FDA Issues Draft Guidance for ALS Drug Development


Agency credits ALS Association proposed guidance, which was funded by Ice Bucket Challenge funds

Washington, D.C. (February 15, 2018) – The ALS Association applauded the Food and Drug Association’s (FDA) announcement Thursday of its proposed industry guidance for the development of drugs to treat ALS. The FDA’s draft guidance is informed by The ALS Association’s first patient-focused guidance for ALS drug development that was submitted to the FDA in late 2017 and that was funded with donations from the ALS Ice Bucket Challenge.

“Despite the availability of some approved therapies, there is an urgent need to identify additional effective treatments for patients with ALS. We’ve been honored to work with the ALS Association to advance these goals. The ALS Association put together a comprehensive proposed draft guidance of their own, funded by the famous ‘ice bucket challenge,’” FDA Commissioner Scott Gottlieb said in a memo released Thursday.

The FDA guidance and the ALS patient-focused guidance complement each other and will work in tandem to provide a roadmap to help researchers in academic institutions and pharmaceutical companies navigate the drug development process. The goal of both these documents is to increase the efficiency, predictability and speed of the drug development process, including clinical trials, and lead to a more effective and earlier assessment of efficacy.

“We want to commend the FDA for moving forward with the first-ever guidance on drug development for people with ALS,” said Calaneet Balas, president and CEO of The ALS Association. “The FDA guidance and the patient-focused guidance both have the potential to speed up the process of drug development, reduce costs, and create incentives for the industry to enter the ALS market and develop new treatments.”

The ALS Association will be submitting comments on the FDA’s draft guidance and encourages others to do so, as well. The proposed guidance will be available for public comment for 60 days after it is placed in the public register. The final FDA guidance could change from the proposal to reflect feedback from the public. The proposed FDA guidance has not been officially published, but it can be viewed here.

The ALS Association worked with more than 100 participants, including industry, clinicians and researchers, the National Institutes of Health, the Centers for Disease Control and Prevention, and people with ALS and their families to develop the proposed patient-focused guidance.

“We are grateful to the many stakeholders who helped draft the patient-focused guidance document, especially people living with ALS,” Balas said. “We also appreciate the commitment of FDA leadership, including Dr. Scott Gottlieb, Commissioner of the Food and Drugs Administration; Janet Woodcock, Director of the Center for Drug Evaluation and Research and Dr. Billy Dunn, Director, Office of Drug Evaluation Division for Neurology Products. We will continue to push and support the FDA to expand its array of projects to accelerate therapies to people with ALS to meet the urgent need for ALS treatments and a cure.

“This is an important step, but it’s critical that our advocates and allies remain engaged during the next few weeks and weigh in during the public comment period to make sure that the community keeps driving this process forward,” Balas added.

ALS Ice Bucket Challenge Year-End Update: Over $94 Million in Commitments Since 2014

In the summer of 2014, the ALS Ice Bucket Challenge inspired 17 million people to upload videos and raised $115 million for The ALS Association. Since then, The ALS Association has committed more than $94 million toward its mission, including over $82 million to fund research and helped forge nine new global research collaborations.

“The Ice Bucket Challenge was a global phenomenon, thanks to the generosity of millions of people, resulting in a tremendous amount of awareness of the disease and spurring a huge increase in our research spending,” said Calaneet Balas, president and CEO of The ALS Association. “Since 2014, we’ve committed over $82 million to fund diverse, cutting-edge research, including over 150 projects in the last year alone. By its nature and design, research requires time, and people with ALS don’t have a lot of time. But we’ve seen exciting breakthroughs from the research we fund – including some that were considered high-risk.”

ALS Ice Bucket Challenge donations have helped lead to the discovery of four new ALS genes, including NEK1, one of the most common. In addition, scientists who are a part of the Neuro Collaborative, which was started with an initial investment of $5 million from ALS Ice Bucket donations, have developed antisense drugs targeting two of the most common ALS genes, C9orf72 and SOD1. Our investment in this innovative technology also translates to other neurodegenerative diseases, making the impact of Ice Bucket donations even greater.

