EU-funded Consortium Led by Immatics and BioNTech to Advance a Novel Class of Fully Personalized Therapeutic Cancer Vaccines into Clinical Trials for Brain Cancer

MAINZ, Germany, October 14, 2014 /PRNewswire/ --

Regulatory authority approves clinical trial for Glioma Actively Personalized Vaccine Consortium (GAPVAC)

First centers to enroll glioblastoma patients opened in
Heidelberg and Tuebingen

European Union supports the initiative with a €6 mn grant

BioNTech AG and immatics biotechnologies GmbH announce today that they are moving a novel concept of fully personalized therapeutic cancer vaccines, Glioma Actively Personalized VAccine Consortium (GAPVAC), into the clinic. The German national authority, the Paul-Ehrlich-Institute (PEI), has approved the start of a phase 1/2 study, GAPVAC-101, which applies for the first time the concept of treating glioblastoma patients based on drugs designed and manufactured for each patient individually according to specific characteristics of their tumor and immune system. The screening of first patients for the trial has commenced at the University Hospital of Heidelberg, Germany, and the University Hospital of Tuebingen, Germany. The complex manufacturing of the personalized vaccines will be performed by the GMP unit of the University of Tuebingen in close cooperation with the "GMP and Core Services platform" of the German Cancer Consortium (DKTK).

GAPVAC is the first EU-funded initiative aimed at clinically developing biomarker-guided actively personalized vaccines (APVACs) to treat patients with glioblastoma. Glioblastoma, an aggressive form of brain cancer with poor prognosis, has a high unmet need and the limited treatments available today have minimal effect on overall survival. The GAPVAC consortium includes 14 organizations in Europe and the United States and is led by BioNTech AG (Vice Coordinator) and immatics biotechnologies GmbH (Coordinator). The consortium is supported by a €6 million grant from the European Union Framework 7 (EU FP7) program.

The clinical trial will recruit up to 30 newly diagnosed glioblastoma patients for the phase 1/2 trial and aims to show that APVACs are well tolerated and induce a strong and specific response against cancer, as well as demonstrating the feasibility of this highly innovative approach. Glioblastoma patients will be immunized with two vaccines specifically prepared for each patient. The first vaccine will be a tailored selection of peptides chosen from a pre-manufactured warehouse supplied by consortium partner BCN Peptides (Barcelona) consisting of approx. 70 peptides based on the target profile of the individual cancer tissue and the ability of the individual's immune system to induce a response to the selected targets. The second vaccine will be based on full next-generation sequencing (NGS)-based genetic analysis of the patient and will comprise peptides newly manufactured by consortium partner University of Tuebingen. The latter vaccine will largely target mutations occurring in the cancer but not in healthy tissue. Both actively personalized vaccines will be designed according to biomarker-guided procedures performed at BioNTech and immatics and will be administered in addition to standard chemotherapy after surgery and initial radio-chemotherapy are completed. The clinical trial is being accompanied by an extensive biomarker program involving the Association of Cancer Immunotherapy (CIMT), a non-profit organization dedicated to the advancement of cancer vaccines.

The clinical trial will be led by chief investigator Prof. Dr. Wolfgang Wick, University of Heidelberg, and co-led by Prof. Dr. Pierre-Yves Dietrich, University of Geneva, both internationally recognized experts in the treatment and immunology of brain cancer.

Prof. Dr. Wolfgang Wick, Chair of the Department of Neurooncology at the University of Heidelberg, said: "The trial concept is exactly the right combination of exceptional science and a rigorous protocol for a disease for which over-simplified strategies have failed in the past. The scientific approach in this trial offers the chance for each involved patient to benefit clinically. In addition, we will learn a lot for future efforts in immunotherapy, bridging the precision of genomic medicine and immunotherapy."  

Prof. Dr. Ugur Sahin, Chief Executive Officer of BioNTech AG and Vice Coordinator of the GAPVAC consortium, noted: "The GAPVAC consortium is the first group in the world that is aiming to turn mutations in glioblastoma into targets for vaccine approaches taking patient care to the next level. This novel approach marks a fundamental paradigm shift from optimizing novel treatments for cohorts of patients towards optimizing drugs for each individual patient. The GAPVAC clinical trial mark a major change in the therapeutic management of cancer patients since the approach is suited to provide a truly individualized treatment."  

