AMX0035 Expanded Access Program Launched for Eligible Patients in US

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by Lindsey Shapiro, PhD |

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Amylyx Pharmaceuticals has launched an expanded access program (EAP) in the U.S. that enables eligible adults with amyotrophic lateral sclerosis (ALS) to access the company’s experimental treatment, AMX0035.

To be eligible for the program (NCT05286372), patients must have experienced their first signs of weakness three years or more prior to entering the program, and have received consistent clinical care from an experienced physician.

Since the therapy has not been tested during pregnancy, women of childbearing age and men seeking to access AMX0035 under the EAP must agree to use appropriate contraception and not plan to have children during treatment or within three months of stopping treatment. Individuals who are already pregnant or breastfeeding are ineligible.

Also ineligible for the program are people with liver and kidney abnormalities and certain other health issues; those dependent on invasive mechanical ventilation; and individuals with a life expectancy under six months. To learn more about the program, contact [email protected].

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The EAP, approved by the U.S. Food and Drug Administration (FDA), is designed to provide earlier access to AMX0035 for patients who are ineligible for the ongoing PHOENIX Phase 3 trial (NCT05021536).

It will run in parallel with the trial and with the FDA’s review of AMX0035 as a treatment for ALS. An online meeting to discuss the approval request is set for March 30, and a decision is expected by June 29.

“We take our responsibilities as part of the ALS community seriously, and we know access to therapies is of the utmost importance,” Machelle Manuel, PhD, Amylyx’s head of global medical affairs, said in a press release.

PHOENIX is enrolling up to 600 adults with ALS, whose symptoms emerged within the previous two years, at sites in the U.S. and Europe. Participants are randomly assigned to receive AMX0035 or a placebo for 48 weeks (about 11 months).

The study’s main goals are to assess safety and changes in a joint assessment of functional decline, measured with the ALS Functional Rating Scale-Revised, and survival. Secondary measures include changes in lung function and quality of life, as well as the need for ventilation and long-term survival.

The company noted that people who are eligible for PHOENIX will be ineligible for the EAP, since the criteria for time since symptom onset do not overlap.

“In designing the EAP, we worked closely with the ALS community — people living with ALS, caregivers, advocacy groups, and clinicians — to take their needs and ideas into account,” said Manuel.

AMX0035 is a fixed-dose oral combination of sodium phenylbutyrate and tauroursodeoxycholic acid, two small molecules that have been used in the clinic and that work together to block stress signals involved in nerve cell death.

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The submission to the FDA was based on top-line results from the Phase 2/3 CENTAUR clinical trial (NCT03127514), which indicated that six months of treatment with AMX0035 was generally safe and significantly more effective than a placebo at slowing the progression of functional disability.

AMX0035 is also under review in the European Union and Canada, based on data from CENTAUR.