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Amylyx Pharmaceuticals Announces First Patients Enroll in Open Label Extension of CENTAUR Phase 2 Clinical Trial of AMX0035 for Treatment of Amyotrophic Lateral Sclerosis


Amylyx Pharmaceuticals, Inc., in collaboration with The ALS Association,
ALS Finding a Cure®, the Northeast ALS Consortium, and the Massachusetts
General Hospital Neurological Clinical Research Institute, today
announced that the first patients to complete the CENTAUR phase II
clinical trial have elected to continue treatment in an open label
extension (OLE) study of AMX0035. All patients who complete the main
phase of the study will be eligible for this extension and will receive
AMX0035 with no placebo comparator.

"The CENTAUR-OLE study provides patients with the option of continuing
treatment once they have completed the 24-week study period," said
Sabrina Paganoni, M.D., Ph.D., Principal Investigator for the study and
physician at the Massachusetts General Hospital and Spaulding
Rehabilitation Hospital. "The study will provide data on AMX0035's
long-term safety and tolerability as well as additional insights into
its efficacy. There is a significant and urgent need for therapies for
ALS, as there are very limited treatment options."

The CENTAUR ("Combination of Phenylbutyrate
and Tauroursodeoxycholic Acid") trial,
which began dosing patients in July 2017, is a randomized, double-blind,
placebo-controlled Phase II clinical trial enrolling 132 ALS patients
nationwide. The trial's primary objectives are to evaluate the safety
and tolerability of AMX0035 and to assess the drug's impact on disease
progression as measured by the revised Amyotrophic Lateral Sclerosis
Functional Rating Scale (ALSFRS-R) over the 24-week study period. The
CENTAUR trial is also the first drug trial to measure patient muscle
strength using an innovative device called ATLIS ("Accurate Test of Limb
Isometric Strength"). Additionally, the trial will include assessments
of promising blood and imaging based biological markers of ALS to
confirm target engagement.

Amylyx CEO and co-founder, Joshua Cohen commented, "We are excited to
offer this extension to the participants in this study and are so
grateful for their commitment." Justin Klee, President and co-founder
added, "Thank you also to The ALS Association, ALS Finding a Cure, the
Northeast ALS Consortium, the Neurological Clinical Research Institute
at MGH, and the ALS community for their outstanding support in helping
us offer this open label extension."

AMX0035 is an oral combination of two small molecules, sodium
phenylbutyrate (PB) and tauroursodeoxycholic acid (TUDCA). Each compound
has shown efficacy in several cellular and animal models of ALS. When
individually tested in ALS clinical trials, PB and TUDCA have both met
endpoints of safety, tolerability, and demonstrated preliminary signs of
efficacy. In preclinical trials, Amylyx has demonstrated a synergistic
effect between the two compounds, suggesting that the combination may
have improved efficacy compared to the individual agents.

More information on the CENTAUR trial can
be found at
NCT03127514. Please contact (617-724-7210), or email Emily Engel at,
if you are interested in participating. Contact information for trial
sites is also available on

Background on ALS
Amyotrophic lateral sclerosis, ALS, is the
most prevalent adult-onset progressive motor neuron disease, affecting
more than 20,000 people in the U.S. and an estimated 450,000 people
worldwide. ALS causes the progressive degeneration of motor neurons,
resulting in rapidly progressing muscle weakness and atrophy that
eventually leads to partial or total paralysis; on average, the disease
is fatal within just three years from onset. There are currently just
three therapies approved specifically for treating ALS—riluzole and the
recently-approved edaravone.

About MGH Neurological Clinical Research Institute (NCRI)
in 1994 at the heart of Massachusetts General Hospital (MGH), the
mission of the NCRI (
is to accelerate translational research in neurological disorders
through initiating and testing novel therapies. The NCRI is a premier
academic Contract Research Organization (CRO) that leverages the vast
disease knowledge of extraordinary physician-investigators to provide
high quality clinical trials design and conduct of multicenter
neurological trials. The NCRI serves as both the Coordinating Center and
a clinical research site for the Northeast ALS Consortium (NEALS) (
a network of ALS specialists at more than 100 locations worldwide.

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

About ALS Finding a Cure®
ALS Finding A Cure® is dedicated
to being a game changer in discovering a cure for this fatal disease.
Founded in 2014 by noted philanthropist and Conair Founder and Chairman,
Leandro (Lee) Rizzuto, as a tribute to Christie Rizzuto, Lee's
daughter-in-law who was diagnosed with ALS in 2009 at the age of 41, and
under the leadership of Peter N. Foss and a team of renowned ALS
researchers, ALS Finding A Cure® is focused on identifying the gaps in
the scientific understanding of ALS that are preventing the development
of a cure. The organization, a program of The Leandro P. Rizzuto
Foundation, collaborates with a wide range of companies, ALS
organizations, other disease research organizations, and ALS patients
and families to ensure research efforts are non-duplicative,
synergistic, and focused on the ultimate goal: finding a cure. For more
information about ALS Finding a Cure®, visit

About Amylyx Pharmaceuticals
Amylyx Pharmaceuticals, Inc. is
a clinical-stage pharmaceutical company currently developing a novel
therapeutic for Amyotrophic Lateral Sclerosis (ALS) and other
neurodegenerative diseases. The company's therapeutic, AMX0035, targets
the neuroinflammation and nerve cell death that characterize these
diseases. AMX0035 is a proprietary combination of existing compounds
that have worked synergistically to prevent cell death and neurotoxic
inflammation in multiple preclinical models. AMX0035 entered a Phase 2
clinical trial (CENTAUR) in ALS patients in June 2017. Learn more at:

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