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
Expansion to APOE3/4 heterozygotes & disease prevention
*Active metabolite of ALZ-801, licensed by Alzheon from Bellus Health/Neurochem in 2013
**Aduhelm Prescribing Information, U.S. FDA 2021
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)
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