Results from the first four regimens of the HEALEY ALS Platform Trial
Sabrina Paganoni1, James Berry1, Melanie Quintana2, Eric Macklin3, Benjamin Saville2, Jinsy Andrews4, Jeremy Shefner5, Christina Fournier6, Suma Babu1, Nicholas Maragakis7, Bjorn Oskarsson8, michelle detry2, Marianne Chase1, Alex Sherman1, Hong Yu1, Lindsay Pothier1, Kristin Drake1, Lori Chibnik9, Marie-Abele Bind1, Matteo Vestrucci2, Anna McGlothlin2, Joseph Marion2, Petra Duda10, Brittany Harvey10, Irfan Qureshi11, Mary Donohue12, Volkan Granit11, Katheryn Grossman11, Robert Glanzman13, Michael Hotchkin14, Y Paul Goldberg15, Melanie Leitner16, Michael Hayden15, Merit Cudkowicz1
1Massachusetts General Hospital, 2Berry Consultants, 3MGH Biostatistics Center, 4Columbia University Medical Center, 5Barrow Neurological Institute, 6Emory University, 7Johns Hopkins University School of Medicine, 8Mayo Clinic, 9Harvard School of Public Health, 10UCB, 11Biohaven Pharmaceuticals, 12Biohaven Pharmaceuticals, Inc., 13Clene Nanomedicine Inc, 14Clene Nanomedicine, Inc., 15Prilenia Therapeutics, 16Accelerating Neuroventures

To report results from the first four regimens of the HEALEY ALS Platform Trial.


The HEALEY ALS Platform Trial is a perpetual adaptive phase 2/3 multi-regimen trial that allows for shared trial infrastructure and use of shared placebo data. The initial four regimens have completed the randomized placebo-controlled treatment period.


The HEALEY ALS Platform Trial is a multicenter, randomized, placebo-controlled, multi-arm trial with a randomization ratio of 3:1 to active drug or matching placebo and interim analyses for early futility. A 24-week randomized placebo-controlled treatment (RCT) period is followed by a long-term open-label extension (OLE). The primary endpoint of the RCT is change in disease severity over time as measured by ALS Functional Rating Scale-Revised (ALSFRS-R) accounting for mortality (analyzed using a Bayesian shared-parameter model). Additional endpoints include respiratory function, muscle strength, survival, and safety. Several exploratory efficacy endpoints and biomarkers are collected.


A total of 653 people with ALS were randomized within the first four regimens of the HEALEY ALS Platform Trial (regimen A: zilucoplan; regimen B: verdiperstat; regimen C: CNM-Au8; regimen D: pridopidine) between July 2020 and Dec 2021. Results for primary, secondary, and exploratory endpoints of the RCT period will be presented.


The HEALEY ALS Platform Trial is evaluating multiple investigational products for the treatment of ALS. Four regimens have concluded the RCT period, a fifth regimen is enrolling new participants (regimen E: trehalose), and more regimens are being added. By testing multiple investigational products concurrently and sequentially, the trial is accelerating drug development for ALS [HEALEY ALS Platform Trial: NCT04297683].