Amylyx Pharmaceuticals, Inc. (NASDAQ: AMLX) (“Amylyx” or the
“Company”) today announced that several abstracts detailing data on
AMX0035 (sodium phenylbutyrate and taurursodiol [PB&TURSO]) and
the Company’s investigational antisense oligonucleotide, AMX0114,
for the potential treatment of amyotrophic lateral sclerosis (ALS)
and other neurodegenerative diseases will be presented at the 34th
International Symposium on ALS/MND. New data using a model for
assessing mortality-adjusted progression (MAP) for clinical trials
of ALS and encore posters will be presented.
Details of the poster presentations are as follows:
Wednesday, December 6, 2023, Session A,
5:45 – 7:30 p.m. CET
- Title: A Joint Model for Assessing Mortality-Adjusted
Progression (MAP) in Amyotrophic Lateral Sclerosis: Application to
Clinical Trials of Sodium Phenylbutyrate and Taurursodiol This
poster features an overview of the MAP model, a method that has
been developed by leading ALS clinicians and statisticians and
detailed in previous publications as an approach to account for
deaths, while providing an interpretable and clinically relevant
ALS Functional Rating Scale–Revised (ALSFRS-R) result. Results from
a post hoc analysis of the Phase 2 CENTAUR trial using this model
are presented, which resulted in a nearly identical statistical
outcome with increased precision as the primary analysis. The
Company intends to use a similar model for the primary efficacy
analysis in the ongoing Phase 3 PHOENIX trial, with results
anticipated in the second quarter of 2024. Poster Number:
CLT-30
- Title: Ongoing and Planned Studies to Further Elucidate
the Efficacy, Safety, and Pharmacokinetics of Sodium Phenylbutyrate
and Taurursodiol in Amyotrophic Lateral Sclerosis (encore) This
poster outlines studies currently underway that further assess
efficacy and safety of AMX0035 in people living with ALS, including
in real-world settings. Poster Number: CLT-10
- Title: Development of a Composite Diagnostic Biomarker
for Amyotrophic Lateral Sclerosis: Experimental Approach and
Progress to Date (encore) One of the key drivers of diagnostic
delay in ALS is the lack of reliable, validated biomarkers to aid
in diagnosis. This poster provides an update on the progress made
in developing a biomarker test that could make the diagnosis of ALS
easier. Poster Number: BIO-14
Thursday, December 7, 2023, Session B,
5:45 – 7:45 p.m. CET
- Title: Real-World Experience and Strategies to Enhance
the Palatability of the Combination Sodium Phenylbutyrate and
Taurursodiol for the Treatment of Amyotrophic Lateral Sclerosis
(encore) Sodium phenylbutyrate and taurursodiol can have a bitter
taste to some people living with ALS, but the combination is
generally well-tolerated with an acceptable safety profile.
Surveying people living with ALS in the U.S. prescribed sodium
phenylbutyrate and taurursodiol, this poster provides information
on what we learned about their real-world experiences related to
product taste. Poster Number: CW-06
Thursday, December 7, 2023, Session B,
Part 1, 5:45 – 6:45 p.m. CET
- Title: Preliminary Experience With Sodium Phenylbutyrate
and Taurursodiol in a United States Expanded Access Program
(encore) In 2022, we completed the largest single-product ALS
Expanded Access Program (EAP) in the U.S. to date to provide
pre-approval access to sodium phenylbutyrate and taurursodiol to
people living with ALS alongside the ongoing Phase 3 PHOENIX trial.
This poster provides initial safety data and learnings gathered
through this program. Poster Number: CLT-11
Thursday, December 7, 2023, Session B,
Part 2, 6:45 – 7:45 p.m. CET
- Title: Update on AMX0114: An Antisense Oligonucleotide
Targeting Calpain-2, a Critical Effector of Axonal Degeneration
(encore) We believe that it is going to take a combination
approach, targeting multiple cellular pathways implicated in
disease pathogenesis, to find a cure for ALS. This poster provides
an update on AMX0114, our internally developed antisense
oligonucleotide (ASO) targeting calpain-2, a critical effector of
axonal degeneration in ALS and other neurodegenerative diseases.
Poster Number: TST-12
For conference information, visit:
https://symposium.mndassociation.org/
A copy of the abstract discussing the MAP model is published
online in Amyotrophic Lateral Sclerosis and Frontotemporal
Degeneration. Additional information, including copies of the
poster presentations, will be made available on the “Publications”
tab of the Amylyx website, following the conclusion of the poster
presentations.
About RELYVRIO®/ALBRIOZA™ /AMX0035
RELYVRIO® (also known as AMX0035), 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 as an investigational drug in
several other regions for the potential treatment of ALS and other
neurodegenerative diseases. 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 ongoing commercialization of RELYVRIO and ALBRIOZA;
expectations regarding the timing of initiation of the Company’s
Phase 3 ORION trial of AMX0035 for the treatment of PSP and of the
results of the Company’s Phase 2 HELIOS trial of AMX0035 for the
treatment of Wolfram syndrome; the timing of the results of the
Company’s Phase 3 PHOENIX trial of AMX0035 for the treatment of
ALS; expectations regarding the results of the Phase 3 PHOENIX
trial and of the potential for future approval of AMX0035 in the
EU; the timing of filing IND applications for AMX0114 and for a new
formation of RELYVRIO; the potential for new pipeline programs and
clinical indications for AMX0035; statements regarding regulatory
developments; the Company’s expectations with respect to its
progress through IND enabling studies of AMX0114 and other
advancements in its pipeline; 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; that data from later-stage trials may not
reflect data from earlier-stage trials, including other
indications; regulatory developments; 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, except as required by
law.
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version on businesswire.com: https://www.businesswire.com/news/home/20231122682452/en/
Media Amylyx Media Team (857) 799-7274
amylyxmediateam@amylyx.com
Investors Lindsey Allen Amylyx Pharmaceuticals, Inc.
(857) 320-6244 Investors@amylyx.com
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