Ice Bucket Dollars at Work: Amylyx Pharmaceuticals CENTAUR Trial is Making Headway

UPDATE: As of March 14, 2019, enrollment for the CENTAUR Phase II clinical trial has been completed.

At the recent Leadership Conference for The ALS Association, Joshua Cohen and Justin Klee, CEO and president, respectively, and founders of Amylyx Pharmaceuticals, updated us on their ongoing CENTAUR phase II clinical trial. The Association is supporting their trial through ALS Ice Bucket Challenge donations, with a $2.96 million grant in partnership with ALS Finding a Cure.

“The ALS Association’s contribution has made a substantial difference in accelerating our clinical study, which, hopefully will result in new insights and a new therapeutic for ALS,” said Cohen. Klee added, “We are enormously grateful for their continuing support and for the support of everybody in the ALS community.”

CENTAUR is testing a drug called AMX0035, a unique combination oral therapy of phenylbutyrate and tauroursodeoxycholic acid that simultaneously targets the mitochondria and endoplasmic reticulum (ER) in neurons. AMX0035’s mechanism is intended to break the cycle of neuroinflammation and neurodegeneration that are important contributors to functional decline in ALS patients. Both compounds showed promising results in separate ALS clinical trials.

Cohen and Klee explained features that make their trial, which began in July 2017 and will enroll 132 ALS patients nationwide, distinctive compared to other ALS clinical trials.

CENTAUR is the first ALS therapeutic trial to use a central institutional review board (cIRB) – a group formally designated to review and monitor biomedical research involving humans – instead of separate boards at each of the 25 trial sites involved in the study. The cIRB is expected to streamline the clinical trial process. The ALS Association funded the development of the central IRB as part of the Northeast ALS Consortium (NEALS) Clinical Trials Network.

CENTAUR is also the first therapeutic trial to utilize ATLIS (Acute Test of Limb Isometric Strength), a new test to measure muscle strength using a “bowflex-like” chair (pictured below). This will complement the use of the traditional ALSFRS-R (ALS Functional Rating Scale-Revised) methods. The ALS Association was the first to grant support of the ATLIS test. The Amylyx team helped manufacture new ATLIS machines, which were donated the clinical trial sites so that ATLIS could be evaluated for all of the patients in the study.

ATLIS machine-Amylyx-Feb 2018

Specific biomarker measurements are also incorporated into the trial to understand whether AMX0035 hits its intended molecular target and works as designed. The trial was designed to facilitate patient access and engagement, and ease of trial site visits. Data from the trial will be publicly available to the ALS research community after the trial has completed.

“I’m delighted that The ALS Association’s investment in both ATLIS and the central IRB for ALS studies is providing benefit to the trial, said Dr. Lucie Bruijn, chief scientist, The ALS Association. We’re pleased to be a significant sponsor for the trial and to provide the necessary infrastructure support through NCRI and NEALS.”

For more information about the trial, read here.
For more information on how to enroll in the trial and site contact information, click here.

To learn more about our progress and monetary commitments since the 2014 Ice Bucket Challenge, please visit the Ice Bucket Challenge Progress site.

Trial sites:
ALS Center at the Swedish Neuroscience Institute
Seattle, Washington 98122
United States

Barrow Neurological Institute
Phoenix, Arizona 85013
United States

Carol and Frank Morsini Center for Advanced Health Care – University of South Florida
Tampa, Florida 33612
United States

Emory University Hospital
Atlanta, Georgia 30322
United States

Forbes Norris MDA/ALS Research Center – California Pacific Medical Center
San Francisco, California 94114
United States

Hennepin County Medical Center
Minneapolis, Minnesota 55415
United States

Johns Hopkins Hospital
Baltimore, Maryland 21287
United States

Massachusetts General Hospital
Boston, Massachusetts 02114
United States

Mount Sinai Beth Israel
New York, New York 10003
United States

Neurology Associates P.C.
Lincoln, Nebraska 68506
United States

Ochsner Neuroscience Institute
New Orleans, Louisiana 70121
United States

Oregon Health & Science University
Portland, Oregon 97239
United States
Temple University Hospital
Philadelphia, Pennsylvania 19140
United States

Texas Neurology, P.A.
Dallas, Texas 75214
United States

The Ohio State University Wexner Medical Center
Columbus, Ohio 43221
United States

The Penn Comprehensive ALS Center
Philadelphia, Pennsylvania 19107
United States

UC Irvine Medical Center
Orange, California 92868
United States

University of Florida Medical Center
Gainesville, Florida 32610
United States

University of Iowa Hospitals and Clinics
Iowa City, Iowa 52242
United States

University of Kentucky Medical Center
Lexington, Kentucky 40536
United States

University of Massachusetts Memorial Medical Center
Worcester, Massachusetts 01655
United States

University of Michigan Medical Center
Ann Arbor, Michigan 48109
United States

University of Texas Health Science Center at San Antonio
San Antonio, Texas 78229
United States

Wake Forest Baptist Medical Center
Winston-Salem, North Carolina 27157
United States
Washington University Medical Center
Saint Louis, Missouri 63110
United States

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