1 billion. GAAP diluted earnings per share for 2019 were $31.42, an increase of 46% over 2018, and Non-GAAP diluted earnings per share increased 28% over the prior year to $33.57.


Our business and cash generation remained strong and provided us with the flexibility to allocate capital to create long-term stockholder value. In 2019 we spent approximately $2.3 billion in research and development and repurchased approximately 24 million shares of our common stock for a total value of approximately $5.9 billion. In addition, we spent approximately $515 million in 2019 on capital expenditures including a significant investment in the large-scale biologics manufacturing facility we are building in Solothurn, Switzerland.


These results reflect the resilience of our multiple sclerosis (MS) business as well as the continued growth of both SPINRAZA and our biosimilars business.

 

 

Capturing the neuroscience opportunity

To review Biogen’s strong 2019 performance – as well as Biogen’s future – let’s consider some key statistics.


It is estimated that approximately 50 million people worldwide suffer from dementia and approximately 10 million suffer from Parkinson’s disease. Neurological disease is the leading cause of disability and the second largest cause of death globally.


Aging populations will almost certainly increase these numbers significantly. It is estimated that the global population over the age of 60 will be nearly 1.5 billion by 2030, and by 2050 those over 60 will be nearly 2 billion, with 1.5 billion over the age of 65.


These numbers are only part of the story. There are important inflection points in medical history when a breakthrough in knowledge or technology generates new ideas and treatments. Consider, for example, the advancements that followed the discoveries of anesthesia, medical imaging, penicillin, organ transplants, HIV treatment and immunotherapy. For Biogen, we believe our expertise and capabilities could lead to the next major inflection point in neuroscience.


Our view is that neurological diseases are deeply connected. As the pathways of disease are interrelated, so are the potential approaches to treating them. Our experience in MS gives our scientists and researchers deeper insights into remyelination and repair, neuroprotection and axonal health, with potential applications in Alzheimer’s disease, Parkinson disease, amyotrophic lateral sclerosis (ALS) and stroke.

 

 

Leading in Alzheimer’s disease

The announcement in October 2019 of our plan to pursue regulatory filing for aducanumab in the U.S. was one of the highlights of our year.


In March 2019, we announced the discontinuation of EMERGE and ENGAGE, our two Phase 3 studies, based on the results of a pre-specified futility analysis that predicted that both studies were unlikely to meet their primary endpoint upon completion. In retrospect, we now know that the result of the futility analysis, based on a smaller and earlier dataset, was incorrect. Following the discontinuation of the studies, additional data from a greater number of patients became available.


A new analysis of this larger dataset, conducted in consultation with the FDA, showed that the Phase 3 EMERGE study met its pre-specified primary and secondary endpoints by showing a significant reduction in clinical decline. And, we believe that results from a subset of patients in the Phase 3 ENGAGE study who received sufficient exposure to high dose aducanumab support the findings from EMERGE, though ENGAGE did not meet its primary endpoint.


Over the past months, we have been actively engaging with the FDA and are working diligently to complete the regulatory filing in the U.S. as soon as possible. We are also actively engaging with regulators in Europe and in Japan based on the positive results of the new findings.


One of our first priorities was to offer eligible patients who had been enrolled in the discontinued aducanumab studies the possibility of restarting the investigational treatment. The first patient in the U.S. returned to dosing in March 2020, and we are also actively working in Europe and Japan to re-open sites.


If approved, aducanumab would become the first therapy to reduce clinical decline in patients at early stages of the disease. While this brings tremendous hope, there remain significant challenges as patients are usually diagnosed late in the progression of the disease. Consequently, we have started working collaboratively with healthcare stakeholders to help support efforts that could enable the system to diagnose patients early enough so they might benefit from potential treatment.


The path for innovation is not straightforward – especially for Alzheimer’s disease research – and aducanumab’s journey has been humbling, fueled by both a drive to address the unmet need and hope. All along, we have worked with determination to follow the science with patients in mind.


We also believe it will take more than one therapy to treat Alzheimer’s disease, so we continue to advance a broad portfolio of potential Alzheimer’s therapies. In March 2019, our collaboration partner Eisai announced the start of CLARITY AD, a Phase 3 study of BAN2401, an anti-amyloid beta antibody co-developed with Biogen to potentially treat patients with early Alzheimer’s disease. In addition, our portfolio includes BIIB080, a tau-targeted antisense oligonucleotide (ASO) in Phase 1; BIIB076, an anti-tau antibody in Phase 1; and gosuranemab (BIIB092), a distinct anti-tau antibody in Phase 2, as well as a number of pre-clinical programs.


