ALBRIOZA Commercial Launch
CENTAUR Trial Results
Statistically Significant Functional Benefit as Measured by the ALSFRS-R, the Gold Standard Clinical Scale in ALS
ALSFRS-R Estimate
35.0-
32.5
30.5
HHH
1A for
T
H+
H
HA
H
Trial of Sodium Phenylbutyrate-Taurursodiol
for Amyotrophic Lateral Sclerosis
Published in The New England Journal of Medicine
Sabrina Paganoni, M.D., Ph.D., Eric A. Macklin, Ph.D., Suzanne Hendrix, Ph.D., James D. Berry, M.D., Michael A.
Elliott, M.D., Samuel Maiser, M.D., Chafic Karam, M.D., James B. Caress, M.D., Margaret A. Owegi, D.O., Adam
Quick, M.D., James Wymer, M.D., Ph.D., Stephen A. Goutman, M.D., Daragh Heitzman, M.D., Terry Heiman-
Patterson, M.D., Carlayne E. Jackson, M.D., Colin Quinn, M.D., Jeffrey D. Rothstein, M.D., Ph.D., Edward J.
Kasarskis, M.D., Ph.D., Jonathan Katz, M.D., Liberty Jenkins, M.D., Shafeeq Ladha, M.D., Timothy M. Miller, M.D.,
Ph.D., Stephen N. Scelsa, M.D., Tuan H. Vu, M.D., Christina N. Fournier, M.D., Jonathan D. Glass, M.D., Kristin M.
Johnson, D.O., Andrea Swenson, M.D., Namita A. Goyal, M.D., Gary L. Pattee, M.D., Patricia L. Andres, M.S., D.P.T.,
Suma Babu, M.B., B.S., M.P.H., Marianne Chase, B.A., Derek Dagostino, B.A., Samuel P. Dickson, Ph.D., Noel
Ellison, M.S., Meghan Hall, M.S., Kent Hendrix, B.S., Gale Kittle, R.N., M.P.H., Michelle McGovern, B.S., Joseph
Ostrow, B.S., Lindsay Pothier, B.A., Rebecca Randall, M.S., R.D., Jeremy M. Shefner, M.D., Ph.D., Alexander V.
Sherman, M.Sc., Eric Tustison, B.A., Prasha Vigneswaran, M.S., Jason Walker, B.S., Hong Yu, M.S., James Chan,
M.A., Janet Wittes, Ph.D., Joshua Cohen, B.S.E., Justin Klee, Sc. B., Kent Leslie, M.S., Rudolph E. Tanzi, Ph.D.,
Walter Gilbert, Ph.D., Patrick D. Yeramian, M.D., David Schoenfeld, Ph.D., and Merit E. Cudkowicz, M.D.
27.5
No. of Participants¹
0
AMX0035
87
CO
6
12
Week
-
84
79
79
75
70
70
Placebo
48
48
48
18
72
67
24
68
88
Ho
64
44
44
44
43
39
38
38
2
AMX0035
Placebo
37
37
2.32 point difference, p=0.03
“Each category in the ALSFRS-R seems
clinically important, and because each
domain includes only five levels that span
O (cannot do) to 4 (normal), prevention of
even 1 unit of worsening in a single
domain seems meaningful and desirable
for individuals with ALS".3
References: 1. Two participants did not have follow-up efficacy assessments and were not included in the efficacy population (modified intention to treat n=135). 2. 77% of participants were on Riluzole or edaravone at or
prior to study entry. 3. FDA Center for Drug Evaluation and Research, Application Number 2091760rig1s000, Office Director Memo, Ellis F. Unger, MD, May 4, 2017.
Paganoni S, et al. New Eng J Med. 2020.
AMYLYX | 6View entire presentation