ALS Ice Bucket Challenge donations have also helped spur advancements in assistive technology to develop flexible, accessible devices to help people living with ALS better communicate to improve their quality of life. ALS Ice Bucket Challenge donations have also helped us increase the number of Certified and Recognized Treatment Centers of Excellence and the size of the grants to those centers. Prior to the Ice Bucket Challenge, we 34 Certified Treatment Centers of Excellence and there were no plans to increase that number. Today, we have 58 Certified centers, in addition to 20 Recognized Treatment Centers. (The primary difference between Certified and Recognized Treatment Centers is that Certified Centers conduct ALS research.)

To see our research accomplishments and highlights for the past year (2017), please click here. To see a specific list of all the projects and project descriptions we have funded since the Ice Bucket Challenge, please click here.

“We fight for treatments and a cure for ALS every single day,” Balas said. “We are the largest private funder of ALS research and are currently funding research projects in eight countries. We are also helping ensure people with ALS receive multidisciplinary care that we know enhances and extends life. And we’re amplifying the voices of more than 16,000 ALS advocates to push for better public policies that can help us fight for treatments and a cure.”

The ALS Association is committed to transparency in how donor dollars are fueling efforts to find treatments and a cure for the disease. And we’re committed to maximizing all donations from the ALS Ice Bucket Challenge and beyond.

Research News: Largest Ever Study of Inherited ALS Identifies New ALS Gene NEK1

Today researchers from Project MinE, a large, international ‘big data’ initiative funded by The ALS Association through ALS Ice Bucket Challenge donations*, shared the exciting news that they have identified a new gene, NEK1, that ranks among the most common genes that contribute to ALS.

It is known that 10 percent of ALS cases are familial, meaning genes are inherited from a family member. The other 90 percent of ALS cases are sporadic, or without a family history. It is very likely that genetics contribute, directly or indirectly, to a much larger percentage of ALS cases. The discovery of NEK1 gives scientists an exciting new target for drug development.

About NEK1

From previous studies, we know that NEK1 functions in multiple roles in neurons (i.e. cells of the brain) including maintaining the neuron’s cytoskeleton, which gives it its shape and promotes transport of molecules within it. It also has roles in regulating the membrane of the mitochondrion, which is the machine that supplies energy to neurons and helps repair DNA that was damaged within. Importantly, all of these cell functions have been found to contribute to ALS in some way and represent potential drug targets. With today’s announcement, we can confirm the association between mutations in NEK1 and ALS.

Project MinE

Project MinE was founded in 2011 by Robbert Jan Stuit and Bernard Muller, two entrepreneurs living with ALS. As of today, Project MinE has achieved 35 percent of its goal to sequence the genomes of 22,500 people – 15,000 people living with ALS and 7,500 people without the disease. It is the first and largest genome sequencing effort to date,  involving 16 countries in a collaborative effort to discover new genes. The concept behind Project MinE is simple: donations contribute directly to DNA sequencing. For example, a donation of $2000 allows for one person’s whole genome to be sequenced.

In the wake of the ALS Ice Bucket Challenge in 2014, The ALS Association proudly contributed $1 million dollars to Project MinE to help spur the U.S. arm of this global initiative, led by Dr. John Landers at University of Massachusetts Medical School and Dr. Jonathan Glass at Emory University. The ALS Ice Bucket Challenge enabled The ALS Association to invest in Project MinE’s work to create large biorepositories to store ALS biosamples that are designed to allow exactly this kind of research and to produce exactly this kind of result.

Next Steps

The discovery of NEK1 highlights the value of ‘big data’ in ALS research. The sophisticated gene analysis that led to this finding is only possible because of the large number of ALS samples collected and made available by researchers by Project MinE. In fact, this discovery was a combined effort of over 80 researchers in 11 countries, including the U.S.

