EU study to build on Phase I trial of Videregen product & generate approval application

The pan-European TETRA consortium, led by Videregen Limited, has been awarded EUR 6.8 million under the Horizon 2020 programme[1] for a pivotal Phase II clinical trial of the company's tissue engineered replacement trachea. The project will start at the beginning of 2016 with the first patients to be treated in 2017, building on the results of the four-patient UK INSPIRE Phase I study[2] The study will focus on the orphan indication of severe structural airway disease (SSAD)[3], a potentially life-threatening condition believed to affect around 12,000 people in Europe. The pivotal trial results are expected to lead to a European Marketing Authorisation application in 2019, with launch of the world's first commercially available tissue engineered organ replacement product in 2020. 

The Phase II trial, which will recruit patients from five clinical sites across Europe, is to assess the safety and efficacy of the replacement trachea product. It will monitor how treatment reduces patients' need for additional interventions such as stents, as well as other factors such as impact on quality of life. The project also involves scale-out of stem cell manufacture from the UCL/Royal Free Hospital London Centre for Cell, Gene and Tissue Therapeutics to establish a second GMP manufacturing site in Munich, creating a pan-European supply chain. Validation of this production and supply system is an objective of the trial, as is demonstration of the product to patients and leading surgeons.  

The 13-partner TETRA consortium brings together cross-disciplinary expertise in the areas of regenerative medicine, tracheal diseases and airway disorders, clinical trials and cell-based tissue engineered product manufacture. The principal investigator is Professor Martin Birchall (UCL, UCL Hospital NHS Foundation Trust) with additional trial sites at the University of Manchester/University Hospital of South Manchester NHS Foundation Trust (UK), Università degli studi di Brescia (Italy), Medical University of Vienna (Austria) and Instytut Gruzlicy I Chorob Pluc (Poland). Other parties include the Cell Therapy Catapult (pan-European regulatory processes and lead clinical research activities), TMC Pharma (regulatory affairs and lead clinical research activities), NHS Blood and Transplant (tissue retrieval and trachea scaffold manufacture), Klinikum Rechts der Isar der Technischen Universität München (stem cell processing and final product release) and Euram (project management). 

Speaking for the consortium, Dr Steve Bloor, CEO of co-ordinator Videregen, said, “Award of this funding under the Horizon 2020 programme is fantastic news for the TETRA consortium, and a major step towards making our replacement trachea available commercially. With state-of-the-art manufacture facilities forming a European supply chain allied to leading clinical trial sites, we expect rapid recruitment of patients with this devastating disease which is associated with costs of EUR 2.6 billion pa in Europe. The results of the trial will also help expand Videregen's technology into other diseases and organ replacement products, and enhance the reputation of Europe in regenerative medicine. The TETRA consortium is very grateful for the support of Horizon 2020, and that of its stakeholders, funders and investors.”

Principal Investigator Professor Martin Birchall said, “We are delighted to be working on a project which will ultimately make the tissue engineered replacement trachea available on widespread basis to patients with SSAD. Designed to be a one-off curative treatment without the need for a lifetime of anti-rejection drugs, it will bring real clinical benefit. Patients will be an important part of the wider TETRA project team, and we are grateful for the support of the National Association of Largyngectomee Clubs.”

(1) This project has received funding from the European Union’s Horizon 2020 research and innovation programme under grant agreement No 681027  (2) For more details of the INSPIRE trial please go to http://www.videregen.com/clinical-trial-for-replacement-trachea-technology-to-start-next-year/ [3] Severe structural airway disease (SSAD) is associated with significant airway obstruction leading to high levels of morbidity and a 50% mortality rate if not treated successfully. Currently patients are subjected to repeated, limited efficacy, surgical interventions, such as stent insertions which ultimately have a high incidence of failure. In contrast, Videregen's tracheal replacement approach, which involves repopulating an acellular trachea 'scaffold' with the patient's own stem cells, is designed to be a one-off curative treatment without the need for a lifetime of anti-rejection drugs thereafter. The overall incidence of severe structural airway disease is estimated at c.11,8273 in EU per year. On average this patient group will spend 22 days in hospital at an average cost of EUR 218,619 per patient translating to an EU-wide population cost of EUR 2.6 billion per year. 

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