In addition to our Alzheimer’s disease pipeline and pre-clinical programs, we have entered into a number of transactions with potential applications in Alzheimer’s disease:

  • In December 2019, we entered into a collaboration with Camp4 Therapeutics, whose platform may bring additional capability in the identification of potential druggable targets for Alzheimer’s disease, among others.
  • In January 2020, we entered into an agreement with Pfizer Inc., which was completed in March, and acquired a Phase 1 asset for the potential treatment of patients with behavioral and neurological symptoms across various psychiatric and neurological diseases, including the treatment of sundowning in Alzheimer’s disease.
  • In February 2020, we announced a global collaboration with Sangamo Therapeutics, Inc., which became effective in April 2020, to develop gene regulation therapies for Alzheimer’s, Parkinson’s, neuromuscular and other neurological diseases.

 

 

 

 

Resilience in MS

In 2019, we remained a global market leader in MS with an approximately 34% market share of the approximately one million MS patients being treated worldwide. Our portfolio ranges from symptomatic treatment to disease-modifying therapies and, with 25 active MS clinical trials, we continue pioneering research across all stages of MS.


In October 2019, together with Alkermes plc, we announced FDA approval for VUMERITY, a novel fumarate treatment for relapsing MS. We are excited about the prospect of this new option for patients. VUMERITY offers the well-characterized efficacy of TECFIDERA, the most prescribed oral medicine for relapsing MS in the U.S., and showed superior patient-reported gastrointestinal tolerability.


We continued to advance the Phase 2b study of opicinumab (anti-LINGO) as a potential remyelinating agent for MS. If successful, opicinumab would represent a first-in-class therapy to potentially repair or restore function in MS patients, an entirely different approach from current disease-modifying therapies.


We recently had two label updates in the European Union to allow, where clinically needed, the use of AVONEX and PLEGRIDY during pregnancy and breastfeeding, and we have several portfolio innovations in progress, such as the evaluation of extended interval dosing with TYSABRI, that we believe are primed to strengthen the business from multiple facets. Looking ahead, our unwavering commitment in MS continues.

 

 

Continued growth and regional expansion in spinal muscular atrophy

SPINRAZA, the first treatment approved for infants, children and adults with spinal muscle atrophy (SMA), continued to grow in the U.S. and even more so outside the U.S. In 2019 full-year SPINRAZA revenues increased 22% from 2018 to $2.1 billion, driven by 9% growth in the U.S. to $933 million and 34% growth outside the U.S. to $1.2 billion.


By the end of 2019 SPINRAZA, was approved in over 50 countries with formal reimbursement in 40 countries, including China. More than 10,000 patients have been treated with SPINRAZA globally, including in clinical studies, the expanded access programs and the post-marketing setting.


Despite progress, a cure has yet to be found for this devastating disease, and our commitment to the SMA community remains unwavering. The results of the NURTURE study in pre-symptomatic infants, presented at the annual Cure SMA meeting in June 2019, showed that treating patients earlier improved outcomes. We are pleased that newborn screening for SMA has increasingly become routine and implemented in 23 states in the U.S. to date. In September 2019, we announced that we plan to initiate DEVOTE, a new Phase 2/3 study evaluating the safety and potential for even greater efficacy of a higher dose of SPINRAZA in the treatment of SMA. The first patient in the study was dosed in March 2020.


SPINRAZA’s success is an example of Biogen’s pioneering science and strong execution capabilities. In less than four years, SPINRAZA has become a foundation of care for SMA, providing life-changing benefits to many patients and turning what was an often-fatal disease for infants with the most severe form of SMA into a potentially survivable condition.

 

 

Double-digit growth in biosimilars

A core part of our strategy is to unlock the potential of biosimilars as a growth driver and as part of our value proposition to support a sustainable healthcare system. Biosimilars are products that have been demonstrated to be similar in efficacy and safety to the originator’s approved biological product, with the advantage that they offer cost savings, providing payers and health systems the budgetary headroom to fund innovation.