Now the next steps are to understand the role of NEK1 in ALS disease. Researchers funded by The ALS Association – using your generous donations – are already making steps towards this by developing novel NEK1 mouse models that will be shared with the entire ALS community. Sharing information is essential to drug discovery in that multiple researchers can all research NEK1 at the same time to uncover potential drug targets.

The discovery of NEK1 through Project MinE’s large, global collaborative effort is a perfect example of how every drop adds up. With this successful ‘big data’ model in hand, be sure to look out for more gene discoveries out of Project MinE in the near future. Together, we will find a treatment and a cure for ALS.

— Lucie Bruijn, Ph.D., M.B.A., Chief Scientist of The ALS Association.

*Funding for Project MinE also came through The ALS Association Georgia Chapter, The ALS Association Greater New York Chapter and New Amsterdam City Swim.


Neuro Collaborative Partner Gladstone Institutes Forms New Collaboration with Biogen In ALS Research

research news banner 2015

The ALS Association is pleased to announce a new collaboration between the Gladstone Institutes in San Francisco, which is a member of The Association-funded Neuro Collaborative and the biotechnology company Biogen in Cambridge, Mass., to discover novel drug targets for the treatment of amyotrophic lateral sclerosis (ALS).

The Neuro Collaborative is an innovative partnership among three California research labs focused on development of new ALS therapies: The Svendsen lab at Cedars-Sinai in Los Angeles, site of the groups’ stem cell core; the Cleveland lab at the University of California San Diego, engaged in development of novel therapeutics; and the Finkbeiner lab at the Gladstone Institutes, which is affiliated with the University of California at San Francisco.

The Neuro Collaborative is funded by The ALS Association with a $5 million grant stemming from the funds raised through the Ice Bucket Challenge. Part of the Neuro Collaborative’s mission is to develop potential therapies that can be delivered to pharmaceutical companies for clinical trials.

Gladstone Senior Investigator Steven Finkbeiner, MD, Ph.D. and his group, invented and use a novel robotic microscopy system, called “Brain Bot,” which allows longitudinal imaging of individual neurons in culture, making target identification and drug discovery faster and more efficient. The lab has identified protein homeostasis as an important factor underlying neurodegeneration. To this end, they are leveraging their microscopy system to develop novel small molecule therapeutics that can clear toxic proteins from neurons and extend their lifespan. In addition, the lab is carrying out screens in search of novel drug targets that could be used to design new therapeutics that mitigate neurodegeneration.

Biogen has carried out genetic screens for genes that affect Drosophila ALS models and identified numerous potential targets that improve ALS-related phenotypes in these models. To evaluate and further validate potential targets for the development of new medicines, Biogen is interested in carrying out secondary screens in mammalian neurons. The new collaboration between the Finkbeiner lab and Biogen will greatly accelerate this effort.

“We are deeply gratified to see the emergence of this new collaboration,” said Lucie Bruijn, Ph.D., M.B.A., Chief Scientist for The ALS Association. “We believe the fastest route to new therapies for ALS is through forging partnerships such as this one, which combines the expertise of a major research laboratory with that of a major biotechnology company, both of which have proven their ability and commitment to advancing the discovery of new ALS treatments.”

“This collaboration offers us the opportunity to bring our state-of-the-art imaging technology fully to bear on the critical challenge for discovery of new ALS drug targets,” Dr. Finkbeiner said.

ALS is a significant focus of drug discovery and development at Biogen, a research-driven biotechnology company that has developed multiple medicines for neurologic conditions. This collaboration is expected to lead to better understanding of disease mechanisms, identification of new drug targets, and accelerate development of new treatments for ALS.

“Biogen is committed to ALS research and collaborating with the world’s best scientists to tackle this devastating disease,” said Spyros Artavanis-Tsakonas, Ph.D., Chief Scientific Officer at Biogen. “We are excited to combine our capabilities in pathway discovery and target validation with the screening technologies and imaging expertise of the Gladstone Institutes’ Neuro Collaborative to find new answers to ALS.”

