Our technology
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We are developing a unique peptide-aptamer, designed to stimulate suppressive regulatory B and T cells. We target regulatory B and T cells that are specific for citrullinated antigens, a hallmark of rheumatoid arthritis (RA). By boosting regulatory B and T cells that recognize citrullinated antigens, we direct their suppressive functions towards the pathogenic RA-specific autoimmune response. This happens while maintaining the capacity of the immune system to respond to foreign antigens such as infections and cancer. Our peptide-aptamer is incorporated into a biodegradable nanoparticle and the whole molecule consisting of peptide-aptamer and nanoparticle is named APT001. The peptide-aptamer is incorporated into the nanoparticle to stabilize it in vivo and to facilitate uptake by immune cells. These immune cells can then restore tolerance in autoimmunity, intervening at the root cause of rheumatoid arthritis. We have in 4 consecutive studies in rodent models of RA demonstrated strong and unforeseen sustained efficacy, suggesting the potential of an immunological reset and restoration of immune homeostasis.
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​Our upcoming studies in animal models of rheumatoid arthritis (RA), systemic lupus erythematosus (SLE), Type 1 Diabetes and more will deliver further evidence for the Mode of Action of our platform as well as uncover future therapy areas as targets for Aptol Pharma.​
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A unique solution
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Current treatment of RA can be divided into three major groups: steroids, non-steroid anti-inflammatory drugs, and disease modifying antirheumatic drugs. Steroids and non-steroid anti-inflammatory drugs are non-disease-specific drugs with severe long term side effects. Therefore, these drugs are used only in limited time and doses.
Biological DMARDs, including monoclonal antibodies targeting tumor necrosis factor, work by inhibiting components of the immune system and hence cause adverse side effects related to immune suppression. Therefore, these drugs are also not suitable for preventing disease progression in the
pre-clinical and early-stage phases of RA, and will rarely cure disease or induce long term remission when stopping treatment.
Our drug candidate is a completely novel approach to RA treatment that targets disease-specific processes and in this way reduces inflammation without affecting the immune system.
This approach (antigen-specific therapy) is being actively explored by others in other autoimmune conditions such as allergy, celiac disease and type 1 diabetes.
The antigen specific therapy approach makes APT001 a first in class drug candidate for the treatment of RA.
The peptide-aptamer technology
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Our technology is based on peptide-aptamers, which are highly attractive assets with the ability to refold into an active conformation.
Additionally, our technology presents:
- A unique mode-of-action including autoantibody targeting and antigen-specific tolerance
- High specificity
- Potential of technology extension to other autoimmune diseases
- Standard, inexpensive synthetic protocols
- High stability under storage
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Read more in our original paper:
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