ALBRIOZA Commercial Launch
Upcoming Pivotal Phase 3 Study in Progressive Supranuclear Palsy (PSP)
PSP is Associated with Tau Protein
Rare neurological disorder that affects body
movements, walking and balance, and eye
movement. Pathology characterized by
widespread neurodegeneration associated
with tau protein deposition in subcortical
regions of the brain.
AMX0035 Demonstrates Potent Impact on Tau4,5
Data from Phase 2 PEGASUS study in Alzheimer's disease
demonstrated that AMX0035 lowered total tau by 64.9 pg/mL and
lowered phosphorylated tau by 14.6 pg/mL at week 24.
Select CSF
Biomarkers
Change from Baseline to
Week 24 LS Mean
Week 24
(95% CI)
p-value
(nominal)
Linear mixed effect
AMX0035
Placebo
Difference between
AMX0035 and Placebo
Estimated prevalence of
* 5-7 in 100,000 worldwide¹
model estimation
PSP is typically fatal
within 5 to 8 years2,3
Total Tau
(pg/mL)
-64.9
8.8
-73.7
(-106.8, -40.7)
<0.0001
Phosphorylated
Tau (pTau181)
(pg/mL)
-14.6
-0.3
-14.4
(-21.5, -7.2)
0.0002
1. Shoeibi A., Olfati N., Litvan I. Frontrunner in translation: Progressive supranuclear palsy. Front. Neurol. 2019;10:1125. doi: 10.3389/fneur.2019.01125. Swallow D.M., Zheng C.S., Counsell C.E. Systematic review of prevalence
studies of progressive supranuclear palsy and corticobasal syndrome. Mov. Disord. Clin. Pract. 2022;9:604-613. doi: 10.1002/mdc3.13489. 2. Williams DR, Lees AJ. Progressive supranuclear palsy: clinicopathological concepts and
diagnostic challenges. Lancet Neurol 2009;8(3):270-279. 3. Guasp M., Molina-Porcel L., Painous C., Caballol N., Camara A., Perez-Soriano A., Sánchez-Gómez A., Garrido A., Muñoz E., Marti M.J. Association of PSP phenotypes with
survival: A brain-bank study. Parkinsonism Relat. Disord. 2021;84:77-81. doi: 10.1016/j.parkreldis.2021.01.015. 4. Analysis conducted with biomarker core lab of the Mass General Institute for Neurodegenerative Disease (MIND) on
CSF samples. Data from Phase 2 PEGASUS trial of AMX0035 for the treatment of Alzheimer's disease. Arnold et al. CTAD 2022. 5. Marie K. Bondulich, Tong Guo, Christopher Meehan, John Manion, Teresa Rodriguez Martin, Jacqueline
C. Mitchell, Tibor Hortobagyi, Natalia Yankova, Virginie Stygelbout, Jean-Pierre Brion, Wendy Noble, Diane P. Hanger, Tauopathy induced by low level expression of a human brain-derived tau fragment in mice is rescued by
phenylbutyrate, Brain, Volume 139, Issue 8, August 2016, Pages 2290-2306, https://doi.org/10.1093/brain/aww137
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