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Amylyx Pharmaceuticals Announces First Patients Dosed in PEGASUS Phase 2 Trial of AMX0035 in Alzheimer’s Disease and Expansion of the Trial

April 3, 2019 Off By BusinessWire

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Amylyx Pharmaceuticals, Inc., in collaboration with the Alzheimer’s
Association, the Alzheimer’s Drug Discovery Foundation (ADDF) and the
Cure Alzheimer’s Fund, today announced the dosing of its first patients
in a recently expanded Phase 2 clinical trial (PEGASUS) to assess
AMX0035 in individuals with Alzheimer’s disease. AMX0035 is Amylyx’s
proprietary two-drug combination therapy in development to prevent nerve
cell death and degeneration.

“The urgent need for a new approach to Alzheimer’s is clearer now than
ever. As this is one of the first major combination therapy clinical
trials for Alzheimer’s Disease, we’re optimistic about AMX0035’s
potential to slow disease progression in individuals through its novel
mechanisms of action,” said Steven E. Arnold, M.D., Translational
Neurology Head of the Interdisciplinary Brain Center at Massachusetts
General Hospital and Harvard Medical School and the study’s principal
investigator.

Alzheimer’s is a complex disease that likely has multiple, interrelated
causes. A growing number of experts believe combination therapy—with
multiple therapeutics—will be required to effectively treat it.

“Although targeting the classic pathologies of Alzheimer’s disease,
beta-amyloid and tau, has proven challenging in clinical trials, this
research has awarded us with a wealth of knowledge in our understanding
of the disease,” said Rudolph Tanzi, Ph.D., of Massachusetts General
Hospital, chair of the Cure Alzheimer’s Fund Research Leadership Group
and chair of the Amylyx SAB. “Cure Alzheimer’s Fund is proud to take
initiative and provide additional funding to expand Amylyx’s trial to
support the development of novel approaches that patients need,” Dr.
Tanzi added.

With additional funding from the Cure’s Alzheimer’s Fund, the PEGASUS
trial has doubled its planned enrollment to 100 participants, with 20
patients enrolled and dosed to date. The trial was first supported in
2017 through the Alzheimer’s Combination Therapy Opportunities (ACTO)
program, a joint research funding initiative created by the Alzheimer’s
Association and the ADDF to support clinical trials combining multiple
treatment approaches.

“There have been trials targeting amyloid, and there have been trials
targeting tau, but this is the first trial to target two novel
mechanisms in tandem,” said Howard Fillit, M.D., the ADDF’s founding
executive director and chief science officer. “The ADDF was one of the
earliest supporters of innovative targets for Alzheimer’s, and we
believe combination therapies are a critical next step in finding
effective treatments for the disease.”

“The rationale for combination therapies has already been demonstrated
in diseases like HIV/AIDS, cancer and heart disease. We believe this
type of approach will also work for Alzheimer’s and other dementias and
are encouraged by the launch of this combination therapy trial,” said
Maria C. Carrillo, Ph.D., Alzheimer’s Association’s chief science
officer. “The Alzheimer’s Association is honored to be at the forefront
of the mission to keep expanding the research pipeline for therapies
that attack this complex disease from a variety of angles.”

PEGASUS is a 3:2 randomized, double-blind, multi-center,
placebo-controlled study evaluating the safety, tolerability and
activity of AMX0035 in patients with late mild cognitive impairment or
early dementia due to Alzheimer’s disease over 24 weeks. The
biomarker-focused trial design will assist in demonstrating the effects
of AMX0035 and its potential in treating Alzheimer’s disease. Study
results are expected in 2020.

“We believe AMX0035 has potential across multiple neurodegenerative
diseases. We’re moving quickly to determine the synergistic efficacy of
these two therapies and deeply value the support from the Alzheimer’s
Drug Discovery Foundation (ADDF), the Alzheimer’s Association and the
Cure Alzheimer’s Fund to expand this unique trial,” said Kent Leslie,
Amylyx’s chief scientific officer.

Alzheimer’s Association

The Alzheimer’s Association is the leading voluntary health organization
in Alzheimer’s care, support and research. It is the largest nonprofit
funder of Alzheimer’s research. The Association’s mission is to
eliminate Alzheimer’s disease through the advancement of research; to
provide and enhance care and support for all affected; and to reduce the
risk of dementia through the promotion of brain health. Its vision is a
world without Alzheimer’s. Visit alz.org or
call 800.272.3900.

Alzheimer’s Drug Discovery Foundation

Founded in 1998 by Leonard A. Lauder and Ronald S. Lauder, the
Alzheimer’s Drug Discovery Foundation is the only philanthropy solely
focused on accelerating the development of drugs to prevent and treat
Alzheimer’s disease. Its venture philanthropy approach and scientific
expertise allows the ADDF to support the most promising ideas around the
world. And 100% of every donation funds drug research programs. To learn
more, visit http://www.alzdiscovery.org.

Cure’s Alzheimer’s Fund

Cure Alzheimer’s Fund is a non-profit dedicated to funding the most
promising research to prevent, slow or reverse Alzheimer’s disease.
Since its founding in 2004, Cure Alzheimer’s Fund has contributed over
$88 million to research, and its funded initiatives have been
responsible for several key breakthroughs – including the groundbreaking
Alzheimer’s in a Dish. Cure Alzheimer’s Fund has received a 4 star
rating for 7 consecutive years regarding its overall financial health
from Charity Navigator. With 100 percent of funds raised going directly
to research, Cure Alzheimer’s Fund has been able to support some of the
best scientific minds in the field of Alzheimer’s research. For more
information, please visit http://www.curealz.org/.

About Amylyx Pharmaceuticals

Amylyx Pharmaceuticals, Inc. is a clinical-stage pharmaceutical company
developing a novel therapeutic for Amyotrophic Lateral Sclerosis (ALS),
Alzheimer’s disease 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 act synergistically to prevent cell death and
neurotoxic inflammation in multiple preclinical models. This press
release and further information about Amylyx can be found at: www.amylyx.com.