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

Objective

The MYASTERIX project will advance a therapeutic vaccine candidate (designated orphan drug) indicated for the autoimmune disease myasthenia 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, administration of the peptides resulted in reduced titres of anti-AChR antibodies and lower numbers of anti-AChR T-cells, 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 objectives of the project are to manufacture 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 action/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 diseases and the concept of targeted therapeutic vaccines could lead to a new class of drugs for the treatment of autoimmune diseases more generally, with a significant impact on innovation, competitiveness and society.

Call for proposal

FP7-HEALTH-2013-INNOVATION-1
See other projects for this call

Funding Scheme

CP-FP - Small or medium-scale focused research project

Coordinator

INSERM TRANSFERT SA
Address
Rue Watt 7
75013 Paris
France
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
EU contribution
€ 355 284,16
Administrative Contact
Florence Chung (Ms.)

Participants (5)

CURAVAC EUROPE SPRL
Belgium
EU contribution
€ 2 101 840,40
Address
Avenue De Villefranche 80
1330 Rixensart
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Administrative Contact
Nicolas Havelange (Mr.)
piCHEM Forschungs und Entwicklungsgmbh
Austria
EU contribution
€ 1 162 000
Address
Kahngasse 20
8045 Graz
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Administrative Contact
Fritz Andreae (Dr.)
ACADEMISCH ZIEKENHUIS LEIDEN
Netherlands
EU contribution
€ 1 061 595,84
Address
Albinusdreef 2
2333 ZA Leiden
Activity type
Higher or Secondary Education Establishments
Administrative Contact
Jan Verschuuren (Prof.)
AEPODIA SA
Belgium
EU contribution
€ 779 999,60
Address
Rue Louis De Geer 6
1348 Ottignies Louvain La Neuve
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Administrative Contact
Charlotte Cuvelier (Mrs.)
UNIVERSITAIR ZIEKENHUIS ANTWERPEN
Belgium
EU contribution
€ 440 000
Address
Drie Eikenstraat 655
2650 Edegem
Activity type
Research Organisations
Administrative Contact
Daisy Van Mieghem (Ms.)