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  • Periodic Report Summary 2 - MYASTERIX (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)
FP7

Myasterix Report Summary

Project ID: 602420
Funded under: FP7-HEALTH
Country: France

Periodic Report Summary 2 - MYASTERIX (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)

Project Context and Objectives:
MYASTERIX project 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. This will provide a more targeted therapeutic approach with fewer and less severe side effects than the existing therapeutics.

The project is divided into 6 main Work Packages (WP): WP1 management, WP2 production, WP3 toxicity study, WP4 phase 1b clinical trial, WP5 phase 2 clinical trial and WP6 exploitation and dissemination

In period 2, the main objectives were:

1. For WP1: to include Antwerp University Hospital (UZA) as a new consortium partner.
2. For WP2: to perform the stability studies on the produced batches and to manufacture new batches of the peptides.
3. For WP4: to perform the phase 1b clinical trial.
4. For WP5: to start planning the phase 2 clinical trial.
5. For WP6:
to communicate on the start of the phase 1b, to the public and with the patient organisations;
to finalise the market analysis;
to go for Protocol Assistance at the European Medicines Agency.

Project Results:
WP2 Production

A prerequisite for launching the phase 1b study was the timely supply of therapeutic vaccine batches for the preclinical toxicity studies and the phase1b clinical trial. piCHEM produced and released the drug substance and drug product following Good Manufacturing Practice (GMP) for the clinical trial. Stability studies for all products used in toxicity and clinical trials have been launched and are ongoing.

WP4 Phase 1b clinical trial

The Clinical Trial Application (CTA) prepared by CuraVac, UZA, Aepodia, LUMC and piCHEM was accepted in October 2015 by the Belgian competent authorities and by the UZA Ethics Committee.

The MYASTERIX project Phase 1b study on patients for the therapeutic vaccine (active targeted immunotherapy) for Myasthenia Gravis (EudraCT 2015-002880-41) was then successfully launched at UZA in November 2015. The first patient was injected in March 2016. The recruitment was greatly facilitated by the involvement of several patient associations. As of the 30th of September 2016, a total of 16 patients have been enrolled, included and dosed into the study of which 14 received more than one injection of CV-MG01 or placebo, 6 patients received the three subcutaneous injections and 3 patients already completed the active part (Part A) and have entered the observational part B of the study. After reviewing the safety and tolerability data at low dose on a first cohort of 8 patients, the data review committee approved the decision to test the high dose regimen in the next cohort of MG patients. Eight patients are currently receiving the high dose regimen of CV-MG01. The results are expected to be available during the second quarter 2017.

The next data review committee meeting is planned for December 2016.

WP5 Phase 2 clinical trial

The consortium is currently preparing for the Phase 2 trial which will take place in the beginning of third quarter 2017. Results of this study are expected for the third quarter 2018.

WP6 Dissemination and exploitation

CuraVac also finalised the market analysis which aimed at building a better understanding of the disease’s socio-economic burden.
The European Medicines Agency (EMA) sent its final report on the Protocol Assistance on the 22nd of October 2015 following a clarification meeting held on the 7th of October at the EMA in London.
Two successful webinars have been organized by/with patient organizations from France (A.M.I.S.) and Belgium/The Netherlands (MG Liga and VSL). The movies are available on YouTube. These webinars were a good opportunity to explain our project to the patient community.
CuraVac remains very active in business development and participated in BIO2015 (Philadelphia), BIO2016 (San Francisco) and Bio-Europe 2015 (Munich). CuraVac is in contact with several companies for an early license agreement on the therapy for a limited territory.

Potential Impact:
The MYASTERIX project is expected to have a major impact on MG patients. Indeed, the project will evaluate the anticipated benefits of a targeted therapeutic approach for MG requiring only three injections which can bring significant and lasting improvement or even a cure to MG patients. We will compare this novel treatment to current options such as general immunosuppression, which cannot cure but only control the disease, and incomplete symptomatic relief, which can only be achieved by the frequent (every 4 hours) administration of drugs like acetylcholinesterase inhibitors. The side effects of the MG therapeutic vaccine are expected to be comparable to current prophylactic vaccines. The vaccine will therefore compare favourably to the current drugs used to treat MG, which cause numerous and severe side effects.

The MYASTERIX project is also expected to have a medico-economic impact on patients and society. Reviews that have evaluated the current treatment strategies for MG, their numerous and severe side effects as well as their costs, indicate that the total average cost of treatment including all known side effects is well above the average cost of MG medication alone. Many patients also remain significantly disabled, which reduces their quality of life and their ability to work, thus causing lost revenue not only for the patients but also for society as a whole. Our approach could generate considerable benefits for patients and society by replacing all the primary and secondary costs with the sole cost of the active targeted immunotherapy and its straightforward three-injection procedure, which have only mild local side effects. Ultimately, if maintenance therapy and hospitalisation costs can be replaced with the costs of CV-MG01, then everyone will win: the patients, the stakeholders and society as a whole.

Project scientific coordinator: Nicolas Havelange
E-mail: n.havelange@curavac.com
Website of the project: www.myasterix.eu
Duration of the project: 5 years

List of Websites:
www.myasterix.eu

Related information

Contact

Florence Chung, (VP European & International Affairs)
Tel.: +33 155030122
Fax: +331 55 03 01 60
E-mail

Subjects

Life Sciences
Record Number: 192387 / Last updated on: 2016-12-15