Community Research and Development Information Service - CORDIS

H2020

FAST-BM Report Summary

Project ID: 735040

Periodic Reporting for period 1 - FAST-BM (FAST-BM: FibroSelect: delivering A Step-change in Biotherapeutic Manufacturing)

Reporting period: 2016-08-01 to 2016-12-31

Summary of the context and overall objectives of the project

Biopharmaceutical therapeutics (biotherapeutics) such as monoclonal antibodies (mAbs) treat or prevent a wide variety of disease states, including cancers, autoimmune, degenerative and cardiovascular diseases. Biotherapeutics manufacture necessitates the use of very large cultures of mammalian cells followed by extensive purification steps (using a combination of unit operations in series), under Good Manufacturing Practice (GMP) conditions. These purification consumables incur the bulk of manufacturing costs - chromatography purification accounts for over 50% of the cost of biotherapuetics . Current processing strategies are limited due to the platform technologies available, which have only seen incremental improvement over the last 20 years, and are now run close to their physical limits to accommodate increasing amounts of product from high titer fermentations. This requires voluminous tanks and significant clean room floor space for larger product pools and buffer volumes, all translating into infrastructural limitations . The linking of standardised process to clinical result, and so commercial success, has made the industry highly risk adverse to the adoption of new technologies. Yet pressure to adopt new technologies is increasingly driven by the need to better understand processes and build in flexibility to both de-risk manufacture and be able to adapt to uncertainties in progression through the clinical trial process and patient population size. This is against a background of payers worldwide becoming more price sensitive and the development of more complex, next generation proteins for gene and T-Cell therapies. Therefore, the biopharmaceutical industry is fast opening up to the adoption of new technologies, as evidenced by the increasing use of disposable equipment such as single-use bioreactors (SUBs; >50% increase in use since 2006 ), which increase facility flexibility and reduce validation costs.
To reduce the manufacturing cost of biotherapeutics, Puridify has developed an innovative bioprocessing platform technology, FibroSelect, a functionalised cellulose chromatography medium to enable high-productivity, product capture and purification that is economic to operate in a disposable manner. This reduces the cost of: a) process development through more powerful tools and critically b) manufacturing through significant reduction in purification consumables costs and ancillary costs such as process validation and buffer usage. This facilitates cost effective manufacture of a wide range of biosimilars and novel biomolecules. FibroSelect has the potential to reduce biotherapeutics manufacturing costs and open up new processing strategies to maximise the output of existing and new facilities. This will play an important role in widening patient access to expensive drugs, and unlock new processing opportunities for “difficult-to-purify” products. Puridify completed a Phase 1 study in order to undertake a detailed investigation of potential routes to market for the platform FibroSelect technology. The expected outcome is a detailed business plan, concurrently giving sufficient information to drive submission of a Phase 2 application to assist the launch of FibroSelect into EU and global marketplaces.
Specific objectives for the feasibility study:
a) Market assessment and prioritisation – to develop early revenue streams to build value whilst partners for large scale commercialisation are identified;
b) Regulatory requirements – to ensure production is in-line with needed standards, and to understand how to best service the regulatory needs for continuous supply (c. 25 years) to the largest customers;
c) Business Models & Exploitation Strategy – to identify scaling partners needed to reach the largest consumers, and the contractual agreements that will underpin these business relationships;
d) Supply chain assessment – to identify reliable suppliers to enable scale-up activities.
Key outputs are a Feasibility report, including a business plan, plus a completed Phase 2 application.

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

Puridify has developed an innovative bioprocessing platform technology, FibroSelect, a functionalised cellulose chromatography medium to enable high-productivity, product capture and purification that is economic to operate in a disposable manner. This reduces the cost of: a) process development through more powerful tools and critically b) manufacturing through significant reduction in purification consumables costs and ancillary costs such as process validation and buffer usage. This facilitates cost effective manufacture of a wide range of biosimilars and novel biomolecules. Over this Phase 1 project Puridify has carried out extensive market research to deliver a report that investigates the commercialisation opportunities for FibroSelect in the near term and forecasts potential to benefit the bioprocess and healthcare industry both in the EU and globally.
The output of the project is this report which provides a route forward for Puridify to drive towards commercialisation with while establishing a base to apply for follow on funding from the Phase II stage of the SME instrument.

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)

Cancer cost the EU €126 billion in 2009, with health care accounting for €51 billion (40%). Productivity losses because of early death cost €42.6 billion and lost working days €9.43 billion. Informal care cost €23.2 billion . At a global level, the economic cost of cancer has been estimated at $1.4 trillion per year . Biopharmaceuticals targeting cancer are the largest segment (52%) of the biotherapeutics pipeline . Antibody-based therapy with mAbs for cancer is one of the most successful and important strategies for treating patients with haematological malignancies and solid tumours (e.g. trastuzumab [Herceptin®], used to treat breast and stomach cancers). However, the cost of biotherapeutics has often been prohibitive, thereby limiting their use, particularly in developing countries. The cost challenges facing biotherapeutic products are as follows: i) Chromatographic purification which accounts for >50% of processing costs. This is important given the increasing price sensitivity of payers worldwide and serves as a limitation to the growth of the biosimilar industry; ii) Increasing diversity and complexity of biotherapeutics: ~40% of biopharma’s product pipeline are large complex proteins (e.g. bispecific antibodies, fusion proteins or viral vectors) which offer promising therapeutic opportunities but cannot be manufactured cost effectively as they do not fit with current bioprocessing platform technologies; iii) Process flexibility: The rapidly evolving industry is moving to more flexible disposable formats to cope with uncertainty in drug development and varying market demand. FibroSelect addresses each point above by i) enabling significant cost reductions%; ii) offering flexibility to design new bioprocessing platforms; iii) being a wholly disposable system where all purifying ligand is expended per-batch purification. It is innovation in the latter area that helps to drive costs down and increase productivity, as the requirement for cleaning columns and re-validating prior to purification of the next batch of biotherapeutic product is ameliorated.
FibroSelect will be the industry’s first true disposable system for biotherapeutic processing and purification. It’s potential to deliver step-change improvements in bioprocessing has been recognised by leading manufacturers of biotherapeutics, who have expressed strong desire to incorporate FibroSelect into their process development once proven via ongoing evaluation studies against their existing platforms.

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