Alzheon to Present Phase 2 Biomarker and Breakthrough Clinical Data for ALZ-801 (Valiltramiprosate) Oral Tablet at the 2022 Alzheimer’s Association International Conference

FRAMINGHAM, Mass.–(BUSINESS WIRE)– Alzheon, Inc.a clinical-stage biopharmaceutical company developing a broad portfolio of product candidates and diagnostic tests for patients suffering from Alzheimer’s disease (AD) and other neurodegenerative disorders, announced today that it will present at the conference 2022 Alzheimer Association International Conference (AAIC) to be July 31-August 4, 2022 in San Diego, California, USA.

Alzheon’s Chief Medical Officer, Susan Abushakra, MD, will present from the podium at the AAIC conference on Sunday, July 31, 2022, at 8 a.m. PT or 11 a.m. ET. This oral presentation will be included in the Hybrid Developing Topics 1 session.

The presentation of the AAIC: Effects of oral ALZ-801, an amyloid oligomer inhibitor, on plasma biomarkers in APOE4 carriers with early-stage Alzheimer’s disease: results from the six-month interim analysis a phase 2 biomarker study, will provide the opportunity to hear Dr. Abushakra discuss the development strategy and path to New Drug Application (NDA) for the ALZ-801 (valiltramiprosate) program. The presentation will also review the mechanism of action of ALZ-801 anti-amyloid oligomer, interim biomarker data from the 6-month interim analysis of the Phase 2 biomarker study fully registered from Alzheon, and updates on the ongoing pivotal Phase 3 APOLLOE4 trial evaluating ALZ-801 oral tablet in Alzheimer’s patients.

“Alzheon has developed a novel precision medicine approach for Alzheimer’s disease by targeting neurotoxic amyloid oligomers. Now, very promising ALZ-801 biomarker data in patients with early Alzheimer’s disease, who are carriers of APOE4, provide further support for this approach and the potential for robust efficacy with convenience. of an oral tablet,” said Susan Abushakra, MD, Aljeon’s chief medical officer. “ALZ-801 significantly reduced p-tau181 plasma concentrations over 6 months of treatment. Importantly, rather than simply slowing cognitive decline as seen with other anti-amyloid agents, subjects treated with ALZ-801 showed significant improvement in memory from baseline over 6 months in parallel with p -tau181 reduction. Combined with a favorable safety profile and absence of vasogenic edema events in ALZ-801 studies, our new biomarker data and promising cognitive benefits support the disease-modifying effect of ALZ -801 in patients with Alzheimer’s disease. These data and ongoing studies position ALZ-801 as the first oral agent capable of slowing or even stopping and preventing Alzheimer’s disease in patients and healthy individuals at risk of contracting the disease. »

ALZ-801 (valiltramiprosate) is an oral agent in Phase 3 development as a modifying therapy for Alzheimer’s disease that blocks the formation of neurotoxic soluble amyloid oligomers that lead to cognitive decline in patients with Alzheimer’s disease. Alzheimers. In mechanism of action studies, ALZ-801 has been shown to completely inhibit amyloid oligomer formation at the Phase 3 clinical dose. ALZ-801 has demonstrated robust efficacy and favorable safety potential in the population at high risk of Alzheimer’s disease – patients carrying two copies of the apolipoprotein ε4 allele (APOE4/4).

“Alzheon has made tremendous progress over the past year, during which we initiated the APOLLOE4 Phase 3 study, reported industry-leading disease-modifying effects through our trial of phase 2 biomarker on oral ALZ-801 in Alzheimer’s disease patients, and initiated a collaboration to commercialize a diagnostic that can measure toxic forms of amyloid in the human brain,” said Martin Tolar, MD, PhD, Founder, President and CEO of Alzheon. “These advances attracted high-profile investors to Alzheon and led to the completion of an oversubscribed Series D round, which, combined with prestigious grants from the National Institute on Aging totaling $51 million, we provides a strong financial position to rapidly complete these trials leading to the NDA submission for oral ALZ-801, as well as the continued expansion of our small molecule product platform for neurodegenerative disorders.

ALZ-801 is in a class of its own as an easy-to-administer oral tablet that has shown the potential for robust efficacy with a favorable safety profile, avoiding the vascular complications of cerebral edema seen with infusions of plaque-removing anti-amyloid antibody. The ongoing, fully recruited Phase 2 biomarker study is evaluating oral ALZ-801 in patients with early AD who carry one or two copies of the ε4 allele of the apolipoprotein E gene. Patients carrying these genotypes together constitute 65 to 70% of patients with Alzheimer’s disease. The APOE4 genotype, the main risk factor for Alzheimer’s disease after aging, is associated with a several times higher brain load of neurotoxic amyloid oligomers.

