Amyloid Oligomers in AD 2018-03-19T13:36:30+00:00

ALZ-801

An Improved Prodrug Formulation

Our lead product candidate, ALZ-801, is a patented, orally administered prodrug of tramiprosate that is designed to inhibit Aβ oligomer formation, a key driver of AD.

ALZ-801 was developed to improve gastrointestinal tolerability and the PK profile of tramiprosate by allowing ALZ-801 to be absorbed through the gut wall in a prodrug inactive form and metabolized into active tramiprosate after absorption into the bloodstream. The data from our oral ALZ-801 Phase 1 clinical program showed favorable safety, improved gastrointestinal tolerability and more consistent plasma levels.

Improvements Enhance Prospects for ALZ-801 Efficacy & Safety

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Accelerating the Development of ALZ-801

We have built our ALZ-801 development program upon the extensive preclinical and clinical data set for tramiprosate. ALZ-801 has an improved PK profile, and through our focus on APOE4/4 homozygous patients who carry a higher Aβ burden, believe that patients will be less likely to be misdiagnosed with AD.

ALZ-801 Biological Pathway

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In October 2017, ALZ-801 was granted Fast Track designation by the U.S. Food and Drug Administration for the treatment of AD. We plan to initiate a Phase 3 trial for ALZ-801 in APOE4/4 homozygous patients with mild AD in 2018. This trial will be an 18-month, double-blind, placebo-controlled Phase 3 trial in APOE4/4 homozygous patients with mild AD. We plan to recruit patients for this study in North America and internationally, and to power the trial to detect efficacy on the co-primary outcomes of ADAS-cog and CDR-SB.

ALZ-801 Phase 3 Study in APOE4/4 with Mild AD

FDA-Accepted Study Design in APOE4/4 AD Patients

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Our Program Builds on Recent Learnings

We believe ALZ-801 has the potential to be differentiated from other emerging therapies targeting AD pathology due to its novel mechanism of action, oral mode of administration, potential efficacy in a genetically-targeted population and observed favorable safety profile. If our development program is successful and ALZ-801 is approved, we believe it has the potential to be among the first drugs to intervene in an underlying mechanism of AD.

Problem Advantage of ALZ-801 Lessons from Other Anti-Amyloid Agents
Efficacy data not supported MOA translates to potential
clinical efficacy target dose
Lack of alignment of MOA with clinical data ( except aducanumab )
Lack of inhibition of Aβ oligomers Inhibits formation of amyloid oligomers Insufficient targeting of Aβ oligomers ( except aducanumab )
Poor brain penetration ~40% Low penetration for antibodies
Lack of target engagement CSF Aβ42 reduction & decrease
in hippocampal atrophy
Plaque lowering on PET imaging
Poorly defined patient population APOE4/4 Mild AD patients APOE4 carriers & non-carriers
Development of vasogenic edema None observed At risk