* SOD1-ALS is a devastating, uniformly fatal, and ultra-rare genetic form of

    ALS estimated to affect less than 1,000 people in Europe(1)
  * QALSODY is Biogen's third rare disease therapy to be approved in the EU,
    demonstrating the company's commitment to addressing diseases with a high
    unmet need
  * With QALSODY, Biogen has helped advance neurofilament as a tool to optimize
    clinical trial design in ALS, offering the potential to expedite further
    breakthroughs in the field

CAMBRIDGE, Mass., May 30, 2024 (GLOBE NEWSWIRE) -- Biogen
(http://www.biogen.com/) Inc. (Nasdaq: BIIB) announced the European Commission
(EC) has granted marketing authorization under exceptional circumstances and
maintained orphan designation for QALSODY(®) (tofersen) for the treatment of
adults with amyotrophic lateral sclerosis (ALS) associated with a mutation in
the superoxide dismutase 1 gene (SOD1-ALS). QALSODY is the first treatment
approved in the European Union to target a genetic cause of ALS, also known as
motor neuron disease (MND).
"The European Commission's approval of QALSODY is a testament to the unwavering
dedication of the ALS community - people living with ALS and their loved ones,
scientists, clinicians, and advocates - who have worked together over the past
two decades to bring forward this important new treatment for the SOD1-ALS
community," said Stephanie Fradette, Pharm.D., Head of the Neuromuscular
Development Unit at Biogen. "We are working with the medical community and local
authorities to bring QALSODY to people living with SOD1-ALS across the region as
quickly as possible."
The marketing authorization for QALSODY is granted under exceptional
circumstances, which is recommended when the benefit/risk assessment of a
treatment is determined to be positive but due to the rarity of the disease, it
is unlikely that comprehensive data can be obtained under normal conditions of
use. The European Medicines Agency (EMA) recommended QALSODY's designation as an
orphan medicinal product be maintained.
"QALSODY's approval represents a paradigm shift in the treatment of SOD1-ALS,
offering hope to patients and loved ones who have long awaited a breakthrough,"
said Philip Van Damme, M.D., Ph.D., Professor of Neurology and Director of the
Neuromuscular Reference Center at the University Hospital Leuven in Belgium.
"The European Academy of Neurology has confirmed new treatment guidelines for
ALS that recognize QALSODY should be offered as first-line treatment for
patients with SOD1-ALS."
The approval of QALSODY is based on the totality of evidence, including the
targeted mechanism of action, biomarker, and clinical data. In the randomized,
double-blind, placebo-controlled Phase 3 VALOR study (n=108), patients were
randomized 2:1 to receive treatment with either QALSODY 100 mg (n=72) or placebo
(n=36) for 24 weeks. The primary efficacy endpoint was the change from baseline
to Week 28 in the ALS Functional Ratings Scale-Revised total score. The results
numerically favored tofersen, but were not statistically significant (ITT
population: tofersen-placebo adjusted mean difference (95% CI): 1.4 (-
1.3, 4.1)). At Week 28, mean plasma neurofilament light chain (NfL), a marker of
axonal injury and neurodegeneration, was reduced by 55% (geometric mean ratio to
baseline) in the tofersen-treated participants (ITT), compared to a 12% increase
with placebo (difference in geometric mean ratios for tofersen to placebo: 60%
(95% CI: 51%, 67%)). Very common adverse reactions (may affect more than 1 in
10 people) reported in QALSODY-treated participants were pain (back pain, pain
in arms or legs), feeling tired, muscle and joint pain, fever, and an increase
in protein and/or white blood cell count occurring in the fluid that surrounds
the brain and spinal cord.
"At EUpALS, we are excited that people with SOD1-ALS in Europe will have access
to QALSODY, the first treatment targeting a genetic cause of ALS. This is a
major milestone for the ALS community, showing that ALS is a treatable disease,"
said Evy Reviers, Chairwoman of the European Organisation for Professionals and
People living with ALS (EUpALS). "As a representative of the European ALS
community, I am excited to enter a new evolution in the common fight against
ALS. We thank Biogen for the many years of scientific and clinical pioneering
efforts that led to this medical success."
Biogen is committed to working closely with all stakeholders to enable access to
this treatment for eligible European patients. Through the Biogen early access
program, about 330 people with SOD1-ALS have received QALSODY across 18 EU
countries. QALSODY is also approved for use in the United States and Biogen is
engaging with regulatory authorities in other regions.
About QALSODY(®) (tofersen)
QALSODY(®) (tofersen) is an antisense oligonucleotide (ASO) designed to bind
to SOD1 mRNA to reduce SOD1 protein production. The U.S. Food and Drug
Administration granted accelerated approval for QALSODY to treat amyotrophic
lateral sclerosis (ALS) in adults who have a mutation in the superoxide
dismutase 1 (SOD1) gene. This indication is approved under accelerated approval
based on reduction in plasma neurofilament light chain (NfL) observed in
patients treated with QALSODY. Continued approval for this indication may be
contingent upon verification of clinical benefit in confirmatory trial(s).(2)
The European Commission granted marketing authorization under exceptional
circumstances and orphan designation for QALSODY.
Biogen licensed QALSODY from Ionis Pharmaceuticals, Inc. under a collaborative
development and license agreement. QALSODY was discovered by Ionis.
In addition to the ongoing open label extension (OLE) of the Phase 3 VALOR
study, QALSODY is being studied in the Phase 3, randomized, placebo-controlled
ATLAS study to evaluate whether QALSODY can delay clinical onset when initiated
in presymptomatic individuals with a SOD1 genetic mutation and biomarker
evidence of disease activity (elevated plasma NfL). More details about ATLAS
(NCT04856982) can be found at clinicaltrials.gov.
(https://www.globenewswire.com/Tracker?data=vZGQA3Iz-TK1B4_39QEg-
MlNGf2qOit7OGLT0WhZfH_Wyn9xb4_0JEDjHYXY7SWUyVxWEXECL6fraG0qjpyMRCYYo_WOYoxBfzEfy
YiCX7OrIfro7Xh_VlNfRLGA7QWx)
About Amyotrophic Lateral Sclerosis and SOD1-ALS