About the ALZ-801
ALZ-801 is an oral agent in Phase 3 development as a potential disease-modifying treatment for AD.1.3 In mechanism of action studies, ALZ-801 has been shown to completely inhibit the formation of neurotoxic soluble amyloid oligomers at the Phase 3 clinical dose.5.6 ALZ-801 works through a new molecular enveloping mechanism of action to completely block the formation of neurotoxic soluble amyloid oligomers in the human brainseven associated with the onset of cognitive symptoms and progression of AD.1–4 ALZ-801 received Fast Track designation from the United States Food and Drug Administration in 2017. Clinical data for ALZ-801 and the Alzheon Safety Database indicate a favorable safety profile.5–7.9 Initial Phase 3 program for ALZ-801 focuses on patients with early AD with APOE4/4 genotype, with future expansion to treatment and prevention of AD in patients carrying one copy of the APOE4 gene and not carriers.1–4

Phase 3 study ALZ-801 APOLOE4
An Efficacy and Safety Study of ALZ-801 in APOE4/4 Early Alzheimer’s Disease Subjects (NCT04770220): This ongoing study is designed to assess the efficacy, safety, biomarkers and imaging effects of an oral dose of 265 mg twice daily of ALZ-801 in AD subjects with the APOE4/4 genotype, who constitute approximately 15% of Alzheimer’s patients. This is a randomized, double-blind trial comparing oral ALZ-801 to placebo treatment over 78 weeks. The APOLLOE4 trial is supported by a $47 million grant from the National Institute on Aging.

Phase 2 ALZ-801 Biomarker Study
Biomarker Effects of ALZ-801 in APOE4 Carriers With Early Alzheimer’s Disease (NCT04693520): This ongoing study is designed to assess the effects of an oral dose of 265 mg twice daily of ALZ-801 on biomarkers of pathology Alzheimer’s disease in subjects with early Alzheimer’s disease, who have either the APOE4/4 or APOE3/4 genotypes, which together constitute 65-70% of patients with Alzheimer’s disease. The study also includes evaluation of the clinical efficacy, safety, tolerability and pharmacokinetic profile of ALZ-801 over 104 weeks of treatment.

About Alzheon
Alzheon, Inc. is a clinical-stage biopharmaceutical company developing a broad portfolio of product candidates and diagnostic tests for patients suffering from Alzheimer’s disease and other neurodegenerative disorders. We are committed to developing innovative medicines by directly treating the underlying pathology of neurodegeneration. Our lead clinical candidate for Alzheimer’s disease, ALZ-801, is an oral agent in phase 3 development as a potential modifying therapy for Alzheimer’s disease. ALZ-801 is an oral small molecule that completely blocks the formation of neurotoxic soluble amyloid oligomers in the brain. Our clinical expertise and technology platform are focused on the development of drug candidates and diagnostic assays using a precision medicine approach based on genetic information and individual biomarkers to advance therapies with the greatest impact for patients. patients.

Alzheon Scientific Publications

1 Tolar M, et al: Neurotoxic soluble amyloid oligomers stimulate the pathogenesis of Alzheimer’s disease and represent a clinically validated target to slow disease progression, International Journal of Molecular Sciences, 2021; 22, 6355.

2 Abushakra S, et al: APOE ε4/ε4 homozygotes with early Alzheimer’s disease exhibit accelerated hippocampal atrophy and cortical thinning correlated with cognitive decline, Alzheimer’s and Dementia, 2020; 6: e12117.

3 Tolar M, et al: Aducanumab, Gantenerumab, BAN2401 and ALZ-801 – the first wave of amyloid-targeting drugs for Alzheimer’s disease with near-term approval potential, Alzheimer’s Disease Research and Therapy, 2020; 12:95.

4 Tolar M, et al: The way forward in Alzheimer’s disease therapy: re-evaluating the amyloid cascade hypothesis, Alzheimer and Dementia, 2019; 1-8.

5 Hi JA, et al: Discovery and identification of an endogenous metabolite of tramiprosate and its prodrug ALZ-801 that inhibits beta-amyloid oligomer formation in the human brain, CNS Drugs, 2018; 32(9): 849-861.

6 Hi JA, et al: Clinical pharmacokinetics and safety of ALZ-801, a new tramiprosate prodrug in development for the treatment of Alzheimer’s disease, Clinical Pharmacokinetics, 2018; 57(3): 315–333.

seven Abushakra S, et al: The clinical effects of tramiprosate in APOE4/4 homozygous patients with mild Alzheimer’s disease suggest disease modifying potential, Journal of Alzheimer’s Disease Prevention, 2017; 4(3): 149-156.

8 Kocis P, et al: Elucidate the mechanism of anti-Aβ42 aggregation of tramiprosate in Alzheimer’s disease: integration of molecular analysis methods, pharmacokinetic and clinical data, CNS Drugs, 2017; 31(6): 495-509.

9 Abushakra S, et al: The clinical benefits of tramiprosate in Alzheimer’s disease are associated with a higher number of APOE4 alleles: the “APOE4 gene dose effect” Journal of Alzheimer’s Disease Prevention, 2016; 3(4): 219-228.

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Source: Alzheon, Inc.

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