Amylyx Pharmaceuticals, Inc. (Nasdaq: AMLX) (“Amylyx” or the
“Company”) today announced the publication of analyses performed on
neuroinflammatory biomarkers using plasma samples from participants
with amyotrophic lateral sclerosis (ALS) from the Phase 2 CENTAUR
trial. These findings were published in the peer-reviewed medical
journal, Journal of Neurology, Neurosurgery and Psychiatry. During
the CENTAUR trial, plasma samples were prospectively collected from
trial participants for future biomarker analyses. Post hoc analyses
were conducted to look at the impact of AMX0035 on biomarkers shown
to correlate with ALS disease progression, including chitinase
biomarkers such as YKL-40 (also known as chitinase-3-like protein
1), chitinase 1 (CHIT1), and the systemic inflammatory biomarker
C-reactive protein (CRP). The results of these post hoc analyses
demonstrated a significant reduction in plasma concentrations of
YKL-40 and CRP, but not CHIT1, over 24 weeks, with reductions
observed as early as Week 12 in participants from the CENTAUR
trial.
“Neuroinflammatory biomarkers are important for assessing
disease progression and therapeutic response in ALS with chitinases
and CRP emerging as potential treatment response biomarkers.
Previous studies have shown that levels of YKL-40 in cerebrospinal
fluid correlate with ALS disease progression rate, severity, and
survival,” said Robert Bowser, PhD, Chief Scientific Officer of
Barrow Neurological Institute. “The learnings from these analyses
are an important step linking the biological impact of AMX0035 with
potential key biomarkers for ALS.”
“Biomarkers linking clinical therapeutic effect with biological
changes are of high interest in ALS, and the findings from these
analyses suggest that YKL-40 could be a treatment-sensitive
biomarker,” said Machelle Manuel, PhD, Head of Global Medical
Affairs at Amylyx. “YKL-40 and CRP concentration decreased
significantly for the group of participants that received AMX0035
compared to placebo and were correlated with disease progression as
measured by the ALSFRS-R, further demonstrating the impact of
AMX0035.”
Of the 135 participants in the modified intent-to-treat
population (ITT) of the CENTAUR trial, 126 had plasma samples
available for these analyses. The analyses showed that geometric
least squares (LS) mean YKL-40 and CRP plasma concentrations were
10% and 17% lower, respectively, at Week 12 and approximately 20%
and 30% lower, respectively, at Week 24 in the AMX0035 versus
placebo group. YKL-40 and CRP concentrations correlated with
ALSFRS-R total score and ALSFRS-R slope. Geometric LS mean CHIT1
plasma levels were not significantly different between treatment
groups. Further analyses of neuroinflammatory biomarkers are
planned in the ongoing Phase 3 PHOENIX trial to confirm these
results.
About RELYVRIO®/ ALBRIOZA™ / ALBRIOZA® / AMX0035
RELYVRIO®, an oral, fixed-dose combination of sodium
phenylbutyrate and taurursodiol (known as ursodoxicoltaurine
outside of the U.S.), is approved to treat amyotrophic lateral
sclerosis (ALS) in adults in the U.S. and approved with conditions
as ALBRIOZA™ for the treatment of ALS in Canada. AMX0035 is being
studied for the potential treatment of other neurodegenerative
diseases, and Amylyx is exploring its treatment in other
populations and regions. The formulation of RELYVRIO, ALBRIOZA, and
AMX0035 are identical.
RELYVRIO® (sodium phenylbutyrate and taurursodiol) Safety
Information for United States
WARNINGS AND PRECAUTIONS
Risk in Patients with Enterohepatic Circulation Disorders,
Pancreatic Disorders, or Intestinal Disorders
RELYVRIO contains taurursodiol, which is a bile acid. In
patients with disorders that interfere with bile acid circulation,
there may be an increased risk for worsening diarrhea, and patients
should be monitored appropriately for this adverse reaction.
Pancreatic insufficiency, intestinal malabsorption, or intestinal
diseases that may alter the concentration of bile acids may also
lead to decreased absorption of either of the components of
RELYVRIO. Because different enterohepatic circulation, pancreatic,
and intestinal disorders have varying degrees of severity, consider
consulting with a specialist. Patients with disorders of
enterohepatic circulation (e.g., biliary infection, active
cholecystitis), severe pancreatic disorders (e.g., pancreatitis),
and intestinal disorders that may alter concentrations of bile
acids (e.g., ileal resection, regional ileitis) were excluded from
the study; therefore, there is no clinical experience in these
conditions.
