The number of CGT trials in China have grown significantly, up 61% since 2014, according to that publication: Cell and Gene Therapy in China.
And most of the trials are focused on chimeric antigen receptor T cells (CAR-T) therapy.
The Chinese authorities’ ability to mobilize and quickly set up the capabilities for R&D required in this space is impressive, said Adlai Goldberg, EY global digital, social and commercial innovation life sciences leader.
“If you look at the number of clinical trials globally in CAR-T, the US has been number one but China is coming in a close second.
“Capital investment, the opportunity to lead in the next generation of therapeutics, with CAR-T being one of those, and the ability for Chinese companies to develop their own IP are some of the drivers for the increased number of CGT trials in China,” Goldberg told BioPharma-Reporter.
Within the active trials for CAR-T, approximately 75% of those are in hematological cancer treatments, reads the EY report. Following the success of CAR-T in hematological or blood tumors, the technology is now shifting to solid tumors. The remaining 25% are spread among various malignant tumor treatments, such as cancer of the gastrointestinal (GI) tract, liver, lungs and breast, noted the analysts.
IP critical for collaboration
The Chinese government is also making significant efforts to strengthen intellectual property (IP), aligning its strategy to drive national innovation including a March 2020 call to strengthen patent laws, finds the report.
This is a positive for multinational pharmaceutical companies looking to enter the Chinese CGT market and many big players have started to explore collaborations and M&A, commented the EY team.
Merck KGaA is collaborating with GenScript to develop and establish a globally recognized CGT manufacturing service in China.
After having produced a with 94% response rate in myeloma patients in 2017 and setting up a 50-50 collaboration with Janssen, Legend Biotech, a China-based, biotech company successfully completed its US IPO in early 2020.
Other local companies have also taken the path of collaborating with large global biopharma firms, including Suzhou Innovent Bio, which formed an R&D collaboration with Roche.
JW Therapeutics — a joint venture established by WuXi AppTec and Juno Therapeutics — completed a US$100m Series B round of financing in June 2020. JW Therapeutics’ lead product, Relma-cel, is an anti-CD19 CAR-T therapy for third-line treatment for relapsed or refractory B-cell lymphoma. The therapy’s new drug application (NDA) has been accepted by China's National Medical Products Administration (NMPA).
Note of caution
The current regulatory requirement in China, however, is less stringent than in the US. This presents a double-edged sword, said Goldberg.
“China has a lot more ability to marshal and mobilize and to fast forward the clinical trial and the approval process. We saw an example of that in terms of the accelerated approach to vaccine development there for COVID-19. The upside to that is they have the opportunity and the infrastructure to move therapeutic development forward much more quickly and actually commercialize these products sooner than we in the US might be able to. Are there downsides to that? One might argue that there would be - caution, safety and security are important elements of any drug development process, particularly for some of these new types of therapies. So, yes, one might well ask if it is all going too fast.”
Multiple factors influence the high cost of CGT therapies in the US, particularly the form and structure of its healthcare system. “When you look at India, it is trying to develop a CAR-T therapy that can be administered for US$50K. China is also exploring ways to supply these therapies at much lower costs, to make them more affordable.”
Goldberg said CAR-T research and development efforts in China are closely aligned with global priorities in this respect, including the development of a ‘universal’ CAR-T, simplifying logistics and the optimization of CAR-T inclusive treatment protocols
“In the CAR T universe, in general, there is an increasing focus on the allogenic approach to that therapy. We are seeing a lot of that type of research taking place right now, both in China and in the US. Part of the larger, greater costs in these therapies - CAR-T but also mRNA therapies and others - is the autologous approach. The industry, overall, is moving towards trying to develop allogenic type CAR-T treatments and I actually believe China might get there before the US.”
The view of healthcare professionals
The general perception from the medical community EY interviewed in China toward CAR-T therapeutics is quite positive. The overall confidence level toward CAR-T therapies from Chinese doctors who have clinical trial experience related to CAR-T trials is high, added Goldberg.
“There was a great deal of pride in terms of what we were able to pick up and acknowledge. China broadly feels it has an opportunity to lead in this space and it is putting a lot of national energy behind doing that. And, as you look at the amount of investment that has gone into furthering that national agenda, it is significant and perhaps deeper that what is even being made available in terms of capital for this in the US and in Western Europe.”
In terms of outlook for CGT in China, the country is expected to continue seeing more research and clinical development activities in CGT, expanding from the current CAR-T dominant space to more diversified modalities.
“In two to three years, we are going to see that move from clinical to commercial and a greater acceleration then in NPD, in four to five years. China, right now, is a sleeping giant in this space. I think its speed and focus and being able to deliver against that are going to have a greater global impact in the years to come.”