Apt001 - Lead Candidate For RA | Aptol Pharma

Our technology

We have developed a unique peptide-aptamer that is designed to induce tolerance and/or suppress citrulline-specific T and B cells and ACPAs.

We incorporate the aptamer into biodegradable nanoparticles that are tuned in size to be taken up by immune cells (specifically macrophages and neutrophil granulocytes).

A schematic structure of the APT001 aptamer-nanoparticle drug candidate is shown in the figure below:

Our upcoming study will verify the following steps of the mode of action for APT001:

Macrophages deliver APT001 into inflamed synovial fluid where it is released.
As a result, our aptamer-nanoparticle binds and prevent the inflammatory activity of ACPA.
Moreover, aptamer-nanoparticle potentially causes tolerance which is a path to stable remission.

A unique solution

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 aptamer technology

Our technology is based on 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