Use in Patients Sensitive to High Sodium Intake
RELYVRIO has a high salt content. Each initial daily dosage of 1
packet contains 464 mg of sodium; each maintenance dosage of 2
packets daily contains 928 mg of sodium. In patients sensitive to
salt intake (e.g., those with heart failure, hypertension, or renal
impairment), consider the amount of daily sodium intake in each
dose of RELYVRIO and monitor appropriately.
ADVERSE REACTIONS
The most common adverse reactions (at least 15% and at least 5%
greater than placebo) with RELYVRIO were diarrhea, abdominal pain,
nausea, and upper respiratory tract infection.
Gastrointestinal-related adverse reactions occurred throughout the
study but were more frequent during the first 3 weeks of
treatment.
Please click here for RELYVRIO Full U.S.
Prescribing Information.
About the CENTAUR Trial
CENTAUR was a multicenter Phase 2 clinical trial in 137
participants with ALS encompassing a 6-month randomized
placebo-controlled phase and an open-label long-term follow-up
phase. The trial met its primary efficacy endpoint of reducing
functional decline as measured by the ALS Functional Rating
Scale-Revised (ALSFRS-R).
Overall, reported rates of adverse events and discontinuations
were similar between AMX0035 and placebo groups during the 24-week
randomized phase; however, gastrointestinal events occurred with
greater frequency (≥2%) in the AMX0035 group. Detailed data from
CENTAUR is published in the New England Journal of Medicine (NEJM)
and Muscle & Nerve.
The CENTAUR trial was funded, in part, by the ALS ACT grant and
the ALS Ice Bucket Challenge, and was supported by The ALS
Association, ALS Finding a Cure (a program of The Leandro P.
Rizzuto Foundation), the Northeast ALS Consortium, and the Sean M.
Healey & AMG Center for ALS at Mass General.
About Amylyx Pharmaceuticals
Amylyx Pharmaceuticals, Inc. is committed to supporting and
creating more moments for the neurodegenerative community through
the discovery and development of innovative new treatments. Amylyx
is headquartered in Cambridge, Massachusetts and has operations in
Canada and EMEA. For more information, visit amylyx.com and follow
us on LinkedIn and X, formerly known as Twitter. For investors,
please visit investors.amylyx.com.
Forward-Looking Statements
Statements contained in this press release and related comments
in our earnings conference call regarding matters that are not
historical facts are “forward-looking statements” within the
meaning of the Private Securities Litigation Reform Act of 1995, as
amended. Because such statements are subject to risks and
uncertainties, actual results may differ materially from those
expressed or implied by such forward-looking statements. Such
statements include, but are not limited to, the potential of
AMX0035 (sodium phenylbutyrate and taurursodiol) as a treatment for
ALS and other neurodegenerative diseases including Wolfram syndrome
and PSP; the Company’s beliefs regarding the benefits of AMX0035 in
ALS and other neurodegenerative diseases, the potential of AMX0035
to be a foundational therapy for ALS the link between biomarker
data and clinical effect in ALS; the potential for new pipeline
programs and clinical indications for AMX0035; statements regarding
regulatory developments; the Company’s expectations regarding its
financial performance; and expectations regarding the Company’s
longer-term strategy. Any forward-looking statements in this press
release and related comments in the Company's earnings conference
call are based on management’s current expectations of future
events and are subject to a number of risks and uncertainties that
could cause actual results to differ materially and adversely from
those set forth in or implied by such forward-looking statements.
Risks that contribute to the uncertain nature of the
forward-looking statements include: the success, cost, and timing
of Amylyx’ program development activities, Amylyx’ ability to
successfully commercialize RELYVRIO in the United States and
ALBRIOZA in Canada, Amylyx’ ability to execute on its commercial
and regulatory strategy, regulatory developments, expectations
regarding the timing of a decision from the EMA regarding AMX0035
for the treatment of ALS, Amylyx’ ability to fund operations, and
the impact that global macroeconomic uncertainty, geopolitical
instability and public health events, such as COVID-19, will have
on Amylyx’ operations, as well as the risks and uncertainties set
forth in Amylyx’ United States Securities and Exchange Commission
(SEC) filings, including Amylyx’ Quarterly Report on Form 10-Q for
the quarter ended September 30, 2023, and subsequent filings with
the SEC. All forward-looking statements contained in this press
release and related comments in our earnings conference call speak
only as of the date on which they were made. Amylyx undertakes no
obligation to update such statements to reflect events that occur
or circumstances that exist after the date on which they were
made.
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version on businesswire.com: https://www.businesswire.com/news/home/20231117036343/en/
Media Amylyx Media Team +1 (857) 799-7274
amylyxmediateam@amylyx.com Investors Lindsey Allen Amylyx
Pharmaceuticals, Inc. +1 (857) 320-6244 Investors@amylyx.com
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