# Cellular Prion Protein Targeting Monoclonal Antibody Antagonist as a Therapy for Alzheimer's Disease

> **NIH NIH R44** · ALLYX BIOSCIENCES, INC. · 2020 · $449,995

## Abstract

SUMMARY/ABSTRACT
An estimated 5.6 million patients are currently living with Alzheimer’s disease and have no disease modifying
therapies available. Without any major breakthroughs in identifying transformative treatments, the burden of
patient care costs to the United States healthcare system is expected to skyrocket as our population ages. The
clinical pipeline in Alzheimer’s disease has been met with high profile failures and setbacks, mostly with
technologies that target removal or lowering of amyloid-β plaques. Novel methods for treating this disease are
desperately needed, specifically those that target mechanisms independent of amyloid-β removal.
Cellular prion protein has been identified as the primary receptor for toxic amyloid-β oligomers and is located at
the synapses of neurons. This receptor has been validated as a key mediator of synapse loss in transgenic
animal models of Alzheimer’s disease. Our product is a high affinity monoclonal antibody inhibitor of cellular
prion protein. By blocking the activity of cellular prion protein, we aim to deliver a disease modifying therapeutic
capable of preventing synapse loss and thereby preserving both cognitive and functional abilities for patients
with Alzheimer’s disease.
Preclinical evaluation of this antibody has been conducted with the murine version of the antibody product. Our
goal for the Phase I segment of this award is to express, purify and characterize the activity of the human
antibody form of the product to ensure drug-like properties in vitro. This will provide validation for large-scale
production, pharmacokinetic and safety evaluation to be carried out during the Phase II segment. The overall
goal of this SBIR is to provide preliminary toxicology data to predict that brain exposure reaches therapeutic
levels at safe and feasible human doses.
The commercial opportunity for a disease modifying therapeutic in Alzheimer’s disease is immense. Peak
worldwide revenues for existing symptomatic Alzheimer’s treatments reached $4.3 B USD. A disease
modifying therapy is expected to reach sales of >$10 B USD per year. Allyx Therapeutics will establish clinical
proof-of-concept in a Phase II clinical trial before partnering with a large pharmaceutical partner for Phase III
development and commercialization.

## Key facts

- **NIH application ID:** 10516260
- **Project number:** 7R44AG069559-02
- **Recipient organization:** ALLYX BIOSCIENCES, INC.
- **Principal Investigator:** Timothy R Siegert
- **Activity code:** R44 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $449,995
- **Award type:** 7
- **Project period:** 2020-09-17 → 2023-05-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10516260

## Citation

> US National Institutes of Health, RePORTER application 10516260, Cellular Prion Protein Targeting Monoclonal Antibody Antagonist as a Therapy for Alzheimer's Disease (7R44AG069559-02). Retrieved via AI Analytics 2026-05-24 from https://api.ai-analytics.org/grant/nih/10516260. Licensed CC0.

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