Alzheon presents positive results of ALZ-801 clinical studies

Pharmacokinetic and safety data from Phase Ib studies with ALZ-801, an optimized prodrug of tramiprosate, presented at the Alzheimer's Association International Conference.

The Phase Ib study results demonstrated favourable gastrointestinal tolerability, consistent and sustained plasma levels, and allowed selection of an ALZ-801 clinical dose that is bioequivalent to the tramiprosate dose that showed promising efficacy in prior Phase III trials. These data are being presented for the first time at the Alzheimer’s Association International Conference (AAIC), held 24–28 July in Toronto, Canada. Alzheon is also making a late-breaking presentation with new analyses of the prior tramiprosate Phase III studies that include: 1) pooled safety analyses from 2,025 subjects across the two studies, 2) centralized assessment of brain MRI for occurrence of vasogenic edema or amyloid-related imaging abnormalities (ARIA), and 3) efficacy analyses in the apolipoprotein E4 (APOE4) subgroups, suggesting promising efficacy and favorable safety profile in Mild and Moderate AD patients with the APOE4/4 genotype.

“We now have all of the necessary clinical, toxicology and tablet data to start the pivotal Phase 3 program with ALZ-801, an oral, amyloid-targeting drug candidate for the treatment of patients with Mild to Moderate Alzheimer’s disease,” said Martin Tolar, Founder, President and CEO of Alzheon. “Our body of ALZ-801 data and analyses suggest a well-differentiated and potentially transformative Alzheimer’s medicine: an oral tablet that targets the underlying amyloid pathology in Alzheimer’s, with compelling clinical efficacy in patients who are apolipoprotein E4 carriers. For the first approval for ALZ-801, we are preparing to initiate the ALZ-801 Phase III registration program in early 2017, focusing on the APOE4/4 homozygous patients that showed the most robust efficacy and represent a genetically-defined population with high unmet medical need.”

Alzheon conducted the two Phase Ib clinical studies of ALZ-801 in healthy elderly volunteers: a single dose tablet bioequivalence study, and a multiple-ascending dose safety, tolerability and pharmacokinetic study. These studies showed that ALZ-801 demonstrates favourable pharmacokinetic (PK) properties, including steady target plasma levels with low inter-subject variability, as well as sustained plasma concentrations up to 24 hours. ALZ-801 showed an equivalent or improved PK profile compared to tramiprosate, including plasma exposures and dose proportionality, thereby allowing bioequivalence and bridging to the prior clinical data with tramiprosate. ALZ-801 also showed a favourable safety and tolerability profile, including improved gastrointestinal tolerability with lower incidence of nausea and vomiting compared to previous studies with tramiprosate.

“We are extremely pleased with these clinical results for ALZ‐801, which show that we have successfully optimized the pharmaceutical, pharmacokinetic and tolerability profile so that ALZ-801 is well positioned to advance into Phase III pivotal studies, and to potentially offer an important advance in AD treatment. In addition, we are excited to show that we have developed a simple, yet superior immediate-release tablet dose that can be used in the Phase III program and subsequently in broad commercial development,” said John Hey, Chief Scientific Officer of Alzheon.