In 2019, our biosimilars business grew 35%, generating $738 million in revenues. More than 200,000 patients were treated with our three anti-tumor necrosis factor (anti-TNF) biosimilars, an increase of approximately 70% versus the prior year. Overall, we estimate that our anti-TNF biosimilars have contributed healthcare savings of approximately €1.8 billion in Europe in 2019.


In December 2019, we bolstered our biosimilar business by securing the exclusive rights to commercialize two potential ophthalmology biosimilar products, SB11 referencing LUCENTIS and SB15 referencing EYLEA, in major markets worldwide, including the U.S., Canada, Europe, Japan and Australia.

 

 

The progress of our pipeline reflects our commitment to bringing potentially innovative new therapies to patients and further supports our goal of building a multi-franchise neuroscience portfolio.

— Michel Vounatsos
Chief Executive Officer

 

 

Advancing significant opportunities for value creation

The progress of our pipeline reflects our commitment to bringing potentially innovative new therapies to patients and further supports our goal of building a multi-franchise neuroscience portfolio.


We are pioneers in neuroscience and are not afraid to go where others won’t. Our focus enables us to leverage the interconnectivity in neuroscience and to develop unique asymmetric core capabilities that we believe may increase the probability of success of our pipeline.


We closed 2019 with a pipeline that included 27 clinical programs, of which 6 are in Phase 3, 12 are in Phase 2 and 9 in Phase 1 – as well as a deep pre-clinical pipeline across multiple modalities. We believe that no other company is as well-positioned to develop potentially breakthrough medicines for patients living with devastating neurological and neurodegenerative diseases. Looking forward, we expect multiple mid- to late-stage readouts by the end of 2021.


We have an unwavering commitment to neuromuscular disorders, and we are inspired by the progress of tofersen (BIIB067), an ASO being studied for the potential treatment of a rare form of ALS in adults with a confirmed superoxide dismutase 1 (SOD1) mutation. At the 71st annual meeting of the American Academy of Neurology in May 2019, we presented positive results of a Phase 1/2 study of tofersen, and we have started enrollment of VALOR, a pivotal Phase 3 study. We believe the Phase 1/2 data further demonstrate the potential of targeting genetic drivers of disease.


In December 2019, we announced positive top-line results from the Phase 2 LILAC study evaluating the efficacy and safety of BIIB059 (anti- BDCA2) in patients with lupus. The study results showed that BIIB059, a monoclonal antibody, demonstrated a statistically significant reduction of disease activity in people with cutaneous lupus and systemic lupus erythematosus, as compared to those who received placebo. There are currently only a limited number of treatment options available to help manage these difficult-to-treat and chronic diseases, and we are excited by the prospect to advance BIIB059 to Phase 3.


We continued to further our pipeline in ophthalmology. In March 2019, we acquired Nightstar Therapeutics plc, a clinical-stage gene therapy company. As a result, we added two mid- to late-stage clinical assets, as well as preclinical programs, that focus on adeno-associated virus treatments for inherited retinal disorders that can lead to blindness. Following the acquisition, we completed enrollment of the Phase 3 STAR study of BIIB111 (timrepigene emparvovec) for the potential treatment of choroideremia, a rare, degenerative, X-linked retinal disorder that leads to blindness and currently has no approved treatments. The study is designed to investigate the safety and efficacy of a single subretinal injection of the gene therapy.


While we hope to continue the clinical trials that we have underway, we expect that COVID-19 precautions may impact the timeline of some of our trials.

 

 

Where science meets humanity

We feel a great sense of responsibility in our role as a corporate citizen to make a positive impact both today and in the future. To do this, we must always consider and act on environmental, social and governance (ESG) issues as an integral part of how we do our business, every day.


In 2019, for the fourth time, Biogen was listed as the number one company for the biotechnology industry in the Dow Jones Sustainability World Indices.


Since 2014, Biogen has been carbon neutral, as reflected in our use of 100% renewable electricity and financially supported carbon offset projects. It is clear that more is needed, and we are working to find solutions that align with the recommendations of climate scientists to move beyond carbon offsets. Also, we treat water as a precious commodity – strictly monitoring and looking for ways to reduce use. We continue to actively employ green chemistry processes and techniques to reduce our waste, water and energy consumption.