The Gladstone-Biogen collaboration is scheduled to start in the first quarter of 2016 and will be led by Dr. Finkbeiner and Dr. Ashkan Javaherian at the Gladstone Institutes along with Dr. Mark Kankel and Dr. Anindya Sen from the Pathway Discovery research group at Biogen. The collaboration will include developing novel cellular models of ALS and building an infrastructure for high-throughput screening to identify new drug targets and develop small molecule therapeutics for ALS.

About ALS
ALS is a progressive neurodegenerative disease that affects nerve cells in the brain and the spinal cord. Eventually, people with ALS lose the ability to initiate and control muscle movement, which often leads to total paralysis and death within two to five years of diagnosis. For unknown reasons, veterans are twice as likely to develop ALS as the general population. There is no cure, and only one drug approved by the U.S. Food and Drug Administration (FDA) modestly extends survival.

About the Gladstone Institutes
To ensure our work does the greatest good, the Gladstone Institutes focuses on conditions with profound medical, economic, and social impact—unsolved diseases of the brain, the heart, and the immune system. Affiliated with the University of California, San Francisco, Gladstone is an independent, nonprofit life science research organization that uses visionary science and technology to overcome disease. Find more information at gladstone.org.

About Biogen
Through cutting-edge science and medicine, Biogen discovers, develops and delivers to patients worldwide innovative therapies for the treatment of neurodegenerative diseases, hematologic conditions and autoimmune disorders. Founded in 1978, Biogen is one of the world’s oldest independent biotechnology companies and patients worldwide benefit from its leading multiple sclerosis and innovative hemophilia therapies. For product labeling, press releases and additional information about the company, please visit www.biogen.com.

Garden City High School Students Raise Over $6K For Greater New York Chapter

Check-Present_1000pxWhen Katherine Squires’ Garden City High School Economics class decided to hold a fundraiser their principal, Mrs. McLaughlin made them a promise. If they could raise $500, then she would take the ALS Ice Bucket Challenge herself.

The small class of five students had decided to raise money for the ALS Association Greater New York Chapter because one student’s mother has ALS, also known as Lou Gehrig’s Disease. When all was said and done, the kids had raised a combined $6,394.17 through a bake sale and an Ice Bucket Challenge of their own.

Their principal made good on her promise, taking the Challenge during school to the delight of the students. We congratulate Katherine and her students for this accomplishment and for their principal for keeping her word to take the ALS Ice Bucket Challenge.

Long Island Man Takes ALS Ice Bucket Challenge Every Day in August For His Dad

brianWhen Brian Poulos was a kid growing up on Long Island the only person he ever heard of who had Lou Gehrig’s Disease was the famous Yankee baseball player himself. “Like everyone else I thought of it as a very rare disease,” Brian said. That was until his own father, Nicholas, a retired school teacher and Vietnam Veteran, started showing symptoms in 2009. His diagnosis of ALS was confirmed at the beginning of last summer, just a month before the ALS Ice Bucket Challenge went viral. Now it seems that everyone in Brian’s social network knows about Lou Gehrig’s Disease or has some connection that he never knew about before.

Brian, a 38-year-old New York State Trooper, and his wife, Amy, Captain of their Walk to Defeat ALS team called “Pa’s PALS,” vowed to do something this year to help raise more money to fight ALS and support the Greater New York Chapter’s research and patient care programs. While watching a presentation at the kick-off event for the Long Island Walk to Defeat ALS this July an idea popped into Brian’s head. He had taken the ALS Ice Bucket Challenge in 2014 like millions of others and he learned from the Chapter’s events staff that it was coming back again. This year he wanted to do something even bigger. He was afraid that his network on social media might miss the banners and posts about joining them in their fight against ALS. To ensure people got the message he decided that he was going to take the ALS Ice Bucket Challenge every day in the month of August. “I wanted to do something to make it tougher to ignore,” he said. “I thought if I do the challenge every day eventually they will click on it and say to themselves, ‘What is this guy up to?’”

