Amylyx Announces $5 Million Series A Financing to Support Phase II Clinical Trial of AMX0035 for Treatment of Amyotrophic Lateral Sclerosis

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Amylyx Pharmaceuticals Inc. announced today that it has completed a $5
million Series A financing to support its upcoming Phase II trial in
patients with Amyotrophic Lateral Sclerosis (ALS). Morningside Venture
led the financing and was joined by the ALS Investment Fund and former
Genzyme CEO Henri Termeer, as well as new and previous private investors.
The financing adds to a recently awarded $2.96 million grant to support
the clinical trial from the ALS Accelerated Therapeutics Initiative,
which is a collaboration between the ALS Association and ALS Finding a
Cure. Overall, Amylyx has raised $10 million in grant funding and
private financing to advance AMX0035. The IND for this Phase II trial is
on schedule for the fourth quarter of 2016 and the trial will start
shortly thereafter. Over 20 clinical sites across the United States have
already expressed interest in participating.
In connection with the financing, Dr. Isaac Cheng, M.D. from Morningside
and Mr. Felix von Coerper from the ALS Investment Fund have joined
Amylyx’s board of directors. Mr. Termeer will become a Board observer.
They will join directors Dr. George Milne, former President of Central
Research and President of Strategic and Operations Management at Pfizer;
Dr. Walter Gilbert, Nobel Laureate and former Biogen CEO; and Stephen D.
Chubb, previous CEO of three publicly-traded biotechnology companies and
previous chairman of the board at Mount Auburn Hospital.
“Amylyx has built a strong rationale for AMX0035 in the treatment of ALS
and has developed an outstanding clinical plan to evaluate its potential
in ALS patients,” said Dr. Isaac Cheng M.D. of Morningside. “There is a
critical unmet need for ALS treatments and Morningside is pleased to
support Amylyx’s program.”
“With AMX0035, Amylyx seeks to advance a promising approach to ALS
therapy, by targeting neuroinflammation and cell death, that is now
poised to enter human studies,” said Mr. von Coerper. “Our investment in
Amylyx is aligned with the Fund’s goals of driving medical advancement
for this terrible disease.”
“Amylyx’s clinical plan is not only designed to validate their
therapeutic candidate, but also push forward important innovation in
trial design and execution in ALS,” said Henri Termeer. “I am excited to
work with Amylyx as they work to make a difference in the lives of these
patients.”
AMX0035 combines two drugs, sodium phenylbutyrate (PB) and
tauroursodeoxycholic acid (TUDCA), that Amylyx has shown act
synergistically in preclinical models of ALS. Previous studies with PB
and TUDCA as single agents demonstrated efficacy in several cellular and
animal models of ALS. In addition, PB and TUDCA have been individually
tested in clinical trials of ALS and both showed safety and tolerability
and preliminary signs of efficacy.
The Phase II clinical trial will test the safety and tolerability of
AMX0035, as well as functional outcomes. Analysis of biomarkers of cell
function, neuronal damage, and inflammation will be included as a major
part of the trial, along with a new measure of muscle strength that has
the potential to be an objective and sensitive assessment of disease
progression.
“We are very pleased with the support and commitment of this group of
highly experienced investors who share the focus of making a significant
difference in this disease,” said Justin Klee, President at Amylyx. Josh
Cohen, CEO added, “We are also thrilled to welcome our new Board
members, and anticipate benefiting from their guidance as we prepare to
enter clinical development with AMX0035.”
ALS is a progressive neurodegenerative disease that affects nerve cells
in the brain and the spinal cord. Eventually, people with ALS lose the
ability to initiate and control muscle movement, which leads to total
paralysis and death, usually within two to five years of diagnosis.
There is no cure, and only one drug approved by the U.S. Food and Drug
Administration modestly extends survival.
About Amylyx Pharmaceuticals
Amylyx is developing a novel
therapeutic for Amyotrophic Lateral Sclerosis (ALS) and other
neurodegenerative diseases that target the neuroinflammation and nerve
cell death that characterize these disorders. The company’s therapeutic
candidate, AMX0035, is a proprietary, fixed-dose combination therapy
that blocks nerve cell death and neurotoxic inflammation. Learn more at:
www.amylyx.com.
About Morningside
Morningside is a diversified investment
group founded in 1986 by the Chan family of Hong Kong. It is engaged
primarily in private equity and venture capital investments. The group
has investments in North America, Europe, across Asia-Pacific, and since
1992, in Mainland China. Morningside is an active investor in
early-stage life science companies formed around new technologies that
represent a high degree of novelty over existing technologies. More
information is available at www.morningside.com.
About ALS Investment Fund
The ALS Investment Fund supports
and finances biotech companies that develop drugs and diagnostics for
Amyotrophic Lateral Sclerosis (ALS), also known as Lou Gehrig’s
disease). The Fund was initiated by 3 Dutch ALS patients and is
supported by a top-tier European VC firm, Life Sciences Partners. The
ALS Investment Fund invests globally, but focuses on Europe and the US.
The fund brings depth of disease-specific knowledge and a deep network
in the space. More information is available at https://www.alsinvestmentfund.com/.