Market Overview

Amylyx Pharmaceuticals Receives FDA Orphan Drug Designation for AMX0035 for the Treatment of Amyotrophic Lateral Sclerosis


Amylyx Pharmaceuticals, Inc., announced today that the U.S. Food and
Drug Administration (FDA) has granted orphan drug designation to
AMX0035, an oral therapeutic in clinical development for the treatment
of amyotrophic lateral sclerosis (ALS). The Company recently began a
Phase 2 clinical study, the CENTAUR trial, to evaluate the safety and
efficacy of AMX0035 in ALS patients.

The U.S. Orphan Drug Act provides incentives to encourage the
development of drugs for diseases affecting fewer than 200,000 people in
the U.S. Orphan drug designation by the FDA conveys up to seven years of
marketing exclusivity if the compound receives regulatory approval, and
offers various development incentives, including tax credits related to
clinical trial expenses, an exemption from the FDA-user fee, and FDA
assistance in clinical trial design.

Background on AMX0035
AMX0035 is a combination of two small
molecules, sodium phenylbutyrate (PB) and tauroursodeoxycholic acid
(TUDCA). Each compound has demonstrated strong efficacy in several
cellular and animal models of ALS. When individually tested in ALS
clinical trials, PB and TUDCA have both shown safety, tolerability, and
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.

About the CENTAUR Trial
CENTAUR is a 24-week, randomized,
double-blind, placebo-controlled Phase II clinical trial in 132
participants with ALS. The trial's primary objectives are to evaluate
the safety and tolerability of AMX0035 and 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 trial will also evaluate the impact of AMX0035 on isometric
strength as measured by ATLIS, respiratory function, and exploratory
biomarkers. There will also be an open label extension to the trial so
that all enrolled patients will have the option of continuing treatment
once the 24-week treatment period concludes. More information on the
CENTAUR trial can be found at or,
NCT03127514. Please contact (855)-437-4823 or email if
you are interested in participating. Contact information for trial sites
will be made available on

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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