The U.S. Food and Drug Administration approved AMX0035 for the treatment of ALS in September 2022. Find answers to frequently asked questions below.
- What is AMX0035 (RELYVRIO)?
- How does AMX0035 work?
- How is AMX0035 administered?
- What have researchers learned about AMX0035 so far?
- How has The ALS Association supported the development of AMX0035?
- How Can I Access AMX0035?
- Learn more about AMX0035
AMX0035 is an investigational drug, also known as RELYVRIO or PB/TURSO. AMX0035 is a combination of sodium phenylbutyrate (PB) and taurursodiol (TURSO).
AMX0035 is designed to slow disease progression by slowing or preventing motor neuron cell death. Motor neurons are the cells that die in ALS.
AMX0035 targets both the endoplasmic reticulum (ER) and mitochondria in ALS in motor neurons. This is important because the ER and mitochondria are connected (via mitochondrial membranes) and both play critical roles in maintaining neuron health and survival.
AMX0035 comes in sachet packets of a powder that is dissolved in room temperature water. It can be administered by mouth or by feeding tube. In the phase 2 trial, the dose regimen was to take the drug once daily for first three weeks and then twice daily if tolerating.
Amylyx published a peer-reviewed paper in the New England Journal of Medicine outlining the results of the CENTAUR phase 2 trial and another outlining the trial’s overall survival data in the journal Muscle and Nerve.
Overall, the trial enrolled 137 people with ALS in 25 medical centers through the Northeast ALS (NEALS) Consortium from June 2017 to September 2019. It showed that AMX0035 was well-tolerated and there were no major safety concerns. Side effect rates were generally similar between the AMX0035 and placebo groups, but the AMX0035 group did report more gastrointestinal issues (for example, diarrhea). AMX0035 also significantly decreased the rate of decline in the Revised ALS Functional Rating Scale (ALSFRS-R) compared to placebo.
Almost three years after the first participant started in CENTAUR, in July 2020, the difference in survival between the AMX0035 group and the placebo group was evaluated. People with ALS who received AMX0035 at the start of the CENTAUR trial lived an average of 6.5 months longer than the comparison placebo group.
The CENTAUR trial included an open label extension phase, where all participants who completed the placebo-controlled phase could enroll and receive AMX0035. Analysis of the data from that study showed that AMX0035 extended life by 10 months and reduced the risk of death, tracheostomy or permanent assisted ventilation, and first hospitalization.
The ALS Association committed $750,000 to Amylyx in funding for AMX0035 research and $1.4M to the NEALS clinical trial consortium to support the phase 2 trial. These grants were made possible through the ALS Ice Bucket Challenge. ALS Finding a Cure also supported this study.
Through a standard repayment clause, the Association could recoup up to 150% of its research investment, which it will reinvest in additional research.
The Association, in collaboration with I AM ALS, submitted a petition to the FDA in November 2020 calling on the agency and Amylyx to act swiftly and with urgency to make AMX0035 available as soon as possible. The petition was signed by more than 50,000 people from across the country who have been affected by ALS.
In September of 2020, The ALS Association and I AM ALS submitted over 50,000 signatures to the FDA calling on the agency to approve AMX0035. In subsequent months, the Association held multiple meetings with FDA officials, including a public We Can’t Wait Action Meeting in May of 2021, so members of the ALS community could speak directly to FDA officials. The Association also provided scientific and regulatory guidance and in 2022, drove over 13,000 individual emails to the FDA urging approval.
After the FDA accepted a New Drug Application for AMX0035 and convened an advisory committee to consider the effectiveness data, the Association empowered more than 13,000 people to write emails to the FDA calling for approval of the treatment.
To ensure every eligible person who can benefit from RELYVRIO (formerly known as AMX0035) will have access to it, Amylyx will provide support to healthcare professionals, people living with ALS, and their loved ones through the Amylyx Care Team (ACT) Support Program.
ACT provides people living with ALS who have been prescribed RELYVRIO, and their loved ones, with a dedicated, single point of contact to guide their treatment journey. ACT will aid with navigating through insurance in an effort to overcome potential barriers to access and provides financial assistance options to eligible individuals with out-of-pocket costs. People with ALS, their caregivers, and healthcare professionals in the U.S. can now call 1-866-318-2989 or email [email protected] to speak with an ACT team member.
Amylyx’s website: www.Amylyx.com
Read more about the PHOENIX phase 3 trial and your eligibility to participate.