Along with Amy, Brian recruited his brother and sister-in-law to help him come up with a plan. They made a list of places Brian could take his ALS Ice Bucket Challenges to increase awareness and create an event-like atmosphere around them. Every one of his challenges is posted on his YouTube channel and shared on Twitter and Facebook. Donations are directed to their Walk Team, “Pa’s PALS” at www.Paspals.com.

Amy helps him set up and film the challenges and he has his nephews follow him to many of the different locations coming along for the ride on his adventure. The effort has picked up steam since his first challenge on August 1st with locations as diverse as the Setauket Fire Department and with the Long Island Ducks mascot “Quacker Jack” on Long Island to the streets of New York City. “I had my 17- and 15-year-old nephews in Times Square at 11pm filming,” he said. “I was the craziest person in Times Square for a little while.” He plans on taking the challenge at a big event at Heritage Park in Mount Sinai, NY and he was approached by News 12 for a story about his effort.

His father Nicholas, who attends the ALSA Certified Center at Stony Brook University Hospital is excited about the project. “He says ‘thank you’ after every one,” Brian said. “And he joins me on as many as he can.” One particular Challenge that Nicholas did with his son was at a Jimmy Buffet concert at Jones Beach where he was also joined by PALS Paul Weisman.

Cindy Keyser-Posner, LMSW, Patient Services Coordinator on Long Island said Nicholas and his wife, Pat, have also come to the Stony Brook Support Group that is held on the first Tuesday of every month. “A very nice family all around and what a terrific thing that their son is taking the challenge every day,” said Cindy.

Since that initial thought, Brian’s idea has taken on a life of its own and people give him suggestions on where to take the Challenge next, even setting them up for him so everything is prepared when he arrives.

The plan worked. So far they have raised almost $7,000 for their Walk to Defeat ALS Team and Brian says he has gotten donations from people on Facebook that he hasn’t seen in years, old high-school friends and people he knows from across the country. “It’s been overwhelming,” he said of his adventure so far. “I’ve met so many people I would never have met. It was something I almost didn’t do. At first, I thought, ‘how am I going to fill up 31 days of this?’ I am happy I did.”

You can watch Brian take the ALS Ice Bucket Challenge everyday in August on his YouTube Channel here and donate to his Long Island Walk to Defeat ALS Team “Pa’s PALS” here. Also, follow Brian on Twitter here.

ALS Ice Bucket Challenge Fuels Research Discovery

Researchers recently announced a significant research discovery expedited by ALS Ice Bucket Challenge donations.

Researchers supported by The ALS Association identified a previously unknown function of an important protein related to amyotrophic lateral sclerosis (ALS). The study, which showed that TDP-43 regulates so-called “cryptic exons,” was published in the journal Science.

The TDP-43 protein is known to bind to and regulate RNA, the cellular messenger that converts genetic instructions into proteins. Most cases of ALS are characterized by accumulation of TDP-43 into clumps, or aggregates, which may indicate the protein is no longer able to carry out its normal function.

In the current study, Association-supported researchers Jonathan Ling, Ph.D., Philip Wong, Ph.D., and colleagues at Johns Hopkins University in Baltimore, MD, showed that a specific function of TDP-43 is to bind to and repress cryptic exons, bits of genetic instruction that are not used to make protein under normal circumstances. The researchers showed that loss of TDP-43 increased the expression of cryptic exons, leading the cell to destroy messenger RNA containing them, thus reducing the amount of normal protein the cell made, and leading to cell death. They also found an abnormal increase in cryptic exon expression in the brains of people with ALS.

In a recent “Ask Me Anything” (AMA) feature on Reddit, Dr. Ling commented: “All of your donations have been amazingly helpful and we have been working tirelessly to find a cure. With the amount of money that the ice bucket challenge raised, I feel that there’s a lot of hope and optimism now for real, meaningful therapies. After all, the best medicines come from a full understanding of a disease and without the financial stability to do high risk, high reward research, none of this would be possible!”

