ALZ-801/valiltramiprosate potentially the first oral disease-modifying treatment for Alzheimer’s disease

ALZ-801 is well differentiated from plaque-clearing antibodies in development for Alzheimer’s disease, due to its novel mechanism of action, oral mode of administration, and potential efficacy in a genetically-targeted population. If our development program is successful and ALZ-801 is approved, it could be among the first disease-modifying drugs for Alzheimer’s disease.

ALZ-801 precision medicine development approach

Fast-to-patient strategy seeking first approval in APOE4/4 homozygotes with Early AD following completion of APOLLOE4 Phase 3 trial

  • Leverage tramiprosate* safety & efficacy database, bridging to ALZ-801 NDA
  • Efficient, well-powered confirmatory trial with NIA grant support
  • Precision medicine focus on patients with high risk of progression
  • Biomarker integration for confidence in success

Expansion to APOE3/4 heterozygotes & disease prevention

  • Phase 2 AD biomarker data to accelerate Phase 3 in APOE3/4 heterozygotes
  • Disease progression biomarkers as surrogates of efficacy to enable Phase 3 & supplemental NDA for AD prevention indication
  • Aducanumab biomarker-based approval established value of AD biomarkers**
*Active metabolite of ALZ-801, licensed by Alzheon from Bellus Health/Neurochem in 2013
**Aduhelm Prescribing Information, U.S. FDA 2021

Click to learn about APOLLOE4

Phase 3 study to evaluate efficacy of ALZ-801 in APOE4/4 Early AD subjects

Initial focus on APOE4/4 patients based on ALZ-801 mechanism of action

  • Greater diagnostic accuracy because >95% are amyloid & tau positive*
  • Strong biologic rationale: 3-fold higher levels of toxic Aβ oligomers in brain compared to non-carriers
  • Alzheimer’s clinical onset ~10 years earlier compared to non-carriers
  • Major unmet medical need
*ALZ-801 Phase 2 biomarker trial includes baseline CSF screening in APOE4/4 and APOE3/4 AD patients (data on file)

APOE4/4 homozygous

ALZ-801/valiltramiprosate oral tablet summary

Precision medicine Phase 3 program in Alzheimer’s

  • Differentiated product profile with first oral tablet advantage
    • Potentially robust disease-modifying efficacy & favorable long-term safety
    • No increased risk of ARIA in APOE4 carriers observed in trials to-date
  • Clinically validated approach with high probability of success
    • Precision medicine focus of confirmatory APOLLOE4 Phase 3 trial
    • Biomarker-enabled program for accelerated development
    • FDA Fast Track designation & substantially de-risked safety profile
  • Expansion to APOE4 carriers reaches over 13 million patients
    • APOE4 carriers with Early AD represent two-thirds of all AD patients
    • Prevention of AD symptoms
  • Positive 24-month Phase 2 results in Early AD supports differentiated profile of oral ALZ-801 & Phase 3 data readout in 3Q 2024

 

ARIA – Amyloid Related Imaging Abnormalities, brain edema & microhemorrhages

ALZ-801 Phase 3 trial in APOE4/4 patients with Early AD

APOLLOE4 pivotal trial fully enrolled with 325 subjects

ALZ-801 Alzheimer's medicine

Reflects current best internal estimates, subject to change; NDA – New Drug Application; sNDA – Supplemental New Drug Application

ALZ-801/valiltramiprosate development strategy