Amyotrophic lateral sclerosis (ALS) is a rare, progressive and fatal
neurodegenerative disease that results in the loss of motor neurons in the brain
and the spinal cord that are responsible for controlling voluntary muscle
movement. People with ALS experience muscle weakness and atrophy, causing them
to lose independence as they steadily lose the ability to move, speak, eat, and
eventually breathe. Average life expectancy for people with ALS is three to five
years from time of symptom onset.(3)
Multiple genes have been implicated in ALS. Genetic testing helps determine if a
person's ALS is associated with a genetic mutation, even in individuals without
a known family history of the disease. Mutations in the SOD1 gene are
responsible for approximately 2% of the estimated 168,000 people who have ALS
globally (SOD1-ALS).(1 )More than 15% of people with ALS are thought to have a
genetic form of the disease; (4) however, they may not have a known family
history of the disease.(1)
In people with SOD1-ALS, mutations in their SOD1 gene cause their bodies to
create a toxic misfolded form of SOD1 protein. This toxic protein causes motor
neurons to degenerate, resulting in progressive muscle weakness, loss of
function, and eventually, death.(4)
Biogen's Continuous Commitment to ALS
For over a decade, Biogen has been committed to advancing ALS research to
provide a deeper understanding of all forms of the disease. The company has
continued to invest in and pioneer research despite making the difficult
decision to discontinue a late-stage ALS asset in 2013. Biogen has applied
important learnings to its portfolio of assets for genetic and other forms of
ALS, with the goal of increasing the probability of bringing a potential therapy
to patients in need. These applied learnings include evaluating genetically
validated targets in defined patient populations, pursuing the most appropriate
modality for each target and employing sensitive clinical endpoints. In addition
to QALSODY, the company has a robust discovery pipeline including efforts to
address TDP43 pathology for the broad ALS population. TDP43 pathology is seen in
97% of ALS cases and is considered a hallmark of the disease.
About Biogen
Founded in 1978, Biogen is a leading biotechnology company that pioneers
innovative science to deliver new medicines to transform patients' lives and to
create value for shareholders and our communities. We apply deep understanding
of human biology and leverage different modalities to advance first-in-class
treatments or therapies that deliver superior outcomes. Our approach is to take
bold risks, balanced with return on investment to deliver long-term growth.
We routinely post information that may be important to investors on our website
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Biogen Safe Harbor
This news release contains forward-looking statements, the potential clinical
effects of QALSODY; the potential benefits, safety and efficacy of QALSODY; the
clinical development program for QALSODY; the identification and treatment of
ALS; our research and development program for the treatment of ALS; the
potential of our commercial business and pipeline programs, including QALSODY;
and risks and uncertainties associated with drug development and
commercialization. These forward-looking statements may be accompanied by words
such as "aim," "anticipate," "believe," "could," "estimate," "expect,"
"forecast," "intend," "may," "plan," "potential," "possible," "will," "would"
and other words and terms of similar meaning. Drug development and
commercialization involve a high degree of risk, and only a small number of
research and development programs result in commercialization of a product.
Results in early-stage clinical trials may not be indicative of full results or
results from later stage or larger scale clinical trials and do not ensure
regulatory approval. You should not place undue reliance on our forward-looking
statements.
These statements involve risks and uncertainties that could cause actual results
to differ materially from those reflected in such statements, including without
limitation, uncertainty of success in the development and potential
commercialization of QALSODY; the risk that we may not fully enroll our clinical
trials or enrollment will take longer than expected; unexpected concerns may
arise from additional data, analysis or results obtained during our clinical
trials; regulatory authorities may require additional information or further
studies, or may fail or refuse to approve or may delay approval of our drug
candidates, including QALSODY; the occurrence of adverse safety events; the
risks of unexpected hurdles, costs or delays; failure to protect and enforce our
data, intellectual property and other proprietary rights and uncertainties
relating to intellectual property claims and challenges; product liability
claims; results of operations and financial condition. The foregoing sets forth
many, but not all, of the factors that could cause actual results to differ from
our expectations in any forward-looking statement. Investors should consider
this cautionary statement, as well as the risk factors identified in our most
recent annual or quarterly report and in other reports we have filed with the
U.S. Securities and Exchange Commission. These statements speak only as of the
date of this news release.
We do not undertake any obligation to publicly update any forward-looking
statements.
References:
1. Brown CA, Lally C, Kupelian V, Flanders WD. Estimated Prevalence and

    Incidence of Amyotrophic Lateral Sclerosis and SOD1 and C9orf72 Genetic
    Variants. Neuroepidemiology. 2021;55(5):342-353. doi: 10.1159/000516752.
    Epub 2021 Jul 9.

2. QALSODY Prescribing Information, Cambridge, MA: Biogen.
3. National Institute of Neurological Disorders and Stroke. Amyotrophic Lateral

    Sclerosis (ALS). Available at: https://www.ninds.nih.gov/health-
    information/disorders/amyotrophic-lateral-sclerosis-als. Accessed: April
    2024.

4. Akcimen F, Lopez ER, Landers JE, et al. Amyotrophic lateral sclerosis:

    translating genetic discoveries into therapies. Nat Rev Genet. 2023.
    https://doi.org/10.1038/s41576-023-00592-y
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