BRINGING BACK
THE BALANCE

Our vision is to re-balance the dysfunctional T cell immune system of patients suffering from autoinflammatory and autoimmune diseases with activated Treg.

Treg

Inflammatory T cells

Treg

Inflammatory T cells

What happens in autoinflammatory and autoimmune diseases?

Inflammatory T cells are part of a healthy immune system. When the system is out of balance, they attack the body’s own cells and thus substantially contribute to autoinflammatory and autoimmune diseases. Dysfunctional Treg are unable to keep the effector T cells in balance.

How relevant is this?

In Europe and USA, more than 25,000 patients per year receive an allogeneic stem cell transplantation. Up to 60% of those patients develop acute GvHD, a life-threatening side effect for which effective treatment is limited.

Autoimmune diseases are very common, affecting about one in ten individuals. Multiple sclerosis (MS) is one of the ten most common autoimmune diseases. The number of patients worldwide has grown to 2.9 million in 2023 with healthcare costs in the US of USD 85.4 billion in 2019.

In GvHD, donor lymphocytes react against tissue antigens in the recipient and cause severe organ and tissue damage, that often takes a chronic course. GvHD is primarily triggered by the donor’s T effector cells, immune cells whose activity is controlled by Treg.

Various therapeutic approaches such as relapse-preventive medications, symptomatic treatments, and rehabilitative measures can help slow the progression of the disease. However, there is no cure for MS. In addition, many of these treatments bear certain risks, especially for women in child-bearing age. Taken together, there is a high medical need for curative new approaches in MS.

Studies suggest that a dysfunction or impaired maturation of Treg cells may contribute to the development of the disease.

Autoinflammatory and autoimmune diseases are being treated by suppressing the immune system. This can lower the immune system’s ability to perform its function in protecting the body, leading to infections or diseases. Immunosuppressants often need to be taken life-long.

OUR TECHNOLOGY

Treg therapies provide a solution to bring back the balance in the T cell immune system in a more gentle and sustainable way. They can reprogram the T cell system without excessive immunosuppression. However, reaching sufficient numbers of Treg is challenging. In addition, many Treg approaches apply genetic manipulation. These processes usually take several weeks, are costly and bear risks.

At ActiTrexx, we have developed a novel platform approach that is different. We don‘t require high numbers of Treg that have been generated by expansion. We take well defined numbers of Treg and activate them with a recombinant CD4-binding Treg activator. Our Treg activator has superior efficacy in Treg activation and the resulting activated Treg show significantly higher suppressive function than resting Treg. The technical platform can be applied ex-vivo or in-vivo for various cellular and biologic products.

With activated Treg, patients will benefit from lowered immunosuppressive burden, receive a curative treatment and an increased quality of life.

Treg

CD4

Treg Activator

Activated Treg

Treg

CD4

Treg
Activator

Activated Treg

ACTILEUCEL

Our lead product Actileucel is a first-in-class allogeneic Treg product.
It does not require tissue (HLA) matching, making it a standardized one-product-fits-all drug.

Activated Treg

GvHD

Multiple Sclerosis

Other autoimmune diseases

The advantages of Actileucel are the following:

  • Standardized
    No personalized medicine. HLA-nonmatched donors. One drug fits all.

  • Fast development
    Highly efficient clinical development started with Actileucel in GvHD.

  • Cost efficient
    24h vein-to-vein time. No genetic engineering, no expansion.

  • Proven
    Pre-clinically proven in mouse models for multi-organ GvHD and asthma. Clinically applied in ongoing phase Ib/II trial for Actileucel (GvHD).

  • Effective
    Unparalleled suppressive function.

OUR PROCESS

Starting from leukapheresis material, Treg are being isolated and activated in a proprietary process. Our process is highly automated and requires no cell expansion or genetic manipulation. The result is an outstandingly pure cellular product with high suppressive function, infusion ready within less than 24 hours.

Donation
of leukocytes

Separation
of Treg

Activation
of Treg

Quality control
of Actileucel

Infusion
of Actileucel

Actileucel is classified by EMA as an Advanced Therapy Medicinal Product (ATMP). The product is a safe and efficient treatment option that is currently in development for patients having received a stem cell transplantation.

Actileucel is being tested in clinical phase Ib/II to prevent GvHD. Further development steps include application of Actileucel for treatment of multiple sclerosis (MS).