Chimeric antigen receptor T cell (CAR-T) therapy is currently the hot topic in the pharma world. The therapy involves treating patients with T cells engineered to express a chimeric antigen receptor that allows the T cells to recognise cancer cells and trigger their disintegration. Autologous CAR-T therapies make use of T cells extracted from each patient. In contrast, allogeneic therapies use T cells obtained from non-patient donors.
Key players in the market
Several hundred CAR-T trials are currently underway in a hotly contested race to market. The first wave technologies being developed mainly originate from academia. In August 2017, Kymriah became the first CAR-T therapy to be authorised by the FDA. Kymriah, an autologous therapy, was developed by Novartis in collaboration with the University of Pennsylvania. Kite Pharma followed closely behind Novartis in obtaining an FDA approval for its CAR-T therapy, Yescarta, in October 2017.
Juno Therapeutics, another front runner, is currently developing CAR-T therapies with the Fred Hutchinson and Memorial Sloan Kettering Cancer Research Centers. Autolus leads the UK CAR-T market, and Bristows was pleased to advise UCL Business on Autolus’ spin-out and initial £30 million equity financing in 2015. Autolus has since raised a further £99 million.
Challenges in delivering CAR-T therapies
CAR-T trials have struggled with poor response levels among some patients. The situation is made more challenging by the lack of reliable bio-markers and reliable pre-clinical CAR-T models.
These low response levels are starting to drive the industry towards combination therapies, increasing the number of collaborations between industry players. This trend will likely accelerate as the industry receives valuable data from the first round of CAR-T trials.
The industry is also wrestling with the complexities of setting up and operating a CAR-T business model that functions ‘from bench to bedside’. A particular challenge is, of course, the manufacture of autologous therapies. In contrast, allogeneic therapies do not require the extraction of patient T cells and so may present lower manufacturing complexity and cost.
CAR-T therapies are expensive to develop and deliver to patients. However, the industry appears confident that a viable financial model can be developed and that payers will support true cures. Novartis has already agreed a “pay-for-performance” pricing structure for Kymriah with the American Centers for Medicare and Medicaid Services (CMS) – the first pay-for-performance agreement CMS has signed.
We are currently witnessing a huge international experiment as the different industry players explore this powerful CAR-T technology. With the approval of Novartis’ Kymriah and Kite Pharma’s Yescarta, the race to develop and launch CAR-T therapies heats up. Patients’ lives will be saved by this technology and the excitement in the industry is palpable.