An Oral Combination of Sodium Phenylbutyrate–Taurursodiol in ALS

In a small, 6-month phase 2 trial with inclusion criteria designed to enroll homogenous and fast progressors, this fixed two-drug combination slowed function decline by 25%, although secondary outcomes did not differ.

To evaluate the safety and efficacy of sodium phenylbutyrate-taurursodiol (AMX0035) for amyotrophic lateral sclerosis (ALS), researchers from the Northeast ALS Consortium and Amylyx collaborated in a multicenter, double-blinded, placebo-controlled trial. They randomized 137 participants to ingest either a dissolvable powder of 3 g sodium phenylbutyrate/1 g taurursodiol (89 patients) or placebo (48 patients) daily for 3 weeks then twice daily for 24 weeks. Eligible participants were enrolled <18 months after ALS onset, had signs in at least three body regions, and had a slow vital capacity >60% of predicted normal based on the patient's age, sex, and height. Most (77%) were taking riluzole, edaravone, or both, with an imbalance in edaravone …

Reviewing Author

Leana Doherty, MD

Disclosures

Editorial BoardsNeurohospitalist Society; InterACTN (Annals of Clinical and Translational Neurology)

Leana Doherty, MD

Disclosures

Editorial BoardsNeurohospitalist Society; InterACTN (Annals of Clinical and Translational Neurology)

An Oral Combination of Sodium Phenylbutyrate–Taurursodiol in ALS

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Reviewing Author