MYASTERIX STRATEGY AND WORKPROGRAMME
MYASTERIX aims to advance a therapeutic vaccine candidate (CV-MG01 with orphan drug status) for the autoimmune disease myasthenia gravis (MG) to human proof-of-concept studies that.
To achieve in 5 years the completion of the study, the workprogramme has been structure in 6 workpackages (WP):
- WP1: This WP will ensure effective and efficient coordination and management throughout the duration of the project.
- WP2: After successful technology transfer for manufacture of the human formulation of the CV-MG01, several batches will be manufactured for toxicity studies, and phase I and phase II trials.
- WP3: As part of preclinical studies, the GLP preclinical regulatory toxicity study will be performed.
- WP4 & WP5: Vaccine safety and efficacy will then be tested in humans. Phase I human pharmacology and therapeutic exploratory trial will be carried out in patients with MG. The study will be placebo controlled and double blind in order to distinguish between (a) the reactogenicity and adverse effects provoked by the adjuvant alone and (b) possible adverse effects of the vaccine. The phase I trial will be followed by a long-term extension follow-up study for the patients under active treatment and by a phase II therapeutic confirmatory trial with a quite similar design to phase I. The primary endpoint will not be safety but efficacy in order to achieve proof of concept at the end of the trial and the project.
- WP6: Dissemination and exploitation will be addressed. This will include the longer-term goal of bringing the vaccine to patients and the market, essentially by optimising the existing project business plan through market analysis (revenue side) and contact with the authorities (cost side).