Watch Jonathan Ling, Ph.D. and Philip Wong, Ph.D. take the ALS Ice Bucket Challenge:

In the wake of announcing a major discovery about the cause of ALS, and a potential treatment strategy, professor Philip Wong and graduate student Jonathan Ling participate in the 2015 Ice Bucket Challenge. Learn more about their discovery here and here.

“This intriguing result provides a new understanding of the normal role of TDP-43,” said ALS Association Chief Scientist Lucie Bruijn, Ph.D., M.B.A. “Because TDP-43 accumulates in most cases of ALS, it is critical that we learn more about its normal functions, and how they are affected in ALS. Replacing those lost functions may be a therapeutic strategy.”

Last year, more than 17 million ALS Ice Bucket Challenge videos were posted to Facebook and these videos were watched by 440 million people a total of 10 billion times. More than $220 million was raised globally with $115 million going to The ALS Association, which is putting these dollars to work funding exciting new initiatives to find treatments and a cure for the disease through research, care services and public policy.

The ALS Ice Bucket Challenge is happening again this August and every August until there is a cure. For more information, please click here.

Biogen, The ALS Association and Columbia University Medical Center Collaborate to Drive Understanding of Genetic Influence in ALS

research news banner 2015CAMBRIDGE, Mass. – Biogen (NASDAQ: BIIB), The ALS Association and Columbia University Medical Center (CUMC) today announced a new collaboration to better understand the differences and commonalities in the ALS disease process and how genes influence the clinical features of the disease. The project, “Genomic Translation for ALS Clinical care” (GTAC), will involve a combination of next generation genetic sequencing and detailed clinical phenotyping in 1500 people with ALS. The goal of the project is to provide a basis for the development of precision medicine, or more individually tailored therapies for ALS.

“We want to bring genomics right to the point of care in ALS where instead of focusing on retrospective DNA samples with limited clinical information, we focus on patients who are under active clinical management,” said ALS Association Chief Scientist Lucie Bruijn, Ph.D., M.B.A. “By focusing on patients seen by participating ALS clinics, this project will allow investigators to ask how different genetic causes of ALS translate into different clinical consequences.”

An explicit aim of the collaboration is to set the stage for a nationwide effort to ensure the genomic characterization of all patients with ALS.

“We know that ALS is not just one disease,” explained Tim Harris, senior vice president, Precision Medicine at Biogen. “This study will help in developing a detailed understanding of how different genes contribute to different clinical forms of ALS. This will in turn help us design better, more focused clinical trials for the development of more effective treatments. This kind of ‘precision medicine,’ in which a treatment is tailored to a person’s unique genetic make-up, is already being used in the cancer field. It is an approach we feel is ready for ALS too.”

“Until recently, most large-scale genomics studies used archived DNA samples, so the findings had minimal impact on patient care,” said David B. Goldstein, Ph.D., professor of genetics and development and director of the Institute for Genomic Medicine at CUMC.  “This project reflects our commitment to using ALS genomic studies to benefit patients directly through diagnoses and to set the stage for genetically stratified clinical trials.”

“This project will provide a clinical deliverable to the 1500 patients that participate in the study.  We will use our extensive database of ALS genomes and exomes to carefully identify definitive genetic risk factors for ALS and these risk factors will be communicated back to participating clinics. The database we create will allow for an unprecedented investigation of the clinical correlates of the genetic causes of ALS.” said Dr. Matthew Harms, M.D., who will lead the project and be joining CUMC in the fall as an assistant professor of neurology.

Patient blood cells will be stored at the Induced Pluripotent Stem Cell (iPSC) Core, a facility supported by the ALS Association, at the Cedars-Sinai Board of Governors Regenerative Medicine Institute. This cell bank will allow researchers to create cell lines for further study, based on leads provided by genome sequencing.