Our employees are actively involved in our corporate responsibility efforts. In September 2019 more than 3,000 Biogen employees across more than 30 countries volunteered their time and energy for our annual Care Deeply Day. Since 2010 this global day of service has supported more than 100 community-based organizations, science education programs, nutrition and food security and other local needs.


In 2019, we took two major steps in providing greater transparency on how critical decisions are made about access to our medicines. In June, we published our updated Pricing Principles that outline how we determine responsible pricing for our therapies, and in December we published our framework of Access Programs for investigational therapies. Our thinking on these very important access matters has been guided by health equity and affordability while sustaining innovation.


The Biogen Foundation supports our commitment to science, technology, engineering and math education (STEM). In 2019 more than 4,220 students participated in education sessions at our Community Labs in our Cambridge, Massachusetts, and Research Triangle Park, North Carolina, locations. Since our Community Labs began, nearly 55,000 children have been engaged in our hands-on programs, and in 2019 54% of those students came from low-income households and/or groups traditionally underrepresented in science.


Biogen’s ongoing success is rooted in the strength of our diverse people and our inclusive culture. We firmly believe that diverse teams drive better performance. Within Biogen today, we are proud of the fact that 46% of our director-level and above positions are held by women, and in the U.S., 26% of director-level and above roles are held by racial or ethnic minorities. Recently, we have taken important steps in setting goals to ensure diversity in our clinical trial programs. This is part of our commitment to address the needs of the patients we serve. In the U.S. alone, we know that African Americans make up only 5% of clinical trial participants, while Hispanic representation is 1%. This is not sufficient representation, a fact we are working to help change.


In 2020 we will continue to advance our corporate responsibility leadership. Ultimately, we believe that by doing the right thing for our community and the world, we can help build sustainable value for all our stakeholders.

 

 

Our multi-front response to the COVID-19 pandemic

Biogen is engaging on many fronts to respond to the COVID-19 global crisis by focusing our efforts on the following major areas.


Through the Biogen Foundation, we have committed $10 million to support the global response efforts and the immediate needs of communities. Our donations are focused on expanding testing options, easing the strain on medical systems and supporting access to necessities like food.


We have directed employees to work from home and provided support, including financial support, to all Biogen employees and their families worldwide to protect their health and safety and prevent the disease from further spreading.


We have deployed our scientific resources and capabilities, which include equipment and supplies, to help support organizations as they work on the front lines to treat and contain the virus.


We are helping to increase the understanding of COVID-19 and advance research efforts and potential therapeutic options. For example, we have entered into a consortium with the Broad Institute of MIT and Harvard, and Partners HealthCare to create an open COVID-19 biobank. We will provide scientific expertise and enable impacted Biogen employees, as well as close contacts, to donate blood samples and related health data, which will then be analyzed by scientists and researchers and will be openly shared with the global scientific community.


Our teams are mobilized as we work to ensure patients continue to have access to our therapies and are closely monitoring developments and potential impacts on our business. As we’ve moved forward through this crisis, the importance of our work and the vital role our team plays in supplying critical therapies for people living with serious neurological and neurodegenerative diseases has become even clearer.

 

 

Looking to the future

Given the fluidity of the current environment, we anticipate that there may be near-term impacts on our business or operations from the COVID-19 pandemic. However, we believe that we have multiple opportunities for long-term value creation as we continue to build a multi-franchise neuroscience portfolio.


As always, we will remain financially disciplined, continue to drive efficiencies and operate with integrity as we aim to continue to deliver long-term value to our stockholders and society. We believe that neuroscience is at an inflection point, and Biogen is at the forefront. We are hopeful about the prospect of creating new sources of value for our stockholders and continuing to deliver on our purpose to lead in this space as we work to develop new therapies for the betterment of humanity.


None of our accomplishments or our prospects for future success would be possible without the commitment of the people of Biogen, the trust of our stockholders and the support all of our stakeholders – scientists, collaboration partners, healthcare providers, advocacy groups, caregivers and patients.


My sincere thanks and appreciation to all of you. Together, we are tackling some of the most difficult and devastating diseases, and I believe we can have a profound, positive impact on society. We are dedicated to working ethically and compliantly with a passion for science to help deliver innovative therapies for patients and value for our stockholders. At Biogen, we are pioneering science for humanity. Millions are waiting for life-changing therapies, which is why we can’t wait. The time is now.