Alzheon Inc.


Alzheon is a pioneering biopharmaceutical company dedicated to developing innovative treatments for neurodegenerative disorders, especially Alzheimer’s disease. They focus on inhibiting the formation of neurotoxic soluble amyloid oligomers, which are key drivers of Alzheimer’s pathology. Their lead candidate, ALZ-801, is an oral, brain-penetrant drug designed to prevent amyloid oligomer formation, slow disease progression, and preserve cognition. The company aims to provide safe, effective, and accessible therapies that address the underlying causes of Alzheimer’s and other neurodegenerative diseases, with a focus on precision medicine approaches targeting high-risk genetic populations such as APOE4/4 homozygotes. They are actively advancing their pipeline, including a promising drug candidate that was previously tested but failed a major study in 2009, which they have since rebuilt to improve delivery and efficacy.

Industries

biotechnology
clinical-trials
health-care
therapeutics

Nr. of Employees

small (1-50)


Patents

Sulfopropanoic acid derivatives for treating neurodegenerative disorders

US-12285400-B2

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Compositions and methods for treating and preventing neurodegenerative disorders

US-12187668-B2

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Methods for treating neurodegenerative disorders

US-12083082-B2

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Methods for treating neurodegenerative disorders

US-12036208-B2

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Methods of treating neurodegenerative disorders in a particular population

US-11191742-B2

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Methods for treating neurodegenerative disorders

US-11077095-B2

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Products

Oral anti-amyloid-oligomer small-molecule prodrug

An oral small-molecule prodrug formulation intended to deliver an active anti-aggregation metabolite to the brain at sustained levels to inhibit formation of neurotoxic soluble amyloid oligomers for early symptomatic neurodegenerative disease.


Services

Design and conduct of multi-site Phase 2/3 clinical trials with genotype-guided enrollment, integrated biomarker and neuroimaging collection, and long-term extension studies.

Preclinical discovery and translational development of small molecules aimed at inhibiting protein oligomer formation, including formulation optimization, PK characterization, and linking biomarker changes to clinical outcomes.

Expertise Areas

  • Clinical trial management for neurodegenerative diseases (Phase 2/3)
  • Precision-medicine trial design and genotype-guided enrollment
  • Biomarker-driven translational research (CSF/plasma amyloid-beta and imaging)
  • Small-molecule discovery for protein aggregation disorders
  • Show More (6)

Key Technologies

  • Oral small-molecule prodrug formulation
  • Selective inhibition of soluble amyloid oligomers
  • Cerebrospinal fluid and plasma amyloid-beta assays
  • MRI volumetric analysis
  • Show More (5)

News & Updates

Alzheon announced the appointment of Susan Flint as Vice President of Clinical Operations, Patrick Kesslak, PhD, as Vice President of Clinical Development & Medical Affairs, and Erwan de Naurois as Vice President of Finance to support the completion of the APOLLOE4 Phase 3 trial and expand its product portfolio.

Alzheon announced it raised $100 million in Series E funding to support the development and commercialization of ALZ-801, including completing the pivotal Phase 3 trial and regulatory preparations.

Alzheon’s experimental drug ALZ-801, a disease-modifying oral treatment targeting amyloid proteins, is expected to be available in about two years. It offers a more user-friendly oral delivery compared to infusion-based therapies and has shown promising results in clinical trials.

ALZ-801 has completed enrollment in its Phase 3 APOLLOE4 trial, evaluating efficacy in APOE4/4 homozygotes with early Alzheimer’s disease. Results are expected in 2024, with plans for NDA submission and potential approval.


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