AMX0114 receives FDA fast track status for the treatment of ALS
Designation makes drug eligible for accelerated approval, priority review
The U.S. Food and Drug Administration (FDA) has granted fast track designation to AMX0114, an experimental therapy being developed by Amylyx Pharmaceuticals, for the treatment of amyotrophic lateral sclerosis (ALS).
Fast track status is designed to facilitate the development and review of treatments that have the potential to address serious or life-threatening conditions and unmet medical needs.
The designation will enable Amylyx to have more frequent meetings and written communication with the FDA to discuss development plans for AMX0114, and it makes the drug eligible for accelerated approval and priority review if certain criteria are met.
“Obtaining FDA Fast Track designation for AMX0114 is an important step forward in our mission to develop potential treatments for people living with ALS,” Camille L. Bedrosian, MD, chief medical officer at Amylyx, said in a company press release. “We look forward to continued interaction with the FDA as we work to expeditiously advance the development of AMX0114.”
Early data from Phase 1 trial of AMX0114 expected this year
ALS is a progressive neurodegenerative disease marked by the death of motor neurons — nerve cells responsible for voluntary movements — which leads to muscle weakness and a range of symptoms. While available treatments can slow disease progression and extend survival, their benefits are limited and there’s no cure.
AMX0114 an antisense oligonucleotide that’s designed to prevent or slow the degeneration of axons, the long extensions of nerve cells that transmit electrical signals, which is a key driver of ALS progression. It works by reducing the production of calpain-2, an enzyme that tends to be elevated in people with ALS and promotes axonal degeneration.
In preclinical studies, AMX0114 lowered calpain-2 levels and reduced neurofilament light chain (NfL), a biomarker of nerve cell injury, leading to increased survival in cell models of ALS.
Prompted by these results, Amylyx began a Phase 1 trial, called LUMINA (NCT06665165), earlier this year to evaluate AMX0114 in people with ALS. The trial, which recently dosed its first participant at a site in Ontario, is expected to enroll about 48 adults in the U.S. and Canada.
This designation from the FDA recognizes both the seriousness of this devastating disorder and the encouraging preclinical evidence supporting AMX0114’s potential to target calpain-2.
Participants are randomly assigned to receive either AMX0114 or a placebo, administered intrathecally, an injection into the spinal canal, once every four weeks. The therapy is being tested across multiple ascending doses, which means that each group of participants will receive a higher dose than the previous group.
The study’s main goal is to assess safety and tolerability, and a secondary goal is to determine how the therapy moves through and affects the body. Researchers will also track calpain-2 and NfL levels, and evaluate changes in patients’ functional abilities throughout the study.
Early data from the first participants are expected later this year.
“This designation from the FDA recognizes both the seriousness of this devastating disorder and the encouraging preclinical evidence supporting AMX0114’s potential to target calpain-2,” Bedrosian said. ”We are committed to advancing AMX0114 as quickly and efficiently as possible, and we continue to anticipate early cohort data from the Phase 1 LUMINA clinical trial later this year.”
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