Alzheon Announces Initiation of Clinical Studies with ALZ-801 Bridging to Pivotal Phase 3 Study in Alzheimer’s Disease

Phase 1b Multiple Dose-Ranging Study and Tablet Bioequivalence Study
Will Provide Clinical Dose and Tablet Formulation Data to Support
ALZ-801 Phase 3 Program

FRAMINGHAM, Mass.–(BUSINESS WIRE)–Alzheon, Inc., a clinical-stage biopharmaceutical company focused on
developing new medicines for patients suffering from Alzheimer’s disease
and other neurological and psychiatric disorders, today announced the
initiation of two clinical studies with ALZ-801. ALZ-801 is a prodrug of
tramiprosate, which is a first-in-class small molecule inhibitor of
amyloid formation and neurotoxicity, being developed for the treatment
of Alzheimer’s disease. These studies will provide final clinical data
necessary to progress ALZ-801 into the Phase 3 program.

“With the robust body of existing positive clinical data in hand, we
believe these studies represent the last clinical steps before we start
the pivotal Phase 3 clinical study with our amyloid-targeting drug
candidate ALZ-801 in Alzheimer’s disease,” explained Martin Tolar, MD,
PhD, Founder, President and Chief Executive Officer of Alzheon.
“Building on the existing positive results related to ALZ-801 which
include the extensive clinical evidence from its active compound
tramiprosate, we have designed ALZ-801 to offer a unique approach and
potentially transformative treatment for patients with Alzheimer’s
disease. We are excited about the prospects for ALZ-801, as we have
optimized its pharmacokinetic and tolerability profile, and designed our
clinical plan building on the subgroup analysis of the Phase 3 studies
with tramiprosate that showed compelling efficacy results in
apolipoprotein E4 positive Alzheimer’s disease subjects.”

The Phase 1b multiple dose-ranging trial is a randomized, double-blind,
placebo-controlled, multiple ascending dose study to evaluate the
safety, tolerability and pharmacokinetics of oral ALZ-801 in healthy
elderly volunteers.

The tablet bioequivalence Phase 1b study is a solid dose formulation
study to examine the pharmacokinetics and food effect of a prototype
tablet formulation with a single oral dose of ALZ-801 in healthy elderly
volunteers, to guide development of final drug product formulation to be
used in the Phase 3 trial and, ultimately, for commercial development.

Completion of these two clinical studies is expected by 1Q 2016.

About Alzheon
Alzheon, Inc. is committed to developing
innovative medicines for patients suffering from Alzheimer’s disease and
other neurological and psychiatric disorders. Our lead clinical
candidate, ALZ-801, is a first in-class, oral inhibitor of amyloid
aggregation and neurotoxicity – hallmarks of Alzheimer’s disease.
ALZ-801 is an optimized, novel prodrug that builds on the established
safety and efficacy profile of the active compound in clinical trials of
more than 2,000 patients. Our mission is to apply our discovery platform
and development experience to unlock new classes of therapeutics that
make a difference in patients’ lives by directly addressing the
underlying pathology of devastating neurodegenerative disorders. For
more information, please visit


For Alzheon, Inc.
In the U.S.
Yates Network
Kathryn Morris, 845-635-9828

Mike Sinclair, +44 (0)20 7318 2955