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  • Periodic Reporting for period 1 - CardAPcells (Cell therapy for patients with chronic heart failure with proprietary cardiac-derived cells (CardAP cells) – “From the Heart to the Heart”)

CardAPcells Report Summary

Project ID: 735261

Periodic Reporting for period 1 - CardAPcells (Cell therapy for patients with chronic heart failure with proprietary cardiac-derived cells (CardAP cells) – “From the Heart to the Heart”)

Reporting period: 2016-07-01 to 2016-12-31

Summary of the context and overall objectives of the project

Chronic heart failure is a life-threatening disease usually without cure and a leading cause of morbidity and mortality. In Germany 45 000 patients (EU: about 140 000) die every year from progressive chronic cardiac insufficiency. There is no curative therapeutic solution, except very rare donor hearts and artificial hearts as bridge devices.
Researchers at the Charité - University Medicine, have identified unique cardiac-specific stromal cells (CardAP cells) with strong efficacy in cardiac stress models. CardAP cells are specialized heart-derived cells with key advantages in safety and efficacy.
CardAP cells fulfil all criteria for cardiac cell therapy and the product manufacturing has been transferred to GMP facilities. CellServe a Charité spin out will develop the CardAP cells for therapy of patients suffering from severe life-threatening cardiomyopathy.
The proposed development of CardAP cells for autologous therapy of patients with severe chronic heart failure will offer millions of patients a therapy, which will regenerate their heart muscle and give them the opportunity to lead a normal life.
Specific objectives for the feasibility study
• Collecting the required data on regulatory issues.
• Calculation of costs for the clinical development, manufacturing and marketing of CardAP cells therapy.
• Development of a strategy to achieve market approval and validation of market introduction in Europe.
• Identification of international collaboration partners to get market approval in the US and Asia.
• Preparation of the clinical Phase I/II study.
• Elaboration of a business plan incl. development strategy, financial projection, market data and detailed regulatory requirements etc.

Work performed from the beginning of the project to the end of the period covered by the report and main results achieved so far

Task 1: Evaluation of regulatory path for hospital exemption for CardAP cells.
Subcontracting of specific regulatory expertise: Applying for Hospital exemption for Advanced Therapies Medicinal Products Guideline (ATMPs): implementation of Art 28(2) of Regulation 1394/2007.
The management of CellServe talked to regulatory experts about the strategy to apply for a hospital exemption. Experts think that the hospital exemption could be applied for, if the clinical data of the phase I study will be convincing and demonstrate that patients will have a clear benefit. The problem using this instrument is the administrative efforts necessary to get a reimbursement because contracts have to be negotiated with hospitals and health insurances.

Task 2: Evaluation of regulatory path and development costs until European market authorisation
CellServe has calculated the costs of the product development until market authorization.
Evaluation of detailed regulation path for European approval and market authorization according ATMP-guideline.
CellServe started with the development of the CardAPcells several years ago. The company considered the ATMP regulation during the whole development process and involved external experts to fulfil all relevant regulations. The last steps in this development process are the clinical studies, which will demonstrate the safety and efficacy of the CardAPcells and the GMP production, which was established by Fraunhofer Institut IZI (FHG-IZI).

Task 3: Development of a business plan for the CardAP cells project.
Regulatory and marketing strategy, financial planning, calculation of financial requirements, time schedule of the development, subcontracting.
In close collaboration with ConsulTech and the subcontractors the management of CellServe developed a business plan, which was integrated into the phase 2 project application. This business plan includes the development strategy, the marketing plan and the financial plan, including the plan how to raise venture capital from private and public investors.

Task 4: Evalution of CardAP manufacturing costs.
CellServe will reduce costs and investments in manufacturing by a cooperation with FHG-IZI as a well established contract manufacturer with a GMP-production.

Task 5: Evaluation of reimbursement in countries such as UK, France, Spain and Italy.
In each country the specific regulations have to be evaluated.
CellServe decided to start marketing under hospital exemption in Germany and not in other European countries, because the German market with 45,000 patients is very large and the time to achieve the full market authorization will be 2023/2024. In this time period many rules could change and it seems to be more reasonable to evaluate the reimbursement systems in more detail in 2019.

Task 6: FTO analysis
A FTO analysis was performed by ConsulTech in December 2016. The report showed that there are hundreds of patents and patent applications with claims concerning cell therapy of heart muscle. None of these describes a cell type with the same properties of CardAP cells. Most of the publications describe the use of stem cells or induced pluripotent stem cells (IPSC). The patent of CellServe/Charité does not infringe any other IP.

Task 7: Completion of the clinical study protocol.
The clinical study protocol was finished and will be up loaded on the EC server and submitted as part of the phase 2 application.

Task 8: Marketing study showing the potential of the CardAP cells.
The marketing study was updated. CardAP cells will have the potential to create a billion $ market, if the clinical results will demonstrate safety and efficacy and if CellServe will collaborate with other companies to market the product in all relevant markets.

Progress beyond the state of the art and expected potential impact (including the socio-economic impact and the wider societal implications of the project so far)

According to European ATMP regulation new cell therapies have to fulfil a lot of different requirements and criteria to prevent damage to patients and to secure high quality standards.
Within the proposed project CellServe will demonstrate the safety of CardAP cells which is one of the demands of regulatory authorities (EMA) and the subcontractor FHG-IZI will demonstrate that it will fulfil all regulatory requirements in the production of the cells in its production facility.
Besides safety of the CardAP cells the clinical study will also show that the therapy will improve the condition of patients receiving these autologous cells. This will be measured by the output of the heart (measurement of ejection fraction (EF) which is the fraction of blood ejected from a ventricle of the heart with each heartbeat).
The results of the clinical study will be evaluated and documented. The final report will be the basis for the decision to continue the clinical development of the CardAP cells and to finance this by venture capital investors.
Fit of concept and objectives into the marketing plan:
• Establishment of a scalable GMP production of CardAP cells (fulfilment of regulatory demand) in order to receive marketing approval by EMA.
• Preparation of a multicenter clinical trial (fulfilment of regulatory demand) to demonstrate safety and efficacy of the product.
• Fulfilment of all regulatory requirements to perform a phase III clinical study.
• Applying for Hospital Exemption for ATMPs (implementation of Art 28(2) of Regulation 1394/2007) for early market entry and early revenues after 3 years. This will generate revenues and will be part of the marketing strategy to involve customers already before marketing approval.
• Venture capital financing of the company by private investors. The achievement of clinical objectives will support the financing of CellServe by private investors.
The number of products according ATMP regulation of the European Union from 2009 is still very small. The regulation complicated the market approval of gene therapy medicines, somatic-cell therapy medicines, tissue-engineered medicines and combined ATMPs.
CardAP cells will be a promising somatic-cell therapy medicine, which will change the therapy of patients with fatal chronic heart failure.

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