MYASTERIX

MYASTERIX (2013-2018)

Clinical safety, immunogenicity and efficacy of a therapeutic vaccine that combines peptides mimicking antigen receptors on autoimmune B and T cells associated with myastenia gravis

 

The MYASTERIX project will advance a therapeutic vaccine candidate (designated orphan drug) indicated for the autoimmune disease myastenia gravis (MG) to clinical proof of concept studies.

 

MG is caused by T cell dependent antibodies that bind to and deplete acetylcholine receptors (AChR) at neuromuscular junctions causing muscle weakness by interfering with neuromuscular transmission and junction architecture. The vaccine candidate comprises two synthetic peptides designed to generate antibodies that bind to autoantibodies and T-cell receptors associated with MG.

These peptides prevented or improved muscle fatigue in a rat model of MG and increased the remission rate to 75% in pet dogs (compared to 17% natural remission rate in historical controls). In both models, administratiion of the peptides resulted in reduced titres of anti-AChR antibodies and lower numbers of anti-AChR T-dellls, based on the induction of antibodies that bound to the corresponding B and T cell antigen receptors. These results suggest that similar antigen receptor mimetic vaccination approaches could drive autoimmune diseases like MG into long-term remission.

The objecitves of the project are to manufacutre toxicology and clinical batches of the vaccine human formulation based on already developed and tested standard operating procedures, to carry out stability and regulatory toxicity testing of the GMP product, to conduct phase I and subsequently phase II clinical trials to demonstrate safety, tolerability and proof of mechanism of acton/concept of the therapeutic vaccine.

The impact on MG patients will´be to offer a targeted therapeutic approach requiring only three injections, bringing significant and lasting improvement or even a cure. MG is a model for many autoimmune deseases and the concept of targeted therapeutic vaccines could lead to anew class of drugs for the treatment of autoimmune diseases more generally, with a significant impact on innovation, competitiveness and society.

 

Our project partners are

- CuraVac Europe SPRL, a Belgian SME specialised in therapeutic vaccines for autoimmune diseases and scientific coordinator

- Leiden University Medical Centre, a referral site for myastenia gravis in the Netherlands

- Aepodia, a Belgian CRO with expertise in First in Man and early phase clinical trials

- Inserm Transfert, a technology transfer and management company based in France

 

This project has received funding from the European Union´s Seventh Framework Programme for research, technological development and demonstration under grant agreement no. 602420.

 

 

 

 

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