A European Perspective on the Cell Therapy Industry

London’s spring weather greeted more than 500 delegates at the 2016 World Stem Cells & Regenerative Medicine Congress (WSCRMC) in May. Leaders of cell and gene therapy companies from across Europe, key representatives from Big Pharma, large organizations such as GE and Thermo Fisher, industry suppliers, and many European government institutions all gathered for two and a half days to discuss the latest developments in the industry.

The Invetech team sat in on many of the discussions and a myriad of topics were discussed. The following is a brief summary of some of our key takeaways from the week.

Big Pharma coming off the bench into the cell therapy game

With a strong voice at the conference, GSK expressed that cell and gene therapy will be the third pillar of healthcare, and their vision is to include cell therapy in mainstream medicine. GSK’s primary, long-term intent is to develop capabilities and platforms by concentrating on orphan diseases with low numbers of patients, and then extend the focus into other therapies for more common indications. GSK has accepted and embraced the reality that the initial indications will not significantly impact revenues and may even cost more to develop and deliver than what can be earned from these early indications.

Pharma is also drawing from the talent of innovators in academia, institutions, and smaller biotech firms. We heard many examples where Pharma expects to leverage its experience in the regulatory environment, scaling-up manufacturing, distribution, and sales as the new technologies are translated to the clinic and commercialization. As an example, GSK shared how they have collaborated with the Telethon Institute on several programs, including retroviral and lentiviral based therapies for ADA-SCID, WAS, MLD, beta thalassemia, MPS1, CGD, and GLD with much success.

The U.K., open for cell therapy business

When a minister comes to a commercial conference, one can surmise the industry is considered important to the economy. George Freeman, Member of Parliament (MP) and Minister for Life Sciences in the U.K., was very bold in his statements identifying the regenerative medicine sector as the new frontier of 21st century life sciences, and the U.K. as the “Spear of Regenerative Medicine”. With 17 universities, five multi-disciplinary hubs, £21 million invested by government, £29 million of industry co-investment, and £54 million in grants has created an exemplary environment for cell and advanced therapies.

The facts show that the U.K. has done a wonderful job supporting the growth of this industry and fostering innovation by applying a carefully planned approach. Indeed, the growth of the sector is significant. It is now home to 62 advanced therapy manufacturers, has formed nine new companies in the last 12 months, and has 51 active clinical trials—eight percent of all clinical trials globally. The U.K. is positioning itself as a leader in Europe with strong co-operation between industry, science, academic, and government institutions.

One key driver in this growth is the Cell and Gene Therapy Catapult. Established in 2012 as an independent center of excellence to advance the growth of the U.K. cell and gene therapy industry, it is bridging the gap between scientific research and full-scale commercialization. It started with just £70 and a mobile phone, but it has grown over the last couple of years into a real driver of growth for the industry—funding and forming new companies, moving research towards commercialization, and soon opening a manufacturing center outside of Stevenage (May 2017). The organization also provides a range of support, including manufacturing on large scale for small and medium enterprises (SMEs).

In a later conversation, a representative from Innovate U.K., Michael Sullivan, explained that any company with a presence in the U.K. can apply for funding and receive advice from any of the Catapult groups. That presence does not need to be the only office the company has, or even the company’s headquarters.

Path to accelerated clinical evaluation is being harmonized across the EU

The cell and advanced therapy industry is very much aware of the Japanese initiative for conditional market approval. The initiative creates higher expectations for many of the other regulatory agencies around the world, including the European Medicines Agency (EMA), to accommodate less conservative methods for evaluation of advanced therapy medicinal products (ATMPs). Some cell therapy companies are flocking to Japan to capitalize on this opportunity to get their therapies in the hands of clinicians and, as a result, delivered to patients more quickly. This enables data acquisition that can inform clinical trial design in the rest of the world, while controlling costs and potentially allowing revenues to be realized much sooner than traditionally possible.

Dr. Paula Salmikangas, the Committee for Advanced Therapies (CAT) chairperson and EMA coordinating senior researcher at the Finnish Medicines Agency, was a guest in London and outlined the harmonized EMA approach for accelerating breakthrough therapies, PRIority MEdicines (PRIME). The program was designed to help SMEs and academic drug developers design robust clinical trials, gather relevant data, and speed regulatory review in order to get promising treatments to patients as quickly as possible. The therapies that qualify for PRIME have access to “very focused scientific advice” and can request the fees for scientific advice at the EMA and national levels be waived. Some of the therapies that have qualified to date include ATMPs.

This newly launched program is another example of the efforts regulatory agencies are making around the world to facilitate novel advanced therapy approvals. Most jurisdictions provide some flavor of accelerated path for market approval; however, the rules differ for qualification, benefits, clinical trial design requirements, and post-market data reporting obligations. These contrasts highlight the need for continued work towards global harmonization.

The CAR-T excitement in Europe continues to accelerate, and the challenges around production and scaling up of these therapies are top of mind

The excitement around the CAR-T therapies was palatable at this conference. More entrants to the field are coming every month and more diseases are being considered targets for immunotherapies. First liquid tumors, now solid tumors, are being successfully treated by modulating patients’ immune systems with genetically modified cells. The number of clinical trials in both Europe and the U.S. has skyrocketed, and the initial results reported are impressive.

However, we are in the early days of CAR-T cell production. One presenter illustrated this basic manufacturing process in need of improvement by comparing today’s production with Ford’s Model-T manual assembly line.

During a panel with representatives from Kite Pharma, Adaptimmune, Cellectis and Autolus, the outlook was unanimous: the manufacturing of T-cells will be one of the most important elements of their organizations’ successes. They also agreed the current processes need to be simplified, automated, and closed. The discussion encompassed the pros and cons of centralized versus decentralized models of manufacturing; however, at least for the time being, the panel participants are opting for central manufacturing for two main reasons: control of product consistency and release testing. Most speakers discussed how they have invested, or are in the process of investing, in building their own manufacturing facilities, including Kite Pharma who has built a facility to treat 4,000 to 5,000 patients annually.

Other speakers during the conference believe a more decentralized model at the point of treatment is the future for CAR-T therapies. One thing all speakers agreed is that they “have to plan for success now” as it pertains to production of CAR-T therapies as the industry is preparing to set a new standard for best practice in oncology.

As more clinical trials are launched, and some therapies are nearing commercial approval, the imperative to deliver life-saving treatments to as many patients as quickly as possible is essential from an ethical and business standpoint.

From Invetech’s experience in working with clients to close cell and advanced therapy manufacturing processes, the priorities are in sync. In fact, correctly timing investment in technology to manufacture a consistent product ensures the reduction of risk that administering a variable product or therapy influences clinical trial results. We have also seen that access to capital is more readily available and is less expensive to negotiate when companies can prove their plans for commercial production are understood and achievable. Finally, resources in the form of personnel costs, number and types of equipment, facility size and clean room grade requirements are drastically reduced, so capital is conserved as the technological solutions are planned and implemented.

In conclusion

A great week with great discussions and insights from a European perspective confirmed once again that this is the best time to be part of this rapidly growing industry. These are extraordinary and exciting therapies that offer the possibility of significant breakthroughs in the field. The impacts, on both changing the landscape to improve patient health and on the corporate bottom line, offer tremendous potential for the health and wealth of everyone involved.