- Topics include safety data from the AMX0035 U.S. Expanded
Access Program, progress on the development of an ALS diagnostic
biomarker test, new in vitro data on AMX0114, and more
Amylyx Pharmaceuticals, Inc. (NASDAQ: AMLX) (“Amylyx” or the
“Company”) today announced the acceptance of several abstracts for
presentation at the hybrid 2023 Northeast ALS Consortium (NEALS)
Annual Meeting. The data being shared are related to 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. AMX0035 is marketed by Amylyx as
RELYVRIO® and is approved to treat ALS in adults in the U.S. and
approved with conditions as ALBRIOZA™ for the treatment of ALS in
Canada. The in-person component of the NEALS Annual Meeting will
take place in Clearwater, Florida October 4-6, 2023.
“We look forward to discussing updates from our research
initiatives, in collaboration with the ALS community, as part of
our collective efforts to contribute to a deeper understanding of
ALS and the treatment and care paradigm,” said Machelle Manuel,
PhD, Head, Global Medical Affairs at Amylyx. “There is still much
to learn about ALS, and research is a critical pillar of how we’ll
deliver on our mission to one day end the suffering caused by
neurodegenerative diseases.”
Details of the poster presentations at the NEALS Annual Meeting
are as follows:
Wednesday, October 4, 2023, 5:15 pm – 7:15
pm ET
- Title: Development of a Composite Diagnostic Biomarker
for ALS: Experimental Approach and Progress to Date 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:
38
- Title: Update on AMX0114: An Antisense Oligonucleotide
Targeting Calpain-2, a Critical Effector of Axonal Degeneration 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: 14
- Title: Novel Neuroprotective Strategies in Human Neuron
Models of ALS/FTD: Evaluating Antisense Oligonucleotide Therapies,
including AMX0114 This poster details findings from a collaboration
with Dr. Sami Barmada and his team at the University of Michigan
School of Medicine in which the impact of ASOs targeting calpain-2,
including AMX0114, on survival were evaluated in human iPSC-derived
motor neurons harboring the ALS-linked TDP43M337V mutation.
Poster Number: 15
Thursday, October 5, 2023, 4:30 pm – 6:30
pm ET
- Title: Real-World Experience and Strategies to Enhance
the Palatability of the Combination Sodium Phenylbutyrate and
Taurursodiol for the Treatment of Amyotrophic Lateral Sclerosis
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: 90
- Title: Preliminary Experience with Sodium Phenylbutyrate
& Taurursodiol in a US Expanded Access Program 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: 123
- Title: An In Vitro Recovery Study of Sodium
Phenylbutyrate and Taurursodiol From 3 Types of Dosing Containers
and Various Percutaneous Endoscopic Gastrostomy Feeding Tubes This
poster details results of an in vitro study evaluating the use of
sodium phenylbutyrate and taurursodiol with different types of
feeding tubes and containers. Poster Number: 124
- Title: Ongoing and Planned Studies to Further Elucidate
the Efficacy, Safety, and Pharmacokinetics of Sodium Phenylbutyrate
and Taurursodiol in Amyotrophic Lateral Sclerosis 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: 125
For conference information, visit:
https://neals.org/als-researchers/annual-neals-meeting
Information about the 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®, 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
explored in other populations and regions, as well as for the
potential treatment of 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 ALS
ALS is a relentlessly progressive and fatal neurodegenerative
disorder caused by motor neuron death in the brain and spinal cord.
Motor neuron loss in ALS leads to deteriorating muscle function,
the inability to move and speak, respiratory paralysis and
eventually, death. More than 90% of people with ALS have sporadic
disease, showing no clear family history. ALS affects approximately
29,000 people in the U.S. and more than 30,000 people are estimated
to be living with ALS in Europe (European Union and United
Kingdom). People living with ALS have a median survival of
approximately two years from diagnosis.
About Amylyx Pharmaceuticals
Amylyx Pharmaceuticals, Inc. is committed to supporting and
creating more moments for the neurodegenerative disease 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 Twitter. For investors,
please visit investors.amylyx.com.
Forward-Looking Statements
Statements contained in this press release 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 approvability of
AMX0035 for conditional marketing authorisation in Europe; the
ability to provide post-authorisation confirmatory data; our plans
to make AMX0035 available in Europe; the ongoing commercialization
of RELYVRIO and ALBRIOZA; the potential continued market acceptance
and market opportunity for RELYVRIO and ALBRIOZA; the potential of
AMX0035 as a treatment for ALS and the Company’s plans to explore
the use of AMX0035 for other neurodegenerative diseases; the
potential of other investigational treatments to be developed to
treat ALS or other neurodegenerative diseases; and expectations
regarding our longer-term strategy. Any forward-looking statements
in this press release 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: Amylyx’ ability
to fund operations, the success, cost, and timing of Amylyx’
program development activities, Amylyx’ ability to execute on its
commercial and regulatory strategy, regulatory developments,
expectations regarding the timing and outcome of EMA’s review of
AMX0035 for the treatment of ALS, Amylyx’ reliance on third
parties, including to conduct clinical trials and manufacture
products, and the effect of global economic uncertainty and
financial market volatility caused by economic effects of rising
inflation and interest rates, the COVID-19 pandemic, geopolitical
instability, changes in international trade relationships and
military conflicts, 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 June 30, 2023, and subsequent filings with the
SEC. All forward-looking statements contained in this press release
speak only as of the date on which they were made. Subject to any
obligations under applicable law, 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/20231002273497/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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