RELYVRIO® and AMX0114 Data to be Presented at 22nd Annual Northeast ALS Consortium (NEALS) Meeting
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
“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
Details of the poster presentations at the NEALS Annual Meeting are as follows:
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 Barmadaand his team at the University of Michigan School of Medicinein 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
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.
RELYVRIO®, an oral, fixed-dose combination of sodium phenylbutyrate and taurursodiol (known as ursodoxicoltaurine outside of the
RELYVRIO® (sodium phenylbutyrate and taurursodiol) Safety Information for
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.
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.
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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
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