Market Overview

Project ALS and Amylyx Enter Collaboration to Test AMX0035


Pre-clinical studies at Columbia University's Motor Neuron Center

Amylyx Phase 2 Clinical Studies of Lead
Therapeutic Compound

Project ALS and Amylyx Pharmaceuticals today announced a collaboration
to undertake pre-clinical studies with Amylyx's oral compound AMX0035 to
advance the understanding of the compound's neurobiological effects. The
studies to be conducted at the Project ALS Pre-Clinical Core at Columbia
University's Motor Neuron Center will complement the company's recently
initiated Phase 2 clinical program of AMX0035 for the treatment of
amyotrophic lateral sclerosis (ALS).

This Smart News Release features multimedia. View the full release here:

The Project ALS Pre-Clinical Core at the Columbia University's Motor
Neuron Center has established a unique integrated and standardized
platform for the testing and validation of new therapeutic strategies in
recognized experimental models of ALS and for biomarker discovery. The
Core, developed in collaboration with Project ALS, will accelerate the
translation of new promising therapies to patients by facilitating
speedy testing of new therapeutic leads discovered by laboratories
studying motor neuron biology, genetics and genomics.

"The collaboration will bring together a promising therapeutic candidate
for a devastating disease with leading edge, fundamental neuroscientific
research at Columbia," said Valerie Estess, director of research for
Project ALS. "The studies in this collaboration will provide greater
insight into the neurobiological effects of AMX0035, and hopefully
optimize its beneficial effects."

The collaboration is an outgrowth of previous studies by The Project ALS
Pre-Clinical Core at Columbia University of tauroursodeoxycholic acid
(TUDCA), one of the components of AMX0035. "We look forward to
evaluating AMX0035 in models of ALS. It is exciting that TUDCA has
independently shown promise in both our labs and in studies conducted by
the company," said Drs. Hynek Wichterle and Serge Przedborski,
co-directors of The Project ALS Pre-Clinical Core and tenure faculty in
the Departments of Pathology and Neurology at Columbia University.

AMX0035 is a combination of two drugs, sodium phenylbutyrate (PB) and
tauroursodeoxycholic acid (TUDCA). Previous studies of PB and TUDCA as
individual agents demonstrated efficacy in cellular and animal models of
ALS. AMX0035 has been shown to synergistically prevent nerve cell death
and neurotoxic inflammation, hallmarks of ALS, in preclinical models. In
addition, PB and TUDCA have been individually tested in ALS clinical
trials and each demonstrated safety, tolerability, and preliminary signs
of efficacy. AMX0035 recently entered Phase 2 clinical development to
evaluate its safety and efficacy in ALS. The trial, called CENTAUR, is a
24-week, randomized, double-blind, placebo-controlled study in 132
participants with ALS. More information on the CENTAUR trial can be
found at,
NCT03127514 and at

"Amylyx is very excited to partner with Project ALS and the researchers
at Columbia's Motor Neuron Center to advance these experiments. We hope
these studies will provide valuable insights into both AMX0035 and ALS
biology that will ultimately improve the lives of patients with ALS,"
said Kent Leslie, Chief Scientific Officer of Amylyx.

About ALS
ALS is a brain disease that is closely related to
Parkinson's, Alzheimer's, and Huntington's. Also known as Lou Gehrig's
disease, ALS targets brain cells called motor neurons. Motor neurons
send messages from the brain to muscles throughout the body. In ALS, as
motor neurons weaken and die, a person progressively loses the ability
to walk, speak, swallow, and breathe. ALS affects adults of all ages,
from teens to seniors.

About Project ALS
Project ALS is a non-profit 501(c)3 that
identifies and funds the most promising scientific research that will
lead to the first effective treatments and a cure for ALS. The Project
ALS Pre-Clinical Core at Columbia University is supported by generous
donations from the Kleiner Family Research Initiative, Stu and Leslie
Hendel, Team Chris Combs, and other donors.

About Motor Neuron Disease Center
The Center for Motor
Neuron Biology and Disease at Columbia University, in short the Motor
Neuron Center, offers an unparalleled pre-clinical academic initiative
integrated with the ALS Clinic Center, the Columbia Precision Medicine
Initiative, the Institute of Genomic Medicine, and the Zuckerman Mind,
Brain, and Behavior Institute.

For the past decade, the Motor Neuron Center faculty members have
utilized existing and novel models of ALS as well as patient derived iPS
cell motor neurons to test a variety of therapeutic strategies ranging
from pharmacological approaches, using small molecules, to gene
therapies, using viral vectors or CRISPR based genome engineering. MNC
scientists work with internationally renowned clinicians experienced in
clinical studies including therapeutic trials for ALS and other
paralytic disorders, eager to provide advice about the feasibility of
therapeutic approaches and to test promising leads in clinical trials.

About Amylyx
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.

View Comments and Join the Discussion!