Notes to editors:

About the project

GAPVAC was launched in 2013 designed to create, manufacture and develop actively personalized vaccines (APVACs) tailored to the individual characteristics of the patient's tumor and immune system. It is based on combining latest state-of-the-art technologies, including next-generation sequencing (NGS), high-sensitivity mass spectrometry and innovative immunomonitoring approaches to generate an optimal therapy for the individual patient.

The consortium is supported by a €6 million grant from the European Union Framework 7 (EU FP7) program.

About the partners

BioNTech uses its next-generation sequencing (NGS) expertise to identify immunogenic tumor mutations and to generate a blueprint for the personalized vaccine that will include patient-specific tumor mutated peptides to be confirmed with regard to their natural presentation by immatics through mass spectrometry. Previously, BioNTech has demonstrated that the integrated use of NGS for genome-wide mutation identification (the "mutanome") followed by mutation-targeting vaccination is feasible and led to tumor control in pre-clinical models.

immatics uses its unique antigen discovery engine XPRESIDENT® to generate a warehouse of tumor-associated peptides (TUMAPs) from which the most suitable for each patient will be selected based on transcriptomic and peptidomic analysis to create the first of two APVACs applied to the patient.

The APVAC "on-demand" manufacturing will be performed by the GMP unit at the Department of Immunology (led by Prof. Dr. Hans-Georg Rammensee and Prof. Dr. Stefan Stevanovic), University of Tuebingen. The peptide warehouse was manufactured by BCN Peptides in Spain, an enterprise focused on peptide synthesis for clinical use. In addition, ten academic partners from Europe and the US are part of the consortium to apply the APVACs to their patients as well as contributing to the project with their own research. These are: Eberhard Karls University Tuebingen (Germany), University Hospital Geneva (Switzerland), University Hospital Heidelberg (Germany), Herlev Hospital/ Rigshospitalet (Denmark), Leiden University Medical Centre (The Netherlands), University of California San Francisco (United States), University Southampton (UK), Technion (Israel) and Vall d'Hebron University Hospital (Spain).

The clinical trial is being accompanied by an extensive biomarker program led by the Association of Cancer Immunotherapy (CIMT), a non-profit organization dedicated to the advancement of cancer vaccines, and immatics to confirm the mechanism-of-action and to identify biomarker signature candidates predicting which patients are most likely to benefit from treatment with APVACs. CIMT will also act as the dissemination platform and will contribute to the biomarker program and regulatory approach through its working parties.

In 2013, the CIMT Regulatory Research Group outlined a regulatory pathway for actively personalized immunotherapies after discussions with the Innovation Task Force of the European Medicines Agency (EMA), the regulatory authority in Europe responsible for marketing approval of new drugs. The results were published by Britten et al., "The regulatory landscape for actively personalized cancer immunotherapies", Nature Biotechnology, Vol. 31 (10), October 2013.

For more information about GAPVAC, visit the consortium website http://www.gapvac.eu.

About BioNTech AG

BioNTech AG (Biopharmaceutical New Technologies) is a leading, immunotherapy company that researches, develops and manufactures innovative, truly individualized, highly potent and well-tolerated immunotherapies for cancer and other diseases. Its focus is on pioneering, disruptive technologies ranging from individualized mRNA-based medicines through highly innovative Chimeric Antigen Receptors /T-cell Receptor-based products and antibody checkpoint immunomodulators. Established by clinicians and scientists, BioNTech is striving to develop uniquely individualized therapies through its pioneering research programs. It currently has two products in clinical testing for cancer, with a further three expected to enter clinical testing in 2014. Its clinical programs are supported by an internal molecular diagnostics-based unit that is preparing to launch its first in-vitro diagnostic for breast cancer. Founded in late 2008, BioNTech is privately held and raised the largest initial financing round in the history of the European biopharmaceutical sector.

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