American clinical-stage biopharmaceutical company Alzheon has recently presented promising interim results from its Phase II Biomarker Trial (NCT04693520) of its oral, brain-penetrant, amyloid-oligomer inhibitor ALZ-801 (valiltramiprosate) at the American Academy of Neurology 2024 annual meeting. ALZ-801 has the potential to be the first oral Aβ-targeting therapy to be approved in the US and EU in early AD, according to GlobalData, a data and analytics company.
Erela Dana, Director of Neurology at GlobalData, comments: “Amyloid beta (Aβ)-targeting therapies hold promise, with this mechanism of action (MOA) recently seeing some success with the FDA’s approval of Biogen’s anti-Aβ monoclonal antibody (mAb) Aduhelm (aducanumab) in 2021, followed by Eisai/Biogen’s anti-Aβ mAb Leqembi (lecanemab) in January 2023.”
ALZ-801 is currently in Phase III, being evaluated for the treatment of apolipoprotein E 4 (APOE4) carrier patients with early AD.
Key opinion leaders (KOLs) previously interviewed by GlobalData highlight that the ALZ-801 target population of early AD patients, who are homozygous for the APOE4 allele, represent a subgroup that has a strong unmet need for novel disease-modifying approaches.
So far, the results over two years of treatment with ALZ-801 have been presented of the four-year open-label biomarker study. Overall, ALZ-801 treatment demonstrated sustained improvement over two years in plasma biomarkers, hippocampal volume, and cognitive effects in the target population.
Dana comments: “There is a strong correlation between the biomarker outcomes and ALZ-801’s cognitive benefits. The presented biomarker results support the disease-modifying effects of ALZ-801 in APOE4 carriers with early AD with promising clinical efficacy and favorable safety, with no observed cases of amyloid-related imaging abnormalities related to underlying vasogenic edema (ARIA-E), a known side effect of the anti-Aβ mAbs, to date.”
Further to its Phase II biomarker study, Alzheon plans to announce the topline results of its APOLLOE4 (NCT04770220) Phase III trial in Q3 2024, with a potential NDA submission to the US FDA later this year.
Dana concludes: “Should Phase III outcomes follow the positive outcomes from the two-year Phase II biomarker data and support the hypotheses that by acting upstream of anti-Aβ mAbs, ALZ-801 could provide significantly improved amyloid clearance, it would pave the way for Alzheon to launch its AD asset in 2025 as the first oral anti-Aβ DMT in the US.”
