For the last couple of years at every annual meeting of the American Society of Hematology (ASH) conference, I have posted an extensive Preview of the CAR T cell therapy landscape and looked at which abstracts piqued my interest.
The roaring 30s CAR
This year the review is the most extensive to date, with more companies, more research groups, more tumour types and way more preclinical research coming through. It’s like a kaleidoscope of ideas cascading through R&D.
The other thing to take note is how fast the field is moving – it’s warp speed now and so much comes through the literature every month on top of that.
So here we go – hold onto your hats as there is a LOT to contemplate this year!
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When we think of pioneers in the CAR T cell therapy space, one person who comes to mind is Waseem Qasim, Professor of Cell and Gene Therapy at the Institute of Child Health at University College London, and a Consultant Immunologist and Pediatrician at Great Ormond Street Hospital (GOSH).
Institute of Child Health
As readers may recall back in 2015, he gave the first allogeneic CAR T cell therapy under compassionate use to an infant with ALL, and in the process undoubtedly saved her life.
The subsequent case report published in Blood was the talk of 2015 annual meeting of the American Society of Hematology (ASH) in Orlando.
The poster focused on the first child that Prof Qasim treated and attracted a phenomenal amount of attention:
Prof Qasim UCART19 #ASH15 Poster
Where are we now with allogeneic CAR T cell therapy?
It’s been 18 months since we spoke to Prof Qasim, so while in London over the summer BSB caught up with him in his office at the Institute of Child Health.
This interview is the first in our latest 3-part mini-series on allogeneic CAR T cell therapy, which runs throughout this week. Here’s a teaser clip:
Kite’s first autologous product, Axi-Cel (in aggressive lymphomas), heads for regulatory approval in the US (PDUFA date November 29th), offering Gilead a hematology launch product with a high unmet need and, presumably, a relatively high price tag to match. Inevitably, some critical attention will subsequently be focused on the pipeline and whether they will move towards allogeneic CAR-T cell therapy (reduces cost of goods and increases profit margin) as well as how the TCR platform in solid tumours will fare.
It’s certainly a timely point to consider allogeneic CAR T cell therapies again given that things are rapidly heating up in the cell therapy niche following the Gilead announcement yesterday that they are acquiring Kite Pharma for $11.9 Billion.
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It was only five years ago that the number of abstracts on CAR T cell therapies at the American Society of Hematology (ASH) ran to a dozen or less. Fast forward to 2016 and we now have tens of them, almost too many to count, let along review quickly and easily.
A scene from ASH 2015…
To give you an idea of the staggering speed of progress, in 2010 it took me less than half an hour to search and read all the CAR T cell abstracts, now it takes nearly a whole day to peruse and review them carefully.
We can’t resist a challenge…
As usual, we will write in more depth from the meeting as the data emerges in real time since many of the abstracts are often placeholders with updated information provided at the conference itself.
For now, here we provide an in-depth preview of the CAR T cell landscape in terms of the players, the products, new scientific research, biomarkers, emerging trends and more in a handy What to Watch For (W2W4) guide on key areas to expect at ASH to enable better enjoyment and awareness as the data rolls out next month.
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Following on from the recent rise in Xoma shares, that I discussed in yesterday’s blog post, the company announced this morning that they have signed a commercialization deal with Les Laboratoires Servier, France’s largest privately held pharmaceutical company (2010 revenue 3.7 billion euros).
According to the press release, the key elements of the deal are as follows:
- XOMA will receive approximately $35 million upfront, up to approximately $470 million in milestone payments and tiered royalties up to a mid-teens percentage rate.
- XOMA retains development and commercialization rights for Behcet’s uveitis and other inflammatory and oncology indications in U.S. and Japan. Servier receives similar rights in the rest of the world.
- Servier will fund the first $50 million of XOMA 052 development expenses and 50% of further expenses for the Behcet’s uveitis indication. XOMA 052 is expected to advance into Phase 3 development in Behcet’s uveitis in 2011.
- Servier will fund development for diabetes and cardiovascular disease indications in exchange for worldwide rights.
- XOMA retains an option to reacquire the development and commercialization rights to the diabetes and cardiovascular indications in the U.S. and Japan by paying an option fee and partial reimbursement of incurred development expenses. If XOMA reacquires these rights, it has the ability to license them to one or more third parties.
This is a large injection of cash into Xoma that will help fund phase 3 clinical trials. The regional deal also keeps open the possibility of licensing U.S. marketing rights to another partner in the future.
The only surprise perhaps is the timing of the announcement, which is in advance of the phase 2 clinical trial results for XOMA 052 in diabetes. The expectation from all the signals is that this data could be positive, although there are no guarantees in clinical research and I’d give this a 50:50 shot at success. It will be interesting to see what happens to XOMA 052 as it moves forward in development.