“The ability to create patient iPS cells from such a genetically well-annotated ALS blood repository will allow us to model causes of motor neuron degeneration in ALS at a scale that has never been possible,” said Dhruv Sareen, Ph.D., who leads the Cedars-Sinai iPSC Core.

Clinical data will be collected and curated through the NeuroBank system at the Massachusetts General Hospital, and cell lines will be developed at Cedars-Sinai in Los Angeles. Currently, participating clinical sites in this effort include the Cedars-Sinai Board of Governors Regenerative Medicine Institute, Columbia University Medical Center, Duke Medical Center, Houston Methodist, the Scotland ALS clinic network, University of Minnesota and Hennepin County Medical Center, University of Utah, University of Washington, and Washington University in St. Louis.

The project is being funded through Biogen’s $30 million strategic alliance with CUMC and $3.5 million from The ALS Association. The ALS Association’s commitment comes from funds raised directly through the Ice Bucket Challenge.

About ALS

ALS (amyotrophic lateral sclerosis) is a progressive neurodegenerative disease that affects neurons (nerve cells) in the brain and the spinal cord. Eventually, people with ALS lose the ability to initiate and control muscle movement, which often leads to total paralysis and death within two to five years of diagnosis. There is no cure and no life-prolonging treatments for the disease.

About the ALS Ice Bucket Challenge

In 2014, $115 million was donated to The ALS Association. This incredible generosity is fueling some exciting new initiatives including this new project, “Genomic Translation for ALS Clinical care” (GTAC). A list of  projects that have been made possible by The ALS Ice Bucket Challenge can be found here.

About The ALS Association

The ALS Association is the only national non-profit organization fighting Lou Gehrig’s Disease on every front.  By leading the way in global research, providing assistance for people with ALS through a nationwide network of chapters, coordinating multidisciplinary care through certified clinical care centers, and fostering government partnerships, The Association builds hope and enhances quality of life while aggressively searching for new treatments and a cure.  For more information about The ALS Association, visit our website at www.alsa.org.

About Biogen

Through cutting-edge science and medicine, Biogen discovers, develops and delivers to patients worldwide innovative therapies for the treatment of neurodegenerative diseases, hematologic conditions and autoimmune disorders. Founded in 1978, Biogen is one of the world’s oldest independent biotechnology companies and patients worldwide benefit from its leading multiple sclerosis and innovative hemophilia therapies. For product labeling, press releases and additional information about the company, please visit www.biogen.com.

About Columbia University Medical Center

Columbia University Medical Center provides international leadership in basic, preclinical, and clinical research; medical and health sciences education; and patient care. The medical center trains future leaders and includes the dedicated work of many physicians, scientists, public health professionals, dentists, and nurses at the College of Physicians and Surgeons, the Mailman School of Public Health, the College of Dental Medicine, the School of Nursing, the biomedical departments of the Graduate School of Arts and Sciences, and allied research centers and institutions. Columbia University Medical Center is home to the largest medical research enterprise in New York City and State and one of the largest faculty medical practices in the Northeast. For more information, visit cumc.columbia.edu orcolumbiadoctors.org.

Biogen Safe Harbor Statement

This press release contains forward-looking statements, including statements about the potential to discover and develop new therapeutics for ALS through its collaboration with the ALS Association and other collaborating institutions mentioned above. These forward-looking statements may be accompanied by such words as “anticipate,” “believe,” “estimate,” “expect,” “forecast,” “intend,” “may,” “plan,” “will” and other words and terms of similar meaning. You should not place undue reliance on these statements. These statements involve risks and uncertainties that could cause actual results to differ materially from those reflected in such statements, including risks and uncertainties associated with drug development and commercialization, Biogen’s dependence on third parties over which it may not always have full control, and the other risks and uncertainties that are described in the Risk Factors section of Biogen’s most recent annual or quarterly report filed with the Securities and Exchange Commission. These statements are based on current beliefs and expectations and speak only as of the date of this press release. Biogen does not undertake any obligation to publicly update any forward-looking statements.